On June 26, 2025 The NextGen EV Therapeutics Forum, hosted by China Medical University and Healthcare System, reported bringing together leading scientists, clinical experts, and biotech industry leaders from Taiwan and the United States to explore the forefront of extracellular vesicle (EV) research and translation (Press release, China Medical University, JUN 26, 2025, View Source [SID1234654146]). The forum showcased emerging EV-based applications in oncology, neurodegenerative disorders, immune modulation, and regenerative medicine, highlighting the rapid advances toward clinical implementation.

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A highlight of the event was the announcement of a strategic partnership between China Medical University Hospital (CMUH) and Shine-On Biomedical Co., Ltd. This collaboration aims to accelerate Taiwan’s leadership in EV-based precision medicine on the global stage. As part of this initiative, Dr. Randy W. Schekman, the 2013 Nobel Laureate in Physiology or Medicine, was appointed International Scientific Advisor. In his keynote address, Dr. Schekman emphasized the critical importance of grounding therapeutic innovation in rigorous basic science, particularly in the evolving roles of EVs in intercellular communication and nucleic acid delivery.

Global Breakthrough: SOB100 Platform Targets HLA-G Tumors

At the center of this three-party collaboration is SOB100, the world’s first engineered exosome platform targeting HLA-G, an immunosuppressive tumor antigen. The platform is capable of crossing the blood-brain barrier and delivering nucleic acid drugs with high specificity, offering a potential therapeutic breakthrough for glioblastoma (GBM) and triple-negative breast cancer (TNBC).

Dr. Hui-Chun Ho, Vice President of Shine-On Biomedical, noted that SOB100 has received U.S. FDA approval to enter Phase I clinical trials and was recently featured in Nature Communications. She emphasized that the platform has demonstrated exceptional targeting precision and delivery efficiency in preclinical studies. With Dr. Schekman’s advises, the company aims to further enhance nucleic acid payload efficiency to unlock even greater therapeutic impact.

U.S.-Taiwan Scientific Exchange and Industry Insight

The forum also featured a keynote speech by Dr. Kenneth Witwer, Professor at Johns Hopkins School of Medicine and President of the International Society for Extracellular Vesicles (ISEV). Dr. Witwer provided a global perspective on the progress and challenges in EV therapeutics and called for greater standardization in EV engineering to support clinical scalability.

CMU System showcased several of its internally developed technologies, including:

In Vivo CAR-T Production via EVs for Solid Tumors
Targeted EV Drug Delivery for Parkinson’s Disease
Mitochondrial EVs from CRISPR-Engineered Stem Cells for MASLD
Engineered EVs for Cardiac Repair in Heart Failure
These innovations demonstrate CMU’s leadership in building an end-to-end EV research and clinical pipeline and underscore its commitment to integrating academia, healthcare, and biotech industry in Taiwan.

Building a Global Innovation Ecosystem for Precision EV Therapeutics

Dr. Mien-Chie Hung, President of China Medical University, remarked, "Extracellular vesicles are one of the most promising delivery vectors for the next generation of precision medicine. This forum not only showcases Taiwan’s research capabilities but also highlights our ability to mobilize global expertise."

Dr. Der-Yang Cho, Superintendent of CMUH, added, "EVs are emerging as a core technology for next-generation therapies. We are committed to building a globally connected precision medicine ecosystem with EVs at its center—driven by translational science and patient benefit."

The forum also gathered leading voices from Taiwan’s top research institutions, including the National Health Research Institutes, Academia Sinica, and National Taiwan University. Prominent researchers such as Prof. Hua-Jung Lee, Prof. Chia-Ning Shen, Prof. Tang-Long Shen, and Prof. Han-Yi Chou shared the latest advancements, strengthening Taiwan’s role in advancing EV-based precision medicine.

On June 26, 2025 Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) reported receipt of a Special Protocol Assessment Agreement Letter from the U.S. Food and Drug Administration (FDA) for ADX-2191 (methotrexate injection, USP), an investigational drug candidate, for the treatment of primary vitreoretinal lymphoma (PVRL), a rare and potentially fatal cancer currently lacking FDA-approved therapy (Press release, Aldeyra Therapeutics, JUN 26, 2025, View Source [SID1234654131]).

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"Primary vitreoretinal lymphoma is treated today off label with intravitreal injections of compounded methotrexate," stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra. "ADX-2191, a novel, vitreous-compatible formulation of methotrexate that is specifically designed for intraocular injection, potentially allows for a reduced injection volume relative to compounding."

Based on a pre-New Drug Application (NDA) meeting with the FDA in December 2022, Aldeyra submitted a literature-based NDA of ADX-2191 for the treatment of PVRL, which was accepted for Priority Review in March 2023. In June 2023, Aldeyra received a Complete Response Letter that stated that the available literature was not sufficient to demonstrate efficacy and that adequate and well-controlled trials were required for approval. The FDA subsequently agreed that a single clinical trial in addition to literature references will be sufficient to support NDA resubmission.

The clinical trial proposed in the Special Protocol Assessment will compare cancer cell clearance after 30 days of therapy in up to 20 patients following 1:1 randomization to receive either a single intraocular injection or eight intraocular injections of ADX-2191. The frequency of methotrexate injections has been linked to cancer cell clearance in patients with PVRL,[1] and approximately five injections are required on average to achieve cancer cell clearance.[2] The clinical trial is expected to begin in the second half of 2025 and conclude in 2026.

About ADX-2191

ADX-2191 (methotrexate injection, USP) is a sterile, non-compounded intravitreal formulation of methotrexate for the potential treatment of specific rare retinal diseases, including primary vitreoretinal lymphoma and retinitis pigmentosa. The ADX-2191 intravitreal formulation is preservative-free, is designed to be vitreous-compatible, and is optimized for excipient composition, viscosity, density, tonicity, pH, concentration, and volume of administration. ADX-2191 has received FDA Orphan Drug Designation for the treatment of primary vitreoretinal lymphoma and retinitis pigmentosa.

About Primary Vitreoretinal Lymphoma

Primary vitreoretinal lymphoma is a rare, aggressive, and potentially fatal retinal cancer that is diagnosed in approximately 300 to 600 patients in the United States per year. The median survival for newly diagnosed patients is less than five years. No approved treatments are currently available, though intravitreal injection of compounded methotrexate represents the current standard of care.

On June 26, 2025 A2 Biotherapeutics, Inc. (A2 Bio), a clinical-stage cell therapy company developing first-in-class logic-gated cell therapies for solid tumors, reported dosing of the first patient in the DENALI-1 (NCT06682793) clinical study. DENALI-1 is a multicenter phase 1/2 dose-escalation clinical study to evaluate the safety and efficacy of A2B395, an allogeneic logic-gated CAR T cell therapy, in participants with solid tumors that express EGFR and have lost HLA-A*02 expression (Press release, A2 Biotherapeutics, JUN 26, 2025, View Source [SID1234654147]). A2B395 is the first allogeneic therapy utilizing the A2 Bio Tmod technology platform to advance into clinical development.

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A2 Bio is advancing A2B395 as a potential new precision cell therapy in participants with solid tumors that express EGFR, including colorectal cancer, non-small cell lung cancer, head and neck squamous cell carcinoma, triple-negative breast cancer, and renal cell carcinoma.

"Dosing of the first patient in DENALI-1 is a key milestone toward providing a precise, novel ‘off the shelf’ CAR T cell therapy for patients with solid tumors that express EGFR. No curative therapies are available for people living with these cancers, and current treatments can be toxic, debilitating, and fatal. All of us at A2 Bio are grateful to the patients, investigators, and clinical care providers participating in DENALI-1," said John Welch, M.D., Ph.D, interim chief medical officer of A2 Bio.

Significant unmet needs remain for people living with EGFR-positive cancers, despite recent advances that have produced new targeted therapies, antibody-based therapies, and immunotherapies.1,2 In the recurrent unresectable, locally advanced, or metastatic setting, the intent of standard of care treatment is typically palliative rather than curative, and has not changed significantly in several decades.

Precision medicine enables efficient identification of patients in the A2 Bio clinical studies. Patients are enrolled in DENALI-1 through BASECAMP-1 (NCT04981119), a master prescreening study that identifies patients with HLA loss of heterozygosity (LOH) at any time in the course of their disease via next-generation sequencing. Upon disease progression, the participant may screen for enrollment in DENALI-1. There is no time requirement between the studies, and patients may go directly from BASECAMP-1 to DENALI-1 based on their own disease course. BASECAMP-1 utilizes artificial intelligence (AI)-enabled precision diagnostics as a cost-effective, high-yield approach to identify eligible patients for all A2 Bio clinical studies.3, 4

In addition to A2B395, A2 Bio continues to advance its clinical development of A2B694, the BASECAMP-1 prescreening study, as well as other preclinical programs as the company pursues additional pipeline expansion opportunities using its proprietary Tmod technology platform. The Tmod platform comprises a suite of technologies that can be used in isolation or in combination, and in both autologous and allogeneic settings, to create novel therapies for various cancers and beyond.

The Tmod platform utilizes a dual-receptor design consisting of an activator that targets tumor cells and a blocker that protects normal cells. This dual-receptor design is intended to provide selective killing of tumor tissues that express EGFR and have lost the HLA-A*02 gene permanently. This novel design is aimed at tackling the fundamental challenge in solid tumor cancer medicines – the ability to selectively kill tumor cells and protect normal cells.

About DENALI-1

DENALI-1 (NCT06682793) is a phase 1/2, open-label, nonrandomized study evaluating the safety and efficacy of A2B395 in adults. Patients are enrolled through BASECAMP-1 (NCT04981119), a master prescreening study that identifies patients with HLA LOH at any time in the course of their disease via next-generation sequencing. Upon disease progression the participant may screen for enrollment in DENALI-1.

More information about DENALI-1 and clinical trial sites participating in the study are available on the A2 Bio clinical trials website.

About the Tmod Platform

A2 Bio has pioneered a precision-targeting cellular system – the Tmod platform – that incorporates two receptors, an activator and a blocker, to aim the powerful armaments of immune cells directly at tumors to unequivocally differentiate tumors from normal tissues. The activator recognizes antigens on tumor cells that trigger their destruction, while the blocker recognizes antigens on normal cells that protect them. This novel blocker technology enables precise, personalized and effective T cell targeting. The blocker component equips Tmod cells with the capacity to identify tumors as distinct from normal cells.

On June 26, 2025 Incyte (Nasdaq:INCY) reported that the Company’s Board of Directors has unanimously appointed Bill Meury as President and Chief Executive Officer (CEO) and a member of the Company’s Board of Directors, effective immediately (Press release, Incyte, JUN 26, 2025, View Source [SID1234654148]).

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Bill Meury succeeds Hervé Hoppenot who will retire from the Company after 11 years of service. Mr. Hoppenot will serve as an advisor to the CEO and will remain as a member of the Board through the end of the year to ensure a smooth transition. In addition, Lead Independent Director Julian Baker has been elected Chairman of the Board of Directors.

Mr. Meury is a proven enterprise leader with expertise in organizational strategy, pipeline and commercial execution and capital allocation. He most recently served as CEO of Anthos Therapeutics, where he successfully scaled the company for its next stage of growth. Prior to that, he was CEO of Karuna Therapeutics, leading its transition into a fully integrated R&D and commercial organization. Before Karuna, Mr. Meury spent more than two decades at Allergan, serving as Chief Commercial Officer, managing a global business with fifty products, $16 billion in revenue and approximately 8,000 employees across a range of therapeutic areas.

"I am honored to join Incyte at this pivotal moment and thank Hervé for his support during this transition," said Mr. Meury. "Incyte’s track record for discovering innovative treatments for complex problems in human health is outstanding. My priority is to build upon our exceptional R&D and commercial capabilities to accelerate new product flow, drive sustainable growth and create value for all stakeholders. I look forward to working with the Incyte team to continue to grow the company for enduring success."

"Bill is a decisive and collaborative leader who possesses the strategic planning and executional skills necessary to accelerate Incyte’s growth and maximize the tremendous opportunities we have in oncology and immunology," said Mr. Baker. "During our thoughtful succession process, Bill’s leadership experience stood out, including his ability to effectively translate scientific breakthroughs into business results. Bill is a great listener, a tireless learner and a direct and open communicator. I believe that he embraces Incyte’s values of hard work, commitment to excellence in science and business and dedication to bringing novel, innovative and life changing medicines to patients in need. In my new role as Chairman, I look forward to working closely with Bill to create long-term value for our shareholders."

"On behalf of the entire Board of Directors, I want to thank Hervé for his unwavering service as Chairman and CEO of Incyte," continued Baker. "It was at Hervé’s request last Fall that the Board of Directors engaged in a thorough succession planning process that led us to today’s transition. Hervé’s leadership and vision have been invaluable over the past decade, growing Incyte into a leading, diversified company. Hervé joined Incyte in 2014 when it was a single product, U.S.-only company. During Hervé’s tenure, Incyte launched six novel medicines plus two new indications for Jakafi, expanded commercial operations into Europe, Japan and Canada and grew revenues from $355 million dollars in 2013 to $4.2 billion today. Hervé fostered a culture of innovation and collaboration that will carry on at Incyte. I would like to thank him for his hard work and dedication to the Company, which I expect will continue through the coming years, and wish him the best in his retirement."

"It has been a privilege to lead Incyte over the past eleven years," said Mr. Hoppenot. "Guided by our mission to Solve On. and relying on great science, together we have achieved remarkable success in our effort to address unmet medical needs. I am proud to retire at a time when Incyte has the strongest management team, internal R&D pipeline and commercial portfolio ever. With Bill’s leadership, I am confident that the Company will continue its legacy of delivering transformative solutions to patients for many years to come."

About Bill Meury

Bill Meury was formerly CEO of Anthos Therapeutic, a Blackstone Life Sciences portfolio company, which was acquired by Novartis in April 2025. Mr. Meury joined Anthos after leading Karuna Therapeutics, a biopharmaceutical company focused on neuroscience, until its merger with Bristol Meyers Squibb in March 2024. Prior to Karuna, he served as a Partner at Hildred Capital Management, a private equity firm focusing on healthcare products and services. Prior to Hildred Capital Management, he was Executive Vice President and Chief Commercial Officer at Allergan, until the time of its acquisition by AbbVie, where he had responsibility for over 50 products with $16 billion in revenue and over $3 billion in operating investment. In this role, he led multiple global divisions totaling approximately 8,000 employees, including marketing, sales, business development, marketing analytics, managed care and customer service. Mr. Meury has experience across a broad range of therapeutics areas and has been involved with over 20 U.S. Food and Drug Administration (FDA) approvals and new product launches. He also served as Allergan’s President, Branded Pharma and Executive Vice President, Commercial, North American Brands, and as Executive Vice President Commercial Operations, at Forest Laboratories prior to its acquisition by Allergan.

Mr. Meury currently serves on the board of directors of the Jed Foundation. He received his B.A. in Economics from the University of Maryland.

About Julian Baker

Julian Baker is Managing Partner of Baker Bros. Advisors LP ("BBA") a biotechnology-focused, long-term investment adviser. Mr. Baker founded BBA, together with his brother and Co-Managing Partner Dr. Felix Baker, in 2000. Prior to BBA, Mr. Baker and his brother were portfolio managers of a biotech-focused investment partnership at Tisch Financial Management from 1994 to 1999. Previously, Mr. Baker was employed from 1988 to 1993 by the private equity investment arm of Credit Suisse First Boston Corporation.

In addition to Incyte, Mr. Baker serves as Chairman of Madrigal Pharmaceuticals, Chairman of Denali Therapeutics and as a Director of Acadia Pharmaceuticals. Mr. Baker holds an A.B. Magna Cum Laude from Harvard University.

On June 26, 2025 Bristol Myers Squibb (NYSE: BMY) reported that the U.S. Food and Drug Administration (FDA) has approved label updates for both of its CAR T cell therapies, Breyanzi (lisocabtagene maraleucel; liso-cel) for the treatment of large B cell lymphoma (LBCL) and other lymphomas and Abecma (idecabtagene vicleucel; ide-cel) for the treatment of multiple myeloma (Press release, Bristol-Myers Squibb, JUN 26, 2025, View Source;Food-and-Drug-Administration-Approves-Streamlined-Patient-Monitoring-Requirements-and-Removal-of-REMS-Programs-within-Bristol-Myers-Squibbs-Cell-Therapy-Labels/default.aspx [SID1234654133]). These label updates reduce certain patient monitoring requirements and remove the Risk Evaluation and Mitigation Strategy (REMS) programs that had been in place since each product was initially approved.

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Despite the transformative potential of cell therapy, only about 2 in 10 eligible patients receive it, due to the confluence of complex logistical and geographic barriers affecting patients and providers. BMS is committed to a long-term goal of expanding access to cell therapy and supports today’s class-wide label updates that will help ease known barriers to treatment and administration while maintaining patient safety.

Across both labels, the FDA has approved the reduction or removal of specific patient monitoring requirements for Breyanzi and Abecma. These prolonged requirements posed burdens on healthcare delivery systems and for certain patients and their care partners, particularly those who live far from certified cell therapy treatment centers. The changes include:

Driving restrictions reduced from 8 weeks to 2 weeks post treatment
Requirement to stay within proximity of a healthcare facility following infusion reduced from 4 weeks to 2 weeks
"CAR T cell therapy is a transformational, potentially life-saving option for patients living with blood cancers, and we are working to challenge current practices, assumptions and barriers that limit access," said Lynelle B. Hoch, president, Cell Therapy Organization, Bristol Myers Squibb. "Today’s FDA-approved label updates reinforce BMS’ continued efforts to collaborate across the healthcare ecosystem, with the ultimate goal of reaching more patients and democratizing access to cell therapy."

The FDA has also approved removal of the REMS requirement from each product label. A REMS program is often required to help mitigate known or potential risks associated with new drugs or therapies. The FDA has since determined that the established management guidelines and extensive experience of the medical hematology/oncology community are sufficient to diagnose and manage the risks of side effects, including cytokine release syndrome (CRS) and neurologic toxicities (NTs), without a REMS for the class of CD19- and BCMA-directed autologous CAR T cell therapies. This change is likely to help further accelerate cell therapy into the community center setting.

Together, these label updates reflect the growing body of clinical and real-world evidence underpinning the favorable efficacy and safety profile of CAR T cell therapy. To date, more than 30,000 patients have been treated with a CAR T cell therapy, with recent studies, including an analysis BMS presented earlier this month at the ASCO (Free ASCO Whitepaper) Annual Meeting, showing that the vast majority of serious adverse events (CRS and NTs) occur within the first two weeks of infusion.

Following this announcement, BMS will work closely with the more than 150 treatment centers currently approved to administer Breyanzi and Abecma to remove the REMS programs. In parallel, BMS is focused on rapidly expanding the geographic footprint of cell therapy, with a renewed effort to add community cancer centers nationwide to administer Breyanzi and Abecma closer to patients, helping further reduce travel time and duration of stay away from home, family and work.

"Living with blood cancer is challenging, but patients and their loved ones still need to maintain jobs, take care of families, and plan for the future," said Sally Werner, chief executive officer, Cancer Support Community. "Today’s announcement reduces some of the most onerous requirements that may have previously discouraged patients, particularly those who live far from a treatment center, from seeking the potentially transformational effects of cell therapy. We applaud any and all efforts to continue to break down barriers, reduce time burden on patients and caregivers, and increase uptake of this life-saving therapy."

As BMS continues to bring cell therapy to more patients, we are committed to working across the healthcare ecosystem to implement these label updates and continue to design and implement measurable programs to increase uptake and equitable access to cell therapy. For a list of programs and services currently offered to support patients through their BMS cell therapy journey, visit celltherapy360.com.