Longeveron Inc. Q3 FY2024 earnings call

• Longeveron is a regenerative medicine company developing Lomecel-B, a cellular therapy with U.S. market potential of $10B to $18B.
• Reached 10th anniversary; Lomecel-B administered to over 540 patients with positive results in trials for Alzheimer's, aging-related frailty, and HLHS.
• HLHS program: ELPIS II over 80% enrolled, Type C meeting with FDA confirmed ELPIS II is pivotal for BLA submission; Devin Blass joined as CTO to lead technology and manufacturing strategy.
• Alzheimer's program: CLEAR MIND Phase 2a results showed slowing of disease worsening; FDA granted RMAT and fast-track designations; plan to meet FDA in Q1 2025 to review clinical and regulatory strategy.
• Expense control: Total operating expenses down 14% Y/Y; cash and cash equivalents as of September 30, 2024, were $22.8 million, expected to fund operations through Q4 2025.

Revenues for the first nine months of 2024 were $1.8 million, up $1.1 million (177%) from the same period in 2023, driven by increased participant demand for frailty and cognitive impairment registry trial in the Bahamas and new contract manufacturing revenue ($0.8 million for the nine months ended September 30, 2024). Total operating expenses through the first nine months declined 14% year-over-year. G&A expenses for the nine-month period ended September 30, 2024, decreased to approximately $7.4 million (vs. $8.9 million in 2023), and R&D expenses decreased approximately $0.8 million (11%) to approximately $6.1 million. Net loss decreased 22% to approximately $11.9 million for the nine months ended September 30, 2024, from $15.3 million in 2023.

• Existing cash and cash equivalents expected to fund the company through the fourth quarter of 2025.
• If ELPIS II trial is successful, plan to initiate a rolling submission of BLA to the FDA in 2026.
• May need to seek additional financing, capital raises, or non-dilutive funding to support ramped-up BLA-enabling activities in 2025.

• Uncertainties in clinical trial outcomes, including potential delays or failures in ELPIS II or Alzheimer's disease trials.
• Regulatory uncertainties related to FDA approval processes for Lomecel-B in HLHS and Alzheimer's.
• Potential need for additional financing beyond current projections to support accelerated BLA-enabling activities.

Q: What would represent an appropriate benchmark from a performance standpoint in the ELPIS II trial that would catalyze the accelerated approval? And what's the current thinking with respect to the path forward for Lomecel-B and Alzheimer's disease?

A: Met with the FDA, confirmed ELPIS II is pivotal for full approval and CMC plan; Alzheimer's program plans a streamlined approach with RMAT designation, aiming for a faster and more economical path to market.

Q: With respect to ELPIS I trial, long-term follow-up, and whether additional cardiac parameters could be measured?

A: Currently only survival and transplant-free survival data; entertaining the idea of retrospectively gathering more granular data on ejection fraction, tricuspid regurgitation, etc., by revising contracts with sites.

Q: Whether the HLHS competitive landscape has meaningfully changed in the recent past, specifically regarding VentriGel?

A: VentriGel is an inert substance; Lomecel-B has multifactorial action (anti-inflammatory, pro-vascular, anti-fibrotic, pro-regenerative); mechanism of action differs, so VentriGel is not a strong competitor at this point