Sarepta Therapeutics Announces Results from Part 2 of the EMBARK Study Demonstrating Sustained Benefits and Disease Stabilization in Ambulatory Individuals with Duchenne Muscular Dystrophy Following Treatment with ELEVIDYS




Sarepta Therapeutics Announces Results from Part 2 of the EMBARK Study Demonstrating Sustained Benefits and Disease Stabilization in Ambulatory Individuals with Duchenne Muscular Dystrophy Following Treatment with ELEVIDYS

– Treatment with ELEVIDYS corresponded with increases on the North Star Ambulatory Assessment (NSAA) at one year in crossover patients, while the study remained blinded

– MRI results at two years in patients treated in Part 1 show minimal muscle pathology progression, aligning closely with observed functional benefits

– Crossover-treated patients show statistically significant benefits of ELEVIDYS treatment on NSAA, Time to Rise (TTR), and 10-meter walk/run (10MWR), when compared to a pre-specified, well-matched external control (EC)

– Part 1-treated patients show sustained expression at week 64, and functional improvements on NSAA, TTR and 10MWR were sustained two years after treatment and show a widening divergence compared to EC

– Safety remained consistent with the profile of ELEVIDYS already established across a broad Duchenne population

– Investor webcast to be held today at 8:30 a.m. ET

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced positive topline results from Part 2 of EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study of ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy.

Crossover-treated patients, those who received a placebo in Part 1 and crossed over at 52 weeks and were treated with ELEVIDYS in Part 2, improved 2.34 points from baseline compared to matched external controls on the North Star Ambulatory Assessment (NSAA) 52 weeks after treatment (P<0.0001), during which time the study remained blinded.

Despite being one year older (average age 7.18 years) than those treated in Part 1 (average age 5.98 years), crossover-treated patients showed clinically meaningful and statistically significant functional benefit for NSAA, Time to Rise (TTR), and 10-meter walk/run (10MWR) function tests compared with a pre-specified, propensity-weighted external control group* (EC).

Crossover-Treated Patients (n=59) vs. EC

Two-Year Results

In patients treated in Part 1, biopsies taken 64 weeks after dosing showed consistent and sustained expression of ELEVIDYS micro-dystrophin compared to week 12 biopsies, as measured by western blot, and provide biological support for observed functional outcomes.

At two years, patients treated in Part 1 of EMBARK showed clinically meaningful and statistically significant functional benefit in NSAA, TTR and 10MWR compared with EC. Furthermore, the least square means (LSM) differences seen between the patients treated in Part 1 and the EC group increase from year one to year two for all three functional outcomes. This indicates that the trajectory of disease in patients treated with ELEVIDYS is continuing to diverge from the natural history of DMD.

Part 1, Year 2 (n=63) ELEVIDYS-Treated vs. EC

Skeletal muscle MRI conducted on patients treated in Part 1 found minimal progression in underlying muscle pathology and remain highly consistent with the functional benefits shown.

“We’re very encouraged to see the results from Part 2 of EMBARK as they further elucidate the impact ELEVIDYS has on disease progression in a blinded, controlled study. Skeletal muscle MRI demonstrates the importance of preserving muscle, and the functional outcome results show disease stabilization sustained through two years after treatment,” said Louise Rodino-Klapac, Ph.D., executive vice president, Head of R&D, Chief Scientific Officer. “Over time, we continue to observe a statistically significant difference favoring ELEVIDYS compared to a well-matched external control on NSAA and timed tests. The consistency and totality of evidence supporting a long-term and clinically meaningful treatment benefit with ELEVIDYS continues to grow. We look forward to sharing more details with the clinical community in upcoming scientific forums.”

No new safety signals were observed, reinforcing the consistent and manageable safety profile of ELEVIDYS to date. Detailed results from Part 2 of the EMBARK study will be shared at future medical meetings.

“As a neuromuscular medicine specialist who has seen patients with Duchenne muscular dystrophy for over three decades, I’ve witnessed firsthand the positive impact of gene therapy on the trajectory of Duchenne,” said Craig McDonald, M.D., professor and chair of the UC Davis Health Department of Physical Medicine and Rehabilitation, and an investigator in the EMBARK study. “These longer-term results are even more striking when compared to external control given the progressive nature of the disease, and we’d expect to see this divergence grow over time. The efficacy of ELEVIDYS gives me great hope as we continue to follow these patients and see others treated in the clinical setting.”

As part of a collaboration agreement signed in 2019, Sarepta is working with Roche to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world.

ELEVIDYS is approved for people living with Duchenne aged four years old and over regardless of their ambulatory status in the U.S., United Arab Emirates (UAE), Qatar, Kuwait, Bahrain and Oman. ELEVIDYS is also approved for the treatment of ambulatory patients aged four through seven years in Brazil and Israel.

*The pre-specified external control used data from five separate studies in Duchenne, comprising DMD controls from two randomized trials and three natural history cohorts who met predefined matching criteria. Comparison of treated and control patients was based on a pre-specified, propensity score weighting approach using age, steroid usage, baseline NSAA and timed function tests in order to balance key prognostic factors between the groups.

Sarepta Investor Call Details

At 8:30 a.m. ET on Jan. 27, 2025, Sarepta will host a conference call and webcast to discuss these results.

The event will be webcast live under the investor relations section of Sarepta’s website at https://investorrelations.sarepta.com/events-presentations and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone.

About EMBARK, Study 9001-301

Study SRP-9001-301, also known as EMBARK, is a multinational, phase 3, randomized, two-part crossover, placebo-controlled study of ELEVIDYS in individuals with Duchenne muscular dystrophy between the ages of 4 to 7 years. The primary endpoint is change from baseline in NSAA Total Score at Week 52 following treatment. Eligible participants received a single dose of ELEVIDYS during either Part 1 or Part 2 of the study.

In Part 1, participants (n=125) were randomized according to age (≥4 to <8 years) or NSAA Total Score at screening (>16 to <29) and received either 1.33 x1014 vg/kg of ELEVIDYS or placebo with a follow-up period for 52 weeks. In Part 2, participants cross over – meaning, those who were previously treated with placebo in Part 1 receive ELEVIDYS and participants who were previously treated with ELEVIDYS receive placebo, with a follow-up period for 52 weeks. All patients remained blinded through Part 1 and Part 2.

Secondary outcome measures in EMBARK include the quantity of shortened dystrophin produced by ELEVIDYS at week 12 as measured by western blot in a subset of participants, timed function tests, stride velocity and validated patient reported outcome measures for mobility and upper limb function. One-year results from the Part 1 placebo-controlled period of the EMBARK study were published in Nature Medicine in October 2024.

About ELEVIDYS (delandistrogene moxeparvovec-rokl)

ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.

ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age.

• For patients who are ambulatory and have a confirmed mutation in the DMD gene
• For patients who are non-ambulatory and have a confirmed mutation in the DMD gene.

The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin (noted hereafter as “micro-dystrophin”) in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

WARNINGS AND PRECAUTIONS:

Infusion-related Reactions:

• Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate.
• ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available.
• Discontinue infusion for anaphylaxis.

Acute Serious Liver Injury:

• Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks.
• Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.
• Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels).
• Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended.

Immune-mediated Myositis:

• In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed.
• Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction.
• Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient’s clinical presentation and medical history if these symptoms occur.

Myocarditis:

• Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials.
• If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath.
• Advise patients to contact a physician immediately if they experience cardiac symptoms.

Preexisting Immunity against AAVrh74:

• In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies.
• Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration.
• ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400.

Adverse Reactions:

• The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia.

Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).

For further information, please see the full Prescribing Information.

About Sarepta Therapeutics

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook.

Internet Posting of Information

We routinely post information that may be important to investors in the ‘For Investors’ section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Forward-Looking Statements

In order to provide Sarepta’s investors with an understanding of its current results and future prospects, this press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “may,” “intends,” “prepares,” “looks,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to ELEVIDYS; the potential benefits of our agreements with strategic partners; and expected milestones and plans, including sharing more details with the clinical community in upcoming scientific forums.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: success in clinical trials does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans for various reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading “Risk Factors” in our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

About Sarepta Therapeutics

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook.

Internet Posting of Information

We routinely post information that may be important to investors in the ‘For Investors’ section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Contacts

Investor:
Ian Estepan

617-274-4052

iestepan@sarepta.com

Media:
Tracy Sorrentino

617-301-8566

tsorrentino@sarepta.com

Kara Hoeger

617-710-3898

khoeger@sarepta.com

Innate Pharma Announces First Patient Dosed in Phase 1 Study of Its Nectin-4 Targeting Antibody Drug Conjugate IPH4502 in Selected Advanced Solid Tumors




Innate Pharma Announces First Patient Dosed in Phase 1 Study of Its Nectin-4 Targeting Antibody Drug Conjugate IPH4502 in Selected Advanced Solid Tumors

• IPH4502 is a novel and differentiated topoisomerase I inhibitor ADC conjugated to exatecan targeting Nectin-4

MARSEILLE, France–(BUSINESS WIRE)–$IPH #ADC–Regulatory News:

Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today announced the first patient was dosed in its Phase 1 study (NCT06781983), investigating the safety and tolerability of IPH4502, an innovative Antibody-Drug Conjugate (ADC), in patients with advanced solid tumors known to express Nectin-4.

IPH4502 is a novel and differentiated topoisomerase I inhibitor ADC designed to precisely target Nectin-4, a cell adhesion molecule that is overexpressed in several types of solid tumors, including urothelial carcinoma (UC), breast cancer, non-small cell lung cancer or gastro-intestinal cancer. IPH4502 targets tumors with a wide range of Nectin-4 expression, supporting its development beyond UC. IPH4502 differentiated topoisomerase I inhibitor payload supports its potential in patients with tumors resistant to MMAE-ADC.

The Phase 1, open-label, multi-center study, includes a Part 1 Dose Escalation and a Part 2 Dose Optimization, and will assess the safety, tolerability, and preliminary efficacy of IPH4502 as a single agent in advanced solid tumors known to express Nectin-4, including but not limited to urothelial carcinoma, non-small cell lung, breast, ovarian, gastric, esophageal, and colorectal cancers. The study plans to enroll approximately 105 patients.

“We are thrilled to initiate the Phase 1 study with IPH4502, a promising therapy for patients with advanced solid tumors known to express Nectin-4,” said Dr Shiraj Sen, Medical Oncologist and Phase 1 Investigator, Director of Clinical Research, NEXT Oncology-Dallas. “IPH4502 is uniquely designed to target tumors with both high and low expression of Nectin-4, offering hope for improved outcomes in a population where effective treatment options are limited, and relapses occur frequently. This study represents an important step forward in advancing innovative care for these patients.”

“The initiation of this Phase 1 trial represents a significant milestone for Innate Pharma as our clinical stage pipeline now includes targeted ADCs. We are optimistic about the potential of IPH4502 to address unmet needs in the treatment of advanced solid tumors, with a well differentiated Nectin-4 targeting and the promise of ADC technology to provide a new therapeutic option for patients,” added Dr Sonia Quaratino, Chief Medical Officer at Innate Pharma.

More information about the trial can be found on clinicaltrials.gov.

About IPH4502

IPH4502 is a novel topoisomerase I inhibitor Antibody Drug Conjugate (ADC) conjugated to exatecan targeting Nectin-4. It is currently investigated in a Phase 1 trial in advanced solid tumors expressing Nectin-4.

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

In non-clinical models, IPH45 is well tolerated and shows anti-tumor activity in vitro and in vivo.

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. 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

Corstasis Therapeutics Inc. Announces FDA Acceptance of New Drug Application for Bumetanide Nasal Spray




Corstasis Therapeutics Inc. Announces FDA Acceptance of New Drug Application for Bumetanide Nasal Spray

PDUFA Target Action Date Set for September 14, 2025

HENDERSON, Nev.–(BUSINESS WIRE)–#corstasistherapeutics–Corstasis Therapeutics Inc. (www.corstasis.com), a clinical-stage MedTech pharmaceutical company developing innovative, practical therapies for fluid overload, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for its novel Bumetanide Nasal Spray (RSQ-777). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 14, 2025.

This NDA acceptance represents a pivotal milestone for Corstasis Therapeutics, signifying the FDA’s determination that the application is sufficiently complete to move forward with a comprehensive review. Bumetanide Nasal Spray (RSQ-777) is being developed as a convenient, outpatient-focused therapy for edema associated with congestive heart failure, as well as liver and kidney disease.

“We are pleased the FDA has accepted our NDA for Bumetanide Nasal Spray,” said Benjamin Esque, CEO of Corstasis Therapeutics Inc. “This milestone brings us closer to providing a simple, easy-to-use outpatient therapy for patients with fluid overload. We look forward to working closely with the FDA throughout the review process.”

“Our aim is to reduce the burden on both patients and the healthcare system by offering treatment options outside the hospital setting—potentially lowering costs and improving quality of life,” added Dr. Brian Kolski, Chief Medical Officer of Corstasis Therapeutics Inc.

About Bumetanide Nasal Spray

Bumetanide Nasal Spray is an investigational new drug designed to enable patients to self-administer non-oral loop diuretic therapy through the nasal mucosa in an outpatient setting.

About Corstasis Therapeutics Inc.

Corstasis Therapeutics Inc. is a clinical-stage MedTech pharmaceutical company focused on maximizing the potential of proven therapeutics. Our mission is to develop and deliver pragmatic, innovative therapies that enhance patient care and reduce healthcare costs. For more information, please visit www.corstasis.com.

#homeiswheretheheartis

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements related to the development, regulatory progress, and potential benefits of Bumetanide Nasal Spray. These statements involve risks and uncertainties, such as the possibility that the FDA may not approve Bumetanide Nasal Spray by the expected date—or at all—and that additional data or analyses may be required. Actual results may differ materially from those expressed or implied by these forward-looking statements. Corstasis Therapeutics Inc. disclaims any obligation to update these statements except as required by law.

Contacts

Media Contact
Ben Esque, CEO

Corstasis Therapeutics Inc.

Phone: 702-541-9222

Email: Info@corstasis.com

Pfizer’s BRAFTOVI® Combination Regimen Demonstrates Improved Response in Patients with BRAF V600E-Mutant Metastatic Colorectal Cancer




Pfizer’s BRAFTOVI® Combination Regimen Demonstrates Improved Response in Patients with BRAF V600E-Mutant Metastatic Colorectal Cancer

• Clinically meaningful and statistically significant results from the Phase 3 BREAKWATER trial show objective response rate of 61% with Pfizer’s BRAFTOVI combination regimen compared to 40% with investigator’s choice of chemotherapy, representing a doubling of the odds of achieving an objective response
• BRAFTOVI combination regimen is the first and only targeted therapy approved by the U.S. FDA for treatment-naïve patients with metastatic colorectal cancer with a BRAF V600E mutation

NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE: PFE) today announced positive results from the Phase 3 BREAKWATER trial evaluating BRAFTOVI^® (encorafenib) in combination with cetuximab (marketed as ERBITUX^®) and mFOLFOX6 (fluorouracil, leucovorin, and oxaliplatin) in patients with metastatic colorectal cancer (mCRC) with a BRAF V600E mutation. At the time of this analysis, the BRAFTOVI combination regimen demonstrated a clinically meaningful and statistically significant improvement in confirmed objective response rate (ORR) assessed by blinded independent central review (BICR) compared to patients receiving chemotherapy with or without bevacizumab (60.9% vs 40.0%, odds ratio =2.443, p=0.0008). These results will be presented today in an oral presentation (Abstract 16) at the 2025 American Society of Clinical Oncology Gastrointestinal Cancer Symposium (ASCO GI) and were simultaneously published in Nature Medicine.

“Despite the high unmet need in this patient population, prior to the recent encorafenib combination regimen approval, there were no approved biomarker-driven therapies indicated for people with previously untreated BRAF V600E-mutant metastatic colorectal cancer,” said Scott Kopetz, M.D., Ph.D., FACP, Professor and Deputy Chair of Gastrointestinal Medical Oncology at The University of Texas MD Anderson Cancer Center and co-principal investigator of the BREAKWATER trial. “These data from the BREAKWATER study show the potential for this targeted treatment regimen to become the new standard of care for people with BRAF V600E-mutant metastatic colorectal cancer, for whom long-term disease control is critical.”

The estimated median duration of response as assessed by BICR was 13.9 months (95% Confidence Interval [CI]: 8.5-not estimable [NE]) with BRAFTOVI plus cetuximab and mFOLFOX6 and 11.1 months (95% CI: 6.7-12.7) with chemotherapy with or without bevacizumab. Of patients on BRAFTOVI plus cetuximab and mFOLFOX6, 22.4% (n=15) had a response lasting 12 months or longer, compared to 11.4% (n=5) with chemotherapy with or without bevacizumab. The median time to response as assessed by BICR was 7.1 weeks (range 5.7-53.7) with BRAFTOVI plus cetuximab and mFOLFOX6 and 7.3 weeks (range 5.4-48.0) with chemotherapy with or without bevacizumab.

Overall survival (OS) data were immature at the time of this analysis but demonstrated a promising trend in favor of BRAFTOVI plus cetuximab and mFOLFOX6 compared to patients receiving chemotherapy with or without bevacizumab. Median OS with BRAFTOVI plus cetuximab with chemotherapy was not estimable (95% CI: 19.8-NE) and 14.6 months (95% CI: 13.4-NE) with chemotherapy with or without bevacizumab (Hazard Ratio [HR]: 0.47, 95% CI: 0.318-0.691). The BREAKWATER trial is ongoing for OS and progression-free survival (PFS), with PFS results expected in 2025.

“These results of this first analysis were the basis for the first approval of a targeted therapy regimen for use in the first-line setting for patients with metastatic colorectal cancer with a BRAF V600E mutation,” said Roger Dansey, M.D., Chief Oncology Officer, Pfizer. “We are highly encouraged by these response results, which are indicative of the clinically meaningful benefit of BRAFTOVI in reducing tumor size or having no detectable cancer, along with the promising interim analysis of overall survival. We look forward to additional read-outs from the BREAKWATER trial this year.”

The safety profile of BRAFTOVI in combination with cetuximab and mFOLFOX6 in the BREAKWATER trial was consistent with the known safety profile of each respective agent. No new safety signals were identified. Serious treatment-emergent adverse events occurred in 37.7% of patients receiving BRAFTOVI in combination with cetuximab and mFOLFOX6 compared to 34.6% of patients receiving chemotherapy with or without bevacizumab.

BRAFTOVI in combination with cetuximab and mFOLFOX6 was granted accelerated approval by the U.S. Food and Drug Administration (FDA) for the treatment of patients with BRAF V600E-mutant mCRC in December 2024. The approval was among the first in the industry to be conducted under the FDA’s Project FrontRunner, which seeks to support the development and approval of new cancer drugs for advanced or metastatic disease. The BREAKWATER data are also being discussed with other regulatory authorities around the world to support potential future additional license applications for the BRAFTOVI combination regimen in this indication.

Pfizer is continuing its commitment to help non-scientists understand the latest findings with the development of abstract plain language summaries (APLS) for company-sponsored research being presented, which are written in non-technical language. Those interested in learning more can visit www.Pfizer.com/apls to access the summaries.

About BREAKWATER

BREAKWATER is a Phase 3, randomized, active-controlled, open-label, multicenter trial of BRAFTOVI with cetuximab, alone or in combination with mFOLFOX6 in participants with previously untreated BRAF V600E-mutant mCRC. Patients were randomized to receive BRAFTOVI 300 mg orally once daily in combination with cetuximab (discontinued after randomization of 158 patients), BRAFTOVI 300 mg orally once daily in combination with cetuximab and mFOLFOX6 (n=236) or mFOLFOX6, FOLFOXIRI, or CAPOX each with or without bevacizumab (control-arm) (n=243). The dual primary endpoints are ORR, which was met at the time of analysis, and PFS as assessed by BICR. OS is a key secondary endpoint.

About Colorectal Cancer (CRC)

CRC is the third most common type of cancer in the world, with approximately 1.8 million new diagnoses in 2022.¹ It is the second leading cause of cancer-related deaths.² Overall, the lifetime risk of developing CRC is about 1 in 24 for men and 1 in 26 for women.² In the U.S. alone, an estimated 154,270 people will be diagnosed with cancer of the colon or rectum in 2025, and approximately 53,000 are estimated to die from the disease each year.³ For 20% of those diagnosed with CRC, the disease has metastasized, or spread, making it harder to treat, and up to 50% of patients with localized disease eventually develop metastases.⁴

BRAF mutations are estimated to occur in 8-12% of people with mCRC and represent a poor prognosis for these patients.⁵ The BRAF V600E mutation is the most common BRAF mutation and the risk of mortality in CRC patients with the BRAF V600E mutation is more than double that of patients with no known mutation present.^5,6 Despite the high unmet need in BRAF V600E-mutant mCRC, prior to December 20, 2024, there were no approved biomarker-driven therapies specifically indicated for people with previously untreated BRAF V600E-mutant mCRC.^7,8

About BRAFTOVI^® (encorafenib)

BRAFTOVI is an oral small molecule kinase inhibitor that targets BRAF V600E. Inappropriate activation of proteins in the MAPK signaling pathway (RAS-RAF-MEK-ERK) has been shown to occur in certain cancers, including CRC.

Pfizer has exclusive rights to BRAFTOVI in the U.S., Canada, Latin America, Middle East, and Africa. Ono Pharmaceutical Co., Ltd. has exclusive rights to commercialize the product in Japan and South Korea, Medison has exclusive rights to commercialize the product in Israel and Pierre Fabre Laboratories has exclusive rights to commercialize the product in all other countries, including Europe and Asia (excluding Japan and South Korea).

INDICATION AND USAGE

BRAFTOVI^® (encorafenib) is indicated, in combination with cetuximab and mFOLFOX6, for the treatment of patients with metastatic colorectal cancer (mCRC) with a BRAF V600E mutation, as detected by an FDA-approved test. This indication is approved under accelerated approval based on response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

BRAFTOVI is also indicated, in combination with cetuximab, for the treatment of adult patients with mCRC with a BRAF V600E mutation, as detected by an FDA-approved test, after prior therapy.

Limitations of Use: BRAFTOVI is not indicated for treatment of patients with wild-type BRAF CRC.

IMPORTANT SAFETY INFORMATION

Refer to the prescribing information for cetuximab and individual product components of mFOLFOX6 for recommended dosing and additional safety information.

WARNINGS AND PRECAUTIONS

New Primary Malignancies: New primary malignancies, cutaneous and non-cutaneous, can occur. In BEACON CRC (previously treated BRAF V600E mutation-positive mCRC), cutaneous squamous cell carcinoma (cuSCC), including keratoacanthoma (KA), occurred in 1.4% of patients with CRC, and a new primary melanoma occurred in 1.4% of patients who received BRAFTOVI in combination with cetuximab. In BREAKWATER (previously untreated BRAF V600E mutation-positive mCRC) skin papilloma was reported in 2.6%, basal cell carcinoma in 1.3%, squamous cell carcinoma of skin in 0.9%, keratoacanthoma in 0.4% and malignant melanoma in situ in 0.4% of patients who received BRAFTOVI in combination with cetuximab and mFOLFOX6. Perform dermatologic evaluations prior to initiating treatment, every 2 months during treatment, and for up to 6 months following discontinuation of treatment. Manage suspicious skin lesions with excision and dermatopathologic evaluation. Dose modification is not recommended for new primary cutaneous malignancies. Based on its mechanism of action, BRAFTOVI may promote malignancies associated with activation of RAS through mutation or other mechanisms. Monitor patients receiving BRAFTOVI for signs and symptoms of non-cutaneous malignancies. Discontinue BRAFTOVI for RAS mutation-positive non-cutaneous malignancies. Monitor patients for new malignancies prior to initiation of treatment, while on treatment, and after discontinuation of treatment.

Tumor Promotion in BRAF Wild-Type Tumors: In vitro experiments have demonstrated paradoxical activation of MAP-kinase signaling and increased cell proliferation in BRAF wild-type cells exposed to BRAF inhibitors. Confirm evidence of BRAF V600E or V600K mutation using an FDA-approved test prior to initiating BRAFTOVI.

Cardiomyopathy: Cardiomyopathy manifesting as left ventricular dysfunction associated with symptomatic or asymptomatic decreases in ejection fraction, has been reported in patients. Assess left ventricular ejection fraction (LVEF) by echocardiogram or multi-gated acquisition (MUGA) scan prior to initiating treatment, 1 month after initiating treatment, and then every 2 to 3 months during treatment. The safety has not been established in patients with a baseline ejection fraction that is either below 50% or below the institutional lower limit of normal (LLN). Patients with cardiovascular risk factors should be monitored closely. Withhold, reduce dose, or permanently discontinue based on severity of adverse reaction.

Hepatotoxicity: Hepatotoxicity can occur. In BREAKWATER (previously untreated BRAF V600E mutation-positive mCRC), the incidence of Grade 3 or 4 increases in liver function laboratory tests in patients receiving BRAFTOVI in combination with cetuximab and mFOLFOX6 was 2.2% for alkaline phosphatase, 1.3% for ALT, and 0.9% for AST. Monitor liver laboratory tests before initiation of BRAFTOVI, monthly during treatment, and as clinically indicated. Withhold, reduce dose, or permanently discontinue based on severity of adverse reaction.

Hemorrhage: In BEACON CRC (previously treated BRAF V600E mutation-positive mCRC), hemorrhage occurred in 19% of patients receiving BRAFTOVI in combination with cetuximab; Grade 3 or higher hemorrhage occurred in 1.9% of patients, including fatal gastrointestinal hemorrhage in 0.5% of patients. The most frequent hemorrhagic events were epistaxis (6.9%), hematochezia (2.3%), and rectal hemorrhage (2.3%). In BREAKWATER (previously untreated BRAF V600E mutation-positive mCRC), hemorrhage occurred in 30% of patients receiving BRAFTOVI in combination with cetuximab and mFOLFOX6; Grade 3 or 4 hemorrhage occurred in 3% of patients. Withhold, reduce dose, or permanently discontinue based on severity of adverse reaction.

Uveitis: Uveitis, including iritis and iridocyclitis, has been reported in patients treated with BRAFTOVI. Assess for visual symptoms at each visit. Perform an ophthalmological evaluation at regular intervals and for new or worsening visual disturbances, and to follow new or persistent ophthalmologic findings. Withhold, reduce dose, or permanently discontinue based on severity of adverse reaction.

QT Prolongation: BRAFTOVI is associated with dose-dependent QTc interval prolongation in some patients. In BREAKWATER (previously untreated BRAF V600E mutation-positive mCRC), an increase of QTcF >500 ms was measured in 3.6% (8/222) of patients receiving BRAFTOVI in combination with cetuximab and mFOLFOX6. Monitor patients who already have or who are at significant risk of developing QTc prolongation, including patients with known long QT syndromes, clinically significant bradyarrhythmias, severe or uncontrolled heart failure and those taking other medicinal products associated with QT prolongation. Correct hypokalemia and hypomagnesemia prior to and during BRAFTOVI administration. Withhold, reduce dose, or permanently discontinue for QTc >500 ms.

Embryo-Fetal Toxicity: BRAFTOVI can cause fetal harm when administered to pregnant women. BRAFTOVI can render hormonal contraceptives ineffective. Advise females of reproductive potential to use effective nonhormonal contraception during treatment with BRAFTOVI and for 2 weeks after the final dose.

Risks Associated with Combination Treatment: BRAFTOVI is indicated for use as part of a regimen in combination with cetuximab, or in combination with cetuximab and mFOLFOX6. Refer to the prescribing information for cetuximab and individual product components of mFOLFOX6 for additional risk information.

Lactation: Advise women not to breastfeed during treatment with BRAFTOVI and for 2 weeks after the final dose.

Infertility: Advise males of reproductive potential that BRAFTOVI may impair fertility.

ADVERSE REACTIONS

BREAKWATER Trial (previously untreated BRAF V600E mutation-positive mCRC)

• Serious adverse reactions occurred in 38% of patients who received BRAFTOVI in combination with cetuximab and mFOLFOX6. Serious adverse reactions in >3% of patients included intestinal obstruction (3.5%) and pyrexia (3.5%).
• Fatal gastrointestinal perforation occurred in 0.9% of patients who received BRAFTOVI in combination with cetuximab and mFOLFOX6.
• Most common adverse reactions (≥25%, all grades) in the BRAFTOVI with cetuximab and mFOLFOX6 arm compared to the control arm (mFOLFOX6 ± bevacizumab or FOLFOXIRI ± bevacizumab or CAPOX ± bevacizumab) were peripheral neuropathy (62% vs 53%), nausea (51% vs 48%), fatigue (49% vs 38%), rash (31% vs 4%), diarrhea (34% vs 47%), decreased appetite (33% vs 25%), vomiting (33% vs 21%), hemorrhage (30% vs 18%), abdominal pain (26% vs 27%), and pyrexia (26% vs 14%).
• Most common laboratory abnormalities (≥10%, grade 3 or 4) in the BRAFTOVI with cetuximab and mFOLFOX6 arm compared to the control arm (mFOLFOX6 ± bevacizumab or FOLFOXIRI ± bevacizumab or CAPOX ± bevacizumab) were: increased lipase (51% vs 25%), decreased neutrophil count (36% vs 34%), decreased hemoglobin (13% vs 5%), decreased white blood cell count (12% vs 7%), and increased glucose (11% vs 2%).

BEACON CRC Trial (previously treated BRAF V600E mutation-positive mCRC)

• Most common adverse reactions (≥25%, all grades) in the BRAFTOVI with cetuximab arm compared to irinotecan with cetuximab or FOLFIRI with cetuximab (control) were: fatigue (51% vs 50%), nausea (34% vs 41%), diarrhea (33% vs 48%), dermatitis acneiform (32% vs 43%), abdominal pain (30% vs 32%), decreased appetite (27% vs 27%), arthralgia (27% vs 3%), and rash (26% vs 26%).
• Other clinically important adverse reactions occurring in <10% of patients who received BRAFTOVI in combination with cetuximab was pancreatitis.
• Most common laboratory abnormalities (all grades) (≥20%) in the BRAFTOVI with cetuximab arm compared to irinotecan with cetuximab or FOLFIRI with cetuximab (control) were: anemia (34% vs 48%) and lymphopenia (24% vs 35%).

DRUG INTERACTIONS

Strong or moderate CYP3A4 inhibitors: Avoid coadministration of BRAFTOVI with strong or moderate CYP3A4 inhibitors, including grapefruit juice. If coadministration is unavoidable, reduce the BRAFTOVI dose.

Strong CYP3A4 inducers: Avoid coadministration of BRAFTOVI with strong CYP3A4 inducers.

Sensitive CYP3A4 substrates: Avoid the coadministration of BRAFTOVI with CYP3A4 substrates (including hormonal contraceptives) for which a decrease in plasma concentration may lead to reduced efficacy of the substrate. If the coadministration cannot be avoided, see the CYP3A4 substrate product labeling for recommendations.

Dose reductions of drugs that are substrates of OATP1B1, OATP1B3, or BCRP may be required when used concomitantly with BRAFTOVI.

Avoid coadministration of BRAFTOVI with drugs known to prolong QT/QTc interval.

View the full Prescribing Information.

About Pfizer Oncology

At Pfizer Oncology, we are at the forefront of a new era in cancer care. Our industry-leading portfolio and extensive pipeline includes three core mechanisms of action to attack cancer from multiple angles, including small molecules, antibody-drug conjugates (ADCs), and bispecific antibodies, including other immune-oncology biologics. We are focused on delivering transformative therapies in some of the world’s most common cancers, including breast cancer, genitourinary cancer, hematology-oncology, and thoracic cancers, which includes lung cancer. Driven by science, we are committed to accelerating breakthroughs to help people with cancer live better and longer lives.

About Pfizer: Breakthroughs That Change Patients’ Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For 175 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. In addition, to learn more, please visit us on www.Pfizer.com and follow us on X at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Disclosure Notice

The information contained in this release is as of January 25, 2025. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about the BRAFTOVI^® (encorafenib) plus cetuximab and mFOLFOX6 combination and an indication in the U.S. for the treatment of metastatic colorectal cancer (mCRC) with a BRAF V600E mutation, as detected by an FDA-approved test, including their potential benefits and discussions with other regulatory authorities to support potential future additional license applications for the BRAFTOVI combination regimen in this indication, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of BRAFTOVI plus cetuximab and mFOLFOX6; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; whether the BREAKWATER trial will meet the primary endpoint of PFS or the secondary endpoint of OS; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any drug applications may be filed in any additional jurisdictions for BRAFTOVI plus cetuximab and mFOLFOX6 for the treatment of patients with metastatic CRC with a BRAFV600E mutation or in any jurisdictions for any other potential indications for BRAFTOVI; whether and when any such other applications may be approved by regulatory authorities, which will depend on a myriad factors, including making a determination as to whether the product’s benefits outweigh its known risks and determination of the product’s efficacy and, if approved, whether BRAFTOVI plus cetuximab and mFOLFOX6 will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of BRAFTOVI or BRAFTOVI plus cetuximab and mFOLFOX6; uncertainties regarding the impact of COVID-19 on Pfizer’s business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023, and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.com.

Erbitux^® is a registered trademark of Eli Lilly and Company and Merck KGaA, Darmstadt, Germany.

References

1. American Cancer Society. Global Cancer Facts & Figures 5th Edition. Available at: https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/global-cancer-facts-and-figures/global-cancer-facts-and-figures-2024.pdf. Last accessed: January 2025.
2. American Cancer Society. Key Statistics for Colorectal Cancer. Available at: https://www.cancer.org/cancer/types/colon-rectal-cancer/about/key-statistics.html. Last accessed: January 2025.
3. American Cancer Society. Cancer Facts & Figures 2025. Available at: https://www.cancer.

Contacts

Media Contact:

+1 (212) 733-1226

PfizerMediaRelations@Pfizer.com

Investor Contact:

+1 (212) 733-4848

IR@Pfizer.com

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Natera Announces Successful Readout of Randomized, Phase III CALGB (Alliance) / SWOG 80702 Clinical Trial in Colorectal Cancer




Natera Announces Successful Readout of Randomized, Phase III CALGB (Alliance) / SWOG 80702 Clinical Trial in Colorectal Cancer

Readout of ~1K patients at ASCO GI 2025 demonstrates that Signatera™ positive patients treated with both chemotherapy and celecoxib had a 40% improvement in overall survival versus chemotherapy alone

Additional ASCO GI poster highlights data from the ALTAIR clinical trial, showing significant clinical benefit in patients with stage IV colorectal cancer who were treated with Trifluridine/Tipiracil (FTD/TPI)

AUSTIN, Texas,–(BUSINESS WIRE)–Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA and genetic testing, announced new data from the randomized, Phase III CALGB (Alliance) / SWOG 80702 study. The study will be presented today, Jan. 25, 2025 as a late-breaking oral presentation at the 2025 American Society of Clinical Oncology’s Gastrointestinal Cancers Symposium (ASCO GI) in San Francisco, CA.

This first-of-its-kind study evaluated whether Signatera-positive patients benefit from an escalation in adjuvant treatment. In the trial, Signatera was used to evaluate the benefit of adding celecoxib, a non-steroidal anti-inflammatory drug (NSAID), to standard of care (SOC) adjuvant chemotherapy with FOLFOX in the management of stage III colorectal cancer (CRC). The pre-specified analysis included approximately 1,000 patients with available post-surgical plasma samples, who were randomized to receive FOLFOX (+/-) celecoxib.

Key findings included:

• Signatera-positivity after surgery was predictive of a disease-free survival (DFS) and overall survival (OS) benefit with the addition of celecoxib to adjuvant FOLFOX. The addition of celecoxib to SOC chemotherapy significantly improved DFS compared to placebo (HR 0.55, 95% CI 0.39-0.80; p=0.001) among Signatera-positive patients with a three-year DFS of 44.1% versus 26.6%. Similar results were seen for OS (HR 0.58, 95% CI 0.38-0.90; p=0.013). No survival benefit was seen by adding celecoxib to chemotherapy in Signatera-negative patients.
• Signatera status after surgery and prior to starting adjuvant therapy was highly predictive of recurrence. Signatera-positivity was significantly associated with worse DFS (HR 7.14, 95% CI: 5.54-9.21; p<0.0001) and OS (HR 6.72, 95% CI: 4.91-9.18; p<0.0001).

“The results from the CALGB (Alliance) / SWOG 80702 study mark an unprecedented moment in personalized medicine for patients with colorectal cancer,” said Alexey Aleshin, M.D., corporate chief medical officer and general manager of oncology for Natera. “We demonstrated Signatera’s ability to predict a benefit in both disease-free survival and overall survival for Signatera-positive patients from the addition of celecoxib, an extremely accessible, affordable, and well-tolerated therapy. These data also offer compelling evidence to address an unmet need in adjuvant colorectal cancer treatment, where there has not been a new drug approval in over 20 years.”

The results of the randomized, double-blind ALTAIR clinical trial will also be presented in a poster today. ALTAIR examined treatment escalation with Trifluridine/Tipiracil (FTD/TPI) in patients with stage I-IV colorectal cancer. In the trial, 243 Signatera-positive patients were randomized to FTD/TPI or placebo over a six-month treatment period. The results showed a trend toward benefit in the FTD/TPI group (median DFS of 9.3 months vs. 5.6 months in the placebo group), although it did not reach statistical significance (HR, 0.79; P = 0.107). There was a significant benefit for resected oligometastatic stage IV patients treated with FTD/TPI, showing a median DFS of 9.76 months as compared to 3.96 months in the placebo group (HR, 0.53; P = 0.012). This presents an opportunity for clinical benefit in stage IV patients who test positive for MRD.

About Signatera

Signatera is a personalized, tumor-informed, molecular residual disease test for patients previously diagnosed with cancer. Custom-built for each individual, Signatera uses circulating tumor DNA to detect and quantify cancer left in the body, identify recurrence earlier than standard-of-care tools, and help optimize treatment decisions. The test is available for clinical and research use and is covered by Medicare for patients with colorectal cancer, breast cancer, ovarian cancer, and muscle-invasive bladder cancer, as well as for immunotherapy monitoring of any solid tumor. Signatera has been clinically validated across multiple cancer types and indications, with published evidence in over 100 peer-reviewed papers.

About Natera

Natera™ is a global leader in cell-free DNA and genetic testing, dedicated to oncology, women’s health, and organ health. We aim to make personalized genetic testing and diagnostics part of the standard of care to protect health and inform earlier, more targeted interventions that help lead to longer, healthier lives. Natera’s tests are validated by more than 250 peer-reviewed publications that demonstrate high accuracy. Natera operates ISO 13485-certified and CAP-accredited laboratories certified under the Clinical Laboratory Improvement Amendments (CLIA) in Austin, Texas, and San Carlos, California. For more information, visit www.natera.com.

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 Natera’s plans, estimates, or expectations will be achieved. These forward-looking statements represent Natera’s expectations as of the date of this press release, and Natera 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. Additional risks and uncertainties are discussed in greater detail in “Risk Factors” in Natera’s recent filings on Forms 10-K and 10-Q, and in other filings Natera makes with the SEC from time to time. These documents are available at www.natera.com/investors and www.sec.gov.

Contacts

Investor Relations: Mike Brophy, CFO, Natera, Inc., 510-826-2350, investor@natera.com
Media: Lesley Bogdanow, VP of Corporate Communications, Natera, Inc., pr@natera.com

Bristol Myers Squibb Presents Results from CheckMate -8HW Analysis Evaluating Opdivo® (nivolumab) plus Yervoy® (ipilimumab) Compared to Opdivo Monotherapy…




Bristol Myers Squibb Presents Results from CheckMate -8HW Analysis Evaluating Opdivo® (nivolumab) plus Yervoy® (ipilimumab) Compared to Opdivo Monotherapy…

Patients experienced a 38% reduction in the risk of disease progression or death when treated with Opdivo plus Yervoy versus Opdivo monotherapy across all lines of therapy at median 47 months of follow-up

Late-breaking data presented in oral session at the 2025 American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium with same-day Publication in The Lancet

CheckMate -8HW previously demonstrated that Opdivo plus Yervoy reduced the risk of disease progression or death by 79% compared to investigator’s choice of chemotherapy

PRINCETON, N.J.–(BUSINESS WIRE)–$BMY #ASCO— 

Bristol Myers Squibb Presents Results from CheckMate -8HW Analysis Evaluating Opdivo^® (nivolumab) plus Yervoy^® (ipilimumab) Compared to Opdivo Monotherapy in Patients with Microsatellite Instability-High or Mismatch Repair Deficient Metastatic Colorectal Cancer

Bristol Myers Squibb (NYSE: BMY) today announced results of an analysis from the three-arm Phase 3 CheckMate -8HW trial evaluating Opdivo^® (nivolumab) plus Yervoy^® (ipilimumab) versus Opdivo monotherapy across all lines of therapy, including first-line, for the treatment of microsatellite instability-high/mismatch repair-deficient (MSI-H/dMMR) metastatic colorectal cancer (mCRC). At a median follow-up of 47 months, Opdivo plus Yervoy demonstrated a statistically significant and clinically meaningful improvement in the dual-primary endpoint of progression-free survival (PFS) as assessed by Blinded Independent Central Review (BICR) versus Opdivo monotherapy (HR 0.62; 95% CI 0.48–0.81; P = 0.0003).

These results will be featured in a late-breaking oral presentation (LBA143) at the American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium on January 25 at 1:00 p.m. Pacific Time, in San Francisco, California and published the same day in The Lancet.

Previous results from CheckMate -8HW, evaluating Opdivo plus Yervoy versus investigator’s choice of chemotherapy, demonstrated that Opdivo plus Yervoy reduced the risk of disease progression or death by 79%. The results were published in The New England Journal of Medicine and were the basis for a European Commission approval for first-line patients with MSI-H/dMMR mCRC in December 2024.

“The benefit of dual inhibition of PD-1 and CTLA-4 has been well established in Phase 3 trials of Opdivo plus Yervoy across a broad range of tumor types, including in MSI-H/dMMR mCRC compared to chemotherapy,” said Dana Walker, M.D., M.S.C.E., vice president, global program lead, late development, oncology, Bristol Myers Squibb. “The results from this analysis of the CheckMate -8HW trial answer affirmatively an important question about whether dual I/O therapy with Opdivo plus Yervoy can also improve outcomes for patients with MSI-H/dMMR mCRC compared with Opdivo alone.”

Results from the CheckMate -8HW trial at a median follow-up of 47 months in patients with MSI-H/dMMR mCRC across all lines of therapy include:

• PFS (progression-free survival; dual primary endpoint): Opdivo plus Yervoy demonstrated a 38% reduction in the risk of disease progression or death versus Opdivo monotherapy [HR 0.62; 95% CI 0.48–0.81; P = 0.0003]. PFS rates at 12-, 24-, and 36-months were also higher compared to Opdivo monotherapy (76%, 71% and 68% versus 63%, 56% and 51%, respectively).
• ORR (overall response rate; secondary endpoint): ORR by BICR was significantly higher with Opdivo plus Yervoy compared to Opdivo monotherapy [71% vs 58%; P = 0.0011].
• Safety: The safety profile for the combination of Opdivo plus Yervoy remained consistent with previously reported data and was manageable with established protocols. Grade 3/4 TRAEs were reported in 22% of patients with Opdivo plus Yervoy and 14% of patients with Opdivo. No new safety signals were identified.

“The data presented today confirm nivolumab plus ipilimumab as a new standard treatment for people living with metastatic colorectal cancer given the evidence observed in both disease-free survival and response rate,” said Thierry Andre, M.D., Head of the Medical Oncology Department, Sorbonne University and Hospital Saint-Antoine, Paris, France. “Importantly, no new safety signals were identified, with a moderate increase of treatment-related adverse events, mostly grade 1 or 2, observed with the combination. Together, these data reaffirm the benefit of dual immunotherapy with nivolumab plus ipilimumab for this population of patients who urgently need improved treatment options.”

The study is ongoing to assess secondary endpoints, including overall survival (OS). Bristol Myers Squibb thanks the patients and investigators involved in the CheckMate -8HW clinical trial.

About CheckMate -8HW

CheckMate -8HW (NCT04008030) is a Phase 3 randomized, open-label trial evaluating Opdivo plus Yervoy compared to Opdivo alone or the investigator’s choice chemotherapy (mFOLFOX-6 or FOLFIRI with or without bevacizumab or cetuximab) in patients with microsatellite instability–high (MSI-H) or mismatch repair deficient (dMMR) unresectable or metastatic colorectal cancer (mCRC).

839 patients were randomized to receive either Opdivo monotherapy (Opdivo 240 mg Q2W for six doses, followed by Opdivo 480 mg Q4W), Opdivo plus Yervoy (Opdivo 240 mg plus Yervoy 1 mg/kg Q3W for four doses, followed by Opdivo 480 mg Q4W), or investigator’s choice of chemotherapy. The dual primary endpoints of the trial are progression-free survival (PFS) per blinded independent central review (BICR) for Opdivo plus Yervoy compared to investigator’s choice of chemotherapy in the first-line setting and PFS per BICR for Opdivo plus Yervoy compared to Opdivo alone across all lines of therapy.

About dMMR or MSI-H Colorectal Cancer

Colorectal cancer (CRC) is cancer that develops in the colon or the rectum, which are part of the body’s digestive or gastrointestinal system. CRC is the third most commonly diagnosed cancer in the world. In 2020, it is estimated that there were approximately 1,931,000 new cases of the disease; it is the second leading cause of cancer-related deaths among men and women combined.

Mismatch repair deficiency (dMMR) occurs when the proteins that repair mismatch errors in DNA replication are missing or non-functional, leading to microsatellite instability-high (MSI-H) tumors. Approximately 5-7% of metastatic CRC patients have dMMR or MSI-H tumors. These patients are less likely to benefit from conventional chemotherapy and typically have a poor prognosis.

Bristol Myers Squibb: Creating a Better Future for People with Cancer

Bristol Myers Squibb is inspired by a single vision — transforming patients’ lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine and, through innovative digital platforms, are turning data into insights that sharpen their focus. Deep understanding of causal human biology, cutting-edge capabilities and differentiated research programs uniquely position the company to approach cancer from every angle.

Cancer can have a relentless grasp on many parts of a patient’s life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. As a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.

About Opdivo

Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.

Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.

In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.

About Yervoy

Yervoy is a recombinant, human monoclonal antibody that binds to the cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4). CTLA-4 is a negative regulator of T-cell activity. Yervoy binds to CTLA-4 and blocks the interaction of CTLA-4 with its ligands, CD80/CD86. Blockade of CTLA-4 has been shown to augment T-cell activation and proliferation, including the activation and proliferation of tumor infiltrating T-effector cells. Inhibition of CTLA-4 signaling can also reduce T-regulatory cell function, which may contribute to a general increase in T-cell responsiveness, including the anti-tumor immune response. On March 25, 2011, the U.S. Food and Drug Administration (FDA) approved Yervoy 3 mg/kg monotherapy for patients with unresectable or metastatic melanoma. Yervoy is approved for unresectable or metastatic melanoma in more than 50 countries. There is a broad, ongoing development program in place for Yervoy spanning multiple tumor types.

INDICATIONS

OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric patients 12 years and older with unresectable or metastatic melanoma.

OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adult and pediatric patients 12 years and older with unresectable or metastatic melanoma.

OPDIVO® is indicated for the adjuvant treatment of adult and pediatric patients 12 years and older with completely resected Stage IIB, Stage IIC, Stage III, or Stage IV melanoma.

OPDIVO® (nivolumab), in combination with platinum-doublet chemotherapy, is indicated as neoadjuvant treatment of adult patients with resectable (tumors ≥4 cm or node positive) non-small cell lung cancer (NSCLC).

OPDIVO® (nivolumab) in combination with platinum-doublet chemotherapy, is indicated for neoadjuvant treatment of adult patients with resectable (tumors ≥4 cm or node positive) non-small cell lung cancer (NSCLC) and no known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements, followed by single-agent OPDIVO® as adjuvant treatment after surgery.

OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.

OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

OPDIVO® (nivolumab) is indicated for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.

OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).

OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with intermediate or poor risk advanced renal cell carcinoma (RCC).

OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).

OPDIVO® (nivolumab) is indicated for the treatment of adult patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.

OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO® (nivolumab) is indicated for the treatment of adult patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.

OPDIVO® (nivolumab) is indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

OPDIVO® (nivolumab), as a single agent, is indicated for the adjuvant treatment of adult patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.

OPDIVO® (nivolumab), in combination with cisplatin and gemcitabine, is indicated as first-line treatment for adult patients with unresectable or metastatic urothelial carcinoma.

OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adult and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

OPDIVO® (nivolumab) is indicated for the treatment of adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.

OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in adult patients who have received neoadjuvant chemoradiotherapy (CRT).

OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC).

OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC).

OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of adult patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.

IMPORTANT SAFETY INFORMATION

Severe and Fatal Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions listed herein may not include all possible severe and fatal immune-mediated adverse reactions.

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue. While immune-mediated adverse reactions usually manifest during treatment, they can also occur after discontinuation of OPDIVO or YERVOY. Early identification and management are essential to ensure safe use of OPDIVO and YERVOY. Monitor for signs and symptoms that may be clinical manifestations of underlying immune-mediated adverse reactions. Evaluate clinical chemistries including liver enzymes, creatinine, adrenocorticotropic hormone (ACTH) level, and thyroid function at baseline and periodically during treatment with OPDIVO and before each dose of YERVOY. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue OPDIVO and YERVOY depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information). In general, if OPDIVO or YERVOY interruption or discontinuation is required, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose immune-mediated adverse reactions are not controlled with corticosteroid therapy. Toxicity management guidelines for adverse reactions that do not necessarily require systemic steroids (e.g., endocrinopathies and dermatologic reactions) are discussed below.

Immune-Mediated Pneumonitis

OPDIVO and YERVOY can cause immune-mediated pneumonitis. The incidence of pneumonitis is higher in patients who have received prior thoracic radiation. In patients receiving OPDIVO monotherapy, immune- mediated pneumonitis occurred in 3.1% (61/1994) of patients, including Grade 4 (<0.1%), Grade 3 (0.9%), and Grade 2 (2.1%). In patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, immune- mediated pneumonitis occurred in 7% (31/456) of patients, including Grade 4 (0.2%), Grade 3 (2.0%), and Grade 2 (4.4%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, immune- mediated pneumonitis occurred in 3.9% (26/666) of patients, including Grade 3 (1.4%) and Grade 2 (2.6%). In NSCLC patients receiving OPDIVO 3 mg/kg every 2 weeks with YERVOY 1 mg/kg every 6 weeks, immune- mediated pneumonitis occurred in 9% (50/576) of patients, including Grade 4 (0.5%), Grade 3 (3.5%), and Grade 2 (4.0%). Four patients (0.7%) died due to pneumonitis.

In Checkmate 205 and 039, pneumonitis, including interstitial lung disease, occurred in 6.0% (16/266) of patients receiving OPDIVO. Immune-mediated pneumonitis occurred in 4.9% (13/266) of patients receiving OPDIVO, including Grade 3 (n=1) and Grade 2 (n=12).

Immune-Mediated Colitis

OPDIVO and YERVOY can cause immune-mediated colitis, which may be fatal. A common symptom included in the definition of colitis was diarrhea. Cytomegalovirus (CMV) infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. In patients receiving OPDIVO monotherapy, immune-mediated colitis occurred in 2.9% (58/1994) of patients, including Grade 3 (1.7%) and Grade 2 (1%). In patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, immune-mediated colitis occurred in 25% (115/456) of patients, including Grade 4 (0.4%), Grade 3 (14%) and Grade 2 (8%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, immune-mediated colitis occurred in 9% (60/666) of patients, including Grade 3 (4.4%) and Grade 2 (3.7%).

Immune-Mediated Hepatitis and Hepatotoxicity

OPDIVO and YERVOY can cause immune-mediated hepatitis. In patients receiving OPDIVO monotherapy, immune-mediated hepatitis occurred in 1.8% (35/1994) of patients, including Grade 4 (0.2%), Grade 3 (1.3%), and Grade 2 (0.4%). In patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, immune- mediated hepatitis occurred in 15% (70/456) of patients, including Grade 4 (2.4%), Grade 3 (11%), and Grade 2 (1.8%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, immune-mediated hepatitis occurred in 7% (48/666) of patients, including Grade 4 (1.2%), Grade 3 (4.9%), and Grade 2 (0.4%).

OPDIVO in combination with cabozantinib can cause hepatic toxicity with higher frequencies of Grade 3 and 4 ALT and AST elevations compared to OPDIVO alone. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. In patients receiving OPDIVO and cabozantinib, Grades 3 and 4 increased ALT or AST were seen in 11% of patients.

Immune-Mediated Endocrinopathies

OPDIVO and YERVOY can cause primary or secondary adrenal insufficiency, immune-mediated hypophysitis, immune-mediated thyroid disorders, and Type 1 diabetes mellitus, which can present with diabetic ketoacidosis. Withhold OPDIVO and YERVOY depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information). For Grade 2 or higher adrenal insufficiency, initiate symptomatic treatment, including hormone replacement as clinically indicated. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism; initiate hormone replacement as clinically indicated. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism; initiate hormone replacement or medical management as clinically indicated. Monitor patients for hyperglycemia or other signs and symptoms of diabetes; initiate treatment with insulin as clinically indicated.

In patients receiving OPDIVO monotherapy, adrenal insufficiency occurred in 1% (20/1994), including Grade 3 (0.4%) and Grade 2 (0.

Contacts

Bristol Myers Squibb


Media Inquiries:
media@bms.com

Investors:
investor.relations@bms.com

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Exelixis Announces Encouraging Results from Phase 1b/2 STELLAR-001 Trial Evaluating Zanzalintinib Alone or in Combination with an Immune Checkpoint Inhibitor in Metastatic Colorectal Cancer at ASCO GI 2025




Exelixis Announces Encouraging Results from Phase 1b/2 STELLAR-001 Trial Evaluating Zanzalintinib Alone or in Combination with an Immune Checkpoint Inhibitor in Metastatic Colorectal Cancer at ASCO GI 2025

– In a subgroup analysis of patients without liver metastases, adding atezolizumab to zanzalintinib led to enhanced progression-free survival and overall survival –

ALAMEDA, Calif.–(BUSINESS WIRE)–Exelixis, Inc. (Nasdaq: EXEL) today announced results from an expansion cohort of the phase 1b/2 STELLAR-001 trial evaluating zanzalintinib alone or in combination with atezolizumab (Tecentriq^®) in patients with previously-treated metastatic colorectal cancer (CRC). The findings will be presented during Poster Session C: Cancers of the Colon, Rectum and Anus, at 7:00 a.m. PT on January 25 at the American Society of Clinical Oncology 2025 Gastrointestinal Cancers Symposium (ASCO GI 2025).

This cohort of the STELLAR-001 trial included 107 patients randomized 1:1 to receive single-agent zanzalintinib or zanzalintinib in combination with atezolizumab. Patients had unresectable, locally advanced or metastatic RAS wild-type CRC that was non-microsatellite instability-high or non-mismatch repair-deficient. The median number of prior lines of therapy was 3.0 for patients treated with zanzalintinib alone and 2.5 for patients treated with zanzalintinib in combination with atezolizumab. Thirty-two percent and 31% of patients did not have liver metastases in baseline scans, respectively.

Both progression-free survival (PFS) and overall survival (OS) were numerically improved by the addition of atezolizumab to zanzalintinib. Detailed efficacy results of the overall population and the subgroup analysis of patients without liver metastases are in Table 1 below.

In a biomarker analysis, a PD-L1 combined positive score greater than 1 was associated with improved PFS and OS in patients treated with zanzalintinib in combination with atezolizumab versus zanzalintinib alone.

“This cohort of the STELLAR-001 trial was designed to inform the contribution of atezolizumab to zanzalintinib in patients with previously treated metastatic colorectal cancer,” said Amy Peterson, M.D., Executive Vice President, Product Development & Medical Affairs, and Chief Medical Officer, Exelixis. “Data from this randomized expansion cohort reaffirms our decision to initiate STELLAR-303 evaluating zanzalintinib in combination with atezolizumab compared with regorafenib in patients with metastatic colorectal cancer, which completed enrollment in August 2024, and we anticipate data from that trial in the second half of 2025, dependent on study event rates.”

Detailed safety results for the overall population are in Table 2 below. Grade 3/4 treatment-related adverse events (AEs) occurred in 40% of patients receiving zanzalintinib alone and 48% of patients receiving zanzalintinib in combination with atezolizumab. One grade 5 treatment-related AE occurred in each group, both of which were also determined by investigator as related to the disease under study. Zanzalintinib was discontinued in 19% of patients receiving zanzalintinib alone and 30% of patients receiving zanzalintinib in combination with atezolizumab. Treatment-related AEs leading to discontinuation of any drug occurred in 8% and 19% of patients, respectively.

About STELLAR-001

STELLAR-001 (NCT03845166) is a global, open-label phase 1b/2 study of zanzalintinib as a single agent or in combination with atezolizumab in patients with inoperable locally advanced or metastatic solid tumors. The trial is divided into two parts: a dose-escalation stage and an expansion cohort stage. The expansion cohorts evaluating zanzalintinib (100 mg) as a single agent or in combination with atezolizumab also include patients with clear cell renal cell carcinoma (RCC), non-clear cell RCC, breast cancer that is hormone receptor-positive and HER-2 negative and castration-resistant prostate cancer. More information about the trial is available at ClinicalTrials.gov.

About STELLAR-303

The global phase 3 pivotal study, STELLAR-303, is evaluating zanzalintinib (100 mg) in combination with atezolizumab compared with regorafenib in patients with metastatic, refractory non-microsatellite instability-high or non-mismatch repair-deficient CRC. The primary endpoint in the study is OS in patients without active liver metastases. If OS is positive in the population of patients without liver metastases, the study will evaluate OS in the intent-to-treat population that includes patients with and without liver metastases. The study completed enrollment in the third quarter of 2024, and preliminary results are expected in the second half of 2025, dependent on study event rates. More information about the trial is available at ClinicalTrials.gov.

About Zanzalintinib

Zanzalintinib is a third-generation oral tyrosine kinase inhibitor that inhibits the activity of receptor tyrosine kinases implicated in cancer growth and spread, including VEGF receptors, MET, AXL and MER. These receptor tyrosine kinases are involved in both normal cellular function and in pathologic processes such as oncogenesis, metastasis, tumor angiogenesis and resistance to multiple therapies, including immune checkpoint inhibitors. With zanzalintinib, Exelixis sought to build upon its extensive experience with the target profile of cabozantinib, the company’s flagship medicine, while improving key characteristics, including pharmacokinetic half-life. Zanzalintinib is currently being developed for the treatment of advanced solid tumors, including genitourinary, colorectal and head and neck cancers. A phase 3 pivotal trial evaluating zanzalintinib compared with everolimus as a first oral therapy in patients with advanced neuroendocrine tumors (NET), regardless of site of origin, is expected to be initiated in the first half of 2025.

About CRC

Colorectal cancer is the third most common cancer and the second leading cause of cancer-related deaths in the U.S.¹ Approximately 154,000 new cases will be diagnosed in the U.S. with around 53,000 expected deaths from the disease in 2025.¹ Colorectal cancer is most frequently diagnosed among people aged 65-74 and is more common in men and in people of non-Hispanic American Indian/Alaska Native descent.² Nearly a quarter of colorectal cancer cases are diagnosed at the metastatic stage, at which point the five-year survival rate is just 15.7%.²

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 data from STELLAR-001 at ASCO GI 2025; the expectation for initial clinical data readouts from STELLAR-303 in the second half of 2025, dependent on study event rates; the therapeutic potential of zanzalintinib, both alone and in combination with atezolizumab, in patients with metastatic CRC; 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’ continuing compliance with applicable legal and regulatory requirements; the potential failure of zanzalintinib, both alone and in combination with atezolizumab to demonstrate safety and/or efficacy in STELLAR-001, STELLAR-303 and 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 zanzalintinib; the costs of conducting clinical trials; Exelixis’ dependence on third-party vendors for the development, manufacture and supply of zanzalintinib; Exelixis’ ability to protect its intellectual property rights; market competition; 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.

TECENTRIQ (atezolizumab) is a registered trademark of Genentech, a member of the Roche Group.

<sup>_________________

1</sup> Key Statistics for Colorectal Cancer. ACS. Available at: https://www.cancer.org/cancer/types/colon-rectal-cancer/about/key-statistics.html. Accessed January 2025.

² Cancer Stat Facts: Colorectal Cancer. SEER. Available at: https://seer.cancer.gov/statfacts/html/colorect.html. Accessed January 2025.

Contacts

Investors Contact:
Susan Hubbard
EVP, Public Affairs and

Investor Relations
Exelixis, Inc.
650-837-8194
shubbard@exelixis.com

Media Contact:
Stekki Millman
Senior Director, Public Affairs
Exelixis, Inc.
650-837-7187
smillman@exelixis.com

Compass Pathways Appoints Steve Levine, M.D., as Chief Patient Officer




Compass Pathways Appoints Steve Levine, M.D., as Chief Patient Officer

Newly created role of Chief Patient Officer reinforces Compass’ vision of working to achieve broad and equitable access to COMP360, if approved

LONDON & NEW YORK–(BUSINESS WIRE)–$CMPS #Biotech–Compass Pathways plc (Nasdaq: CMPS) (“Compass”), a biotechnology company dedicated to accelerating access to evidence-based innovation in mental health, today announced the appointment of Steve Levine, M.D., as Chief Patient Officer, effective immediately. In this newly developed executive team role, Dr. Levine will be responsible for ensuring that patient-centric strategies are embedded across the organization and that treatments and initiatives developed by the company are focused on addressing unmet needs in current clinical care and are designed to improve patient outcomes.

“Steve has spent the past four years working to deeply understand barriers to care for patients living with serious mental health conditions and he is committed to helping find solutions to address those barriers,” said Kabir Nath, CEO of Compass Pathways. “Steve shares a passion for our mission to bring better options to people who urgently need them, and I am delighted to welcome him onto the executive leadership team and into the newly developed Chief Patient Officer role.”

Dr. Levine has been a member of the Compass team since 2020, serving as Senior Vice President of Patient Access. Dr. Levine is a board-certified psychiatrist who has spent his career working across multiple facets of the healthcare system to improve people’s lives through creating access to innovation. Dr. Levine completed his internship and residency in psychiatry at New York – Presbyterian Hospital/Weill Cornell Medical Center. He then completed a fellowship subspecialty training in psychosomatic medicine/psycho-oncology at Memorial Sloan Kettering Cancer Center/New York – Presbyterian Hospital. Prior to Compass, he led the company he founded in 2010, Actify Neurotherapies, that built new models of care delivery across the US for interventional psychiatry treatments. He has published extensively in both peer-reviewed journals and popular media, presented to both professional and lay audiences around the world, served in leadership roles for professional societies and not-for-profit entities, and received numerous awards for leadership and service. Dr. Levine is also an adjunct professor at Rutgers Medical School.

Offering congratulations to Dr. Levine on his expanded role as Chief Patient Officer at Compass Pathways, Debbie Plotnick, Executive Vice President for State and Federal Advocacy at Mental Health America (MHA), stated, “Listening to people impacted by mental health conditions and prioritizing their needs and desires for new types of therapies, and working to assure access to them, are shared core values of Compass and MHA.”

“I’ve spent my career pursuing better outcomes and experiences for those living with serious mental health conditions. At Compass, I believe we have an opportunity to transform lives by creating, and making accessible, truly new options,” said Dr. Levine. “I am excited about the opportunity that I have in this new role to help transform patient care.”

About Compass Pathways

Compass Pathways plc (Nasdaq: CMPS) is a biotechnology company dedicated to accelerating patient access to evidence-based innovation in mental health. Our focus is on improving the lives of those who are living with mental health challenges and who are not helped by existing standards of care. We are pioneering the development of a new model of psilocybin treatment, in which our proprietary formulation of synthesized psilocybin, COMP360, is administered in conjunction with psychological support. COMP360 has Breakthrough Therapy designation from the US Food and Drug Administration (FDA) and has received Innovative Licensing and Access Pathway (ILAP) designation in the UK for treatment-resistant depression (TRD).

Compass is headquartered in London, UK, with offices in New York and San Francisco in the US. Our vision is a world of mental wellbeing.

Forward-looking statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. In some cases, forward-looking statements can be identified by terminology such as “may”, “might”, “will”, “could”, “would”, “should”, “expect”, “intend”, “plan”, “objective”, “anticipate”, “believe”, “contemplate”, “estimate”, “predict”, “potential”, “continue” and “ongoing,” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. Forward-looking statements include express or implied statements relating to, among other things, statements regarding our expectations regarding the impact of executive team appointments; the safety or efficacy of investigational COMP360 psilocybin treatment as a treatment for mental health conditions, including TRD, PTSD or anorexia nervosa; the potential for the pivotal phase 3 program in TRD or other trials to support regulatory filings and approvals; our ability to obtain regulatory approval and adequate coverage and reimbursement; our ability to transition from a clinical-stage to a commercial-stage organization and effectively launch a commercial product, if regulatory approval is obtained; and our expectations regarding the benefits of our investigational COMP360 psilocybin treatment . The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Compass’s control and which could cause actual results, levels of activity, performance or achievements to differ materially from those expressed or implied by these forward-looking statements.

These risks, uncertainties, and other factors include, among others: uncertainties associated with risks related to clinical development which is a lengthy and expensive process with uncertain outcomes, and therefore our clinical trials may be delayed or terminated and may be more costly than expected; the results of early-stage clinical trials of our investigational COMP360 psilocybin treatment may not be predictive of the results of later stage clinical trials; our need for substantial additional funding to achieve our business goals and if we are unable to obtain this funding when needed and on acceptable terms, we could be forced to delay, limit or terminate our clinical trials; our efforts to obtain marketing approval from the applicable regulatory authorities in any jurisdiction for our investigational COMP360 psilocybin treatment may be unsuccessful; our efforts to commercialize and obtain coverage and reimbursement for our investigational COMP360 psilocybin treatment, if approved, may be unsuccessful; our ability to successfully manage executive team changes and our ability to retain key personnel; and those risks and uncertainties described under the heading “Risk Factors” in Compass’s most recent annual report on Form 10-K or quarterly report on Form 10-Q, the prospectus supplement related to the proposed public offering we plan to file and in other reports we have filed with the U.S. Securities and Exchange Commission (“SEC”), which are available on the SEC’s website at www.sec.gov. Except as required by law, Compass disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Compass’s current expectations and speak only as of the date hereof.

Contacts

Enquiries
Media: media@compasspathways.com
Investors: Stephen Schultz, stephen.schultz@compasspathways.com, +1 401 290 7324

Revelation Biosciences Starts its PRIME Phase 1b Clinical Study of Gemini in CKD Patients




Revelation Biosciences Starts its PRIME Phase 1b Clinical Study of Gemini in CKD Patients

– Dosing to Commence Mid-February –

– Topline data expected Mid-Year –

SAN DIEGO–(BUSINESS WIRE)–$REVB #AKI—Revelation Biosciences, Inc. (NASDAQ: REVB) (the “Company” or “Revelation”), a clinical-stage life sciences company that is focused on harnessing the power of trained immunity for the treatment of disease, announced today that it has started its PRIME (PReconditioning IMmunostimulatory Evaluation) Phase 1b clinical study of escalating doses of intravenously administered Gemini in patients with Stage 3 and 4 Chronic Kidney Disease (CKD). The US based multi-site placebo-controlled study will enroll up to forty patients in five cohorts of single escalating doses.

“I am delighted to see the Revelation teams’ hard work culminating with the start of the PRIME study.” said James Rolke, Chief Executive Officer of Revelation. “We look forward to the results and plan to educate the public over the next few months on the importance of the planned biomarkers as it relates to CKD treatment and prevention of AKI.”

Top-line data including safety, tolerability, and biomarkers of target activity are expected by mid-year. Data from the PRIME clinical study will support future development in both the GEM-CKD and GEM-AKI programs.

About AKI

AKI, also known as acute renal failure, is defined as a rapid loss of kidney function. AKI causes a build-up of waste products in blood and makes it more difficult for kidneys to maintain the correct balance of fluid in the body. AKI can also have a significant impact on other organs such as the brain, heart, and lungs.

About CKD

Chronic kidney disease is a pervasive problem in the United States and world-wide. CKD is due to chronic inflammation and can be initiated and propagated in several ways. One prevalent condition is the high blood sugar levels associated with diabetes (either Type 1 or Type 2). High blood sugar is toxic to kidney cells creating stress which imitates the inflammatory process leading to the demise of these cells with subsequent fibrosis ultimately resulting in continuous loss of kidney function over time. High arterial blood pressure is another source of stress that initiates the inflammatory process leading to CKD. Other risk factors include heart disease, obesity, family history of CKD or older age. Progression of chronic kidney damage often leads to end stage renal disease with the need for renal replacement therapy (dialysis or transplantation), resulting in significant morbidity and mortality for affected patients. Kidney diseases are the leading cause of death in the United States.

About Gemini

Gemini is an intravenously administrated, proprietary formulation of phosphorylated hexaacyl disaccharide (PHAD^®) that reduces the damage associated with inflammation by reprograming the innate immune system to respond to stress (trauma, infection, etc.) in an attenuated manner. Revelation has conducted multiple preclinical studies demonstrating the therapeutic potential of Gemini in the target indications. Earlier this year Revelation announced positive Phase 1 clinical data for intravenous treatment with Gemini. The primary safety endpoint was met in the Phase 1 study, and results demonstrated statistically significant pharmacodynamic activity as observed through expected changes in multiple biomarkers including upregulation of IL-10.

Gemini is being developed for multiple indications including as a pretreatment to prevent or reduce the severity and duration of acute kidney injury (GEMINI-AKI program), and as pretreatment to prevent or reduce the severity and duration of post-surgical infection (GEMINI-PSI program). In addition, Gemini may be a treatment to stop or slow the progression of chronic kidney disease (GEMINI-CKD program).

About Revelation Biosciences, Inc.

Revelation Biosciences, Inc. is a clinical stage life sciences company focused on harnessing the power of trained immunity for the prevention and treatment of disease using its proprietary formulation Gemini. Revelation has multiple ongoing programs to evaluate Gemini, including as a prevention for post-surgical infection, as a prevention for acute kidney injury, and for the treatment of chronic kidney disease.

For more information on Revelation, please visit www.RevBiosciences.com.

Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These forward-looking statements are generally identified by the words “anticipate”, “believe”, “expect”, “estimate”, “plan”, “outlook”, and “project” and other similar expressions. We caution investors that forward-looking statements are based on management’s expectations and are only predictions or statements of current expectations and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from those anticipated by the forward-looking statements. Revelation cautions readers not to place undue reliance on any such forward looking statements, which speak only as of the date they were made. The following factors, among others, could cause actual results to differ materially from those described in these forward-looking statements: the ability of Revelation to meet its financial and strategic goals, due to, among other things, competition; the ability of Revelation to grow and manage growth profitability and retain its key employees; the possibility that the Revelation may be adversely affected by other economic, business, and/or competitive factors; risks relating to the successful development of Revelation’s product candidates; the ability to successfully complete planned clinical studies of its product candidates; the risk that we may not fully enroll our clinical studies or enrollment will take longer than expected; risks relating to the occurrence of adverse safety events and/or unexpected concerns that may arise from data or analysis from our clinical studies; changes in applicable laws or regulations; expected initiation of the clinical studies, the timing of clinical data; the outcome of the clinical data, including whether the results of such study is positive or whether it can be replicated; the outcome of data collected, including whether the results of such data and/or correlation can be replicated; the timing, costs, conduct and outcome of our other clinical studies; the anticipated treatment of future clinical data by the FDA, the EMA or other regulatory authorities, including whether such data will be sufficient for approval; the success of future development activities for its product candidates; potential indications for which product candidates may be developed; the ability of Revelation to maintain the listing of its securities on NASDAQ; the expected duration over which Revelation’s balances will fund its operations; and other risks and uncertainties described herein, as well as those risks and uncertainties discussed from time to time in other reports and other public filings with the SEC by Revelation.

Contacts

Mike Porter

Investor Relations

Porter LeVay & Rose Inc.

Email: mike@plrinvest.com

Chester Zygmont, III

Chief Financial Officer
Revelation Biosciences Inc.

Email: czygmont@revbiosciences.com

Glox Therapeutics Awarded Share of £3M Collaborative Discovery Programme Funding from Cystic Fibrosis Antimicrobial Resistance Syndicate




Glox Therapeutics Awarded Share of £3M Collaborative Discovery Programme Funding from Cystic Fibrosis Antimicrobial Resistance Syndicate

• £500K grant to advance discovery and development of effective precision antibiotics targeting antimicrobial-resistant lung infections in people with cystic fibrosis
• Programme delivered by CF AMR Syndicate offers cross-sector collaboration and support to address urgent unmet need

GLASGOW, Scotland–(BUSINESS WIRE)–#AMR–Glox Therapeutics, a company pioneering the development of precision antibiotic therapies based on naturally occurring bacteriocins, today announced it has been awarded a share of the £3 million Collaborative Discovery Programme (CDP) launched by the Cystic Fibrosis Antimicrobial Resistance (CF AMR) Syndicate with funding from the medical research charity, LifeArc. Glox Therapeutics will receive up to £500,000 of the fund to accelerate the development of its novel precision antibiotics, helping to address the growing need for effective treatments to overcome antimicrobial-resistant lung infections in people with cystic fibrosis (CF).

Over 162,000+ people are estimated to be living with CF globally*. The thick mucus that lines the lungs of people with CF can be difficult to clear, often resulting in infections due to pathogens, such as Pseudomonas aeruginosa, becoming embedded. This typically requires antibiotic treatment; however, the bacteria may become resistant to antibiotics over time, leading to antimicrobial resistance (AMR).

Glox Therapeutics’ proprietary protein bacteriocin engineering platform allows for the development of potent therapeutics that selectively eradicate drug-resistant pathogenic bacteria. These novel antibiotics do so without damaging the human microbiome, offering efficacious therapies to treat infections associated with CF.

The CDP is led by the CF AMR Syndicate, a cross-sector initiative driven by Medicines Discovery Catapult, LifeArc and Cystic Fibrosis Trust. The CF AMR Syndicate combines the expertise of leaders in CF and AMR from industry, academia and the clinic, with insights from people living with CF to accelerate CF antimicrobial drug discovery and development. This collaborative approach will allow Glox Therapeutics to benefit from expert support and resources to expedite the discovery and development of new and effective treatment options for people with CF lung infections.

Dr James Clark, CEO and Co-founder, Glox Therapeutics, said: “There is a critical need for new and effective therapeutics to tackle the rise of antimicrobial-resistant lung infections in people with CF. We are grateful to LifeArc for the CDP funding, recognising the potential of Glox Therapeutics’ unique platform to address this urgent unmet need. The collaborative support, disease insights and expert guidance of the CF AMR Syndicate will be incredibly valuable as we advance the development of our precision antibiotics to help improve the lives of patients.”

Dr Catherine Kettleborough, Head of Chronic Respiratory Infection Translational Challenge, LifeArc, said: “Collaboration is essential to unlocking new avenues in drug discovery, especially in areas like CF where AMR poses such significant challenges. Funding research through the CF AMR Syndicate will play a crucial role in catalysing scientific progress, and we’re thrilled to support Glox Therapeutics in their work.”

Dr Beverley Isherwood, Strategy Leader, Infectious Diseases, Medicines Discovery Catapult, said: “We are delighted to be working with innovative companies like Glox Therapeutics to accelerate vital research and find new treatments for people with CF. By supporting awardees through the early development phases to generate essential data packages, the CF AMR Syndicate’s CDP will position these projects to attract onward funding and investment for further development.”

Dr Paula Sommer, Head of Research and Partnerships, Cystic Fibrosis Trust, said: “For people with CF, lung infections can cause major disruptions to day-to-day life and may lead to permanent lung damage. These infections are hard to treat due to AMR, which is why we’re delighted to see the CF AMR Syndicate support these projects that will develop urgently needed new antimicrobial treatments for CF.”

For further information about Glox Therapeutics’ precision antibiotics, please visit: https://gloxtherapeutics.com/platform/

For details of the CF AMR Syndicate’s CDP, please visit: https://cfamr.org.uk/collaborative-discovery-programme/

*Worldwide rates of diagnosis and effective treatment for cystic fibrosis (J Cyst Fibros, 2022)

Notes to Editors

Please contact Codon Communications for high-resolution images.

About Glox Therapeutics www.gloxtherapeutics.com

Glox Therapeutics is developing a pipeline of engineered precision bacteriocins targeting pathogens that cause serious infections associated with high levels of antimicrobial resistance (AMR) and mortality. These novel precision antibiotics have potent narrow-spectrum activity to target Gram-negative AMR pathogens without collateral damage to the wider human microbiome.

Founded in 2023, the company is built out of over 20 years of research at the Universities of Glasgow and Oxford from globally recognised expertise in elucidating bacteriocin structure and function to treat bacterial infections. It is uniquely positioned to address the AMR public health crisis which is predicted to surpass 10 million deaths globally per year at a cost of $100 trillion dollars by 2050.

The company is supported by a highly expert network of scientific advisers and has received investment and support from high-profile investors, the Boehringer Ingelheim Venture Fund and Scottish Enterprise, as well as grant funding from PACE.

Follow Glox Therapeutics on LinkedIn GLOX Therapeutics Ltd. and X (Twitter) @GloxThera

About CF AMR Syndicate www.cfamr.org.uk

The CF AMR Syndicate is a cross-sector initiative that brings together leading experts in CF and AMR from industry, academia and the clinic with people with CF. The Syndicate’s aim is to accelerate the translation of CF antimicrobials and diagnostics to the clinic and bring new and effective treatment options to people with CF. The Syndicate was established in 2019 and is jointly managed by Medicines Discovery Catapult, Cystic Fibrosis Trust and LifeArc.

About LifeArc

LifeArc is a self-funded, not-for-profit medical research charity. We take scientific ideas out of the lab and help turn them into medical breakthroughs that can be life-changing for patients. We have been doing this for more than 25 years and our work has resulted in five licensed medicines, including cancer drug pembrolizumab (Keytruda^®), lecanemab for Alzheimer’s (Leqembi), and Carbaplex (Bruker) for diagnosing antibiotic resistance.

Our teams are experts in drug and diagnostics discovery, technology transfer, and intellectual property. Our work is in translational science — bridging the gap between academic research and clinical development, providing funding, research and expert knowledge, all with a clear and unwavering commitment to having a positive impact on patient lives.

LifeArc is a company limited by guarantee (registered in England and Wales under no. 2698321) and a charity (registered in England and Wales under no. 1015243 and in Scotland under no. SC037861).

Find out more about our work on www.lifearc.org or follow us on LinkedIn or X.

About Cystic Fibrosis Trust

Cystic Fibrosis Trust is the charity uniting people to stop cystic fibrosis (CF). We fund vital research, improve care, speak out and race towards effective treatments for all.

Cystic Fibrosis Trust is here to make sure everyone with cystic fibrosis can live without limits. Since 1964, we’ve supported people with cystic fibrosis to live longer, healthier lives — and we won’t stop until everyone can live without limits imposed by CF. Through our research goals we will accelerate progress towards a future where everyone with CF can live a life unlimited. We will develop new and improved treatments for everyone, find better ways to diagnose and treat lung infections, treat all CF symptoms throughout the body and enable people with CF to live longer, healthier lives. These goals are informed by the research priorities of the CF community.

To achieve them we will fund world class research, build effective partnerships, provide internationally recognised infrastructure and harness high quality healthcare data. People with CF will be involved in contributing to and shaping all of these activities every step of the way. Find out more and get involved: www.cysticfibrosis.org.uk

About Medicines Discovery Catapult

Medicines Discovery Catapult (MDC) is a national Life Sciences service dedicated to turning drug discovery into commercial breakthroughs.

At the frontier of drug discovery, MDC works with entrepreneurial scientists to make every move count. It validates their ideas, de-risks investments, and feeds insights back into the sector to drive productivity and impact.

MDC creates momentum through its unique blend of discovery expertise, technology, insights, and sector-leading partnerships. Where there is unmet patient need, MDC stimulates innovation through its National Programmes.

MDC has helped over 300 companies raise more than £1.2bn of R&D investment.

Its approach to drug discovery drives game-changing breakthroughs and improves patients’ lives.

More information:

• Part of the Innovate UK Catapult Network
• Based in Alderley Park, Cheshire
• For more information, visit https://md.catapult.org.uk/

Contacts

Glox Therapeutics

Dr James Clark

Email: info@gloxtherapeutics.com

Codon Communications
Dr Michelle Ricketts

Tel: +447789053885

Email: michelle.ricketts@codoncommunications.com