PENMENVY, GSK’s 5-in-1 Meningococcal Vaccine, Approved by US FDA to Help Protect Against MenABCWY




PENMENVY, GSK’s 5-in-1 Meningococcal Vaccine, Approved by US FDA to Help Protect Against MenABCWY

• Vaccine helps protect against five common disease-causing serogroups of Neisseria meningitidis (A, B, C, W, and Y)
• Broad serogroup coverage in one vaccine reduces injections to help improve vaccination rates and help protect more US adolescents and young adults

PHILADELPHIA–(BUSINESS WIRE)–#Adolescents–GSK plc (LSE/NYSE: GSK) today announced that the US Food and Drug Administration (FDA) has approved PENMENVY (Meningococcal Groups A, B, C, W, and Y Vaccine) for use in individuals aged 10 through 25 years. The vaccine targets five major serogroups of Neisseria meningitidis (A, B, C, W, and Y) which commonly cause invasive meningococcal disease (IMD).^1,2

The vaccine combines the antigenic components of GSK’s two well-established meningococcal vaccines, BEXSERO (Meningococcal Group B Vaccine) and MENVEO (Meningococcal [Groups A, C, Y, and W-135] Oligosaccharide Diphtheria CRM₁₉₇ Conjugate Vaccine). The regulatory application was supported by positive results from two phase III trials [NCT04502693; NCT04707391], which evaluated the vaccine’s safety, tolerability, and immune response in over 4,800 participants aged 10-25 years. The safety data demonstrated that the vaccine has a safety profile consistent with GSK’s licensed meningococcal vaccines.^3-5

Tony Wood, Chief Scientific Officer, GSK, said: “We are excited about the opportunities ahead to help improve meningococcal vaccination coverage in the United States, especially for IMD caused by serogroup B. Building on our global leadership in meningococcal vaccination and our longstanding commitment to address unmet need in disease prevention, we aim to help protect more teens and young adults at a life stage when they are at an increased risk.”

Integrating GSK’s MenABCWY vaccine into healthcare provider practices could simplify meningococcal vaccination delivery and help protect more US adolescents against these five common disease-causing serogroups – A, B, C, W, and Y – for which the US Centers for Disease Control and Prevention (CDC) have issued recommendations.⁶ Although MenB is the leading cause of IMD among this population, less than 13% receive the recommended two-dose vaccination series; around 32% receive at least one dose.^7,8 Three of every four MenB doses currently administered in the US are manufactured by GSK,⁹ positioning the company well to lead in the US market as MenB-containing vaccinations must be completed with the same manufacturer’s MenB vaccine.

Judy Klein, President and Founder of Unity Consortium, a non-profit organization focused on adolescent health and immunization in the US, said: “The consequences of IMD can be devastating for those who contract it, for their families and friends. We welcome new tools to help protect more adolescents from meningococcal disease. Pentavalent MenABCWY vaccines could help address the disease by providing protection against the five vaccine-preventable serogroups in one vaccine and making it easier for adolescents to get the coverage they need.”

At its meeting on February 26, 2025, the CDC’s Advisory Committee on Immunization Practices (ACIP) is expected to vote on recommendations for the appropriate use of GSK’s MenABCWY vaccine in adolescents and young adults.

About IMD

IMD is an uncommon but serious illness that can lead to death for up to one in six of those who contract it in as little as 24 hours from onset, despite treatment.^10,11 IMD is easily misdiagnosed, with early symptoms often mistaken for the flu.^11,12 Approximately one in five survivors may experience long-term consequences such as brain damage, amputations, hearing loss, and nervous system problems.^10,12 Although anyone can get IMD, adolescents and young adults between the ages of 16 and 23 years are one of the groups at highest risk due to common behaviors that help transmit the bacteria that cause IMD such as living in close quarters like college dormitories, kissing and sharing drinks, utensils, or smoking devices.^13,14

About PENMENVY (Meningococcal Groups A, B, C, W, and Y Vaccine)

GSK’s MenABCWY vaccine is an injectable suspension for intramuscular use. The vaccine is supplied as one vial of lyophilized MenACWY Component (powder) which is reconstituted at the time of use with the accompanying prefilled syringe of MenB Component (liquid). It is indicated in the US for active immunization to prevent invasive disease caused by Neisseria meningitidis serogroups A, B, C, W, and Y. It is approved in the US for use in individuals aged 10 through 25 years. The US Prescribing Information is available here.¹⁵

Important Safety Information for PENMENVY in the US

The following is based on the US Prescribing Information for PENMENVY. Please consult the full Prescribing Information for additional safety information.

• Do not administer PENMENVY to individuals with a severe allergic reaction (e.g., anaphylaxis) to a previous dose of PENMENVY, to any component of this vaccine, or to any other diphtheria toxoid-containing vaccine
• Syncope (fainting) has occurred in association with administration of PENMENVY
• PENMENVY may not protect all vaccine recipients and may not provide protection against all meningococcal serogroup B strains
• Immunocompromised persons, including those receiving immunosuppressive therapy, may have reduced immune responses to PENMENVY
• Persons with certain complement deficiencies and persons receiving treatment that inhibits terminal complement activation are at increased risk for invasive disease caused by N. meningitidis, including disease caused by serogroups A, B, C, W, and Y, even if they develop antibodies following vaccination with PENMENVY
• Guillain-Barré syndrome (GBS) has been reported in temporal relationship following administration of a U.S.-licensed meningococcal quadrivalent polysaccharide conjugate vaccine. The decision by the healthcare professional to administer PENMENVY to persons with a history of GBS should take into account the expected benefits and potential risks
• The most commonly reported solicited adverse reactions in individuals aged 10 through 25 years after Dose 1 and Dose 2: pain at the injection site, fatigue, headache, myalgia, nausea, erythema, and swelling. The most commonly reported solicited adverse reactions in MenACWY conjugate vaccine-experienced individuals aged 15 through 25 years after Dose 1 and Dose 2: pain at the injection site, headache, fatigue, myalgia, and nausea

About BEXSERO (Meningococcal Group B Vaccine)

GSK’s MenB vaccine has received regulatory approval in over 55 countries, including the US, and is used in 18 national immunization programs worldwide for the prevention of IMD caused by Neisseria meningitidis serogroup B. More than 110 million doses have been distributed worldwide since 2015.¹⁶ It is supported by clinical data supporting its effectiveness in helping to protect adolescents and young adults against diverse disease-causing strains of MenB, with a well-characterized safety profile. In the US, this vaccine has received regulatory approval for active immunization to prevent invasive disease caused by Neisseria meningitidis serogroup B and is approved for use in individuals aged 10 through 25 years. The US Prescribing Information is available here.¹⁷

Important Safety Information for BEXSERO in the US

The following is based on the US Prescribing Information for BEXSERO. Please consult the full Prescribing Information for additional safety information.

• Do not administer BEXSERO to individuals with a history of a severe allergic reaction (e.g., anaphylaxis) to any component of BEXSERO or after a previous dose of BEXSERO
• The tip cap of the prefilled syringe may or may not be made with natural rubber latex. Natural rubber latex may cause allergic reactions
• Syncope (fainting) can occur in association with administration of BEXSERO
• BEXSERO may not protect all vaccine recipients and may not provide protection against all meningococcal serogroup B strains
• Some individuals with altered immunocompetence may have reduced immune responses to BEXSERO
• Individuals with certain complement deficiencies and individuals receiving treatment that inhibits terminal complement activation (for example, eculizumab) are at increased risk for invasive disease caused by N. meningitidis serogroup B even after being vaccinated with BEXSERO
• The most commonly reported solicited adverse reactions: pain at the injection site, fatigue, headache, nausea, erythema, myalgia, and swelling

About MENVEO (Meningococcal [Groups A, C, Y, and W-135] Oligosaccharide Diphtheria CRM197 Conjugate Vaccine)

GSK’s MenACWY vaccine has received regulatory approval in over 60 countries, including the US, with more than 80 million doses distributed worldwide since 2010.¹⁸ It offers evidence of immunogenicity with a well-characterized safety profile. In the US, this vaccine has received regulatory approval for active immunization to prevent IMD caused by Neisseria meningitidis serogroups A, C, Y, and W in individuals from 2 months through 55 years of age. MENVEO does not prevent N. meningitidis serogroup B infections. The US Prescribing Information is available here.¹⁹

Important Safety Information for MENVEO in the US

The following is based on the US Prescribing Information for MENVEO. Please consult the full Prescribing Information for additional safety information

• Do not administer MENVEO to individuals with a severe allergic reaction (e.g., anaphylaxis) to a previous dose of MENVEO, to any component of this vaccine, or to any other diphtheria toxoid-containing vaccine
• Syncope (fainting) has occurred in association with administration of MENVEO
• Some individuals with altered immunocompetence, including some individuals receiving immunosuppressant therapy, may have reduced immune responses to MENVEO
• Individuals with certain complement deficiencies and individuals receiving treatment that inhibits terminal complement activation (for example, eculizumab) are at increased risk for invasive disease caused by Neisseria meningitidis serogroups A, C, Y, and W, even after being vaccinated with MENVEO
• Guillain-Barré syndrome has been reported in temporal relationship following administration of another US-licensed meningococcal quadrivalent polysaccharide conjugate vaccine
• Apnea following intramuscular vaccination has been observed in some infants born prematurely
• Common solicited adverse reactions: at 2 months of age – tenderness and erythema at injection site, irritability, sleepiness, persistent crying, change in eating habits, vomiting, and diarrhea; at 7 months through 23 months of age – tenderness and erythema at injection site, irritability, sleepiness, persistent crying, change in eating habits, and diarrhea; at 2 through 10 years of age – injection site pain, erythema, irritability, induration, sleepiness, malaise, and headache. Among adolescents and adults aged 11 through 55 years were pain at the injection site, headache, myalgia, malaise, and nausea – similar rates were observed following a booster dose
• In two clinical studies, there were no notable differences in frequency and severity of solicited adverse reactions in individuals who received MENVEO 1-vial presentation compared to individuals who received the 2-vial presentation
• Vaccination with MENVEO may not result in protection in all vaccine recipients

About GSK

GSK is a global biopharma company with a purpose to unite science, technology, and talent to get ahead of disease together. Find out more at gsk.com.

Cautionary statement regarding forward-looking statements

GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described under Item 3.D “Risk factors” in GSK’s Annual Report on Form 20-F for 2023, and GSK’s Q4 Results for 2024.

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References:

1. Centers for Disease Control and Prevention. About Meningococcal Disease. Available at: https://www.cdc.gov/meningococcal/about/index.html?CDC_AA_refVal=https%3A%2F%2Fwww.cdc.gov%2Fmeningococcal%2Fabout%2Fcauses-transmission.html. Accessed February 2025.
2. European Centers for Disease Control and Prevention. Factsheet about meningococcal disease. Available at: https://www.ecdc.europa.eu/en/meningococcal-disease/factsheet. Accessed February 2025.
3. GSK. GSK’s 5-in-1 meningococcal ABCWY vaccine candidate accepted for regulatory review by US FDA. Available at: https://www.gsk.com/en-gb/media/press-releases/gsk-s-5-in-1-meningococcal-abcwy-vaccine-candidate-accepted-for-regulatory-review-by-us-fda/. Accessed February 2025.
4. NIH. Effectiveness of GlaxoSmithKline Biologicals S.A.’s Meningococcal Group B and Combined ABCWY Vaccines in Healthy Adolescents and Young Adults, ClinicalTrials.gov. Available at: https://clinicaltrials.gov/study/NCT04502693. Accessed February 2025.
5. NIH. Immunogenicity and Safety Study of GSK’s MenABCWY Vaccine in Healthy Adolescents and Adults Previously Primed With MenACWY Vaccine, ClinicalTrials.gov. Available at: https://www.clinicaltrials.gov/study/NCT04707391. Accessed February 2025.
6. Centers for Disease Control and Prevention. Meningococcal Vaccine Recommendations. Available at: https://www.cdc.gov/meningococcal/hcp/vaccine-recommendations/index.html. Accessed February 2025.
7. Cheng WY, et al. Determinants of Meningococcal ACWY vaccination in adolescents in the US: completion and compliance with the CDC recommendations. Hum Vaccin Immunother. 2020;16(1):176-188.
8. Centers for Disease Control and Prevention. National Vaccination Coverage Among Adolescents Aged 13–17 Years — National Immunization Survey-Teen, United States, 2023. Available at: https://www.cdc.gov/mmwr/volumes/73/wr/mm7333a1.htm#:~:text=Among%20adolescents%20aged%2013%E2%80%9317%20years%20included%20in%20the%202023,view%2Fcdc%2F159388). Accessed February 2025.
9. Based on information licensed from IQVIA: IQVIA, DDD, Meningococcal B market all channels, period January – December 2024, reflecting estimates of real-world activity. All rights reserved.
10. World Health Organisation. Meningitis fact sheet. Available at: https://www.who.int/news-room/fact-sheets/detail/meningitis. Accessed April 2024.
11. Thompson MJ, et al. Clinical recognition of meningococcal disease in children and adolescents. Lancet. 2006;367(9508):397-403.
12. Marshall GS, et al. Understanding the Sequelae of Invasive Meningococcal Disease in the United States. Infect Dis Ther. 2024;13(11):2213-2220.
13. European Centers for Disease Control and Prevention. Outbreak of invasive meningococcal disease in the EU associated with a mass gathering event, the 23rd World Scout Jamboree, in Japan. 21 August 2015. Available at: https://www.ecdc.europa.eu/sites/default/files/media/en/publications/Publications/Meningococcal-disease-scouts-EU-August-2015.pdf. Accessed February 2025.
14. Centers for Disease Control and Prevention. Risk Factors for Meningococcal Disease. Available at: https://www.cdc.gov/meningococcal/risk-factors/index.html. Accessed February 2025.
15. GSK. US Prescribing Information for Penmenvy. Available at: https://gskpro.com/content/dam/global/hcpportal/en_US/Prescribing_Information/Penmenvy/pdf/PENMENVY.PDF. Accessed February 2025.
16. GSK Data on File. Number of Bexsero doses shipped from 2015 to November 2023 REF-219766
17. GSK. US Prescribing Information for Bexsero. Available at: gskpro.com/content/dam/global/hcpportal/en_US/Prescribing_Information/Bexsero/pdf/BEXSERO.PDF. Accessed February 2025.
18. GSK Data on File. Menveo Doses Shipped from 2010 to end of 2022 REF-195452
19. GSK. Prescribing Information for Menveo. Available at: gskpro.com/content/dam/global/hcpportal/en_US/Prescribing_Information/Menveo/pdf/MENVEO.PDF. Accessed February 2025.

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LimmaTech Vaccinates First Participants in Phase 1 Study of Staphylococcus aureus Vaccine Candidate LBT-SA7




LimmaTech Vaccinates First Participants in Phase 1 Study of Staphylococcus aureus Vaccine Candidate LBT-SA7

• CARB-X awards LimmaTech US$6.5 million to advance clinical development of LBT-SA7

ZURICH–(BUSINESS WIRE)–LimmaTech Biologics AG, a clinical-stage biotech company developing vaccines for the prevention of life-threatening diseases, announced today that the first participants have been vaccinated in a Phase 1 controlled study of its multivalent vaccine candidate, LBT-SA7. The candidate is designed to prevent skin and soft tissue infections (SSTIs) caused by the bacterial pathogen Staphylococcus aureus (S. aureus). In this context, the company also announced the award of US$6.5 million from the Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) to advance the clinical development of LBT-SA7.

S. aureus infections pose a significant global health challenge, causing an estimated 1 million deaths annually. Notably, 90% of all community-acquired S. aureus infections are SSTIs. The absence of a vaccine to prevent S. aureus, coupled with only limited treatment options – particularly against multidrug-resistant strains of the pathogen, often described as methicillin-resistant S. aureus (MRSA) – highlights the urgent need for effective preventive solutions. LimmaTech’s vaccine candidate, LBT-SA7, is the first multivalent vaccine entirely based on secreted antigens to address this critical need. LBT-SA7 contains weakened forms of the pathogen’s toxins, referred to as toxoids, designed to prevent infections by neutralizing the toxins secreted by S. aureus. This approach offers a promising solution to combat the widespread bacterial threat.

LimmaTech started a Phase 1 clinical trial (NCT06719219) in the U.S. after receiving a Fast Track designation from the U.S. Food and Drug Administration (FDA). This first-in-human study aims to evaluate the safety and immunogenicity of LBT-SA7 against S. aureus. It is a randomized, double-blinded, and controlled dose-escalation study expected to enroll 130 healthy adults aged 18-50 years. Initial results are anticipated in the second half of 2025.

“Developing an S. aureus vaccine has long been a significant scientific challenge,” explained Dr. Patricia Martin-Killias, Chief Operating Officer of LimmaTech. “We believe LBT-SA7 has the potential to provide a much-needed solution for those suffering from S. aureus infections. We are excited to launch the first-in-human clinical trial for LBT-SA7, bringing us closer to addressing an urgent global health challenge.”

“We are grateful for the significant support from CARB-X, which is not only instrumental in accelerating the clinical development of our S. aureus vaccine candidate LBT-SA7 but also underscores the importance of our mission to develop efficient solutions for preventing microbial infections and protecting from their threatening consequences for affected people,” added Dr. Franz-Werner Haas, Chief Executive Officer of LimmaTech.

Research reported in this press release is supported by CARB-X. CARB-X’s funding for this project is provided in part with federal funds from the U.S. Department of Health and Human Services (HHS); Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority; under agreement number: 75A50122C00028, and by awards from Wellcome (WT224842), Germany’s Federal Ministry of Education and Research (BMBF), and the UK Department of Health and Social Care as part of the Global Antimicrobial Resistance Innovation Fund (GAMRIF). The content of this press release is solely the responsibility of the authors and does not necessarily represent the official views of CARB-X or any of its funders.

About Staphylococcus aureus

Staphylococcus aureus (S. aureus), a Gram-positive bacterial pathogen, affects approximately 30% of the human population while causing a spectrum of infections, from SSTI to severe conditions like pneumonia and bloodstream infections. S. aureus is the leading cause of antimicrobial resistance (AMR)-attributed fatalities with community-acquired and hospital-acquired infections being the most prevalent. SSTIs caused by S. aureus range from mild to severe and entail microbial invasion into the skin layers and underlying soft tissues. Traditional antibiotic treatments, both oral therapy and intravenous administration reserved for severe cases, have become increasingly less effective due to the rise of antibiotic resistance. S. aureus has been designated as a “high priority” pathogen by the World Health Organization (WHO), underscoring the urgency for innovative vaccine approaches and effective treatment strategies.

About LimmaTech Biologics AG

LimmaTech Biologics is at the forefront of combating the global antimicrobial resistance epidemic based on its unparalleled track record in vaccine technology and clinical candidate development. The company is leveraging its proprietary self-adjuvanting and multi-antigen vaccine platform alongside additional disease-specific vaccine approaches to prevent increasingly untreatable microbial infections. With decades of expertise and an expanding, robust pipeline, the LimmaTech team is dedicated to generating protective solutions to deliver transformative value worldwide. LimmaTech Biologics is backed by specialist healthcare investors, including Adjuvant Capital, AXA IM Alts, Novo Holdings REPAIR Impact Fund, and Tenmile. For more information, please visit www.lmtbio.com.

Contacts

LimmaTech Biologics AG
Franz-Werner Haas, CEO

E-Mail: media@lmtbio.com

For media enquiries
Jacob Verghese or Anja Heuer

Trophic Communications

Phone: +49 151 7441 6179

Email: limmatech@trophic.eu

New ‘Super Test’ for Prostate Cancer Developed in the UK by EDX Medical




New ‘Super Test’ for Prostate Cancer Developed in the UK by EDX Medical

CAMBRIDGE, England–(BUSINESS WIRE)–#EDXMedical–Scientists in Cambridge have developed a new ‘super test’ for prostate cancer in an effort to revolutionise screening and diagnosis of the disease and accelerate personalised treatment for patients.

The test identifies the presence or absence of cancerous cells, signs of early and late-stage cancer, whether it is slow or aggressive as well as genetic and hereditary risks in the patient.

The new test involves studying the most comprehensive combination of clinically-validated prostate-related biomarkers currently known, in both blood and urine samples. The interpretation of these biomarkers using a proprietary AI-driven algorithm highlights early signs of cancer and characteristic features that can guide treatment selection.

There are 55,000 new cases of prostate cancer in the UK each year, more than 330,000 across European Union countries and more than one million men undergoing treatment at any one time.

More than 100 clinically validated biomarkers are measured in the new test – which has been developed by EDX Medical Group plc. The biomarkers used in the test are then analysed by the specially created AI-powered algorithm which produces a detailed report of results for doctors. Currently available advanced tests which rely on up to 20 biomarkers per test.

EDX Medical scientists expect the test to consistently deliver exceptionally high accuracy with levels of sensitivity and specificity of between 96-99% across an extended age-range and diverse ethnic groups. By comparison, current standard of care prostate testing, including prostate specific antigen (PSA) tests and biopsies, can be below 50%.

The non-invasive ‘super test’ will detect various sub-types of prostate cancer determining key features particularly important for patients in non-caucasian higher risk groups.

The super test takes a ‘multi-omics’ approach and comprises a combination of multiple proteomic, transcriptomic, genetic/hereditary and epigenetic biomarker signatures which provide detailed biological data. A comprehensive list of phenotypic and symptom data is added to the biomarker data and is simultaneously analysed by the AI algorithm.

Individually, these biomarkers have all been clinically validated and published and in previous trials on more than 31,000 positive prostate cancer samples as well as more than 100,000 control non-cancer samples.

A highly accurate prostate cancer test will provide significant benefits for seemingly well 45-70 year-old men and also for healthcare providers. The increased accuracy should reduce the requirement to run unnecessary MRI scans. The need for highly invasive digital rectal examinations (DRE) will also be dramatically reduced.

The new test is being developed at the Cambridge laboratory of EDX Medical Group which develops and supplies digitally enhanced diagnostic tools for cancer, cardiovascular and infectious diseases. The company has filed a patent application for the test and the AI algorithm with the European Patent Office.

Prof Sir Chris Evans, founder and chief scientific officer of EDX Medical, said: “We have been studying this area intensively and are tremendously excited by what we believe is a truly game-changing test. Every indication thus far shows it will be the most accurate and sensitive screening test available and will be transformative in tackling prostate cancer in men who may have no idea if anything is wrong with them.

“Our integrated approach highlights the potential of combining these molecular signatures, offering a powerful, non-invasive diagnostic tool that can certainly improve clinical outcomes and help personalise treatment for patients. The incorporation of all these biomarkers into routine screening could revolutionise prostate cancer management by enabling earlier detection and more accurate risk prediction. What sets this test apart is the use of so many biomarkers with best-in-class instrument and reagent technology and our bespoke AI algorithm.”

Sir Chris Hoy, who has been diagnosed with prostate cancer and supports campaigns to raise awareness and encourage early diagnosis, said: “Prof Sir Chris Evans and his team encouraged and supported me greatly after my initial diagnosis and I know they have some amazing people and a great commitment to finding better ways to diagnose and treat prostate and other cancers. I now know there is a need for better and more accurate prostate cancer screening tests and I wholeheartedly welcome this initiative by Sir Chris’ EDX.”

Rio Ferdinand, former Manchester United and England captain, whose mother and first wife Rebecca died of cancer, said: “Those of us who have lost a loved one to cancer and have campaigned for research and more awareness know full well the need for better and earlier testing across a range of cancers. Ethnic minority men not only have twice the risk of getting prostate cancer, they also have higher rates of stage 3 and 4 and more aggressive cancer. We know if it can be caught early these men can be mostly cured, but it’s not, and too many men are dying unnecessarily. I believe a test as good as Sir Chris’ EDX one can one day put a stop to this and I look forward to its launch later this year.”

Andy Taylor, guitarist with Duran Duran who was diagnosed with prostate cancer at 55, said: “Prof Sir Chris helped me immensely with my cancer treatment and this revolutionary new test from his labs is simply brilliant. To know your prostate cancer status, stage and type and all your genetics when you were unaware there was a problem at all is a life saver and game-changer. It is so accurate and comprehensive, it can spot many early prostate cancers, save many lives and save a fortune in fruitless treatments: this is priceless.”

Dr Mike Hudson, chief executive of EDX Medical said: “I’m confident that the EDX testing strategy will define a new standard for the early detection and characterisation of emergent, prostate cancer, and provide unique insights to guide optimal treatment selection.”

EDX Medical’s scientific team will validate further clinical data over coming months prior to seeking regulatory approval from the Medicines & Healthcare Products Regulatory Agency (MHRA) and the US Food and Drug Administration (FDA) with a view to launching the test later this year or early 2026.

NOTES TO EDITORS

About multi-omics:

Multi-omics’ combines genomic, transcriptomic and proteomic analyses plus information on additional gene expression products and clinical data to provide significantly enhanced information which can guide clinical decision-making.

About PSA testing

The vast majority of men rely on PSA tests as their first assessment of prostate cancer but elevated PSA is regarded as unreliable for definitive cancer diagnosis. Research has shown that ~70% of men with elevated PSA levels do not have cancer at all whereas around 20% of men who show normal or low PSA levels do have cancer – and often an aggressive sub-type which is only discovered later when limited time remains for effective treatment..

About EDX Medical Group plc

The EDX Medical Group plc is listed on the Apex Segment of the AQSE Growth Market (TIDM: EDX). EDX Medical was founded by Professor Sir Christopher Evans, OBE, a medical and life sciences entrepreneur with more than 30 years of experience, together with CEO, Dr Mike Hudson.

By translating clinical insights into pragmatic solutions combining advanced biological and digital technologies, EDX Medical seeks to cost effectively improve the detection and characterisation of disease in order to personalise treatment in a timely fashion. Early disease detection and biologically-based personal treatment optimisation is considered to be the most impactful way of reducing deaths and lowering the cost of healthcare globally.

EDX Medical Group provides doctors, hospitals and insurers/payers with access to a portfolio of the best clinical diagnostics products and services. The Company operates its own facilities in Cambridge and Oxford, UK, and has a number of significant strategic with world leading companies and organisations in their respective fields.

www.edxmedical.com

Contacts

EDX Medical Group Plc
Dr Mike Hudson (Chief Executive Officer)

+44 (0)7812 345 301

Oberon Capital
Nick Lovering (Corporate Adviser)

Adam Pollock (Corporate Broking)

Mike Seabrook (Corporate Broking)

+44 (0)20 3179 5300

IFC Advisory (Investor Relations)

Tim Metcalfe

Graham Herring

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Media House International

Ramsay Smith

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Gary McQueen

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gary@mediahouse.co.uk

Innate Pharma Announces U.S. FDA Granted Breakthrough Therapy Designation to Lacutamab for Relapsed or Refractory Sézary Syndrome




Innate Pharma Announces U.S. FDA Granted Breakthrough Therapy Designation to Lacutamab for Relapsed or Refractory Sézary Syndrome

• Designation is based on TELLOMAK Phase 2 results demonstrating efficacy and a favorable safety profile in patients with advanced Sézary syndrome heavily pre-treated, post- mogamulizumab.
• Breakthrough Therapy Designation is intended to accelerate the development and regulatory review in the U.S. of drugs that are intended to treat a serious condition; adding to a Fast Track designation by the U.S. FDA received in 2019 as well as a PRIME designation by European Medicines Agency in 2020
• Innate continues to align with regulatory agencies around the confirmatory Phase 3 trial in Cutaneous T Cell Lymphoma and is actively seeking for a partner

MARSEILLE, France–(BUSINESS WIRE)–Regulatory News:

Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to lacutamab, an anti-KIR3DL2 cytotoxicity-inducing antibody, for the treatment of adult patients with relapsed or refractory (r/r) Sézary Syndrome (SS) after at least 2 prior systemic therapies including mogamulizumab.

The BTD is granted based on Phase 1 study results as well as results from the Phase 2 TELLOMAK study, where lacutamab demonstrated encouraging efficacy and a favorable safety profile in heavily pretreated, post-mogamulizumab patients with advanced Sézary syndrome.

“There is a high unmet medical need for patients with Sézary syndrome. In this aggressive and rare form of cutaneous T-cell lymphoma, patients in advanced disease often experience very poor quality of life and are in strong need of new, targeted treatment options,” commented Sonia Quaratino, MD, Chief Medical Officer of Innate Pharma. “The Breakthrough Therapy Designation underscores lacutamab’s potential to transform the patient’s care by achieving clinically meaningful efficacy and favorable safety profile compared to available therapies. This is an important step in Innate’s strategy for lacutamab. We are excited to work with the U.S. FDA to accelerate the development of this therapy.”

A Breakthrough Therapy Designation by the FDA is intended to accelerate the development and regulatory review in the U.S. of drugs that are intended to treat a serious condition and that have shown encouraging early clinical results, which may demonstrate substantial improvement on a clinically significant endpoint over available medicines.

Lacutamab previously received a Fast Track designation by the FDA in 2019 for the treatment of adult patients with relapsed or refractory Sézary syndrome who have received at least two prior systemic therapies as well as a PRIME designation by European Medicines Agency in 2020.

Innate continues to align with the regulatory agencies around the confirmatory Phase 3 trial in CTCL and is actively seeking for a partner.

About Cutaneous T-Cell Lymphoma:

Cutaneous T-Cell Lymphoma (CTCL) is a heterogeneous group of non-Hodgkin’s lymphomas which arise primarily in the skin and are characterized by the presence of malignant clonal mature T-cells. CTCL accounts for approximately 4% of all non-Hodgkin’s lymphomas and has a median age at diagnosis of 55-65 years. Mycosis fungoides, and Sézary syndrome, its leukemic variant, are the most common CTCL subtypes. The overall 5-year survival rate, which depends in part on disease subtype, is approximately 10% for Sézary syndrome. There are approximately 6,000 new CTCL cases in Europe and the United States per year.

About Sézary syndrome:

Sézary syndrome is the leukemic variant of CTCL. Patients often experience very poor quality of life with severe and debilitating pruritus (chronic itchy skin). Despite recent advancements, Sézary syndrome is associated with a high relapse rate with currently available therapies.

About Lacutamab:

Lacutamab is a first-in-class anti-KIR3DL2 humanized cytotoxicity-inducing antibody that is currently in clinical trials for treatment of cutaneous T-cell lymphoma (CTCL), an orphan disease, and peripheral T cell lymphoma (PTCL). Rare cutaneous lymphomas of T lymphocytes have a poor prognosis with few efficacious and safe therapeutic options at advanced stages.

KIR3DL2 is an inhibitory receptor of the KIR family, expressed by approximately 65% of patients across all CTCL subtypes and expressed by up 90% of patients with certain aggressive CTCL subtypes, in particular Sézary syndrome. It is expressed by up to 50% of patients with mycosis fungoides and peripheral T-cell lymphoma (PTCL). It has a restricted expression on normal tissues.

Lacutamab is granted European Medicines Agency (EMA) PRIME designation and US Food and Drug Administration (FDA) granted Fast Track designation and Breakthrough Therapy Designation for the treatment of patients with relapsed or refractory Sézary syndrome who have received at least two prior systemic therapies. Lacutamab is granted orphan drug status in the European Union and in the United States for the treatment of CTCL.

About TELLOMAK:

TELLOMAK (NCT03902184) is a global, open-label, multi-cohort Phase 2 clinical trial in patients with Sézary syndrome and mycosis fungoides (MF) in the United States and Europe. Specifically:

• Cohort 1: lacutamab being evaluated as a single agent in approximately 60 patients with Sézary syndrome who have received at least two prior systemic therapies, including mogamulizumab. The Sézary syndrome cohort of the study could enable the registration of lacutamab in this indication.
• Cohort 2: lacutamab being evaluated as a single agent in patients with MF that express KIR3DL2, as determined at baseline with a Simon 2-stage design.
• Cohort 3: lacutamab being evaluated as a single agent in patients with MF that do not express KIR3DL2, as determined at baseline, with a Simon-2 stage design.
• All comers: lacutamab being evaluated as a single agent in patients with both KIR3DL2 expressing and non-expressing MF to explore the correlation between the level of KIR3DL2 expression and treatment outcomes utilizing a formalin-fixed paraffin embedded (FFPE) assay under development as a companion diagnostic.

The trial is fully enrolled. The primary endpoint of the trial is objective global response rate. Key secondary endpoints are progression-free survival, duration of response, overall survival, quality of life, pharmacokinetics and immunogenicity and adverse events.

About Innate Pharma

Innate Pharma S.A. is a global, clinical-stage biotechnology company developing immunotherapies for cancer patients. Its innovative approach aims to harness the innate immune system through three therapeutic approaches: multi-specific NK Cell Engagers via its ANKET^® (Antibody-based NK cell Engager Therapeutics) proprietary platform and Antibody Drug Conjugates (ADC) and monoclonal antibodies (mAbs).

Innate’s portfolio includes several ANKET^® drug candidates to address multiple tumor types as well as IPH4502, a differentiated ADC in development in solid tumors. In addition, anti-KIR3DL2 mAb lacutamab is developed in advanced form of cutaneous T cell lymphomas and peripheral T cell lymphomas, and anti-NKG2A mAb monalizumab is developed with AstraZeneca in non-small cell lung cancer.

Innate Pharma is a trusted partner to biopharmaceutical companies such as Sanofi and AstraZeneca, as well as leading research institutions, to accelerate innovation, research and development for the benefit of patients.

Headquartered in Marseille, France with a US office in Rockville, MD, Innate Pharma is listed on Euronext Paris and Nasdaq in the US.

Learn more about Innate Pharma at www.innate-pharma.com and follow us on LinkedIn and X.

Information about Innate Pharma shares

Disclaimer on forward-looking information and risk factors

This press release contains certain forward-looking statements, including those within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. The use of certain words, including “anticipate,” “believe,” “can,” “could,” “estimate,” “expect,” “may,” “might,” “potential,” “expect” “should,” “will,” or the negative of these and similar expressions, is intended to identify forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, these forward-looking statements are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those anticipated. These risks and uncertainties include, among other things, the uncertainties inherent in research and development, including related to safety, progression of and results from its ongoing and planned clinical trials and preclinical studies, review and approvals by regulatory authorities of its product candidates, the Company’s reliance on third parties to manufacture its product candidates, the Company’s commercialization efforts and the Company’s continued ability to raise capital to fund its development. For an additional discussion of risks and uncertainties, which could cause the Company’s actual results, financial condition, performance or achievements to differ from those contained in the forward-looking statements, please refer to the Risk Factors (“Facteurs de Risque”) section of the Universal Registration Document filed with the French Financial Markets Authority (“AMF”), which is available on the AMF website http://www.amf-france.org or on Innate Pharma’s website, and public filings and reports filed with the U.S. Securities and Exchange Commission (“SEC”), including the Company’s Annual Report on Form 20-F for the year ended December 31, 2023, and subsequent filings and reports filed with the AMF or SEC, or otherwise made public by the Company. References to the Company’s website and the AMF website are included for information only and the content contained therein, or that can be accessed through them, are not incorporated by reference into, and do not constitute a part of, this press release.

In light of the significant uncertainties in these forward-looking statements, you should not regard these statements as a representation or warranty by the Company or any other person that the Company will achieve its objectives and plans in any specified time frame or at all. The Company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

This press release and the information contained herein do not constitute an offer to sell or a solicitation of an offer to buy or subscribe to shares in Innate Pharma in any country.

Contacts

For additional information, please contact:

Investors

Innate Pharma
Henry Wheeler

Tel.: +33 (0)4 84 90 32 88

Henry.wheeler@innate-pharma.fr

Media Relations

NewCap
Arthur Rouillé

Tel.: +33 (0)1 44 71 00 15

innate@newcap.eu

New Five-Year Sotyktu (deucravacitinib) Data Show Consistent Safety and Durable Response Rates in Moderate-to-Severe Plaque Psoriasis




New Five-Year Sotyktu (deucravacitinib) Data Show Consistent Safety and Durable Response Rates in Moderate-to-Severe Plaque Psoriasis

No new safety signals observed at Year 5 in the POETYK PSO long-term extension trial, consistent with the established Sotyktu safety profile

Following five years of continuous Sotyktu treatment, clinical response was maintained in nearly half of patients for Psoriasis Area and Severity Index (PASI) 90 in the POETYK PSO long-term extension trial

PRINCETON, N.J.–(BUSINESS WIRE)–$BMY #BMS—Bristol Myers Squibb (NYSE:BMY) today announced new five-year results from the POETYK PSO long-term extension (LTE) trial of Sotyktu (deucravacitinib) treatment in adult patients with moderate-to-severe plaque psoriasis. The safety profile of Sotyktu remained consistent through five years with more than 5,000 patient-years of exposure in the trial, with no new safety signals identified. In patients who were treated continuously with Sotyktu, clinical response rates were maintained from Year 1 to Year 5, including Psoriasis Area and Severity Index (PASI) 75, PASI 90 and static Physician’s Global Assessment (sPGA) 0/1 (clear/almost clear).

These data were presented at the Winter Clinical Dermatology Conference – Hawaii (WCH) in Big Island, Waikoloa Village, HI taking place February 14-19, 2025.

“Today’s findings demonstrate the continued long-term safety and efficacy profile of Sotyktu, with patients maintaining skin clearance over five years,” said Mark Lebwohl, MD, dean of Clinical Therapeutics at the Kimberly and Eric J. Waldman Department of Dermatology at the Icahn School of Medicine at Mount Sinai and an investigator and paid consultant for Bristol Myers Squibb. “These results further support the role of Sotyktu, the first TYK2 inhibitor available for patients living with moderate-to-severe plaque psoriasis, as a potential oral standard of care.”

Clinical efficacy outcomes were sustained in patients who were continuously treated with Sotyktu for PASI 75 (72.1%, Year 1; 67.3%, Year 5), PASI 90 (45.9%, Year 1; 46.3%, Year 5) and sPGA 0/1 (57.5%, Year 1; 52.6%, Year 5).

The efficacy analysis included 513 patients who received continuous Sotyktu treatment from Day 1 in the pivotal POETYK PSO-1 and POETYK PSO-2 trials and transitioned to the POETYK PSO-LTE trial, while the safety analysis included 1,519 patients who received at least one dose of Sotyktu during the trials. The patients in this five-year analysis completed 256 weeks of treatment. Efficacy was analyzed using the modified nonresponder imputation (mNRI) method.

“These positive five-year results build upon the established profile of Sotyktu, a first-in-class TYK2 inhibitor, as a transformative oral treatment for psoriasis,” said Edgar Charles, MD, vice president and senior global program lead, Early & Late Development Immunology, Bristol Myers Squibb. “As the leader in TYK2 innovation, we continue our relentless pursuit of bold science to elevate new standards of care for the patients we serve.”

Sotyktu is approved in numerous countries around the world for the treatment of adults with moderate-to-severe plaque psoriasis.

Bristol Myers Squibb thanks the patients and investigators involved in the POETYK PSO clinical trial program.

About the POETYK PSO Clinical Trial Program

PrOgram to Evaluate the efficacy and safety of Sotyktu (deucravacitinib), a selective TYK2 inhibitor (POETYK) PSO-1 (NCT03624127) and POETYK PSO-2 (NCT03611751) were global Phase 3 studies designed to evaluate the efficacy of Sotyktu compared to placebo and Otezla^® (apremilast), and the safety of Sotyktu, in patients with moderate-to-severe plaque psoriasis. Both POETYK PSO-1, which enrolled 666 patients, and POETYK PSO-2, which enrolled 1,020 patients, were multicenter, randomized, double-blind trials that evaluated Sotyktu (6 mg once daily) compared to placebo and Otezla (30 mg twice daily). POETYK PSO-2 included a randomized withdrawal and retreatment period after Week 24.

The co-primary endpoints of both POETYK PSO-1 and POETYK PSO-2 were the percentage of patients who achieved Psoriasis Area and Severity Index (PASI) 75 response and those who achieved static Physician’s Global Assessment (sPGA) score of 0 or 1 (clear/almost clear) at Week 16 versus placebo. Key secondary endpoints of the trials included the percentage of patients who achieved PASI 75 and sPGA 0/1 compared to Otezla at Week 16 and other measures evaluating Sotyktu versus placebo and Otezla.

Across both clinical trials and timepoints, significantly more Sotyktu-treated patients achieved a sPGA score of 0/1, PASI 75 response and PASI 90 response. Responses persisted through Week 52, as 82% (187/228) of patients who achieved PASI 75 with Sotyktu at Week 24 maintained their response at Week 52 in POETYK PSO-1. In POETYK PSO-2, 80% (119/148) of patients who continued Sotyktu maintained PASI 75 response compared to 31% (47/150) of patients who were withdrawn from Sotyktu.

Following the 52-week POETYK PSO-1 and POETYK PSO-2 trials, patients could enroll in the ongoing POETYK PSO long-term extension (LTE) trial (NCT04036435) and receive open-label Sotyktu 6 mg once-daily. In the LTE trial, 1,221 patients were enrolled and received at least one dose of Sotyktu. Efficacy was analyzed utilizing treatment failure rules (TFR) method of imputation, along with sensitivity analyses using modified non-responder imputation and as-observed analysis, which have been used in similar analyses with other agents.

In addition to POETYK PSO-1, POETYK PSO-2 and POETYK PSO-LTE, Bristol Myers Squibb has evaluated Sotyktu in two other Phase 3 studies in psoriasis: POETYK PSO-3 (NCT04167462) and POETYK PSO-4 (NCT03924427).

About Psoriasis

Psoriasis is a widely prevalent, chronic, systemic immune-mediated disease that substantially impairs patients’ physical health, quality of life and work productivity. Psoriasis is a serious global problem, with at least 100 million people worldwide impacted by some form of the disease, including around 14 million people in Europe and approximately 7.5 million people in the United States. Nearly one-quarter of people with psoriasis have cases that are considered moderate-to-severe. Up to 90 percent of patients with psoriasis have psoriasis vulgaris, or plaque psoriasis, which is characterized by distinct round or oval plaques typically covered by silvery-white scales. Despite the availability of effective systemic therapy, many patients with moderate-to-severe plaque psoriasis remain undertreated or even untreated and are dissatisfied with current treatments. People with psoriasis report an impact on their emotional well-being, straining both personal and professional relationships and causing a reduced quality of life. Psoriasis is associated with multiple comorbidities that may impact patients’ well-being, including psoriatic arthritis, cardiovascular disease, metabolic syndrome, obesity, diabetes, inflammatory bowel disease and depression.

About Sotyktu (deucravacitinib)

Sotyktu (deucravacitinib) is an oral, selective, allosteric tyrosine kinase 2 (TYK2) inhibitor with a unique mechanism of action, representing a new class of small molecules. It is the first selective TYK2 inhibitor in clinical studies across multiple immune-mediated diseases. Bristol Myers Squibb scientists designed Sotyktu to selectively target TYK2, thereby inhibiting signaling of interleukin (IL)-23, IL-12 and Type 1 interferons (IFN), key cytokines involved in the pathogenesis of multiple immune-mediated diseases. Sotyktu achieves a high degree of selectivity by binding to the regulatory domain of TYK2, resulting in allosteric inhibition of TYK2 and its downstream functions. Sotyktu selectively inhibits TYK2 at physiologically relevant concentrations. At therapeutic doses, Sotyktu does not inhibit JAK1, JAK2 or JAK3.

Sotyktu is approved in numerous countries around the world for the treatment of adults with moderate-to-severe plaque psoriasis.

Bristol Myers Squibb: Pursuing Bold Science in Immunology to Transform Patients’ Lives

Bristol Myers Squibb is inspired by a single vision – transforming patients’ lives through science. For people living with immune-mediated diseases, the debilitating reality of enduring chronic symptoms and disease progression can make simple tasks and daily life a challenge. Driven by our deep understanding of the immune system that spans over 20 years of experience, we continue to pursue bold science as we work to deliver life-changing medicines that elevate new standards of care across rheumatology, dermatology and pulmonology. Our sequential immunotherapy research framework aims to address the root cause of disease by controlling inflammation, resetting the immune system and promoting immune homeostasis with the goal of achieving transformational efficacy. By continuously pushing the boundaries of scientific knowledge, we strive to bring forward tailored approaches, treatments and combinations that may lead to durable remissions, improved quality of life and functional cures. Our collaborations with patients, caregivers, healthcare providers and researchers inform our patient-centric approach as we aim to break efficacy ceilings and deliver what matters most – the promise of living a better life.

SOTYKTU U.S. INDICATION

SOTYKTU^® (deucravacitinib) is indicated for the treatment of moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy.

Limitations of Use:

SOTYKTU is not recommended for use in combination with other potent immunosuppressants.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

SOTYKTU is contraindicated in patients with a history of hypersensitivity reaction to deucravacitinib or to any of the excipients in SOTYKTU.

WARNINGS AND PRECAUTIONS

Hypersensitivity: Hypersensitivity reactions such as angioedema have been reported. If a clinically significant hypersensitivity reaction occurs, institute appropriate therapy and discontinue SOTYKTU.

Infections: SOTYKTU may increase the risk of infections. Serious infections have been reported in patients with psoriasis who received SOTYKTU. The most common serious infections reported with SOTYKTU included pneumonia and COVID-19. Avoid use of SOTYKTU in patients with an active or serious infection. Consider the risks and benefits of treatment prior to initiating SOTYKTU in patients:

• with chronic or recurrent infection
• who have been exposed to tuberculosis
• with a history of a serious or an opportunistic infection
• with underlying conditions that may predispose them to infection.

Closely monitor patients for the development of signs and symptoms of infection during and after treatment. A patient who develops a new infection during treatment should undergo prompt and complete diagnostic testing, have appropriate antimicrobial therapy initiated and be closely monitored. Interrupt SOTYKTU if a patient develops a serious infection. Do not resume SOTYKTU until the infection resolves or is adequately treated.

Viral Reactivation

Herpes virus reactivation (e.g., herpes zoster, herpes simplex) was reported in clinical trials with SOTYKTU. Through Week 16, herpes simplex infections were reported in 17 patients (6.8 per 100 patient-years) treated with SOTYKTU, and 1 patient (0.8 per 100 patient-years) treated with placebo. Multidermatomal herpes zoster was reported in an immunocompetent patient. During PSO-1, PSO-2, and the open-label extension trial, the majority of patients who reported events of herpes zoster while receiving SOTYKTU were under 50 years of age. The impact of SOTYKTU on chronic viral hepatitis reactivation is unknown. Consider viral hepatitis screening and monitoring for reactivation in accordance with clinical guidelines before starting and during therapy with SOTYKTU. If signs of reactivation occur, consult a hepatitis specialist. SOTYKTU is not recommended for use in patients with active hepatitis B or hepatitis C.

Tuberculosis (TB): In clinical trials, of 4 patients with latent TB who were treated with SOTYKTU and received appropriate TB prophylaxis, no patients developed active TB (during the mean follow-up of 34 weeks). One patient, who did not have latent TB, developed active TB after receiving 54 weeks of SOTYKTU. Evaluate patients for latent and active TB infection prior to initiating treatment with SOTYKTU. Do not administer SOTYKTU to patients with active TB. Initiate treatment of latent TB prior to administering SOTYKTU. Consider anti-TB therapy prior to initiation of SOTYKTU in patients with a past history of latent or active TB in whom an adequate course of treatment cannot be confirmed. Monitor patients for signs and symptoms of active TB during treatment.

Malignancy including Lymphomas: Malignancies, including lymphomas, were observed in clinical trials with SOTYKTU. Consider the benefits and risks for the individual patient prior to initiating or continuing therapy with SOTYKTU, particularly in patients with a known malignancy (other than a successfully treated non-melanoma skin cancer) and patients who develop a malignancy when on treatment with SOTYKTU.

Rhabdomyolysis and Elevated CPK: Treatment with SOTYKTU was associated with an increased incidence of asymptomatic creatine phosphokinase (CPK) elevation and rhabdomyolysis compared to placebo.

Discontinue SOTYKTU if markedly elevated CPK levels occur or myopathy is diagnosed or suspected. Instruct patients to promptly report unexplained muscle pain, tenderness or weakness, particularly if accompanied by malaise or fever.

Laboratory Abnormalities: Treatment with SOTYKTU was associated with increases in triglyceride levels. Periodically evaluate serum triglycerides according to clinical guidelines during treatment. SOTYKTU treatment was associated with an increase in the incidence of liver enzyme elevation compared to placebo. Evaluate liver enzymes at baseline and thereafter in patients with known or suspected liver disease according to routine management. If treatment-related increases in liver enzymes occur and drug-induced liver injury is suspected, interrupt SOTYKTU until a diagnosis of liver injury is excluded.

Immunizations: Prior to initiating therapy with SOTYKTU, consider completion of all age-appropriate immunizations according to current immunization guidelines including prophylactic herpes zoster vaccination. Avoid use of live vaccines in patients treated with SOTYKTU. The response to live or non-live vaccines has not been evaluated.

Potential Risks Related to JAK Inhibition: It is not known whether tyrosine kinase 2 (TYK2) inhibition may be associated with the observed or potential adverse reactions of Janus Kinase (JAK) inhibition. In a large, randomized, postmarketing safety trial of a JAK inhibitor in rheumatoid arthritis (RA), patients 50 years of age and older with at least one cardiovascular risk factor, higher rates of all-cause mortality, including sudden cardiovascular death, major adverse cardiovascular events, overall thrombosis, deep venous thrombosis, pulmonary embolism, and malignancies (excluding non-melanoma skin cancer) were observed in patients treated with the JAK inhibitor compared to those treated with TNF blockers. SOTYKTU is not approved for use in RA.

ADVERSE REACTIONS

Most common adverse reactions (≥1% of patients on SOTYKTU and more frequently than with placebo) include upper respiratory infections, blood creatine phosphokinase increased, herpes simplex, mouth ulcers, folliculitis and acne.

SPECIFIC POPULATIONS

Pregnancy: Available data from case reports on SOTYKTU use during pregnancy are insufficient to evaluate a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Report pregnancies to the Bristol-Myers Squibb Company’s Adverse Event reporting line at 1-800-721-5072.

Lactation: There are no data on the presence of SOTYKTU in human milk, the effects on the breastfed infant, or the effects on milk production. SOTYKTU is present in rat milk. When a drug is present in animal milk, it is likely that the drug will be present in human milk. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for SOTYKTU and any potential adverse effects on the breastfed infant from SOTYKTU or from the underlying maternal condition.

Hepatic Impairment: SOTYKTU is not recommended for use in patients with severe hepatic impairment.

SOTYKTU is available in 6 mg tablets.

Please see U.S. Full Prescribing Information, including Medication Guide, for SOTYKTU.

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, X, YouTube, Facebook and Instagram.

Otezla^® (apremilast) is a registered trademark of Amgen Inc.

Cautionary Statement Regarding Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on historical performance and current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, that results of future post-marketing studies will be consistent with the results of this study, that Sotyktu (deucravacitinib) for the indication described in this release may not be commercially successful, any marketing approvals, if granted, may have significant limitations on their use, and that continued approval of Sotyktu for such indication may be contingent upon verification and description of clinical benefit in additional confirmatory trials. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Bristol Myers Squibb’s business and market, particularly those identified in the cautionary statement and risk factors discussion in Bristol Myers Squibb’s Annual Report on Form 10-K for the year ended December 31, 2024, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, Bristol Myers Squibb undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

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FDA Approves Samsung Bioepis’ OSPOMYV™, XBRYK™ (denosumab-dssb), a Biosimilar to Prolia and Xgeva




FDA Approves Samsung Bioepis’ OSPOMYV™, XBRYK™ (denosumab-dssb), a Biosimilar to Prolia and Xgeva

• OSPOMYV™ and XBRYK™ approved by the U.S. Food and Drug Administration (FDA) for all indications referencing Prolia and Xgeva, respectively
• Samsung Bioepis’ first endocrinology biosimilar to be approved by the FDA – widening its therapeutic areas and portfolios

INCHEON, Korea–(BUSINESS WIRE)–Samsung Bioepis Co., Ltd. today announced that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for OSPOMYV™ (denosumab-dssb; SB16; 60 mg pre-filled syringe) and XBRYK™ (denosumab-dssb; SB16; 120 mg vial), biosimilars referencing Prolia and Xgeva respectively. In addition, the FDA granted a provisional determination for both Ospomyv and Xbryk’s interchangeability designation.

OSPOMYV, referencing Prolia, has been approved for the treatment of postmenopausal women with osteoporosis at high risk for fracture, treatment to increase bone mass in men with osteoporosis at high risk for fracture, treatment of glucocorticoid-induced osteoporosis in men and women at high risk for fracture, treatment to increase bone mass in men at high risk for fracture receiving androgen deprivation therapy for nonmetastatic prostate cancer and for the treatment to increase bone mass in women at high risk for fracture receiving adjuvant aromatase inhibitor therapy for breast cancer.

XBRYK, referencing Xgeva, has been approved for the prevention of skeletal-related events in patients with multiple myeloma and in patients with bone metastases from solid tumors, treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity, and for the treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy.

“The FDA approval of OSPOMYV and XBRYK marks a key step in improving patient access and alleviating treatment cost for patients with osteoporosis and cancer-related bone loss in the US. By providing quality-proven biosimilars, we are helping to address a critical healthcare need and reduce the burden of skeletal fractures that impact patients’ quality of life,” said Byoungin Jung, Vice President and Regulatory Affairs Team Leader at Samsung Bioepis. “This achievement underscores our commitment to healthcare innovation through biosimilars and our mission to meet the growing needs in critical therapeutic areas.”

The FDA approval was based on totality of evidence including analytical, non-clinical data, and clinical data. A randomized, double-blind, three-arm, parallel group, single-dose Phase 1 study demonstrated the pharmacokinetic (PK) equivalence between SB16, EU-sourced denosumab (EU-DEN), and US-sourced denosumab (US-DEN) in healthy male participants. The primary PK endpoints were met, in terms of area under the concentration-time curve (AUC) from time zero to infinity, AUC from time zero to the last quantifiable concentration, and maximum serum concentration.¹ In addition, a randomized, double-blind, multi-center Phase 3 study demonstrated equivalent efficacy and comparable safety, immunogenicity, PK, and pharmacodynamics (PD) profiles between SB16 and reference denosumab (DEN) in postmenopausal osteoporosis (PMO) patients. The primary endpoint was met in terms of percent (%) change from baseline in lumbar spine bone mineral density (BMD) at Month 12, and a follow-up up to Month 18 demonstrated switching to SB16 from DEN were comparable up to Month 18 in terms of efficacy, PK, PD, safety and immunogenicity.^2,3

OSPOMYV and XBRYK, marks Samsung Bioepis’ 9^th and 10^th FDA approved medicines as well as the company’s first FDA approval for an endocrinology biosimilar, setting another milestone for the company to broaden its biosimilar portfolio that cover a spectrum of therapeutic areas including immunology, oncology, ophthalmology, hematology and endocrinology.

OSPOMYV™ (denosumab-dssb) injection, for subcutaneous use

Ospomyv is a RANK ligand (RANKL) inhibitor indicated for treatment:

• of postmenopausal women with osteoporosis at high risk for fracture
• to increase bone mass in men with osteoporosis at high risk for fracture
• of glucocorticoid-induced osteoporosis in men and women at high risk for fracture
• to increase bone mass in men at high risk for fracture receiving androgen deprivation therapy for nonmetastatic prostate cancer
• to increase bone mass in women at high risk for fracture receiving adjuvant aromatase inhibitor therapy for breast cancer

SELECTED SAFETY INFORMATION

CONTRAINDICATIONS

• Hypocalcemia
• Pregnancy
• Known hypersensitivity to denosumab products

WARNINGS AND PRECAUTIONS

• Hypocalcemia: Pre-existing hypocalcemia must be corrected before initiating Ospomyv. May worsen, especially in patients with renal impairment. Adequately supplement all patients with calcium and vitamin D. Concomitant use of calcimimetic drugs may also worsen hypocalcemia risk. Evaluate for presence of chronic kidney disease mineral-bone disorder. Monitor serum calcium.
• Same Active Ingredient: Patients receiving Ospomyv should not receive other denosumab products concomitantly.
• Hypersensitivity including anaphylactic reactions may occur. Discontinue permanently if a clinically significant reaction occurs.
• Osteonecrosis of the jaw: Has been reported with denosumab products. Monitor for symptoms.
• Atypical femoral fractures: Have been reported. Evaluate patients with thigh or groin pain to rule out a femoral fracture.
• Multiple vertebral fractures have been reported following treatment discontinuation. Patients should be transitioned to another antiresorptive agent if Ospomyv is discontinued.
• Serious infections including skin infections: May occur, including those leading to hospitalization. Advise patients to seek prompt medical attention if they develop signs or symptoms of infection, including cellulitis.
• Dermatologic reactions: Dermatitis, rashes, and eczema have been reported. Consider discontinuing Ospomyv if severe symptoms develop.
• Severe bone, joint, muscle pain may occur. Discontinue use if severe symptoms develop.
• Suppression of bone turnover: Significant suppression has been demonstrated. Monitor for consequences of bone over-suppression.

These highlights do not include all the information needed to use OSPOMYV safely and effectively. See full prescribing information for OSPOMYV HERE, which includes the Boxed Warning, Medication Guide and Instructions for Use.

XBRYK™ (denosumab-dssb) injection, for subcutaneous use

Xbryk is a RANK ligand (RANKL) inhibitor indicated for:

• Prevention of skeletal-related events in patients with multiple myeloma and in patients with bone metastases from solid tumors.
• Treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity.
• Treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy.

SELECTED SAFETY INFORMATION

CONTRAINDICATIONS

• Hypocalcemia
• Known clinically significant hypersensitivity to denosumab products

WARNINGS AND PRECAUTIONS

• Same Active Ingredient: Patients receiving Xbryk should not receive other denosumab products concomitantly.
• Hypersensitivity reactions including anaphylaxis may occur. Discontinue permanently if a clinically significant reaction occurs.
• Hypocalcemia: Denosumab products can cause severe symptomatic hypocalcemia. Fatal cases have been reported with denosumab products use. Correct hypocalcemia prior to initiating Xbryk. Monitor calcium levels during therapy, especially in the first weeks of initiating therapy, and adequately supplement all patients with calcium and vitamin D.
• Osteonecrosis of the jaw (ONJ) has been reported in patients receiving denosumab products. Perform an oral examination prior to starting Xbryk. Monitor for symptoms. Avoid invasive dental procedures during treatment with Xbryk.
• Atypical femoral fracture: Evaluate patients with thigh or groin pain to rule out a femoral fracture.
• Hypercalcemia Following Treatment Discontinuation in Patients with Giant Cell Tumor of Bone and in Patients with Growing Skeletons: Monitor patients for signs and symptoms of hypercalcemia, and manage as clinically appropriate.
• Multiple Vertebral Fractures (MVF) Following Treatment Discontinuation: When Xbryk treatment is discontinued, evaluate the individual patient’s risk for vertebral fractures.
• Embryo-Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential of potential risk to the fetus and to use effective contraception.

These highlights do not include all the information needed to use XBRYK safely and effectively. See full prescribing information for XBRYK HERE, which includes the Boxed Warning, Medication Guide and Instructions for Use.

About Samsung Bioepis Co., Ltd.

Established in 2012, Samsung Bioepis is a biopharmaceutical company committed to realizing healthcare that is accessible to everyone. Through innovations in product development and a firm commitment to quality, Samsung Bioepis aims to become the world’s leading biopharmaceutical company. Samsung Bioepis continues to advance a broad pipeline of biosimilar candidates that cover a spectrum of therapeutic areas, including immunology, oncology, ophthalmology, hematology, nephrology, and endocrinology. For more information, please visit: www.samsungbioepis.com and follow us on social media – X, LinkedIn.

¹ Lee HA, Kim S, Seo H, Kim S. A phase I, randomized, double-blind, single-dose pharmacokinetic study to evaluate the biosimilarity of SB16 (proposed denosumab biosimilar) with reference denosumab in healthy male subjects. Expert Opin Investig Drugs. 2023 Jul-Dec;32(10):959-966. doi: 10.1080/13543784.2023.2273510. Epub 2023 Nov 6. PMID: 37870163.

² Langdahl B, Chung YS, Plebanski R, Czerwinski E, Dokoupilova E, Supronik J, Rosa J, Mydlak A, Rowińska-Osuch A, Baek KH, Urboniene A, Mordaka R, Ahn S, Rho YH, Ban J, Eastell R. Proposed Denosumab Biosimilar SB16 vs Reference Denosumab in Postmenopausal Osteoporosis: Phase 3 Results Up to Month 12. J Clin Endocrinol Metab. 2024 Sep 7:dgae611. doi: 10.1210/clinem/dgae611. Epub ahead of print. PMID: 39243386.

³ Richard Eastell, Bente Langdahl, Yoon-Sok Chung, Rafal Plebanski, Edward Czerwinski,Eva Dokoupilova, Jerzy Supronik, Jan Rosa, Andrzej Mydlak, Anna Rowinska-Osuch, Ki-Hyun Baek, Audrone Urboniene, Robert Mordaka, Sohui Ahn, Young Hee Rho, Jisuk Ban. A Randomized, Double-blind, Phase III Study to Compare SB16 (Proposed Denosumab Biosimilar) to Reference Denosumab in Patients with Postmenopausal Osteoporosis: 18-Month Results. Oral presentation at 2024 European Calcified Tissue Society (ECTS) Congress. May 25-28, 2024. Marseille, France.

Contacts

MEDIA CONTACT
Yoon Kim, yoon1.kim@samsung.com
Anna Nayun Kim, nayun86.kim@samsung.com

Samsung Bioepis Gains European Commission (EC) Approval for Denosumab Biosimilar (OBODENCE™, XBRYK™)




Samsung Bioepis Gains European Commission (EC) Approval for Denosumab Biosimilar (OBODENCE™, XBRYK™)

• OBODENCE™ and XBRYK™ approved by the European Commission (EC) for all indications referencing Prolia and Xgeva, respectively
• Marks Samsung Bioepis’ 10^th and 11^th product and first endocrinology treatment approved by the EC – adding to its growing portfolio of biosimilars

INCHEON, Korea–(BUSINESS WIRE)–Samsung Bioepis Co., Ltd. today announced that the European Commission (EC) has granted marketing authorization for OBODENCE™ (60mg pre-filled syringe) and XBRYK™ (120mg vial), denosumab biosimilars referencing Prolia and Xgeva – formerly referred to as SB16.

OBODENCE, referencing Prolia, has been approved for the treatment of osteoporosis in postmenopausal women and in men at increased risk of fractures, treatment of bone loss associated with hormone ablation in men with prostate cancer at increased risk of fractures, and treatment of bone loss associated with long-term systemic glucocorticoid therapy in adult patients at increased risk of fracture.

XBRYK, referencing Xgeva, has been approved for the prevention of skeletal related events (pathological fracture, radiation to bone, spinal cord compression or surgery to bone) in adults with advanced malignancies involving bone, and treatment of adults and skeletally mature adolescents with giant cell tumour of bone that is unresectable or where surgical resection is likely to result in severe morbidity.

Osteoporosis is a major concern in Europe as it results in 4.3 million fragility fractures and health care costs in excess of €56 billion annually. Less than half of women at high risk of fracture are treated despite the high cost of fractures and the availability of affordable medications.¹ In addition, skeletal related events (SREs) resulting from bone metastases can lead to severe pain, increased risk of death, increased health care costs and reduced quality of life.²

The EC approval was based on totality of evidence including analytical, non-clinical data, and clinical data. A randomized, double-blind, three-arm, parallel group, single-dose Phase 1 study demonstrated the pharmacokinetic (PK) equivalence between SB16, EU-sourced denosumab (EU-DEN), and US-sourced denosumab (US-DEN) in healthy male participants. The primary PK endpoints were met, in terms of area under the concentration-time curve (AUC) from time zero to infinity, and maximum serum concentration.³ In addition, a randomized, double-blind, multi-center Phase 3 study demonstrated equivalent efficacy and comparable safety, immunogenicity, PK, and pharmacodynamics (PD) profiles between SB16 and reference denosumab (DEN) in postmenopausal osteoporosis (PMO) patients. The primary endpoint was met in terms of percent (%) change from baseline in lumbar spine bone mineral density (BMD) at Month 12, and a follow-up up to Month 18 demonstrated switching to SB16 from DEN were comparable up to Month 18 in terms of efficacy, PK, PD, safety and immunogenicity.^4,5

OBODENCE and XBRYK, marking Samsung Bioepis’ 10^th and 11^th EC approvals as well as the company’s first EC approval for an endocrinology biosimilar, add to the company’s growing portfolio in new therapeutic areas.

About Samsung Bioepis Co., Ltd.

Established in 2012, Samsung Bioepis is a biopharmaceutical company committed to realizing healthcare that is accessible to everyone. Through innovations in product development and a firm commitment to quality, Samsung Bioepis aims to become the world’s leading biopharmaceutical company. Samsung Bioepis continues to advance a broad pipeline of biosimilar candidates that cover a spectrum of therapeutic areas, including immunology, oncology, ophthalmology, hematology, nephrology, and endocrinology. For more information, please visit: www.samsungbioepis.com and follow us on social media – X, LinkedIn.

¹ International Osteoporosis Foundation. SCORECARD FOR OSTEOPOROSIS IN EUROPE: SCOPE 2021 Summary Report. Available at: https://www.osteoporosis.foundation/scope-2021. Accessed January 2025.

² So A, Chin J, Fleshner N, Saad F. Management of skeletal-related events in patients with advanced prostate cancer and bone metastases: Incorporating new agents into clinical practice. Can Urol Assoc J. 2012 Dec;6(6):465-70. doi: 10.5489/cuaj.12149. PMID: 23282666; PMCID: PMC3526633.

³ Lee HA, Kim S, Seo H, Kim S. A phase I, randomized, double-blind, single-dose pharmacokinetic study to evaluate the biosimilarity of SB16 (proposed denosumab biosimilar) with reference denosumab in healthy male subjects. Expert Opin Investig Drugs. 2023 Jul-Dec;32(10):959-966. doi: 10.1080/13543784.2023.2273510. Epub 2023 Nov 6. PMID: 37870163.

⁴ Langdahl B, Chung YS, Plebanski R, Czerwinski E, Dokoupilova E, Supronik J, Rosa J, Mydlak A, Rowińska-Osuch A, Baek KH, Urboniene A, Mordaka R, Ahn S, Rho YH, Ban J, Eastell R. Proposed Denosumab Biosimilar SB16 vs Reference Denosumab in Postmenopausal Osteoporosis: Phase 3 Results Up to Month 12. J Clin Endocrinol Metab. 2024 Sep 7:dgae611. doi: 10.1210/clinem/dgae611. Epub ahead of print. PMID: 39243386.

⁵ Richard Eastell, Bente Langdahl, Yoon-Sok Chung, Rafal Plebanski, Edward Czerwinski, Eva Dokoupilova, Jerzy Supronik, Jan Rosa, Andrzej Mydlak, Anna Rowinska-Osuch, Ki-Hyun Baek, Audrone Urboniene, Robert Mordaka, Sohui Ahn, Young Hee Rho, Jisuk Ban. A Randomized, Double-blind, Phase III Study to Compare SB16 (Proposed Denosumab Biosimilar) to Reference Denosumab in Patients with Postmenopausal Osteoporosis: 18-Month Results. Oral presentation at 2024 European Calcified Tissue Society (ECTS) Congress. May 25-28, 2024. Marseille, France.

Contacts

MEDIA CONTACT
Yoon Kim, yoon1.kim@samsung.com
Anna Nayun Kim, nayun86.kim@samsung.com

New Data Demonstrated Best-in-Class Potential for Casdatifan, a HIF-2a Inhibitor, in Patients with Metastatic Kidney Cancer




New Data Demonstrated Best-in-Class Potential for Casdatifan, a HIF-2a Inhibitor, in Patients with Metastatic Kidney Cancer

• A 9.7-month median progression-free survival (mPFS) was reached for the 50mg twice-daily (BID) casdatifan monotherapy cohort of the Phase 1/1b ARC-20 study; mPFS was not yet reached for other cohorts
• Across all three monotherapy cohorts presented, casdatifan demonstrated improvements in the rate of primary progression, overall response rate (ORR) and progression-free survival (PFS) relative to published data from studies with HIF-2a inhibitors to date
• Arcus will host a conference call to discuss these data at 5:00 AM PT / 8:00 AM ET on Tuesday, February 18, 2025

HAYWARD, Calif.–(BUSINESS WIRE)–Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for people with cancer, today presented new data for casdatifan, a HIF-2a inhibitor with best-in-class potential, in an oral plenary session by Dr. Toni K. Choueiri, Dana-Farber Cancer Institute, at the 2025 American Society of Clinical Oncology (ASCO) Genitourinary (GU) Cancers Symposium.

“The newest data are from the 100mg cohort using the tablet formulation and the expected go-forward dose for pivotal studies, which showed a 33% confirmed response rate despite the relatively short follow-up,” said Richard Markus, M.D., Ph.D., chief medical officer at Arcus Biosciences. “Casdatifan continues to be well tolerated, with a very low discontinuation rate, supporting its strong potential in combination therapy. We look forward to sharing initial results for the casdatifan plus cabozantinib cohort later this year.”

ARC-20 is a Phase 1/1b dose-escalation and expansion study. New data include mPFS and ORR for the 50mg BID cohort, and ORR for the 50mg once-daily (QD) and 100mg QD (tablet) cohorts, all of which evaluated casdatifan in patients with metastatic clear cell renal cell carcinoma (ccRCC), most of whom had progressed on at least two prior lines of therapy, including both an anti-PD-1 and a VEGFR tyrosine kinase inhibitor (TKI) therapy. The patient population was heavily pretreated; more than half (52-59%) of subjects received at least three prior lines of therapy and approximately one quarter (24-29%) had received at least four prior lines of therapy. Most patients (70-76%) had an International Metastatic Renal Cell Carcinoma Database Consortium (IMDC) risk factor of intermediate or poor.

Casdatifan showed improvement in primary progressive disease rate (progressed at or before their first disease assessment), ORR and mPFS relative to published data from studies with HIF-2a inhibitors to date. At the time of data cut off (DCO, January 3, 2025), most patients (81-87%) experienced disease control with either a partial response or stable disease, and most were still on treatment. The median duration of response had not been reached, with all but two of the 26 responders across all three cohorts still on treatment.

No unexpected safety signals were observed at the time of DCO, and casdatifan had an acceptable and manageable safety profile across all doses. Across all three cohorts, only one patient discontinued treatment as a result of anemia and only two due to hypoxia. A summary of the efficacy and safety results is below.

CI: confidence interval; NE: not estimable

^a Efficacy-evaluable population for this expansion cohort is defined as all eligible participants who received any study treatment and have at least one post-baseline efficacy assessment, or who discontinued study treatment due to progressive disease or death.

^b Majority of patients (n=21) were still on treatment at time of DCO.

^c In the 50mg BID cohort, one unconfirmed responder remains on treatment. In the 50mg QD cohort, one unconfirmed responder became a confirmed responder after the DCO, increasing the cORR to 32%.

^d Unconfirmed best overall response.

^e Includes two patients with radiological progressive disease and two patients who had clinical progression before the first scan.

^a The safety-evaluable population included all dose expansion enrolled patients who received any amount of any study treatment.

Arcus is pursuing a broad development program in both the immuno-oncology (IO)-naive and post-IO settings with differentiated combinations to maximize the opportunity for casdatifan in ccRCC. These studies include:

• Arcus’s planned Phase 3 study, PEAK-1, which will evaluate casdatifan in combination with cabozantinib versus cabozantinib monotherapy as a first- or second-line treatment in patients with metastatic ccRCC who have previously received anti-PD-1 therapy. The primary endpoint will be PFS with a key secondary endpoint of overall survival.
• A planned Phase 1b study operationalized by AstraZeneca (part of the eVOLVE portfolio) to evaluate casdatifan in combination with volrustomig, an investigational anti-PD-1/CTLA-4 bispecific antibody.
• Initiation of two additional cohorts in ARC-20 to evaluate casdatifan in first-line settings.

Investors may dial in to the conference call at +1 404 975 4839 (local) or +1 833 470 1428 (toll-free) using Conference ID: 331780 on Tuesday, February 18, 2025, at 5:00 AM PT / 8:00 AM ET. Participants may also register for the call online using the following link: https://events.q4inc.com/attendee/364282703. To access the live webcast and accompanying slide presentation, please visit the “Investors & Media” section of the Arcus Biosciences website at www.arcusbio.com. A replay will be available following the live event.

About Casdatifan (AB521)

Casdatifan is a small-molecule inhibitor of HIF-2a, a transcription factor responsible for activating multiple tumor growth pathways in hypoxic and pseudo-hypoxic tumor environments. By selectively binding HIF-2a, casdatifan is designed to shut down hypoxic oncogenesis and key oncogenic pathways leading to cancer cell death. Clear cell RCC (ccRCC) is almost universally associated with HIF-2a dysregulation. Casdatifan is currently being evaluated in ARC-20, a Phase 1/1b study in renal cell carcinoma and other cancers.

Casdatifan is an investigational molecule. Approval from any regulatory authority for its use has not been received, and its safety and efficacy have not been established.

About RCC

According to the American Cancer Society, kidney cancer is among the top 10 most commonly diagnosed forms of cancer among both men and women in the U.S., and an estimated 80,980 Americans will be diagnosed with kidney cancer in 2025. Clear cell RCC is the most common type of kidney cancer in adults. If detected in its early stages, the five-year survival rate for RCC is high; for patients with advanced or late-stage metastatic RCC, however, the five-year survival rate is only 18%. In 2022, approximately 32,200 patients with advanced kidney cancer required systemic therapy in the U.S., with over 20,000 patients receiving first-line treatment.

About Arcus Biosciences

Arcus Biosciences is a clinical-stage, global biopharmaceutical company developing differentiated molecules and combination medicines for people with cancer. In partnership with industry collaborators, patients and physicians around the world, Arcus is expediting the development of first- or best-in-class medicines against well-characterized biological targets and pathways and studying novel, biology-driven combinations that have the potential to help people with cancer live longer. Founded in 2015, the company has expedited the development of multiple investigational medicines into clinical studies, including new combination approaches that target TIGIT, PD-1, HIF-2a, CD73, dual A2a/A2b receptor, CD39 and AXL. For more information about Arcus Biosciences’s clinical and preclinical programs, please visit www.arcusbio.com.

Forward Looking Statements

This press release contains forward-looking statements. All statements regarding events or results to occur in the future contained herein are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, the statements in Dr. Markus’s quotes and statements regarding: the potency, efficacy or safety of casdatifan, including its potential for a best-in-class profile and potential as a combination therapy; and Arcus’s development plans for the casdatifan program, including expected timing and design for new studies and cohorts and plans for generating data to support initiation of future studies. All forward-looking statements involve known and unknown risks and uncertainties and other important factors that may cause Arcus’s actual results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to risks associated with: interim data not being replicated in future studies evaluating the same investigational molecules or regimen; the unexpected emergence of adverse events or other undesirable side effects with casdatifan; risks associated with manufacturing or supplying product for such clinical trials; uncertainties in timelines associated with the conduct of clinical studies and with respect to the regulatory application process; difficulties associated with the management of the collaboration activities with our strategic partners or expanded clinical programs; changes in the competitive landscape for Arcus’s programs; and the inherent uncertainty associated with pharmaceutical product development and clinical trials. Risks and uncertainties facing Arcus are described more fully in the “Risk Factors” section of Arcus’s most recent periodic report filed with the U.S. Securities and Exchange Commission. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date of this press release. Arcus disclaims any obligation or undertaking to update, supplement or revise any forward-looking statements contained in this press release except to the extent required by law.

The Arcus name and logo are trademarks of Arcus Biosciences, Inc. All other trademarks belong to their respective owners.

Contacts

Investor Inquiries:
Pia Eaves

VP of Investor Relations & Strategy

(617) 459-2006

peaves@arcusbio.com

Media Inquiries:
Holli Kolkey

VP of Corporate Affairs

(650) 922-1269

hkolkey@arcusbio.com

Maryam Bassiri

AD, Corporate Communications

(510) 406-8520

mbassiri@arcusbio.com

Exelixis Announces Final Five-Year Follow-up Results from CheckMate -9ER Trial Evaluating CABOMETYX® (cabozantinib) in Combination with Opdivo® (nivolumab) in Patients with Advanced Kidney Cancer at ASCO GU 2025




Exelixis Announces Final Five-Year Follow-up Results from CheckMate -9ER Trial Evaluating CABOMETYX® (cabozantinib) in Combination with Opdivo® (nivolumab) in Patients with Advanced Kidney Cancer at ASCO GU 2025

– After more than five years of follow-up, CABOMETYX in combination with Opdivo continued to show survival benefit compared with sunitinib –

– Long-term efficacy seen across subgroups, including site of metastases –

ALAMEDA, Calif.–(BUSINESS WIRE)–Exelixis, Inc. (Nasdaq: EXEL) today announced final results from the phase 3 CheckMate -9ER pivotal trial evaluating CABOMETYX^® (cabozantinib) in combination with Opdivo^® (nivolumab) versus sunitinib for patients with previously untreated advanced renal cell carcinoma (RCC). After more than five years of follow-up, the findings demonstrated that efficacy benefits with CABOMETYX in combination with Opdivo were sustained long term. These results, including subgroup analyses, will be presented at 8:10 a.m. PT on February 15 during Oral Abstract Session C: Renal Cell Cancer and Testicular Cancer at the American Society of Clinical Oncology 2025 Genitourinary Cancers Symposium (ASCO GU).

“In this evolving treatment landscape for renal cell carcinoma, patients are looking for options that have shown improved survival time in the long-term,” said Robert J. Motzer, M.D., Kidney Cancer Section Head, Genitourinary Oncology Service, Memorial Sloan Kettering Cancer Center. “These final five-year results from CheckMate -9ER demonstrated the durable clinical benefits of cabozantinib in combination with nivolumab—including for those with organ metastases or intermediate- or poor-risk disease classifications—and continue to support this combination regimen as a valuable first-line option for this patient population.”

At a median follow-up of 67.6 months, CABOMETYX in combination with Opdivo improved progression-free survival (PFS; hazard ratio [HR]: 0.58; 95% confidence interval [CI]: 0.49-0.70) and overall survival (OS; HR: 0.79; 95% CI: 0.65-0.96) compared with sunitinib in the intent-to-treat population. A subgroup analysis by International Metastatic RCC Database Consortium (IMDC) risk showed PFS and objective response rates (ORR) favored CABOMETYX in combination with Opdivo versus sunitinib regardless of IMDC risk group. Detailed results are shown in Table 1.

In an analysis by baseline metastases sites, PFS, OS and ORR favored the combination regimen versus sunitinib in all three subgroups (liver, bone and lung). Detailed results are shown in Table 2.

“With now more than five years of follow-up, these results continue to support CABOMETYX in combination with Opdivo as a treatment regimen that can have enduring survival benefits for patients with previously untreated advanced kidney cancer,” said Amy Peterson, M.D., Executive Vice President, Product Development & Medical Affairs, and Chief Medical Officer, Exelixis. “The efficacy was sustained across multiple subgroups, further underscoring the potential of this regimen to benefit a broad population with variable disease burden. We are proud to have established such a compelling standard of care for this community and remain committed to developing much-needed treatment options for all patients living with advanced cancers.”

Safety and tolerability with long-term follow-up were manageable and consistent with previous analyses. No new safety signals were reported. Grade 3/4 adverse events (AEs) occurred in 68% of patients treated with CABOMETYX in combination with Opdivo versus 55% of patients treated with sunitinib, with the most frequent being diarrhea (7% versus 5%, respectively), palmar-plantar erythrodysesthesia (8% versus 8%), hypertension (13% versus 13%), fatigue (3% versus 5%), thrombocytopenia (<1% versus 5%) and alanine aminotransferase increased (6% versus 1%). One treatment-related death per investigator occurred with CABOMETYX in combination with Opdivo versus three with sunitinib. Treatment-related AEs leading to discontinuation occurred in 28% of patients treated with CABOMETYX in combination with Opdivo versus 11% of patients treated with sunitinib.

About CheckMate -9ER

CheckMate -9ER is an open-label, randomized, multi-national phase 3 trial evaluating patients with previously untreated advanced or metastatic RCC. A total of 651 patients (23% favorable risk, 58% intermediate risk, 20% poor risk; 25% tumor PD-L1≥1%) were randomized to receive CABOMETYX in combination with Opdivo (n=323) versus sunitinib (n=328). The primary endpoint is PFS. Secondary endpoints include OS and ORR. The primary efficacy analysis is comparing the doublet combination versus sunitinib in all randomized patients. The trial is sponsored by Bristol Myers Squibb and Ono Pharmaceutical Co. and co-funded by Exelixis, Inc., Ipsen Pharma SAS and Takeda Pharmaceutical Company Limited.

About RCC

Kidney cancer is among the top ten most commonly diagnosed forms of cancer among both men and women in the U.S.¹ An estimated 80,980 Americans will be diagnosed with kidney cancer in 2025.¹ If detected in its early stages, the five-year survival rate for RCC is high; for patients with advanced or late-stage metastatic RCC, however, the five-year survival rate is only 18%.² In 2024, approximately 33,200 patients with advanced kidney cancer required systemic therapy in the U.S., with over 21,000 patients receiving first-line treatment.³

About CABOMETYX^® (cabozantinib)

In the U.S., CABOMETYX tablets are approved as monotherapy for the treatment of patients with advanced RCC and in combination with nivolumab as a first-line treatment for patients with advanced RCC; for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib; and for adult and pediatric patients 12 years of age and older with locally advanced or metastatic differentiated thyroid cancer (DTC) that has progressed following prior VEGFR-targeted therapy and who are radioactive iodine-refractory or ineligible. CABOMETYX tablets have also received regulatory approvals in over 65 countries outside the U.S. and Japan, including the European Union. In 2016, Exelixis granted Ipsen Pharma SAS exclusive rights for the commercialization and further clinical development of cabozantinib outside of the U.S. and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the U.S.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Hemorrhage: Severe and fatal hemorrhages occurred with CABOMETYX. The incidence of Grade 3 to 5 hemorrhagic events was 5% in CABOMETYX patients in RCC, HCC, and DTC studies. Discontinue CABOMETYX for Grade 3 or 4 hemorrhage and prior to surgery as recommended. Do not administer CABOMETYX to patients who have a recent history of hemorrhage, including hemoptysis, hematemesis, or melena.

Perforations and Fistulas: Fistulas, including fatal cases, occurred in 1% of CABOMETYX patients. Gastrointestinal (GI) perforations, including fatal cases, occurred in 1% of CABOMETYX patients. Monitor patients for signs and symptoms of fistulas and perforations, including abscess and sepsis. Discontinue CABOMETYX in patients who experience a Grade 4 fistula or a GI perforation.

Thrombotic Events: CABOMETYX increased the risk of thrombotic events. Venous thromboembolism occurred in 7% (including 4% pulmonary embolism) and arterial thromboembolism in 2% of CABOMETYX patients. Fatal thrombotic events occurred in CABOMETYX patients. Discontinue CABOMETYX in patients who develop an acute myocardial infarction or serious arterial or venous thromboembolic events that require medical intervention.

Hypertension and Hypertensive Crisis: CABOMETYX can cause hypertension, including hypertensive crisis. Hypertension was reported in 37% (16% Grade 3 and <1% Grade 4) of CABOMETYX patients. Do not initiate CABOMETYX in patients with uncontrolled hypertension. Monitor blood pressure regularly during CABOMETYX treatment. Withhold CABOMETYX for hypertension that is not adequately controlled with medical management; when controlled, resume at a reduced dose. Permanently discontinue CABOMETYX for severe hypertension that cannot be controlled with anti-hypertensive therapy or for hypertensive crisis.

Diarrhea: Diarrhea occurred in 62% of CABOMETYX patients. Grade 3 diarrhea occurred in 10% of CABOMETYX patients. Monitor and manage patients using antidiarrheals as indicated. Withhold CABOMETYX until improvement to ≤ Grade 1, resume at a reduced dose.

Palmar-Plantar Erythrodysesthesia (PPE): PPE occurred in 45% of CABOMETYX patients. Grade 3 PPE occurred in 13% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 PPE or Grade 3 PPE.

Hepatotoxicity: CABOMETYX in combination with nivolumab can cause hepatic toxicity with higher frequencies of Grades 3 and 4 ALT and AST elevations compared to CABOMETYX alone.

Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes than when the drugs are administered as single agents. For elevated liver enzymes, interrupt CABOMETYX and nivolumab and consider administering corticosteroids.

With the combination of CABOMETYX and nivolumab, Grades 3 and 4 increased ALT or AST were seen in 11% of patients. ALT or AST >3 times ULN (Grade ≥2) was reported in 83 patients, of whom 23 (28%) received systemic corticosteroids; ALT or AST resolved to Grades 0-1 in 74 (89%). Among the 44 patients with Grade ≥2 increased ALT or AST who were rechallenged with either CABOMETYX (n=9) or nivolumab (n=11) as a single agent or with both (n=24), recurrence of Grade ≥2 increased ALT or AST was observed in 2 patients receiving CABOMETYX, 2 patients receiving nivolumab, and 7 patients receiving both CABOMETYX and nivolumab. Withhold and resume at a reduced dose based on severity.

Adrenal Insufficiency: CABOMETYX in combination with nivolumab can cause primary or secondary adrenal insufficiency. For Grade 2 or higher adrenal insufficiency, initiate symptomatic treatment, including hormone replacement as clinically indicated. Withhold CABOMETYX and/or nivolumab and resume CABOMETYX at a reduced dose depending on severity.

Adrenal insufficiency occurred in 4.7% (15/320) of patients with RCC who received CABOMETYX with nivolumab, including Grade 3 (2.2%), and Grade 2 (1.9%) adverse reactions. Adrenal insufficiency led to permanent discontinuation of CABOMETYX and nivolumab in 0.9% and withholding of CABOMETYX and nivolumab in 2.8% of patients with RCC.

Approximately 80% (12/15) of patients with adrenal insufficiency received hormone replacement therapy, including systemic corticosteroids. Adrenal insufficiency resolved in 27% (n=4) of the 15 patients. Of the 9 patients in whom CABOMETYX with nivolumab was withheld for adrenal insufficiency, 6 reinstated treatment after symptom improvement; of these, all (n=6) received hormone replacement therapy and 2 had recurrence of adrenal insufficiency.

Proteinuria: Proteinuria was observed in 8% of CABOMETYX patients. Monitor urine protein regularly during CABOMETYX treatment. For Grade 2 or 3 proteinuria, withhold CABOMETYX until improvement to ≤ Grade 1 proteinuria; resume CABOMETYX at a reduced dose. Discontinue CABOMETYX in patients who develop nephrotic syndrome.

Osteonecrosis of the Jaw (ONJ): ONJ occurred in <1% of CABOMETYX patients. ONJ can manifest as jaw pain, osteomyelitis, osteitis, bone erosion, tooth or periodontal infection, toothache, gingival ulceration or erosion, persistent jaw pain, or slow healing of the mouth or jaw after dental surgery. Perform an oral examination prior to CABOMETYX initiation and periodically during treatment. Advise patients regarding good oral hygiene practices. Withhold CABOMETYX for at least 3 weeks prior to scheduled dental surgery or invasive dental procedures, if possible. Withhold CABOMETYX for development of ONJ until complete resolution, resume at a reduced dose.

Impaired Wound Healing: Wound complications occurred with CABOMETYX. Withhold CABOMETYX for at least 3 weeks prior to elective surgery. Do not administer CABOMETYX for at least 2 weeks after major surgery and until adequate wound healing. The safety of resumption of CABOMETYX after resolution of wound healing complications has not been established.

Reversible Posterior Leukoencephalopathy Syndrome (RPLS): RPLS, a syndrome of subcortical vasogenic edema diagnosed by characteristic findings on MRI, can occur with CABOMETYX. Evaluate for RPLS in patients presenting with seizures, headache, visual disturbances, confusion, or altered mental function. Discontinue CABOMETYX in patients who develop RPLS.

Thyroid Dysfunction: Thyroid dysfunction, primarily hypothyroidism, has been observed with CABOMETYX. Based on the safety population, thyroid dysfunction occurred in 19% of patients treated with CABOMETYX, including Grade 3 in 0.4% of patients.

Patients should be assessed for signs of thyroid dysfunction prior to the initiation of CABOMETYX and monitored for signs and symptoms of thyroid dysfunction during CABOMETYX treatment. Thyroid function testing and management of dysfunction should be performed as clinically indicated.

Hypocalcemia: CABOMETYX can cause hypocalcemia. Based on the safety population, hypocalcemia occurred in 13% of patients treated with CABOMETYX, including Grade 3 in 2% and Grade 4 in 1% of patients. Laboratory abnormality data were not collected in CABOSUN.

In COSMIC-311, hypocalcemia occurred in 36% of patients treated with CABOMETYX, including Grade 3 in 6% and Grade 4 in 3% of patients.

Monitor blood calcium levels and replace calcium as necessary during treatment. Withhold and resume at reduced dose upon recovery or permanently discontinue CABOMETYX depending on severity.

Embryo-Fetal Toxicity: CABOMETYX can cause fetal harm. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Verify the pregnancy status of females of reproductive potential prior to initiating CABOMETYX and advise them to use effective contraception during treatment and for 4 months after the last dose.

ADVERSE REACTIONS

The most common (≥20%) adverse reactions are:

CABOMETYX as a single agent: diarrhea, fatigue, PPE, decreased appetite, hypertension, nausea, vomiting, weight decreased, and constipation.

CABOMETYX in combination with nivolumab: diarrhea, fatigue, hepatotoxicity, PPE, stomatitis, rash, hypertension, hypothyroidism, musculoskeletal pain, decreased appetite, nausea, dysgeusia, abdominal pain, cough, and upper respiratory tract infection.

DRUG INTERACTIONS

Strong CYP3A4 Inhibitors: If coadministration with strong CYP3A4 inhibitors cannot be avoided, reduce the CABOMETYX dosage. Avoid grapefruit or grapefruit juice.

Strong CYP3A4 Inducers: If coadministration with strong CYP3A4 inducers cannot be avoided, increase the CABOMETYX dosage. Avoid St. John’s wort.

USE IN SPECIFIC POPULATIONS

Lactation: Advise women not to breastfeed during CABOMETYX treatment and for 4 months after the final dose.

Hepatic Impairment: In patients with moderate hepatic impairment, reduce the CABOMETYX dosage. Avoid CABOMETYX in patients with severe hepatic impairment.

Please see accompanying full Prescribing Information https://www.cabometyx.com/downloads/CABOMETYXUSPI.pdf.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.FDA.gov/medwatch or call 1-800-FDA-1088.

About Exelixis

Exelixis is a globally ambitious oncology company innovating next-generation medicines and regimens at the forefront of cancer care. Powered by drug discovery and development excellence, we are rapidly evolving our product portfolio to target an expanding range of tumor types and indications with our clinically differentiated pipeline of small molecules, antibody-drug conjugates and other biotherapeutics. This comprehensive approach harnesses decades of robust investment in our science and partnerships to advance our investigational programs and extend the impact of our flagship commercial product, CABOMETYX^® (cabozantinib). Exelixis is driven by a bold scientific pursuit to create transformational treatments that give more patients hope for the future. For information about the company and its mission to help cancer patients recover stronger and live longer, visit www.exelixis.com, follow @ExelixisInc on X (Twitter), like Exelixis, Inc. on Facebook and follow Exelixis on LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including, without limitation, statements related to: the presentation of final results from the CheckMate -9ER trial at ASCO GU 2025; the therapeutic potential of cabozantinib in combination with nivolumab and Exelixis’ belief that the regimen may provide enduring survival benefits for patients with previously untreated advanced kidney cancer; Exelixis’ belief in the ability of the regimen to benefit a broad population with variable disease burden; Exelixis’ commitment to developing much-needed treatment options for all patients living with advanced cancers; and Exelixis’ scientific pursuit to create transformational treatments that give more patients hope for the future. Any statements that refer to expectations, projections or other characterizations of future events or circumstances are forward-looking statements and are based upon Exelixis’ current plans, assumptions, beliefs, expectations, estimates and projections. Forward-looking statements involve risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in the forward-looking statements as a result of these risks and uncertainties, which include, without limitation: the availability of data at the referenced times; complexities and the unpredictability of the regulatory review and approval processes in the U.S. and elsewhere; Exelixis’ and Bristol Myers Squibb’s continuing compliance with applicable legal and regulatory requirements; the potential failure of cabozantinib in combination with nivolumab to demonstrate safety and/or efficacy in future clinical testing; unexpected concerns that may arise as a result of the occurrence of adverse safety events or additional data analyses of clinical trials evaluating cabozantinib; the costs of conducting clinical trials; Exelixis’ dependence on third-party vendors for the development, manufacture and supply of cabozantinib; Exelixis’ and Bristol Myers Squibb’s ability to protect their respective intellectual property rights; market competition, including the potential for competitors to obtain approval for generic versions of CABOMETYX; changes in economic and business conditions; and other factors affecting Exelixis and its development programs detailed from time to time under the caption “Risk Factors” in Exelixis’ most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q, and in Exelixis’ future filings with the Securities and Exchange Commission. All forward-looking statements in this press release are based on information available to Exelixis as of the date of this press release, and Exelixis undertakes no obligation to update or revise any forward-looking statements contained herein, except as required by law.

Exelixis, the Exelixis logo and CABOMETYX are registered U.S. trademarks of Exelixis.

______________________________

¹ Cancer Facts & Figures 2025. ACS. Available at: https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2025/2025-cancer-facts-and-figures-acs.pdf. Accessed February 2025.

² Survival Rates for Kidney Cancer. ACS. Available at: https://www.cancer.org/cancer/types/kidney-cancer/detection-diagnosis-staging/survival-rates.html. Accessed February 2025.

³ Citeline’s Datamonitor Healthcare: Renal Cell Carcinoma. March 2023 (internal data on file).

Contacts

Investors Contact:
Susan Hubbard
EVP, Public Affairs and
Investor Relations
Exelixis, Inc.
(650) 837-8194
shubbard@exelixis.com

Media Contact:
Claire McConnaughey
Senior Director, Public Affairs
Exelixis, Inc.
(650) 837-7052
cmcconn@exelixis.com

U.S. FDA Grants Full Approval of Deciphera’s ROMVIMZA™ (vimseltinib) for the Treatment of Symptomatic Tenosynovial Giant Cell Tumor (TGCT)




U.S. FDA Grants Full Approval of Deciphera’s ROMVIMZA™ (vimseltinib) for the Treatment of Symptomatic Tenosynovial Giant Cell Tumor (TGCT)

In the MOTION Phase 3 study, ROMVIMZA met primary endpoint of improved objective response rate (ORR) compared to placebo and all key secondary endpoints with statistically significant and clinically meaningful improvements in quality-of-life measures, and demonstrated well-tolerated safety profile

OSAKA, Japan & WALTHAM, Mass.–(BUSINESS WIRE)–Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President: Toichi Takino; “Ono”) announced that the U.S. Food and Drug Administration (FDA) has approved ROMVIMZA™ (vimseltinib), a kinase inhibitor, for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity. The FDA previously granted Fast Track designation and Priority Review for ROMVIMZA, which was developed by Deciphera Pharmaceuticals, Inc. (“Deciphera”), a wholly owned subsidiary of Ono.

“The approval of ROMVIMZA provides a new, much-needed, well-tolerated, and effective treatment option for people suffering from TGCT,” said Hans Gelderblom, M.D., Ph.D., Chair of the Department of Medical Oncology at Leiden University Medical Center. “TGCT adversely affects the lives of patients, causing significant pain, limited mobility, and stiffness. The MOTION Phase 3 study demonstrated ROMVIMZA’s ability to shrink tumors along with being the first well-tolerated agent to demonstrate significant improvement in a number of other important quality-of-life measures without any observed liver injury as seen with other approved TGCT treatment. ROMVIMZA is a differentiated treatment that has the potential to address the significant unmet needs of the TGCT community.”

“The FDA approval of ROMVIMZA for TGCT is a crucial advancement for the TGCT community and we believe ROMVIMZA has the potential to become the new standard of care for people with TGCT for which surgical resection will potentially cause worsening functional limitation or severe morbidity. This is also an important milestone for our organization, as it is the second approved therapy discovered using Deciphera’s proprietary switch-control kinase inhibitor platform,” said Ryota Udagawa, President and Chief Executive Officer of Deciphera Pharmaceuticals. “I’d like to extend my gratitude to the patients, families, caregivers, and healthcare providers who contributed to the success in ROMVIMZA’s clinical studies. Their commitment, along with the dedication of the Deciphera and Ono teams, enabled us to advance this impactful new treatment, which we look forward to delivering to patients.”

TGCT is a rare, non-malignant tumor that forms within or near joints. TGCT arises from the dysregulation of the CSF1 gene, resulting in an overproduction of CSF1. If left untreated or if the tumor repeatedly recurs, it can lead to damage and degeneration in the affected joint and surrounding tissues, potentially causing significant disability.

The FDA approval was based on the efficacy and safety results from the pivotal Phase 3 MOTION study of ROMVIMZA in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib allowed), compared to placebo, as well as the Phase 1/2 study of ROMVIMZA. In MOTION, ROMVIMZA demonstrated a statistically significant and clinically meaningful ORR at Week 25 in the intent-to-treat (ITT) population, as assessed by blinded independent radiologic review (IRR) per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1), versus placebo (40% in ROMVIMZA arm vs 0% in placebo arm, p <0.0001). The primary endpoint was supported by statistically significant and clinically meaningful improvements in active range of motion, patient-reported physical functioning, and patient-reported pain observed in the vimseltinib arm compared to the placebo arm at week 25. The safety profile of ROMVIMZA is manageable and consistent with results previously disclosed in the Phase 1/2 clinical trial.

Deciphera Pharmaceuticals plans to make ROMVIMZA commercially available in the U.S. next week. Learn more at www.ROMVIMZA.com.

In July of 2024, the Company announced the marketing authorization application (MAA) for ROMVIMZA for the treatment of patients with TGCT was accepted and is under review by the European Medicines Agency (EMA).

Deciphera is committed to supporting TGCT patients and providers in navigating coverage and access to ROMVIMZA. As part of that commitment, Deciphera AccessPoint^TM, a patient support program, is available to provide comprehensive access and financial assistance programs for eligible patients. For more information, visit DecipheraAccessPoint.com or call 1-833-4DACCES (1-833-432-2237), Monday-Friday, 8:00 AM to 8:00 PM Eastern Time (ET).

About MOTION Study

The MOTION study is a two-part, randomized, double-blind, placebo-controlled Phase 3 clinical study to assess the efficacy and safety of vimseltinib in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib allowed). The primary endpoint of the study is an objective response rate (ORR) at Week 25 in the intent-to-treat (ITT) population, as assessed by blinded independent radiologic review (IRR) per using Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1), versus placebo. The secondary endpoints include ORR per tumor volume score (TVS), active range of motion (ROM), physical function, stiffness, quality of life, and pain, all assessed at Week 25.

This study consists of two Parts. In Part 1, patients were randomized to receive either vimseltinib or placebo for 24 weeks. In Part 2, patients randomized to placebo in Part 1 have the option to receive vimseltinib, and all patients receive vimseltinib for a long-term period in an open-label setting.

ROMVIMZA (vimseltinib) capsules

INDICATIONS AND USAGE

ROMVIMZA is indicated for treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Hepatotoxicity

• Cases of serious and fatal liver injury have occurred with the use of another kinase inhibitor that targets CSF1R. Serious and fatal liver injury have not been observed with ROMVIMZA.
• Elevated AST and ALT can occur with ROMVIMZA.
• Avoid ROMVIMZA in patients with pre-existing increased serum transaminases; total bilirubin or direct bilirubin (>ULN); or active liver or biliary tract disease, including ALP.
• Monitor liver function tests prior to initiation of ROMVIMZA, twice a month for the first two months and once every 3 months for the first year of therapy and as clinically indicated thereafter. Withhold and reduce the dose, or permanently discontinue ROMVIMZA based on the severity of the hepatotoxicity.

Embryo-Fetal Toxicity:

• ROMVIMZA may cause fetal harm when administered to pregnant women. Advise pregnant women on the potential risk to the fetus.
• Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with ROMVIMZA and for 1 month after the last dose.

Allergic Reactions to FD&C Yellow No.5 (Tartrazine) and No. 6 (Sunset Yellow FCF):

• ROMVIMZA 20 mg capsule contains FD&C Yellow No. 5 (tartrazine) which may cause allergic reactions (including bronchial asthma) in certain susceptible patients. FD&C Yellow No. 5 (tartrazine) sensitivity is frequently seen in patients who also have aspirin sensitivity.
• Advise patients that ROMVIMZA 14 mg and 20 mg capsules contain FD&C Yellow No.6 (Sunset Yellow FCF), which may cause allergic reactions.

Increased Creatinine without Affecting Renal Function:

• Increases in serum creatinine can occur with the use of ROMVIMZA. These increases in serum creatinine may not be associated with changes in renal function. Increases in creatinine reversed upon ROMVIMZA discontinuation. During ROMVIMZA treatment, use alternative measures that are not based on serum creatinine to assess renal function.

Adverse Reactions:

The most common (≥20%) adverse reactions, including laboratory abnormalities that occurred in patients receiving ROMVIMZA were increased AST, periorbital edema, fatigue, rash, increased cholesterol, peripheral edema, face edema, decreased neutrophils, decreased leukocytes, pruritus, and increased ALT.

Drug Interactions:

• P-glycoprotein (P-gp) substrates: Avoid concomitant use of ROMVIMZA with P-gp substrates. If concomitant use cannot be avoided, take ROMVIMZA at least 4 hours prior to P-gp substrates.
• Breast Cancer Resistance Protein (BCRP) substrates: Avoid concomitant use of ROMVIMZA with BCRP substrates.
• Organic Cation Transporter 2 (OCT2) substrates: Avoid concomitant use of ROMVIMZA with OCT2 substrates.
• Concomitant use of vimseltinib with P-gp substrates, BCRP substrates or OCT2 substrates may increase exposure of these substrates.

Lactation: Advise females not to breastfeed during treatment with ROMVIMZA.

Please see the accompanying full Prescribing Information.

About Tenosynovial Giant Cell Tumor (TGCT)

TGCT is a rare disease caused by a translocation in colony-stimulating factor 1 (CSF1) gene resulting in overexpression of CSF1 and recruitment of colony-stimulating factor 1 receptor (CSF1R)-positive inflammatory cells into the lesion. TGCT is a rare, non-malignant tumor that develops inside or near joints. TGCT is caused by dysregulation of the CSF1 gene leading to overproduction of CSF1. TGCT is also known as giant cell tumor of the tendon sheath (GCT-TS) or pigmented villonodular synovitis (PVNS), a diffuse-type of TGCT. Although non-malignant, these tumors can grow and cause damage to surrounding tissues and structures inducing pain, swelling, and limitation of movement of the joint. Surgery is the main treatment option; however, these tumors tend to recur, particularly in diffuse-type TGCT. If untreated or if the tumor continually recurs, damage and degeneration may occur in the affected joint and surrounding tissues, which may cause significant disability. For a subset of patients, surgical resection will potentially cause worsening functional limitation or severe morbidity, systemic treatment options are limited and a new therapeutic option for TGCT is needed.

About Deciphera Pharmaceuticals Inc.

(As of June 11, 2024, Deciphera became a member of Ono Pharmaceutical Co., Ltd.)

Deciphera is a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer. We are leveraging our proprietary switch-control kinase inhibitor platform and deep expertise in kinase biology to develop a broad portfolio of innovative medicines. In addition to advancing multiple product candidates from our platform in clinical studies, QINLOCK^® (ripretinib) is Deciphera’s switch-control inhibitor approved in many countries including the European Union and the United States for the treatment of fourth-line gastrointestinal stromal tumor (GIST). ROMVIMZA™ (vimseltinib) is a kinase inhibitor approved in the United States for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity. For more information, visit www.deciphera.com and follow us on LinkedIn and Twitter (@Deciphera).

Cautionary Note Regarding Forward-Looking Statements

In this press release, statements made with respect to current plans, estimates, strategies and beliefs, and other statements that are not historical facts are forward-looking statements about the future performance of the company. These statements are based on current assumptions and beliefs in light of the information currently available and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in the business environment in the pharmaceutical market and amendments to relevant laws and regulations, (ii) disruptions to product supply due to stagnation or delays in production caused by natural disasters, fires, etc., (iii) the possibility that sales activities for new and existing products may not achieve the expected results, (iv) the emergence of new side effects in post-marketing drugs, and (v) infringements of intellectual property rights by third parties. Information about pharmaceutical products included in this press release is not intended to constitute an advertisement or medical advice.

Contacts

Ono Pharmaceutical Co., Ltd.
Corporate Communications

public_relations@ono-pharma.com

Deciphera Pharmaceuticals, Inc.
Investor Relations:

Maghan Meyers

Argot Partners

Deciphera@argotpartners.com
212-600-1902

Media:

David Rosen

Argot Partners

david.rosen@argotpartners.com
646-461-6387