Longeveron Secures FDA Type C Meeting Before ELPIS II Pivotal Data in Q3 2026

Longeveron Inc. is advancing its rare disease program after receiving confirmation of a Type C meeting with the U.S. FDA, scheduled for late March. This regulatory milestone comes strategically ahead of the anticipated Q3 2026 data readout from ELPIS II, the company’s pivotal Phase 2 clinical trial of Laromestrocel (Lomecel-B), an investigational allogeneic stem-cell therapy designed for Hypoplastic Left Heart Syndrome (HLHS).

Understanding HLHS: Why This Therapy Matters

Hypoplastic Left Heart Syndrome represents one of the most critical congenital heart defects, where the left side of the heart fails to develop adequately. The condition carries severe clinical implications: even with current three-stage surgical reconstruction protocols, approximately 50% of affected infants reach adolescence. This sobering survival statistic underscores the urgent clinical need for innovative therapeutic approaches beyond traditional surgical intervention.

Laromestrocel functions as an allogeneic medicinal signaling cell therapy engineered to restore cardiac function through two primary mechanisms: promoting tissue repair and reducing inflammatory responses. The FDA has recognized the therapy’s potential by granting multiple designations including Rare Pediatric Disease status, Orphan Drug designation, and Fast Track classification for HLHS treatment.

The Strategic Role of Type C Meeting in BLA Pathway

Longeveron’s request for the Type C meeting reflects strategic planning around the regulatory pathway. The company seeks FDA guidance on critical regulatory components of the ELPIS II program, specifically focusing on clinical efficacy endpoints and the statistical analysis plan (SAP) that will support a future Biologics License Application (BLA).

This meeting represents a prudent step: by obtaining regulatory feedback now, Longeveron aims to align its data collection and analysis framework with FDA expectations before submitting definitive trial results. Should Q3 2026 data demonstrate clinically meaningful benefits, the company can move forward with a traditional BLA submission with greater regulatory clarity. The March meeting builds upon prior FDA interactions where the agency has already provided guidance on ELPIS II’s pivotal status, trial design considerations, and chemistry-manufacturing-controls (CMC) and potency assay evaluations.

ELPIS II Trial Design and Patient Population

The ELPIS II Phase 2b trial has enrolled 40 pediatric patients across twelve leading pediatric and infant care centers throughout the United States. The study operates as a collaboration between Longeveron and the National Heart, Lung, and Blood Institute, supported through grants from the National Institutes of Health.

The trial’s primary objectives center on measuring cardiac functional outcomes, assessing survival rates without requiring heart transplantation, and evaluating long-term ventricular performance. If trial results support efficacy and safety, these data could form the foundation for full traditional BLA submission, potentially positioning Laromestrocel as the first regenerative therapy option available for this vulnerable pediatric population.

Longeveron plans to issue a regulatory update following receipt of official FDA meeting minutes. Currently, the company’s stock has traded within a $0.49 to $1.92 range over the past 12 months, with recent trading near $0.60 per share.