Heart Failure

May 01, 2025 -- BioCardia, Inc. [NASDAQ: BCDA], a global leader in cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary diseases, today announced the first patient enrolled in the Phase III randomized procedure placebo-controlled double-blind CardiAMP HF II clinical trial of the Company’s lead autologous cell therapy program for the treatment of heart failure at BayCare Morton Plant Hospital in Clearwater, Florida. “We are excited to lead enrollment in the CardiAMP HF II trial and be able to offer this cell therapy study to our heart failure patients. This investigational therapy has been shown to enhance microvascular function and demonstrated promising clinical outcomes in the recently completed CardiAMP HF Trial, particularly for the patients we are targeting for this trial,” said Dr. Leslie Miller, M.D., Chief of the Congestive Heart Failure and Heart Function Clinic at BayCare Morton Plant Hospital in Clearwater, Florida. Dr. Miller is also a CardiAMP HF II Trial Principal Investigator and member of the Executive Steering Committee. “The unique ability to personalize this treatment based on a patient’s own cellular characteristics make CardiAMP cell therapy attractive for treating heart failure. The approach preserves all therapeutic options for our patients, including repeated cell therapy, and avoids the need for immune suppression. I congratulate and thank our team at Morton Plant Hospital for achieving this important milestone in enrolling the first patient in the world’s first confirmatory Phase 3 cardiac cell therapy trial for heart failure.” “Our first CardiAMP HF II study procedure went smoothly and per protocol,” said Dr. Rakesh Sharma, M.D., Interventional Cardiologist at BayCare Morton Plant Hospital in Clearwater, and CardiAMP HF II investigator. “Our efforts in cardiac cell therapy over many years have supported the potential of this CardiAMP cell therapy where cells are harvested, processed, and delivered into the heart muscle by our research team. I value the Helix minimally invasive cell intramyocardial delivery platform for its ability to enable targeting of specific segments in the heart with confidence and find the new Morph DNA steerable navigation catheter to be an elegant enhancement.” “We are thankful for the BayCare Morton Plant team led by Dr. Miller and Dr. Sharma as well as the many other world-class clinical teams engaged in the CardiAMP cell therapy trials,” said Peter Altman, PhD, BioCardia President and Chief Executive Officer. “We are preparing regulatory submissions in Japan and the USA intended to seek separate approvals of the CardiAMP cell therapy and Helix biotherapeutic delivery platform based on our existing TABMMI1, TAC-HFT2,3, and CardiAMP HF4 clinical data. The CardiAMP HF II trial is intended to collect confirmatory evidence to support this autologous cell therapy with respect to approvals, adoption, and reimbursement.” CardiAMP HF II is a 250-patient randomized multicenter procedure placebo-controlled study of the CardiAMP autologous cell therapy as a one-time treatment for patients with ischemic heart failure with reduced ejection fraction (HFrEF) on guideline directed medical therapy having elevated NTproBNP. The study is intended to confirm the safety and efficacy results in these patients observed in the CardiAMP HF study4. The CardiAMP HF II study uses a similar three-tier composite primary outcome measure to CardiAMP HF, with tier 1 all cause death, tier 2 nonfatal major adverse cardiac events, but with tier 3 using a validated quality of life patient self-assessment instrument. In CardiAMP HF, this composite efficacy endpoint was achieved with statistical significance in the patients with elevated NTproBNP that are the focus of the CardiAMP HF II study (p=0.02). Advances in this therapeutic approach in CardiAMP HF II include using the cell population analysis at screening to define treatment doses, which enables more patients to be eligible for the therapy, and improvements to the Helix system, which include the introduction of the FDA approved Morph DNA steerable platform. Granted FDA Breakthrough designation, CardiAMP Cell Therapy uses a patient’s own bone marrow cells delivered to the heart in a minimally invasive, catheter-based procedure to potentially stimulate the body’s natural healing response to increase capillary density, reduce tissue fibrosis, and ultimately treat microvascular dysfunction. The mechanisms that lead to microvascular dysfunction, including fibrotic, inflammatory, apoptotic, and endothelial autonomic dysfunction, are all targets of CardiAMP cell therapy, largely through production of growth factors, cytokines, chemokines, and other factors that directly counteract each of these mechanisms. The CardiAMP clinical development for heart failure is supported by the Maryland Stem Cell Research Fund and is reimbursed by Centers for Medicare and Medicaid Services (CMS). BioCardia, Inc., headquartered in Sunnyvale, California, is a global leader in cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary disease. CardiAMP® autologous and CardiALLO™ allogeneic cell therapies are the Company’s biotherapeutic platforms with three clinical stage product candidates in development. These therapies are enabled by its Helix™ biotherapeutic delivery and Morph® vascular navigation product platforms. References: de la Fuente LM, Stertzer SH, Argentieri J, et al. Transendocardial autologous bone marrow in myocardial infarction induced heart failure, two-year follow-up in an open-label phase I safety study (the TABMMI study). EuroIntervention. 2011 Nov;7(7):805-12. Heldman AW, DiFede DL, Fishman JE, et al. Transendocardial mesenchymal stem cells and mononuclear bone marrow cells for ischemic cardiomyopathy: the TAC-HFT randomized trial. JAMA. 2014 Jan 1;311(1):62-73. Wong Po Foo et al., The transendocardial autologous cells in ischemic heart failure trial bone marrow mononuclear cells (TAC-HFT-BMC) randomized placebo controlled blinded study, Regenerative Medicine 2015, 10(7s), S169. Raval A, on behalf of the CardiAMP HF Investigators. A Double-blind, Randomized Controlled Trial of an Autologous Cell Therapy in Patients with HFrEF: Principal Results from the CardiAMP-HF Trial, American College of Cardiology Scientific Sessions, March 30, 2025. 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Cytokinetics\' addition of a REMS plan constituted a \"major\" application amendment, triggering a three-month delay.\n To REMS, or not to REMS.That, apparently, was the question for Cytokinetics, which has seen any potential approval for its experimental heart drug delayed by an extra three months over an unusual safety program omission and addition. Aficamten, a cardiac myosin inhibitor, had been on the books with the FDA for a potential approval by (or before) Sept. 26 for obstructive hypertrophic cardiomyopathy.This disease, characterized by the blood flow from the heart being reduced by the thickening of the heart\'s muscular wall, already has an approved treatment on the market in the form of Bristol Myers Squibb’s cardiac myosin inhibitor Camzyos.Cytokinetics was hoping to get into this market that has seen Camyzos bring in just over $600 million last year, but, now, those hopes will be on hold a while longer.The whole issue revolves around REMS, the shorthand for the FDA\'s Risk Evaluation and Mitigation Strategy programs. REMS are typically demanded by the FDA for potentially risky drugs and are a common requirement for heart disease meds.In a twist of fate, BMS also had a similar FDA REMS delay back in 2021, but its medicine was ultimately approved in 2022.In the U.S., Camzyos can only be used via a REMS program because it can cause the heart to become too relaxed, increasing the risk of heart failure. In its statement released after-hours Thursday, May 1, Cytokinetics said it had submitted its new drug application with the FDA without a plan for REMS, seemingly with the agency’s blessing. But not so fast: The FDA has now looked over the application and in fact does want a REMS plan “based on the inherent characteristics of aficamten,\" the company explained. While Cytokinetics has now provided the plan, the addition has been regarded as a “Major Amendment to the NDA,” according to the biotech, thus adding a mandatory three months to the review. The drug\'s decision is now expected by Dec. 26.Analysts at Evercore ISI were perplexed, saying in a note to clients: “We didn’t see this one coming. The most surprising part to us is that [aficamten\'s] NDA was originally submitted without an accompanying REMS. Was that actually the FDA’s guidance to the company or some sort of miscommunication? We assume the former given that the application was accepted.”The analysts added, however, that “the REMS was not expected to be the main differentiator for [aficamten].\" Shares in the biotech were down around 10% premarket. “We remain confident in the distinct benefit-risk and pharmaceutic profile of aficamten and continue to expect a differentiated label and risk mitigation profile upon its potential approval by FDA,” said Robert Blum, Cytokinetics’ president and CEO, in the release.“We look forward to continuing our constructive engagement with the FDA regarding the NDA for aficamten.”

PITTSBURGH and BOSTON, May 1, 2025 /PRNewswire/ -- Synhale Therapeutics Inc. (Synhale), a virtual, clinical-stage biotech company committed to transforming Pulmonary Hypertension (PH), announced the acquisition of Telaglenastat (CB-839), the first-in-class glutaminase inhibitor. Synhale is advancing into a Phase 2 clinical program across PH Groups 1-4. "This acquisition represents a unique opportunity to rapidly deliver clinical data addressing a disease with high mortality and substantial market potential," said Chad D. Holland, President & CEO of Synhale. "Leveraging Telaglenastat's prior human clinical experience, our capital-efficient model aims to accelerate transformative therapy to patients while maximizing shareholder value." Stephen Chan, MD, PhD, Founder of Synhale, added, "The unmet patient need in PH is immense, particularly in Groups 2 and 3, where heart failure with preserved ejection fraction (HFpEF) and chronic lung disease remain deadly and underserved. PH with HFpEF carriers a five-year mortality approaching 50%, with similarly grim outcomes in COPD and other lung diseases. By targeting glutaminase activity, we address a fundamental metabolic pathway across all PH groups, offering hope to patients with few options." PH, a severe condition, is characterized by remodeling and stiffening in the pulmonary vasculature and often dramatically increases mortality risk when combined with other conditions. Current classifications fail to reflect the molecular drivers of PH, leaving treatment gaps. Synhale's approach with Telaglenastat targets elevated glutaminase activity, a key driver of vascular, cardiac, and pulmonary pathology, potentially benefiting not only rare Pulmonary Arterial Hypertension (Group 1) and chronic thromboembolic disease (Group 4) but also more prevalent PH linked to heart failure (Group 2) and chronic lung disease (Group 3). Telaglenastat, previously dosed in over 800 patients during oncology development, is Phase 2-ready with extensive preclinical data for PH. Synhale's clinical advancement strategy addresses an estimated $12B-$24B market opportunity across PH Groups 1-4. "We are embarking on our next round of fundraising to advance Telaglenastat through critical clinical milestones," added Holland. "Our virtual operating model enables us to be capital-efficient while maintaining world-class expertise focused on delivering breakthrough therapies for PH patients." About Synhale Therapeutics Synhale Therapeutics Inc. is a virtual biotech company dedicated to transforming Pulmonary Hypertension treatment by targeting elevated glutaminase activity—a key driver of vascular stiffness, fibrosis, and proliferation. Synhale's mission is to redefine PH treatment through mechanism-driven therapies that address the root cause of this disease. For Media & Investor Inquiries: Chad D. Holland [email protected] 617-758-8643 SOURCE Synhale Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED