Longeveron Reports Positive FDA Type C Meeting on BLA Pathway for Lomecel-B in HLHS

Longeveron Inc. (NASDAQ: LGVN), a clinical-stage biotechnology company focused on regenerative medicine, has announced a successful Type C meeting with the U.S. Food and Drug Administration (FDA). This meeting centered on the advancement of Lomecel-B™, an investigational cellular therapy, currently under evaluation in a Phase 2b clinical trial (ELPIS II) for hypoplastic left heart syndrome (HLHS).

The company and the FDA reached a critical agreement on the primary and secondary endpoints for the ELPIS II trial. The FDA has indicated that, subject to certain conditions being met, the ELPIS II study could be considered pivotal. If the study results are positive, it will pave the way for a Biological License Application (BLA) submission for full traditional approval. Among other requirements, Longeveron must submit its prespecified Statistical Analysis Plan (SAP) and Chemistry, Manufacturing, and Controls (CMC) readiness plan, including data on Lomecel-B™ stability and comparability, for FDA review.

"This alignment with the FDA on our Lomecel-B™ program for HLHS represents a significant step forward," said Wa’el Hashad, CEO of Longeveron. "While there is still considerable work ahead, the possibility of ELPIS II forming the basis for a BLA submission shortens the timeline for potential approval of Lomecel-B™ as an adjunct therapy for HLHS."

The ELPIS II trial builds on the positive outcomes of ELPIS I, where children treated with Lomecel-B™ showed a 100% transplant-free survival rate up to five years of age, compared to an approximate 20% mortality rate based on historical data. The ELPIS II trial is being conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) with grants from the National Institutes of Health (NIH).

Longeveron's Lomecel-B™ program for HLHS has received three FDA designations: Orphan Drug, Fast Track, and Rare Pediatric Disease. If Lomecel-B™ receives FDA marketing approval for HLHS, Longeveron could be eligible for a Priority Review Voucher under the Rare Pediatric Disease designation. 

Lomecel-B™ is an allogeneic investigational product derived from medicinal signaling cells (MSCs) isolated from the bone marrow of young, healthy adult donors. These MSCs play a crucial role in the body's natural repair mechanisms, exhibiting potential for cardiac regenerative therapy. They respond to injury or disease sites by secreting bioactive factors that have anti-inflammatory and regenerative properties. Longeveron believes that Lomecel-B™ could have multiple mechanisms of action, contributing to anti-inflammatory and pro-vascular regenerative responses, which may be applicable to a variety of rare and aging-related diseases.

HLHS is a rare congenital heart defect affecting about 1,000 infants annually in the U.S. Infants born with this condition have an underdeveloped left ventricle, creating a life-threatening situation as the heart cannot pump sufficient blood throughout the body. The standard treatment involves a complex three-stage heart reconstruction surgery within the first five years of life. However, even with this treatment, only 50% to 60% of these infants survive to adolescence due to right ventricular failure. Improving right ventricular function is thus essential to enhancing both short- and long-term outcomes for these patients.

Longeveron focuses on developing regenerative medicines to address unmet medical needs. Its leading investigational product, Lomecel-B™, is an MSC therapy with promising applications across various disease areas due to its pro-vascular, pro-regenerative, anti-inflammatory, and tissue-healing properties. The company's pipeline includes indications for HLHS, Alzheimer’s disease, and aging-related frailty. Lomecel-B™ development programs have received five crucial U.S. FDA designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease for HLHS, and Regenerative Medicine Advanced Therapy (RMAT) and Fast Track for Alzheimer's disease.

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