FDA Approves PIMJETMI® (pimicotinib) for Patients with Tenosynovial Giant Cell Tumor




FDA Approves PIMJETMI® (pimicotinib) for Patients with Tenosynovial Giant Cell Tumor

• PIMJETMI is a new once-daily oral systemic treatment option for certain U.S. patients with symptomatic tenosynovial giant cell tumor (TGCT)
• In the Phase 3 MANEUVER trial, PIMJETMI demonstrated early¹ and durable² tumor response with clinically meaningful improvements in all clinical outcome assessments, including physical function and pain
• PIMJETMI has shown high objective response rate (ORR) with a manageable safety profile

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

DARMSTADT, Germany–(BUSINESS WIRE)–Merck, a leading science and technology company, announced today that the U.S. Food and Drug Administration (FDA) has approved PIMJETMI^® (pimicotinib), a once-daily oral colony stimulating factor-1 receptor (CSF-1R) inhibitor, as a systemic treatment for adults with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection may cause worsening functional limitation or severe morbidity.

“People living with TGCT have long faced difficult treatment decisions as they navigate a disease that can significantly affect their daily lives including their mobility,” said Danny Bar-Zohar, CEO Healthcare and Member of the Executive Board for the Healthcare business of Merck. “Translating scientific innovation into clinically meaningful advances for people living with rare tumors is a strategic imperative for our company, as we continue our commitment to addressing significant unmet needs. With the approval of PIMJETMI in the U.S., we strengthen our leadership position and growing portfolio in rare tumors and bring real hope to patients and their families.”

TGCT is a rare, locally aggressive tumor occurring in or around the joints leading to progressive worsening of symptoms including pain and reduced mobility of the affected joint, significantly impacting physical function in what is typically an otherwise healthy and young population. It has been reported that up to half of people with TGCT can be initially misdiagnosed, resulting in delays in the appropriate disease management.³ If left untreated or in recurrent cases, TGCT may result in cumulative morbidity and irreversible damage to the bone, joint and surrounding tissues.

“The results of the Phase 3 MANEUVER trial demonstrate that PIMJETMI elicits durable² tumor response with a manageable tolerability profile,” said Vinod Ravi, MD, MS, MBA, Department of Sarcoma Medical Oncology, Division of Cancer Medicine, UT MD Anderson, an investigator on the MANEUVER trial. “This combination of efficacy and safety data will potentially address the needs of people living with TGCT in a once-daily treatment option.”

FDA approval is based on primary and longer-term results from the global Phase 3 MANEUVER study. PIMJETMI demonstrated a statistically significant improvement in objective response rate (ORR) at week 25 as assessed by blinded independent review committee (BIRC) based on RECIST v1.1 compared with placebo (56% vs. 3% for placebo; p<0.0001). PIMJETMI also demonstrated clinically meaningful and statistically significant improvements as compared to placebo across secondary endpoints relevant to patients’ daily lives, improving relative range of motion (p=0.0003), physical functioning (p=0.0074), and pain (p<0.0001). The MANEUVER study was presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. In the longer-term follow up (median 14.3 months for patients initially randomized to pimicotinib) presented at the European Society for Medical Oncology (ESMO) Congress in 2025, the median duration of response was not reached. The majority (77%) of patients with response at 25 weeks maintained clinical benefit after an additional 12 months of follow up, demonstrating the durability² of response with PIMJETMI.

In MANEUVER, pimicotinib was well-tolerated, with no evidence of cholestatic hepatotoxicity or hair/skin hypopigmentation. During the randomized, double-blind treatment phase of the trial, treatment-emergent adverse events (TEAEs) leading to treatment discontinuation occurred in one patient (1.6%) treated with pimicotinib; TEAEs leading to dose reduction occurred in 7.9% (n=5) of pimicotinib-treated patients.

“TGCT can profoundly affect patients’ daily lives through persistent pain, swelling, and loss of joint function,” said Sydney Stern, PhD, MS, TGCT Support, a Program of the Life Raft Group. “For symptomatic patients who are not likely to benefit from surgery alone, there remains need for treatment options which are tolerable and effectively address the burden of disease, shrink their TGCT, and improve pain and function.”

The FDA approval of PIMJETMI builds on growing momentum for the Merck rare tumors portfolio that included two other drug approvals in the past three years including OGSIVEO^®, the first FDA-approved treatment for adults with progressing desmoid tumors who require systemic therapy. The PIMJETMI approval follows its approval in China by the National Medical Products Administration (NMPA) in December 2025, in Canada by Health Canada in June 2026 for the treatment of adult patients with symptomatic TGCT, as well as in Switzerland by its own independent national regulatory authority, the Swissmedic in October 2026.

The application for PIMJETMI was reviewed under Project Orbis, an international initiative of the FDA Oncology Center of Excellence. Application reviews are ongoing for partners of Project Orbis, an international framework for collaboration.

¹ In the global Phase 3 MANEUVER clinical trial, the effect of pimicotinib had an early onset, with 41.3% (26 of 63) of patients experiencing objective response to therapy after 13 weeks.
² The majority (77%) of patients with response at 25 weeks maintained this clinical benefit after an additional 12 months of treatment.

³ Stern S, et al. Localized and diffuse tenosynovial giant cell tumor: real-world results from a patient observational registry. Future Oncol. 2025;21(12):1501-1510.

About MANEUVER

The pivotal, global, Phase 3 MANEUVER study is a three-part, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of PIMJETMI in patients with TGCT who were eligible for systemic therapy and have not received prior anti-CSF-1/CSF-1R therapy.

The ongoing study is being conducted by Abbisko Therapeutics and enrolled patients globally in USA, Canada and Europe (n=49) and China (n=45).

In the double-blind Part 1, 94 patients were randomized 2:1 to receive either 50 mg QD of PIMJETMI (n=63) or placebo (n=31) for 24 weeks. In addition to the primary endpoint of ORR at week 25, as measured by RECIST v1.1 by BICR, secondary endpoints include tumor volume score (TVS), relative range of motion, pain by Brief Pain Inventory (BPI), and physical function measured by Patient-Reported Outcomes Measurement Information System (PROMIS-PF).

After the double-blind Part 1, all eligible patients from either arm could continue to the open-label Part 2 and receive up to 24 weeks of pimicotinib treatment. Patients who completed Part 2 could then enter the open-label extension phase (Part 3) for extended treatment and safety follow-up.

About PIMJETMI^® (pimicotinib)

PIMJETMI, developed by Abbisko Therapeutics, is a novel, orally administered, highly selective and potent small-molecule inhibitor of CSF-1R. PIMJETMI has been approved for the treatment of adult patients with symptomatic TGCT for which surgical resection will potentially cause functional limitation or relatively severe morbidity by China National Medical Products Administration (NMPA) and Health Canada. PIMJETMI has received priority medicine (PRIME) designation from the European Medicines Agency (EMA). Merck holds worldwide commercialization rights for PIMJETMI.

About Merck

Merck, a leading science and technology company, operates across life science, healthcare and electronics. More than 62,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From providing products and services that accelerate drug development and manufacturing as well as discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2025, Merck generated sales of € 21.1 billion in 65 countries.

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

All Merck press releases are distributed by e-mail at the same time they become available on the Merck website. Please go to www.merckgroup.com/subscribe to register online, change your selection or discontinue this service.

Contacts

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

Investor Relations

investor.relations@merckgroup.com
Phone: +49 6151 72-3321

Qureight’s Fibrosis Imaging Biomarker Selected by Contineum Therapeutics as a Secondary Endpoint in Phase 2 PROPEL-IPF Clinical Trial




Qureight’s Fibrosis Imaging Biomarker Selected by Contineum Therapeutics as a Secondary Endpoint in Phase 2 PROPEL-IPF Clinical Trial

• Qureight’s Fibr8 model to provide structural assessment as secondary endpoint in Contineum’s Phase 2 PROPEL-IPF clinical trial examining the safety and efficacy of PIPE-791 in idiopathic pulmonary fibrosis (IPF) patients
• Announcement comes after a recent European Respiratory Journal viewpoint by Qureight CSO identifying fibrosis quantification as the key structural imaging biomarker needed for antifibrotic trials
• PIPE-791 targets lysophosphatidic acid receptor 1 (LPAR1), a clinically validated target for IPF

CAMBRIDGE, England & SAN DIEGO–(BUSINESS WIRE)–Qureight, an end-to-end imaging company providing enterprise-grade imaging and precision endpoints for clinical trials for lung and heart disease, and Contineum Therapeutics, Inc. (Contineum) (NASDAQ:CTNM), a clinical-stage biopharmaceutical company pioneering differentiated small molecule therapies for inflammatory and fibrotic diseases with significant unmet need, today announced that Qureight’s Fibr8™ quantitative imaging biomarker is being used as a secondary endpoint in Contineum’s PROPEL-IPF Phase 2 clinical trial examining the safety and efficacy of PIPE-791, Contineum’s lead drug candidate, in idiopathic pulmonary fibrosis (IPF) patients.

Forced Vital Capacity (FVC), the primary endpoint in PROPEL-IPF, provides an established measure of lung function in IPF patients. Qureight’s AI-powered Fibr8™ imaging biomarker complements FVC by measuring the extent of fibrosis visible on High-Resolution Computed Tomography (HRCT), providing an assessment of structural disease burden. By examining both physiological and quantitative imaging measurements, PROPEL-IPF will investigate treatment effects on both lung function and imaging-defined measures of fibrosis progression, potentially generating valuable evidence to support the development of imaging endpoints for future clinical trials.

PROPEL-IPF (NCT07284459) is a global, randomised, double-blind, placebo-controlled Phase 2 clinical trial evaluating the efficacy, safety, and tolerability of once-daily oral PIPE-791 with 26 weeks of treatment in approximately 324 people with IPF. PIPE-791 targets the lysophosphatidic acid receptor 1 (LPAR1), a clinically validated target for IPF.

Qureight recently published a Viewpoint in the European Respiratory Journal advocating for the integration of quantitative fibrosis measurements alongside established physiological endpoints in antifibrotic clinical trials¹. PROPEL-IPF represents an important opportunity to try to advance this approach by potentially generating prospective evidence on the relationship between treatment-related changes in lung fibrosis and pulmonary function. This would strengthen the evidence base for quantitative CT as a sensitive measure of therapeutic response and support Qureight’s longer-term ambition to pursue formal regulatory qualification of imaging biomarkers as endpoints in antifibrotic drug development.

Simon Walsh, MD, PhD, Chief Scientific Officer, Qureight, commented: “PROPEL-IPF is a strong example of the endpoint model we believe the field needs: preserve FVC as the functional anchor, while measuring fibrosis directly on HRCT. The combination has the potential to help reveal whether a therapy is slowing the structural disease process as well as its downstream functional consequences.”

Timothy Watkins, MD, MSc, Chief Medical Officer, Contineum Therapeutics, added: “Qureight’s approach to quantitatively measure fibrosis could provide important insights in PROPEL-IPF. We are excited for our team to partner with the clinical scientists at Qureight, leveraging this imaging technology platform to enable lung-specific mechanistic insight into the effects of PIPE-791. Ultimately, this measure has the potential to serve as an additional means by which we can assess the impact of PIPE-791 treatment on underlying fibrosis pathology in IPF.”

1. Press release (2 November 2026) Qureight publishes in the European Respiratory Journal calling for fibrosis burden to become the principal structural imaging biomarker for antifibrotic trials

Forward-Looking Statements

Certain statements contained in this press release, other than historical information, constitute forward-looking statements of Contineum Therapeutics, Inc. (“Contineum”, the “Company”, “we”, “our”, or “us”) within the meaning of the federal securities laws. Forward-looking statements include, but are not limited to, implied or express statements regarding the Company’s PROPEL-IPF clinical trial; the Company’s use of Qureight’s Fibr8 model in its PROPEL-IPF clinical trial and its ability to provide a complementary structural assessment of disease burden or to generate or provide valuable evidence or important insights; the future expectations, plans and prospects for the Company; the indications, anticipated benefits of, and market opportunities for the Company’s drug candidates; the Company’s business strategies and plans; and the quotations of the Company’s management. These statements involve known and unknown risks, uncertainties and other important factors that are in some cases beyond the Company’s control and may cause its actual results, events, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These risks and uncertainties, include, but are not limited to, the following: the Company is heavily dependent on the success of PIPE-791 which is in the early stages of clinical development, and PIPE-791, and the Company’s other drug candidates, may not progress through clinical development or receive regulatory approval; the results of preclinical studies and clinical trials, including those conducted by third parties, may not be predictive of future results and unexpected adverse side effects or inadequate efficacy of the Company’s drug candidates may limit their development, regulatory approval and/or commercialization; the timing and outcome of research, development and regulatory review is uncertain; the FDA or comparable foreign regulatory authorities may disagree as to the design or implementation of our proposed clinical trials and/or require us to conduct additional studies or clinical trials or provide additional patient data, which could be costly, time-consuming and difficult to complete; clinical trials and preclinical studies may not proceed at the time or in the manner expected, or at all; the Company may use its capital resources sooner than expected and they may be insufficient to allow the Company to achieve its anticipated milestones; the potential for the Company’s programs and prospects to be negatively impacted by developments relating to the Company’s competitors, including the results of studies or regulatory determinations relating to the Company’s competitors; risks associated with reliance on third parties to successfully conduct clinical trials; the Company has incurred significant operating expenses since inception and it expects that its operating expenses will continue to significantly increase for the foreseeable future; the Company’s ability to operate in a competitive industry and compete successfully against competitors that have greater resources than the Company does; the Company may be unable to obtain, maintain and enforce intellectual property protection for its technology and drug candidates; and unstable market and economic conditions and military conflicts may adversely affect the Company’s business and financial condition and the broader economy and biotechnology industry. Additional risks and uncertainties that could affect the Company’s business, operations and results are included under the captions, “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in the Company’s periodic filings and in other filings that the Company makes with the Securities and Exchange Commission (SEC) from time to time, which are available on the Company’s website at www.contineum-tx.com under the Investor section and on the SEC’s website at www.sec.gov. Accordingly, readers should not rely upon forward-looking statements as predictions of future events. Except as required by applicable law, the Company undertakes no obligation to update publicly or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Contacts

For further information, please contact:

Zyme Communications
Katie Odgaard

Email: katie.odgaard@zymecommunications.com
Tel: +44 (0)7787 502 947

Qureight
Rebecca Simmons, COO

Email: r.simmons@qureight.com

Contineum
Steve E. Kunszabo, Senior Director, Investor Relations & Corporate Communications

Email: skunszabo@contineum-tx.com
Tel: +1 858 649 1158

Avanzanite Announces Exclusive Licence Agreement to Commercialise Zemcelpro® in Europe




Avanzanite Announces Exclusive Licence Agreement to Commercialise Zemcelpro® in Europe

• Zemcelpro is authorised in the European Union for eligible adults with blood cancers who need an allogeneic stem cell transplant and have no other suitable donor cells available.
• Avanzanite secures exclusive commercialisation rights to Zemcelpro across 32 countries covering the European Economic Area, UK and Switzerland including potential indication expansion.
• Avanzanite will become the EU marketing authorisation holder following regulatory transfer and support a planned Phase 3 trial.
• Zemcelpro becomes Avanzanite’s fifth rare disease launch and first advanced therapy medicinal product (ATMP).

AMSTERDAM–(BUSINESS WIRE)–#NoOneLeftBehind–Avanzanite Bioscience B.V. (“Avanzanite” or “Company”) a European pharmaceutical company focused on rare diseases, today announced an exclusive licence agreement with Montreal-based Cordex Biologics, an ExCellThera company (“ExCellThera”), a leader in enhanced blood stem cell therapies, to commercialise Zemcelpro^®. Zemcelpro received conditional marketing authorisation from the European Commission on 25 August 2025 for the treatment of adult patients with haematological malignancies requiring an allogeneic haematopoietic stem cell transplant (allo-HSCT) following myeloablative conditioning for whom no other type of suitable donor cells is available.

Under the agreement, Avanzanite will lead commercialisation across 32 countries including the European Economic Area, UK and Switzerland. Avanzanite will become the holder of the existing EU marketing authorisation for Zemcelpro and plans to submit marketing authorisation applications in the UK and Switzerland. ExCellThera will retain responsibility for manufacturing and supply and will continue to lead global development across potential indication expansion in specific patients.

Furthermore, Avanzanite will contribute to the cost of a planned Phase 3 trial evaluating Zemcelpro against haploidentical or mismatched unrelated donor transplantation in adult patients with high-and very high-risk acute leukaemias and myelodysplastic syndromes (MDS). The trial is intended to provide confirmatory evidence of efficacy and safety and may support a potential indication expansion.

“This partnership reflects why Avanzanite was founded: to unlock the full potential of innovative medicines for patients with rare diseases in Europe,” said Adam Plich, Co-founder and CEO, Avanzanite Bioscience. “We believe that while discovering breakthrough medicines requires one kind of genius, ensuring those medicines reach patients who can benefit from them requires another. This partnership unites these strengths in a shared sense of purpose.” Plich continues: “Our commitment also extends to supporting the funding of the planned Phase 3 trial to gather further evidence and explore the therapy’s potential for broader use.”

Zemcelpro has orphan drug designation and was part of the European Medicines Agency’s PRIority MEdicines (PRIME) scheme during development. This scheme provides early and enhanced scientific and regulatory support for promising medicines addressing unmet medical needs.

“Our team and collaborators have worked relentlessly for many years to advance Zemcelpro from scientific discovery towards patient care,” said David Millette, CEO of ExCellThera. “Avanzanite brings a pan-European footprint with deep expertise in rare disease launches and market access. We believe they are the ideal partner to help turn the promise of this science into impact for patients in need while allowing us to continue focusing on scientific and clinical development.”

“For patients with blood cancers who require a donor stem cell transplant, finding suitable donor cells is a vital step in the treatment pathway,” said Dr Jurjen Versluis, Internist-Haematologist, Erasmus MC Cancer Institute, Rotterdam, the Netherlands. “When no other suitable donor cells are available, additional transplant options are important. Erasmus MC contributed to the clinical trials evaluating Zemcelpro, and we are pleased to have played a role in its clinical development.”

The agreement advances Avanzanite’s ambition to become a European rare disease champion. With this fifth rare disease launch in four years, Zemcelpro extends the company’s haematology portfolio into stem cell therapy and haemato-oncology, alongside its ophthalmology and nephrology franchises. Founded in 2022, Avanzanite now has more than 110 employees across Europe and serves more than 400 patients.

About Zemcelpro^®

Zemcelpro is an allogeneic haematopoietic stem cell therapy authorised as a medicinal product in the European Union for use in eligible adults requiring allogeneic haematopoietic stem cell transplantation (allo-HSCT). It is made specifically for each patient from a single umbilical cord blood unit and contains two components: expanded CD34+ haematopoietic stem cells using proprietary UM171 technology (dorocubicel) and unexpanded CD34–cells. Following infusion, the CD34+ stem cells travel to the bone marrow where they divide and mature into specialised blood and immune cells. The unexpanded CD34–cells, consisting primarily of CD3+ T cells, play a complementary role by supporting immune reconstitution and providing graft-versus-leukaemia effects post-transplantation.

About Allogeneic Stem Cell Transplantation

Each year around 15,000 adults in the licensed territories require allogeneic haematopoietic stem cell transplantation (HSCT) as a potentially curative treatment for blood cancers such as acute myeloid leukaemia (AML), acute lymphoblastic leukaemia (ALL), myelodysplastic syndromes (MDS), lymphoma and other diseases. However, one third of these patients urgently need a transplant but have no matched related donor. Most receive transplants from haploidentical or mismatched unrelated donors, which have an increased risk of serious complications, including graft-versus-host disease (GVHD) and infections, contributing to non-relapse mortality. Some are unable to receive a transplant at all and face the risk of relapse with little prospect of survival.

About Avanzanite Bioscience

Avanzanite is a next-generation pharmaceutical company purpose-built to unlock the full potential of rare disease medicines across Europe. Founded in 2022 and headquartered in Amsterdam, the Netherlands, Avanzanite was created around a belief that scientific innovation creates patient impact only when medicines reach those who can benefit from them. Through tailored partnerships, biopharma innovators entrust Avanzanite with the European future of their medicines. Avanzanite combines pan-European expertise, bold commercial and access strategy, together with high-performance execution, to transform Europe’s complexity into opportunity, ensuring no patient is left behind and creating lasting long-term value for partners.

For more information, visit www.avanzanite.com and follow us on LinkedIn.

About ExCellThera

ExCellThera is a world leader in enhanced blood stem cell therapies. Its technology platform is designed to expand HSCs from any source while counteracting the cellular stress associated with cell culture or gene editing.

For additional information, visit www.excellthera.com and follow on LinkedIn.

Contacts

Avanzanite Bioscience B.V.

Phone: +31 20 301 21 13

Email: media@avanzanite.com
Website: www.avanzanite.com

Almirall 9M 2026 Results




Almirall 9M 2026 Results

Almirall continues to deliver solid results at 9M 2026, growing its European Dermatology business 17.6% YoY, increasing total Net Sales by 8.3%, and EBITDA by 22.7% – reiterating full-year guidance

• Total Almirall Net Sales increased by 8.3% YoY to €889.2 MM, while EBITDA increased by 22.7% reaching €221.7 MM, delivering 24.9% margin based on strong commercial and operational execution.
• The European Dermatology business increased 17.6% YoY to €520.3 MM, supported by the continued performance of key growth drivers.
• The biologics portfolio keeps driving Almirall’s growth, with Net Sales of Ebglyss® increasing 75.0% YoY to €132.1 MM, and Ilumetri® increasing 11.5% YoY to €190.6 MM. The scientific evidence base for Ebglyss® was further strengthened at EADV 2026, with 5-year ADlong results confirming long-term disease control in moderate-to-severe atopic dermatitis, and ADtouch data (by Eli Lilly) showing rapid and significant improvements in hand and foot atopic dermatitis.
• The broader dermatology portfolio maintains strong performance, including Wynzora® (increasing 15.4% to €29.3 MM) and Klisyri® (increasing 18.4% to €23.8 MM).
• Almirall continues to advance its innovation strategy with six potentially disruptive programmes – four currently in PoC/Phase II and two additional PoC studies planned to be started towards the end of the year – targeting five dermatological diseases in total, including the rare disease pyoderma gangrenosum with the anti-IL-1RAP mAb.
• Reiterated guidance for the full year 2026: 9-12% Net Sales growth and €270 MM to €290 MM EBITDA.

BARCELONA, Spain–(BUSINESS WIRE)–Almirall, S.A. (ALM), a global pharmaceutical company dedicated to medical dermatology, today announced its results for the first nine months of 2026. Net Sales rose 8.3% YoY to €889.2 MM and EBITDA grew 22.7% to €221.7 MM, representing a margin of 24.9%. With steady performance in line with its long-term growth trajectory, Almirall reiterated its full-year 2026 guidance.

The European Dermatology business remained the main engine of Almirall’s growth, with Net Sales up 17.6% YoY to €520.3 MM on the back of continued momentum across the company’s key growth drivers.

The biologics portfolio continued its positive performance as a key contributor to the overall business growth. Net Sales of Ebglyss® (lebrikizumab, moderate-to-severe atopic dermatitis) reached €132.1 MM, up 75.0% YoY, as its market share continued to increase across launched markets. Ilumetri® (tildrakizumab, moderate-to-severe plaque psoriasis) demonstrated continued growth with sales increasing 11.5% YoY to €190.6 MM, further strengthening its established position in the psoriasis segment.

The Ebglyss® scientific evidence base was further strengthened at the 2026 European Academy of Dermatology and Venereology (EADV) Congress. Late-breaking 5-year results from the ADlong study confirmed long-term disease control in moderate-to-severe atopic dermatitis, while ADtouch data, presented by Almirall’s partner Eli Lilly and Company, showed rapid and significant improvements in moderate-to-severe hand and foot atopic dermatitis, highlighting the efficacy of lebrikizumab in hard-to-treat areas.

The broad dermatology portfolio continued to contribute to Almirall’s sustained growth. Wynzora® (psoriasis) sales increased by 15.4% YoY to €29.3 MM and Klisyri® (actinic keratosis) sales increased by 18.4% to €23.8 MM.

Almirall continues to advance its innovation strategy and is progressing six potentially disruptive programmes – four currently in PoC/Phase II studies, and two additional PoC studies to be started towards the end of the year, together targeting five dermatological diseases. Almirall also announced pyoderma gangrenosum, a rare and debilitating inflammatory skin disease, as the target indication for its anti-IL-1RAP monoclonal antibody.

2026 Full Year Guidance

Full year guidance for 2026: Net Sales: 9% to 12% growth.

EBITDA between €270 MM to €290 MM.

R&D pipeline

Innovation remains central to Almirall’s growth strategy and its ambition to lead in medical dermatology. The company’s R&D efforts are rooted in a deep scientific understanding of skin diseases, with the aim of turning research into meaningful new options for patients and dermatologists. In the first nine months of 2026, R&D investment represented approximately 12% of Net Sales.

Four PoC/Phase II studies are currently under way:

• an anti-IL-1RAP and an anti-IL-21 monoclonal antibodies targeting hidradenitis suppurativa
• an IL-2muFc mutant fusion protein programme targeting alopecia areata and atopic dermatitis

Two further PoC studies are planned to be started towards the end of the year:

• systemic lupus erythematosus (SLE) with cutaneous manifestations, within the IL-2muFc fusion protein
• pyoderma gangrenosum, a rare and debilitating inflammatory skin disease, with the anti-IL-1RAP monoclonal antibody

The first read-outs are expected in this quarter.

During the third quarter, Almirall entered into a collaboration and licensing agreement with CrystalO for the discovery and development of novel small molecules in medical dermatology, leveraging CrystalO’s proprietary ion-channel technology in combination with Almirall’s development and commercial capabilities.

Alongside its pipeline, Almirall is building further clinical and real-world evidence for Ebglyss® (lebrikizumab) and Ilumetri® (tildrakizumab) through lifecycle management programmes and collaborative activities with partners Lilly and Sun Pharma.

Partnership with the dermatology and science communities

Almirall works closely with dermatologists, patients, researchers and strategic partners to advance medical dermatology. This collaborative approach is at the core of Almirall’s strategy in medical dermatology and supports scientific exchange, deepens knowledge of skin diseases and keeps the company’s innovation focused on what matters most to patients and the physicians who treat them.

As Gold Sponsor of the 2026 EADV Congress in Vienna, Almirall presented more than 20 abstracts, including late-breaking ADlong results that highlighted sustained improvement in skin signs and itch with up to five years of treatmentⁱ. Almirall also hosted two expert-led, educational symposia on AD and PsO, an interactive industry hub session focused on AD and a dedicated R&D corner at the exhibition booth providing opportunities for scientific exchange on the new evidence and its relevance to clinical practice.

Investor Calendar 2027

FY 2026 Financial Results – February 22^nd 2027

Q1 2027 Financial Results – May 10^th 2027

H1 2027 Financial Results – July 23^rd 2027

Q3 2027 Financial Results – November 15^th 2027

About Almirall

Almirall is a global biopharmaceutical company dedicated to medical dermatology. We closely collaborate with leading scientists, healthcare professionals, and patients to deliver our purpose: to transform the patients’ world by helping them realize their hopes and dreams for a healthy life. We advance the scientific understanding of skin diseases to develop innovative and differentiated treatments and solutions that address patients’ needs. Our clinical development portfolio includes four ongoing proof-of-concept (PoC)/Phase II studies and two further PoC studies planned across key areas of medical dermatology.

Almirall, founded in 1944 and headquartered in Barcelona, is publicly traded on the Spanish Stock Exchange (ticker: ALM, total revenue in 2025: €1114.5 MM, over 2100 employees globally). Almirall products help to improve the lives of patients every day and are available in over 100 countries.

For more information, please visit https://www.almirall.com/

Legal notice:

This document includes only summary information and is not intended to be exhaustive. The facts, figures, and opinions contained in this document, in addition to the historical ones, are “forward-looking statements.” These statements are based on the information currently available and the best estimates and assumptions that the Company considers reasonable. These statements involve risks and uncertainties beyond the control of the Company. Therefore, actual results may differ materially from those declared by such forward-looking statements. The Company expressly disclaims any obligation to revise or update any forward-looking statements, goals, or estimates contained in this document to reflect any changes in the assumptions, events, or circumstances on which such forward-looking statements are based, unless required by the applicable law.

Contacts

Corporate Communications:
corporate.communication@almirall.com
Phone: +34 93 291 35 08


Investor Relations
investors@almirall.com
Phone: (+34) 93 291 30 87

Alight Appoints Vikram Kalra as India Country Leader to Advance Global Delivery and Transformation




Alight Appoints Vikram Kalra as India Country Leader to Advance Global Delivery and Transformation

Seasoned executive will lead Alight’s India strategy, performance and continued evolution as a global delivery and capability hub

DEERFIELD, Ill.–(BUSINESS WIRE)–Alight, Inc. (NYSE: ALIT), a leading benefits administration provider of health, wealth, and leave solutions, today announced the appointment of Vikram Kalra as India Country Leader. Kalra will lead the strategy, performance and continued evolution of Alight’s India offices, with a focus on service and operational excellence, strong execution and expanding India’s contribution as a global delivery and capability hub.

Kalra brings more than 25 years of global experience in enterprise transformation and operational excellence across organizations including Mercer, Aon and MetLife. He has led multidisciplinary teams and complex portfolios spanning insurance, health, pension and retirement benefits and retail banking.

In his new role, Kalra will bring Alight’s businesses and functions in India together around a unified strategy and execution agenda, while strengthening the connection between India and the company’s global organization. His priorities will include client delivery, technology, talent and capability development, productivity, culture, risk and the colleague experience. Kalra’s appointment is part of Alight’s continued focus on strengthening leadership, improving execution and bringing critical client-service capabilities under closer Alight ownership.

“India is critical to how we serve our clients and to Alight’s continued transformation,” said Allison Bassiouni, Chief Delivery Officer at Alight. “We are building on our momentum in India and constantly striving to deliver with excellence for our clients. Vikram’s operational and transformation expertise combined with his client focus will bolster the already strong foundation we’ve built in India.”

Kalra will report to Bassiouni and lead the India Leadership Team, bringing together leaders across delivery, technology, human resources, finance and legal. A near-term priority will be strengthening the integration of Alight’s India operations as the company builds on its recent work to bring critical client-service capabilities closer to Alight, with a focus on stronger accountability, collaboration and execution.

The transition is designed to strengthen direct accountability, collaboration and access to Alight leadership, tools, training and innovation investments. Kalra will focus on building on that foundation as Alight continues to strengthen India’s role in global delivery, technology, talent, capability and innovation.

“India has deep talent, strong capabilities and a growing role in Alight’s global organization,” said Kalra. “I look forward to building on those strengths with our leadership team to deliver excellent execution, stronger connections across our teams and better outcomes for our clients. I’m excited to work with colleagues across India and around the world to build on what is already here and create the next chapter for Alight in India.”

About Alight Solutions

Alight is a leading benefits administration provider of health, wealth, leave and point solutions for many of the world’s largest organizations and over 30 million people. Through the administration of employee benefits, Alight helps clients gain a benefits advantage while building a healthy and financially secure workforce by unifying the benefits ecosystem across health, wealth, wellbeing, absence management and navigation. Our Alight Worklife® platform empowers employers to gain a deeper understanding of their workforce and engage them throughout life’s most important moments with personalized benefits management and data-driven insights, leading to increased employee wellbeing, engagement and productivity. Learn more at alight.com.

Contacts

Media
Stephanie Lerdall

mediarelations@alight.com

Veradermics to Hold Conference Call to Discuss 12-Month Long-Term Results from Phase 3 ‘302’ Clinical Trial of VDPHL01 in Males with Mild-to-Moderate Pattern Hair Loss




Veradermics to Hold Conference Call to Discuss 12-Month Long-Term Results from Phase 3 ‘302’ Clinical Trial of VDPHL01 in Males with Mild-to-Moderate Pattern Hair Loss

Conference call will take place on Monday, November 9, 2026, at 8:00 am ET

NEW HAVEN, Conn.–(BUSINESS WIRE)–Veradermics, Incorporated (NYSE: MANE), a dermatologist-founded, late-stage biopharmaceutical company focused on developing innovative therapeutics for pattern hair loss, today announced it will host an investor call and live webcast on Monday, November 9, 2026, at 8:00 am ET to review topline results from Part B of its randomized, double-blind, placebo-controlled registration-directed Phase 3 clinical trial (Study ‘302’) evaluating VDPHL01, a proprietary, extended-release oral minoxidil formulation, in over 500 males with mild-to-moderate pattern hair loss.

Webcast Details:

The event and the accompanying slides will be available on the Events page in the Investors section of the company’s website. A replay will be available following the call.

About VDPHL01

VDPHL01 (extended-release minoxidil tablet) is a proprietary investigational, oral non-hormonal drug in Phase 3 development for pattern hair loss in both women and men. VDPHL01 leverages extended-release technology to deliver a minoxidil product with the potential for improved efficacy and safety. The proprietary extended-release formulation utilizes a gel matrix designed to deliver long-lasting, steady release of minoxidil for sustained absorption. VDPHL01 has been shown to avoid the high peak concentrations of immediate-release oral minoxidil, while extending time above the minimum hair growth threshold to increase time for hair to grow. If approved, VDPHL01 would be the only FDA-approved oral non-hormonal treatment for pattern hair loss in both male and female patients. VDPHL01 is protected by a broad library of patents and patent applications related to the key innovations of VDPHL01. The earliest expiring patent term is 2043.

About Pattern Hair Loss

Pattern hair loss, also known as androgenetic alopecia, affects an estimated 80 million people in the United States (30 million women and 50 million men). Pattern hair loss can have a significant impact on quality of life, affecting an individual’s mental health and relationships. People with pattern hair loss often experience depression, low self-esteem, and social withdrawal. There have been no new FDA-approved prescription medicines for pattern hair loss in nearly 30 years. In addition, current treatments include over-the-counter “nutraceuticals” that produce inconsistent results and contribute to high dissatisfaction among patients and healthcare providers. The prevalence of pattern hair loss and the market demand for new treatments contribute to making it the largest aesthetics market worldwide, projected to reach approximately $30 billion by 2028.

About Veradermics, Inc.

Veradermics is a dermatologist-founded, late clinical-stage biopharmaceutical company focused on developing innovative therapeutics for pattern hair loss. Veradermics aims to develop a focused portfolio of aesthetic dermatology product candidates targeting high-prevalence dermatologic conditions, with potential selective development of medical dermatology product candidates. Its lead program, VDPHL01, is being developed as an oral, non-hormonal treatment for men and women with pattern hair loss, to reduce the barriers to wide adoption of chronic hair loss therapy and potentially transform pattern hair loss treatment. VDPHL01 is an oral, extended-release proprietary formulation of minoxidil, a proven hair growth agent, designed to maximize minoxidil’s impact on hair restoration while minimizing the risk of cardiac activity. For additional information, visit www.veradermics.com and follow us on LinkedIn and Instagram.

Contacts

Media:

Catherine Collier Kyroulis

917-886-5586

media@veradermics.com

Investors:

investors@veradermics.com

Vir Biotechnology Announces Primary Endpoint Met in Pivotal Phase 3 ECLIPSE 1 Trial for Elebsiran and Tobevibart in Chronic Hepatitis Delta




Vir Biotechnology Announces Primary Endpoint Met in Pivotal Phase 3 ECLIPSE 1 Trial for Elebsiran and Tobevibart in Chronic Hepatitis Delta

• Trial met all Phase 3 endpoints and regimen is well-tolerated with all patients completing 48 weeks of treatment
• 100% of patients achieved virologic response at Week 24 with 79% of patients reaching undetectable virus (as defined by HDV RNA TND) at Week 48
• Comprehensive regulatory filing package planned for U.S. FDA submission mid-2027
• Conference call to discuss topline ECLIPSE 1 results and expanded SOLSTICE Phase 2 data scheduled for November 9, 2026, at 8:30 a.m. ET / 5:30 a.m. PT

SAN FRANCISCO–(BUSINESS WIRE)–Vir Biotechnology, Inc. (Nasdaq: VIR), today announced positive topline results from the pivotal Phase 3 ECLIPSE 1 trial evaluating monthly subcutaneous administration of elebsiran and tobevibart in adults with chronic hepatitis delta (CHD). Topline results were presented in an oral session at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting^® 2026, in Denver, Colorado.

“For people living with CHD, the goal is not simply to reduce the amount of virus, but to drive it to levels where it is no longer detectable since ongoing HDV replication drives liver disease progression,” said Nancy Reau, Associate Director of Solid Organ Transplantation and Section Chief of Hepatology at Rush University Medical Center. “The high number of participants who achieved undetectable virus and normalized ALT levels in ECLIPSE 1 shows that the combination regimen of elebsiran and tobevibart could offer a meaningful new treatment option for people living with this serious, progressive disease.”

ECLIPSE 1 Week 48 Topline Data

ECLIPSE 1 enrolled a total of 124 patients, randomized 2:1, to receive monthly subcutaneous elebsiran and tobevibart or delayed treatment, with the delayed treatment group initiating therapy at Week 12. The trial enrolled a broad and diverse CHD population across multiple countries and included patients with advanced disease.

The trial’s primary endpoint was met, with a 44% response rate in patients receiving elebsiran and tobevibart achieving undetectable virus as defined by hepatitis delta virus RNA target not detected (HDV RNA TND) plus alanine aminotransferase (ALT) normalization at Week 48, compared with 0% in the delayed treatment group at Week 12 (p<0.0001).

The trial also met its key secondary endpoint, with a 79% response rate in patients achieving undetectable virus at Week 48 versus 0% in the delayed treatment group. Undetectable virus was observed across patients regardless of cirrhosis status, with HDV RNA TND achieved in 85% of cirrhotic patients (n=46) and 73% of non-cirrhotic patients (n=37). Topline data are summarized in the below table, “Phase 3 Efficacy Endpoints.”

The regimen was well-tolerated with a favorable safety profile. There was one Grade 3 treatment-related adverse event (TRAE; neutrophil count decrease, which resolved in 15 days) and no treatment interruptions or discontinuations. Most TRAEs were mild to moderate and transient. The most common TRAE was flu-like symptoms, which predominantly occurred at first dose with the majority resolved within 24 to 48 hours.

Phase 3 Efficacy Endpoints

“Our ambition in hepatitis delta has always been to fundamentally change what patients can expect from treatment. These compelling ECLIPSE 1 results deliver on the potential for elebsiran’s and tobevibart’s complementary mechanisms to drive the virus to undetectable levels, potentially lowering the risk of progression to cirrhosis, liver cancer and liver-related mortality,” said Marianne De Backer, President and Chief Executive Officer of Vir Biotechnology. “We believe the robust viral suppression combined with the flexibility of monthly at-home or healthcare provider administration has the potential to position elebsiran and tobevibart as the best-in-disease treatment for hepatitis delta.”

The elebsiran and tobevibart regimen is also being evaluated in the ECLIPSE 2 and ECLIPSE 3 pivotal trials with data expected in the first quarter of 2027. Data from all three trials will support a comprehensive global regulatory filing package planned for U.S. FDA submission in mid-2027.

The significant unmet need in CHD and the potential for the combination of elebsiran and tobevibart to provide a differentiated treatment option has been recognized by the U.S. Food and Drug Administration (FDA) with Breakthrough Therapy and Fast Track designations, and by the European Medicines Agency (EMA) with Priority Medicines (PRIME) and orphan drug designations.

In December 2025, Vir Biotechnology entered into an exclusive licensing agreement with Norgine, under which Norgine will be responsible for the commercialization of elebsiran and tobevibart in Europe, Australia and New Zealand, subject to receipt of the necessary marketing authorizations.

Conference Call

Vir Biotechnology will host a conference call to discuss the ECLIPSE 1 topline results and expanded SOLSTICE data at 8:30 a.m. ET / 5:30 a.m. PT on Monday, November 9. A live webcast will be available at https://investors.vir.bio and will be archived for 30 days.

About the ECLIPSE Registrational Program

ECLIPSE is a registrational program to evaluate the safety and efficacy of elebsiran in combination with tobevibart in patients with chronic hepatitis delta (CHD). ECLIPSE includes three randomized, controlled trials designed to evaluate the combination therapy in comparison to delayed treatment or bulevirtide. ECLIPSE 1 (NCT06903338) is a Phase 3 trial evaluating the safety and efficacy of elebsiran in combination with tobevibart compared to delayed treatment in the U.S. or other regions where bulevirtide use is limited. ECLIPSE 2 (NCT07128550) is a Phase 3 trial evaluating the efficacy and safety of switching to elebsiran and tobevibart in people with CHD who have not achieved viral suppression with bulevirtide therapy. ECLIPSE 1 and 2 are designed to provide the registrational efficacy and safety data needed for potential submission to global regulatory agencies. ECLIPSE 3 (NCT07142811) is a pivotal head-to-head trial evaluating combination elebsiran and tobevibart compared with bulevirtide in bulevirtide-naïve patients, and it is designed to provide important supportive data to help establish access and reimbursement in key markets.

About Elebsiran and Tobevibart

Elebsiran and tobevibart are investigational agents being evaluated as a novel combination regimen administered monthly as two separate sequential subcutaneous injections for the treatment of chronic hepatitis delta (CHD). The combination is designed to disrupt the hepatitis delta virus (HDV) life cycle at multiple points by addressing both viral entry and the sustained presence of hepatitis B surface antigen (HBsAg) that enables ongoing HDV replication.

Elebsiran is an investigational hepatitis B virus-targeting small interfering ribonucleic acid (siRNA) licensed from Alnylam Pharmaceuticals, Inc. It is designed to degrade hepatitis B virus RNA transcripts and limit the production of HBsAg.

Tobevibart is an investigational broadly neutralizing monoclonal antibody (mAb) targeting HBsAg. It is designed to inhibit the entry of hepatitis B and hepatitis delta viruses into hepatocytes and to reduce the level of circulating viral and subviral particles in the blood. Tobevibart was identified using Vir Biotechnology’s proprietary mAb discovery platform. The Fc domain has been engineered to increase immune engagement and clearance of HBsAg immune complexes and incorporates Xencor’s Xtend™ technology to extend half-life.

About Chronic Hepatitis Delta (CHD)

CHD is the most severe form of chronic viral hepatitis¹ and was recently classified as carcinogenic by the International Agency for Research on Cancer.² People living with the disease rapidly progress to cirrhosis, liver failure³ and liver-related death.¹ Because ongoing hepatitis delta virus (HDV) replication drives disease progression, achieving undetectable virus, as defined by HDV RNA TND (target not detected), is considered an important virologic marker associated with improved clinical outcomes in CHD.⁴ Individuals with CHD who have detectable HDV RNA are at a higher risk of experiencing any liver-related event, including developing compensated and decompensated cirrhosis, hepatocellular carcinoma, liver transplantation and mortality, compared to patients with undetectable HDV RNA.⁴ There are currently limited approved treatments in the U.S. and globally.

About Vir Biotechnology, Inc.

Vir Biotechnology, Inc. is a clinical-stage biopharmaceutical company focused on powering the immune system to transform lives by discovering and developing medicines for serious infectious diseases and cancer. Its clinical-stage portfolio includes programs for chronic hepatitis delta and multiple PRO-XTEN^® dual-masked T-cell engagers across validated targets in solid tumor indications. Vir Biotechnology also has a preclinical portfolio of programs across a range of oncologic malignancies. Vir Biotechnology routinely posts information that may be important to investors on its website.

Forward-looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “should,” “could,” “may,” “might,” “will,” “plan,” “potential,” “aim,” “expect,” “anticipate,” “promising” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements regarding: the therapeutic potential of the elebsiran and tobevibart regimen to treat patients living with CHD and lower the risk of progression to cirrhosis, liver cancer and liver-related mortality; Vir Biotechnology’s belief that robust viral suppression combined with the flexibility of monthly at-home or healthcare provider administration has the potential to position the regimen as a best-in-disease treatment; Vir Biotechnology’s clinical development plans and expectations for the elebsiran and tobevibart regimen, including protocols for and enrollment into ongoing and planned clinical trials, target endpoints and data readouts (including from the ECLIPSE 2 and ECLIPSE 3 pivotal trials in the first quarter of 2027), as well as plans for a U.S. FDA regulatory submission in mid-2027; Vir Biotechnology’s strategy and plans; and any assumptions underlying any of the foregoing. Many factors may cause differences between current expectations and actual results, including, without limitation: unexpected safety or efficacy data or results observed during clinical studies or in data readouts, including the occurrence of adverse safety events; risks of unexpected costs, delays or other unexpected hurdles; challenges in accessing manufacturing capacity; clinical site activation rates or clinical enrollment rates that are lower than expected; the timing and outcome of Vir Biotechnology’s planned interactions with regulatory authorities, as well as general difficulties in obtaining any necessary regulatory approvals; successful development and/or commercialization of alternative product candidates by Vir Biotechnology’s competitors, as well as changes in expected or existing competition; geopolitical changes or other external factors; and unexpected litigation or other disputes. In light of these risks and uncertainties, the events or circumstances referred to in the forward-looking statements may not occur. 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 studies may not be indicative of full results or results from later stage or larger scale clinical studies and do not ensure regulatory approval. The actual results may vary from the anticipated results, and the variations may be material. You are cautioned not to place undue reliance on any scientific data presented or these forward-looking statements, which are based on Vir Biotechnology’s available information, expectations and assumptions as of the date of this press release. Other factors that may cause Vir Biotechnology’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Vir Biotechnology’s filings with the U.S. Securities and Exchange Commission, including the section titled “Risk Factors” contained therein. Except as required by law, Vir Biotechnology assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.

Vir Biotechnology has exclusive rights to the universal PRO-XTEN^® masking platform for oncology and infectious disease. PRO-XTEN^® is a trademark of Amunix Pharmaceuticals, Inc., a Sanofi company.

Contacts

Media Contact
Caren Scannell

Director, Communications

cscannell@vir.bio

Investor Contact
Kiki Patel, PharmD

Head of Investor Relations

kpatel@vir.bio

Vir Biotechnology Announces Expanded Phase 2 SOLSTICE Data for Elebsiran and Tobevibart in Chronic Hepatitis Delta, Showing Deep and Sustained Viral Suppression, and Exploratory Finite Treatment Data




Vir Biotechnology Announces Expanded Phase 2 SOLSTICE Data for Elebsiran and Tobevibart in Chronic Hepatitis Delta, Showing Deep and Sustained Viral Suppression, and Exploratory Finite Treatment Data

• Data presented at AASLD The Liver Meeting demonstrated 100% of participants (28/28) who completed Week 96 of combination therapy reached undetectable virus (as defined by HDV RNA TND)
• Separate, exploratory analysis suggests curative potential with 10/10 patients who discontinued treatment after at least 96 weeks of undetectable virus showing sustained viral response (SVR) after 12 to 48 weeks of follow-up
• Conference call to discuss topline ECLIPSE 1 results and expanded SOLSTICE data scheduled for November 9, 2026, at 8:30 a.m. ET / 5:30 a.m. PT

SAN FRANCISCO–(BUSINESS WIRE)–Vir Biotechnology, Inc. (Nasdaq: VIR), today announced new, expanded data from the Phase 2 SOLSTICE trial evaluating monthly subcutaneous administration of elebsiran and tobevibart in adults with chronic hepatitis delta (CHD). These data include new analyses presented in oral sessions at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting^® 2026, in Denver, Colorado.

“We are seeing rapid viral suppression that deepens over time, alongside improvement in liver fibrosis and function, giving us a more complete view of what sustained control of HDV could mean for patients,” said Tarik Asselah, M.D., Ph.D., Professor of Hepatology at the Hôpital Beaujon, APHP, Clichy, France, and at the University of Paris-Cité, and Head of the unit Viral Hepatitis UMR1149 at INSERM, France. “Furthermore, the profound antiviral potency with patients achieving virus undetectability provides optimism for the potential of elebsiran and tobevibart to offer finite treatment. As the clinical experience with elebsiran and tobevibart continues to grow, so does our optimism about the possibility to meaningfully change the trajectory of this serious disease.”

Additional Week 96 data from the Phase 2 SOLSTICE trial show that treatment with elebsiran and tobevibart drove rapid HDV RNA suppression, with 100% (28/28) of patients who completed Week 96 of combination therapy reaching undetectable viremia.

Deep virologic response to undetectable levels was accompanied by encouraging findings across measures of liver health. Among patients with paired liver stiffness measurements, 89% (24/27) had improved or stable liver stiffness at Week 96. Liver stiffness is a noninvasive measure associated with liver inflammation and fibrosis, and its improvement or stabilization alongside sustained HDV RNA suppression provides additional insight into the potential impact of treatment on the underlying liver disease.

With extended follow-up, the regimen continued to be generally well-tolerated. Adverse events were mild to moderate with no study drug-related discontinuations or serious adverse events.

SOLSTICE Exploratory Analysis of Finite Treatment

Beyond the expanded SOLSTICE data presented at AASLD, new and early exploratory analyses suggest curative potential of the regimen. The SOLSTICE trial design allows for exploration of the durability of profound viral suppression following treatment discontinuation. Patients who achieve and maintain undetectable virus for at least 96 weeks are eligible to discontinue elebsiran and tobevibart treatment and are followed to assess for sustained viral response (SVR). SVRx is defined at the number of weeks (x) after last dose.

Ten patients, 7 of whom are cirrhotic, chose to discontinue treatment, with follow-up assessment ranging from 12 to 48 weeks as of the data cutoff. All 10 patients achieved SVR12. Among those reaching later follow-up timepoints, 7 achieved SVR24 and 1 achieved SVR48. All 10 patients maintained stable ALT levels following treatment discontinuation with no viral rebound or ALT flares observed in any of the patients. All 10 patients are continuing follow-up to later time points.

“These preliminary findings show the potential for a finite treatment approach that goes beyond suppressing HDV to potentially achieving cure,” said David Liu, Chief Medical Officer of Vir Biotechnology. “For patients who have faced the prospect of living with this chronic disease, that possibility could fundamentally change what is achievable in hepatitis delta.”

The elebsiran and tobevibart regimen is being evaluated in the ECLIPSE registrational program, and the company announced topline Phase 3 results from the pivotal ECLIPSE 1 trial earlier today showing the trial met all of its study endpoints. Data from all three trials will support a comprehensive global regulatory filing package planned for U.S. FDA submission in mid-2027.

The significant unmet need in CHD and the potential for the combination of elebsiran and tobevibart to provide a differentiated treatment option has been recognized by the U.S. Food and Drug Administration (FDA) with Breakthrough Therapy and Fast Track designations, and by the European Medicines Agency (EMA) with Priority Medicines (PRIME) and orphan drug designations.

In December 2025, Vir Biotechnology entered into an exclusive licensing agreement with Norgine, under which Norgine will be responsible for the commercialization of elebsiran and tobevibart in Europe, Australia and New Zealand, subject to receipt of the necessary marketing authorizations.

Conference Call

Vir Biotechnology will host a conference call to discuss the ECLIPSE 1 topline results and expanded SOLSTICE data at 8:30 a.m. ET / 5:30 a.m. PT on Monday, November 9. A live webcast will be available at https://investors.vir.bio and will be archived for 30 days.

About the ECLIPSE Registrational Program

ECLIPSE is a registrational program to evaluate the safety and efficacy of elebsiran in combination with tobevibart in patients with chronic hepatitis delta (CHD). ECLIPSE includes three randomized, controlled trials designed to evaluate the combination therapy in comparison to delayed treatment or bulevirtide. ECLIPSE 1 (NCT06903338) is a Phase 3 trial evaluating the safety and efficacy of elebsiran in combination with tobevibart compared to delayed treatment in the U.S. or other regions where bulevirtide use is limited. ECLIPSE 2 (NCT07128550) is a Phase 3 trial evaluating the efficacy and safety of switching to elebsiran and tobevibart in people with CHD who have not achieved viral suppression with bulevirtide therapy. ECLIPSE 1 and 2 are designed to provide the registrational efficacy and safety data needed for potential submission to global regulatory agencies. ECLIPSE 3 (NCT07142811) is a pivotal head-to-head trial evaluating combination elebsiran and tobevibart compared with bulevirtide in bulevirtide-naïve patients, and it is designed to provide important supportive data to help establish access and reimbursement in key markets.

About Elebsiran and Tobevibart

Elebsiran and tobevibart are investigational agents being evaluated as a novel combination regimen administered monthly as two separate sequential subcutaneous injections for the treatment of chronic hepatitis delta (CHD). The combination is designed to disrupt the hepatitis delta virus (HDV) life cycle at multiple points by addressing both viral entry and the sustained presence of hepatitis B surface antigen (HBsAg) that enables ongoing HDV replication.

Elebsiran is an investigational hepatitis B virus-targeting small interfering ribonucleic acid (siRNA) licensed from Alnylam Pharmaceuticals, Inc. It is designed to degrade hepatitis B virus RNA transcripts and limit the production of HBsAg.

Tobevibart is an investigational broadly neutralizing monoclonal antibody (mAb) targeting HBsAg. It is designed to inhibit the entry of hepatitis B and hepatitis delta viruses into hepatocytes and to reduce the level of circulating viral and subviral particles in the blood. Tobevibart was identified using Vir Biotechnology’s proprietary mAb discovery platform. The Fc domain has been engineered to increase immune engagement and clearance of HBsAg immune complexes and incorporates Xencor’s Xtend™ technology to extend half-life.

About Chronic Hepatitis Delta (CHD)

CHD is the most severe form of chronic viral hepatitis¹ and was recently classified as carcinogenic by the International Agency for Research on Cancer.² People living with the disease rapidly progress to cirrhosis, liver failure³ and liver-related death.¹ Because ongoing hepatitis delta virus (HDV) replication drives disease progression, achieving undetectable virus, as defined by HDV RNA TND (target not detected), is considered an important virologic marker associated with improved clinical outcomes in CHD.⁴ Individuals with CHD who have detectable HDV RNA are at a higher risk of experiencing any liver-related event, including developing compensated and decompensated cirrhosis, hepatocellular carcinoma, liver transplantation and mortality, compared to patients with undetectable HDV RNA.⁴ There are currently limited approved treatments in the U.S. and globally.

About Vir Biotechnology, Inc.

Vir Biotechnology, Inc. is a clinical-stage biopharmaceutical company focused on powering the immune system to transform lives by discovering and developing medicines for serious infectious diseases and cancer. Its clinical-stage portfolio includes programs for chronic hepatitis delta and multiple PRO-XTEN^® dual-masked T-cell engagers across validated targets in solid tumor indications. Vir Biotechnology also has a preclinical portfolio of programs across a range of oncologic malignancies. Vir Biotechnology routinely posts information that may be important to investors on its website.

Forward-looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “should,” “could,” “may,” “might,” “will,” “plan,” “potential,” “aim,” “expect,” “anticipate,” “promising” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements regarding: the therapeutic potential of the elebsiran and tobevibart regimen to treat patients living with CHD and change the trajectory of the disease, including further exploration of the regimen as a finite treatment that can possibly achieve cure; Vir Biotechnology’s clinical development plans and expectations for the elebsiran and tobevibart regimen, including protocols for and enrollment into ongoing and planned clinical trials, target endpoints and data readouts, as well as plans for a U.S. FDA regulatory submission in mid-2027; Vir Biotechnology’s strategy and plans; and any assumptions underlying any of the foregoing. Many factors may cause differences between current expectations and actual results, including, without limitation: unexpected safety or efficacy data or results observed during clinical studies or in data readouts, including the occurrence of adverse safety events; risks of unexpected costs, delays or other unexpected hurdles; challenges in accessing manufacturing capacity; clinical site activation rates or clinical enrollment rates that are lower than expected; the timing and outcome of Vir Biotechnology’s planned interactions with regulatory authorities, as well as general difficulties in obtaining any necessary regulatory approvals; successful development and/or commercialization of alternative product candidates by Vir Biotechnology’s competitors, as well as changes in expected or existing competition; geopolitical changes or other external factors; and unexpected litigation or other disputes. In light of these risks and uncertainties, the events or circumstances referred to in the forward-looking statements may not occur. 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 studies may not be indicative of full results or results from later stage or larger scale clinical studies and do not ensure regulatory approval. The actual results may vary from the anticipated results, and the variations may be material. You are cautioned not to place undue reliance on any scientific data presented or these forward-looking statements, which are based on Vir Biotechnology’s available information, expectations and assumptions as of the date of this press release. Other factors that may cause Vir Biotechnology’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Vir Biotechnology’s filings with the U.S. Securities and Exchange Commission, including the section titled “Risk Factors” contained therein. Except as required by law, Vir Biotechnology assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.

Considerations and Limitations of the Finite Treatment Data

The SOLSTICE data described in this press release are preliminary and represent an interim snapshot of the first 10 patients who meet protocol-specified eligibility criteria and consent to treatment discontinuation. The finite treatment analysis is exploratory and is based on interim data that have not undergone a formal data cut. Accordingly, the participants and outcomes presented may not be representative of the broader SOLSTICE study population or of results that may be observed with additional participants or longer follow-up. Follow-up remains ongoing and incomplete, including with respect to potential delayed hepatic flares following treatment discontinuation, and safety data have not been fully assessed. The data remain subject to ongoing data review, cleaning and analysis and may change as the study continues. These data should not be interpreted as a complete assessment of the safety or efficacy of the investigational regimen or as establishing a sustained treatment-free response or cure.

Vir Biotechnology has exclusive rights to the universal PRO-XTEN^® masking platform for oncology and infectious disease. PRO-XTEN^® is a trademark of Amunix Pharmaceuticals, Inc., a Sanofi company.

Contacts

Media Contact
Caren Scannell

Director, Communications

cscannell@vir.bio

Investor Contact
Kiki Patel, PharmD

Head of Investor Relations

kpatel@vir.bio

Arrowhead Pharmaceuticals Presents Plozasiran Data Highlighting Sustained Triglyceride Reductions and Reduced Acute Pancreatitis Rates




Arrowhead Pharmaceuticals Presents Plozasiran Data Highlighting Sustained Triglyceride Reductions and Reduced Acute Pancreatitis Rates

ARO-DIMER-PA Data Demonstrating Simultaneous Reductions in LDL Cholesterol and Triglycerides at American Heart Association Scientific Sessions 2026

• Pooled SHASTA-3 and SHASTA-4 analysis showed plozasiran reduced acute pancreatitis event rates by 78% overall and 91% in sHTG patients with a recent history of acute pancreatitis versus placebo
• PALISADE open-label extension showed sustained reductions in triglycerides and APOC3 with plozasiran through up to 36 months of treatment in patients with FCS
• Single doses of investigational dual-functional ARO-DIMER-PA produced mean LDL-C reductions of up to 65% and median triglyceride reductions of up to 78% from baseline

PASADENA, Calif.–(BUSINESS WIRE)–Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today presented results on two of the RNA interference (RNAi) based therapies in its cardiometabolic pipeline at the American Heart Association (AHA) Scientific Sessions 2026 in Chicago. The presentations included multiple studies of plozasiran in patients with familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG), and interim results from a Phase 1/2a study of investigational ARO-DIMER-PA in patients with mixed hyperlipidemia.

Christie Ballantyne, M.D., Chief of Cardiology and Cardiovascular Research, Baylor College of Medicine, and Director, Center for Cardiometabolic Disease Prevention at the Houston Methodist DeBakey Heart and Vascular Center, Houston, TX, presented results from the pooled analysis of SHASTA-3 and SHASTA-4 evaluating acute pancreatitis risk and the effects of plozasiran in patients with severe hypertriglyceridemia (sHTG) at a late-breaking oral session.

Luke Laffin, M.D., Co-Director, Center for Blood Pressure Disorders, and Medical Director of Cardiac Rehabilitation at the Cleveland Clinic, Cleveland, OH, presented the ARO-DIMER-PA interim results across single-dose cohorts, including LDL-C and TG reductions, effects on ApoB and remnant cholesterol, and a fuller characterization of safety and tolerability at the same late-breaking oral session.

“Together, these data demonstrate the breadth of our cardiometabolic portfolio and our progress toward addressing serious consequences of lipid disorders,” said Chris Anzalone, Ph.D., President and Chief Executive Officer at Arrowhead. “Plozasiran’s reduction in acute pancreatitis event rates in SHASTA-3 and SHASTA-4, alongside sustained triglyceride lowering through up to three years of treatment in PALISADE, reinforces its potential to make a meaningful difference for patients. With ARO-DIMER-PA, we are extending the capabilities of our TRiM™ (Targeted RNAi Molecule) platform by targeting two genes with a single RNAi molecule, with the goal of addressing both LDL cholesterol and triglyceride-rich lipoproteins in people with mixed hyperlipidemia.”

“These findings highlight the importance of looking beyond triglyceride levels alone to understand a patient’s risk of acute pancreatitis,” said James Hamilton, M.D., MBA, Chief Medical Officer and Head of R&D at Arrowhead. “In the SHASTA-3 and SHASTA-4 pooled analysis, plozasiran reduced acute pancreatitis event rates by 78% overall versus placebo and by 91% among patients who had experienced pancreatitis within five years before screening. The PALISADE long-term extension found sustained triglyceride and APOC3 reductions through up to three years of treatment in patients with FCS. Meanwhile, the early ARO-DIMER-PA results show the potential to address multiple lipid abnormalities with a single molecule, supporting further evaluation of this approach for patients with mixed hyperlipidemia.”

Plozasiran Data at AHA Scientific Sessions 2026

Select SHASTA-3/4 Pooled Analysis

Results from the pooled analysis of SHASTA-3 and SHASTA-4 evaluating acute pancreatitis risk and the effects of plozasiran in patients with severe hypertriglyceridemia (sHTG) were presented. The analysis examined the individual and combined associations of baseline TG level and a history of acute pancreatitis (AP) within the previous five years with subsequent AP events over 12 months and evaluated the effects of plozasiran across these risk strata.

The studies randomized 757 adults with severe hypertriglyceridemia to plozasiran 25 mg or placebo administered every three months. The pooled analysis identified recent AP history as an important marker of subsequent AP risk across the severe hypertriglyceridemia spectrum, including among patients with baseline TG levels from 500 to 880 mg/dL.

Across the pooled population, plozasiran reduced the rate of all adjudicated AP events by 78% versus placebo. Among patients with a history of prior AP within the previous five years, the AP event rate was reduced by 91%. Among patients with prior AP and baseline TG levels from 500 to 880 mg/dL, the event rate was reduced by 82%. No AP events occurred among 35 plozasiran-treated patients with both prior AP and TG levels ≥880 mg/dL, compared with five events among 17 placebo-treated patients. These findings highlight the importance of considering both TG level and recent AP history when assessing AP risk in patients with sHTG.

In the pooled safety population, TEAEs occurred in 73% of patients in both groups, and serious TEAEs occurred in 8.3% of plozasiran-treated patients and 10% of placebo-treated patients. Worsening glycemic control was reported in 14% and 8.7%, respectively. Three deaths in the plozasiran group were attributed to pre-existing cardiovascular or hematologic disease and assessed as unrelated to study treatment.

PALISADE Open-Label Extension

The PALISADE open-label extension evaluated longer-term plozasiran treatment in patients with clinically diagnosed or genetically confirmed familial chylomicronemia syndrome (FCS). Through 24 months of the extension, representing up to 36 months of total treatment, median reductions from baseline were 76% for TGs and 89% for APOC3. At Month 24 of the extension, 57% of patients achieved TG levels < 880 mg/dL and 48% achieved levels < 500 mg/dL. AP events remained infrequent during longer-term follow-up. The results were presented by Robert Rosenson, M.D., Dr. Rony Shimony Distinguished Professor of Cardiovascular Medicine at the Icahn School of Medicine at Mount Sinai and System Director of Metabolism and Lipids for the Mount Sinai Health System, New York, NY.

During the extension, three adjudicated AP events were reported: one mild incident event in a patient who continued plozasiran and two recurrent events in a patient who switched from placebo to plozasiran. The extension had no concurrent placebo control, and its findings are descriptive.

Among 63 patients in the extension safety analysis, TEAEs occurred in 49 (78%), serious TEAEs in seven (11%), and TEAEs leading to study drug discontinuation in three (5%). Increased HbA1c was reported in five patients (8%), with elevated HbA1c associated with two discontinuations. One death due to cardiac arrhythmia was assessed by the investigator and sponsor as unrelated to plozasiran. The study reported no clinically meaningful hematologic, hepatic, or renal laboratory findings considered related to plozasiran and no cases met Hy’s law criteria.

Select ARO-DIMER-PA Single-Ascending Dose Phase 1/2a Interim Results

Today’s presentation included single ascending dose data across doses of 25 mg, 50 mg, 100 mg, 200 mg, and 400 mg. The baseline and safety tables report 21 ARO-DIMER-PA-treated participants and 10 placebo-treated participants. The primary endpoint evaluates safety and tolerability, and secondary efficacy endpoints include changes from baseline in PCSK9, APOC3, LDL-C, and TGs.

ARO-DIMER-PA Data at AHA Scientific Sessions 2026

A single dose of ARO-DIMER-PA produced simultaneous suppression of PCSK9 and APOC3, accompanied by reductions in low-density lipoprotein cholesterol (LDL-C) and triglycerides (TGs). The study team reported mean LDL-C reductions from baseline ranging from 26% to 65% and median TG reductions ranging from 56% to 78%, together with reductions in apolipoprotein B (ApoB) and remnant cholesterol. These findings provide clinical proof of concept for simultaneously targeting two complementary lipid pathways with a single RNAi therapeutic.

Across the reported dose cohorts and available follow-up, the presentation summarized the following reductions from baseline:

• PCSK9: mean reductions of 50% to 89%.
• APOC3: mean reductions of 43% to 95%.
• LDL-C: mean reductions of 26% to 65%.
• Triglycerides: median reductions of 56% to 78%.
• ApoB: mean reductions of 32% to 66%.
• Remnant cholesterol: mean reductions of 52% to 73%.

These interim results reflect different dose cohorts and available assessment times, with participant numbers varying by endpoint and visit. The reported lipid changes represent changes from baseline and are not placebo-adjusted treatment effects. Longer-term studies are needed to establish optimal dosing and further characterize safety, tolerability, and durability of treatment effects, as well as potential cardiovascular benefit.

Safety and Tolerability

In the single escalating dose cohorts, treatment-emergent adverse events (TEAEs) were reported in all 21 ARO-DIMER-PA-treated participants and nine of 10 placebo-treated participants. Most participants had a maximum TEAE severity of Grade 1 or Grade 2. No serious adverse events, deaths, or Grade 4 or Grade 5 TEAEs were reported. One participant in the 400 mg cohort experienced Grade 3 syncope six hours after administration.

Injection-site erythema was the most commonly reported adverse event, occurring in 20 of 21 ARO-DIMER-PA-treated participants and no placebo-treated participants. Other reported events included headache and pyrexia. Hyperglycemia was reported in three ARO-DIMER-PA-treated participants, although no clinically meaningful changes in HbA1c were observed. HbA1c follow-up measurements were reported at 12 weeks for placebo and the 25 mg, 50 mg, 100 mg, and 200 mg groups, and at eight weeks for one participant in the 400 mg group. One participant in the 200 mg cohort had an investigator-reported aminotransferase elevation. No clear pattern of hepatotoxicity was observed, and no cases met Hy’s law criteria. No clinically meaningful platelet changes were observed. Occasional transient elevations in high-sensitivity C-reactive protein occurred around dosing, with no apparent sustained elevation over time.

About SHASTA-3 and SHASTA-4 Phase 3 Studies

SHASTA-3 (NCT06347003) and SHASTA-4 (NCT06347016) are global double-blind, placebo-controlled, Phase 3 studies to evaluate the efficacy and safety of plozasiran in adults with severe hypertriglyceridemia. Between the two studies, approximately 750 participants were randomized to receive 4 doses (once every 3 months) of 25 mg plozasiran or placebo. The primary endpoint is percent change in fasting serum triglyceride levels from baseline to month 12 compared to placebo. After month 12, eligible participants are offered an opportunity to continue in an optional open-label extension.

About Severe Hypertriglyceridemia

Severe hypertriglyceridemia (sHTG) is characterized by triglyceride (TG) levels greater than 500 mg/dL (5.65 mmol/L), with the most severe form being familial chylomicronemia syndrome (FCS) where TGs typically exceed 880 mg/dL (9.94 mmol/L). SHTG significantly increases the risk of acute pancreatitis (AP), which can often include recurrent attacks requiring repeat hospital admissions and worsening outcomes. AP risk is proportional to the number, characteristics, and concentration of triglyceride rich lipoproteins (TRLs), particularly chylomicrons, and increases as TGs rise. Elevated TGs can also increase the risk of atherosclerotic cardiovascular disease (ASCVD). Limited treatment options exist to sustainably reduce TGs below guideline directed risk thresholds.

About the PALISADE Study

The PALISADE study (NCT05089084) is a Phase 3 placebo-controlled study to evaluate the efficacy and safety of plozasiran in adults with genetically confirmed or clinically diagnosed FCS. The primary endpoint of the study is percent change from baseline in fasting TG versus placebo at Month 10. A total of 75 patients distributed across 39 different sites in 18 countries were randomized to receive 25 mg plozasiran, 50 mg plozasiran, or matching placebo once every three months. Participants who completed the randomized period were eligible to continue in a 2-part extension period, where all participants receive plozasiran.

About Familial Chylomicronemia Syndrome

Familial chylomicronemia syndrome (FCS) is a severe and rare genetic disease often caused by various monogenic mutations. FCS leads to extremely high triglyceride (TG) levels, typically over 880 mg/dL. Such severe elevations can lead to various serious signs and symptoms including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive issues. Currently, the therapeutic options that can adequately treat FCS are limited.

About ARO-DIMER-PA and the Phase 1/2a Study

ARO-DIMER-PA is an investigational, liver-targeted, dual-functional siRNA therapeutic designed to silence expression of both PCSK9 and APOC3 in hepatocytes. Enabled by Arrowhead’s proprietary Targeted RNAi Molecule (TRiM™) platform, it is designed to address complementary pathways involved in LDL cholesterol and triglyceride-rich lipoprotein metabolism with a single molecule.

ARO-DIMER-PA-1001 (NCT07223658) is a placebo-controlled, dose-escalation Phase 1/2a study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and lipid effects of single and multiple doses of ARO-DIMER-PA in adults with mixed hyperlipidemia. The study includes single-ascending-dose and multiple-ascending-dose parts, with participants enrolled at 11 sites in Australia, New Zealand, and Georgia. The findings presented at AHA are interim single-dose results.

About Mixed Hyperlipidemia

Mixed hyperlipidemia is characterized by elevated LDL cholesterol and triglyceride-rich lipoproteins, both of which contribute to atherosclerotic cardiovascular disease risk. Treatment often requires multiple therapies to address these lipid pathways. ARO-DIMER-PA is being evaluated as a potential single-agent approach to reducing both LDL cholesterol and triglycerides. Cardiovascular outcome benefit has not been established for ARO-DIMER-PA.

About Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals (NASDAQ: ARWR) is a commercial-stage pharmaceutical company developing medicines that treat intractable diseases by silencing the genes that cause them, harnessing the natural RNA interference (RNAi) mechanism. The company has built a broad portfolio of clinical and commercial RNAi therapeutics through its industry-leading targeted RNAi molecule (TRiM™) platform, which can precisely silence genes in a wide range of cell types, including liver, lung, muscle, adipose, and central nervous system tissue. At Arrowhead, we rapidly advance potential best- and first-in-class RNAi treatments for diseases with significant unmet medical need, because every day matters to the patients we serve.

For more information, please visit www.arrowheadpharma.com, or follow us on X (formerly Twitter) at @ArrowheadPharma, LinkedIn, Facebook, and Instagram. To be added to the Company’s email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts.

Forward Looking Statements

Safe Harbor Statement under the Private Securities Litigation Reform Act:

This news release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline, products or product candidates, or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about our beliefs and expectations regarding the long-term impacts of plozasiran on patient health and the health care system; our beliefs and expectations regarding the pricing, value, or expected timing for availability of our drugs and drug candidates; our beliefs and expectations regarding the potential safety, efficacy, and therapeutic benefit of ARO-DIMER-PA and plozasiran in sHTG; the timing of any regulatory submissions for plozasiran in sHTG; and our beliefs and expectations around the potential uses and value of the TRiM™ platform. These statements are based upon our current expectations and speak only as of the date hereof. Actual results or outcomes may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties including but not limited to the safety and efficacy of our products and product candidates, pricing and reimbursement decisions related to our products, demand for our products, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.

Source: Arrowhead Pharmaceuticals, Inc.

Contacts

Arrowhead Pharmaceuticals, Inc.

Vince Anzalone, CFA

+1 626-304-3400

ir@arrowheadpharma.com

Paul Graves

+1 626-304-3400

media@arrowheadpharma.com

Investors

LifeSci Advisors, LLC

Brian Ritchie

+1 212-915-2578

britchie@lifesciadvisors.com

Media

LifeSci Communications, LLC

Kendy Guarinoni, Ph.D.

+1 724-910-9389

kguarinoni@lifescicomms.com

Aqtual Presents Data Linking Blood-Based Biomarkers to Tissue Biology and Treatment Response in Rheumatoid Arthritis




Aqtual Presents Data Linking Blood-Based Biomarkers to Tissue Biology and Treatment Response in Rheumatoid Arthritis

Second poster at ACR Convergence 2026 shows PRIMA-102 study participants are broadly representative of routine care, supporting clinical validation and clinical utility efforts

HAYWARD, Calif. & ORLANDO, Fla.–(BUSINESS WIRE)–Aqtual, Inc., a precision medicine company developing active chromatin cell-free DNA (cfDNA) diagnostics, today announced data that advance the understanding of why patients with rheumatoid arthritis (RA) respond differently to treatment and support the continued development of Aqtual’s RA treatment selection test, a blood-based biomarker test designed to help physicians choose among TNF inhibitors, JAK inhibitors, and abatacept for patients with RA before starting or switching treatment. Two posters being presented at the American College of Rheumatology (ACR) Convergence 2026, November 6–10 in Orlando, Florida, show that signals detected in blood reflect joint tissue biology associated with response to different RA medications, and that participants in Aqtual’s prospective PRIMA-102 study (NCT05936970) can be linked to real-world data to extend follow-up. Aqtual is also exhibiting at ACR at Booth #4119.

“We enrolled more than 1,400 patients as they started a new rheumatoid arthritis treatment. Even before the first dose, their blood carried biological signals associated with treatment response, with distinct patterns across drug classes,” said Diana Abdueva, PhD, co-founder and Chief Executive Officer of Aqtual. “These findings lay the foundation for choosing RA treatments based on biology rather than trial and error, and we’re now working to bring that science into clinical practice.”

Both posters draw on PRIMA-102, a prospective, multicenter study that enrolled more than 1,400 adults with RA at more than 50 sites across 22 states as they started a new treatment.

The first poster reports an interim analysis of baseline plasma samples from 416 PRIMA-102 participants who initiated treatment with a TNF inhibitor (n=156), JAK inhibitor (n=135), or abatacept (n=125). Using active chromatin cfDNA (cfDNAac) profiling across approximately 37,000 gene promoters, researchers identified largely distinct, drug-class-specific gene regulatory signatures associated with clinical response, defined as achieving a minimal clinically important difference (MCID) in the Clinical Disease Activity Index (CDAI) at 12 weeks. Response was associated with immune-rich biology for TNF inhibitors, cytokine-signaling biology for JAK inhibitors, and adaptive immune regulatory biology for abatacept, while nonresponse was associated with stromal, myeloid, and fibroblast-related biology. These signatures showed strong concordance with previously published synovial tissue pathotypes, supporting the potential of blood-based cfDNAac profiling to reflect tissue-relevant biology associated with treatment response.

The second poster, developed in collaboration with investigators from the University of Alabama at Birmingham and the non-profit Foundation for Advancing Science, Technology, Education and Research (FASTER), evaluated the representativeness of PRIMA-102 participants compared with RA patients receiving routine clinical care at the same rheumatology practices. Researchers compared PRIMA-102 participants with patients in electronic health record (EHR) and commercial insurance claims data. The populations were broadly similar in age, sex, and body mass index, although PRIMA-102 participants had higher disease activity, greater prior disease-modifying antirheumatic drug (DMARD) use, and lower oral glucocorticoid use. Researchers also assessed the feasibility of linking clinical study participants to real-world data using privacy-preserving patient matching methods. Among 1,131 PRIMA-102 participants at 20 participating sites, 580 (51.3%) were linked to at least one real-world data source. Matching was highly accurate: 99.0% of EHR matches were unique one-to-one matches, and 95.6% of claims matches were confirmed by an RA diagnosis. The findings demonstrate the feasibility of linking clinical research data with real-world patient records to extend longitudinal follow-up and augment model training and clinical validation data while informing clinical utility study plans, including potential external synthetic comparator cohorts.

“In our recent 2026 Arthritis Care & Research study of more than 37,000 newly diagnosed RA patients, TNF inhibitors, JAK inhibitors, and abatacept accounted for most first- and second-line advanced therapy choices, TNF inhibitor cycling remained common, and switching rates varied more than four-fold between rheumatologists,” said Jeffrey R. Curtis, MD, MS, MPH, Professor of Medicine at the University of Alabama at Birmingham. “That variation in part reflects the absence of a test to guide treatment switching. A blood test that accurately predicts response across RA drug classes would greatly inform the decisions that rheumatologists make most often.”

Presentation Details

Poster 1: Baseline cfDNA Active Chromatin Profiles Reflect Synovial Pathotype-Associated Biology Distinguishing Response to Targeted Treatments in Rheumatoid Arthritis (Abstract: #0080)

• Date: Sunday, November 8, 2026
• Time: 10:30 AM–12:30 PM ET
• Session: Poster Session A
• Category: Rheumatoid Arthritis: Translational Models and Mechanisms

Poster 2: Representativeness of a U.S. Biomarker Discovery Cohort Compared to Real-World Data from Enrolling U.S. Rheumatology Practices (Abstract: #2408)

• Date: Tuesday, November 10, 2026
• Time: 10:30 AM–12:30 PM ET
• Session: Poster Session C
• Category: Rheumatoid Arthritis: Treatment

Aqtual is also expanding strategic biopharmaceutical partnerships across chronic diseases and oncology. The company’s active chromatin cell-free DNA platform is giving drug developers a new, blood-based window into disease biology in cancer and autoimmune conditions beyond RA, no biopsy required. For more information on Aqtual, including a platform technology whitepaper, upcoming conferences, and details on the most recent scientific publications, please visit www.aqtual.com

About Aqtual, Inc.

Aqtual, Inc. is a precision medicine company developing products for chronic disease management and oncology utilizing a novel cell-free DNA-based platform. Aqtual’s proprietary platform evaluates protein regulation, epigenetics, and transcriptomics solely using cell-free DNA fragments found in the blood. The platform yields efficient and robust real-time analysis of disease and treatment while overcoming the limitations of previous cell-free DNA methodologies.

Contacts

Media Contact
Andrea Vuturo

Vuturo Group for Aqtual

andrea@vuturo.com
+1 (415) 689-8414