Longeveron’s Q4 Earnings Call: PDCM PRV Pursuit and BLA Timeline Shifts Highlight Contradictions

Date of Call: Mar 17, 2026

Financials Results

• Revenue: $1.2M for 2025, down 50% YOY from $2.4M in 2024

Guidance:

• Anticipate results from ELPIS-II (pivotal phase 2b in HLHS) in Q3 2026, with potential BLA preparation if positive.
• Cash runway extended into Q4 2026 following a $15.9M private placement.
• Plan to initiate BLA submission for HLHS in 2027, aiming to leverage rolling submission and priority review eligibility.
• Target BLA submission for PDCM in 2027, following a single pivotal phase 2 registrational study planned for 2026.
• Plan to secure a PRV for HLHS (eligible upon BLA approval) with potential sale to investors.

Business Commentary:

Financial Performance and Capital Resources:

• Longeveron reported a 50% decrease in revenues for 2025, from $2.4 million in 2024 to $1.2 million, primarily due to reduced demand for clinical trial and contract manufacturing services.
• The company increased its net loss by 41%, from $16 million in 2024 to $22.7 million in 2025, driven by higher general and administrative expenses and increased research and development costs.
• Despite the decline in revenue, Longeveron completed a private placement that raised $15.9 million, providing financial runway into the fourth quarter of 2026.

HLHS Program and Regulatory Pathway:

• The ELPIS-II clinical trial for HLHS has completed enrollment, with top-line results anticipated in the third quarter of 2026.
• The trial may be considered pivotal, which could accelerate the regulatory pathway for laromestrocel, potentially leading to the preparation of a Biologics License Application (BLA).
• The company is eligible for a Priority Review Voucher (PRV) upon BLA approval, which could be sold or used for future therapies.

PDCM Program and Development Strategy:

• The IND application for laromestrocel as a treatment for PDCM became effective in July 2025, allowing advancement directly into a single pivotal phase 2 registrational clinical trial.
• The trial is designed to assess the efficacy of laromestrocel in reducing heart transplant and hospitalization rates, with planning to initiate in 2027.
• The program has the potential to receive a PRV, similar to the HLHS program.

Strategic Partnerships and Future Funding:

• Longeveron plans to pursue strategic partnerships for its Alzheimer's disease and age-related frailty programs to accelerate development and commercialization.
• The company's recent private placement included an agreement to pursue a sale of a PRV, with investors entitled to 50% of the proceeds.
• The focus on partnerships is driven by the need to increase capital use efficiency and leverage larger organizations' resources.

Sentiment Analysis:

Overall Tone: Positive

• CEO Stephen Willard stated, 'This is an extraordinarily exciting time for Longeveron.' He highlighted 'strengthening of our balance sheet,' 'support of high quality fundamental investors,' and 'anticipated near-term pivotal clinical data' as key positives. The company is 'on the cusp of pivotal data' and sees 'significant opportunity' in stem cell therapies.

Q&A:

• Question from Raghuram Selvaraju (H.C. Wainwright & Co.): Could you enumerate potential areas of inquiry for laromestrocel eligible for PRVs beyond HLHS?
  Response: PDCM (pediatric dilated cardiomyopathy) is a separate PRV opportunity, with the company planning to seek it.

• Question from Raghuram Selvaraju (H.C. Wainwright & Co.): What non-dilutive capital sources beyond PRVs could fund Alzheimer’s disease and age-related frailty initiatives?
  Response: Strategic licensing partnerships are the priority for funding these programs, leveraging the CEO's background and existing interest from partners.

• Question from Boobalan Pachaiyappan (Roth Capital Partners): If HLHS data is positive in Q3 2026, how soon can you file for BLA, and will it be rolling submission with priority review?
  Response: Eligible for rolling submission and priority review; aim to initiate regulatory process as quickly as possible after data readout, targeting BLA submission in 2027.

• Question from Boobalan Pachaiyappan (Roth Capital Partners): Could the PRV sunset date in September 2029 pose challenges in monetizing at a premium?
  Response: Prices have remained strong around the $200M range recently; expect them to remain strong as the 2029 date approaches.

• Question from Boobalan Pachaiyappan (Roth Capital Partners): What is the next step, timeline, design, and endpoints for the PDCM program, and is the goal to prevent heart transplantation?
  Response: Plan feasibility assessment in 2026, trial initiation in 2027; use a hierarchical composite endpoint including transplant and hospitalization, aiming to reverse disease and prevent transplant.

• Question from Boobalan Pachaiyappan (Roth Capital Partners): What is the strategy for the female sexual dysfunction patent, and is it a partnered or self-developed program?
  Response: Sees high unmet need; views it as a partnership opportunity rather than self-development, given the need for further studies and regulatory pathway establishment.

Contradiction Point 1

Strategy for Pediatric Indication (PDCM)

Shift from evaluating partnerships to explicitly pursuing a PRV.

Raghuram Selvaraju (H.C. Wainwright & Co.) - Raghuram Selvaraju (H.C. Wainwright & Co.)

2025Q4: Beyond HLHS, the Pediatric Dilated Cardiomyopathy (PDCM) program is eligible for a separate PRV, which the company will pursue. - [Stephen Willard](CFO)

Could you elaborate on the commercial perspectives for scaled-up manufacturing and CMC of laromestrocel if approved in HLHS, and what other potential areas for laromestrocel might be eligible for Priority Review Vouchers (PRVs)? - Raghuram Selvaraju (H.C. Wainwright)

20251105-2025 Q3: In rare pediatric diseases, the company seeks partnerships both outside and within the U.S. - [Than Powell](CFO)

Contradiction Point 2

BLA Submission Timeline for HLHS

Inconsistency regarding the feasibility of a 2026 BLA submission.

Boobalan Pachaiyappan (Roth Capital Partners) - Boobalan Pachaiyappan (Roth Capital Partners)

2025Q4: The goal is to initiate the BLA process as quickly as possible thereafter, targeting submission in 2027. - [Joshua Hare](CFO) and [Nataliya Agafonova](CFO)

Assuming positive data in Q3 2026, how soon can you file for a BLA, and will it be on a rolling basis with priority review? - Boobalan Pachaiyappan (ROTH Capital Partners)

20251105-2025 Q3: A rolling submission is possible, but the full submission is likely not achievable by end of 2026. - [Than Powell](CFO)

Contradiction Point 3

Regulatory Pathway Support for PDCM

Contradiction on whether a positive HLHS trial directly supports PDCM approval.

Boobalan Pachaiyappan (Roth Capital Partners) - Boobalan Pachaiyappan (Roth Capital Partners)

2025Q4: The company is **eligible for rolling submission and priority review** due to the rare pediatric disease designation. - [Joshua Hare](Chief Medical Officer) and [Nataliya Agafonova](Chief Medical Officer)

Assuming positive HLHS program data in Q3 2026, how soon can you file a BLA, will it be on a rolling basis, and is priority review expected? - Raghuram Selvaraju (H.C. Wainwright & Co, LLC)

2025Q2: A positive outcome in HLHS would **definitely be supportive** for the PDCM regulatory review process. - [Nataliya Agafonova](Chief Medical Officer)

Contradiction Point 4

Timeline for BLA Submission

Contradiction on the availability of the regulatory pathway (PREB) for rolling BLA submission.

Boobalan Pachaiyappan (Roth Capital Partners) - Boobalan Pachaiyappan (Roth Capital Partners)

2025Q4: The company is **eligible for rolling submission** due to the rare pediatric disease designation. - [Joshua Hare](Chief Medical Officer) and [Nataliya Agafonova](Chief Medical Officer)

Assuming positive data in Q3 2026 for the HLHS program, how soon can you file for a BLA, will it be on a rolling basis, and do you expect a priority review? - Boobalan Pachaiyappan (ROTH Capital Partners, LLC)

2025Q2: The **current PREB program is set to sunset in September 2026** unless reauthorized by Congress. - [Mohamed Wa'el Ahmed Hashad](CEO)

Contradiction Point 5

PRV Program Eligibility and Strategy

Contradiction on the current eligibility status and strategy for obtaining a PRV.

Raghuram Selvaraju (H.C. Wainwright & Co.) - Raghuram Selvaraju (H.C. Wainwright & Co.)

2025Q4: Beyond HLHS, the Pediatric Dilated Cardiomyopathy (PDCM) program is eligible for a separate PRV, which the company will pursue. - [Stephen Willard](CFO)

Could you elaborate on the commercial perspectives for scaled-up manufacturing and CMC of laromestrocel if approved in HLHS, as well as other potential PRV-eligible areas beyond HLHS? - Ram Selvaraju (H.C. Wainwright)

2025Q1: The current PRV program expires in September 2026. Longeveron believes there is strong bipartisan support for reauthorization, and it is a top priority. - [Wa'el Hashad](CEO)