Longeveron Announces Positive Type C Meeting With FDA Regarding Pathway to BLA

Longeveron Inc. recently announced the positive Type C meeting with the US FDA supporting the advancement of Lomecel-B, a proprietary, scalable, allogeneic, investigational cellular therapy currently being evaluated in a Phase 2b clinical trial (ELPIS II) for hypoplastic left heart syndrome (HLHS).

The company and the FDA reached foundational alignment on the primary endpoint and secondary endpoints for ELPIS II. The FDA confirmed that, with several conditional requirements to be accomplished, ELPIS II may be deemed pivotal, and, if positive, acceptable for Biological License Application (BLA) submission for full traditional approval. Among other items, the Company will need to submit its prespecified Statistical Analysis Plan (SAP) and Chemistry, Manufacturing and Controls (CMC) readiness plan, including Lomecel-B stability and comparability data, to the FDA for prior review.

“We are pleased to have alignment with FDA on the development pathway for our Lomecel-B development program in HLHS, which has a devastating impact on patients and their families,” said Wa’el Hashad, Chief Executive Officer of Longeveron. “While we have a lot of work yet to do, the potential for ELPIS II to serve as the foundation for a BLA submission significantly reduces the time to reach submission and potential approval of Lomecel-B as an HLHS adjunct therapy.”

ELPIS II builds on the positive clinical results of ELPIS I, in which children in the trial experienced 100% transplant-free survival up to five years of age after receiving Lomecel-B compared to an approximate 20% mortality rate observed from historical control data.

ELPIS II is being conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) through grants from the National Institutes of Health (NIH).

The Lomecel-B HLHS program has received three FDA designations: Orphan Drug designation, Fast Track designation and Rare Pediatric Disease designation. Under the Rare Pediatric Disease designation, if Longeveron were to receive FDA marketing approval for Lomecel-B for HLHS, the company could be eligible to receive a Priority Review Voucher.

Lomecel-B is an allogeneic, investigational product made from specialized cells isolated from the bone marrow of young healthy adult donors. These specialized cells, known as medicinal signaling cells (MSCs), are essential to the endogenous biological repair mechanism. MSCs have shown high promise for cardiac regenerative therapy (Kaushal and Wehman 2015; Wehman et al. 2016). They also have been shown to respond to sites of injury or disease and secrete bioactive factors that are anti-inflammatory and regenerative. Longeveron believes that Lomecel-B may have multiple potential mechanisms of action that may lead to anti-inflammatory, pro-vascular regenerative responses, and therefore may have broad application for a range of rare and aging related diseases.

HLHS is a rare congenital heart defect that effects approximately 1,000 infants per year in the US. Infants with HLHS are born with an underdeveloped left ventricle, which creates a life-threatening condition due to the heart’s inability to pump adequate amounts of blood throughout the body. The current treatment requires infants to undergo a complex three-stage heart reconstruction surgery process over the first five years of their life. Even with this comprehensive treatment, only 50% to 60% of infants survive to adolescence due to right ventricular failure. There is clearly an important unmet medical need to improve right ventricular function in these infants to positively impact both short- and long-term patient outcomes.

Longeveron is a clinical stage biotechnology company developing regenerative medicines to address unmet medical needs. The company’s lead investigational product is Lomecel-B, an allogeneic medicinal signaling cell (MSC) therapy product isolated from the bone marrow of young, healthy adult donors. Lomecel-B has multiple potential mechanisms of action encompassing pro-vascular, pro-regenerative, anti-inflammatory, and tissue repair and healing effects with broad potential applications across a spectrum of disease areas. Longeveron is currently pursuing three pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer’s disease, and Aging-related Frailty. Lomecel-B development programs have received five distinct and important US FDA designations: for the HLHS program – Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation; and, for the AD program – Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation. For more information, visit www.longeveron.com.