Longeveron Inc. Q1 FY2026 earnings call

• Strategic Repositioning and Capital Raising

  • New CEO Stephen Willard led a comprehensive review of company assets, development pipeline, and strategic plan after taking office in February 2026
  • The company reoriented to a capital-efficient, asset-light operating model focused on securing strategic licensing partnerships for lead product LaramastroCell across all four development programs
  • Longevron successfully secured new investment capital from top-tier life science investment funds including Coastlands Capital, Janus Henderson Investors, Logos Capital, and Kalahaua Capital
  • The company will participate in the 2026 Bio International Convention in San Diego in June to hold partnership discussions for all four stem cell programs

• Pipeline and Clinical Development Updates

  • The lead indication hypoplastic left heart syndrome (HLHS) Phase 2b ELPAS-2 trial completed enrollment of 40 patients in June 2025, with top-line data readout expected in August 2026; this is the company's top near-term priority
  • A recent Type C meeting with the FDA confirmed the agency does not consider the original primary endpoint (right ventricle ejection fraction) appropriate to demonstrate efficacy, and will not designate the ongoing trial as pivotal as agreed in 2024; the FDA has agreed to meet after trial completion to align on a future development path, and the company is capturing all objective clinical endpoints (all-cause mortality, transplant-free survival, major adverse cardiac events) as recommended by the FDA
  • The investigational new drug (IND) application for pediatric dilated cardiomyopathy (PDCM) became effective in July 2025, allowing direct advancement to a single Phase 2 registration trial; trial planning and preparation will continue in 2026, with study initiation expected in 2027, and trial endpoints have already been agreed upon with the FDA
  • Additional pipeline indications include Alzheimer's disease and aging-related frailty, representing multiple independent value creation opportunities

• Intellectual Property and Regulatory Positioning

  • LaramastroCell is supported by a robust IP portfolio of 52 issued patents and over 60 pending patents worldwide
  • The therapy has received five FDA expedited designations, including Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations, which reinforce its clinical promise and regulatory positioning
  • The four initial indications address estimated total market opportunity of up to $11 billion across all programs

Longevron reported total revenue of $0.4 million for Q1 2026, flat year-over-year compared to $0.4 million in Q1 2025. Clinical trial revenue contributed $0.4 million (100% of total revenue) in Q1 2026, an increase of $0.1 million (46% YoY) driven by higher participant demand for the company's Bahamas registry trial. Contract manufacturing revenue contributed $20,000 (5% of total revenue) in Q1 2026, a decrease of $0.1 million (84% YoY) due to reduced third-party client demand. General and administrative expenses were $2.7 million in Q1 2026, a 7% ($0.2 million) decrease YoY, driven by lower 2025 annual performance bonuses partially offset by higher professional fees. Research and development expenses were $2.3 million in Q1 2026, an 8% ($0.2 million) decrease YoY, driven by lower bonuses and a 2025 non-recurring patent amortization charge partially offset by higher personnel and clinical spend ahead of the ELPAS-2 data readout. Net loss was $4.7 million for Q1 2026, a 6% ($0.3 million) decrease YoY. As of March 31, 2026, cash and cash equivalents totaled $15.8 million.

• Based on current operating budgets and cash flow forecasts, existing cash and cash equivalents ($15.8 million as of March 31, 2026) are expected to fund operating expenses and capital requirements into the fourth quarter of 2026
  • Top-line data readout from the ELPAS-2 Phase 2b HLHS trial remains on track for August 2026
  • PDCM trial initiation is expected to occur in 2027, following completion of planning and feasibility work in 2026
  • A pre-BLA meeting for HLHS is currently expected to be scheduled in 2027, after submission and discussion of ELPAS-2 results, to review full BLA readiness including clinical and CMC components

• The FDA does not accept the original primary endpoint of the ELPAS-2 HLHS trial, and the trial is no longer designated as pivotal by the agency while it remains ongoing; approval of LaramastroCell for HLHS is dependent on demonstrating efficacy via new clinical endpoints and reaching agreement with the FDA on a path forward after data readout
  • HLHS is a pioneering indication with limited existing clinical precedent, creating uncertainty around what magnitude of benefit is required to satisfy FDA requirements for approval
  • The company's cash runway only extends into Q4 2026, creating reliance on successful partnership out-licensing or additional capital raising to fund ongoing operations beyond that point
  • Regulatory feedback for the HLHS program could introduce unplanned changes to development timelines and costs for other LaramastroCell indications, even though programs are structurally independent

Q: What are the expected follow-up steps and timeline with the FDA after ELPAS-2 top-line results are released, and what impact has recent FDA feedback on HLHS had on post-approval requirements and the PDCM program? / A: Management will share results with the FDA immediately after release and request a meeting, which will be either a Type C meeting for general alignment or a Type B meeting to discuss BLA plans if results are strongly positive. A pre-BLA meeting to review full clinical and CMC readiness is expected in 2027. A long-term patient extension study to track 10-year transplant-free survival was already proposed to and accepted by the FDA in 2024, so post-approval requirements have not changed as a result of recent feedback. For PDCM, while HLHS results will inform some clinical planning, the programs are independent with different patient populations and administration routes, and the primary trial endpoint for PDCM has already been agreed upon with the FDA, so no major changes to plans are expected.

Q: If a composite endpoint including 12-month transplant-free survival, hospitalization length, and MACE is used for ELPAS-2, what level of benefit is needed to convince the FDA, and what insights came from the recent published CHILD study? / A: As of the current blinded data, two deaths have been recorded in the trial, and the company is powering the trial to show a reduction in average 12-month post-procedure hospitalization from the historical standard of 30 days to 15 days, and already has enough adjudicated MACE events to demonstrate a meaningful difference between treatment and control groups. The concurrently-enrolled CHILD study, which ran at the same centers as ELPAS-2 with the same patient population, informed the company's selection of MACE components and endpoint design, and the higher event rate observed in the CHILD study standard of care group aligns with the assumptions management is using for ELPAS-2.

Q: What endpoints will be most important to payers for coverage if LaramastroCell is approved, and will placebo patients have access to the therapy via compassionate use if ELPAS-2 data is positive? / A: Payer coverage will be most strongly predicted by clinically meaningful outcomes, specifically longer transplant-free survival (which is critical given the limited supply of donor hearts) and reduced hospitalization length, both of which are included as core trial endpoints. Right ventricular ejection fraction will still be collected as a secondary endpoint, and long-term data will be used to explore its future use as a surrogate endpoint. Management currently does not have an active crossover or compassionate use extension program for placebo patients, but making the therapy available to patients is a core company priority, and the topic will be prioritized for discussion if ELPAS-2 data is positive.