ADARx Pharmaceuticals Announces First Patient Dosed in Phase 3 STOP-HAE Clinical Trial and Orphan Drug Designation for ADX-324, a Long-Acting siRNA in Development for the Prophylactic Treatment of Hereditary Angioedema (HAE)




ADARx Pharmaceuticals Announces First Patient Dosed in Phase 3 STOP-HAE Clinical Trial and Orphan Drug Designation for ADX-324, a Long-Acting siRNA in Development for the Prophylactic Treatment of Hereditary Angioedema (HAE)

– Next-generation siRNA therapeutic candidate with the potential for twice-yearly dosing –

SAN DIEGO, Oct. 22, 2025 (GLOBE NEWSWIRE) — ADARx Pharmaceuticals, Inc. (ADARx), a late-stage clinical biotechnology company developing next-generation RNA therapeutics, today announced that the first patient has been dosed in its Phase 3 STOP-HAE clinical trial evaluating ADX-324 in patients with hereditary angioedema (HAE). Additionally, ADARx announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ADX-324 for the treatment of patients with HAE.

“Despite advances in prophylactic therapy, most HAE patients remain at risk of breakthrough attacks and do not achieve sustained, long-term attack-free control. In addition, the currently approved prophylaxis treatments require monthly or more frequent injections. We believe the FDA’s Orphan Drug Designation for ADX-324 underscores the continued need for additional treatment options and highlights the potential of ADX-324 to provide extended attack-free periods with substantially reduced dosing frequency,” said Zhen Li, President and Chief Executive Officer of ADARx. “Dosing the first patient in our ADX-324 Phase 3 clinical trial is a significant milestone for ADARx and we look forward to advancing this program for the HAE community.”

The Phase 3 STOP-HAE clinical trial of ADX-324 is a randomized, double-blind, placebo-controlled study designed to evaluate the efficacy of ADX-324 in preventing HAE attacks in adults with Type I and Type II HAE. The trial will also assess safety, pharmacokinetics (PK), pharmacodynamics (PD), and patient reported health-related quality of life (HRQoL). Approximately 90 patients will be enrolled and randomized to receive either ADX-324 300 mg every 6 months, ADX-324 240 mg every 3 months, or placebo during the study. Patients completing the trial will be eligible to enroll in a long-term-open-label extension study.

Enrollment for this Phase 3 program follows positive Phase 1/2 data demonstrating that ADX-324 achieved significant and durable suppression of prekallikrein (PKK) protein levels with a favorable safety profile.

For more information about the clinical trial, visit https://stophae.com.

About HAE and ADX-324

HAE is a rare genetic disorder characterized by recurrent, unpredictable attacks of swelling that can be painful, disabling, and life-threatening. These attacks result from dysregulation of the kallikrein-kinin system (KKS), which regulates blood pressure, inflammation, coagulation and pain. PKK is a critical protein in the plasma kallikrein pathway that activates a second protein called kallikrein, which, if present, produces bradykinin, a potent vasodilator. A dysfunctional KKS leads to excessive release of bradykinin which causes the swelling attacks in HAE.

ADX-324 is an investigational siRNA therapy designed to inhibit PKK generation at the mRNA level and reduce the production of plasma PKK, thereby averting bradykinin generation and potentially preventing HAE attacks. Compared to currently approved prophylactic treatments, ADX-324 is expected to decrease PKK to a greater degree, offering the potential for greater and more durable control of kallikrein activity, which is expected to result in a higher proportion of patients remaining attack-free with a less frequent dosing regimen.

About ADARx Pharmaceuticals 

ADARx Pharmaceuticals is a late-stage clinical biotechnology company dedicated to transforming cutting-edge science into next-generation RNA medicines across a broad range of therapeutic areas. We have developed proprietary technology to control the expression of specific disease drivers with highly selective RNA targeted therapies with the goal of delivering life-changing treatments for patients with urgent unmet medical needs. ADARx is focused on advancing and expanding a deep pipeline of highly potent, durable and selective RNA-targeted therapeutic candidates, developing product candidates for the treatment of complement-mediated, genetic, cardiovascular, thrombotic, central nervous system and metabolic (obesity) diseases. In addition to our wholly-owned programs, we have entered into a collaboration and license option agreement with AbbVie to develop siRNA therapeutics across multiple disease areas, including neuroscience, immunology and oncology. Follow ADARx on LinkedIn.

CONTACT: Contacts

Investors: ir@adarx.com 

Media: teri@redhousecomms.com 

SELLAS Life Sciences to Participate in the J.P. Morgan U.S. Opportunities Forum




SELLAS Life Sciences to Participate in the J.P. Morgan U.S. Opportunities Forum

Company to conduct 1×1 meetings with investors on Wednesday, November 12th

NEW YORK, Oct. 22, 2025 (GLOBE NEWSWIRE) — SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS’’ or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced that Dr. Angelos Stergiou, President and Chief Executive Officer of SELLAS, will participate in the J.P. Morgan U.S. Opportunities Forum, which is being held on Wednesday, November 12, at the Kimpton EPIC Hotel in Miami, Florida.

J.P. Morgan U.S. Opportunities Forum Details:

Format: 1×1 investor meetings

Date: Wednesday, November 12, 2025

Location: Kimpton EPIC Hotel, Miami, FL

Registration Link: Click Here or please contact your J.P. Morgan representative

About SELLAS Life Sciences Group, Inc.

SELLAS is a late-stage clinical biopharmaceutical company focused on the development of novel therapeutics for a broad range of cancer indications. SELLAS’ lead product candidate, GPS, is licensed from Memorial Sloan Kettering Cancer Center and targets the WT1 protein, which is present in an array of tumor types. GPS has the potential as a monotherapy and combination with other therapies to address a broad spectrum of hematologic malignancies and solid tumor indications. The Company is also developing SLS009 (tambiciclib) – potentially the first and best-in-class differentiated small molecule CDK9 inhibitor with reduced toxicity and increased potency compared to other CDK9 inhibitors. Data suggests that SLS009 demonstrated a high response rate in AML patients with unfavorable prognostic factors including ASXL1 mutation, commonly associated with poor prognosis in various myeloid diseases. For more information on SELLAS, please visit www.sellaslifesciences.com.

Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical facts are “forward-looking statements,” including those relating to future events. In some cases, forward-looking statements can be identified by terminology such as “plan,” “expect,” “anticipate,” “may,” “might,” “will,” “should,” “project,” “believe,” “estimate,” “predict,” “potential,” “intend,” or “continue” and other words or terms of similar meaning. These statements include, without limitation, statements related to the GPS clinical development program, including the REGAL study and the timing of future milestones related thereto. These forward-looking statements are based on current plans, objectives, estimates, expectations, and intentions, and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties with oncology product development and clinical success thereof, the uncertainty of regulatory approval, and other risks and uncertainties affecting SELLAS and its development programs as set forth under the caption “Risk Factors” in SELLAS’ Annual Report on Form 10-K filed on March 20, 2025 and in its other SEC filings. Other risks and uncertainties of which SELLAS is not currently aware may also affect SELLAS’ forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof. SELLAS undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations, or other circumstances that exist after the date as of which the forward-looking statements were made.

Investor Contact
John Fraunces
Managing Director
LifeSci Advisors, LLC
jfraunces@lifesciadvisors.com

Alvotech Announces Changes in Global Business Development and Commercial Operations Team




Alvotech Announces Changes in Global Business Development and Commercial Operations Team

REYKJAVIK, Iceland, Oct. 22, 2025 (GLOBE NEWSWIRE) — Alvotech (NASDAQ: ALVO), a global biotech company specializing in the development and manufacture of biosimilar medicines for patients worldwide, today announced changes to its global business development and commercial operations team. Anil Okay, Chief Commercial Officer, is stepping down to serve as Chief Executive Officer of Adalvo. Trisha Durant has joined Alvotech as Senior Vice President, Global Business Development and Commercial Operation, ex-North America. Harshika Sarbajna is Senior Vice President Commercial, North America. Agne Pasko is Vice President Head of Business Development.

“I want to thank Anil for his dedication and success in leading the expansion of Alvotech’s commercial partnerships and operations. It is good to know that Adalvo, a company I founded and has been dear to my heart, is now in very capable hands,” said Róbert Wessman, chairman and CEO of Alvotech. “I’m confident that with Trisha joining the team to manage commercial operations outside of North America, Harshika in charge of North American partnerships and Agne leading business development globally, we have a stellar leadership team for continuing commercial success of Alvotech.”

Trisha Durant has 18 years of experience in the pharmaceutical industry, with an exemplary track record in strategy development, execution, forging strategic alliances, driving revenue growth and structuring high-impact commercial teams. She joins Alvotech from Biocon, where she served most recently as Head of Europe, leading commercial expansion. Prior to this, Trisha was at McKesson for 10 years, in a range of global roles and in Ernst & Young’s Global Tax Advisory practice for 6 years. Trisha is based in the United Kingdom.

Harshika Sarbajna joined Alvotech’s commercial team in 2022, from Sandoz where she led biosimilars and specialty products in the U.S. Prior to that she was the Global Head of Marketing and Strategy for Sandoz, within the anti-infectives and biosimilars businesses, based in Munich, Germany. In a career spanning over 18 years, she has also led strategy and strategic partnerships for Dr. Reddy’s and worked as a management consultant at Parthenon for the U.S., Middle East and Asia. Harshika is based in the United States.

Agne Pasko joined Alvotech in 2019, and has held several senior roles in Commercial Operations, Supply Chain and Business Development, driving revenue growth, new partnerships and portfolio expansion. Prior to joining Alvotech she managed development and marketing for 3P Pharmaceuticals, Northway Biotech and Stem Cell RC. Agne is based in Spain.

About Alvotech
Alvotech is a biotech company, founded by Robert Wessman, focused solely on the development and manufacture of biosimilar medicines for patients worldwide. Alvotech seeks to be a global leader in the biosimilar space by delivering high quality, cost-effective products, and services, enabled by a fully integrated approach and broad in-house capabilities. Two biosimilars, to Humira® (adalimumab) and Stelara® (ustekinumab) are already approved and marketed in multiple global markets. The current development pipeline includes nine disclosed biosimilar candidates aimed at treating autoimmune disorders, eye disorders, osteoporosis, respiratory disease, and cancer. Alvotech has formed a network of strategic commercial partnerships to provide global reach and leverage local expertise in markets that include the United States, Europe, Japan, China, and other Asian countries and large parts of South America, Africa and the Middle East. Alvotech’s commercial partners include Teva Pharmaceuticals, a US affiliate of Teva Pharmaceutical Industries Ltd. (US), STADA Arzneimittel AG (EU), Fuji Pharma Co., Ltd (Japan), Advanz Pharma (EEA, UK, Switzerland, Canada, Australia and New Zealand), Dr. Reddy’s (EEA, UK and US), Biogaran (FR), Cipla/Cipla Gulf/Cipla Med Pro (Australia, New Zealand, South Africa/Africa), JAMP Pharma Corporation (Canada), Yangtze River Pharmaceutical (Group) Co., Ltd. (China), DKSH (Taiwan, Hong Kong, Cambodia, Malaysia, Singapore, Indonesia, India, Bangladesh and Pakistan), YAS Holding LLC (Middle East and North Africa), Abdi Ibrahim (Turkey), Kamada Ltd. (Israel), Mega Labs, Stein, Libbs, Tuteur and Saval (Latin America) and Lotus Pharmaceuticals Co., Ltd. (Thailand, Vietnam, Philippines, and South Korea). Each commercial partnership covers a unique set of product(s) and territories. Except as specifically set forth therein, Alvotech disclaims responsibility for the content of periodic filings, disclosures and other reports made available by its partners. For more information, please visit https://www.alvotech.com. None of the information on the Alvotech website shall be deemed part of this press release.

For more information, please visit our investor portal, and our website or follow us on social media on LinkedIn, Facebook, Instagram and YouTube.

Alvotech Forward Looking Statements
Certain statements in this communication may be considered “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements generally relate to future events or the future financial operating performance of Alvotech and may include, for example, Alvotech’s expectations regarding competitive advantages, business prospects and opportunities including pipeline product development, future plans and intentions, results, level of activities, performance, goals or achievements or other future events, regulatory submissions, review and interactions, the potential approval and commercial launch of its product candidates, the timing of regulatory approval, and market launches. In some cases, you can identify forward-looking statements by terminology such as “may”, “should”, “expect”, “intend”, “will”, “estimate”, “anticipate”, “believe”, “predict”, “potential”, “aim” or “continue”, or the negatives of these terms or variations of them or similar terminology. Such forward-looking statements are subject to risks, uncertainties, and other factors which could cause actual results to differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon estimates and assumptions that, while considered reasonable by Alvotech and its management, are inherently uncertain and are inherently subject to risks, variability, and contingencies, many of which are beyond Alvotech’s control. Factors that may cause actual results to differ materially from current expectations include, but are not limited to: (1) the ability to raise substantial additional funding, which may not be available on acceptable terms or at all; (2) the ability to maintain stock exchange listing standards; (3) changes in applicable laws or regulations; (4) the possibility that Alvotech may be adversely affected by other economic, business, and/or competitive factors; (5) Alvotech’s estimates of expenses and profitability; (6) Alvotech’s ability to develop, manufacture and commercialize the products and product candidates in its pipeline; (7) actions of regulatory authorities, which may affect the initiation, timing and progress of clinical studies or future regulatory approvals or marketing authorizations; (8) the ability of Alvotech or its partners to respond to inspection findings and resolve deficiencies to the satisfaction of the regulators; (9) the ability of Alvotech or its partners to enroll and retain patients in clinical studies; (10) the ability of Alvotech or its partners to gain approval from regulators for planned clinical studies, study plans or sites; (11) the ability of Alvotech’s partners to conduct, supervise and monitor existing and potential future clinical studies, which may impact development timelines and plans; (12) Alvotech’s ability to obtain and maintain regulatory approval or authorizations of its products, including the timing or likelihood of expansion into additional markets or geographies; (13) the success of Alvotech’s current and future collaborations, joint ventures, partnerships or licensing arrangements; (14) Alvotech’s ability, and that of its commercial partners, to execute their commercialization strategy for approved products; (15) Alvotech’s ability to manufacture sufficient commercial supply of its approved products; (16) the outcome of ongoing and future litigation regarding Alvotech’s products and product candidates; (17) the impact of worsening macroeconomic conditions, including rising inflation and interest rates and general market conditions, conflicts in Ukraine, the Middle East and other global geopolitical tension, on the Company’s business, financial position, strategy and anticipated milestones; and (18) other risks and uncertainties set forth in the sections entitled “Risk Factors” and “Cautionary Note Regarding Forward-Looking Statements” in documents that Alvotech may from time to time file or furnish with the SEC. There may be additional risks that Alvotech does not presently know or that Alvotech currently believes are immaterial that could also cause actual results to differ from those contained in the forward-looking statements. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. You should not place undue reliance on forward-looking statements, which speak only as of the date they are made. Alvotech does not undertake any duty to update these forward-looking statements or to inform the recipient of any matters of which any of them becomes aware of which may affect any matter referred to in this communication. Alvotech disclaims any and all liability for any loss or damage (whether foreseeable or not) suffered or incurred by any person or entity as a result of anything contained or omitted from this communication and such liability is expressly disclaimed. The recipient agrees that it shall not seek to sue or otherwise hold Alvotech or any of its directors, officers, employees, affiliates, agents, advisors, or representatives liable in any respect for the provision of this communication, the information contained in this communication, or the omission of any information from this communication.

ALVOTECH INVESTOR RELATIONS AND GLOBAL COMMUNICATIONS
Benedikt Stefansson, VP
alvotech.ir@alvotech.com

Alvotech Announces Changes in Global Business Development and Commercial Operations Team




Alvotech Announces Changes in Global Business Development and Commercial Operations Team

REYKJAVIK, ICELAND (OCTOBER 21, 2025) — Alvotech (NASDAQ: ALVO), a global biotech company specializing in the development and manufacture of biosimilar medicines for patients worldwide, today announced changes to its global business development and commercial operations team. Anil Okay, Chief Commercial Officer, is stepping down to serve as Chief Executive Officer of Adalvo. Trisha Durant has joined Alvotech as Senior Vice President, Global Business Development and Commercial Operation, ex-North America. Harshika Sarbajna is Senior Vice President Commercial, North America. Agne Pasko is Vice President Head of Business Development.

“I want to thank Anil for his dedication and success in leading the expansion of Alvotech’s commercial partnerships and operations. It is good to know that Adalvo, a company I founded and has been dear to my heart, is now in very capable hands,” said Róbert Wessman, chairman and CEO of Alvotech. “I’m confident that with Trisha joining the team to manage commercial operations outside of North America, Harshika in charge of North American partnerships and Agne leading business development globally, we have a stellar leadership team for continuing commercial success of Alvotech.”

Trisha Durant has 18 years of experience in the pharmaceutical industry, with an exemplary track record in strategy development, execution, forging strategic alliances, driving revenue growth and structuring high-impact commercial teams. She joins Alvotech from Biocon, where she served most recently as Head of Europe, leading commercial expansion. Prior to this, Trisha was at McKesson for 10 years, in a range of global roles and in Ernst & Young’s Global Tax Advisory practice for 6 years. Trisha is based in the United Kingdom.

Harshika Sarbajna joined Alvotech’s commercial team in 2022, from Sandoz where she led biosimilars and specialty products in the U.S. Prior to that she was the Global Head of Marketing and Strategy for Sandoz, within the anti-infectives and biosimilars businesses, based in Munich, Germany. In a career spanning over 18 years, she has also led strategy and strategic partnerships for Dr. Reddy’s and worked as a management consultant at Parthenon for the U.S., Middle East and Asia. Harshika is based in the United States.

Agne Pasko joined Alvotech in 2019, and has held several senior roles in Commercial Operations, Supply Chain and Business Development, driving revenue growth, new partnerships and portfolio expansion. Prior to joining Alvotech she managed development and marketing for 3P Pharmaceuticals, Northway Biotech and Stem Cell RC. Agne is based in Spain.

About Alvotech
Alvotech is a biotech company, founded by Robert Wessman, focused solely on the development and manufacture of biosimilar medicines for patients worldwide. Alvotech seeks to be a global leader in the biosimilar space by delivering high quality, cost-effective products, and services, enabled by a fully integrated approach and broad in-house capabilities. Two biosimilars, to Humira® (adalimumab) and Stelara® (ustekinumab) are already approved and marketed in multiple global markets. The current development pipeline includes nine disclosed biosimilar candidates aimed at treating autoimmune disorders, eye disorders, osteoporosis, respiratory disease, and cancer. Alvotech has formed a network of strategic commercial partnerships to provide global reach and leverage local expertise in markets that include the United States, Europe, Japan, China, and other Asian countries and large parts of South America, Africa and the Middle East. Alvotech’s commercial partners include Teva Pharmaceuticals, a US affiliate of Teva Pharmaceutical Industries Ltd. (US), STADA Arzneimittel AG (EU), Fuji Pharma Co., Ltd (Japan), Advanz Pharma (EEA, UK, Switzerland, Canada, Australia and New Zealand), Dr. Reddy’s (EEA, UK and US), Biogaran (FR), Cipla/Cipla Gulf/Cipla Med Pro (Australia, New Zealand, South Africa/Africa), JAMP Pharma Corporation (Canada), Yangtze River Pharmaceutical (Group) Co., Ltd. (China), DKSH (Taiwan, Hong Kong, Cambodia, Malaysia, Singapore, Indonesia, India, Bangladesh and Pakistan), YAS Holding LLC (Middle East and North Africa), Abdi Ibrahim (Turkey), Kamada Ltd. (Israel), Mega Labs, Stein, Libbs, Tuteur and Saval (Latin America) and Lotus Pharmaceuticals Co., Ltd. (Thailand, Vietnam, Philippines, and South Korea). Each commercial partnership covers a unique set of product(s) and territories. Except as specifically set forth therein, Alvotech disclaims responsibility for the content of periodic filings, disclosures and other reports made available by its partners. For more information, please visit https://www.alvotech.com. None of the information on the Alvotech website shall be deemed part of this press release.

For more information, please visit our investor portal, and our website or follow us on social media on LinkedIn, Facebook, Instagram and YouTube.

Alvotech Forward Looking Statements
Certain statements in this communication may be considered “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements generally relate to future events or the future financial operating performance of Alvotech and may include, for example, Alvotech’s expectations regarding competitive advantages, business prospects and opportunities including pipeline product development, future plans and intentions, results, level of activities, performance, goals or achievements or other future events, regulatory submissions, review and interactions, the potential approval and commercial launch of its product candidates, the timing of regulatory approval, and market launches. In some cases, you can identify forward-looking statements by terminology such as “may”, “should”, “expect”, “intend”, “will”, “estimate”, “anticipate”, “believe”, “predict”, “potential”, “aim” or “continue”, or the negatives of these terms or variations of them or similar terminology. Such forward-looking statements are subject to risks, uncertainties, and other factors which could cause actual results to differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon estimates and assumptions that, while considered reasonable by Alvotech and its management, are inherently uncertain and are inherently subject to risks, variability, and contingencies, many of which are beyond Alvotech’s control. Factors that may cause actual results to differ materially from current expectations include, but are not limited to: (1) the ability to raise substantial additional funding, which may not be available on acceptable terms or at all; (2) the ability to maintain stock exchange listing standards; (3) changes in applicable laws or regulations; (4) the possibility that Alvotech may be adversely affected by other economic, business, and/or competitive factors; (5) Alvotech’s estimates of expenses and profitability; (6) Alvotech’s ability to develop, manufacture and commercialize the products and product candidates in its pipeline; (7) actions of regulatory authorities, which may affect the initiation, timing and progress of clinical studies or future regulatory approvals or marketing authorizations; (8) the ability of Alvotech or its partners to respond to inspection findings and resolve deficiencies to the satisfaction of the regulators; (9) the ability of Alvotech or its partners to enroll and retain patients in clinical studies; (10) the ability of Alvotech or its partners to gain approval from regulators for planned clinical studies, study plans or sites; (11) the ability of Alvotech’s partners to conduct, supervise and monitor existing and potential future clinical studies, which may impact development timelines and plans; (12) Alvotech’s ability to obtain and maintain regulatory approval or authorizations of its products, including the timing or likelihood of expansion into additional markets or geographies; (13) the success of Alvotech’s current and future collaborations, joint ventures, partnerships or licensing arrangements; (14) Alvotech’s ability, and that of its commercial partners, to execute their commercialization strategy for approved products; (15) Alvotech’s ability to manufacture sufficient commercial supply of its approved products; (16) the outcome of ongoing and future litigation regarding Alvotech’s products and product candidates; (17) the impact of worsening macroeconomic conditions, including rising inflation and interest rates and general market conditions, conflicts in Ukraine, the Middle East and other global geopolitical tension, on the Company’s business, financial position, strategy and anticipated milestones; and (18) other risks and uncertainties set forth in the sections entitled “Risk Factors” and “Cautionary Note Regarding Forward-Looking Statements” in documents that Alvotech may from time to time file or furnish with the SEC. There may be additional risks that Alvotech does not presently know or that Alvotech currently believes are immaterial that could also cause actual results to differ from those contained in the forward-looking statements. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. You should not place undue reliance on forward-looking statements, which speak only as of the date they are made. Alvotech does not undertake any duty to update these forward-looking statements or to inform the recipient of any matters of which any of them becomes aware of which may affect any matter referred to in this communication. Alvotech disclaims any and all liability for any loss or damage (whether foreseeable or not) suffered or incurred by any person or entity as a result of anything contained or omitted from this communication and such liability is expressly disclaimed. The recipient agrees that it shall not seek to sue or otherwise hold Alvotech or any of its directors, officers, employees, affiliates, agents, advisors, or representatives liable in any respect for the provision of this communication, the information contained in this communication, or the omission of any information from this communication.

ALVOTECH INVESTOR RELATIONS AND GLOBAL COMMUNICATIONS
Benedikt Stefansson, VP
alvotech.ir@alvotech.com

Attachments

• Trisha Durant
• Harshika Sarbajna
• Agne Pasko

smartbax announces a €4.7 M Pre-Series A round to advance novel antibiotic compound through preclinical stage




smartbax announces a €4.7 M Pre-Series A round to advance novel antibiotic compound through preclinical stage

• Lead antibacterial compound against a novel target in Gram-negative bacteria validated in infection models
• Progressing a platform of small-molecule enzymatic activators that induce self-digestion of bacteria as novel mode of action against multi-drug resistant Gram-negative and Gram-positive bacteria
• Funding round led by Anobis Asset and Bayern Kapital; second closing remains open

Munich, Germany, Oct. 22, 2025 (GLOBE NEWSWIRE) — smartbax, a biotech company developing next-generation antibiotics against multi-drug resistant bacteria, today announced the successful first closing of its €4.7 M Pre-Series A financing round. The round was led by new investors Anobis Asset and Bayern Kapital, with participation from UnternehmerTUM Funding for Innovators as well as existing investors HTGF – High-Tech Gründerfonds and Boehringer Ingelheim Venture Fund (BIVF). A second closing of the round remains open to investors.

smartbax will use the funds to progress its proprietary pipeline of small-molecule antibiotics designed to overcome bacterial resistance with innovative approaches and novel mechanisms of action. The lead candidate is an inhibitor that blocks a previously unexplored step in the synthesis of lipopolysaccharides (LPS), key structural components of the outer membrane in Gram-negative bacteria. This new inhibitor has already demonstrated in vivo proof of concept, including activity against multi-drug resistant strains, shows potential as an orally available drug, and will now be advanced through preclinical development.

In parallel, smartbax is advancing its platform of small-molecule activators of bacterial hydrolases. Rather than inhibiting bacterial functions like traditional antibiotics, these compounds stimulate hydrolase activity, causing bacteria to digest themselves from within. This innovative mode of action has not been exploited in commercial antibiotics to date and offers a promising strategy to overcome established resistance mechanisms. smartbax has identified two activator classes, effective against different targets in Gram-positive and Gram-negative bacteria, both of which display encouraging drug-like properties, are able to eliminate biofilms and show no development of resistance. The company will further develop these candidates toward lead selection and in vivo proof of concept using the current funds.

“Small-molecule antibiotics remain one of the most effective tools in combating the rapidly growing threat of antimicrobial resistance. smartbax is currently the only German biotech dedicated exclusively to developing these crucial tools, and we are proud to advance complementary approaches with both a classical inhibitor against a novel target and enzyme activators with a truly novel mode of action in the antibiotic realm,” said Dr. Robert Macsics, CEO of smartbax. “Our programs focus on WHO priority pathogens and aim to provide new treatment options for critically ill patients who currently have limited alternatives. We are delighted to have assembled such a strong consortium of investors who share our commitment to addressing this urgent public health threat.”

Martin Falk, managing director at Anobis Asset, said: “Antibiotic resistance is one of the most urgent medical challenges of our time, and there is a clear need for new therapeutic approaches. In Germany alone, nearly 10,000 people die each year as a direct consequence of infections with multi-drug resistant bacteria; many more are hospitalized and often face lengthy recovery times. We are proud to support a team focused on developing solutions that could help patients and protect public health worldwide.”

Monika Steger, Managing Partner at Bayern Kapital, commented: “Rising bacterial resistance to antibiotics poses an enormous burden on global healthcare. smartbax is tackling this problem with two novel drug approaches that are already showing great potential at their current stage. At the same time, the market for new antibiotics is opening up a highly attractive growth area with great economic opportunities. Our investment in smartbax is therefore a promising investment in the local biotech ecosystem and the resilience of our healthcare system.”

Inga vom Holtz, Director Investments at UnternehmerTUM Funding for Innovators, added: “smartbax has grown from academic research into a biotech company with a clear focus on antibiotic innovation. We are pleased to join this financing round and to support a team that is advancing both classical inhibitors and entirely new antibacterial mechanisms with enzyme activators, and we are proud that such innovation has its origins at the Technical University Munich.”

About smartbax

smartbax is developing a new generation of antibiotics to address the increasing spread of multi-drug resistant bacteria. Their team of experts is advancing a complementary pipeline of small molecules against novel bacterial targets and with innovative modes of action to prevent resistance. Their lead program is a new inhibitor of lipopolysaccharide synthesis in Gram-negative bacteria. Moreover, the company specializes in the tailored activation of enzymatic pathways that trigger bacterial self-digestion, as this approach is particularly promising in the context of difficult-to-treat biofilms. Two activators are in development, targeting both Gram-positive bacteria and Gram-negative bacteria individually.

Knowing that preventing a resistance crisis tomorrow means taking action today, smartbax was founded in 2021 as a spin-off of the Technical University Munich (TUM) with the aim of making innovative research applicable in creating novel antibiotics that add true value for patients worldwide.

For more information visit our website: https://www.smartbax.de/

Media contact:
Dr. Regina Lutz, Katja Arnold
MC Services AG
smartbax@mc-services.eu

About Anobis Asset

As a Freiburg-based single family office with a background in the pharmaceutical industry, Anobis Asset is actively exploring investment opportunities across the healthcare and life sciences landscape, with a focus on innovation-driven ventures and long-term partnerships.

About Bayern Kapital

Bayern Kapital is the venture capital company of the Free State of Bavaria and, with 700 million euros under management, investments of up to 25 million euros per company, over 110 active portfolio companies and 30 years of experience in the VC business, is one of the most active, financially strongest and most experienced investors in the DACH region. Bayern Kapital invests in a consortium with private investors in Bavarian high-tech and deep-tech start-ups and scale-ups with innovative business models from all sectors, from the seed and scale-up phase through to exit.

Since its foundation in 1995, Bayern Kapital has invested over 500 million euros of its own equity capital in more than 320 start-ups and scale-ups from sectors such as life sciences, software & IT, materials & new materials, nanotechnology and environmental technology. This has created over 12,000 permanent jobs in sustainable companies in Bavaria.

Examples of numerous pioneering success stories that Bayern Kapital has supported from an early stage include Casavi, Catalym, commercetools, egym, EOS, Fazua, Immunic, Isar Aerospace, IQM, Parcellab, Proglove, Qbilon, Riskmethods, SimScale, Sirion, tado, Tubulis, Marvel Fusion, Proxima Fusion, plancq, Akhetonics and many more. bayernkapital.de 

About UnternehmerTUM Funding for Innovators

UnternehmerTUM Funding for Innovators is the pre-seed fund of UnternehmerTUM, Europe’s foremost center for innovation and business creation. It invests early – often as the first institutional investor – in deep tech founders building category-defining companies. The fund specializes in hard tech and the code that powers it, backing scalable B2B technologies with billion-euro potential and helping founders move breakthrough science from lab to market. UnternehmerTUM Funding for Innovators offers founder-friendly convertible loans and is committed to follow-on investments, ensuring its companies have the resources to scale their ambitions.

funding.unternehmertum.de 

About HTGF – High-Tech Gründerfonds 

HTGF is one of the leading and most active early-stage investors in Germany and Europe, financing startups in the fields of Deep Tech, Industrial Tech, Climate Tech, Digital Tech, Life Sciences and Chemistry. With its experienced investment team, HTGF supports startups in all phases of their development into international market leaders. HTGF invests in pre-seed and seed phases and can participate significantly in later-stage financing rounds. Since its inception in 2005, HTGF has financed around 800 startups and achieved 200 successful exits. HTGF has a fund volume of over 2 billion euros.

Fund investors in the public-private partnership include the Federal Ministry for Economic Affairs and Energy, KfW Capital as well as 45 companies and family offices.  

For more information, please visit HTGF.de or follow us on LinkedIn.   

About Boehringer Ingelheim Venture Fund

The Boehringer Ingelheim Venture Fund (BIVF), established in 2010, is dedicated to investing in groundbreaking biotechnology companies that are at the forefront of therapeutic and digital innovations, aiming to advance biomedical research. With a commitment to revolutionizing the standard of care, the BIVF fosters long-term partnerships with scientists and entrepreneurs. The BIVF’s focus is on nurturing disease-modifying therapeutic concepts and facilitating their clinical application. The BIVF prioritizes the translation of first-in-class concepts that address significant medical needs in fields such as oncology, immunology, regenerative medicine, neurodegeneration, infectious diseases, and digital health technologies. These innovative concepts often encompass novel platform technologies designed to tackle targets and diseases that were previously considered untreatable.

With a fund volume of EUR 350 million, the BIVF operates as an evergreen fund, continually reinvesting to fuel its mission. The partners of the BIVF gain from the fund’s deep expertise in drug discovery & development, translational science, and management, along with access to a network of experts within the Boehringer Ingelheim organization. Currently, the BIVF supports a diverse portfolio of over 40 companies, leveraging its extensive experience to drive progress in healthcare.

For additional information, please visit boehringer-ingelheim-venture.com/

Onco3R Therapeutics reports first pre-clinical data from its best-in-class precision oncology portfolio at the 2025 AACR-NCI-EORTC symposium




Onco3R Therapeutics reports first pre-clinical data from its best-in-class precision oncology portfolio at the 2025 AACR-NCI-EORTC symposium

• Three posters to be presented featuring pre-clinical data of potential best-in-class FGFR3 selective inhibitor, SMARCA2 selective inhibitor, and P53 Y220C reactivator
• Candidates for FGFR3 and SMARCA2 programs identified: IND enabling studies anticipated in mid-2026

Leuven, Belgium, October 22, 2025. Onco3R Therapeutics, a clinical-stage immunology and oncology biotech company dedicated to transforming patients’ lives with best-in-class medicines, today announced that it will present preclinical data from its FGFR3, SMARCA2 and P53 Y220C small molecules programs in 3 posters at the EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics, taking place October 22-26, 2025, in Boston.

“First generation precision medicines are often suboptimal in the clinic due to low target coverage, off-target toxicity and emergence of resistance. At Onco3R, our vision is to design best-in-class medicines to address the unmet needs left by first generation drugs and unlock the full potential of therapeutic targets” said François Gonzalvez, PhD, CSO and co-Founder of Onco3R Therapeutics.

“We are thrilled to present the first preclinical data from our lead oncology programs FGFR3, SMARCA2 and P53 Y220C. Each program has identified best-in-class molecules which offer the potential to deliver transformational efficacy and improved tolerability for patients. Our FGFR3 and SMARCA2 candidates, G-012 and G-141 respectively, have reached the optimum potency and selectivity profile to mitigate dose-limiting toxicities while maintaining maximum target coverage. This has translated into robust anti-tumor activity in vivo. The poster presentations will highlight data supporting the advancement of these two candidates towards the clinic, as well as the discovery of unique small molecule P53 reactivators.”

 “These compelling preclinical results further validate our patient-centric drug discovery approach, which integrates deep translational science with rational, structure-based and AI-augmented drug design”, Pierre Raboisson, PhD, CEO and co-Founder of Onco3R Therapeutics said. “We look forward to advancing these two candidates and remain on track to initiate IND-enabling studies in mid-2026. The identification of these candidates, alongside the continued clinical progress of our SIK3 inhibitor O3R-5671 in autoimmune indications, reinforces Onco3R’s strong strategic position. With a robust pipeline and clear execution momentum, we are confidently advancing toward our next value-driving milestones.”

Presentation details

Title: Discovery of Best-in-Class FGFR3 small molecule inhibitors with high isoform selectivity and activity against gatekeeper mutations
Session:                                         
Session: Poster Session C                          
Session Date and Time: Saturday, October 25, 12:30-4pm
Presenting author: Sandrine Vendeville, PhD

Key findings from preclinical studies include:

• G-012 demonstrated best-in-class potency and selectivity with favorable drug-like properties.
• Based on translational modelling, the compound reached the optimal selectivity against other FGFR isoforms to mitigate off-target toxicity and maintain maximal target coverage.
• G-012 showed robust anti-proliferative activity in FGFR3-driven cancer cells and induced significant tumor regression in vivo.
• G-012 is currently advancing in 14 days toxicology studies.
• IND-enabling studies are anticipated in mid-2026.

Title: Discovery of novel SMARCA2 small molecule inhibitors with best-in-class potency and selectivity for the treatment of SMARCA4-mutant cancers
Session: Poster Session C                          
Session Date and Time: Saturday, October 25, 12:30-4pm
Presenting author: Lijs Beke, PhD

Key findings from preclinical studies include:

• G-141 combined best-in-class cellular potency and selectivity to allow optimal target coverage and unlock the full therapeutic potential of SMARCA2 inhibition.
• The compound showed synthetic lethality in SMARCA4-deficient cells and induced robust anti-tumor activity in vivo without signs of SMARCA4-related toxicity.
• G-141 showed favorable drug-like properties and is currently advancing in 14 days toxicology studies.
• IND-enabling studies are anticipated in mid-2026.

Title: Discovery of a Best-in-Class small molecule p53 Y220C reactivator: Breaking through the potency ceiling
Session: Poster Session C                          
Session Date and Time: Saturday, October 25, 12:30-4pm
Presenting author: François Gonzalvez, PhD

Key findings from preclinical studies include:

• Onco3R patient-centric drug discovery approach identified unique small molecule P53 reactivators with best-in-class cellular potency.
• Onco3R leads exhibit the optimal potency and residence time to induce deep and sustain target engagement and fully unlock the tumor suppressive function of P53 in cells.  
• This translated into robust anti-proliferative activity in P53 Y220C mutant cancer cell lines (single digit nanomolar IC50s) and tumor regression in a Y220C P53 mutant xenograft model.
• Further characterization of the lead candidates is ongoing.

About Onco3R Therapeutics
At Onco3R Therapeutics, we are driven by our purpose to transform the lives of patients with autoimmune diseases and cancer through precision-designed, best-in-class therapies. With over 150 years of combined R&D experience, our team brings deep expertise in disease biology, drug discovery & development, and translational science. We focus on clinically validated targets and select the right therapeutic modality, small or large molecules, to address the underlying disease biology with best-in-class therapies. Our mission is to develop safer, more effective medicines in oncology and immunology that truly make a difference for patients. By integrating learnings from past clinical challenges and applying cutting-edge technologies, we aim to de-risk clinical development and accelerate the delivery of innovative treatments with real-world impact. The company is based in the biotech cluster in Leuven, Belgium. For more information, visit www.onco3r.com or follow us on LinkedIn.

About O3R-5671
O3R-5671 has been developed based on more than 12 years of preclinical and clinical data on SIK inhibitors for autoimmune diseases. O3R-5671 is a highly selective SIK3 inhibitor, designed to avoid the toxicities associated with inhibiting SIK1 and SIK2. Furthermore, O3R-5671 does not inhibit other kinases and has demonstrated a highly attractive profile in an extensive safety panel. Preclinical data demonstrated that O3R-5671 inhibits the release of the inflammatory cytokines TNFα and IL-23 and promotes the release of the immunomodulatory cytokine IL-10. These data, along with data from animal models of autoimmune diseases, indicate that O3R-5671 has the potential to treat a variety of autoimmune diseases including ulcerative colitis, Crohn’s Disease, psoriasis, psoriatic arthritis and rheumatoid arthritis. O3R-5671 is currently being investigated in a first-in-human study in healthy volunteers with a SAD-MAD design.

Attachment

• AACR-EORTC-NCI Meeting 2025_final

Elevara Medicines Raises $70 Million Series A to Advance Phase 2 Rheumatoid Arthritis Trial and Expand Pipeline




Elevara Medicines Raises $70 Million Series A to Advance Phase 2 Rheumatoid Arthritis Trial and Expand Pipeline

• Co-led by Forbion and Sofinnova Partners, along with founding investor Monograph Capital, and Weatherden
• Innovative lead program, ELV001, in-licensed by Elevara from Japan’s Teijin Pharma with good tolerability and safety, plus early signs of efficacy in Phase 1b
• Disrupting the treatment paradigm for patients with incomplete response to Standard of Care (Methotrexate + Tumor Necrosis Factor inhibitors (TNFi))

London, UK – 22 October 2025 – Elevara Medicines (“Elevara”), a clinical-stage biotech developing therapies for rheumatoid arthritis (RA) and chronic inflammatory diseases, today announced the close of a $70 million Series A financing. The round was co-led by Forbion and Sofinnova Partners, with participation from founding investor, Monograph Capital.

The Series A proceeds will fund Elevara’s upcoming Phase 2 clinical trial of its lead candidate ELV001 and support exploratory programs in additional chronic inflammatory conditions and women’s health. The START-SYNERGY Phase 2 trial will enroll approximately 180 patients with inadequate response to methotrexate and TNF inhibitors, with recruitment expected to begin before the end of 2025.  

Emma Tinsley, CEO of Elevara Medicines, added, “We have created Elevara together with Monograph Capital because we believe ELV001 could have a fundamental impact on RA patients who fail to achieve disease remission through immune targeted approaches. This $70 million financing provides us with the resources to rapidly advance ELV001 through Phase 2 and to expand into other chronic inflammatory indications. We are grateful for the support of our investors and are excited to take this molecule forward with Teijin Pharma.”

Rheumatoid Arthritis has historically been treated purely as an immune disease; while effective therapies have been developed, they typically hit an efficacy ceiling. Elevara is approaching RA by targeting synovial fibroblasts which contribute to local inflammation, pain, swelling, and stiffness in the joint. 

ELV001, a first-in-class oral CDK4/6 inhibitor, selectively targets fibroblast-like synoviocytes (FLS), a key driver of RA pathology, without affecting the immune system. Elevara aims to significantly disrupt early line RA treatment by seeking clinical remission in combination with standard of care. ELV001 Phase 1 data demonstrated favorable safety and tolerability, along with encouraging early efficacy signals. The data also supports the potential of ELV001 to act in combination with existing immune agents to achieve full remission.

Professor Dominique Baeten, MD, PhD, Elevara’s CMO, added: “The current standard of care isn’t achieving low disease activity and remission in a large proportion of patients with RA. Teijin has built an exceptional data package of preclinical and clinical work with the highest level of scientific rigor and discipline. By targeting the synovial fibroblast rather than immune cells, ELV001 is uniquely positioned to work with approved immunomodulators for RA in early line therapy.” 

Elevara was founded by Weatherden with operational support and funding from Monograph Capital. Elevara gained exclusive global rights to develop, commercialize and manufacture ELV001 from Teijin Pharma, part of the Teijin Group (TSE: 3401), which has dedicated over a decade of scientific work to the program to date. 

The Elevara board will be composed of Emma Tinsley (CEO of Elevara), Tim Funnell (Monograph Capital), Vanessa Carle (Forbion), Maina Bhaman (Sofinnova Partners), and Gijs van den Brink (Independent Director).

Vanessa Carle, Principal at Forbion noted, “Together with Sofinnova Partners and Monograph Capital, we look forward to working closely with the Elevara team to advance this highly differentiated asset through Phase 2 in RA and unlock its full potential for patients with chronic inflammatory diseases.”

Maina Bhaman, Partner at Sofinnova Partners, commented, “The Elevara team has moved with remarkable speed to secure ELV001 and prepare it for later stage clinical trials. We are delighted to be part of a strong investment syndicate backing this novel approach to RA treatment.”

-Ends-

Notes to Editors

Elevara Medicines

Elevara is a clinical stage company developing new therapeutics to treat rheumatoid arthritis and other chronic inflammatory conditions. The Company’s lead program, ELV001 is a potent, selective and well differentiated first-in-class oral CDK4/6 inhibitor which will be entering Phase 2 by the end of 2025. The ELV001 program is being positioned as an add-on oral therapy for RA patients with an incomplete response (IR) to a TNF inhibitor (TNFi), preventing cycling between multiple immunosuppressive therapies. The company was co-founded by Weatherden (a UK based clinical advisory group) and Monograph Capital. The Company raised $70m in a Series A in September 2025 and its investors are Sofinnova Partners, Forbion and Monograph Capital. For more information, visit www.elevara.com.

Forbion

Forbion is a leading global venture capital firm with deep expertise in Europe and offices in Naarden, the Netherlands, Munich, Germany, and Boston, USA. Forbion invests in innovative biotech companies, managing approximately €5 billion across multiple fund strategies covering all stages of (bio)pharmaceutical drug development. In addition to its human health focus, Forbion also invests in planetary health solutions through its BioEconomy strategy. The firm’s team of over 30 investment professionals has a strong track record, with more than 130 investments across 11 funds, resulting in numerous approved therapies and successful exits. Forbion is a signatory to the UN Principles for Responsible Investment and operates a joint venture with BGV for seed and early-stage investments in the Benelux and Germany regions. 

Monograph Capital

Monograph Capital is a life sciences venture firm who find and fund groundbreaking life sciences companies to transform promising science into real, life-changing treatments across the world. Founded in 2021, with offices in London and San Francisco, Monograph invests in life sciences companies that seek to create new or improved therapeutics and related technology-enabled service businesses. The Monograph team brings a wealth of expertise and a proven track record in building successful companies. The team’s collective experience spans various domains, including biotechnology, pharmaceuticals, medical devices, and digital health. For more information, please visit: www.monograph.bio.

Sofinnova Partners

Sofinnova Partners is a leading European venture capital firm in life sciences, specializing in healthcare and sustainability. Based in Paris, London, and Milan, the firm brings together a team of professionals from all over the world with strong scientific, medical, and business expertise. Sofinnova Partners is a hands-on company builder across the entire value chain of life sciences investments, from seed to later-stage. The firm actively partners with ambitious entrepreneurs as a lead or cornerstone investor to develop transformative innovations that have the potential to positively impact our collective future.

Founded in 1972, Sofinnova Partners is deeply established in Europe, with 50 years of experience backing over 500 companies and creating market leaders around the globe. Today, Sofinnova Partners manages over €4 billion in assets. For more information, please visit: sofinnovapartners.com.

Teijin Pharma

Guided by its long-term vision to become “a company that solves challenges faced by patients, families, and communities in need of greater support,” Teijin Pharma focuses on rare and intractable diseases. Leveraging its foundation in home healthcare, the company aims to deliver new value by integrating pharmaceuticals and medical devices, enabling patients to receive safe and continuous treatment in the comfort of their own homes.

About the Teijin Group

Teijin (TSE: 3401) is a technology-driven global group with two core businesses: high-performance materials and healthcare solutions. Established in 1918 as Japan’s first rayon manufacturer, Teijin today comprises some 150 companies employing 20,000 people. Teijin is committed to its Purpose, “Pioneering solutions together for a healthy planet.” Teijin works together with employees and external partners to achieve its Long-Term Vision, “To be a company that supports the society of the future.” Teijin posted consolidated revenue of JPY 1,005.5 billion and total assets of JPY 1,061.3 billion in the fiscal year ending March 31, 2025. Visit www.teijin.com.

Weatherden

Weatherden is a global clinical development consultancy helping biotech and pharma realize the full potential of their science and investment. Weatherden combine scientific, regulatory, and commercial expertise to guide smarter, faster decisions across the clinical development journey—from translational research to registrational trials.

The Weatherden team are seasoned experts who have built companies, led global programs, secured investment, and navigated regulators. ​Weatherden thrives as an integrated, collaborative team. We do not believe in one-size-fits-all solutions. Instead, we tailor our work to each task – whether applying foresight to anticipate risks, integrating disciplines to ensure continuity, or aligning development with the commercial realities that drive investment and partnerships. Our strength lies in adapting these capabilities to solve the hardest problems at the right time, with integrity, precision and value.

Weatherden partners with clients to solve the hardest problems at the right time—balancing strategic foresight with operational precision. For more information, please visit: www.weatherden.co.uk.

CONTACT: 
For more information, please contact:

Elevara Medicines
Emma Tinsley, CEO
Professor Dominque Baeten, CMO
info@elevara.com

Vigo Consulting
Melanie Toyne-Sewell / Rozi Morris
Elevara@vigoconsulting.com
melanie.toyne-sewell@vigoconsulting.com
+44 7890 022 814

Press Release: Sanofi’s efdoralprin alfa met all primary and key secondary endpoints in alpha-1 antitrypsin deficiency emphysema phase 2 study




Press Release: Sanofi’s efdoralprin alfa met all primary and key secondary endpoints in alpha-1 antitrypsin deficiency emphysema phase 2 study

Sanofi’s efdoralprin alfa met all primary and key secondary endpoints in alpha-1 antitrypsin deficiency emphysema phase 2 study

• Efdoralprin alfa demonstrated superiority in a head-to-head study versus a standard of care plasma-derived therapy
• Results reinforce the potential of efdoralprin alfa to be the first restorative recombinant therapy that normalizes and maintains functional AAT levels
• Phase 2 data support both three-week and four-week dosing regimens for efdoralprin alfa – a potentially significant improvement in convenience – compared to a plasma-derived therapy dosed weekly

Paris, October 22, 2025. Positive results from the global ElevAATe phase 2 study (clinical study identifier: NCT05856331) showed that efdoralprin alfa (SAR447537, formerly known as INBRX-101), met all primary and key secondary endpoints when dosed every three weeks (Q3W) or four weeks (Q4W) in adults with alpha-1 antitrypsin deficiency (AATD) emphysema, a rare disease. Efdoralprin alfa is an investigational recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein. It demonstrated a statistically significant greater mean increase in functional AAT levels within normal range as measured by trough concentrations at steady state compared to those receiving weekly plasma-derived augmentation therapy at week 32 [p<0.0001]. The study also met key secondary endpoints, demonstrating superior mean increase in fAAT average concentration as well as higher percentage of days above the lower limit of the normal range for both Q3W and Q4W dosing.

The recombinant efdoralprin alfa was well tolerated with a similar adverse event profile to plasma-derived therapy. Additional safety follow-up will be assessed in the ElevAATe OLE phase 2 study (clinical study identifier: NCT05897424).

“These data demonstrate that efdoralprin alfa achieved consistently higher normal functional AAT levels, with less frequent dosing, compared to a current standard of care,” said Christopher Corsico, Global Head of Development at Sanofi. “This is particularly meaningful as maintaining protective protein levels is the cornerstone of pulmonary management of AATD and currently available treatments require weekly therapeutic infusions. The ElevAATe results represent the potential for efdoralprin alfa to be a restorative recombinant therapeutic option for the AATD community, reinforcing our commitment to develop treatments for both rare and respiratory conditions with great unmet medical need.”

“AATD is a debilitating condition that can be challenging to treat,” said Igor Barjaktarevic, MD, PhD, Associate Professor, David Geffen School of Medicine at UCLA and primary investigator on the ElevAATe phase 2 study. “Achieving and maintaining normal AAT levels with less frequent dosing and with complete independence from blood donation programs would be a welcome change to the current treatment experience for people living with AATD. With the current standard of care, patients reach but do not maintain normal protein levels between the infusions, leaving a remaining unmet need. I’m encouraged by the ElevAATe trial results and what efdoralprin alfa could mean for the AATD community.”

Efdoralprin alfa was previously granted fast track and orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of AATD emphysema. Efdoralprin alfa is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority. Sanofi plans to present the data at a forthcoming medical meeting and engage with global regulatory authorities on the appropriate next steps.

About AATD
AATD is a rare, inherited disorder characterized by low levels or absence of AAT, a protein produced by the liver that protects the lungs from inflammation and damage.^1,3 The disease causes progressive deterioration of the tissue of the lungs and liver.^1,4 Without adequate AAT levels, affected individuals often experience lung damage and develop COPD, including emphysema, and in severe forms of the disease, patients can sometimes require lung transplantation. Plasma-derived therapies were introduced in 1987 to treat the condition but since then, no new therapies have been introduced. About 235,000 people worldwide live with AATD, with nearly 100,000 people in the US, but about 90% of individuals with AATD are likely undiagnosed.

About efdoralprin alfa
Efdoralprin alfa (SAR447537, formerly known as INBRX-101) is a recombinant human AAT-Fc fusion protein being investigated as a restorative therapy in adults with AATD emphysema, with Q3W or Q4W dosing. The investigational treatment is being studied to restore functional AAT levels to the normal range and inhibit neutrophil elastase, an enzyme that can cause lung tissue damage in patients with AATD. Efdoralprin alfa was granted fast track designation and orphan drug designation by the FDA for the treatment of AATD emphysema.

About ElevAATe
The ElevAATe phase 2 study was a double-blind, randomized study evaluating efdoralprin alfa versus a standard of care plasma-derived augmentation therapy in patients with AATD emphysema. ​Ninety-seven patients were randomized 2:2:1 to receive efdoralprin alfa every three weeks or every four weeks, or plasma-derived augmentation therapy once weekly. The primary endpoint was the mean change in average fAAT concentrations as measured from baseline to average serum trough fAAT concentrations at steady state in patients treated with efdoralprin alfa Q3W or Q4W compared with weekly plasma-derived augmentation therapy, following a treatment period of up to 32 weeks. Key secondary endpoints included mean change in serum fAAT concentration from baseline to fAAT average concentrations at steady state and percentage of days that steady-state functional AAT levels were above the lower limit of the normal range.

About Sanofi
Sanofi is an R&D driven, AI-powered biopharma company committed to improving people’s lives and delivering compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people’s lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

Media Relations
Sandrine Guendoul | +33 6 25 09 14 25 | sandrine.guendoul@sanofi.com
Evan Berland | +1 215 432 0234 | evan.berland@sanofi.com
Léo Le Bourhis | +33 6 75 06 43 81 | leo.lebourhis@sanofi.com
Victor Rouault | +33 6 70 93 71 40 | victor.rouault@sanofi.com
Timothy Gilbert | +1 516 521 2929 | timothy.gilbert@sanofi.com
Léa Ubaldi | +33 6 30 19 66 46 | lea.ubaldi@sanofi.com

Investor Relations
Thomas Kudsk Larsen | + 44 7545 513 693 | thomas.larsen@sanofi.com
Alizé Kaisserian | + 33 6 47 04 12 11 | alize.kaisserian@sanofi.com
Felix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.com
Keita Browne | + 1 781 249 1766 | keita.browne@sanofi.com
Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com
Tarik Elgoutni | + 1 617 710 3587 | tarik.elgoutni@sanofi.com
Thibaud Châtelet | + 33 6 80 80 89 90 | thibaud.chatelet@sanofi.com
Yun Li | +33 6 84 00 90 72 | yun.li3@sanofi.com

Sanofi forward-looking statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2024. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

All trademarks mentioned in this press release are the property of the Sanofi group.

Attachment

• Press Release

Ipsen to acquire ImCheck Therapeutics, expanding its leadership in oncology, strengthening its pipeline




Ipsen to acquire ImCheck Therapeutics, expanding its leadership in oncology, strengthening its pipeline

• Acquisition focused on lead clinical-stage program ICT01 in acute myeloid leukemia, where data from the ongoing Phase I/II EVICTION trial showed high treatment response
• ICT01 has the potential to be a new standard of care in combination in first line unfit acute myeloid leukemia, an aggressive blood cancer affecting older adults
• Ipsen to acquire all issued and outstanding shares of ImCheck Therapeutics, for which ImCheck Therapeutics’ shareholders will be eligible to receive a closing purchase price of 350 million euros and downstream payments contingent upon achievement of regulatory and sales-based milestones

PARIS AND MARSEILLE, FRANCE, 22 October 2025 – Ipsen (Euronext: IPN; ADR: IPSEY) and ImCheck Therapeutics today announced they have entered into a definitive share purchase agreement in which Ipsen will acquire all issued and outstanding shares of ImCheck Therapeutics, a private French biotechnology company pioneering next-generation immuno-oncology therapies. The anticipated acquisition is focused on the lead Phase I/II program ICT01 in first line acute myeloid leukemia (AML)³ patients who are ineligible for intensive chemotherapy. ICT01 is a first-in-class monoclonal antibody targeting BTN3A, a key immune-regulatory molecule broadly expressed across cancer, and received Orphan Drug Designations from the U.S. Food and Drug Administration and European Medicines Agency in July 2025.

Many AML patients are unable to tolerate intensive chemotherapy and must rely on lower-intensity options, which often deliver limited and short-lived benefit.² This high-risk, unfit population continues to face a significant unmet medical need, highlighting the urgency for new therapies that can improve survival and quality of life. 

“At completion, the acquisition of ImCheck Therapeutics presents an opportunity for us to expand our pipeline in oncology and reinforces our commitment to deliver transformative therapies to the people who need them most,” said David Loew, CEO, Ipsen. “We feel confident that with the ICT01 promising data combined with Ipsen’s global development and commercialization expertise, we are well positioned to start a Phase IIb/III trial in 2026.” 

Interim data (n=45) orally presented at the annual American Society of Clinical Oncology 2025¹ from the Phase I/II EVICTION trial showed treatment with ICT01 in combination with venetoclax and azacitidine (Ven-Aza) achieved very encouraging high responses. In this single-arm trial, treatment response nearly doubled relative to those seen in historical standard of care data across all molecular subtypes in newly diagnosed patients including sub-types typically less responsive to standard of care (Ven-Aza).² ICT01 in combination with Ven-Aza was also shown to be well tolerated, underscoring ICT01’s potential as a novel immunotherapy to improve outcomes for patients with AML. 

“We are thrilled to become part of Ipsen, a company whose ambition for transformative care matches our commitment to bringing innovative treatments to patients. This transaction recognizes groundbreaking science originating from French academia,” said Pierre d’Epenoux, CEO, ImCheck Therapeutics. “It also highlights the exceptional work the ImCheck team and our partners have achieved to advance the understanding of butyrophilns and gamma delta T cells. Joining Ipsen will help us accelerate ICT01 toward registrational studies and commercialization. I remain grateful to the patients and investors for their contributions to furthering ImCheck’s pioneering science.”

Transaction details
Under the terms of the agreement, through a wholly owned affiliate of Ipsen SAS, shareholders of ImCheck Therapeutics will receive a 350 million euros payment on a cash-free and debt-free basis at closing of the transaction, and deferred payments contingent upon the achievement of specified regulatory approvals and sales-based milestones, for a total potential consideration up to 1 billion euros.

The transaction is expected to close by the end of Q1 2026, subject to fulfilment of customary closing conditions including the expiration or termination of any required regulatory and governmental approvals under French and U.S. regulations.

Advisors
Allen & Overy Shearman (Paris) is acting as legal counsel to Ipsen. Centerview Partners is acting as exclusive financial advisor to ImCheck Therapeutics with Goodwin (London) and Dentons (Paris) acting as legal counsel.

About the EVICTION trial
EVICTION is a first-in-human, dose-escalation (Part 1) and cohort-expansion (Part 2) clinical trial of ICT01 in patients with various advanced relapsed or refractory solid or hematologic cancers that have exhausted standard-of-care treatment options, as well as newly-diagnosed AML. More information on the EVICTION trial can be found at clinicaltrials.gov (NCT04243499).

About ICT01
ICT01 is a humanized, anti-BTN3A (also known as CD277) monoclonal antibody that promotes the recognition and elimination of tumor cells by γ9δ2 T cells, which are responsible for immunosurveillance of malignancy and infections. The three isoforms of BTN3A targeted by ICT01 are overexpressed on many solid tumors (e.g., melanoma, urothelial cell, colorectal, ovarian, pancreatic, and lung cancer) and hematologic malignancies (e.g., leukemia and lymphomas) and are also expressed on the surface of innate (e.g., γδ T cells and NK cells) and adaptive immune cells (T cells and B cells). Binding to BTN3A is essential for the activation of the anti-tumor immune response of γ9δ2 T cells. By altering the conformation of BTN3A, ICT01 promotes this binding, thereby selectively activating circulating γ9δ2 T cells. This leads to migration of γ9δ2 T cells out of the circulation and into the tumor tissue, and triggers a downstream immunological cascade through secretion of pro-inflammatory cytokines, including but not limited to IFNγ and TNFα, further augmenting the anti-tumor immune response. Anti-tumor activity and efficacy of ICT01 have been shown in patients across several cancer indications.

About Ipsen

We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. 

Our pipeline is fueled by internal and external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries.

Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com.

About ImCheck Therapeutics
ImCheck Therapeutics is developing a new generation of immunotherapeutic antibodies targeting butyrophilins, a novel superfamily of immunomodulators. By unlocking the power of γ9δ2 T cells, ImCheck’s innovative approach has the potential to transform treatments across oncology, autoimmune, and infectious diseases. 

The lead clinical-stage program, ICT01, has been advancing to late-stage trials, demonstrating a unique mechanism of action that modulates both innate and adaptive immunity. These “first-in-class” activating antibodies may deliver superior clinical outcomes compared to first-generation immunotherapy approaches, in particular in rationale combinations with immune checkpoint inhibitors and immunomodulatory anti-cancer drugs. Additionally, ImCheck’s pipeline compounds are progressing toward clinical development for autoimmune and infectious diseases. 

The company benefits from the pioneering research of Prof. Daniel Olive (Institut Paoli Calmettes, INSERM, CNRS, Aix-Marseille University), a global leader in γ9δ2 T cells and butyrophilins, as well as the expertise of a seasoned management team and the commitment of leading French, European and U.S. investors including Kurma Partners, Eurazeo, Bpifrance through its Innobio 2 and Large Venture funds, Andera Partners, Pfizer Ventures, Gimv, EQT Life Sciences, Earlybird, Wellington Partners, Pureos Bioventures, Invus, Agent Capital, Boehringer Ingelheim Venture Fund, Alexandria Venture Investments, and Blood Cancer United (previously LLS)®.

For further information: https://www.imchecktherapeutics.com/     

Ipsen Contacts

Investors
Henry Wheeler         henry.wheeler@ipsen.com       +33 7766471149
Khalid Deojee          khalid.deojee@ipsen.com        +33 666019526

Media
Sally Bain                sally.bain@ipsen.com                 +1 8573200517
Anne Liontas            anne.liontas.ext@ipsen.com        +33 0767347296

ImCheck Contacts

Media
Gretchen Schweitzer (EU and US)        ImCheck@trophic.eu                  +49 1728618540
Céline Voisin (France)                        Imcheck@atcg-partners.com        +33 (0)6 62 12 53 39

References

1. Dumas et al. γ9δ2 T-cell activation with ICT01 combined with azacitidine-venetoclax for older/unfit adults with newly diagnosed AML: preliminary efficacy and dose selection in Phase 1/2 study EVICTION. ASCO 2025. Available here: https://www.ImChecktherapeutics.com/fileadmin/Posters_Prez/ASCO2025-ICT01-AzaVen.pdf
2. Kone AS, Ait Ssi S, Sahraoui S, Badou A. BTN3A: A Promising Immune Checkpoint for Cancer Prognosis and Treatment. Int J Mol Sci. 2022;23(21):13424. Published 2022 Nov 3. doi:10.3390/ijms232113424
3. Short NJ, Rytting ME, Cortes JE. Acute myeloid leukaemia. Lancet. 2018;392(10147):593-606. doi:10.1016/S0140-6736(18)31041-9

Disclaimers and/or forward-looking statements

The forward-looking statements, objectives and targets contained herein are based on Ipsen’s management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect Ipsen’s future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words ‘believes’, ‘anticipates’ and ‘expects’ and similar expressions are intended to identify forward-looking statements, including Ipsen’s expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external-growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by Ipsen. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising medicine in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. Ipsen must face or might face competition from generic medicine that might translate into a loss of market share. Furthermore, the research and development process involves several stages each of which involves the substantial risk that Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation and risks arising from unexpected regulatory or political changes such as changes in tax regulation and regulations on trade and tariffs, such as protectionist measures, especially in the United States; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen’s patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen’s activities and financial results. Ipsen cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen’s partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen’s business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen’s business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen’s latest Universal Registration Document, available on ipsen.com.

Attachment

• Ipsen PR_Ipsen to acquire ImCheck Therapeutics_22102025