UMass Chan Medical School

ALY-301 is a first-in-class bispecific antibody targeting c-Kit and CD-203c with mast cell selectivity Designed to enable safe dosing in chronic conditions and deliver clinical proof-of concept in a Phase 1/1b study BOSTON and LAUSANNE, Switzerland, June 10, 2025 /PRNewswire/ -- Alys Pharmaceuticals, Inc. ("Alys"), an immune-dermatology focused company, today announces the submission of a Clinical Trial Application (CTA) to Germany's Paul-Ehrlich-Institut (PEI) to initiate a Phase 1/1b study of ALY-301 in healthy volunteers and patients suffering from Cold Urticaria, a subtype of Chronic Inducible Urticaria (CIndU). Cold Urticaria is a chronic skin disease where red, itchy wheals or hives appear after the skin is exposed to cold temperatures. It is a well-established clinical model for mast cell-mediated diseases in general, and chronic spontaneous urticaria in particular. Current and emerging solutions do not address the significant unmet medical needs experienced by patients. In conjunction with limited treatment options, the Urticaria market is projected to reach multiple billions within the next ten years. ALY-301 is the first mast cell-selective c-Kit inhibitor to enter the clinic. It is a cis-targeting bispecific antibody binding c-Kit and CD-203c, allowing selective targeting and depletion of mast cells over other c-Kit positive cells. This differentiated profile of ALY-301 is expected to offer a significantly improved safety profile over conventional c-Kit targeting agents and enable safe, chronic dosing in multiple mast-cell dependent diseases such as Chronic Urticaria. The trial will evaluate the safety and efficacy of ALY-301 at multiple sites across Germany. This CTA submission is the first clinical program to emerge from Alys' Granular platform, which is focused on the precise targeting of mast cell biology through advanced antibody engineering. Professor Brian Kim, Sol and Clara Kest Professor of Dermatology and Vice Chair of Research at Icahn School of Medicine at Mount Sinai and Head of Alys Pharmaceuticals Scientific Advisory Board, said: "ALY-301 offers a truly differentiated approach. It is the only c-Kit antibody designed to target activated mast cells while avoiding off-target effects. Unleashing the proven efficacy of c-Kit inhibition on mast cells, while sparing all other cell-types that have been associated with significant side effects, should result in a transformational drug profile for ALY-301 and could have a revolutionary impact on Chronic Urticaria and several other allergic indications." Professor Martin Metz, Professor of Dermatology at the Institute of Allergology at the Charité – Universitätsmedizin Berlin & Fraunhofer Institute for Translational Medicine and Pharmacology, Allergology and Immunology and Principal Investigator for ALY-301, commented: "The concept behind ALY-301, to enable safe and effective chronic dosing of a c-Kit agent through mast-cell selectivity, has been strongly supported across all non-clinical studies. It is very exciting to start the clinical journey today, especially since this first clinical trial will yield clinical outcomes from Urticaria patients." Thibaud Portal, Co-Founder and Chief Operating Officer of Alys Pharmaceuticals, added: "Recently, there has been intense attention to solving Urticaria and other allergic diseases, with keen interest in c-Kit targeting. Data have demonstrated the remarkable potency of the mode of action, but indiscriminate c-Kit targeting has been coupled with chronic safety challenges. ALY-301 is a first-in-class approach that combines the power of c-Kit inhibition with selectivity and safety. With its expected safety margin and broad applicability across multiple diseases, this marks another pivotal moment for Alys in our journey to bring innovative treatments to patients." This study marks the second major clinical milestone for Alys Pharmaceuticals. The Alys pipeline includes a diverse portfolio of assets targeting unmet needs in indications such as atopic dermatitis, chronic spontaneous urticaria, vitiligo, and systemic mastocytosis. The Company remains on track to deliver multiple clinical Proof-of-Concept (POC) readouts by 2027. About Alys Pharmaceuticals Alys Pharmaceuticals, Inc. ("Alys") is a Boston and Lausanne-based cutting edge pure-play immuno-dermatology company. Alys is backed by international investment firm Medicxi with $100M financing. Alys has a world class leadership team that brings together experts across dermatology and advanced scientific fields, including co-founders John Harris (UMass Chan Medical School), Brian Kim (Icahn School of Medicine at Mount Sinai), Lars French (LMU, Munich), Craig Mello (UMass Chan Medical School), Eric Deutsch (Gustave Roussy) and Mark Prausnitz (Georgia Institute of Technology). Alys is led by dermatology specialist, co-founder, and COO Thibaud Portal. Its pipeline includes programs targeting multiple dermatological indications, including atopic dermatitis, vitiligo, chronic urticaria, psoriasis and mastocytosis. The Company entered the clinic in Q1 2025 with the siRNA platform developed by its affiliate Aldena Therapeutics, which offers the potential for long-lasting treatments that could transform the dermatology treatment landscape and is now advancing its mastocyte-selective therapies developed by affiliate Granular Therapeutics. For further information, please visit . SOURCE Alys Pharmaceuticals WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

NEW YORK, May 29, 2025 /PRNewswire/ -- Breakthrough T1D, formerly JDRF, the leading global type 1 diabetes (T1D) research and advocacy organization, is pleased to announce two exciting developments at the Breakthrough T1D Center of Excellence in New England that bolster its commitment to accelerating scientific breakthroughs on the path to cures for T1D. In recognition of the steadfast support of Breakthrough T1D volunteer leader John Cammett, the Breakthrough T1D Center of Excellence in New England has been named after his mother, who lived with T1D for nearly 60 years. The center will now be known as the Breakthrough T1D Barbara Dewey Cammett Center of Excellence in New England. John Cammett helped establish the Breakthrough T1D Center of Excellence in New England and has provided foundational support for Project ACT, Breakthrough T1D's initiative to make curative cell therapies for T1D a reality. "Throughout my childhood, I admired the way my mother lived a full life while navigating her type 1 diabetes. When I was diagnosed with type 1 diabetes as an adult, she became even more of an inspiration to me," said John Cammett. "She helped fuel my passion and commitment to doing all I can to support Breakthrough T1D and help find cures for this disease. The Breakthrough T1D Center of Excellence in New England is driving cell therapy research that may one day allow all of us to walk away from type 1 diabetes. I'm honored that my mother's legacy will be part of this groundbreaking effort." After five years of steady progress and growth in multiple research fronts, the Breakthrough T1D Barbara Dewey Cammett Center of Excellence in New England has been awarded additional funding over the next four years to maintain its momentum in accelerating cell therapy research in pursuit of cures for T1D. Since its inception, it has made significant progress advancing islet cell-based T1D research, enhancing the understanding of the immune response following islet transplantation to prevent rejection, and creating genetically modified islet cells that could withstand the immune attack after transplantation into people living with T1D. The continued funding will support research that builds on this work to further advance cell therapy research with a focus on refined approaches to make islet cells healthier and more resilient to the immune system and metabolic and infectious stressors. It will continue to be led by Dr. David Harlan at UMass Chan Medical School. Significantly, Dr. Harlan and co-investigators have expanded the Center, bringing in leading experts in innovative areas to complement the existing team. "Accelerating cell therapies from our Centers of Excellence is a key priority area for Breakthrough T1D, and one we believe will lead us to cures for type 1 diabetes," said Breakthrough T1D vice president of research Esther Latres, PhD. "Our Centers of Excellence allow for unique collaboration, innovation, and career development that make the accomplishment of joint projects that could not be achieved as individual efforts possible. The Breakthrough T1D Barbara Dewey Cammett Center of Excellence in New England is well-positioned to address the remaining key challenges in cell therapy that provide benefits to the entire type 1 diabetes community." The center's new name announcement coincides with a generous $1 million gift from the Bukhman Foundation for the Breakthrough T1D Barbara Dewey Cammett Center of Excellence in New England. Established in 2023, the Bukhman Foundation supports medical research, access to quality education, and strengthening arts and culture. Breakthrough T1D Centers of Excellence align with the organization's research strategy and seek to leverage the expertise, specialty, and resources of world-class institutions and researchers to accelerate T1D treatments and therapies. Bringing together leading experts from diverse fields and multiple institutions, each Breakthrough T1D Center of Excellence builds on established expertise to accelerate T1D advances. The Center of Excellence model is designed to maximize resources, remove barriers, link research efforts around the world, develop the next generation of scientific leadership, and foster maximum collaboration to drive progress as quickly as possible. The Breakthrough T1D Barbara Dewey Cammett Center of Excellence is one of five around the world, powering advances to deliver cures and life-improving breakthroughs for T1D. About Breakthrough T1D, Formerly JDRF As the leading global type 1 diabetes research and advocacy organization, Breakthrough T1D helps make everyday life with type 1 diabetes better while driving toward cures. We do this by investing in the most promising research, advocating for progress by working with government to address issues that impact the T1D community, and helping educate and empower individuals facing this condition. About Type 1 Diabetes (T1D) T1D is an autoimmune condition that causes the pancreas to make very little insulin or none at all. This leads to dependence on insulin therapy and the risk of short and long-term complications, which can include highs and lows in blood sugar; damage to the kidneys, eyes, nerves, and heart; and even death. Globally, it impacts nearly 9 million people. Many believe T1D is only diagnosed in childhood and adolescence, but diagnosis in adulthood is common and accounts for nearly 50% of all T1D diagnoses. The onset of T1D has nothing to do with diet or lifestyle. While its causes are not yet entirely understood, scientists believe that both genetic factors and environmental triggers are involved. There is currently no cure for T1D. Contact: Casey Fielder 509-651-0087 [email protected] SOURCE Breakthrough T1D, Formerly JDRF WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

None Tadaaki Taniguchi, M.D., Ph.D., Chief Research & Development Officer, AstellasWith more than 40,000 delegates due to gather in Chicago this week for the American Society of Clinical Oncology (ASCO) Annual Meeting, the global oncology community is poised to shape the future of cancer care.And with good reason – cancer remains one of the biggest healthcare challenges facing the world today, with one in five developing it during their lifetime. Yet, in my role as Chief Research and Development Officer at Astellas, I am optimistic. As an industry we’re making important headway – harnessing the full power of science, technology and collaboration to deliver groundbreaking discoveries that have the potential to transform the lives of millions. But it will require us to draw on diverse approaches, technologies and resources to truly tackle cancer.Designing the oncology medicines of the futureMy time as a surgical oncologist brought home how devastating cancer can be. Astellas’ deep heritage in oncology and landmark therapies that have transformed treatment expectations are a key part of why I joined. Today, we are advancing several next-generation modalities which could potentially reshape cancer care, particularly in hard-to-treat cancers, as part of our long-term R&D strategy.Targeted Protein Degradation (TPD)Protein degradation could change treatment expectations in many types of diseases by unlocking and degrading targets previously considered undruggable.Advanced technologies are fuelling progress in this space. We have been combining in-house drug discovery capabilities – including state-of-the-art modelling, highly efficient molecular synthesis technologies, robotics and artificial intelligence (AI) – to create optimal TPDs quickly and efficiently. This allowed rapid progress of our lead TPD program into the clinic, one which targets KRAS G12D mutant protein, a key driver of cancer that is highly prevalent in pancreatic, colorectal and non-small cell lung cancers. As we look forward, these advanced capabilities will continue to strengthen the future of TPDs.Immuno-oncology (IO)We are also working to activate and enhance the immune system in novel ways, to reinvigorate its ability to discover, disarm and destroy more cancers in more patients. We’re advancing some of the most cutting-edge IO modalities, including immunostimulatory antibody drug conjugates, T-cell engagers and bispecific antibodies, to do just this. Our goal is to create a range of therapies that target multiple aspects of tumor biology and different stages of the cancer immune cycle. Our lead program is a bispecific immune cell engager which targets claudin 18.2, a protein that is highly expressed in multiple gastrointestinal tumors, and others, which still have significant unmet needs.Cancer is complex and persistent – we can’t cure it aloneCollaboration underpins many of oncology’s greatest success stories. At Astellas, we recognize innovation can come from anywhere, whether it’s academia, a biotech startup, regulatory bodies, health professionals and more. Which is why we have invested in a global network of innovation hubs and partner with leading institutions, such as Mass General Brigham and UMass Chan Medical School.Delivering highly complex and competitive next-generation therapies also requires increasingly specialized expertise. At Astellas, we recently established research centers of excellence to centralize our leading capabilities and accelerate progress with our partners – for example, to advance oncology research in antibody-based approaches, cancer cell therapy, engineered small molecules like TPDs, and more.Successful collaborations thrive when there is a balance between academic freedom and commercial objectives, empowering researchers to drive groundbreaking science within a culture of continuous learning, all while ensuring real-world impact.Urgency, agility and a flexible approach are the keys to success in cancerBreakthroughs will come when we work together. If we can combine this mindset with passion, persistence and urgency, we can make significant progress in detecting cancer early and discovering new treatments no matter the stage. By doing so, we can bring hope and change the outlook for millions of people courageously facing this disease.Learn more about Astellas in oncology, here, and find out about our unique approach, here.