Stoke Therapeutics and Biogen Present Data that Further Support the Disease-Modifying Potential of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome, at the 2025 American Epilepsy Society (AES) Annual Meeting




Stoke Therapeutics and Biogen Present Data that Further Support the Disease-Modifying Potential of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome, at the 2025 American Epilepsy Society (AES) Annual Meeting

—Long-term Phase 1/2a and open label extension (OLE) data for zorevunersen on top of standard of care anti-seizure medicines (ASMs) demonstrate durable seizure reductions, including increases in seizure-free days, in addition to improvements in cognition, behavior and quality of life—

—Propensity score weighted analysis comparing the effects of zorevunersen to natural history showed reductions in seizures and improvements in cognition and behavior with dose levels and timepoints similar to and consistent with the ongoing Phase 3 EMPEROR study—

—Analysis of electroencephalogram (EEG) supports a disease-modifying mechanism of action—

BEDFORD, Mass. & CAMBRIDGE, Mass.–(BUSINESS WIRE)–Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, and Biogen Inc. (Nasdaq: BIIB) today announced data presentations that further support the potential of zorevunersen, an investigational antisense oligonucleotide, as a disease-modifying medicine for Dravet syndrome. These data were presented at the 2025 American Epilepsy Society (AES) Annual Meeting in Atlanta, Georgia.

Long-term results from the ongoing Phase 1/2a and open label extension (OLE) studies demonstrated durable seizure reductions, including increases in seizure-free days, in addition to improvements in cognition, behavior and quality of life in patients treated with zorevunersen on top of standard of care anti-seizure medicines (ASMs). A new propensity score weighted analysis provides the first direct comparison between patients with Dravet syndrome treated with zorevunersen and a matched cohort from the BUTTERFLY natural history study and showed reductions in seizures and improvements in cognition and behavior with dose levels and timepoints similar to and consistent with the ongoing Phase 3 EMPEROR study.

“Over the last four years we have gained an increasing appreciation for the potential to change the course of Dravet syndrome with new disease-modifying medicines. Open-label data from the zorevunersen clinical studies have been highly encouraging, bringing hope and anticipation to the Dravet community,” said M. Scott Perry, M.D., Head of Neurosciences and Director of the Jane and John Justin Institute for Mind Health and Medical Director of the Genetic Epilepsy Clinic at Cook Children’s Medical Center. “In addition, the new propensity score weighted analysis comparing zorevunersen treatment to patients treated with the current standard of care gives us further context for the improvements we’re seeing in patients living with this devastating disease.”

Propensity Score Weighted Analysis

A propensity score weighted analysis, designed to mimic randomization by adjusting for baseline differences between treated and untreated (natural history) patient groups, showed patients receiving two loading doses of zorevunersen (70mg) experienced statistically significant reductions in major motor seizure frequency at six months compared to natural history. Six months is consistent with the Week 28 Phase 3 primary endpoint measuring the effects of zorevunersen on seizure frequency. With continued dosing at 45mg, improvements in five key assessments of cognition and behavior as measured by Vineland-3 were shown at 18 months, with several reaching statistical significance. At the 18-month timepoint, cumulative dosing is similar to and consistent with a key secondary endpoint in the Phase 3 EMPEROR study. Durable effects were demonstrated through 24 months, the longest evaluable timepoint in the BUTTERFLY natural history study.

“Data from this analysis, which allows us to match patients treated with zorevunersen directly to natural history, add to a growing body of information shaping our understanding of Dravet syndrome and the effects of zorevunersen over time,” said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. “As we continue to progress our Phase 3 EMPEROR program and await safety and efficacy data from this larger, sham-controlled study, results presented at AES give us additional confidence in what zorevunersen may one day offer to patients and their families.”

“In addition to these important efficacy findings, we are also encouraged by the accumulating long-term safety data, with some patients treated with up to 15 doses over more than four years,” said Katherine Dawson, M.D., Head of the Therapeutics Development Unit at Biogen. “The totality of clinical data, in addition to the new EEG findings, demonstrates zorevunersen’s potential to restore protein function and address the underlying cause of Dravet syndrome to improve debilitating symptoms like seizures and cognitive impairment.”

Electroencephalogram (EEG) Results

Data from an analysis of EEGs performed in patients treated with zorevunersen highlighted the dose-dependent effects of zorevunersen in decreasing abnormal brain activity that is persistently higher in patients with Dravet syndrome. The analysis also showed that a reduction in abnormal EEG activity in the brain was associated with an increased probability of achieving a meaningful reduction in seizure frequency.

Summary of Zorevunersen Safety Data

Eighty-one patients received at least one dose of zorevunersen and have been evaluated for safety, and more than 800 doses have been administered to date. Zorevunersen has been generally well tolerated across the Phase 1/2a and OLE studies. Study drug related treatment emergent adverse events (TEAEs) were observed in 30% (24/81) and 53% (40/75) of patients treated in the Phase 1/2a and OLE studies, respectively. The most common study drug related TEAE was CSF protein elevations reported in 14% (11/81) of patients in the Phase 1/2a studies and 45% (33/75) of patients in the OLE studies. CSF protein elevations (>50 mg/dL) occurred in 42% (34/81) of patients in the Phase 1/2a studies and 86% (62/72) of patients in the OLE studies. No related clinical manifestations have been observed although one patient discontinued treatment due to elevated CSF protein levels. Treatment-emergent serious adverse events (TESAEs) were reported in 22% (18/81) and 29% (22/75) of patients in the Phase 1/2a and OLE studies, respectively, all of which were assessed to be unrelated to zorevunersen except one patient who experienced SUSARs. Three patient deaths have been reported across the Phase 1/2a and OLE studies, two from sudden unexpected death in epilepsy (SUDEP) and one from malnutrition. All were unrelated to zorevunersen.

Details of the AES Presentations:

• Title: Zorevunersen Continues to Demonstrate Potential as a Disease-modifying Therapy in Long-term Open-label Extension Studies of Patients with Dravet Syndrome
  
  Oral Presentation Date & Time: Friday, December 5, 3:30-5:55 PM EST
  
  Oral Presenter: M. Scott Perry, M.D., Head of Neurosciences and Director of the Jane and John Justin Institute for Mind Health and Medical Director of the Genetic Epilepsy Clinic at Cook Children’s Medical Center

• Title: Zorevunersen Demonstrates Disease-modifying Potential in Patients with Dravet Syndrome with Increases in Seizure-free Days, Improvements in Quality of Life, and Benefits in Overall Clinical Status
  
  Poster Presentation Date & Time: Saturday, December 6, 12:00-2:00 PM EST
  
  Poster Presenter: Kelly Knupp, M.D., MSCS, Professor of Pediatrics and Neurology at the University of Colorado Anschutz and the Dravet Program Director and Epilepsy Program Lead at Children’s Hospital Colorado
  
  Poster Number: 1.379

• Title: Zorevunersen Continues to Demonstrate Potential as a Disease-modifying Therapy in Long-term Open-label Extension Studies of Patients with Dravet Syndrome
  
  Poster Presentation Date & Time: Sunday, December 7, 12:00-2:00 PM EST
  
  Poster Presenter: M. Scott Perry, M.D., Head of Neurosciences and Director of the Jane and John Justin Institute for Mind Health and Medical Director of the Genetic Epilepsy Clinic at Cook Children’s Medical Center
  
  Poster Number: 2.341

• Title: Spectral Electroencephalogram Abnormalities Across Development in Patients with Dravet Syndrome
  
  Poster Presentation Date & Time: Sunday, December 7, 12:00-2:00 PM EST
  
  Poster Presenter: Pieter van Mierlo, Founder and Chief Scientific Officer, Epilog, Clouds of Care, Associate Professor, Ghent University
  
  Poster Number: 2.432

• Title: Electrophysiological Improvements in Patients with Dravet Syndrome Following Treatment with Zorevunersen, an Investigational Antisense Oligonucleotide
  
  Poster Presentation Date & Time: Monday, December 8, 12:00-1:45 PM EST
  
  Poster Presenter: Nigel Colenbier, Senior Data Scientist, Epilog, Clouds of Care
  
  Poster Number: 3.489

About Dravet Syndrome

Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by recurrent seizures as well as significant cognitive and behavioral impairments. Most cases of Dravet are caused by mutations in one copy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in neuronal cells in the brain. Even when treated with the best available anti-seizure medicines (ASMs), up to 57 percent of patients with Dravet syndrome do not achieve ≥50 percent reduction in seizure frequency. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. Developmental and cognitive impairments often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP; up to 20 percent of children and adolescents with Dravet syndrome die before adulthood due to SUDEP, prolonged seizures, seizure-related accidents or infections¹. Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan. ² There are no approved disease-modifying therapies for people living with Dravet syndrome.

About Zorevunersen

Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing functional NaV1.1 protein production in brain cells from the non-mutated (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition, and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights.

About the EMPEROR Study

The EMPEROR Phase 3 Study (NCT06872125) is a global, double-blind, sham-controlled study evaluating the efficacy, safety, and tolerability of zorevunersen in children ages 2 to <18 with Dravet syndrome with a confirmed variant in the SCN1A gene not associated with gain-of-function. The trial is currently enrolling patients within the United States and is expected to enroll participants across Japan, United Kingdom and European Union, with participants being randomized 1:1 to receive either zorevunersen via intrathecal administration or a sham comparator for a 52-week treatment period following an 8-week baseline period. Following the completion of the study, eligible participants will be offered ongoing treatment with zorevunersen as part of an OLE study. The primary endpoint of the study is percent change from baseline in major motor seizure frequency at week 28 in patients receiving zorevunersen as compared to sham. The key secondary endpoints are the durability of effect on major motor seizure frequency and improvements in behavior and cognition as measured by Vineland-3 subdomains, including expressive communication, receptive communication, interpersonal relationships, coping skills and personal skills. Additional endpoints include safety, Clinician Global Impression of Change (CGI-C), Caregiver Global Impression of Change (CaGI-C), and the Bayley Scales of Infant Development (BSID-IV). For more information, visit https://www.emperorstudy.com/.

About Stoke Therapeutics

Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine. Using Stoke’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke’s first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke’s initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke’s proprietary approach. Stoke is headquartered in Bedford, Massachusetts. For more information, visit https://www.stoketherapeutics.com/.

About Biogen

Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients’ lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth.

We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media – Facebook, LinkedIn, X, YouTube.

Stoke Therapeutics Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the ability of zorevunersen to treat the underlying causes of Dravet syndrome and reduce seizures or show improvements in behavior and cognition at the indicated dosing levels or at all; the potential benefits, safety and efficacy of zorevunersen; the timing and expected progress of clinical trials, data readouts, regulatory meetings, regulatory decisions and other presentations; and the participation of scientists associated with Stoke making presentations at AES 2025 and the presentation of data at AES 2025. Statements including words such as “plan,” “potential,” “will,” “continue,” “expect,” or similar words and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they prove incorrect or do not fully materialize, could cause Stoke’s results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: Stokes ability to advance, obtain regulatory approval and ultimately commercialize its product candidates; that if Biogen were to breach or terminate the collaboration, Stoke would not obtain the anticipated financial or other benefits; the possibility that Stoke and Biogen may not be successful in their development of zorevunersen and that, even if successful, they may be unable to successfully commercialize zorevunersen; positive results in a clinical trial may not be replicated in subsequent trials or successes in early stage clinical trials may not be predictive of results in later stage trials; Stoke’s ability to protect its intellectual property; Stoke’s ability to fund development activities and achieve development goals to mid-2028; and the other risks and uncertainties described under the heading “Risk Factors” in its Annual Report on Form 10-K for the year ended December 31, 2024, its quarterly reports on Form 10-Q, and the other documents it files with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and Stoke undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.

Biogen Safe Harbor

This news release contains forward-looking statements, including, among others, relating to: the potential clinical effects of zorevunersen; the potential for zorevunersen to improve outcomes and for patients of Dravet syndrome; the potential benefits, safety and efficacy of zorevunersen and continued treatment with zorevunersen; potential regulatory discussions, submissions and approvals and the timing thereof; the treatment of Dravet syndrome; the anticipated benefits, risks and potential of Biogen’s collaboration arrangements with Stoke Therapeutics; the potential of Biogen’s commercial business and pipeline programs, including zorevunersen; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “forecast,” “goal,” “guidance,” “hope,” “intend,” “may,” “objective,” “outlook,” “plan,” “possible,” “potential,” “predict,” “project,” “prospect,” “should,” “target,” “will,” “would” or the negative of these words or other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements.

These forward-looking statements are based on management’s current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward-looking statements will be realized in whole or in part. We caution that these statements are subject to risks and uncertainties, many of which are outside of our control and could cause future events or results to differ materially from those stated or implied in this document, including, among others, uncertainty of our long-term success in developing, licensing, or acquiring other product candidates or additional indications for existing products; expectations, plans, prospects and timing of actions relating to product approvals, approvals of additional indications for our existing products, sales, pricing, growth, reimbursement and launch of our marketed and pipeline products; the potential impact of increased product competition in the biopharmaceutical and healthcare industry, as well as any other markets in which we compete, including increased competition from new originator therapies, generics, prodrugs and biosimilars of existing products and products approved under abbreviated regulatory pathways; our ability to effectively implement our corporate strategy; difficulties in obtaining and maintaining adequate coverage, pricing, and reimbursement for our products; the drivers for growing our business, including our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks related to commercialization of biosimilars, which is subject to such risks related to our reliance on third-parties, intellectual property, competitive and market challenges and regulatory compliance; the risk that positive results in a clinical trial may not be replicated in subsequent or confirmatory trials or success in early stage clinical trials may not be predictive of results in later stage or large scale clinical trials or trials in other potential indications; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; and the occurrence of adverse safety events, restrictions on use with our products, or product liability claims; and any other risks and uncertainties that are described in other reports we have filed with the U.S. Securities and Exchange Commission, which are available on the SEC’s website at www.sec.gov.

These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our subsequent reports on Form 10-Q. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise.

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From time to time, we have used, or expect in the future to use, our investor relations website (investors.biogen.com), the Biogen LinkedIn account (linkedin.com/company/biogen-) and the Biogen X account (https://x.com/biogen) as a means of disclosing information to the public in a broad, non-exclusionary manner, including for purposes of the SEC’s Regulation Fair Disclosure (Reg FD). Accordingly, investors should monitor our investor relations website and these social media channels in addition to our press releases, SEC filings, public conference calls and websites, as the information posted on them could be material to investors.

References:

1. Symonds, J. et al. Early childhood epilepsies: epidemiology, classification, aetiology, and socio-economic determinants. Brain. 2021;144(9):2879-2891.
2. Based on Stoke Therapeutics’ preliminary estimates, which scaled annual incidence to prevalence using country-specific live birth rates over the past 85 years and adjusted for Dravet-specific mortality.

Contacts

Stoke Media & Investor Contacts:
Susan Willson

Vice President, Corporate Communications

swillson@stoketherapeutics.com
415-509-8202

Doug Snow

Director, Communications & Investor Relations

IR@stoketherapeutics.com
508-642-6485

Biogen Media Contact:
Madeleine Shin

Public.affairs@biogen.com
+ 1 781 464 3260

Biogen Investor Contact:
Tim Power

IR@biogen.com
+1 781 464 2442

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Amylyx Pharmaceuticals Announces New Safety and Tolerability Cohort 1 Data of AMX0114 in ALS from First-in-Human LUMINA Trial




Amylyx Pharmaceuticals Announces New Safety and Tolerability Cohort 1 Data of AMX0114 in ALS from First-in-Human LUMINA Trial

– AMX0114 was generally well-tolerated, with no treatment-related serious adverse events

– Amylyx will proceed with opening enrollment of second cohort

– Data are being presented at the 36^th International Symposium on ALS/MND

CAMBRIDGE, Mass.–(BUSINESS WIRE)–$AMLX—Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced the presentation of early safety and tolerability data from its Phase 1 LUMINA trial of AMX0114 and results from ongoing work characterizing biomarkers of AMX0114 target engagement at the 36^th International Symposium on ALS/MND (MNDA) held from December 5-7 in San Diego, California. AMX0114 was generally well-tolerated in LUMINA trial participants enrolled in cohort 1 (n=12), with no treatment-related serious adverse events (SAEs). Based on these data, Amylyx expects to begin enrolling the second cohort of participants in Canada later this month and in the U.S. in January.

“LUMINA is a first-in-human study, and we are encouraged by these data as we continue to advance AMX0114 as a potential treatment for this rapidly progressive disease with high unmet need. The safety and tolerability analysis allows LUMINA to proceed with its next cohort of participants, which is critical given that this community has no time to wait,” said Sabrina Paganoni, MD, PhD, principal investigator of the LUMINA trial, investigator at the Sean M. Healey & AMG Center for ALS at Mass General Brigham, and member of the Scientific Advisory Board of the Network of Excellence for ALS (NEALS).

The LUMINA trial (NCT06665165) is a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose clinical trial of AMX0114 – an investigational, potent antisense oligonucleotide (ASO) targeting calpain-2 – in people living with amyotrophic lateral sclerosis (ALS). LUMINA is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114 in people living with ALS and assessing both novel and broadly researched ALS biomarkers, including change from baseline in neurofilament light chain (NfL) levels. Amylyx expects biomarker data from the first cohort of LUMINA participants (n=12) in the first half of 2026.

“We appreciate the partnership with LUMINA sites and participants to achieve complete enrollment of the first cohort. In addition, we are pleased that to date no drug-related SAEs or dose-limiting toxicities were observed, which represent important early steps in this study,” said Camille L. Bedrosian, MD, Chief Medical Officer at Amylyx. “AMX0114 is designed to inhibit calpain-2, a calcium-activated protease that is one of the fundamental drivers of axonal degeneration and consequent disease progression in ALS. Preclinical studies have demonstrated that treatment with AMX0114 resulted in potent, dose-dependent, and durable reduction in calpain-2 protein levels, translating to improved neuronal survival and reductions in extracellular NfL levels. We look forward to presenting cohort 1 biomarker data at a medical meeting in the first half of next year.”

The MNDA presentation details are as follows:

Friday, December 5, 2025, Session A, 5:30-7pm Pacific Time:

Title: A Phase 1, Multicenter, Randomized, Placebo-Controlled Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of AMX0114 in Amyotrophic Lateral Sclerosis (LUMINA)

Presenting Author: Sabrina Paganoni, MD, PhD, Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Spaulding Rehabilitation Hospital, Harvard Medical School

Poster Number: CLT-14

Saturday, December 6, 2025, Session 6B: Biomarkers, 10:50-11:10am Pacific Time:

Title: Characterizing the CSF Biomarker Signature of Calpain-2 Activity in ALS and the Biomarker Impact of Calpain-2 Inhibition in a Preclinical Model of ALS

Presenting Author: Robert Bowser, PhD, Barrow Neurological Institute and nVector, Inc.

For MNDA conference information, visit https://symposium.mndassociation.org/. Abstracts have been published as an online open access supplement of Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. The presentations will be available on the “Events & Presentations” tab of the Amylyx website.

About AMX0114

AMX0114 is an investigational antisense oligonucleotide (ASO) with U.S. Food and Drug Administration (FDA) Fast Track designation for the potential treatment of ALS. AMX0114 targets calpain-2 (CAPN2), a calcium-activated protease that is one of the fundamental drivers of axonal degeneration and consequent disease progression in ALS. In preclinical studies, treatment with AMX0114 resulted in potent, dose-dependent, and durable reduction in CAPN2 mRNA and calpain-2 protein levels in disease-relevant cell models of axonal degeneration. This translated to improved neuronal survival, including in a model of TDP-43 ALS, and reductions in extracellular neurofilament light (NfL) levels across multiple disease models and paradigms of neuronal injury. AMX0114 was generally well-tolerated in LUMINA trial participants enrolled in cohort 1 (n=12), with no treatment-related serious adverse events (SAEs).

About LUMINA

The Phase 1 LUMINA clinical trial (NCT06665165) is a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose trial evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114 in people living with ALS. LUMINA will also assess change from baseline in calpain-2 levels, neurofilament light (NfL) levels, and other pharmacodynamic biomarkers of ALS. LUMINA is anticipated to enroll approximately 48 adult participants. Participants will be randomized 3:1 to receive AMX0114 or placebo by intrathecal administration once every four weeks for a total of up to 4 doses. Study sites are active in Canada and the United States, and the first cohort has been fully enrolled. Amylyx expects to begin enrolling the second cohort of participants in Canada in December and in the U.S. in January.

About ALS

Amyotrophic lateral sclerosis (ALS, also known as motor neuron disease) is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. One of the ways ALS progresses is through axonal degeneration, which disrupts neural connectivity and contributes significantly to disease pathology. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually, death. More than 90% of people with ALS have sporadic disease, showing no clear family history.

About Amylyx Pharmaceuticals

At Amylyx, our mission is to usher in a new era of treating diseases with high unmet needs. Where others see challenges, we see opportunities that we pursue with urgency, rigorous science, and unwavering commitment to the communities we serve. We are currently focused on three investigational therapies across several neurodegenerative and endocrine diseases in which we believe they can make the greatest impact. For more information, visit amylyx.com and follow us on LinkedIn and X. For investors, please visit investors.amylyx.com.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, Amylyx’ expectations regarding: the potential for AMX0114 as a treatment for ALS, the expected timeline for enrollment of future cohorts, and the expected timeline for data readout. Any forward-looking statements in this press release and related comments in the Company’s earnings conference call are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx’ program development activities; Amylyx’ ability to execute on its regulatory development plans and expectations regarding the timing of results from its planned data announcements and initiation of clinical studies; the risk that early-stage results may not reflect later-stage results; Amylyx’ ability to fund operations, and the impact that global macroeconomic uncertainty, geopolitical instability, and public health events will have on Amylyx’ operations, as well as the risks and uncertainties set forth in Amylyx’ United States Securities and Exchange Commission (SEC) filings, including Amylyx’ Annual Report on Form 10-K for the year ended December 31, 2024, and subsequent filings with the SEC. All forward-looking statements contained in this press release and related comments in our earnings conference call speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Contacts

Media
Amylyx Media Team

(857) 799-7274

amylyxmediateam@amylyx.com

Investors
Lindsey Allen

(857) 320-6244

Investors@amylyx.com

Actio Biosciences Announces Preclinical Data Highlighting Ability of ABS-1230 to Potently and Selectively Inhibit KCNT1 and Significantly Reduce Seizure Activity




Actio Biosciences Announces Preclinical Data Highlighting Ability of ABS-1230 to Potently and Selectively Inhibit KCNT1 and Significantly Reduce Seizure Activity

Data to be Presented During an Oral Platform Presentation at the American Epilepsy Society Annual Meeting

SAN DIEGO–(BUSINESS WIRE)–Actio Biosciences, a clinical-stage biotechnology company advancing the translation of genetic insights into novel precision medicines, today announced preclinical data related to the company’s co-lead candidate ABS-1230, a selective small molecule KCNT1 inhibitor for the treatment of KCNT1-related epilepsy. The data are being presented today, December 5, in a platform presentation by Actio CSO David Breckenridge, Ph.D., from 3:30-5:45 p.m. at the American Epilepsy Society (AES) Annual Meeting in Atlanta.

The preclinical data support the therapeutic potential of ABS-1230 to reduce seizures by selectively inhibiting mutations of the KCNT1 gene, which cause overactivation of the potassium channel and abnormal electrical brain activity, leading to a severe, early-onset epileptic encephalopathy. Actio is currently advancing ABS-1230 in a Phase 1a healthy volunteer clinical trial and plans to initiate a Phase 1b/2a clinical trial in patients with KCNT1-related epilepsy in 2026.

“Development of treatments for KCNT1-related epilepsy has been limited by the large number of distinct pathogenic mutations, many of which are resistant to known inhibitors,” said David Goldstein, Ph.D., CEO of Actio. “In our preclinical work, ABS-1230 has consistently demonstrated its ability to potently and selectively inhibit KCNT1 and pathogenic mutations leading to notable reductions in seizures in a mouse model. We’re pleased to share these data today, which support ABS-1230 as a targeted therapy with the potential to address the immense burden and unmet need for patients with KCNT1-related epilepsy. Our Phase 1a trial of ABS-1230 in healthy volunteers is ongoing, and we look forward to reporting data from that study in early 2026 and advancing into a first-in-patient trial next year.”

Presentation Highlights

In preclinical human and mouse models, administration of ABS-1230 led to:

• Potent and selective inhibition of KCNT1 and known pathogenic mutations
• High oral bioavailability and brain penetration across species
• Significant dose-dependent reduction of seizures in a KCNT1 mouse model relative to vehicle control treatment

About ABS-1230

ABS-1230 is designed to be a potent and selective orally available small molecule inhibitor of the KCNT1 ion channel and is initially in clinical development for the treatment of KCNT1-related epilepsy. The KCNT1 gene provides instructions for making potassium channels that allow potassium ions to flux out of cells, especially neurons in the brain. Gain-of-function mutations in the KCNT1 gene are the known genetic cause of KCNT1-related epilepsy — a rare, severe and often fatal form of pediatric epilepsy. The company has initiated a Phase 1a trial of ABS-1230 in healthy adult volunteers. Actio is also conducting preclinical studies to evaluate the therapeutic potential of ABS-1230 in more prevalent genetic epilepsies with related underlying biology. The FDA has granted ABS-1230 Rare Pediatric Disease, Fast Track and Orphan Drug designations for the treatment of KCNT1-related epilepsy.

About KCNT1-related Epilepsy

KCNT1-related epilepsy is a rare and often fatal pediatric developmental epileptic encephalopathy with a U.S. prevalence Actio estimates to be 2,500 patients. KCNT1-related epilepsy patients typically have a normal prenatal course before developing sporadic, asynchronous focal seizures. Seizure onset is often in the first six months of life, and the median age of onset is approximately 3.5 weeks. Seizure frequency tends to increase over time and can reach up to 50 seizures per day (15-20 per day on average). Other common symptoms and complications of KCNT1-related epilepsy include developmental delays and profound cognitive impairments and neurological deficits. There are currently no approved therapies for KCNT1-related epilepsy.

About Actio Biosciences

Actio Biosciences is a clinical-stage biotechnology company advancing the translation of genetic insights into novel precision medicines. The company applies deep expertise in Mendelian genetics, drug discovery and data science to identify targets whose biology underlies rare diseases with high unmet need and holds promise for the treatment of more prevalent indications. By precisely targeting the root causes of rare diseases, Actio generates biological insights to enable expansion of development into more prevalent indications.

The company is advancing two lead clinical-stage programs, ABS-1230, initially being developed for the treatment of KCNT1-related epilepsy, and ABS-0871, initially being developed for the treatment of Charcot-Marie-Tooth disease type 2C including other TRPV4-related neuromuscular disorders (collectively, CMT2C). Actio plans to pursue development of both lead programs in more prevalent indications such as additional genetic epilepsies for ABS-1230 and overactive bladder for ABS-0871. In addition to Actio’s two lead programs, Actio has also launched a third program for a rare genetic epilepsy. For more information, please visit ActioBiosciences.com and follow the company on LinkedIn and X.

Contacts

Contact
Katie Engleman, 1AB

katie@1abmedia.com

Investor Contact:
Renee Leck, THRUST

renee@thrustsc.com

Zoetis Receives Health Canada Approval for Portela™(relfovetmab injection) to Alleviate Pain Associated with Osteoarthritis in Cats




Zoetis Receives Health Canada Approval for Portela™(relfovetmab injection) to Alleviate Pain Associated with Osteoarthritis in Cats

PARSIPPANY, N.J.–(BUSINESS WIRE)–$ZTS #animalhealth—Zoetis Inc. today announced that Health Canada has approved Portela^™ (relfovetmab injection) for the alleviation of pain associated with osteoarthritis (OA) in cats. Designed to provide three months of OA pain relief with a single injection, Portela targets anti-nerve growth factor (NGF), which is a key mediator of OA pain and inflammation.

In a nine-month field trial in Europe, Portela demonstrated effectiveness in alleviating OA pain and was found to be well tolerated, including by cats identified with kidney disease at IRIS stage 1, 2 or 3¹. Portela is also approved in the European Union (EU), and Zoetis anticipates making Portela commercially available in Canada and the EU in 2026.

“Health Canada’s approval of Portela marks a significant step forward in managing osteoarthritis-related pain in cats,” said Rob Polzer, Ph.D., Executive Vice President and President, Research and Development at Zoetis. “Thanks to Portela’s long-lasting antibody and its unique binding site to NGF, veterinarians and cat owners in Canada now have a new, innovative way to address osteoarthritis pain. This achievement underscores our ongoing dedication to developing new therapies that improve the health and quality of life for companion animals.”

Osteoarthritis as a Significant Unmet Medical Need

OA is a common, chronic and progressive joint disease characterized by the inflammation and breakdown of joints, leading to pain and mobility issues. Although the disease cannot be cured, much can be done to control the associated pain and improve quality of life. Even with up to 40% of all cats having clinical signs of OA^{2 3}, pet owners often overlook these signs, and as a result only 18% of affected cats are diagnosed with OA pain by veterinary professionals⁴. Identifying and treating OA pain is important because chronic pain has a negative impact on many aspects of a cat’s health. In addition to gait and movement, chronic pain impacts sleep, relationships, and cognition⁵. Portela now provides a convenient, long-acting therapy to reduce OA pain – particularly beneficial for cats that are difficult to medicate regularly.

Building on a Legacy of Innovation

Zoetis continues to advance care for animals around the globe with a robust pipeline fueled by lifecycle innovation, geographic expansion and new product innovation. Portela joins Solensia^® (frunevetmab injection) in the company’s growing portfolio of OA pain products for cats. Like Solensia, Portela is a monoclonal antibody that targets nerve growth factor (NGF); however, Portela is designed to alleviate pain associated with OA for a longer period of time by binding to a different site on NGF.

“Portela is a new therapy designed to support long-term comfort and mobility for cats—with the added benefit that pet owners only need to bring their cats in for an injection once every three months. This is a testament to the value we place at Zoetis on meaningful innovation in feline medicine, solving for the unmet needs of our patients and their families,” said Richard Goldstein, DVM, DACVIM, DECVIM-CA, Global Chief Medical Officer and Head of Medical Affairs at Zoetis. “We look forward to providing veterinarians with another innovative tool, adding to our wide feline portfolio, to help them improve the quality of life for the cats in their care.”

About Portela

Portela (relfovetmab injection) is a monoclonal antibody therapy that binds to nerve growth factor to reduce NGF’s effects. Portela is indicated for the alleviation of pain associated with osteoarthritis in cats. Portela is administered subcutaneously once every three months. Portela should not be used in cats with known hypersensitivity to relfovetmab, or to any of the excipients; or in breeding, pregnant or lactating cats, or in cats less than 12 months of age. The following clinical signs have been reported in cats receiving Portela: pain upon injection, skin infection, dermatitis, hair loss, and anorexia.

About Zoetis

As the world’s leading animal health company, Zoetis is driven by a singular purpose: to nurture our world and humankind by advancing care for animals. After innovating ways to predict, prevent, detect, and treat animal illness for more than 70 years, Zoetis continues to stand by those raising and caring for animals worldwide – from veterinarians and pet owners to livestock producers. The company’s leading portfolio and pipeline of medicines, vaccines, diagnostics and technologies make a difference in over 100 countries. A Fortune 500 company, Zoetis generated revenue of $9.3 billion in 2024 with approximately 13,800 employees. For more information, visit www.zoetis.com.

DISCLOSURE NOTICES

Forward-Looking Statements: This press release contains forward-looking statements which reflect the current views of Zoetis with respect to: our business plans or prospects; expectations regarding products, product approvals or licenses, products under development; and other future events. These statements are not guarantees of future performance or actions. Forward-looking statements are subject to risks and uncertainties. If one or more of these risks or uncertainties materialize, or if management’s underlying assumptions prove to be incorrect, actual results may differ materially from those contemplated by a forward-looking statement. Forward-looking statements speak only as of the date on which they are made. Zoetis expressly disclaims any obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise. A further list and description of risks, uncertainties and other matters can be found in our most recent Annual Report on Form 10-K, including in the sections thereof captioned “Forward-Looking Statements and Factors That May Affect Future Results” and “Item 1A. Risk Factors,” in our Quarterly Reports on Form 10-Q and in our Current Reports on Form 8-K. These filings and subsequent filings are available online at www.sec.gov, www.zoetis.com, or on request from Zoetis.

All trademarks are the property of Zoetis Services LLC or a related company or a licensor unless otherwise noted.

1. IRIS stages of kidney disease. Accessed August 21, 2025. https://static1.squarespace.com/static/666b9ecb4064a156963b4162/t/66a6dbc90ca6986e1b5c06bd/1722211273243/2_IRIS_Staging_of_CKD_2023.pdf
2. Enomoto M, Mantyh PW, Murrell J, Innes JF, Lascelles BDX. Anti-nerve growth factor monoclonal antibodies for the control of pain in dogs and cats. Vet Rec. 2019;184(1):23.
3. L.I. Slingerland, H.A.W. Hazewinkel, B.P. Meij, Ph. Picavet, G. Voorhout, Cross-sectional study of the prevalence and clinical features of osteoarthritis in 100 cats. The Veterinary Journal, Volume 187, Issue 3, 2011, Pages 304-309.
4. Solensia Veterinarian A&U MR (MARC 2025)
5. Lascelles BDX, Brown DC, Conzemius MG, Gill M, Oshinsky ML, Sharkey M. Measurement of chronic pain in companion animals: discussions from the Pain in Animals Workshop (PAW) 2017. Vet J. 2019;250(8):71-78.

ZTS-COR

ZTS-IR

ZTS-CA

ZTS-PS

Contacts

Media Contacts:

Jennifer Albano

862-399-0810 (o)

jennifer.albano@zoetis.com

Laura Panza

973-975-5176 (o)

laura.panza@zoetis.com

Investor Contacts:

Steve Frank

973-822-7141 (o)

steve.frank@zoetis.com

Nick Soonthornchai

973-443-2792 (o)

nick.soonthornchai@zoetis.com

Guardant Health to Present 14 Abstracts Demonstrating the Power of Multiomic Liquid Biopsy Tests in Predicting Outcomes and Tailoring Treatment for Early and Metastatic Breast Cancer at the 2025 San Antonio Breast Cancer Symposium




Guardant Health to Present 14 Abstracts Demonstrating the Power of Multiomic Liquid Biopsy Tests in Predicting Outcomes and Tailoring Treatment for Early and Metastatic Breast Cancer at the 2025 San Antonio Breast Cancer Symposium

• Presentations highlight advancements in cancer recurrence detection with Guardant Reveal and real-time treatment response monitoring with Guardant360 Liquid

PALO ALTO, Calif.–(BUSINESS WIRE)–$GH–Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, will present a total of 14 abstracts with its research collaborators from multiple studies demonstrating the value of its tissue-free liquid biopsy tests at the 2025 San Antonio Breast Cancer Symposium (SABCS), taking place Dec. 9–12, 2025.

Oral presentations will highlight results generated with Guardant Reveal, the company’s tissue-free test for detecting minimal residual disease (MRD) and Guardant360 Liquid, the company’s comprehensive multiomic profiling test, both leveraging Guardant’s proprietary epigenomic technology powered by the Guardant Infinity Smart Platform. Collectively, these studies demonstrate how blood-based testing can uncover risk, guide treatment selection, and influence long-term patient management across the breast cancer continuum, from early-stage disease through metastatic settings.

Guardant’s MRD presentations reinforce the value of tissue-free circulating tumor DNA (ctDNA) testing for both neoadjuvant treatment response assessment and post-treatment surveillance in early-stage breast cancer. In a retrospective analysis of HER2-positive patients, ctDNA measured with Guardant Reveal demonstrated strong prognostic significance in the neoadjuvant setting, showing a correlation with pathological complete response and three-year invasive disease-free survival. In an independent study of triple-negative breast cancer patients, Guardant Reveal accurately predicted relapse risk and detected ctDNA more frequently and earlier than orthogonal methods.

Together, these findings demonstrate the validity of Guardant Reveal as a reliable approach for ctDNA detection and monitoring in both neoadjuvant and surveillance settings.

“Breast cancer care increasingly depends on understanding tumor biology at every stage of disease, and Guardant’s data continues to reaffirm the growing value of multiomic liquid biopsy as a powerful, non-invasive tool to equip providers with the data necessary to tailor therapy and treatment,” said Dr. Craig Eagle, Chief Medical Officer at Guardant Health. “We look forward to sharing our findings demonstrating the power of blood in helping provide clinicians with deeper clarity and more confident decision-making for their patients.”

Other data highlights to be presented include:

• Data showcasing the clinical utility of Guardant Reveal in predicting outcomes in patients with early-stage HER2-positive breast cancer. In this analysis, early ctDNA clearance throughout treatment was linked to better outcomes, underscoring ctDNA as a sensitive marker of treatment response and highlighting the value of tissue-free testing for real-time response monitoring.
• Data supporting the clinical value potential of non-invasive, methylation-based, continuous breast cancer subtype monitoring by Guardant360 Liquid to uncover tumor evolution and heterogeneity that may guide more precise treatment decisions. In this analysis, Guardant360 Liquid showed agreement to tissue-based monitoring and replicated previously reported accuracy.
• Data showcasing Guardant360 Liquid methylation-based breast cancer subtyping feature as a noninvasive liquid tool to dynamically reassess ER, HER2 and triple negative status in patients who are resistant to ER therapy but don’t carry an ESR1 mutation. In this study, Guardant360 Liquid predicted real-world outcomes in previously AI-treated patients receiving subsequent lines of therapy.

Guardant-Led Research at SABCS 2025

The full abstracts for Guardant Health and a list of all abstracts being presented at SABCS 2025 can be found on the SABCS website.

For information and updates from the conference, visit booth #942 and follow Guardant Health on LinkedIn, X (Twitter) and Facebook.

About Guardant Health

Guardant Health is a leading precision oncology company focused on guarding wellness and giving every person more time free from cancer. Founded in 2012, Guardant is transforming patient care and accelerating new cancer therapies by providing critical insights into what drives disease through its advanced blood and tissue tests, real-world data and AI analytics. Guardant tests help improve outcomes across all stages of care, including screening to find cancer early, monitoring for recurrence in early-stage cancer, and treatment selection for patients with advanced cancer. For more information, visit guardanthealth.com and follow the company on LinkedIn, X (Twitter) and Facebook.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of federal securities laws, including statements regarding the potential utilities, values, benefits and advantages of Guardant Health’s liquid biopsy tests or assays, which involve risks and uncertainties that could cause the actual results to differ materially from the anticipated results and expectations expressed in these forward-looking statements. These statements are based on current expectations, forecasts and assumptions, and actual outcomes and results could differ materially from these statements due to a number of factors. These and additional risks and uncertainties that could affect Guardant Health’s financial and operating results and cause actual results to differ materially from those indicated by the forward-looking statements made in this press release include those discussed under the captions “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operation” and elsewhere in its Annual Report on Form 10-K for the year ended December 31, 2024, and any current and periodic reports filed with or furnished to the Securities and Exchange Commission thereafter. The forward-looking statements in this press release are based on information available to Guardant Health as of the date hereof, and Guardant Health disclaims any obligation to update any forward-looking statements provided to reflect any change in its expectations or any change in events, conditions, or circumstances on which any such statement is based, except as required by law. These forward-looking statements should not be relied upon as representing Guardant Health’s views as of any date subsequent to the date of this press release.

Contacts

Investor Contact:
Zarak Khurshid

investors@guardanthealth.com

Media Contact:
Meaghan Smith

press@guardanthealth.com

Natera Acquires Foresight Diagnostics




Natera Acquires Foresight Diagnostics

Expands Natera’s lead in solid tumor MRD, acquiring ultrasensitive phased variant technology with LOD95 of 0.3 parts per million (ppm) and detection below 0.1 ppm¹

Cutting-edge IP adds to Natera’s portfolio of >500 issued or pending patents

Accelerates Natera’s expansion into lymphoma, where Foresight has developed a strong clinical position

AUSTIN, Texas & BOULDER, Colo.–(BUSINESS WIRE)–Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA and precision medicine, and Foresight Diagnostics, a leader in ultrasensitive molecular residual disease (MRD) detection, today announced that Natera has completed a transaction to acquire Foresight.

Foresight is a cancer diagnostics company and CLIA-registered laboratory. The company’s circulating tumor DNA (ctDNA)-based MRD tests leverage its patented PhasED-Seq™ technology, targeting phased variants. With this technology, Foresight has reported performance with LOD95 of 0.3 parts per million and detection below 0.1 ppm¹.

Foresight was founded by Stanford University physicians and scientists, Maximilian Diehn, M.D., Ph.D., Ash Alizadeh, M.D., Ph.D., and David Kurtz, M.D., Ph.D., together with Jake Chabon, Ph.D., Foresight’s chief scientific officer and chief executive officer. The company has authored more than 40 scientific publications and presentations and partnered with more than 30 biopharma and academic researchers.

Strategic Rationale

The transaction combines Natera’s leading commercial and operational infrastructure for the delivery of personalized MRD testing with Foresight’s unique phased variant technology and leadership in lymphoma. It builds on Natera’s broad intellectual property portfolio for tumor-informed and personalized MRD products including in phased variants, and promises to accelerate MRD adoption in lymphoma and other solid tumor types.

• Signatera™ platform with phased variants: The integration of phased variants into the Signatera platform will further differentiate and strengthen test performance across solid tumors. This enhanced version is available immediately for research use for biopharma and academic partners and is expected to be launched for clinical use in 2026.
• Leadership in lymphoma: The transaction builds on Foresight’s clinical research momentum in B-cell lymphomas, a large patient population with more than 75,000 new cases annually in the U.S.² Earlier this year, Foresight data provided the foundation for the inclusion of ctDNA MRD into the National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines for diffuse large B-cell lymphoma. Additionally, Foresight’s CLARITY™ MRD assay for lymphoma is being used in three prospective MRD-driven clinical trials informing treatment decisions for patients. Foresight CLARITY joins Natera’s extensive MRD product portfolio and will continue to support clinical trials, translational research and future applications.

At the American Society of Hematology (ASH) Annual Meeting beginning on December 6, Natera and Foresight will have a total of 15 abstracts featuring Signatera and PhasED-Seq, including seven oral presentations.

Transaction Terms

Natera has closed the acquisition of Foresight in an all-stock transaction consisting of a $275 million upfront with an additional $175 million in earnouts tied to the achievement of revenue- and reimbursement-based milestones.

“This acquisition reinforces Natera’s position at the forefront of precision oncology,” said Steve Chapman, chief executive officer of Natera. “Foresight’s phased variant technology and leadership in lymphoma complement Natera’s strong capabilities in personalized MRD testing, improving the value we can deliver to patients, clinicians, biopharma partners and the broader healthcare system.”

“Foresight’s mission has always been to improve the lives of cancer patients worldwide through innovative diagnostics,” said Chabon. “As we join Natera, I’m deeply grateful to our employees, partners and investors who have helped bring us to this moment. Together, we can realize this mission on a far greater scale, accelerating the pace of discovery across both hematologic and solid tumors.”

Gibson, Dunn & Crutcher LLP is serving as legal counsel to Natera. Wilson Sonsini Goodrich & Rosati is serving as legal counsel to Foresight, while Centerview Partners LLC is acting as its financial advisor.

References

1. Cabel L, Jeon YJ, Parikh AR, et al. Ultrasensitive Phased Variant Detection Enables Improved ctDNA-Based MRD Assessment Across Solid Tumors. Presented at: European Society for Medical Oncology (ESMO) Annual Meeting; 2024.
2. American Cancer Society. Types of B-cell Lymphoma. American Cancer Society. https://www.cancer.org/cancer/types/non-hodgkin-lymphoma/about/b-cell-lymphoma.html. Accessed November 26, 2025.

About Natera

Natera™ is a global leader in cell-free DNA and precision medicine, 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 supported by more than 325 peer-reviewed publications that demonstrate excellent performance. 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, including those regarding the expected benefits of the Foresight acquisition, integration of technologies, clinical and commercial opportunities, and future performance, represent Natera’s expectations as of the date of this press release, and are based on current expectations and are subject to risks and uncertainties that could cause actual results to differ materially, including the ability to integrate Foresight’s business, realize anticipated benefits, achieve expected clinical or commercial outcomes, obtain or maintain reimbursement coverage, and respond to competitive or regulatory developments. 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., investor@natera.com
Media: Lesley Bogdanow, VP of Corporate Communications, Natera, Inc., pr@natera.com

Viridian Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)




Viridian Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

WALTHAM, Mass.–(BUSINESS WIRE)–Viridian Therapeutics, Inc. (Nasdaq: VRDN), a biotechnology company focused on discovering, developing, and commercializing potentially best-in-class medicines for serious and rare diseases, today announced that the Compensation Committee of the company’s Board of Directors, made up entirely of independent directors, approved the grant of non-qualified stock options to purchase an aggregate of 171,900 shares of the company’s common stock to 3 new employees (the “Inducement Grants”) on December 1, 2025 (the “Grant Date”). The Inducement Grants have been granted outside of the company’s Amended and Restated 2016 Equity Incentive Plan (the “Plan”) but remain subject to the terms and conditions of such Plan. The Inducement Grants were granted as an inducement material to these individuals entering into employment with Viridian in accordance with Nasdaq Listing Rule 5635(c)(4).

The Inducement Grants have an exercise price per share that is equal to the closing price of Viridian’s common stock on the Grant Date. The Inducement Grants will vest over a four-year period, with 25% of the shares vesting on the one-year anniversary of the employee’s start date, and thereafter the remainder of the shares vest in 36 equal monthly installments, subject to each employee’s continued employment with Viridian through the applicable vesting dates.

About Viridian Therapeutics

Viridian is a biopharmaceutical company focused on discovering, developing, and commercializing potential best-in-class medicines for patients with serious and rare diseases. Viridian’s expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates for previously validated drug targets in commercially established disease areas.

Viridian is advancing multiple candidates in the clinic for the treatment of patients with thyroid eye disease (TED). The company conducted a pivotal program for veligrotug (VRDN-001), including two global phase 3 clinical trials (THRIVE and THRIVE-2), to evaluate its efficacy and safety in patients with active and chronic TED. Both THRIVE and THRIVE-2 reported positive topline data, meeting all the primary and secondary endpoints of each study. Viridian is also advancing VRDN-003 as a potential best-in-class subcutaneous therapy for the treatment of TED, including two ongoing global phase 3 pivotal clinical trials, REVEAL-1 and REVEAL-2, to evaluate the efficacy and safety of VRDN-003 in patients with active and chronic TED.

In addition to its TED portfolio, Viridian is advancing a novel portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008, which has the potential to be developed in multiple autoimmune diseases.

Viridian is based in Waltham, Massachusetts. For more information, please visit www.viridiantherapeutics.com. Follow Viridian on LinkedIn and X.

Contacts

Investors
Greg Rossino

grossino@viridiantherapeutics.com

Media
Lisa Lopez

llopez@viridiantherapeutics.com

Ossium Health to Present New Clinical Data on Its Organ Donor Bone Marrow at ASH 2025




Ossium Health to Present New Clinical Data on Its Organ Donor Bone Marrow at ASH 2025

SAN FRANCISCO–(BUSINESS WIRE)–Ossium Health, a bioengineering company that developed a first-of-its-kind bank of bone marrow available on-demand, will present new clinical data at the at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition held December 6-9, 2025 in Orlando, Florida.

Ossium’s key abstract includes clinical data from four patients with hematologic malignancies that were treated with hematopoietic stem cells derived from the vertebral bodies of deceased organ donors. All four patients achieved rapid neutrophil engraftment, platelet recovery, and full donor chimerism. All cases of acute GVHD were successfully managed with steroids and no chronic GVHD was reported. By Day 180, all evaluatable patients were alive and relapse-free. These early results indicate that Ossium’s deceased-donor-derived bone marrow is a promising new graft source that supports robust engraftment while addressing both relapse and GVHD prevention.

The abstract is now available online:

High-dose cryopreserved bone marrow from deceased donors may reduce relapse in HLA-mismatched allogeneic transplantation: Day 180 post-transplant HOPE update
Abstract Number: 5976

Presenter: Sagar Munjal, MD, MS (Chief Medical Officer, Ossium Health)

Date/Time: Monday, December 8, 06:00 PM – 08:00 PM EST

Poster Session: 721. Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Toxicities: Poster III

Ossium’s presence at ASH 2025 underscores the company’s commitment to improving access, speed, and quality in hematopoietic cell transplantation. By presenting new clinical and scientific data and engaging directly with leaders in the hematology community, the company aims to raise awareness to how organ donor bone marrow is driving meaningful therapeutic advantages and overcoming longstanding donor availability limitations.

“We’re excited to share our latest clinical insights with the hematology community at ASH,” said Kevin Caldwell, Ossium’s CEO, Co-Founder & President. “Our findings demonstrate the potential of organ donor bone marrow to transform patient outcomes, and we look forward to engaging with clinicians and researchers at our booth.”

In addition to its presentation, Ossium Health will host an exhibit at Booth 112, where attendees can learn more about the company’s organ donor-derived bone marrow and its clinical programs.

About Ossium Health

Ossium Health is a bioengineering company that leverages its proprietary organ donor bone marrow banking platform to develop stem cell therapies for patients with life-threatening hematologic conditions, organ transplant rejection, and musculoskeletal defects. Founded in 2016, the company is led by Kevin Caldwell, Chief Executive Officer, President and Co-Founder, and Erik Woods, Chief Science Officer, Executive Vice President and Co-Founder. Ossium Health’s manufacturing facility is registered with the FDA and its laboratory is certified under the Clinical Laboratory Improvement Amendments (CLIA). For more information, please visit https://ossiumhealth.com.

Follow the company on LinkedIn at https://www.linkedin.com/company/ossiumhealth/ and X at https://x.com/ossiumhealth.

Contacts

Jane Griffin

Ossium Health, Inc.

415-513-5535

press@ossiumhealth.com

Bristol Myers Squibb’s Breyanzi Approved by the U.S. FDA as the First and Only CAR T Cell Therapy for Adults with Relapsed or Refractory Marginal Zone Lymphoma (MZL)




Bristol Myers Squibb’s Breyanzi Approved by the U.S. FDA as the First and Only CAR T Cell Therapy for Adults with Relapsed or Refractory Marginal Zone Lymphoma (MZL)

In the MZL cohort of TRANSCEND FL, Breyanzi delivered deep and durable responses in 95.5% of patients while demonstrating a consistent safety profile

Breyanzi is now the only CAR T cell therapy approved by the FDA for five cancer types, the most of any CD19-directed CAR T cell therapy

PRINCETON, N.J.–(BUSINESS WIRE)–$BMY #Breyanzi—Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has granted approval of Breyanzi^® (lisocabtagene maraleucel; liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy, for the treatment of adult patients with relapsed or refractory (R/R) marginal zone lymphoma (MZL) who have received at least two prior lines of systemic therapy. Breyanzi is administered as a one-time infusion*. Please see the Important Safety Information section below, including Boxed WARNINGS for Breyanzi regarding Cytokine Release Syndrome (CRS), Neurologic Toxicities, and Secondary Hematological Malignancies.

“The FDA approval of Breyanzi for relapsed or refractory marginal zone lymphoma further solidifies it as the leading CD19-directed CAR T cell therapy covering the broadest range of B-cell malignancies. This approval in a fifth cancer type reflects our bold vision to bring the transformational potential of cell therapy to more patients,” said Lynelle B. Hoch, president, Cell Therapy Organization, Bristol Myers Squibb. “Breyanzi is the first and only CAR T cell therapy approved for this patient population, demonstrating Bristol Myers Squibb’s deep commitment to expanding access and reaching as many patients as possible with this innovative, practice-changing treatment.”

This approval of Breyanzi is based on results from the MZL cohort in TRANSCEND FL, an open-label, multicenter, multi-cohort, single-arm study. Based on the U.S. Prescribing Information (USPI), in patients treated with Breyanzi in the third-line plus setting and included in the primary efficacy analysis set (n=66), the overall response rate (ORR) was 95.5% (95% CI: 87.3-99.1). ORR was defined as the percentage of patients achieving a partial or complete response per the Lugano criteria assessed by an Independent Review Committee (IRC) per CT. The complete response (CR) rate was 62.1% (95% CI: 49.3-73.8). The median duration of response (mDOR) was not reached (95% CI: 25.59-NR), with 90.1% of responders remaining in response at 24 months.

“Patients living with marginal zone lymphoma, a subtype of indolent non-Hodgkin lymphoma, generally see success with initial therapy, but a subset of patients ultimately experience multiple relapses over the course of many years, creating a pressing need for new treatment options with durable outcomes,” said M. Lia Palomba, M.D., TRANSCEND FL study investigator and lymphoma and cell therapy specialist, Memorial Sloan Kettering Cancer Center. “The FDA approval of liso-cel in relapsed or refractory marginal zone lymphoma is a significant advancement in redefining the treatment landscape and providing patients with an option that has demonstrated high rates of responses with an established safety profile.”

The safety profile of Breyanzi in R/R MZL was consistent with prior reports from trials in other indications. In the MZL cohort of the TRANSCEND FL study, any grade cytokine release syndrome (CRS) occurred in 76% of patients, including Grade ≥3 CRS in 4.5% of patients. Any grade nervous system disorders included: headache (21%), tremor (21%), encephalopathy (21%), dizziness (16%), and aphasia (10%), including Grade ≥3 headache (1.5%) and Grade ≥3 encephalopathy (1.5%). Patients in the MZL cohort of the TRANSCEND FL study were treated in the inpatient and outpatient setting since the safety profile of Breyanzi allows for the option of outpatient treatment and adverse event management for appropriate patients.

Breyanzi is broadly covered by commercial and government insurance programs in the U.S. Bristol Myers Squibb offers various programs and resources to address the needs of patients and care partners that support their CAR T cell therapy treatment journey. Bristol Myers Squibb also supports the patient and physician treatment experience by providing Cell Therapy 360, a digital service platform, which optimizes access to relevant information, manufacturing updates, and patient and care partner support.

*The treatment process includes blood collection, CAR T cell creation, potential bridging therapy, lymphodepletion, administration, and side-effect monitoring.

About TRANSCEND FL

TRANSCEND FL (NCT04245839) is an open-label, global, multicenter, Phase 2, single-arm study to determine the efficacy and safety of Breyanzi in patients with relapsed or refractory indolent B-cell non-Hodgkin lymphoma, including follicular lymphoma and marginal zone lymphoma. The primary outcome measure is overall response rate. Secondary outcome measures include complete response rate, duration of response, and progression-free survival.

About MZL

Marginal zone lymphoma (MZL) is a subtype of non-Hodgkin lymphoma (NHL), accounting for about 7% of all NHL cases. Most patients with MZL are diagnosed at a median age of 67 years. MZL develops when white blood cells cluster together to form lumps in a person’s lymph nodes or organs. Initial therapy often leads to remission, but relapse is common, sometimes occurring several times over many years. A small portion of MZL cases transform into diffuse large B-cell lymphoma, a more aggressive lymphoma.

About Breyanzi

Breyanzi is a CD19-directed CAR T cell therapy with a 4-1BB costimulatory domain, which enhances the expansion and persistence of the CAR T cells. Breyanzi is made from a patient’s own T cells, which are collected and genetically reengineered to become CAR T cells that are then delivered via infusion as a one-time treatment. The treatment process includes blood collection, CAR T cell creation, potential bridging therapy, lymphodepletion, administration, and side-effect monitoring.

Breyanzi is approved in the U.S. for the treatment of relapsed or refractory large B-cell lymphoma (LBCL) after at least one prior line of therapy, has received accelerated approval for the treatment of relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) after at least two prior lines of therapy, relapsed or refractory follicular lymphoma (FL) after two or more prior lines of systemic therapy, and is approved for the treatment of relapsed or refractory mantle cell lymphoma (MCL) after at least two prior lines of systemic therapy. Breyanzi is also approved in Japan, the European Union (EU), Switzerland, Israel, the United Kingdom (UK), and Canada for the treatment of relapsed or refractory LBCL after at least one prior line of therapy; in Japan for the treatment of patients with relapsed or refractory high-risk FL after one prior line of systemic therapy, and in patients with relapsed or refractory FL after two or more lines of systemic therapy; in the EU, Switzerland, Israel, and the UK for the treatment of relapsed or refractory FL after two or more lines of systemic therapy; and in the EU and Israel for the treatment of relapsed or refractory MCL after at least two lines of systemic therapy including a Bruton’s tyrosine kinase (BTK) inhibitor.

Bristol Myers Squibb’s clinical development program for Breyanzi includes clinical studies in several types of lymphoma. For more information, visit clinicaltrials.gov.

Breyanzi U.S. FDA-Approved Indications

BREYANZI is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:

• adult patients with large B-cell lymphoma (LBCL), including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B, who have:
  • refractory disease to first-line chemoimmunotherapy or relapse within 12 months of first-line chemoimmunotherapy; or
  • refractory disease to first-line chemoimmunotherapy or relapse after first-line chemoimmunotherapy and are not eligible for hematopoietic stem cell transplantation (HSCT) due to comorbidities or age; or
  • relapsed or refractory disease after two or more lines of systemic therapy.

Limitations of Use: BREYANZI is not indicated for the treatment of patients with primary central nervous system lymphoma.

• adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least 2 prior lines of therapy, including a Bruton tyrosine kinase (BTK) inhibitor and a B-cell lymphoma 2 (BCL-2) inhibitor. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
• adult patients with relapsed or refractory follicular lymphoma (FL) who have received 2 or more prior lines of systemic therapy. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
• adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have received at least 2 prior lines of systemic therapy, including a Bruton tyrosine kinase (BTK) inhibitor.
• adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least 2 prior lines of systemic therapy.

Breyanzi U.S. Important Safety Information

WARNING: CYTOKINE RELEASE SYNDROME, NEUROLOGIC TOXICITIES, AND SECONDARY HEMATOLOGICAL MALIGNANCIES

• Cytokine Release Syndrome (CRS), including fatal or life-threatening reactions, occurred in patients receiving BREYANZI. Do not administer BREYANZI to patients with active infection or inflammatory disorders. Treat severe or life-threatening CRS with tocilizumab with or without corticosteroids.
• Neurologic toxicities, including fatal or life-threatening reactions, occurred in patients receiving BREYANZI, including concurrently with CRS, after CRS resolution, or in the absence of CRS. Monitor for neurologic events after treatment with BREYANZI. Provide supportive care and/or corticosteroids as needed.
• T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including BREYANZI.

Cytokine Release Syndrome

Cytokine release syndrome (CRS), including fatal or life-threatening reactions, occurred following treatment with BREYANZI. In clinical trials of BREYANZI, which enrolled a total of 769 patients with non-Hodgkin lymphoma (NHL), CRS occurred in 56% of patients, including ≥ Grade 3 CRS in 3.4% of patients. The median time to onset was 5 days (range: 1 to 63 days). CRS resolved in 99% of patients with a median duration of 5 days (range: 1 to 37 days). One patient had fatal CRS and 5 patients had ongoing CRS at the time of death. The most common manifestations of CRS (≥10%) were fever, hypotension, chills, tachycardia, hypoxia, and headache.

Serious events that may be associated with CRS include cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), cardiac arrest, cardiac failure, diffuse alveolar damage, renal insufficiency, capillary leak syndrome, hypotension, hypoxia, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS).

Ensure that 2 doses of tocilizumab are available prior to infusion of BREYANZI.

Neurologic Toxicities

Neurologic toxicities that were fatal or life-threatening, including immune effector cell-associated neurotoxicity syndrome (ICANS), occurred following treatment with BREYANZI. Serious events including cerebral edema and seizures occurred with BREYANZI. Fatal and serious cases of leukoencephalopathy, some attributable to fludarabine, also occurred.

In clinical trials of BREYANZI, CAR T cell-associated neurologic toxicities occurred in 32% of patients, including ≥ Grade 3 cases in 10% of patients. The median time to onset of neurotoxicity was 8 days (range: 1 to 63 days). Neurologic toxicities resolved in 88% of patients with a median duration of 7.5 days (range: 1 to 119 days). Of patients developing neurotoxicity, 83% also developed CRS.

The most common neurologic toxicities (≥5%) included encephalopathy, tremor, aphasia, delirium, and headache.

CRS and Neurologic Toxicities Monitoring

Monitor patients daily for at least 7 days following BREYANZI infusion for signs and symptoms of CRS and neurologic toxicities and assess for other causes of neurological symptoms. Continue to monitor patients for signs and symptoms of CRS and neurologic toxicities for at least 2 weeks after infusion and treat promptly. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated. Manage neurologic toxicity with supportive care and/or corticosteroid as needed. Advise patients to avoid driving for at least 2 weeks following infusion. Counsel patients to seek immediate medical attention should signs or symptoms of CRS or neurologic toxicity occur at any time.

Hypersensitivity Reactions

Allergic reactions may occur with the infusion of BREYANZI. Serious hypersensitivity reactions, including anaphylaxis, may be due to dimethyl sulfoxide (DMSO).

Serious Infections

Severe infections, including life-threatening or fatal infections, have occurred in patients after BREYANZI infusion. In clinical trials of BREYANZI, infections of any grade occurred in 33% of patients, with Grade 3 or higher infections occurring in 12% of all patients. Grade 3 or higher infections with an unspecified pathogen occurred in 7%, bacterial infections in 3.5%, viral infections in 2%, and fungal infections in 0.7% of patients. One patient who received 4 prior lines of therapy developed a fatal case of John Cunningham (JC) virus progressive multifocal leukoencephalopathy 4 months after treatment with BREYANZI. One patient who received 3 prior lines of therapy developed a fatal case of cryptococcal meningoencephalitis 35 days after treatment with BREYANZI.

Febrile neutropenia developed after BREYANZI infusion in 8% of patients. Febrile neutropenia may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated.

Monitor patients for signs and symptoms of infection before and after BREYANZI administration and treat appropriately. Administer prophylactic antimicrobials according to standard institutional guidelines. Avoid administration of BREYANZI in patients with clinically significant, active systemic infections.

Viral reactivation: Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, can occur in patients treated with drugs directed against B cells. In clinical trials of BREYANZI, 35 of 38 patients with a prior history of HBV were treated with concurrent antiviral suppressive therapy. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing. In patients with prior history of HBV, consider concurrent antiviral suppressive therapy to prevent HBV reactivation per standard guidelines.

Prolonged Cytopenias

Patients may exhibit cytopenias not resolved for several weeks following lymphodepleting chemotherapy and BREYANZI infusion. In clinical trials of BREYANZI, Grade 3 or higher cytopenias persisted at Day 29 following BREYANZI infusion in 35% of patients, and included thrombocytopenia in 25%, neutropenia in 22%, and anemia in 6% of patients. Monitor complete blood counts prior to and after BREYANZI administration.

Hypogammaglobulinemia

B-cell aplasia and hypogammaglobulinemia can occur in patients receiving BREYANZI. In clinical trials of BREYANZI, hypogammaglobulinemia was reported as an adverse reaction in 9% of patients. Hypogammaglobulinemia, either as an adverse reaction or laboratory IgG level below 500 mg/dL after infusion, was reported in 30% of patients. Monitor immunoglobulin levels after treatment with BREYANZI and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement as clinically indicated.

Live vaccines: The safety of immunization with live viral vaccines during or following BREYANZI treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during BREYANZI treatment, and until immune recovery following treatment with BREYANZI.

Secondary Malignancies

Patients treated with BREYANZI may develop secondary malignancies. T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including BREYANZI. Mature T cell malignancies, including CAR-positive tumors, may present as soon as weeks following infusion, and may include fatal outcomes. Monitor lifelong for secondary malignancies. In the event that a secondary malignancy occurs, contact Bristol-Myers Squibb at 1-888-805-4555 for reporting and to obtain instructions on collection of patient samples for testing.

Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome (IEC-HS)

Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome (IEC-HS), including fatal or life-threatening reactions, occurred following treatment with BREYANZI. Seven out of 769 (0.9%) patients with R/R NHL exposed to BREYANZI developed IEC-HS. Time to onset of IEC-HS ranged from 7 to 32 days. Of the 7 patients, 3 patients developed IEC-HS with overlapping occurrence of CRS and neurotoxicity, 2 patients developed IEC-HS with overlapping occurrence of neurotoxicity, and 1 patient developed IEC-HS with overlapping occurrence of CRS. IEC-HS was fatal in 2 of 7 patients. One patient had fatal IEC-HS and one had ongoing IEC-HS at time of death. IEC-HS is a life-threatening condition with a high mortality rate if not recognized and treated early. Treatment of IEC-HS should be administered per current practice guidelines.

Adverse Reactions

The most common adverse reaction(s) (incidence ≥30%) in:

• LBCL are fever, CRS, fatigue, musculoskeletal pain, and nausea. The most common Grade 3-4 laboratory abnormalities include lymphocyte count decrease, neutrophil count decrease, platelet count decrease, and hemoglobin decrease.
• CLL/SLL are CRS, encephalopathy, fatigue, musculoskeletal pain, nausea, edema, and diarrhea. The most common Grade 3-4 laboratory abnormalities include neutrophil count decrease, white blood cell decrease, hemoglobin decrease, platelet count decrease, and lymphocyte count decrease.
• FL is CRS. The most common Grade 3-4 laboratory abnormalities include lymphocyte count decrease, neutrophil count decrease, and white blood cell decrease.
• MCL are CRS, fatigue, musculoskeletal pain, and encephalopathy. The most common Grade 3-4 laboratory abnormalities include neutrophil count decrease, white blood cell decrease, and platelet count decrease.
• MZL is CRS. The most common Grade 3-4 laboratory abnormalities include lymphocyte count decrease, neutrophil count decrease, and white blood cell decrease.

Please see full Prescribing Information, including Boxed WARNINGS and Medication Guide.

Bristol Myers Squibb: Unlocking the Full Potential of Cell Therapy

A pioneer in harnessing the immune system to fight cancer and an established leader in cell therapy, Bristol Myers Squibb is uniquely positioned to unlock the full potential of this technology across blood cancers and within new frontiers, including autoimmune disease.

Bristol Myers Squibb is currently the only company with two approved CAR T cell therapies with two distinct targets, available in major markets around the world. Our bold vision for the future is one in which hundreds of thousands of patients can be treated with cell therapy’s transformational potential.

The building blocks to realize this ambition—a promising and differentiated pipeline, extensive translational and clinical data sets, a deep bench of talent, and robust manufacturing capabilities—are in our cells. We are laser-focused on advancing the field of cell therapy toward a true revolution for patients. Learn more about the science behind cell therapy and ongoing progress at Bristol Myers Squibb here.

About Bristol Myers Squibb

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

Cautionary Statement Regarding Forward-Looking Statements

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

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IDE034, a Bispecific ADC Licensed by Biocytogen to IDEAYA, Receives FDA IND Clearance




IDE034, a Bispecific ADC Licensed by Biocytogen to IDEAYA, Receives FDA IND Clearance

BEIJING–(BUSINESS WIRE)–Biocytogen Pharmaceuticals (Beijing) Co., Ltd. (Biocytogen, HKEX: 02315), announced that its partner IDEAYA Biosciences, Inc. (Nasdaq: IDYA), a precision oncology company, has received the clearance of an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) for the initiation of a Phase 1 clinical trial of IDE034, a potential first-in-class B7H3/PTK7 bispecific antibody-drug conjugate (ADC). IDEAYA expects to begin patient enrollment in Q1 2026, initially evaluating patients with solid tumors known to express B7H3 and PTK7, including lung, colorectal, head and neck, and ovarian/gynecological cancers.

IDE034 is a bispecific B7H3/PTK7 TOP1 ADC, independently developed by Biocytogen and licensed to IDEAYA in July 2024. The IND clearance marks an important milestone for this licensed program, supporting subsequent clinical development of IDE034, while highlighting Biocytogen’s technical capabilities in bispecific ADC discovery and development.

Dr. Yuelei Shen, Chairman and CEO of Biocytogen, said: “The IND clearance for IDE034 is an important milestone in the development of this first-in-class TOP1 ADC licensed project, representing a significant advancement for IDEAYA in expanding its clinical pipeline with bispecific, precision-targeted strategies. It also validates our RenLite^® platform and proprietary linker-payload technologies that enable the discovery and optimization of bispecific ADCs. We look forward to seeing IDE034 demonstrate clinical potential across multiple B7H3/PTK7 co-expressing solid tumors, offering new therapeutic options for patients.”

Preclinical studies have shown that IDE034 monotherapy induces deep and durable tumor regressions in multiple B7H3/PTK7-positive tumor models, demonstrating strong anti-tumor activity. In addition, IDEAYA plans to explore combination strategies with its PARG inhibitor IDE161 to enhance the durability of response and intends to present additional preclinical data supporting the combination rationale at a major medical conference in H1 2026. B7H3 and PTK7 are co-expressed in lung, colorectal, and head and neck cancers at approximately 30%, 46%, and 27%, respectively, indicating the broad clinical potential of IDE034.

Looking ahead, Biocytogen will continue to provide high-quality source antibodies through RenBiologics to support the clinical translation of licensed projects and actively explore additional early-stage assets for external licensing opportunities.

About Biocytogen

Biocytogen (HKEX: 02315) is a global biotechnology company that drives the research and development of novel antibody-based drugs with innovative technologies. Founded on gene editing technology, Biocytogen leverages genetically engineered proprietary RenMice^® platforms for fully human monoclonal/bispecific/multispecific antibody discovery, bispecific antibody-drug conjugate discovery, nanobody discovery and TCR mimic antibody discovery, and has established a sub-brand, RenBiologics™, to explore global partnerships for an off-the-shelf library of >1,000,000 fully human antibody sequences against over 1000 targets for worldwide collaboration. As of June 30, 2025, approximately 280 therapeutic antibody and multiple clinical asset co-development/out-licensing/transfer agreements and over 50 target-nominated RenMice^® licensing projects have been established around the globe, including several partnerships with multinational pharmaceutical companies (MNCs). Biocytogen pioneered the generation of drug target knock-in humanized models for preclinical research, and currently provides a few thousand off-the-shelf animal and cell models under the company’s sub-brand, BioMice™, along with preclinical pharmacology and gene-editing services for clients worldwide. Headquartered in Beijing, Biocytogen has branches in China (Haimen Jiangsu, Shanghai), USA (Boston, San Francisco, San Diego), and Germany (Heidelberg). For more information, please visit https://biocytogen.com.

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Antibody assets and platforms: BD-Licensing@biocytogen.com
Media: pr@bbctg.com.cn