CD11a

Cryopreserved allogeneic Treg infusions associated with slowed disease progression and decreased plasma neurofilament level in ALS HOUSTON, April 22, 2025 /PRNewswire/ -- New findings published today in NEJM Evidence reveal promising clinical outcome for Cellenkos Inc.'s cryopreserved, allogeneic, T regulatory (Treg) cells derived from umbilical cord blood, for the treatment of Amyotrophic Lateral Sclerosis (ALS)—a neurodegenerative disease with limited therapeutic options. The study reports that patients receiving multiple intravenous infusions of this "off-the-shelf" Treg therapy showed a measurable slowdown in disease progression. Prior to therapy, participants' ALS Functional Rating Scale-Revised (ALSFRS-R) scores declined at an average rate of −1.66 points per month. During treatment, that rate slowed significantly to −0.41 points per month, with a modest post-treatment decline of −0.60 points per month—suggesting sustained benefit. Clinical improvements were accompanied by reductions in plasma neurofilament levels, a biomarker correlated with neuronal injury. The Treg therapy was administered in an outpatient setting via peripheral IV, with each patient receiving a standardized fixed dose of 100 million cells. The regimen included weekly infusions for the first month, followed by monthly doses for six months, with optional continued dosing based on physician discretion. The therapy required no lymphodepletion, immunosuppression, interleukin-2, or HLA matching, and patients were discharged the same day. Importantly, no dose-limiting toxicities were reported. Six participants, with a median age of 48.5 years (range: 27–66), and a median baseline ALSFRS-R score of 31.5 (range: 23–43), received a median of 11 infusions (range: 6–22), and all were alive at the time of last follow-up. Among participants with sufficient follow-up data (n=4), the median follow-up duration was 18 months. "These results are both encouraging and transformative for those battling ALS," said Dr. Simrit Parmar, Founder of Cellenkos and faculty member at Texas A&M University. "Unlike conventional cell therapies that require complex, individualized cell harvesting and hospitalization, our Treg therapy is cryopreserved, ready-to-use, and outpatient-based. It eliminates the need for HLA matching and preconditioning. These findings offer a compelling proof of concept and lay the foundation for our next-generation neurotropic Tregs, CK0803." CK0803 Tregs, now in clinical trials, are modified to overexpress CD11a and CXCR3, improving their ability to migrate to inflamed brain regions, specifically, inflamed microglia — potentially enabling direct targeting of ALS-related pathology. These findings offer renewed hope for patients, families, and researchers working toward more accessible, non-invasive, and effective treatments for ALS. About Amyotrophic Lateral Sclerosis (ALS) ALS is a progressive neurodegenerative disease that affects nerve cells responsible for voluntary muscles. As the disease advances, individuals experience worsening muscle weakness, paralysis, and eventual respiratory failure. Most patients succumb to ALS within three to five years of diagnosis. Currently, there is no cure. About Cellenkos®, Inc. Cellenkos is a clinical-stage biotech company, advancing allogeneic, off-the-shelf, cord blood-derived T regulatory cell therapies for rare inflammatory and autoimmune diseases. Its pipeline includes: CK0801 for Aplastic Anemia CK0802 for Acute Respiratory Distress Syndrome CK0803 for ALS CK0804 for Myelofibrosis Using its proprietary CRANE® platform, Cellenkos tailors tissue directed Treg therapies that require no HLA or ABO matching, can be administered in outpatient settings, and are optimized for rapid, point of care delivery. Contact [email protected] SOURCE Cellenkos, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

NATICK, Mass.--( BUSINESS WIRE )-- AffyImmune Therapeutics , a clinical-stage biotechnology company committed to developing its novel, first-in-class ICAM-1 targeting chimeric antigen receptor (CAR) T cell therapy for solid tumors, today announced the appointment of Rick Rutter, Ph.D., and Jorge Nieva, M.D., to its Board of Directors. The addition of these distinguished leaders in clinical development and manufacturing strengthens AffyImmune’s commitment to advancing its AIC100 program in advanced thyroid cancers and non-small cell lung cancer (NSCLC). “AffyImmune is nearing completion of our Phase 1 trial and advancing toward determining the recommended Phase 2 dose in thyroid cancers, while defining the path for NSCLC. Expanding the board to include Rick and Jorge cannot be timelier. Not only will they bring decades of experience in their respective domains, they will also strengthen the board’s ability to steer and govern strategic matters. The management team and board will work closely together to deliver lifesaving cancer therapeutics for patients who currently have no effective treatments,” said Simone Song, Chairwoman, AffyImmune. “We are thrilled to welcome Rick and Jorge to our board at such an important time for AffyImmune, as we advance our CAR T cell product candidate AIC100 towards registrational studies for advanced thyroid cancers, and plan for our Phase 1 trial for non-small cell lung cancer,” said Daniel Janse, CEO, AffyImmune. “Their insight and counsel as members of our board will be invaluable as we validate a commercially robust CAR T cell manufacturing process and focus our NSCLC clinical strategy on patients that will receive the greatest benefit from AIC100.” Rick Rutter, Ph.D., brings over 40 years of experience in the pharmaceutical and biotech industries, with a deep expertise in CMC regulatory strategy and drug product manufacturing. Dr. Rutter has held several key positions, including Executive Vice President of Biotherapeutics Pharmaceutical Sciences at Pfizer. He currently serves as an expert consultant and advises both public and private companies on CMC and supply chain management. Jorge Nieva, M.D., is Associate Professor of Clinical Medicine at the Keck School of Medicine of the University of Southern California and Section Head of Lung and Head/Neck Tumors at the Norris Comprehensive Cancer Center. He served as a standing member of the U.S. Food and Drug Administration’s Oncology Drug Advisory Committee from 2021-2024. Dr. Nieva’s clinical research teams have been recognized for excellence in the conduct of clinical trials from the National Cancer Institute and the American Association of Oncology Research. “I am delighted to join AffyImmune’s Board and to work with the management team to bring industry-best CMC and supply chain practices, and ready AIC100 for registrational trials and commercial launch,” said Dr. Rutter. “I look forward to sharing my insights and supporting the company as it delivers timely and high-quality autologous CAR T cell products to advanced thyroid cancer and NSCLC patients with significant unmet medical need.” “Joining AffyImmune’s Board of Directors is an exciting opportunity to contribute to the expansion of AIC100 beyond advanced thyroid cancers, into larger indications such as NSCLC,” said Dr. Nieva. “I look forward to working alongside such a dedicated team to drive forward their innovative CAR T therapies. AffyImmune’s technology has the unique potential to broadly revolutionize treatment for patients with solid tumors.” About AffyImmune Therapeutics AffyImmune is a clinical-stage biopharmaceutical company committed to developing a first-in-class, ICAM-1 targeting and affinity-tuned LFA-1 binder CAR T therapy to transform the lives of patients with advanced solid cancers. The Company’s proprietary technology utilizing ICAM-1/LFA-1 interaction can potentially address the critical CAR T challenges such as toxicity, antigen loss, T-cell trafficking and exhaustion, while also allowing in vivo tracking of the CAR T cells. Its lead candidate, AIC100, is currently in a Phase 1 study to treat patients with advanced thyroid cancers, including advanced differentiated thyroid cancer and anaplastic thyroid cancer. AffyImmune is developing a focused pipeline in ICAM-1-positive cancers with high unmet medical need. AffyImmune is based in Natick, MA. For more information, visit www.affyimmune.com .

NATICK, Mass.--( BUSINESS WIRE )-- AffyImmune , a clinical-stage biotechnology company committed to developing novel, first-in-class chimeric antigen receptor (CAR) T cell therapies, announced today the designation of Regenerative Medicine Advanced Therapy (RMAT) by the U.S. Food and Drug Administration (FDA) for its CAR T-cell product candidate, AIC100 as a potential treatment for patients with recurrent anaplastic thyroid cancer (ATC), the most aggressive form of the disease. “We believe this important recognition from the FDA further supports the therapeutic potential of AIC100 to change the current treatment paradigm in advanced thyroid cancer and potentially other forms of aggressive solid tumors,” said Daniel Janse, Ph.D., CEO at AffyImmune. “RMAT designation was granted following the FDA’s review of safety and efficacy data from the first ten patients dosed with AIC100 in our Phase 1 study. We believe the RMAT designation reinforces the potential ability of AIC100 to meet the high unmet medical need in recurrent ATC, an aggressive disease where a standard of care is currently not available.” RMAT designation was designed to expedite the development and review of regenerative medicine therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious condition, and preliminary clinical evidence indicates the therapy has the potential to address unmet medical needs for the disease. Sponsor companies receiving RMAT designation can benefit from increased interactions with the FDA involving senior managers, with the goal of expediting drug development. “We remain focused on advancing the development of AIC100 for patients and families living with this devastating cancer,” said Sonal Gupta, M.D., Ph.D., Senior Vice President and Head of Clinical Development. “Receiving RMAT designation helps facilitate this goal by enabling increased dialogue with the FDA to expedite our development plan for our affinity-tuned CAR T therapy. We look forward to working closely with the FDA and other regulatory agencies as we continue to advance this program.” At ASCO 2024, AffyImmune reported interim results from their Phase 1 study evaluating the safety and efficacy of AIC100, an ICAM-1 targeting and affinity-tuned LFA-1 binder CAR T-cell therapy, in patients with advanced thyroid cancer. Notably, a metabolic complete response was achieved in one patient with ATC, the most aggressive form of the disease. For more information about the Phase 1 study, visit www.clinicaltrials.gov (NCT04420754). About AffyImmune Therapeutics AffyImmune is a clinical-stage biopharmaceutical company committed to developing a first-in-class, ICAM-1 targeting and affinity-tuned LFA-1 binder CAR T therapy to transform the lives of patients with advanced solid cancers. The Company’s proprietary technology utilizing ICAM-1/LFA-1 interaction can potentially address the critical CAR T challenges such as toxicity, antigen loss, T-cell trafficking and exhaustion, while also allowing in vivo tracking of the CAR T cells. Its lead candidate, AIC100, is currently in a Phase 1 study to treat patients with advanced thyroid cancers including advanced differentiated thyroid cancer and ATC. AffyImmune is developing a focused pipeline in ICAM-1-positive cancers with high unmet medical need. AffyImmune is based in Natick, Massachusetts. For more information, visit www.affyimmune.com .