Longeveron (NASDAQ:LGVN) said it is narrowing its near-term focus on the upcoming data readout from its ELPIS II trial in hypoplastic left heart syndrome while pursuing a more partnership-oriented strategy for its stem cell therapy programs.
On the company’s first-quarter 2026 results and business update call, Chief Executive Officer Stephen Willard said Longeveron has taken “decisive steps to reposition the company for long-term value creation” since he became CEO in February. Those steps include a review of the company’s assets and strategy, a focus on raising new capital, and a move toward what he described as a more “capital-efficient, asset-light operating model.”
The company plans to attend the BIO International Convention in San Diego from June 22 through June 25, 2026, where Willard said it will meet with global pharmaceutical executives to discuss potential partnerships and strategic opportunities.
ELPIS II Data Expected in August
Chief Medical Officer Nataliya Agafonova said Longeveron’s main clinical priority is ELPIS II, a Phase 2 trial evaluating laromestrocel in infants with hypoplastic left heart syndrome, or HLHS. Enrollment of 40 patients was completed in June 2025, and top-line results are expected in August 2026.
Agafonova said the company recently completed a Type C meeting with the U.S. Food and Drug Administration regarding the HLHS program. According to Agafonova, the FDA acknowledged that HLHS is a rare disease associated with significant morbidity and mortality and a high unmet medical need. However, she said the agency stated that right ventricle ejection fraction, the primary endpoint in ELPIS II, is not an appropriate endpoint to demonstrate efficacy.
Agafonova said Longeveron agreed that right ventricle ejection fraction alone was insufficient as a primary efficacy endpoint and was prepared to discuss other potential endpoints. But she said the FDA indicated that because an interim analysis mandated and conducted by the National Institutes of Health had occurred while the company remained blinded, a new primary endpoint could not be agreed to while the study is ongoing.
As a result, Agafonova said the FDA no longer refers to ELPIS II as a pivotal trial, which she said had been discussed at a 2024 Type C meeting. Still, she said the FDA agreed to meet with Longeveron again after the trial is completed to review the results and discuss a potential path forward.
Agafonova said the FDA indicated that objective measures such as all-cause mortality, cardiac transplant-free survival, cardiac transplantation events and well-defined major adverse cardiac events could be informative of efficacy in ELPIS II. She said Longeveron is capturing those measures and intends to submit a sponsor statistical analysis plan to the FDA for review and approval.
The company remains optimistic that ELPIS II results and other evidence could support a Biologics License Application following the August readout, Agafonova said.
Analysts Press Management on Regulatory Path
During the question-and-answer session, H.C. Wainwright analyst Raghuram Selvaraju asked about the likely next steps with the FDA after the ELPIS II readout. Willard said the company would move “immediately” to provide top-line results to the agency and seek a meeting. Agafonova said the type of meeting would depend on the results, adding that if results are “overwhelmingly positive,” the company would likely seek a Type B meeting to discuss the possibility of a future BLA filing. She said a pre-BLA meeting would likely occur in 2027, after the company is prepared to discuss clinical results as well as chemistry, manufacturing and controls matters.
Agafonova also discussed possible post-marketing requirements if laromestrocel were approved in HLHS. She said the company has proposed a long-term extension study to collect data from patients in ELPIS I and ELPIS II, including long-term transplant-free survival. She said the FDA accepted the concept and that Longeveron is working on operational implementation.
In response to questions from Roth Capital Partners, Agafonova said Longeveron is evaluating a composite endpoint that could include hospitalization days and major adverse cardiac events. She said the company has seen two deaths in the blinded trial data so far and that hospitalization days may carry significant weight in the composite endpoint. Based on literature, she said HLHS patients may spend about 30 days in the hospital in the 12 months after the Glenn procedure, and Longeveron is currently powering for a reduction to 15 days.
Joshua Hare, Longeveron’s co-founder, chief science officer and executive chairman, said the recently published CHILD Study informed the company’s thinking around ELPIS II endpoints. He said the CHILD Study included patients enrolled at the same centers and around the same period as ELPIS II and showed events that align with those Longeveron is tracking in ELPIS II.
Pediatric Dilated Cardiomyopathy Program Advances
Agafonova said Longeveron is also preparing for development of laromestrocel in pediatric dilated cardiomyopathy, a rare pediatric cardiovascular disease in which heart chamber muscles become enlarged or stretched. She said nearly 40% of children with the condition require a heart transplant or die within two years of diagnosis.
The company’s investigational new drug application for laromestrocel in pediatric dilated cardiomyopathy became effective in July 2025, Agafonova said. She said the IND allows the company to advance directly into a single Phase 2 registrational trial. Longeveron expects planning and preparation for the study in 2026, with potential initiation in 2027.
Asked whether the FDA feedback on ELPIS II would affect the pediatric dilated cardiomyopathy program, Agafonova said the diseases are distinct, with different patient populations and routes of administration, though learnings from HLHS may inform future clinical messaging. Willard said the company would be careful to ensure the FDA is “completely comfortable” with endpoints in that program. Hare added that the pediatric dilated cardiomyopathy trial already has a clinical endpoint agreed upon with the agency.
First-Quarter Financial Results
Chief Financial Officer Lisa Locklear said first-quarter 2026 revenue was $0.4 million, consisting of $0.4 million in clinical trial revenue and $20,000 in contract manufacturing revenue. Revenue in the same period of 2025 was also $0.4 million, made up of $0.3 million in clinical trial revenue and $0.1 million in contract manufacturing revenue.
- Clinical trial revenue rose $0.1 million, or 46%, year over year, driven by greater participant demand for the Bahamas Registry Trial.
- Contract manufacturing revenue fell $0.1 million, or 84%, due to lower demand from third-party clients.
- General and administrative expenses decreased to $2.7 million from $2.9 million.
- Research and development expenses fell to $2.3 million from $2.5 million.
- Net loss was $4.7 million, compared with $5.0 million in the prior-year quarter.
Locklear said Longeveron had $15.8 million in cash and cash equivalents as of March 31, 2026. Based on its current operating budget and cash flow forecast, the company expects its cash to fund operating expenses and capital expenditures into the fourth quarter of 2026.
Hare said laromestrocel is supported by 52 issued patents and more than 60 pending patents worldwide. He said the therapy has received five FDA expedited designations, including Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations.
Willard closed the call by emphasizing the August HLHS data as a key milestone, while noting that the company has “four shots on goal” across HLHS, pediatric dilated cardiomyopathy, Alzheimer’s disease and aging-related frailty.
About Longeveron (NASDAQ:LGVN)
Longeveron Inc is a clinical-stage biotechnology company focused on the development and commercialization of allogeneic cellular therapies designed to address aging-related and inflammatory conditions. The company’s primary therapeutic candidate, Lomecel-B, is an off-the-shelf mesenchymal stem cell product derived from bone marrow. Through its proprietary manufacturing process, Longeveron aims to produce a consistent, scalable cell therapy platform with potential applications in multiple disease areas.
Longeveron’s pipeline encompasses several ongoing and completed clinical studies.
