Abcuro Presents Interim Phase 1 Data Evaluating Ulviprubart in Patients with T Cell Large Granular Lymphocytic Leukemia at the 67th American Society of Hematology Annual Meeting




Abcuro Presents Interim Phase 1 Data Evaluating Ulviprubart in Patients with T Cell Large Granular Lymphocytic Leukemia at the 67th American Society of Hematology Annual Meeting

NEWTON, Mass.–(BUSINESS WIRE)–Abcuro, Inc., a late-stage clinical biotechnology company developing potentially first-in-class immunotherapies designed to benefit people living with debilitating and progressive rare autoimmune diseases and for other indications where certain cytotoxic T cells are pathogenic, today presented interim data from the ongoing Phase 1/2 clinical trial evaluating ulviprubart (ABC008) in patients with T cell large granular lymphocytic leukemia (T-LGLL) with anemia and/or neutropenia, in an oral presentation at the 67^th American Society of Hematology (ASH) Annual Meeting and Exposition, taking place December 6-9, 2025 in Orlando, Florida.

Ulviprubart is a potentially first-in-class, potent, monoclonal antibody targeting KLRG1, a novel mechanism of action to drive selective depletion of highly differentiated T cells and modify disease progression. Ulviprubart was designed to target KLRG1-expressing T cells while sparing B and regulatory T cells that are required to maintain normal immune system homeostasis.

“The data presented at ASH continue to support the potential of ulviprubart to selectively target and deplete highly differentiated T cells that drive debilitating diseases like T-LGLL. At the interim analysis, ulviprubart had an acceptable safety profile and was generally well tolerated across ascending doses, further supporting ulviprubart’s potential in treating patients with T-LGLL,” said H. Jeffrey Wilkins, MD, Chief Medical Officer of Abcuro.

T-LGLL is a hematological cancer driven by pathogenic expansion of immune cells that are frequently KLRG1+, resulting in neutropenia and anemia. Neutropenia can lead to frequent infections, a major cause of premature death in patients with T-LGLL, while anemia results in transfusion dependence in approximately one-third of patients.

Key Highlights from Oral Presentation:

The Phase 1/2 clinical trial (NCT05532722) is an open label, ascending dose study of ulviprubart patients with T-LGLL. The primary objective is to evaluate safety and tolerability. Secondary objectives include evaluating initial efficacy and pharmacokinetic/pharmacodynamic (PK/PD) profile of ulviprubart.

As of the data cut-off date of November 15, 2025:

• 21 patients were enrolled and evaluable for safety. 95% (n=20) of patients had neutropenia and 57% (n=12) of patients had anemia at baseline.
• 62% (n=13) of patients achieved >12 weeks of Q4W dosing on ulviprubart.
  • Among evaluable patients, seven experienced sustained depletions of >50% of CD8⁺ CD57⁺ KLRG1⁺ T cells at two or more consecutive visits. Three patients experienced sustained depletions of >90% of both CD8⁺ CD57⁺ KLRG1⁺ T cells and the CD8⁺ CD57⁺ parent population.
  • Among evaluable patients, all had neutropenia and nine patients had anemia at baseline.
    • With treatment, seven patients had a neutropenia response, defined as an absolute neutrophil count (ANC) increase of ≥50% from baseline for ≥4 weeks or ANC levels ≥1000 cells/µL for ≥4 weeks.
    • With treatment, two patients had an anemia response, defined as hemoglobin increased ≥1 g/dL for ≥4 weeks and not attributable to transfusion or growth factor.

Overall, ulviprubart was well tolerated at increasing doses and had an acceptable safety profile. Most treatment-related treatment-emergent adverse events (TEAE) were mild or moderate in severity. One patient experienced a Grade 3 infusion-related reaction, the only serious treatment-related TEAE observed on therapy.

About Ulviprubart

Ulviprubart (ABC008) is potentially a first-in-class, potent, monoclonal antibody targeting KLRG1, a novel proposed mechanism of action to drive selective depletion of highly differentiated T cells and modify disease progression. KLRG1 is a cell surface receptor predominantly expressed on highly differentiated T cells, while moderately or minimally expressed on other immune cells. Ulviprubart was designed to selectively deplete KLRG1-expressing T cells while sparing B and regulatory T cells that are required to maintain normal immune system homeostasis.

About Abcuro

Abcuro is a late-stage clinical biotechnology company developing potentially first-in-class immunotherapies designed to benefit people living with debilitating and progressive rare autoimmune diseases and for other indications where certain cytotoxic T cells are pathogenic. Our product candidate, ulviprubart (formerly referred to as ABC008), is a humanized monoclonal antibody that targets pathogenic T cells that express killer cell lectin-like receptor G1 (“KLRG1”), referred to as KLRG1+ T cells. Ulviprubart is designed to selectively target and deplete well-differentiated cytotoxic KLRG1+ T cells where KLRG1 is highly expressed, while sparing other immune cells, which we believe will offer improvements in safety and tolerability as compared to other T cell depleting approaches. Ulviprubart is currently being evaluated in a registrational Phase 2/3 clinical trial in people with inclusion body myositis (IBM), and a Phase 1/2 clinical trial in people with T cell large granular lymphocytic leukemia (T-LGLL).

For more information, visit us on LinkedIn and at abcuro.com.

Contacts

Matthew DeYoung

Investor Relations and Media

Argot Partners

abcuro@argotpartners.com

PepGen Appoints Joseph Vittiglio, Esq., as Chief Business and Legal Officer




PepGen Appoints Joseph Vittiglio, Esq., as Chief Business and Legal Officer

BOSTON–(BUSINESS WIRE)–PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced the appointment of Joseph Vittiglio, Esq., as Chief Business and Legal Officer. Mr. Vittiglio brings more than two decades of executive leadership experience across legal, compliance, corporate development, and corporate governance within public biotechnology companies.

“Joe is a deeply experienced legal and business leader with a proven track record guiding companies through complex transactions, regulatory milestones, financings, product launches, and strategic partnerships,” said James McArthur, PhD, President and Chief Executive Officer of PepGen. “We are excited to welcome him to PepGen’s executive leadership team as we continue to make significant progress advancing our PGN-EDODM1 program and prepare for the anticipated 2026 readouts from the 5 mg/kg and 10 mg/kg cohorts in our FREEDOM2-EDODM1 multiple ascending dose study in DM1 patients.”

Mr. Vittiglio joins PepGen after providing extensive consulting support to the Company in 2025. He most recently served as Chief Business and Legal Officer and Corporate Secretary at bluebird bio, where he led global legal, compliance, and business development; supported the approval and launch of three U.S. gene therapy products; advanced ex-U.S. partnering; and completed more than $400 million in financings. Prior to bluebird, he served as Chief Business and Legal Officer at Finch Therapeutics, guiding the company’s IPO, building its public-company governance and compliance functions, and overseeing a global IP portfolio of more than 50 patent families. Previously, he was Chief Business Officer and General Counsel at AMAG Pharmaceuticals, supporting major corporate transactions, multiple product launches, significant financings, and all legal, compliance, and business development for a commercial organization generating over $400 million annually. Earlier in his career, he held senior legal roles at Flexion Therapeutics, AVEO Pharmaceuticals, and Oscient Pharmaceuticals, after beginning his legal career at Mintz. Mr. Vittiglio holds a J.D. from Northeastern University School of Law and a B.A. in International Relations from Tufts University.

About PepGen

PepGen Inc. is a clinical-stage biotechnology company developing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, the Company is generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases.

For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X.

Contacts

Investor Contact
Laurence Watts

New Street Investor Relations

laurence@newstreetir.com

Media Contact
Julia Deutsch

Lyra Strategic Advisory

Jdeutsch@lyraadvisory.com

Kallyope Announces Positive Results from Phase 2b Study of Elismetrep for the Acute Treatment of Migraine




Kallyope Announces Positive Results from Phase 2b Study of Elismetrep for the Acute Treatment of Migraine

Novel mechanism of TRPM8 antagonism may offer new option for large number of patients suffering from debilitating migraines

Full data to be presented at a major medical meeting in 2026

Kallyope plans to initiate registrational studies in mid-2026

NEW YORK–(BUSINESS WIRE)–Kallyope, a late-stage biotechnology company leveraging unique insights into neural signaling pathways to develop innovative therapies for migraine and metabolism, today announced positive results from a Phase 2b study of the company’s lead candidate elismetrep, an oral Transient Receptor Potential Melastatin 8 (TRPM8) antagonist being studied for the acute treatment of migraine. In the study, elismetrep delivered a favorable clinical profile through its novel mechanism, supporting its potential as a new therapeutic option for the large number of patients who are suffering from the debilitating effects of migraine.

“Migraines impact one in six Americans, leading to debilitating pain, nausea, and sensitivity that can strike at a moment’s notice and prevent people from participating fully in their lives. Migraines are estimated to have an economic impact of $36 billion annually in the U.S. alone,” said Jay Galeota, CEO and President, Kallyope. “As the only TRPM8 antagonist being studied for the acute treatment of migraine, elismetrep has demonstrated the potential to bring relief and a sense of control back to the lives of millions of people who suffer from migraines. We look forward to initiating registrational studies in 2026 and working with great urgency to bring this important potential therapy to patients in need.”

The double-blind, placebo-controlled, Phase 2b dose-ranging study randomized 431 patients in the United States. Key measures were standard for clinical studies in the acute treatment of migraine and included pain freedom, pain relief, and freedom from most bothersome migraine-associated symptom. The performance of elismetrep across all endpoints was competitive with marketed therapies. No safety signals were observed, and the drug was well tolerated. Tolerability-related adverse events were mostly mild.

“Our Phase 2b data strongly support elismetrep’s ability to deliver on key measures in the assessment of migraine drugs and potentially become an important part of the standard of care,” said Brett Lauring, MD, PhD, Chief Medical Officer, Kallyope. “We look forward to sharing additional details on the novel TRPM8 mechanism, elismetrep, and expanded clinical analyses at an upcoming medical meeting.”

Currently, only around 30% of patients achieve good outcomes with any particular medication used in the acute treatment of migraine. This creates a highly dissatisfied population that switches between medications to seek relief. Elismetrep targets Transient Receptor Potential Melastatin 8 (TRPM8), an ion channel protein with a strong genetic association to migraine and has demonstrated robust efficacy in preclinical models of migraine. TRPM8 is expressed in trigeminal sensory neurons involved in migraine which are distinct from those targeted by existing therapies such as antagonists of calcitonin gene-related peptide (CGRP), suggesting potential as a combination treatment.

“Migraines are among the leading causes of disability globally, and suboptimal treatment of migraine through existing drug classes results in reduced quality of life for millions of people worldwide,” said Peter Goadbsy, MD, PhD, King Abdullah University of Science and Technology, Saudi Arabia, and King’s College London, UK. “Given the variable and inadequate response to existing therapies, patients and physicians urgently need new approaches to managing this debilitating condition, making elismetrep a compelling addition to the innovation pipeline in migraine.”

About Kallyope

Kallyope is a late-stage biotechnology company using unique insights into neural signaling pathways to develop innovative therapies for migraine and metabolism, which affect hundreds of millions of people globally. Kallyope’s lead candidate elismetrep (K-304) is in development for the acute treatment of migraines. The company’s metabolic pipeline includes candidates against a novel target identified and validated by the company’s Klarity™ platform. Kallyope was founded by world-leading neuroscientists and continues to explore the role of neural circuits in driving disease. For more information, visit www.kallyope.com.

Contacts

Media Contact:
Ten Bridge Communications

Michael Galfetti

TBCKallyope@tenbridgecommunications.com

Investor Contact:
Kallyope

ir@kallyope.com

Appointment of Francis Van Parys as Chief Executive Officer




Appointment of Francis Van Parys as Chief Executive Officer

OXFORD, England–(BUSINESS WIRE)–#Nanopore–Oxford Nanopore Technologies, the company delivering a new generation of molecular sensing technology based on nanopores, today announces the appointment of Francis Van Parys as Chief Executive Officer (“CEO”). Francis will join the Company and the Board as an Executive Director on 2 March 2026.

Francis brings more than 20 years of experience leading multi-billion-dollar life science businesses, with a strong track record of scaling innovation-driven organisations through commercial and operational excellence. He is currently President and CEO of Radiometer, a global leader in acute care diagnostics and part of Danaher Corporation. Previously, Francis held senior leadership roles at Cytiva and GE Healthcare, driving sustained growth and building high-performing teams across Europe, Asia, and North America.

Francis Van Parys will succeed Gordon Sanghera, who has led Oxford Nanopore since its inception in 2005. Gordon will step down from the Board at close of business on 2 March 2026 and will remain as an employee of the Company in an advisory capacity through to early 2027 to ensure a smooth handover.

Duncan Tatton-Brown, Chair of Oxford Nanopore, remarked: “We are delighted to welcome Francis Van Parys as the next CEO. We believe his global life sciences leadership and deep regulatory expertise, combined with a proven ability to scale innovation-driven businesses make him a strong fit for the next phase of growth. His appointment follows a comprehensive succession process, led by the Nomination Committee with an independent search firm. The Board looks forward to working with Francis as he builds on strong foundations of innovation to lead the Company into its next chapter of growth and impact.

“I would like to reiterate my thanks to Gordon for his leadership, vision and unwavering commitment to building Oxford Nanopore over the last 20 years.”

Francis Van Parys commented: “I’m excited to join Oxford Nanopore at such an important stage in its development. The Company is delivering strong growth, underpinned by its differentiated sensing platform and expanding global customer base. With a substantial market opportunity ahead, I look forward to building on this strong foundation, driving innovation that shapes trends in global genomics and enhancing operational execution across the business to deliver value for the Company and for all our stakeholders.”

Gordon Sanghera, CEO of Oxford Nanopore, added: “Leading Oxford Nanopore for more than two decades has been an extraordinary privilege. From an idea that single-molecule sensing could be done differently, we’ve built a company that created a new category of multi-omic analysis. I’m confident Francis will build on the strong foundations of innovation and growth to lead Oxford Nanopore to even greater success.”

About Oxford Nanopore Technologies plc:

Oxford Nanopore Technologies’ goal is to bring the widest benefits to society through enabling the analysis of anything, by anyone, anywhere. The Group has developed a new generation of nanopore- based sensing technology that is currently used for real-time, high-performance, accessible, and scalable analysis of DNA and RNA. The technology is used in more than 125 countries, to understand the biology of humans, plants, animals, bacteria, viruses and environments as well as to understand diseases such as cancer. Oxford Nanopore’s technology also has the potential to provide broad, high impact, rapid insights in a number of areas including healthcare, food and agriculture.

For more information please visit: www.nanoporetech.com.

Contacts

For further information, please contact:
Oxford Nanopore Technologies plc

Investors: ir@nanoporetech.com
Media: media@nanoporetech.com

Teneo (communications adviser to the Company)
Tom Murray, Lisa Jarrett-Kerr

+44 (0) 20 7353 4200

OxfordNanoporeTechnologies@teneo.com

PureTech Announces Successful End-of-Phase 2 Meeting with FDA for Deupirfenidone (LYT-100) in Idiopathic Pulmonary Fibrosis




PureTech Announces Successful End-of-Phase 2 Meeting with FDA for Deupirfenidone (LYT-100) in Idiopathic Pulmonary Fibrosis

Feedback from U.S. Food and Drug Administration (FDA) supports advancement into a pivotal Phase 3 trial and a 505(b)(2) regulatory pathway

Phase 3 SURPASS-IPF trial remains on track to be initiated by PureTech’s Founded Entity, Celea Therapeutics, in the first half of 2026

BOSTON–(BUSINESS WIRE)–PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the “Company”), a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value, today announced the successful completion of the End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) regarding the development of deupirfenidone (LYT-100) for the treatment of idiopathic pulmonary fibrosis (IPF). Deupirfenidone is being advanced by Celea Therapeutics, a Founded Entity established by PureTech to lead its late-stage development and potential commercialization.

“Our discussion with the FDA was productive and provided helpful feedback on key elements of our Phase 3 program and the overall data expectations for registration,” said Sven Dethlefs, Ph.D., Chief Executive Officer of Celea Therapeutics. “The forthcoming Phase 3 SURPASS-IPF trial builds on the strong foundation established by the Phase 2b ELEVATE IPF trial, which demonstrated deupirfenidone’s robust and durable treatment effect as a monotherapy and its potential to become a new standard of care. In shaping the Phase 3 design, we incorporated learnings from recent IPF trials and collaborated closely with patients and clinicians to reflect the latest thinking in the field. We are now advancing this pivotal program with urgency to bring forward a therapy with the potential to stabilize lung function and meaningfully improve care for people with IPF.”

The pivotal Phase 3 SURPASS-IPF trial will be a global, randomized, double-blind, head-to-head trial comparing deupirfenidone 825 mg three times-a-day (TID) to pirfenidone 801 mg TID in adults with IPF who are not on background therapy. The primary efficacy endpoint is the change from baseline in absolute forced vital capacity (FVC) at week 52, which will assess the superiority of deupirfenidone compared with pirfenidone. The 52-week trial will use the same active comparator and dosing regimen as the Phase 2b ELEVATE-IPF trial, providing continuity and confidence that the favorable safety profile and strong treatment effect observed previously can be replicated and confirmed in a larger, global population. Based on feedback from the FDA, PureTech believes that the results from this single Phase 3 trial, if successful, and supported by the totality of data from the overall deupirfenidone development program, could complete the data package required to support potential registration of deupirfenidone via a streamlined 505(b)(2) pathway.

The EOP2 meeting was supported by results from the global Phase 2b randomized, double-blind, active- and placebo-controlled, dose-ranging ELEVATE IPF trial. In that trial, participants treated with deupirfenidone 825 mg TID experienced a slower rate of lung function decline, as measured by change from baseline of Forced Vital Capacity (FVC), at 26 weeks versus those who were treated with pirfenidone 801 mg TID or placebo (-21.5 mL vs. -51.6 mL vs. -112.5 mL, respectively), with a 91 mL difference between deupirfenidone 825 mg and placebo at 26 weeks. Following the completion of the blinded portion of the trial, 170 participants (more than 90%) enrolled in the open-label extension. Those who continued treatment with deupirfenidone 825 mg TID maintained a robust treatment effect and experienced an overall FVC decline of -32.8 mL over a 52-week period,¹ which is similar to the expected natural decline in lung function in healthy older adults over that time (approximately -30.0 mL to -50.0 mL).²

PureTech’s Founded Entity, Celea Therapeutics, expects to finalize financing in early 2026 to support the initiation of the Phase 3 SURPASS-IPF trial in the first half of 2026.

About Deupirfenidone (LYT-100)

Deupirfenidone (LYT-100) is in development as a potential new standard of care for the treatment of idiopathic pulmonary fibrosis (IPF). It is a next generation antifibrotic and a deuterated form of pirfenidone, one of three FDA-approved therapies for IPF. The uptake and adherence to approved antifibrotics has historically been limited by a tradeoff between modest efficacy and tolerability, and only ~25% of people with IPF in the U.S. had ever received treatment as of 2019.³

Deupirfenidone may overcome these limitations. In the global Phase 2b ELEVATE IPF trial, deupirfenidone demonstrated the potential to stabilize lung function decline over at least 26 weeks as a monotherapy while maintaining a favorable safety and tolerability profile. Initial data from an ongoing open-label extension study suggest this effect may be sustained through at least 52 weeks. These findings support the potential for deupirfenidone to offer a meaningful advance for people living with this progressive and deadly disease. Beyond IPF, deupirfenidone may also address multiple underserved fibrotic conditions, including progressive fibrosing interstitial lung diseases.

About Idiopathic Pulmonary Fibrosis (IPF)

Idiopathic pulmonary fibrosis (IPF) is a rare, progressive, and fatal lung disease characterized by irreversible scarring of lung tissue that leads to a steady decline in lung function. Median survival following diagnosis is estimated to be two to five years, and currently there is no cure.⁴

About Celea Therapeutics

Celea Therapeutics is dedicated to advancing transformative treatments for people with serious respiratory diseases. Drawn from the Latin word for “sky,” the name reflects the company’s mission to rise above the status quo and deliver therapies that change lives. The company’s lead program, deupirfenidone (LYT-100), is a Phase 3-ready therapeutic candidate with the potential to set a new standard of care for idiopathic pulmonary fibrosis (IPF) and other fibrotic lung diseases.

Celea was founded by and is currently a wholly-owned subsidiary of PureTech Health plc (Nasdaq: PRTC, LSE: PRTC), a biotherapeutics company dedicated to giving life to science. PureTech’s innovative R&D model drives the creation of Founded Entities like Celea, enabling the advancement of highly promising medicines to patients in a capital-efficient manner. For more information, please visit www.celeatx.com.

About PureTech Health

PureTech Health is a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value. We do this through a proven, capital-efficient R&D model focused on opportunities with validated pharmacology and untapped potential to address significant patient needs. This strategy has produced dozens of therapeutic candidates, including three that have received U.S. FDA approval. By identifying, shaping, and de-risking these high-conviction assets, and scaling them through dedicated structures backed by external capital, we accelerate their path to patients while creating sustainable value for shareholders.

For more information, visit www.puretechhealth.com or connect with us on X (formerly Twitter) @puretechh.

Cautionary Note Regarding Forward-Looking Statements

This press release contains statements that are or may be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements that relate to continued development of and regulatory interactions related to deupirfenidone, the potential of deupirfenidone in IPF and other indications, our expectations around our therapeutic candidates and approach towards addressing major diseases, our plans to advance our programs and deliver on our milestones, our future plans, prospects, developments, and strategies. The forward-looking statements are based on current expectations and are subject to known and unknown risks, uncertainties and other important factors that could cause actual results, performance and achievements to differ materially from current expectations, including, but not limited to, those risks, uncertainties and other important factors described under the caption “Risk Factors” in our Annual Report on Form 20-F for the year ended December 31, 2024 filed with the SEC and in our other regulatory filings. These forward-looking statements are based on assumptions regarding the present and future business strategies of the Company and the environment in which it will operate in the future. Each forward-looking statement speaks only as at the date of this press release. Except as required by law and regulatory requirements, we disclaim any obligation to update or revise these forward-looking statements, whether as a result of new information, future events or otherwise.

Contacts

PureTech
Public Relations

publicrelations@puretechhealth.com
Investor Relations

IR@puretechhealth.com

UK/EU Media
Ben Atwell, Rob Winder

+44 (0) 20 3727 1000

puretech@fticonsulting.com

US Media
Justin Chen

jchen@tenbridgecommunications.com

Merz Therapeutics to Present Broad Range of Clinical, Real-World and Mechanistic Data at TOXINS 2026




Merz Therapeutics to Present Broad Range of Clinical, Real-World and Mechanistic Data at TOXINS 2026

• Merz Therapeutics to present more than 20 abstracts and posters at the TOXINS 2026 8^th International Conference, taking place January 14-17 2026 in Madrid, Spain.

FRANKFURT, Germany–(BUSINESS WIRE)–Merz Therapeutics, a leading player in neurology-focused specialty pharma, today announced that the company will present more than 20 clinical and non-clinical abstracts and posters spanning spasticity, movement disorders, and neurotoxin science at the TOXINS 2026 8^th International Conference, taking place January 14–17, 2026, in Madrid, Spain. These presentations underscore the company’s commitment to addressing unmet needs in neurological disorders.

“Our research reflects a relentless focus on improving patient outcomes through innovation in neurotoxin science,” said Dr. Stefan Albrecht, Chief Scientific and Medical Officer at Merz Therapeutics. “By presenting these new data at TOXINS 2026, we aim to foster scientific exchange and advance treatment strategies that address real-world challenges for patients and clinicians.”

Merz Therapeutics will share new findings reflecting its continued dedication to advancing neurotoxin science. These include:

Lower Limb Spasticity

• IncobotulinumtoxinA for the Treatment of Lower Limb Spasticity in Children and Adolescents with Cerebral Palsy: Evaluation of Lower Limb IncobotulinumtoxinA Efficacy (ELLIE)
  
  Authors: Marta Banach, Iryna Makedonska, Veronika Mykhaylenko, Angelika Hanschmann, Thorin Geister, Andrzej Dekundy
  
  Presenter: Sebastian Schröder, MD (Germany)
  
  Location and Time: Clinical Track 1B – Thursday, 15 January 2026, 17:40 – 17:55 p.m.

Upper Limb Spasticity – Shoulder Management

• Pathways for diagnosis and multimodal management, including botulinum neurotoxin therapy, in shoulder conditions following central lesions
  
  Authors: Bo Biering-Sørensen, Carlos Cordero-García, Chris Boulias, Damon Hoad, Djamel Bensmail, Franco Molteni, François Genêt, Jörg Wissel, Philippe Marque, Steffen Berweck, Jorge Jacinto
  
  Location and Time: Poster and Exhibit Hall, 14-17 January 2026

Cervical Dystonia

• Real-world Evidence of Longevity of BoNT/A in Cervical Dystonia (RELY-CD)
  
  Authors: Benjamin Wäschle, John Ih Lee, Tristan Kölsche, Robin Jansen, Piotr Sobolewski, Sara Sánchez Valiente, Eva López Valdés, Pablo Mir, Silvia Jesús, Elena Ojeda Lepe, Ewa Papuć, Pilar Sánchez Alonso, Gabriel Salazar, Georg Comes, Holger Stark, Philipp Albrecht
  
  Location and Time: Poster and Exhibit Hall, 14-17 January 2026

Pain

• Evaluating the efficacy and safety of IncobotulinumtoxinA in adults with moderate to severe chronic peripheral neuropathic pain – The Phase 2 PaiNT Study Design
  
  Authors: Nadine Attal, Irena Pulte, Ilona Bicker, Ralf Baron, Nanna B. Finnerup, Thorin L. Geister, Eric Viel
  
  Location and Time: Poster and Exhibit Hall, 14-17 January 2026

Immunogenicity and Secondary Treatment Failure

• A systematic review and meta-analysis of neutralizing antibodies after treatment with abobotulinumtoxinA, incobotulinumtoxinA and onabotulinumtoxinA across multiple indications
  
  Authors: Uwe Walter, Phillipp Albrecht, Warner Carr, Harald Hefter
  
  Location and Time: Poster and Exhibit Hall, 14-17 January 2026
• Secondary treatment failure with abobotulinumtoxinA, incobotulinumtoxinA and onabotulinumtoxinA: A systematic review and meta-analysis
  
  Authors: Uwe Walter, Phillipp Albrecht, Warner Carr, Harald Hefter
  
  Location and Time: Poster and Exhibit Hall, 14-17 January 2026

Toxins congress attendees can connect with Merz Therapeutics at the booth to learn more about the company, its pipeline and ongoing research.

About Merz Therapeutics

Merz Therapeutics GmbH is dedicated to delivering better outcomes for more patients. With science as its foundation and the patient experience as its focus, the company relentlessly pursues innovative treatments and partnerships to address unmet needs in movement disorders, neurodegenerative conditions, liver disease, and other health conditions that severely impact patients’ quality of life.

Merz Therapeutics is headquartered in Frankfurt am Main, Germany, and is active in more than 80 countries. Merz Therapeutics GmbH is part of the Merz Group, a privately held, family-owned company with a 117-year legacy. With passion and purpose, Merz Therapeutics continues to advance care in specialty neurology in ways that benefit both patients and society.

Please visit www.merztherapeutics.com.

Contacts

Press Contact:
Merz Therapeutics GmbH

Luke Anthony Mircea-Willats

Global Communications

luke.mirceawillats@merz.ch

Sensorion Reports Independent Data Monitoring Committee Raises No Safety Concerns and Agrees to Continue SENS-501’s Audiogene Phase 1/2 Trial




Sensorion Reports Independent Data Monitoring Committee Raises No Safety Concerns and Agrees to Continue SENS-501’s Audiogene Phase 1/2 Trial

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

Sensorion (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company specializing in the development of novel therapies to restore, treat and prevent hearing loss disorders, today announced that the Data Monitoring Committee (DMC) supports the continuation of the Audiogene Phase 1/2 clinical trial of SENS-501, the Company’s gene therapy program being developed to treat a specific form of congenital deafness linked to mutations in the OTOF (otoferlin) gene. Based on the DMC’s safety observations to date which include good procedural tolerance, the Committee has raised no safety concerns.

The Audiogene trial is designed to evaluate the safety, tolerability and efficacy of intra-cochlear administration of SENS-501 for the treatment of OTOF gene-mediated hearing loss in paediatric patients aged 6 to 31 months at the time of treatment. By intervening during the critical early window of brain plasticity and enrolling infants who have not yet received a cochlear implant, the study aims to isolate the therapeutic effect of SENS-501 as monotherapy. The trial consists of two sequential dose-escalation cohorts, each involving unilateral injection to allow a clear assessment of the kinetics of hearing restoration. Moreover, Audiogene aims to evaluate the usability, the clinical and the technical performances of the injection system in development.

On December 4^th, the DMC reviewed the first part of the SENS-501 Audiogene Phase 1/2 clinical trial including cohort 2 which completed enrolment in July 2025. This cohort includes three patients aged between 6 and 31 months who received the dose 2 of SENS-501 (4.5E11 vg/vector/ear). Across both the first and second cohorts, the surgical procedure was well tolerated, and intra-cochlear administration of SENS-501 was uneventful, and no serious adverse events or serious side effects have been reported.

“The safety findings combined with the early efficacy signals observed so far in infants and toddlers support further clinical investigation of SENS-501,” said Nawal Ouzren, Chief Executive Officer of Sensorion. “Treating otoferlin-mediated congenital deafness before cochlear implantation and during heightened neural plasticity is essential to fully evaluate the potential of gene therapy as a monotherapy. We will continue to generate rigorous and comprehensive data to guide development decisions for this important patient population.”

In children with age and baseline hearing characteristics comparable to those reported in recent publications, Sensorion has observed in the second cohort early directional improvements in two of the three treated patients by Month 3 on pure-tone audiometry. At Month 3, Patient 4 demonstrated a behavioural threshold of approximately 60 dB HL at the best-performing frequency, while Patient 5 demonstrated approximately 70 dB HL at the best-performing frequency. Follow-up is ongoing to assess the durability of these effects and the potential for further clinically meaningful functional gains.

The Company will review the upcoming six-month efficacy data and will communicate during Q1 2026 once the dataset has reached sufficient maturity.

About SENS-501

SENS-501 (OTOF-GT) is an innovative gene therapy program developed to treat a specific form of congenital deafness linked to mutations in the OTOF (otoferlin) gene. This gene plays a key role in the transmission of auditory signals between the hair cells of the inner ear and the auditory nerve. When this gene is defective, affected individuals are born with severe to profound hearing loss. The aim of SENS-501 (OTOF-GT) is to restore hearing by introducing a functional copy of the OTOF gene directly into hair cells via viral vector technology (AAV). This therapy aims to restore the normal process of converting sound into electrical signals, enabling patients to regain their hearing ability. Currently in the clinical research phase, this gene therapy program represents significant hope for families affected by this rare form of genetic deafness. SENS-501 (OTOF-GT) embodies a commitment to scientific innovation in the field of hearing, with the potential to dramatically improve the quality of life of patients suffering from genetic deafness. This gene therapy for patients suffering from otoferlin deficiency has been developed in the framework of RHU AUDINNOVE, a consortium composed of Sensorion with the Necker Enfants Malades Hospital, the Institut Pasteur, and the Fondation pour l’Audition. The project is partially financed by the French National Research Agency, through the “investing for the future” program (ref: ANR-18-RHUS-0007). The OTOF gene targeted by the Audiogene trial was discovered in 1999 at the Institut Pasteur, by Prof. Christine Petit’s team (Institut reConnect, Institut de l’Audition, Pasteur Institute), who also unraveled the pathophysiology of the corresponding deafness (DFNB9).

About the Audiogene Trial

Audiogene aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of SENS-501 for the treatment of OTOF gene-mediated hearing loss in infants and toddlers aged 6 to 31 months at the time of gene therapy treatment. By targeting the first years of life, when brain plasticity is optimal, the chances of these young children with pre-linguistic hearing loss acquiring normal speech and language are maximized. The study comprises two cohorts of two doses followed by an expansion cohort at the selected dose. While safety will be the primary endpoint of the first part of the dose escalation study, auditory brainstem response (ABR) will be the primary efficacy endpoint of the second part of the expansion. Audiogene will also evaluate the clinical safety, performance and ease-of-use of the delivery system developed by Sensorion.

About Sensorion

Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat, and prevent hearing loss disorders, a significant global unmet medical need. Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases, enabling it to select the best targets and mechanisms of action for drug candidates.

It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness, developed in the framework of its broad strategic collaboration focused on the genetics of hearing with the Institut Pasteur. SENS-501 (OTOF-GT) currently being developed in a Phase 1/2 clinical trial, targets deafness caused by mutations of the gene encoding for otoferlin and GJB2-GT targets hearing loss related to mutations in GJB2 gene to potentially address important hearing loss segments in adults and children. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses.

Sensorion’s portfolio also comprises programs of a clinical-stage small molecule, SENS-401 (Arazasetron), for the treatment and prevention of hearing loss disorders. Sensorion’s small molecule progresses in a Phase 2 proof of concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity (CIO) for the preservation of residual hearing. Sensorion, with partner Cochlear Limited, has completed in 2024 a Phase 2a study of SENS-401 for the residual hearing preservation in patients scheduled for cochlear implantation. A Phase 2 study of SENS-401 was also completed in Sudden Sensorineural Hearing Loss (SSNHL) in January 2022.

www.sensorion.com

Label: SENSORION

ISIN: FR0012596468

Mnemonic: ALSEN

Disclaimer

This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2024 full year report published on March 14, 2025, and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements. This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform oneself of any such local restrictions and comply therewith.

Contacts

Investor Relations
Sensorion

Nicolas Bogler, Investor Relations and Communication

ir.contact@sensorion-pharma.com

Press Relations
Maarc

Bruno Arabian / 00 33(0)6 87 88 47 26

bruno.arabian@maarc.fr
Nicolas Entz / 00 33 (0)6 33 67 31 54

nicolas.entz@maarc.fr

Galderma Welcomes Increased Equity Investment From L’Oréal




Galderma Welcomes Increased Equity Investment From L’Oréal

L’Oréal to acquire an additional 10% stake in Galderma recognizing its scientific leadership in dermatology and long-term growth potential

Ad hoc announcement pursuant to Art. 53 LR

ZUG, Switzerland–(BUSINESS WIRE)–Galderma Group AG (SIX:GALD), the pure-play dermatology category leader, today announced that L’Oréal Groupe intends to increase its equity investment in the company, acquiring an additional 10% stake from Sunshine SwissCo GmbH (a consortium led by EQT, Abu Dhabi Investment Authority (ADIA), and Auba Investment Pte. Ltd., acting as sellers).

Following the transaction, which is subject to customary approvals, L’Oréal’s total shareholding in Galderma will rise to 20%, building on its initial investment made in August 2024. The transaction is expected to close in Q1 2026.

In connection with this increased investment, Galderma will consider nominating two non-independent board candidates from L’Oréal, replacing the board members representing the consortium led by EQT, at the 2026 Annual General Meeting.

Additionally, Galderma and L’Oréal aim at exploring additional scientific research projects of mutual interest. This ongoing collaboration brings together Galderma’s scientific leadership in dermatology and L’Oréal’s expertise as the world’s leading beauty player. With a shared focus on skincare and skin health, innovation, and long-term growth, Galderma and L’Oréal are well positioned in the fast-growing, consumer-focused dermatology and beauty markets.

About Galderma

Galderma (SIX: GALD) is the pure-play dermatology category leader, present in approximately 90 countries. We deliver an innovative, science-based portfolio of premium flagship brands and services that span the full spectrum of the fast-growing dermatology market through Injectable Aesthetics, Dermatological Skincare and Therapeutic Dermatology. Since our foundation in 1981, we have dedicated our focus and passion to the human body’s largest organ – the skin – meeting individual consumer and patient needs with superior outcomes in partnership with healthcare professionals. Because we understand that the skin we are in shapes our lives, we are advancing dermatology for every skin story. For more information: www.galderma.com.

Forward-looking statements

Certain statements in this announcement are forward-looking statements. Forward-looking statements are statements that are not historical facts and may be identified by words such as “plans”, “targets”, “aims”, ” believes”, “expects”, “anticipates”, “intends”, “estimates”, “will”, “may”, “continues”, “should” and similar expressions. These forward-looking statements reflect, at the time, Galderma’s beliefs, intentions and current targets/ aims concerning, among other things, Galderma’s results of operations, financial condition, industry, liquidity, prospects, growth and strategies and are subject to change. The estimated financial information is based on management’s current expectations and is subject to change. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial consequences of the plans and events described herein. Actual results may differ from those set forth in the forward-looking statements as a result of various factors (including, but not limited to, future global economic conditions, changed market conditions, intense competition in the markets in which Galderma operates, costs of compliance with applicable laws, regulations and standards, diverse political, legal, economic and other conditions affecting Galderma’s markets, and other factors beyond the control of Galderma). Neither Galderma nor any of their respective shareholders (as applicable), directors, officers, employees, advisors, or any other person is under any obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. You should not place undue reliance on forward-looking statements, which speak of the date of this announcement. Statements contained in this announcement regarding past trends or events should not be taken as a representation that such trends or events will continue in the future. Some of the information presented herein is based on statements by third parties, and no representation or warranty, express or implied, is made as to, and no reliance should be placed on, the fairness, reasonableness, accuracy, completeness or correctness of this information or any other information or opinions contained herein, for any purpose whatsoever. Except as required by applicable law, Galderma has no intention or obligation to update, keep updated or revise this announcement or any parts thereof.

Contacts

For further information:

Media

Christian Marcoux, M.Sc.

Chief Communications Officer

christian.marcoux@galderma.com
+41 76 315 26 50

Richard Harbinson

Corporate Communications Director

richard.harbinson@galderma.com
+41 76 210 60 62

Investors

Emil Ivanov

Head of Strategy, Investor Relations and ESG

emil.ivanov@galderma.com
+41 21 642 78 12

Jessica Cohen

Investor Relations and Strategy Director

jessica.cohen@galderma.com
+41 21 642 76 43

Hercules Pharmaceuticals Approved as Authorized Distributor in Apexus 340B Prime Vendor Program




Hercules Pharmaceuticals Approved as Authorized Distributor in Apexus 340B Prime Vendor Program

NEW YORK–(BUSINESS WIRE)–Hercules Pharmaceuticals (Hercules), a national leader in pharmaceutical distribution, today announced that it has been approved as an authorized distributor in the 340B Prime Vendor Program distribution network, administered by Apexus, HRSA’s designated prime vendor for the 340B Drug Pricing Program.

Hercules joins the 340B Prime Vendor Program distribution network as an independent distributor with a governance model built for precision and transparency. The company’s disciplined operational framework, supported by advanced eligibility insight, standardized pricing processes, and clear reporting, provides hospitals and pharmacy partners with reliable 340B execution and consistent access to medications. By improving eligibility accuracy, reinforcing pricing integrity, and helping ensure that purchases align with the correct outpatient locations, Hercules delivers the analytics and operational clarity that enable hospitals to focus on patient care while giving manufacturers confidence in a well-governed distribution environment.

“We are honored to be approved as an authorized distributor within the 340B Prime Vendor Program distribution network,” said Sara Amani, Founder and CEO of Hercules. “At its core, the 340B program strengthens the ability of hospitals and health systems to care for the patients who rely on them most. Our approach is rooted in ethical access to medicine, operational precision, and a deep commitment to supporting providers as they stretch every dollar toward patient care. This authorization affirms our dedication to transparent, provider-centered distribution that enhances continuity of access for the patients and communities our partners serve.”

This step reflects a shared interest in supporting a 340B distribution infrastructure that is efficient, responsive, and focused on continuity of access to medicines. Hercules looks forward to contributing to the collaborative efforts that strengthen program integrity and promote best-practice execution within a well-established industry framework. As hospital systems face increasing complexity in administering 340B, spanning diversified outpatient footprints, contract pharmacy oversight, and heightened audit expectations, Hercules’ participation in the Prime Vendor Program brings a more stable, disciplined, and transparent channel to support their needs.

By leveraging the Apexus Prime Vendor Program’s negotiated value, distribution solutions, and technical assistance, Hercules will provide covered entities with a purchasing and compliance experience that reinforces operational continuity and accuracy, helping ensure that patients across diverse health system settings maintain uninterrupted access to the medicines they need.

About Hercules Pharmaceuticals

Hercules Pharmaceuticals is a national pharmaceutical distributor and steward of AriaGPO, a provider-aligned group purchasing organization (GPO). With a mission-driven focus and a commercial model built for resilience, Hercules helps healthcare providers reduce concentration risk, enhance financial performance, and ensure consistent access to critical therapies.

Hercules’ national distribution platform, bolstered by its proprietary GPO, AriaGPO, offers a distinct alternative to traditional models by combining structural agility with an elevated standard of service. Rather than relying on consolidated pipelines, Hercules operates as a true risk mitigation channel, providing redundancy and strategic diversification to health systems and pharmacies nationwide. The company’s direct relationships with a global network of U.S.-licensed manufacturers enable faster, more transparent sourcing and supply continuity.

Its fulfillment operations are built to flex across classes of trade, from large health systems and oncology clinics to specialty and retail partners, with customized logistics tailored to each provider. Central to Hercules’ differentiation is its High-Touch Partnership Model: every partner is supported by a dedicated client operations team that ensures seamless onboarding, responsive issue resolution, and personalized account management. These services are further enhanced by Aria’s embedded contracting and formulary tools, which align purchasing strategies with real-time clinical and financial objectives.

This integrated model empowers providers to enhance control over their supply chain strategy while securing uninterrupted access to high-value medicines. Hercules serves hospitals, health systems, specialty pharmacies, community practices, and retail providers across the U.S.

For more information, visit www.herculesrx.com and www.ariagpo.com.

Contacts

Media Contact:

press@herculesrx.com
1-800-815-5800

Sonrotoclax Data at ASH 2025 Confirm Foundational Potential Across B-cell Malignancies




Sonrotoclax Data at ASH 2025 Confirm Foundational Potential Across B-cell Malignancies

Novel BCL2 inhibitor sonrotoclax monotherapy demonstrates deep and durable clinical responses in R/R MCL and R/R CLL

Sonrotoclax in combination with BRUKINSA demonstrated rapid MRD negativity in treatment-naive CLL, regardless of high-risk features

SAN CARLOS, Calif.–(BUSINESS WIRE)–$ONC #BeOne—BeOne Medicines Ltd. (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, today announced new data on sonrotoclax, a next-generation investigational BCL2 inhibitor, demonstrating meaningful clinical benefit as monotherapy and in combination across B-cell malignancies. These data were featured at the 67th American Society of Hematology (ASH) Annual Meeting & Exposition in Orlando, Florida. The five presentations highlight durable responses in heavily pretreated patients with relapsed/refractory (R/R) mantle cell lymphoma (MCL) and additional studies showing deep, rapid, and sustained undetectable minimal residual disease (uMRD) rates with sonrotoclax-based combinations in patients with treatment-naive chronic lymphocytic leukemia (CLL), highlighting the foundational potential of this medicine.

“The data we’re presenting at ASH 2025 are redefining what physicians can expect from sonrotoclax as a next-generation BCL2 inhibitor,” said Amit Agarwal, M.D., Ph.D., Chief Medical Officer, Hematology, BeOne. “Our data demonstrates that sonrotoclax has succeeded where others have failed, achieving deep and durable responses as a monotherapy in both R/R CLL and MCL and notably fast kinetics as a combination therapy in treatment-naïve CLL. With these results, we believe sonrotoclax will become a foundational medicine in B-cell malignancies, potentially transforming outcomes for patients worldwide.”

Sonrotoclax could become the first BCL2 inhibitor indicated for R/R MCL in the U.S., based on data showing an overall response rate (ORR) of 52.4%. (Oral Presentation: 663; December 7 from 5:00-5:15 PM EST)

In this Phase 1/2, global, multicenter, single-arm, open-label study (NCT05471843), ORR by IRC was 52.4% (95% CI, 42.4-62.4) with a complete response (CR) rate of 15.5% (95% CI, 9.1-24.0) in patients with R/R MCL post-treatment with anti-CD20 therapy and a BTK inhibitor treated with 320 mg of sonrotoclax (n=103). Notably, ORR by IRC benefit was consistent across patients with high-risk disease subtypes, including patients with TP53 mutation, a key prognostic marker for MCL. In this patient group, ORR by IRC was 59.1% (95% CI, 36.3-79.3).

At a median study follow-up of 14.2 months (range, 0.3-24.9 months), the median duration of response (DOR) by IRC was 15.8 months (95% CI, 7.4 months-NE) and has yet to reach full maturity. The median time to response (TTR) was 1.9 months (range 1.6-6.5 months), and the median progression-free survival (PFS) was 6.5 months (95% CI: 4.0-10.4).

Treatment with sonrotoclax monotherapy was generally well tolerated, and adverse events were manageable. The most common grade ≥3 treatment-emergent adverse events (TEAEs) in greater than 10% of patients were neutropenia (19.1%), infections (16.5%), and pneumonia (10.4%).

These data are under Priority Review by the U.S Food and Drug Administration for potential accelerated approval.

“Achieving deep and durable responses in relapsed or refractory mantle cell lymphoma after BTK inhibitor therapy has been a long-standing challenge,” said Michael Wang, M.D., Professor, Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, and presenting author of the study. “In this analysis, sonrotoclax monotherapy demonstrated meaningful and lasting responses in heavily pretreated patients, including those with high-risk disease. These findings are highly encouraging and suggest this next-generation BCL2 inhibitor could play a foundational role in improving outcomes for patients with limited treatment options.”

Sonrotoclax combinations demonstrate fast responses with unmatched uMRD rates and notably better kinetics than current options

BGB-11417-101 (NCT04277637) is an ongoing, phase 1/1b, dose-escalation/expansion study in patients with B-cell malignancies. Results presented at ASH showcase data from sonrotoclax combinations in patients with treatment-naive (TN) CLL/SLL. Notable highlights include:

• Sonrotoclax plus zanubrutinib (Poster Presentation: 3891)
  • In 135 efficacy-evaluable patients, ORR was 100%, with CR/CR with incomplete count recovery (CRi) in 55% of the 320-mg cohort. Median TTR was 2.6 months (range, 1.5-10.8 months).
  • At 48 weeks of combination treatment, the uMRD4 rate in the 320-mg cohort was 91% and uMRD rates continue to increase over time, with 98% of patients achieving uMRD4 by 96 weeks.
    • Median time from the initiation of the combination to uMRD4 was 4.5 months.
  • With a median study follow-up of 30.9 months, no progression events have been observed in the 320-mg cohort, including in the 40% of patients (34 patients) who had electively discontinued treatment.
  • The combination was generally well tolerated, with no TEAEs leading to death, or clinical or laboratory tumor lysis syndrome (TLS).

• Sonrotoclax plus obinutuzumab (Oral Presentation: 793)
  • In the 320 mg efficacy-evaluable cohort (n=30), the ORR was 93%, with CR/CRi in 43% of patients.
  • The median time from reaching sonrotoclax target dose to uMRD was 2.3 months (range, 1.4-5.6 months) in the 320-mg cohort.
  • The combination was generally well tolerated, with no sonrotoclax discontinuations or deaths due to TEAEs.
  • Updated efficacy and safety data will be presented on Monday, December 8, 10:30 AM–10:45 AM EST.

• Sonrotoclax plus zanubrutinib and obinutuzumab (Poster Presentation: 3890)
  • In 15 efficacy-evaluable patients, the ORR was 100%, with a CR/CRi rate of 40%.
  • Of the MRD-evaluable patients (n=10), 100% achieved uMRD4, discontinued treatment as mandated by the protocol, and remain in remission; 80% of patients achieved uMRD6.
  • With a median study follow-up of approximately 18.0 months, no PFS events have occurred.
  • The combination was generally well tolerated, and no deaths or discontinuations of any study drug due to TEAEs were observed.

Sonrotoclax monotherapy achieves an ORR by IRC of 76%, with a CR/CRi of 19%, in patients with R/R CLL/SLL, demonstrating rapid and deep responses (Poster Presentation: 5666)

BGB-11417-202 (NCT05479994) is an open-label, phase 2, and a potential registrational study evaluating the efficacy and safety of sonrotoclax in 100 heavily pretreated patients with R/R CLL/SLL. At a median follow-up of 14.4 months (range, 0.2-27.5 months), primary analysis results show:

• Similar ORR and CR responses were seen in patients with unmutated IGHV, del(17p) and/or TP53 mutation, and BTK mutation. Median TTR was 3.7 months (range, 1.3-11.1 months).
• The best blood uMRD rate was 49.0% (n=49/100). Median time to blood uMRD4 was 5.8 months (range, 3-12 months).
• Sonrotoclax monotherapy was well tolerated, and toxicities were manageable and no clinical TLS occurred.
• Updated data from the abstract will be presented on Monday, December 8, 6:00-8:00 PM EST. Additionally, these data are under review by China’s National Medical Products Administration (NMPA) for potential accelerated approval.

For more information about our presence at the 2025 ASH Annual Meeting and Exposition, please visit our meeting hub: congress.beonemedicines.com.

About Mantle Cell Lymphoma

Mantle cell lymphoma (MCL) is a rare subtype of aggressive B-cell non-Hodgkin lymphoma (NHL)¹ that develops in B-cells located in the mantle zone of the lymph nodes. MCL accounts for approximately 5% of all NHL cases globally², affecting an estimated 28,000 people³. MCL is often diagnosed at advanced stages⁴ and nearly all MCL patients will eventually develop refractory or relapsed (R/R) disease.⁵ The five-year survival rate for MCL is approximately 50%, reflecting the urgent need for new therapeutic options.⁶

About Chronic Lymphocytic Leukemia

Chronic lymphocytic leukemia (CLL) is a life-threatening cancer of adults. It is a type of mature B-cell malignancy in which abnormal leukemic B lymphocytes (a type of white blood cells) arise from the bone marrow and flood peripheral blood, bone marrow, and lymphoid tissues.^7,8 CLL is the most common type of leukemia in adults, accounting for about one-third of new cases.^2,9

About Sonrotoclax (BGB-11417)

Sonrotoclax is a next-generation and potentially best-in-class investigational B-cell lymphoma 2 (BCL2) inhibitor with a unique pharmacokinetic and pharmacodynamic profile. Laboratory studies during early drug development have shown that sonrotoclax is a highly potent and specific BCL2 inhibitor with a short half-life and no drug accumulation. Sonrotoclax has shown promising clinical activity across a range of B-cell malignancies and is in development as a monotherapy and in combination with other therapeutics, including BRUKINSA. Notably, in early clinical trials, sonrotoclax plus BRUKINSA has demonstrated rapid and unprecedented rates of undetectable minimal residual disease (uMRD) in treatment-naïve patients with CLL. To date, more than 2,200 patients have been enrolled across the broad sonrotoclax global development program.

The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) for sonrotoclax for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL). In addition, the FDA has granted sonrotoclax Fast Track Designation for MCL and Waldenström macroglobulinemia, as well as Orphan Drug Designation for the treatment of adult patients with MCL, WM, multiple myeloma, acute myeloid leukemia, and myelodysplastic syndrome.

The information provided in this press release is intended for a global audience.

About BeOne

BeOne Medicines is a global oncology company domiciled in Switzerland that is discovering and developing innovative treatments that are more accessible to cancer patients worldwide. With a portfolio spanning hematology and solid tumors, BeOne is expediting development of its diverse pipeline of novel therapeutics through its internal capabilities and collaborations. With a growing global team of nearly 12,000 colleagues spanning six continents, the Company is committed to radically improving access to medicines for far more patients who need them.

To learn more about BeOne, please visit www.beonemedicines.com and follow us on LinkedIn, X, Facebook and Instagram.

Forward-Looking Statement

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the potential benefits of sonrotoclax; BeOne’s expectations regarding sonrotoclax’s clinical development, regulatory milestones, submissions and approvals; and BeOne’s plans, commitments, aspirations and goals under the caption “About BeOne.” Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeOne’s ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeOne’s ability to achieve commercial success for its marketed medicines and drug candidates, if approved; BeOne’s ability to obtain and maintain protection of intellectual property for its medicines and technology; BeOne’s reliance on third parties to conduct drug development, manufacturing, commercialization, and other services; BeOne’s limited experience in obtaining regulatory approvals and commercializing pharmaceutical products; BeOne’s ability to obtain additional funding for operations and to complete the development of its drug candidates and achieve and maintain profitability; and those risks more fully discussed in the section entitled “Risk Factors” in BeOne’s most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeOne’s subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeOne undertakes no duty to update such information unless required by law.

To access BeOne media resources, please visit our Newsroom.

Contacts

Investor Contact
Liza Heapes

+1 857-302-5663

ir@beonemed.com

Media Contact
Kyle Blankenship

+1 667-351-5176

media@beonemed.com