Datopotamab Deruxtecan New BLA Submitted for Accelerated Approval in the U.S. for Patients with Previously Treated Advanced EGFR-Mutated Non-Small Cell Lung Cancer




Datopotamab Deruxtecan New BLA Submitted for Accelerated Approval in the U.S. for Patients with Previously Treated Advanced EGFR-Mutated Non-Small Cell Lung Cancer

• Daiichi Sankyo and AstraZeneca’s new application is based on the TROPION-Lung05 phase 2 trial and supported by data from additional trials including TROPION-Lung01
• Previously submitted BLA based on TROPION-Lung01 phase 3 trial for patients with nonsquamous NSCLC has been voluntarily withdrawn

TOKYO & BASKING RIDGE, N.J.–(BUSINESS WIRE)–Daiichi Sankyo (TSE: 4568) and AstraZeneca (LSE/STO/Nasdaq: AZN) have submitted a new Biologics License Application (BLA) for accelerated approval in the U.S. for datopotamab deruxtecan (Dato-DXd) for the treatment of adult patients with locally advanced or metastatic epidermal growth factor receptor-mutated (EGFR-mutated) non-small cell lung cancer (NSCLC) who have received prior systemic therapies, including an EGFR-directed therapy.

The companies have voluntarily withdrawn the BLA in the U.S. for datopotamab deruxtecan for patients with advanced or metastatic nonsquamous NSCLC based on the TROPION-Lung01 phase 3 trial.

The decision to submit a new BLA for EGFR-mutated NSCLC and withdraw the previously submitted BLA for nonsquamous NSCLC was informed by feedback from the U.S. Food and Drug Administration (FDA).

The new BLA is based on results from the TROPION-Lung05 phase 2 trial and supported by data from the TROPION-Lung01 phase 3 and TROPION-PanTumor01 phase 1 trials. New results from a pooled analysis of patients with previously treated EGFR-mutated NSCLC in the TROPION-Lung05 and TROPION-Lung01 trials will be featured in a late-breaking presentation at the upcoming European Society for Medical Oncology (ESMO) Asia 2024 Congress (LBA7).

Datopotamab deruxtecan is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed by Daiichi Sankyo and AstraZeneca.

“Treating EGFR-mutated non-small cell lung cancer is incredibly challenging following disease progression given that the complexity and variability of these mutations often lead to resistance,” said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. “The potential approval of datopotamab deruxtecan could offer renewed hope for patients with this formidable disease.”

“TROPION-Lung01 was designed to test the potential to improve upon standard-of-care chemotherapy in a broad, previously treated, advanced lung cancer patient population. The results, together with data from TROPION-Lung05, showed an especially pronounced benefit for patients with an EGFR mutation which informed our discussions with the FDA and the decision to seek accelerated approval of datopotamab deruxtecan in this patient population,” said Susan Galbraith, MBBChir, PhD, Executive Vice President, Oncology R&D, AstraZeneca. “TROPION-Lung01 has also provided exciting exploratory data supporting our biomarker development, which will be validated in ongoing and planned phase 3 lung cancer trials.”

Daiichi Sankyo and AstraZeneca are evaluating datopotamab deruxtecan alone and with osimertinib, AstraZeneca’s EGFR tyrosine kinase inhibitor (TKI), as treatment for patients with advanced or metastatic EGFR-mutated nonsquamous NSCLC in the ongoing TROPION-Lung14 and TROPION-Lung15 phase 3 trials. In addition, ongoing phase 3 trials in first-line advanced or metastatic nonsquamous NSCLC, AVANZAR and TROPION-Lung10, have the potential to validate the QCS (quantitative continuous scoring) biomarker for TROP2 identified in an exploratory analysis of TROPION-Lung01. An additional trial in patients with biomarker-positive tumors in the second-line nonsquamous NSCLC setting is also planned.

About TROPION-Lung05

TROPION-Lung05 is a global, multicenter, single-arm, open-label phase 2 trial evaluating the efficacy and safety of datopotamab deruxtecan in patients with locally advanced or metastatic NSCLC with actionable genomic alterations who have progressed on or after one regimen of platinum-based chemotherapy and at least one TKI (with or without other systemic therapies). Patients receiving up to four prior lines of treatment with tumors with one or more genomic alterations including EGFR, ALK, ROS1, NTRK, BRAF, RET or MET were eligible for the trial.

The primary trial endpoint of TROPION-Lung05 is objective response rate (ORR) as assessed by blinded independent central review (BICR). Secondary efficacy endpoints include duration of response (DoR), disease control rate (DCR), clinical benefit rate (CBR), progression-free survival (PFS), time to response (TTR), overall survival (OS) and safety.

TROPION-Lung05 enrolled 137 patients globally in Asia, Europe and North America. For more information visit ClinicalTrials.gov.

About TROPION-Lung01

TROPION-Lung01 is a global, randomized, multicenter, open-label phase 3 trial evaluating the efficacy and safety of datopotamab deruxtecan versus docetaxel in adult patients with locally advanced or metastatic NSCLC with and without actionable genomic alterations who require systemic therapy following prior treatment. Patients with actionable genomic alterations were previously treated with an approved targeted therapy and platinum-based chemotherapy. Patients without known actionable genomic alterations were previously treated, concurrently or sequentially, with platinum-based chemotherapy and a PD-1 or PD-L1 inhibitor.

The dual primary endpoints of TROPION-Lung01 are PFS as assessed by BICR and OS. Key secondary endpoints include investigator-assessed PFS, ORR, DoR, TTR, and DCR as assessed by both BICR and investigator, and safety.

TROPION-Lung01 enrolled approximately 600 patients in Asia, Europe, North America, Oceania and South America. For more information visit ClinicalTrials.gov.

Primary PFS results and interim OS results from TROPION-Lung01 were presented at the 2023 ESMO (#ESMO23) Congress. Final OS results were presented at IASLC 2024 World Conference on Lung Cancer hosted by the International Association for the Study of Lung Cancer (#WCLC24) and simultaneously published in the Journal of Clinical Oncology in September 2024.

About TROPION-PanTumor01

TROPION-PanTumor01 is a first-in-human, open-label, two-part, multicenter phase 1 trial evaluating the safety and preliminary efficacy of datopotamab deruxtecan in patients with advanced solid tumors that have relapsed or are refractory to standard treatment or for which no standard treatment is available. The dose escalation portion of the trial enrolled patients with NSCLC to assess the safety and tolerability of datopotamab deruxtecan to determine the recommended dose for expansion (6 mg/kg). The dose expansion part of TROPION-PanTumor01 is enrolling several different cohorts including patients with NSCLC, triple negative breast cancer, HR positive, HER2 low or negative breast cancer, small cell lung cancer, urothelial, gastric, pancreatic, castration resistant prostate and esophageal cancer.

Safety endpoints include dose-limiting toxicities and serious adverse events. Efficacy endpoints include ORR, DoR, TTR, PFS and OS. Pharmacokinetic, biomarker and immunogenicity endpoints also are being evaluated.

TROPION-PanTumor01 enrolled approximately 900 patients in Asia and North America. For more information visit ClinicalTrials.gov.

About Advanced Non-Small Cell Lung Cancer

Nearly 2.5 million lung cancer cases were diagnosed globally in 2022.¹ Lung cancer is broadly split into small or non-small cell lung cancer, the latter accounting for about 80% of cases.² Approximately 10 to 15% of patients with NSCLC in the U.S. and Europe, and 30 to 40% of patients in Asia have an EGFR mutation.^3,4 The majority of EGFR mutations occur in tumors of nonsquamous histology.⁵

For patients with tumors that have an EGFR mutation, the established first-line treatment in the metastatic setting is an EGFR-TKI.⁶ While EGFR TKIs have improved outcomes in the first-line setting, most patients eventually experience disease progression and receive chemotherapy.^7,8,9,10

TROP2 is a protein broadly expressed in the majority of NSCLC tumors.¹¹ There is currently no TROP2 directed ADC approved for the treatment of lung cancer.^6,12

About Datopotamab Deruxtecan (Dato-DXd)

Datopotamab deruxtecan (Dato-DXd) is an investigational TROP2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, datopotamab deruxtecan is one of six DXd ADCs in the oncology pipeline of Daiichi Sankyo, and one of the most advanced programs in AstraZeneca’s ADC scientific platform. Datopotamab deruxtecan is comprised of a humanized anti-TROP2 IgG1 monoclonal antibody, developed in collaboration with Sapporo Medical University, attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

A comprehensive global clinical development program is underway with more than 20 trials evaluating the efficacy and safety of datopotamab deruxtecan across multiple cancers, including NSCLC, triple negative breast cancer and HR positive, HER2 low or negative breast cancer. The program includes seven phase 3 trials in lung cancer and five phase 3 trials in breast cancer evaluating datopotamab deruxtecan as a monotherapy and in combination with other anticancer treatments in various settings.

About the Daiichi Sankyo and AstraZeneca Collaboration

Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialize ENHERTU in March 2019 and datopotamab deruxtecan (Dato-DXd) in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights for each ADC. Daiichi Sankyo is responsible for the manufacturing and supply of ENHERTU and datopotamab deruxtecan.

About the ADC Portfolio of Daiichi Sankyo

The Daiichi Sankyo ADC portfolio consists of seven ADCs in clinical development crafted from two distinct ADC technology platforms discovered in-house by Daiichi Sankyo.

The ADC platform furthest in clinical development is Daiichi Sankyo’s DXd ADC Technology where each ADC consists of a monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. The DXd ADC portfolio currently consists of ENHERTU, a HER2 directed ADC, and datopotamab deruxtecan (Dato-DXd), a TROP2 directed ADC, which are being jointly developed and commercialized globally with AstraZeneca. Patritumab deruxtecan (HER3-DXd), a HER3 directed ADC, ifinatamab deruxtecan (I-DXd), a B7-H3 directed ADC, and raludotatug deruxtecan (R-DXd), a CDH6 directed ADC, are being jointly developed and commercialized globally with Merck. DS-3939, a TA-MUC1 directed ADC, is being developed by Daiichi Sankyo.

The second Daiichi Sankyo ADC platform consists of a monoclonal antibody attached to a modified pyrrolobenzodiazepine (PBD) payload. DS-9606, a CLDN6 directed PBD ADC, is the first of several planned ADCs in clinical development utilizing this platform.

Datopotamab deruxtecan, ifinatamab deruxtecan, patritumab deruxtecan, raludotatug deruxtecan, DS-3939 and DS-9606 are investigational medicines that have not been approved for any indication in any country. Safety and efficacy have not been established.

About Daiichi Sankyo

Daiichi Sankyo is an innovative global healthcare company contributing to the sustainable development of society that discovers, develops and delivers new standards of care to enrich the quality of life around the world. With more than 120 years of experience, Daiichi Sankyo leverages its world-class science and technology to create new modalities and innovative medicines for people with cancer, cardiovascular and other diseases with high unmet medical need. For more information, please visit www.daiichisankyo.com.

Contacts

Media Contacts:

Global/US:
Jennifer Brennan

Daiichi Sankyo, Inc.

jennifer.brennan@daiichisankyo.com
+1 908 900 3183 (mobile)

Japan:
Daiichi Sankyo Co., Ltd.

DS-PR@daiichisankyo.co.jp

Investor Relations Contact:
DaiichiSankyoIR@daiichisankyo.co.jp

Pimicotinib Significantly Improved Outcomes for Patients with Tenosynovial Giant Cell Tumor in a Global Phase III Trial




Pimicotinib Significantly Improved Outcomes for Patients with Tenosynovial Giant Cell Tumor in a Global Phase III Trial

• Phase III MANEUVER study met primary endpoint, with an objective response rate at week 25 of 54.0% for pimicotinib versus 3.2% for placebo (p<0.0001)
• Statistically significant and clinically meaningful improvements also seen in all key secondary endpoints, including pain and stiffness
• Oral, once-daily treatment with pimicotinib was well-tolerated, with very low rates of discontinuation due to treatment-related adverse events
• Pimicotinib is being developed by Abbisko Therapeutics Co., Ltd.; Merck holds commercial license for pimicotinib in Chinese mainland, Hong Kong, Macau, and Taiwan, with option for rest of world

Not intended for Canada-, UK- or US-based media

DARMSTADT, Germany–(BUSINESS WIRE)–Merck, a leading science and technology company, today announced that the Phase III MANEUVER trial of pimicotinib, an investigational medicine being developed by Abbisko Therapeutics Co., Ltd., met its primary endpoint, demonstrating significant improvement in objective response rate (ORR) in patients with tenosynovial giant cell tumor (TGCT). The ORR for pimicotinib at week 25 was 54.0% compared with 3.2% for placebo (p<0.0001).

Notably, the study also showed that treatment with pimicotinib provided statistically significant and clinically meaningful improvements in secondary endpoints associated with important patient outcomes in TGCT, including stiffness by Numeric Rating Scale (NRS; -3.00 mean change from baseline vs. -0.57 for placebo, p<0.0001) and pain by Brief Pain Inventory (BPI; -2.32 vs. 0.23 mean change from baseline, p<0.0001). Further efficacy and safety data from the MANEUVER study will be presented at an upcoming medical conference.

“TGCT tends to be a disease of the young. This rare, benign tumor that grows in and around the joints primarily affects young and middle-aged adults in their working years. The swelling, pain, stiffness and limited mobility caused by the disease can have a significant impact on the ability to perform daily activities, limiting patients’ work and social lives. Treatment often involves surgery, yet the high recurrence rate and potential complications from repeated surgical interventions can be very challenging for patients to deal with, creating an urgent need for systemic therapy that could control tumor growth,” said Professor Niu Xiaohui, Director of the Bone and Soft Tissue Tumour Diagnosis and Research Centre at Beijing Jishuitan Hospital. “Based on these new data from the MANEUVER study, together with once-daily oral administration that may promote long-term adherence and pimicotinib’s selective inhibition of CSF-1R, this investigational medicine has the potential to establish a new treatment paradigm for patients with TGCT.”

In MANEUVER, pimicotinib was well-tolerated, and the safety profile was consistent with previously reported data, with no evidence of cholestatic hepatotoxicity. Treatment-emergent adverse events (TEAEs) leading to treatment discontinuation occurred in 1.6% (n=1) of patients treated with pimicotinib; TEAEs leading to dose reduction occurred in 7.9% (n=5) of pimicotinib-treated patients.

“There is a tremendous unmet need for effective, well-tolerated systemic treatment for TGCT, a disease primarily caused by CSF-1 overexpression that leads to joint tissue thickening and overgrowth, severely impacting patients’ lives. These Phase III data from MANEUVER confirm results of Abbisko’s Phase I study, indicating that targeting CSF-1R with pimicotinib has the potential to offer a new treatment option for patients. As we work with Abbisko to review the data from this study and prepare to share it with regulators in China, we are focused on our shared goal of bringing pimicotinib to patients in need,” said Danny Bar-Zohar, Global Head of Research & Development and Chief Medical Officer for the Healthcare business sector of Merck.

“As the first global trial to enroll both Asian and Western patients with TGCT in balanced proportions across multiple regions, MANEUVER is a landmark global study that allows for detailed outcome comparisons. This can facilitate a deeper understanding of disease characteristics and potential similar response across different populations,” said Yaochang Xu, Chairman and CEO of Abbisko Therapeutics. “This unique study shows that pimicotinib has the potential to provide a novel oral small molecule therapy option for TGCT patients, representing a key advancement within the emerging class of CSF-1R inhibitors. We look forward to collaborating with Merck as we pursue registration of pimicotinib as the first therapy option indicated for the systemic treatment of TGCT in China.”

Pimicotinib is an investigational orally administered, highly selective and potent small-molecule inhibitor of colony stimulating factor-1 receptor (CSF-1R) being developed by Abbisko. In December 2023, Abbisko Therapeutics and Merck entered into an agreement granting an exclusive license to commercialize products comprising or containing pimicotinib for all indications in Chinese mainland, Hong Kong, Macau, and Taiwan, with an option for the rest of world including the US.

About MANEUVER

The pivotal Phase III MANEUVER study is a three-part, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of pimicotinib in patients with TGCT who are eligible for systemic therapy and have not received prior anti-CSF-1/CSF-1R therapy. The study is being conducted in China (n=45), Europe (n=28), and the US and Canada (n=21).

In the double-blind Part 1, 94 patients were randomized 2:1 to receive either 50 mg QD of pimicotinib (n=63) or placebo (n=31) for 24 weeks. The primary endpoint is objective response rate (ORR) at week 25 as measured by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 by blinded independent central review in the intent-to-treat (ITT) population. Secondary endpoints include tumor volume score, active range of motion, stiffness by Numeric Rating Scale (NRS), pain by Brief Pain Inventory (BPI), and physical function measured by PROMIS.

After the double-blind Part 1, eligible patients may continue to the open-label Part 2 for up to 24 weeks of dosing. Patients who complete Part 2 may then enter the open-label extension phase (Part 3) for extended treatment and safety follow-up.

About Tenosynovial Giant Cell Tumor

Tenosynovial giant cell tumor (TGCT) is a rare, benign and locally aggressive disease that originates in the synovial lining of joints, bursae, and tendon sheaths, leading to thickening and overgrowth of these tissues. TGCT is life-limiting, causing joint pain, stiffness, swelling, and reduced range of motion, significantly impacting the daily activities and quality of life in the primarily working-age population that it affects. If left untreated or in recurrent cases, TGCT can result in irreversible damage to the bone, joint and surrounding tissues.

Currently, surgery is the primary treatment option for TGCT. However, surgical resection is often limited by risk of post-surgical recurrence and associated morbidity. This highlights the unmet need for well-tolerated and effective systemic treatment in TGCT.

About Pimicotinib (ABSK021)

Pimicotinib (ABSK021), which is being developed by Abbisko Therapeutics, is a novel, orally administered, highly selective and potent small-molecule inhibitor of CSF-1R. Pimicotinib has been granted breakthrough therapy designation (BTD) by China National Medical Products Administration (NMPA) and the US Food and Drug Administration (FDA) and priority medicine (PRIME) designation from the European Medicines Agency (EMA) for the treatment of patients with TGCT that are not amenable to surgery.

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About Merck

Merck, a leading science and technology company, operates across life science, healthcare and electronics. Around 63,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From providing products and services that accelerate drug development and manufacturing as well as discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2023, Merck generated sales of € 21 billion in 65 countries.

Scientific exploration and responsible entrepreneurship have been key to Merck’s technological and scientific advances. This is how Merck has thrived since its founding in 1668. The founding family remains the majority owner of the publicly listed company. Merck holds the global rights to the Merck name and brand. The only exceptions are the United States and Canada, where the business sectors of Merck operate as MilliporeSigma in life science, EMD Serono in healthcare, and EMD Electronics in electronics.

Contacts

Media Relations

gangolf.schrimpf@merckgroup.com
Phone: +49 151 1454 9591

Investor Relations

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Phone: +49 6151 72-3321

CTP Builds 30,000 sqm of Distribution Space for Redcare Pharmacy, Europe’s Leading Online Pharmacy




CTP Builds 30,000 sqm of Distribution Space for Redcare Pharmacy, Europe’s Leading Online Pharmacy

AMSTERDAM–(BUSINESS WIRE)–Regulatory News:

CTP, Europe’s largest listed developer, owner, and manager of industrial and logistics properties by gross lettable area (GLA), today began the construction of a new distribution centre of more than 30,000 sqm at CTPark Blatnice in the Czech Republic for Redcare Pharmacy. The building is scheduled for completion in July 2025.

Redcare Pharmacy N.V. is the leading e-pharmacy in Europe, currently active in seven countries: Germany, Austria, France, Belgium, Italy, the Netherlands and Switzerland. Today, Redcare Pharmacy offers almost 12 million active customers a wide range of more than 150,000 products at attractive and fair prices. This logistics centre is being developed to serve the Austrian market of Redcare Pharmacy and deliver orders faster in the future to their customers in Austria.

“We are thrilled to be able to develop such an important project together with Redcare Pharmacy. CTPark Blatnice will provide an optimal environment for development thanks to its modern facilities and strategic location, as well as our speed and expertise in delivering large and technically challenging projects. The emphasis on sustainability and innovative technologies such as heat pumps or photovoltaic panels show CTP’s commitment to ESG as a long-term owner of industrial and logistics real estate, said Jakub Kodr, Head of Business Development CTP Czech Republic.

“Finding a partner who understands the specific needs of the pharmaceutical industry was crucial for us. CTP not only has experience in this sector, but offered us premises with a high standard of sustainability and technologies that ensure safe and efficient storage of pharmaceuticals. We look forward to working together and successfully delivering this project,” comments Theresa Holler, COO and responsible pharmacist of Redcare Pharmacy.

The project is characterized by its emphasis on sustainability and modern technologies, which are required for the storage standards of pharmaceutical products. The warehouse will be equipped with indoor climate control and innovative technologies such as ground-to-air heat pumps, thermal sensors or special protective films on the skylights to ensure optimal indoor temperature control and protect the space from overheating. Such a concept guarantees that the warehouse and pharmacy operations will meet the strict standards and regulations of the State Institute for Drug Control (SÚKL).

CTP is focusing on the use of renewable energy sources in the project, including the planned installation of photovoltaic panels on the roof of the building. In addition, the project includes electric vehicle charging stations, advanced technologies for rainwater retention and reuse, and biodiversity measures such as pools, lizard houses and habitats to protect local fauna.

CTPark Blatnice is a modern logistics park located directly on the D5 motorway, 17 km from Pilsen and only 7 km from the container terminal in Nýřany. The park offers excellent conditions for domestic and international logistics operations. It currently occupies an area of 58,600 sqm with a planned expansion of another 20,748 sqm. Thanks to its proximity to technical universities and good connections to transport infrastructure, the park is also well placed to attract a skilled workforce.

About CTP

CTP is Europe’s largest listed owner, developer, and manager of logistics and industrial real estate by gross lettable area, owning 12.6 million sqm of GLA across 10 countries as of 30 September 2024. CTP certifies all new buildings to BREEAM Very good or better and earned a negligible-risk ESG rating by Sustainalytics, underlining its commitment to being a sustainable business. For more information, visit CTP’s corporate website: www.ctp.eu

Contacts

FOR MEDIA ENQUIRIES:
Patryk Statkiewicz, Group Head of Marketing & PR

Mobile: +31 (0) 629 596 119

Email: patryk.statkiewicz@ctp.eu

SEC Newgate

James Carnegie

Mobile: +44 (0)7827 486 224

Email: CTP@SECNewgate.co.uk

FOR ANALYST AND INVESTOR ENQUIRIES:
Maarten Otte, Head of Investor Relations

Mobile: +420 730 197 500

Email: maarten.otte@ctp.eu

Bristol Myers Squibb Showcases the Continued Strength of its Cardiovascular Portfolio with New Clinical and Real-World Data at American Heart Association Scientific Sessions 2024




Bristol Myers Squibb Showcases the Continued Strength of its Cardiovascular Portfolio with New Clinical and Real-World Data at American Heart Association Scientific Sessions 2024

Real-world and long-term extension data, including updated results from post-launch evaluation of REMS Program, bolster growing body of evidence supporting the efficacy and safety profile of CAMZYOS^® (mavacamten)

PRINCETON, N.J.–(BUSINESS WIRE)–$BMY #AHA—Bristol Myers Squibb (NYSE: BMY) today announced the presentation of data across its cardiovascular portfolio at the American Heart Association (AHA) Annual Scientific Sessions, taking place November 16-18, 2024, in Chicago, Illinois. New analyses include updated results from the nearly two-year post-launch evaluation of the CAMZYOS^® (mavacamten) Risk Evaluation and Mitigation Strategy (REMS) Program and real-world and long-term extension data reinforcing the efficacy and safety profile of CAMZYOS, as well as data on behalf of the BMS-Pfizer Alliance on ELIQUIS^® (apixaban) and the BMS-Johnson & Johnson Collaboration on milvexian.

“We look forward to AHA where we will share data that build on our 70-year legacy of pioneering cardiovascular research and delivering paradigm-changing medicines to address the growing burden of cardiovascular disease,” said Roland Chen, MD, senior vice president and head of Immunology, Cardiovascular & Neuroscience (ICN) Development at Bristol Myers Squibb. “Data to be presented at the meeting highlight our commitment to improving clinical outcomes for patients around the world by delivering transformational therapies, like CAMZYOS, the first and only approved cardiac myosin inhibitor.”

With inclusion in both the ESC and AHA/ACC clinical guidelines as a recommended option for when symptoms persist after first-line therapy, CAMZYOS is a standard of care for NYHA class II-III symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

Research to be presented at the meeting supports the growing body of evidence of CAMZYOS, including compliance with the REMS Program. These data include:

• An updated analysis of results from the post-launch evaluation of the CAMZYOS REMS Program spanning nearly 2-years (22-months).
• A featured science presentation of the 128-week analysis (nearly 2.5 years) of the VALOR-HCM long-term study analyzing the efficacy and safety profile of CAMZYOS in reducing patient eligibility and/or decision to proceed with septal reduction therapy (SRT) in patients with symptomatic oHCM.
• New real-world evidence on CAMZYOS’ long-term effectiveness and safety profile, including data from MARVEL-HCM—the largest observational study on CAMZYOS’ effectiveness—and from COLLIGO-HCM, a real-world study examining racially diverse patients and those with higher disease burden than typically seen in clinical trials.

Abstracts sponsored by Bristol Myers Squibb, the BMS-Pfizer Alliance and the BMS-Johnson & Johnson Collaboration to be presented at AHA Scientific Sessions 2024 can be found below. Complete abstracts may be accessed online here. Visit this page on BMS.com for more information on Bristol Myers Squibb’s scientific approach and resources on cardiovascular diseases.

About CAMZYOS^® (mavacamten)

CAMZYOS^® (mavacamten) is the first and only cardiac myosin inhibitor approved in the U.S., indicated for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms, and in the European Union, indicated for the treatment of symptomatic (NYHA, class II-III) obstructive HCM in adult patients. It has also received regulatory approvals in countries and regions across five continents. CAMZYOS is an allosteric and reversible inhibitor selective for cardiac myosin. CAMZYOS modulates the number of myosin heads that can enter “on actin” (power-generating) states, thus reducing the probability of force-producing (systolic) and residual (diastolic) cross-bridge formation. Excess myosin actin cross-bridge formation and dysregulation of the super-relaxed state are mechanistic hallmarks of HCM. CAMZYOS shifts the overall myosin population towards an energy-sparing, recruitable, super-relaxed state. In HCM patients, myosin inhibition with CAMZYOS reduces dynamic left ventricular outflow tract (LVOT) obstruction and improves cardiac filling pressures.

IMPORTANT SAFETY INFORMATION

WARNING: RISK OF HEART FAILURE

CAMZYOS reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction.

Echocardiogram assessments of LVEF are required prior to and during treatment with CAMZYOS. Initiation of CAMZYOS in patients with LVEF <55% is not recommended. Interrupt CAMZYOS if LVEF is <50% at any visit or if the patient experiences heart failure symptoms or worsening clinical status.

Concomitant use of CAMZYOS with certain cytochrome P450 inhibitors or discontinuation of certain cytochrome P450 inducers may increase the risk of heart failure due to systolic dysfunction; therefore, the use of CAMZYOS is contraindicated with the following:

• Moderate to strong CYP2C19 inhibitors or strong CYP3A4 inhibitors
• Moderate to strong CYP2C19 inducers or moderate to strong CYP3A4 inducers

Because of the risk of heart failure due to systolic dysfunction, CAMZYOS is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the CAMZYOS REMS PROGRAM.

CONTRAINDICATIONS

CAMZYOS is contraindicated with concomitant use of:

• Moderate to strong CYP2C19 inhibitors or strong CYP3A4 inhibitors
• Moderate to strong CYP2C19 inducers or moderate to strong CYP3A4 inducers

WARNINGS AND PRECAUTIONS

Heart Failure

CAMZYOS reduces systolic contraction and can cause heart failure or totally block ventricular function. Patients who experience a serious intercurrent illness (e.g., serious infection) or arrhythmia (e.g., atrial fibrillation or other uncontrolled tachyarrhythmia) are at greater risk of developing systolic dysfunction and heart failure.

Assess the patient’s clinical status and LVEF prior to and regularly during treatment and adjust the CAMZYOS dose accordingly. New or worsening arrhythmia, dyspnea, chest pain, fatigue, palpitations, leg edema, or elevations in N-terminal pro-B-type natriuretic peptide (NT-proBNP) may be signs and symptoms of heart failure and should also prompt an evaluation of cardiac function.

Asymptomatic LVEF reduction, intercurrent illnesses, and arrhythmias require additional dosing considerations.

Initiation of CAMZYOS in patients with LVEF <55% is not recommended. Avoid concomitant use of CAMZYOS in patients on disopyramide, ranolazine, verapamil with a beta blocker, or diltiazem with a beta blocker as these medications and combinations increase the risk of left ventricular systolic dysfunction and heart failure symptoms and clinical experience is limited.

CYP 450 Drug Interactions Leading to Heart Failure or Loss of Effectiveness

CAMZYOS is primarily metabolized by CYP2C19 and CYP3A4 enzymes. Concomitant use of CAMZYOS and drugs that interact with these enzymes may lead to life-threatening drug interactions such as heart failure or loss of effectiveness.

Advise patients of the potential for drug interactions, including with over-the-counter medications (such as omeprazole, esomeprazole, or cimetidine). Advise patients to inform their healthcare provider of all concomitant products prior to and during CAMZYOS treatment.

CAMZYOS Risk Evaluation and Mitigation Strategy (REMS) Program

CAMZYOS is only available through a restricted program called the CAMZYOS REMS Program because of the risk of heart failure due to systolic dysfunction. Notable requirements of the CAMZYOS REMS Program include the following:

• Prescribers must be certified by enrolling in the REMS Program.
• Patients must enroll in the REMS Program and comply with ongoing monitoring requirements.
• Pharmacies must be certified by enrolling in the REMS Program and must only dispense to patients who are authorized to receive CAMZYOS.
• Wholesalers and distributors must only distribute to certified pharmacies.

Further information is available at www.CAMZYOSREMS.com or by telephone at 1-833-628-7367.

Embryo-Fetal Toxicity

CAMZYOS may cause fetal toxicity when administered to a pregnant female, based on animal studies. Confirm absence of pregnancy in females of reproductive potential prior to treatment and advise patients to use effective contraception during treatment with CAMZYOS and for 4 months after the last dose. CHCs containing a combination of ethinyl estradiol and norethindrone may be used with CAMZYOS. However, CAMZYOS may reduce the effectiveness of combined hormonal contraceptives (CHC). If these CHCs are used, advise patients to add nonhormonal contraception (such as condoms) during concomitant use and for 4 months after the last dose of CAMZYOS.

ADVERSE REACTIONS

In the EXPLORER-HCM trial, adverse reactions occurring in >5% of patients and more commonly in the CAMZYOS group than in the placebo group were dizziness (27% vs 18%) and syncope (6% vs 2%). There were no new adverse reactions identified in VALOR-HCM.

Effects on Systolic Function

In the EXPLORER-HCM trial, mean (SD) resting LVEF was 74% (6) at baseline in both treatment groups. Mean (SD) absolute change from baseline in LVEF was -4% (8) in the CAMZYOS group and 0% (7) in the placebo group over the 30-week treatment period. At Week 38, following an 8-week interruption of trial drug, mean LVEF was similar to baseline for both treatment groups. In the EXPLORER-HCM trial, 7 (6%) patients in the CAMZYOS group and 2 (2%) patients in the placebo group experienced reversible reductions in LVEF <50% (median 48%: range 35-49%) while on treatment. In all 7 patients treated with CAMZYOS, LVEF recovered following interruption of CAMZYOS.

DRUG INTERACTIONS

Potential for Other Drugs to Affect Plasma Concentrations of CAMZYOS

CAMZYOS is primarily metabolized by CYP2C19 and to a lesser extent by CYP3A4 and CYP2C9. Inducers and inhibitors of CYP2C19 and moderate to strong inhibitors or inducers of CYP3A4 may affect the exposures of CAMZYOS.

Impact of Other Drugs on CAMZYOS:

• Moderate to Strong CYP2C19 Inhibitors or Strong CYP3A4 Inhibitors: Concomitant use increases CAMZYOS exposure, which may increase the risk of heart failure due to systolic dysfunction. Concomitant use is contraindicated.
• Moderate to Strong CYP2C19 Inducers or Moderate to Strong CYP3A4 Inducers: Concomitant use decreases CAMZYOS exposure, which may reduce CAMZYOS’ efficacy. The risk of heart failure due to systolic dysfunction may increase with discontinuation of these inducers as the levels of induced enzyme normalizes. Concomitant use is contraindicated.
• Weak CYP2C19 Inhibitors or Moderate CYP3A4 Inhibitors: Concomitant use with a weak CYP2C19 inhibitor or a moderate CYP3A4 inhibitor increases CAMZYOS exposure, which may increase the risk of adverse drug reactions. Initiate CAMZYOS at the recommended starting dose of 5 mg orally once daily in patients who are on stable therapy with a weak CYP2C19 inhibitor or a moderate CYP3A4 inhibitor. Reduce dose of CAMZYOS by one level (i.e., 15 to 10 mg, 10 to 5 mg, or 5 to 2.5 mg) in patients who are on CAMZYOS treatment and intend to initiate a weak CYP2C19 inhibitor or a moderate CYP3A4 inhibitor. Schedule clinical and echocardiographic assessment 4 weeks after inhibitor initiation, and do not up-titrate CAMZYOS until 12 weeks after inhibitor initiation. Avoid initiation of concomitant weak CYP2C19 and moderate CYP3A4 inhibitors in patients who are on stable treatment with 2.5 mg of CAMZYOS because a lower dose is not available.

Potential for CAMZYOS to Affect Plasma Concentrations of Other Drugs

CAMZYOS is an inducer of CYP3A4, CYP2C9, and CYP2C19. Concomitant use with CYP3A4, CYP2C19, or CYP2C9 substrates may reduce plasma concentration of these drugs. Closely monitor when CAMZYOS is used in combination with CYP3A4, CYP2C19, or CYP2C9 substrates unless otherwise recommended in the Prescribing Information.

Certain Combined Hormonal Contraceptives (CHC): Progestin and ethinyl estradiol are CYP3A4 substrates. Concomitant use of CAMZYOS may decrease exposures of certain progestins, which may lead to contraceptive failure. CHCs containing a combination of ethinyl estradiol and norethindrone may be used with CAMZYOS, but if other CHCs are used, advise patients to add nonhormonal contraception (such as condoms) during concomitant use and for 4 months after the last dose of CAMZYOS.

Drugs That Reduce Cardiac Contractility

Expect additive negative inotropic effects of CAMZYOS and other drugs that reduce cardiac contractility. Avoid concomitant use of CAMZYOS in patients on disopyramide, ranolazine, verapamil with a beta blocker, or diltiazem with a beta blocker as these medications and combinations increase the risk of left ventricular systolic dysfunction and heart failure symptoms and clinical experience is limited.

If concomitant therapy with a negative inotrope is initiated, or if the dose of a negative inotrope is increased, monitor LVEF closely until stable doses and clinical response have been achieved.

SPECIFIC POPULATIONS

Pregnancy

CAMZYOS may cause fetal harm when administered to a pregnant female. Advise pregnant females about the potential risk to the fetus with maternal exposure to CAMZYOS during pregnancy. There is a pregnancy safety study for CAMZYOS. If CAMZYOS is administered during pregnancy, or if a patient becomes pregnant while receiving CAMZYOS or within 4 months after the last dose of CAMZYOS, healthcare providers should report CAMZYOS exposure by contacting Bristol Myers Squibb at 1-800-721-5072 or www.bms.com.

Lactation

The presence of CAMZYOS in human or animal milk, the drug’s effects on the breastfed infant, or the effects on milk production are unknown. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for CAMZYOS and any potential adverse effects on the breastfed child from CAMZYOS or from the underlying maternal condition.

Females and Males of Reproductive Potential

Confirm absence of pregnancy in females of reproductive potential prior to initiation of CAMZYOS. Advise females of reproductive potential to use effective contraception during treatment with CAMZYOS and for 4 months after the last dose. CHCs containing a combination of ethinyl estradiol and norethindrone may be used with CAMZYOS. However, CAMZYOS may reduce the effectiveness of certain other combined hormonal contraceptives (CHC). If these CHCs are used, advise patients to add nonhormonal contraception (such as condoms) during concomitant use and for 4 months after the last dose of CAMZYOS.

Please see U.S. Full Prescribing Information, including Boxed WARNING and Medication Guide.

About ELIQUIS ^® (apixaban)

ELIQUIS^® is an oral selective Factor Xa inhibitor. By inhibiting Factor Xa, a key blood clotting protein, ELIQUIS decreases thrombin generation and blood clot formation. ELIQUIS is approved for multiple indications in the U.S. based on efficacy and safety data from multiple Phase 3 clinical trials. ELIQUIS is a prescription medicine indicated to reduce the risk of stroke and systemic embolism in patients with non-valvular atrial fibrillation (NVAF); for the prophylaxis of deep vein thrombosis (DVT), which may lead to pulmonary embolism (PE), in patients who have undergone hip or knee replacement surgery; for the treatment of DVT and PE; and to reduce the risk of recurrent DVT and PE, following initial therapy. ELIQUIS continues to be developed and commercialized by The Bristol Myers Squibb-Pfizer Alliance.

ELIQUIS Important Safety Information

Indications

ELIQUIS is indicated to reduce the risk of stroke and systemic embolism in patients with nonvalvular atrial fibrillation. ELIQUIS is indicated for the prophylaxis of deep vein thrombosis (DVT), which may lead to pulmonary embolism (PE), in patients who have undergone hip or knee replacement surgery. ELIQUIS is indicated for the treatment of DVT and PE, and to reduce the risk of recurrent DVT and PE following initial therapy.

Important Safety Information

WARNING: (A) PREMATURE DISCONTINUATION OF ELIQUIS INCREASES THE RISK OF THROMBOTIC EVENTS, (B) SPINAL/EPIDURAL HEMATOMA

(A) Premature discontinuation of any oral anticoagulant, including ELIQUIS, increases the risk of thrombotic events. If anticoagulation with ELIQUIS is discontinued for a reason other than pathological bleeding or completion of a course of therapy, consider coverage with another anticoagulant.

(B) Epidural or spinal hematomas may occur in patients treated with ELIQUIS who are receiving neuraxial anesthesia or undergoing spinal puncture. These hematomas may result in long-term or permanent paralysis. Consider these risks when scheduling patients for spinal procedures. Factors that can increase the risk of developing epidural or spinal hematomas in these patients include:

• use of indwelling epidural catheters
• concomitant use of other drugs that affect hemostasis, such as nonsteroidal anti-inflammatory drugs (NSAIDs), platelet inhibitors, other anticoagulants
• a history of traumatic or repeated epidural or spinal punctures
• a history of spinal deformity or spinal surgery
• optimal timing between the administration of ELIQUIS and neuraxial procedures is not known

Monitor patients frequently for signs and symptoms of neurological impairment. If neurological compromise is noted, urgent treatment is necessary.

Consider the benefits and risks before neuraxial intervention in patients anticoagulated or to be anticoagulated.

CONTRAINDICATIONS

• Active pathological bleeding
• Severe hypersensitivity reaction to ELIQUIS (e.g., anaphylactic reactions)

WARNINGS AND PRECAUTIONS

• Increased Risk of Thrombotic Events after Premature Discontinuation: Premature discontinuation of any oral anticoagulant, including ELIQUIS, in the absence of adequate alternative anticoagulation increases the risk of thrombotic events. An increased rate of stroke was observed during the transition from ELIQUIS to warfarin in clinical trials in atrial fibrillation patients. If ELIQUIS is discontinued for a reason other than pathological bleeding or completion of a course of therapy, consider coverage with another anticoagulant.
• Bleeding Risk: ELIQUIS increases the risk of bleeding and can cause serious, potentially fatal, bleeding.
• Concomitant use of drugs affecting hemostasis increases the risk of bleeding, including aspirin and other antiplatelet agents, other anticoagulants, heparin, thrombolytic agents, SSRIs, SNRIs, and NSAIDs.
• Advise patients of signs and symptoms of blood loss and to report them immediately or go to an emergency room. Discontinue ELIQUIS in patients with active pathological hemorrhage.
• The anticoagulant effect of apixaban can be expected to persist for at least 24 hours after the last dose (i.e., about two half-lives). An agent to reverse the anti-factor Xa activity of apixaban is available. Please visit www.andexxa.com for more information on availability of a specific reversal agent.
• Spinal/Epidural Anesthesia or Puncture: Patients treated with ELIQUIS undergoing spinal/epidural anesthesia or puncture may develop an epidural or spinal hematoma which can result in long-term or permanent paralysis.

  The risk of these events may be increased by the postoperative use of indwelling epidural catheters or the concomitant use of medicinal products affecting hemostasis. Indwelling epidural or intrathecal catheters should not be removed earlier than 24 hours after the last administration of ELIQUIS. The next dose of ELIQUIS should not be administered earlier than 5 hours after the removal of the catheter.

Contacts

Bristol Myers Squibb

Media Inquiries:
media@bms.com

Investors:
investor.relations@bms.com

Read full story here

Novotech Recognized for Excellence in Business Expansion, Innovation, and Marketing at the 2024 Clinical Trials Arena Excellence Awards




Novotech Recognized for Excellence in Business Expansion, Innovation, and Marketing at the 2024 Clinical Trials Arena Excellence Awards

SYDNEY–(BUSINESS WIRE)–#ClinicalTrials–Novotech, the global full-service clinical Contract Research Organization (CRO), has received multiple awards in the 2024 Clinical Trials Arena Excellence Awards, recognizing the achievements of our people in Business Expansion, Innovation, and Marketing. These awards highlight our commitment to advancing the clinical trial landscape and delivering specialized solutions across the biotech sector.

“We’re grateful for this recognition, which is a direct result of our team’s effort and commitment to our mission,” states Dr. John Moller, CEO, Novotech. “It’s a reminder of what we can accomplish when we work together, and we look forward to continuing this momentum.”

The Clinical Trials Arena Excellence Awards celebrate the pharmaceutical industry’s top achievements, identifying organizations that drive progress and innovation. Novotech’s accolades reflect our proactive strategies, technological advancements, and strong international presence.

Highlights of Novotech’s Award-Winning Strategies:

• Business Expansion – Novotech has significantly extended our reach through strategic acquisitions, including NCGS, EastHORN, and CBR International. These acquisitions have strengthened our operational capabilities and broadened service offerings, ensuring localized expertise in key biotech hubs. The launch of the Early Phase Strategic Delivery Unit (EP SDU) enhances trial support for biotech companies by leveraging Australia and New Zealand’s clinical excellence and regulatory efficiencies.

• Innovation in Data-Driven Trials – Novotech’s innovation award reflects its adoption of advanced technologies, including AI and big data analytics, which streamline trial design and execution. Partnerships with Medidata and ObvioHealth have strengthened our capacity for virtual trials and predictive analytics, leading to better site selection, faster patient recruitment, and improved trial outcomes.

• Strategic Marketing Leadership – Under the direction of Toyna Chin, Global Director of Marketing, Novotech’s rebranding has elevated the company’s brand position as a global CRO leader. Strategic, multi-channel campaigns have increased brand recognition and positioned Novotech as a trusted partner, particularly within the US and European markets.

“I am deeply honored to accept this recognition, but it truly reflects the remarkable dedication and talent of our entire marketing organization. Their relentless effort and collaboration are what make our success possible. This award is a tribute to the passion and unity that drives us forward, proving that true marketing excellence is always the result of a collective vision and shared purpose.” – Toyna Chin, Global Director of Marketing, Novotech

About Novotech Novotech-CRO.com

Founded in 1997, Novotech is a global full-service clinical Contract Research Organization (CRO) focused on partnering with biotech companies to accelerate the development of advanced and novel therapeutics at every phase.

Recognized for its industry-leading contributions, Novotech has received numerous prestigious awards, including the Frost & Sullivan 2024 Global Biotech CRO of the year award, Clinical Trials Arena Excellence Awards 2024, 2024 Employer of Choice, 2024 Great Place to Work in the US, 2024 Brandon Hall Gold Award, CRO Leadership Award 2023, the Asia Pacific Cell & Gene Therapy Clinical Trials Excellence 2023, the Asia-Pacific Contract Research Organization Company of the Year Award since 2006.

The Company offers a comprehensive suite of services including laboratories, Phase I facilities, drug development consulting, regulatory expertise, and has experience with over 5,000 clinical projects, including Phase I to Phase IV clinical trials and bioequivalence studies. With a presence in 34 office locations and a dedicated team of 3,000+ professionals worldwide, Novotech is a trusted end-to-end strategic partner of choice.

For more information or to speak to an expert team member visit www.Novotech-CRO.com

Contacts

Media Contact
Toyna Chin
mediacontact@novotech-cro.com
USA: +1 415 364 8135

Almirall’s Nine-Month 2024 Results: Almirall Reports Strong Performance at Q3 2024 – Driven by Successful Commercial Execution of the Dermatology Portfolio, Fuelled by Biologics Growth




Almirall’s Nine-Month 2024 Results: Almirall Reports Strong Performance at Q3 2024 – Driven by Successful Commercial Execution of the Dermatology Portfolio, Fuelled by Biologics Growth

• Positive growth trajectory maintained with Net Sales increase of 7.9% to a total of €727.6 MM, EBITDA of €142.2 MM (+2.9% YoY) driven by increased overall sales and strong performance of the biologics portfolio. Full year guidance for 2024 reiterated (Net Sales and EBITDA).
• Biologics performance continued to be the key growth engine for Almirall, enabled by strong commercial execution across EU markets: the successful Ebglyss^® launch generated total sales of €20.4 MM in the first nine months in Europe. Geographic expansion is on track with recent launches in Norway, UK, Spain, Denmark, and the Czech Republic. Consistent growth of Ilumetri® with 24.8% increase in sales vs 9M 2023 (total of €152.5 MM year to date).
• Strong performance of Almirall´s broad dermatology product portfolio further drives the company´s relevance to patients and dermatologists. Wynzora® growing 62.7% YoY to a total of €19.2 MM, and Klisyri® growing 14.0% YoY to a total of €16.4 MM. The total medical dermatology portfolio grew 17.3% to a total of €404.9 MM.
• Continued R&D pipeline progress with the recent launch of Klisyri large field in the USA, completion of the decentralised regulatory procedure for efinaconazole in Europe, and start of the Phase I of the readthrough inducer for rare dermatology indications.

BARCELONA, Spain–(BUSINESS WIRE)–Almirall, S.A. (ALM), a global biopharmaceutical company based in Barcelona, today announced its year to date financial results at the third quarter of 2024. Almirall continued to deliver strong sales growth in the first nine months of 2024 which were driven by the dermatology business, and especially the biologics portfolio in Europe. Net Sales increased by 7.9% YoY to a total of €727.6 MM, EBITDA was €142.2 MM (increase of 2.9% YoY) driven by higher sales growth, with a gross margin of 64,8%. Dermatology sales in Europe increased by 21.9% to a total of €355.2 MM. Almirall re-iterates its guidance for 2024 delivering high single digit net sales growth and an EBITDA between €175 MM and €190 MM.

The successful launch of Ebglyss® in the first markets in Europe generated €20.4 MM in the initial nine months of 2024, representing a 34% sales increase quarter-on-quarter. The potential of lebrikizumab as a first line treatment for moderate to severe atopic dermatitis is demonstrated by the steadily increasing prescriptions in Germany, high product awareness, and strong feedback received from dermatologists and their patients – leading to significant gain of share in the dynamic market segment. The geographic expansion of lebrikizumab after the initial launch in Germany is progressing well with recent launches in Norway, UK, Spain, Denmark, and the Czech Republic. Ebglyss® is on track to deliver against expectations for 2024 and anticipated peak sales supported by continued significant investment behind the launch by Almirall.

Ilumetri®, for the treatment of psoriasis, continued to deliver very strong results gaining market share in key countries across Europe. Total sales of tildrakizumab in the first 9 months of 2024 were €152.5 MM representing a 24.8% growth year-on-year. With its steadily growing market share, Ilumetri® has cemented its strong position within the leading class of the Anti-IL 23 psoriasis treatments and is on track to achieve €250 MM peak sales.

Almirall´s broad dermatology product portfolio continues to show strong performance growing 17.3% to a total of €404.9 MM in the first nine months of 2024 further driving the company´s relevance to patients and dermatologists. Wynzora® grew 62.7% YoY to a total of €19.2 MM, and Klisyri® grew 14.0% YoY to a total of €16.4 MM.

“Our continued strong performance at Q3 2024 is fuelled by the growth of our biologics: the sustained performance of Ilumetri®, and the positive trajectory of Ebglyss®. In combination with the success story of our broad dermatology portfolio in Europe, this growth is proof of our strong commercial capabilities and builds the foundation of our leadership in medical dermatology in line with our company ambition. In the 80^th anniversary of Almirall, we are pleased to confirm our guidance for 2024 based on this strong business performance while we continue to invest significantly in supporting our launches and R&D to advance science and to innovate for future growth.” Carlos Gallardo, Almirall Chairman and CEO

R&D pipeline

Almirall’s continued investment in its leading R&D capabilities, and the medical dermatology pipeline are closely aligned with the company’s long-term view on its contributions and commitment to positively impacting patients and society. In the YTD2024 Almirall invested 12.4% of Net Sales in R&D (representing an increase of 14.9% year on year) and major achievements in Q3 2024 – in addition to progress of the early-stage pipeline – included the completion of the decentralised regulatory procedure of our efinaconazole asset for the treatment of onychomycosis in Europe. The launch of Klisyri® large field in the USA following the approval by the FDA in June increases the potential of this product given the dermatologists’ treatment needs and the growing prevalence of Actinic Keratosis in an ageing population. Clinical testing to expand the Klisyri® indication to include large field in Europe are under way. Phase I clinical testing of the readthrough inducer for rare dermatology indications was initiated (aimed at developing an oral therapy to overcome nonsense mutations that cause a premature stop codon resulting in nonfunctional protein production). Lifecycle management activities will further strengthen Almirall´s key priority products with several ongoing clinical studies supporting Ebglyss® and Ilumetri®.

Financial highlights (€ rounded million)

2024 Full Year Guidance

Full year guidance reiterated: Net Sales growth of high-single digit and EBITDA between €175 MM – €190 MM.

Partnership with the dermatology community

Almirall´s close collaborations with dermatologists and life-science experts continue to be a key cornerstone for the company´s dedication to and success in medical dermatology. These partnerships expand Almirall´s focus on fostering scientific exchange and to advance the understanding of skin diseases, treatment options, and their impact on patients. In October, Almirall hosted the 3^rd ImmunoSkin Conference in Barcelona, which brought together leading experts and dermatologists to advance the understanding of immune-mediated skin diseases and their treatment. Almirall had a significant presence at the 33^rd congress of the European Academy of Dermatology and Venerology (EADV) in September. Amongst the 34 abstracts presented were new data showing that Ebglyss® provided sustained disease control for up to three years in more than 80% of adults and adolescents with moderate-to-severe atopic dermatitis. New data on Ilumetri® from the POSITIVE study showed the significant impact of the treatment for moderate to severe psoriasis on patients’ wellbeing.

At the Fall Clinical conference in October, Almirall´s partner Lilly presented important data showing the strong performance of Ebglyss® in patients with skin of colour, and in patients who had previously discontinued treatment with dupilumab.

About Almirall

Almirall is a global pharmaceutical company dedicated to medical dermatology. We closely collaborate with leading scientists, healthcare professionals, and patients to deliver our purpose: to transform the patients’ world by helping them realize their hopes and dreams for a healthy life. We are at the forefront of science to deliver ground-breaking, differentiated medical dermatology innovations that address patients´ needs.

Almirall, founded in 1944 and headquartered in Barcelona, is publicly traded on the Spanish Stock Exchange (ticker: ALM). Almirall (total revenue in 2023: €898.8 MM, 1900 employees globally) has direct presence in 21 countries and marketed products in over 100.

For more information, please visit almirall.com.

Legal Notice

This document includes only summary information and does not intend to be comprehensive. Facts, figures and opinions contained herein, other than historical, are “forward-looking statements”. These statements are based on currently available information and on best estimates and assumptions believed to be reasonable by the Company. These statements involve risks and uncertainties beyond the Company’s control. Therefore, actual results may differ materially from those stated by such forward-looking statements. The Company expressly disclaims any obligation to review or update any forward-looking statements, targets or estimates contained in this document to reflect any change in the assumptions, events or circumstances on which such forward-looking statements are based unless so required by applicable law.

Contacts

Media:
Corporate Communications

Carla Arrieta

corporate.communication@almirall.com
Tel.: (+34) 673 05 37 67

Investors & Corporate Communications:
Almirall

Pablo Divasson del Fraile

investors@almirall.com
Tel.: (+34) 93 291 30 87

Oncoinvent Announces Promising Interim Results for Radspherin® as a Potential Treatment for Peritoneal Carcinomatoses




Oncoinvent Announces Promising Interim Results for Radspherin® as a Potential Treatment for Peritoneal Carcinomatoses

OSLO, Norway–(BUSINESS WIRE)–Oncoinvent ASA, a clinical stage company advancing alpha emitter therapy across a variety of solid cancers, today announced an interim data readout of the Phase 1/2a studies of Radspherin® for the treatment of peritoneal carcinomatoses. The studies were closed for recruitment at the end of 2023, and patients are currently in long-term follow-up. The readout confirmed the previously published results on efficacy and adds further confidence in the ongoing randomized controlled Phase 2 clinical trial for Radspherin® in patients with ovarian cancer.

In the first interim readout from the Phase 1 study in ovarian cancer, only 1 patient out of the 10 receiving the selected dose (10%) had peritoneal recurrence at the 12-month interim readout, compared to an expected recurrence rate of 25% in similar populations [1-3]. Correspondingly, in the second intermediate readout of the colorectal cancer study, only 3 of 20 patients (15%) receiving the selected dose of 7 MBq had peritoneal recurrence after the full 18 months follow-up period. With current standard therapy, the expected peritoneal recurrence rate is approximately 50% after 18 months [4]. 36 patients with colorectal cancer have received the selected dose, with results from the final 16 patients still pending full follow-up.

“We are excited to announce the interim results of our Phase1/2a studies of Radspherin®, confirming a positive efficacy signal and demonstrating Radspherin’s® ability to enable local control in the peritoneum, thus ensuring patients remain in remission after surgery,” said Oystein Soug, Chief Executive Officer of Oncoinvent. “These positive results increase confidence in the further development of Radspherin® as an effective treatment option for patients suffering from peritoneal carcinomatoses, a condition that is notoriously difficult to treat effectively with systemic therapy.”

“Patients with ovarian cancer often have peritoneal carcinomatoses already at diagnosis, giving them higher recurrence rate and worse prognosis – so an effective treatment targeted specifically at these carcinomatoses could reduce recurrence and prolong survival,” says Yun Wang, MD PhD, Department of Gynecologic Oncology, The Norwegian Radium Hospital, Oslo University Hospital, principal investigator in the RAD-18-001 study.

About Radspherin

Radspherin® is an investigational radiopharmaceutical designed for the local treatment of cancer that has spread to body cavities. It consists of billions of calcium carbonate microparticles containing the radioactive material radium-224. The mode of action is the decay of radium-224 emitting alpha-particles, a highly potent form of ionizing radiation. Radspherin® is investigated in ongoing clinical studies to treat peritoneal carcinomatoses from ovarian and colorectal cancer and it is administered intraperitoneally after surgical resection with removal of all macroscopic tumors.

About the RAD-18-001 and RAD-18-002 study

Two early phase studies with Radspherin® in patients with peritoneal carcinomatoses have completed recruitment at the end of 2023 with 68 patients treated. One study in patients with peritoneal carcinomatoses from platinum sensitive recurrent epithelial ovarian, fallopian tube or primary peritoneal carcinoma (RAD-18-001, n=21, phase 1) scheduled for secondary cytoreduction, and one in patients with peritoneal carcinomatoses from colorectal cancer (RAD-18-002, n=47, phase 1/2a) scheduled for cytoreduction and HIPEC. The two studies were designed to evaluate the dose, safety and tolerability, dosimetry, and signal of efficacy of Radspherin®.

About Oncoinvent

Oncoinvent ASA is a clinical stage, radiopharmaceutical company developing innovative treatments for solid cancers. The technology platform is focused on the use of alpha-emitting radionuclides to deliver powerful radiation directly to cancer cells. The Company’s lead product candidate, Radspherin®, is being advanced through clinical development by a team with experience from all stages of radiopharmaceutical development. Internal manufacturing and supply chain capabilities have been established, which now have the capacity to supply Radspherin® for multi-center phase 2 clinical studies.

Forward-Looking Statements

All statements other than statements of historical facts contained in this press release are forward-looking statements and are not a representation that Oncoinvent’s plans, estimates, or expectations will be achieved. These forward-looking statements represent Oncoinvent’s expectations as of the date of this press release, and Oncoinvent disclaims any obligation to update the forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual results to differ materially, including with respect to whether the results of clinical or other studies will support the use of our product offerings, the impact of results of such studies, our expectations of the reliability, accuracy and performance of our tests, or of the benefits of our tests and product offerings to patients, providers and payers.

References

[1] Coleman, R. L., Spirtos, N. M., Enserro, D., Herzog, T. J., Sabbatini, P., Armstrong, D. K., … & Mannel, R. S. (2019). Secondary surgical cytoreduction for recurrent ovarian cancer. New England Journal of Medicine, 381(20), 1929-1939.

[2] Harter, P., Sehouli, J., Vergote, I., Ferron, G., Reuss, A., Meier, W., … & Du Bois, A. (2021). Randomized trial of cytoreductive surgery for relapsed ovarian cancer. New England Journal of Medicine, 385(23), 2123-2131.

[3] Shi, T., Zhu, J., Feng, Y., Tu, D., Zhang, Y., Zhang, P., … & Zang, R. (2021). Secondary cytoreduction followed by chemotherapy versus chemotherapy alone in platinum-sensitive relapsed ovarian cancer (SOC-1): a multicentre, open-label, randomised, phase 3 trial. The Lancet Oncology, 22(4), 439-449.

[4] Quénet, F., Elias, D., Roca, L., Goéré, D., Ghouti, L., Pocard, M., … & Patrick, R. A. T. (2021). Cytoreductive surgery plus hyperthermic intraperitoneal chemotherapy versus cytoreductive surgery alone for colorectal peritoneal metastases (PRODIGE 7): a multicentre, randomised, open-label, phase 3 trial. The Lancet Oncology, 22(2), 256-266.

Contacts

Oystein Soug, Chief Executive Officer

Email: soug@oncoinvent.com

Tore Kvam, Chief Financial Officer

Email: kvam@oncoinvent.com

IR enquiries:

Courtney Mogerley, Precision AQ

Email: Courtney.Mogerley@precisionaq.com

A2 Bio Highlights Progress of Tmod™ CAR T-Cell Clinical Programs During 2024 Annual Meeting of the Society for Immunotherapy of Cancer




A2 Bio Highlights Progress of Tmod™ CAR T-Cell Clinical Programs During 2024 Annual Meeting of the Society for Immunotherapy of Cancer

Oral presentation provides update on enhancements to improve participant diversity and enrollment in BASECAMP-1 prescreening study

BASECAMP-1 abstract named a ‘Top 100’ abstract by SITC

A2 Bio shares enrollment updates on EVEREST-1 and EVEREST-2 clinical studies

HOUSTON–(BUSINESS WIRE)–#ColorectalCancer–A2 Biotherapeutics, Inc. (A2 Bio), a clinical-stage cell therapy company developing first-in-class logic-gated cell therapies to selectively target tumor cells and protect normal cells, today shared progress of its Tmod™ CAR T-cell clinical programs during the 39^th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) taking place November 8-10, 2024. In an oral presentation today, the company shared continued progress in increasing the diversity of participants enrolled in the BASECAMP-1 nationwide prescreening study for all Tmod™ CAR T-cell trials. A2 Bio also shared posters highlighting early safety and biomarker data from the ongoing EVEREST-1 clinical study, an enrollment update for the EVEREST-2 clinical study, and updates on adaptations to boost potency and preserve selectivity of its Tmod™-based precision cell therapies.

The BASECAMP-1 abstract (number 589: “BASECAMP-1 is an efficient pre-screening study that identifies patients with HLA LOH and provides mutational, RNA-Seq, and microbiome data for precision logic-gated CAR T therapeutic trials”) was recognized by SITC as a top 100 abstract during the 2024 annual meeting.

“A2 Bio is very pleased to present progress of our clinical trials at SITC 2024 as we advance our innovative Tmod™ logic-gated CAR T-cell therapies to help patients with today’s most challenging cancers. We are pioneering a new participant-recruitment model to enhance safety, efficiency and diversity in the interventional trials of our Tmod™ precision cell therapies,” said William Go, M.D., Ph.D., chief medical officer of A2 Bio.

The Tmod™ platform solves the problem of on-target, off-tumor toxicity with a dual-receptor system that targets two (or more) antigens to confer selective tumor killing. Normal cells are protected from cytotoxicity by the Tmod™ blocker that recognizes antigens expressed only on normal tissues.¹ A2 Bio is sponsoring two investigational Tmod™ therapies in separate trials: EVEREST-1 that utilizes A2B530 to target carcinoembryonic antigen (CEA); and EVEREST-2 that utilizes A2B694 to target mesothelin (MSLN). Patients are enrolled in EVEREST-1 and EVEREST-2 through the BASECAMP-1 prescreening study. BASECAMP-1 utilizes artificial intelligence (AI)-enabled precision diagnostics in partnership with Tempus AI, as a cost-effective, high-yield approach to identify eligible patients for all A2 Bio clinical studies.²

BASECAMP-1 Oral Presentation Summary

In an oral presentation today, Julian R. Molina, M.D., Ph.D., professor of oncology, Mayo Clinic, Rochester, Minn., presented an update on the BASECAMP-1 prescreening study, highlighting innovative advancements that improve participant diversity in clinical trials and operational efficiency in patient recruitment. BASECAMP-1 is an ongoing, nationwide prescreening study featuring an innovative approach to overcome the operational burden of finding eligible participants for precision medicine studies. BASECAMP-1 uses a single next-generation sequencing (NGS) test from Tempus AI to efficiently screen for participants with tumor-associated HLA-A*02 LOH. Such screening identifies participants eligible for multiple current and future interventional trials of Tmod™ logic-gated CAR T therapies, improves trial diversity, and enhances the dataset and statistical power for translational studies. As of September 1, 2024, 70 participants have been enrolled in the BASECAMP-1 prescreening study.

A participants-matching program has been implemented to accelerate the identification and enrollment of participants whose tumors have HLA-A*02 LOH. Careful evaluation of the Tmod™ blocker has demonstrated that it functions well against HLA-A*02 subtypes beyond HLA-A*02:01, thus providing the impetus to enroll additional participants with broader ethnic and racial diversity. Based on these data, eligibility criteria have been amended to enroll patients with germline HLA-A*02:XX heterozygosity. As of July 1, 2024, this amendment has led to the potential enrollment of 16% more Hispanic, 43% more African American, and 112% more Asian and Pacific Islander participants, improving the racial and ethnic diversity of the BASECAMP-1 study population. Furthermore, information captured in the BASECAMP-1 study provides a large dataset for correlative analysis to further characterize tumors of patients with and without LOH.

“The wealth of data generated from the multicenter BASECAMP-1 prescreening study will enable more efficient identification and enrollment of patients undergoing cancer treatment at leading academic centers across the United States. Additional strategies to enhance access to BASECAMP-1 include increasing the geographic location of study sites; leveraging NGS across the US in both academic and community practices; and creating materials to help patients understand complex clinical trials,” Dr. Molina said.

EVEREST-1 Poster Presentation Summary

In a poster presentation today, Patrick M. Grierson, M.D., Ph.D., assistant professor in the division of oncology of Washington University in St. Louis, shared safety and early biomarker data from EVEREST-1 (abstract number 588), the first clinical study sponsored by A2 Bio. EVEREST-1 is a first-in-human, phase 1/2, multicenter, open label, nonrandomized study to evaluate the safety and efficacy of a single dose of A2B530 Tmod™ CAR T cells in adults with recurrent unresectable, locally advanced, or metastatic non-small cell lung, colorectal, pancreatic, or other solid tumors associated with CEA expression.

The first EVEREST-1 patient was dosed in May 2023 and, as of September 1, 2024, 14 patients have been enrolled (four patients with pancreatic cancer and 10 patients with colorectal cancer). Of these, two pancreatic cancer patients have reached one-year survival post-infusion. Pharmacokinetic data from 14 patients show a potential dose-response, with higher doses appearing to have an effect on the peak expansion of Tmod™ cells. There have been no reports of dose-limiting toxicities, grade >3 serious adverse events, or neurotoxicity related to A2B530, and there have been no significant safety issues in patients at their one-year follow-up visit and beyond. Dose escalation is ongoing, and the maximum tolerated dose has not yet been reached. To date, treatment with A2B530 appears to have a manageable safety profile and to be tolerable.

EVEREST-2 Poster Presentation Summary

In a poster presentation on November 8, Dr. Molina shared an enrollment update on EVEREST-2 (abstract number 627), a seamless, phase 1/2, open-label, nonrandomized study to evaluate the safety and efficacy of A2B694 in adult patients with solid tumors. EVEREST-2 is the second interventional clinical study sponsored by A2 Bio. The first patient was enrolled in EVEREST-2 in April 2024, and dose escalation is ongoing.

For more information about ongoing A2 Bio clinical trials, please visit https://www.a2bioclinicaltrials.com/.

About EVEREST-1

EVEREST-1 (NCT05736731) is a seamless Phase 1/2 study for A2B530, an autologous logic-gated investigational cell therapy developed from the A2 Bio proprietary Tmod™ platform. The Tmod™ platform provides selective killing of tumor cells and protection of normal cells via a dual-receptor design consisting of an activator that targets tumor cells and a blocker that protects normal cells. A2B530 consists of an activator that targets carcinoembryonic antigen (CEA) and a blocker that targets HLA-A*02. HLA-A*02 is lost in tumor cells and present in normal cells in the eligible patient population. The study is recruiting participants with non-small cell lung, colorectal and pancreatic cancers.

About EVEREST-2

EVEREST-2 (NCT06051695) is a seamless Phase 1/2 study for A2B694, an autologous logic-gated investigational cell therapy developed from the A2 Bio proprietary Tmod™ platform. The Tmod™ platform provides selective killing of tumor cells and protection of normal cells via a dual-receptor design consisting of an activator that targets tumor cells and a blocker that protects normal cells. A2B694 consists of an activator that targets mesothelin (MSLN) and a blocker that targets HLA-A*02. HLA-A*02 is lost in tumor cells and present in normal cells in the eligible patient population. The study is recruiting participants with non-small cell lung, colorectal, pancreatic, ovarian and mesothelioma cancers.

About BASECAMP-1

BASECAMP-1 (NCT04981119) is a prescreening study to identify patients for potential treatment in A2 Bio clinical trials. It is a novel approach to help optimize patient treatment outcomes by enabling patients’ immune cells to be stored in their healthiest state earlier in their course of cancer treatment. Next-generation sequencing is used to identify patients who have lost HLA-A*02, the biomarker of interest for the A2 Bio studies. Patients then undergo leukapheresis to collect, process, and store patient T cells for future Tmod™ CAR T cell therapy. BASECAMP-1 is currently enrolling participants with non-small cell lung, colorectal, pancreatic, ovarian and mesothelioma cancers.

About the Tmod™ Platform

A2 Bio has pioneered a precision-targeting cellular system – the Tmod™ platform – that incorporates two receptors, an activator and a blocker, to aim the powerful armaments of immune cells directly at tumors to unequivocally differentiate tumors from normal tissues. The activator recognizes antigens on tumor cells that trigger their destruction, while the blocker recognizes antigens on normal cells that protect them. This novel blocker technology enables precise, personalized and effective T cell targeting. The blocker component equips Tmod™ cells with the capacity to identify tumors as distinct from normal cells.

About A2 Bio

A2 Biotherapeutics, Inc. (A2 Bio) is a clinical-stage biotech company developing first-in-class logic-gated cell therapies to address the high unmet need in cancers. A2 Bio invented the proprietary Tmod™ cell therapy platform to tackle the fundamental challenge in cancer treatment—the ability of cancer medicines to distinguish between tumor and normal cells. For more information, please visit the company’s website at www.a2bio.com.

References

¹ Breanna DiAndreth, Agnes E. Hamburger, Han Xu, Alexander Kamb: The Tmod cellular logic gate as a solution for tumor-selective immunotherapy. Clinical Immunology, Volume 241, 2022, 109030.

² Lozac’hmeur A, Danek T, Yang Q, Rosasco MG, Welch JS, Go WY, Ng EW, Mardiros A, Maloney DG, Garon EB, Kirtane K, Simeone DM, Molina JR, Salahudeen AA, Stein MM, Hecht JR. Detecting HLA loss of heterozygosity within a standard diagnostic sequencing workflow for prognostic and therapeutic opportunities. NPJ Precis Oncol. 2024 Aug 5;8(1):174. doi: 10.1038/s41698-024-00665-z. PMID: 39103508; PMCID: PMC11300791.

Contacts

Andrea Greif

Jeff Winton Associates

andrea@jeffwintonassociates.com
914-772-3027

Mary-Frances Faraji

Jeff Winton Associates

maryfrances@jeffwintonassociates.com
908-334-7693

Innate Pharma Highlights Data From Its Innovative Oncology Portfolio Selected for the SITC Annual Meeting 2024




Innate Pharma Highlights Data From Its Innovative Oncology Portfolio Selected for the SITC Annual Meeting 2024

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

Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today announced that new preclinical data supporting the clinical development of its proprietary next generation antibody-drug conjugate (ADC) and innovative tetra-specific ANKET^® will be presented at the SITC Annual Meeting 2024.

“We are thrilled to share our latest preclinical data at the SITC Annual Meeting, highlighting the potential of IPH6501, our tetra-specific NK cell engager and IPH4502, our innovative ADC targeting Nectin-4. These findings underscore our commitment to advancing next-generation immunotherapies and reflect significant progress in the development of our drug candidates. We look forward to engaging with the scientific community as we continue to push the boundaries of next generation immunotherapies,” commented Pr. Eric Vivier, Chief Scientific Officer of Innate Pharma.

Details of the presentations

Society for Immunotherapy of Cancer (SITC) 39^th Annual Meeting | 6-10 November, Houston, Texas and Virtual

• Harnessing NK Cells in Cancer Therapies
  Session 107d: NK Cells and Innate Immunity
  
  Presentation Type: Oral Presentation
  
  Session Date and Time: Friday, Nov. 8, 2024, 3:50-5:25pm CST
  
  Presentation Time: 4:35pm CST
  
  Speaker: Eric Vivier, Chief Scientific Officer, Innate Pharma (co-chair of the session)

• Preclinical Characterization of IPH6501: A Novel IL2v-Armed Tetraspecific NK Cell Engager Targeting CD20 in Relapsed or Refractory B cell Non-Hodgkin Lymphoma Subtypes and Post-CAR-T Therapy.
  Abstract Number: 1083
  
  Presentation Type: Poster Presentation
  
  Primary Category: Immuno-Conjugates and Chimeric Molecules
  
  Poster Presentation Day: Friday, Nov. 8, 2024

• IPH45, a next-generation antibody-drug conjugate (ADC) targeting Nectin-4
  Abstract Number: 1056
  
  Presentation Type: Poster Presentation
  
  Primary Category: Immuno-Conjugates and Chimeric Molecules
  
  Poster Presentation Day: Saturday, Nov. 9, 2024
  

More information can be found on the JITC website.

Protein & Antibody Engineering Summit (PEGS) Europe | November 5-7, Barcelona, Spain and virtual

In addition, the presentation entitled « A Next-Generation ADC for Nectin-4 Expressing Tumours: Preclinical Characterisation of IPH45, a Novel and Differentiated Exatecan-Based ADC Targeting Nectin-4 » was presented at the PEGS Europe Summit. The presentation is available on the Company website, in the publications section.

About IPH4502

IPH4502 is a novel topoisomerase I inhibitor Antibody Drug Conjugate (ADC) conjugated to exatecan targeting Nectin-4.

Nectin-4 is a cell membrane adhesion protein overexpressed in several solid tumors, including urothelial, breast, lung, ovarian, and pancreatic cancers, with limited expression in normal tissues.

In non-clinical models, IPH4502 is well tolerated and shows anti-tumor efficacy in vitro and in vivo.

In September 2024, the U.S Food and Drug Administration cleared Innate’s investigational new drug (IND) application to initiate a Phase 1 clinical study of IPH4502 in Nectin-4 expressing solid tumor indications.

About IPH6501

IPH6501 is the first Antibody-based NK cell Engager Therapeutic to co-engage activating receptors on NK cells (NKp46 and CD16), IL-2R (but not a subunit) through a variant of human IL-2, and a tumor antigen (CD20) via a single molecule, hence providing proliferation and activation signals targeted to NK cells and promoting their cytotoxic activity against CD20 expressing malignant cells.

IPH6501 has shown better anti-tumor efficacy than approved benchmark antibodies in preclinical tumor models (Demaria, EHA 2023).

IPH6501 is currently being evaluated in a Phase 1/2 multicenter trial (NCT06088654), investigating the safety and tolerability of IPH6501 in patients with Relapsed and/or Refractory CD20-expressing B-cell Non-Hodgkin’s Lymphoma.

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: monoclonal antibodies, multi-specific NK Cell Engagers via its ANKET^® (Antibody-based NK cell Engager Therapeutics) proprietary platform and Antibody Drug Conjugates (ADC).

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

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

Square Knot Health Improves Access to Kidney Transplants, Results Reported at the American Society of Nephrology (ASN) Kidney Week Conference




Square Knot Health Improves Access to Kidney Transplants, Results Reported at the American Society of Nephrology (ASN) Kidney Week Conference

BOSTON–(BUSINESS WIRE)–#AvoidDialysis—Square Knot Health, a leader in technology-enabled kidney transplant care, has achieved industry leading results with a unique model of care that helps patients navigate the complex world of kidney transplantation.

Nearly 25% of patients referred to Square Knot received a kidney transplant in 1.6 years (on average), and more than half of these received a kidney from a living donor, far exceeding typical outcomes. The results were presented on October 26, 2024, at the 47th Annual Kidney Week Conference in San Diego. https://asn.apprisor.org/index.cfm?k=6q06blxkdi.

Square Knot had a dramatic impact on patients who were previously unable to gain access to the transplant list due to overweight, medical challenges, failure to complete tasks, or geographic isolation and other social determinants of health. With the support of Square Knot, 42% of these patients were added to the transplant waitlist, and 19% received a transplant.

Square Knot Health employs Transplant Peer Navigators (TPNs), telehealth, and artificial intelligence applied to medical records. Barriers to transplant are identified and removed, living donors are identified and supported, and patients are given the ongoing guidance they need to succeed.

“We work independently of, but in close collaboration with, multiple transplant centers. This approach allows us to move patients to the center where they are most likely to succeed. Ten of twenty-five patients who were transplanted in this initial report had difficulties with their initial center but were transplanted at a second or third transplant center with our guidance,” explained Dr. Eliot Heher, Square Knot Health founder and practicing transplant nephrologist. “A number of patients in this cohort remain on the transplant list and we continue to support them as they more towards a future transplant.”

Jullie Hoggan, Square Knot’s co-founder and lead navigator, herself a kidney transplant recipient, explains: “Our patients love the support from someone who has been through this difficult process. They express appreciation for the personalized care and easy-to-understand information provided. We are confident our involvement advances equity in kidney transplantation.”

Specialized AI for Kidney Transplant Assessments

Alongside our excellent results, Square Knot Health has developed a proprietary kidney transplant AI model that helps us quickly scale our impact and support care partners. Patients with chronic kidney disease have exceptionally long and complex medical records that are often scattered across multiple sites. Square Knot deploys AI to automate medical record retrieval, analyze patient candidacy and readiness for transplant, and produce documentation customized for adult nephrologists, transplant centers, and other providers. A top AI research team, patients.app, is embedded within Square Knot Health.

The Clinical and Economic Burden of Chronic Kidney Disease

More than 500,000 patients in the United States require chronic, maintenance kidney dialysis despite clear evidence that kidney transplantation is the optimal treatment for kidney failure. Up to 75% of patients with kidney failure are never referred for transplant, and 80% of those who are referred are neither listed nor transplanted 1 year later. Only 3% of patients who develop kidney failure in the United States each year receive transplants as their first therapy, while the remaining 97% of patients receive dialysis.

The chronic kidney disease market is expected to grow at a compound annual growth rate (CAGR) of 19.6% between 2023 and 2033. Its economic costs to the U.S. healthcare system continue to rise, with Medicare spending over $95 billion in 2022 to treat those with chronic kidney disease and approximately $53 billion on those with kidney failure.

Kidney transplants are more cost-effective than dialysis and improve patient survival and quality of life, mainly when performed before the start of dialysis.

Cost:

Medicare: Medicare spends over $89,000 per year per dialysis patient but only $35,000 per year per kidney transplant patient.

Break-even point: Health plans and patients can recoup the cost of a transplant in as little as 1.7 years, compared to dialysis costs.

Economic Impact: Transplanting patients on the waiting list could contribute over $63 billion to the US economy.

Results from American Society of Nephrology Kidney Week: https://asn.apprisor.org/index.cfm?k=6q06blxkdi.

For more information, www.squareknothealth.com.

Contacts

Melissa Coleman

Corporate Communications

702-357-0997

mcoleman@squareknothealth.com