Longeveron (NASDAQ:LGVN) said it expects to report top-line data in mid-September from its Phase IIb ELPIS II trial of laromestrocel in hypoplastic left heart syndrome, or HLHS, as the cell-therapy developer focuses its resources on what it described as its most important near-term catalyst.
HLHS is a rare congenital heart defect in which the left ventricle is severely underdeveloped or absent. Longeveron is evaluating laromestrocel as an adjunct treatment in the disease, while also developing the therapy for Alzheimer’s disease, pediatric dilated cardiomyopathy and age-related frailty.
ELPIS II Readout and Regulatory Discussions
Chief Medical Officer Dr. Nataliya Agafonova said the company had discussed endpoint strategy with the FDA and incorporated agency feedback into its statistical analysis plan. The company submitted the plan to the FDA for review and was awaiting further feedback, she said.
Agafonova said Longeveron expects to proceed with its planned database lock and pre-specified analysis if it does not receive additional FDA comments before the lock. The company expects database lock on Aug. 31 and availability of top-line results in September.
The timing shifted from an earlier August expectation because of delays involving month-12 MRI assessments for the last patients treated, according to Agafonova. She said the delay was not related to unresolved statistical analysis plan issues.
Longeveron said it has captured objective outcomes including all-cause mortality, cardiac transplant-free survival, cardiac transplantation events and defined major adverse cardiac events. Agafonova said the FDA had indicated these clinically significant measures would be considered exploratory, but that the agency had requested the company share trial results for potential approval considerations.
In response to analyst questions, Agafonova said that if right ventricular ejection fraction does not reach statistical significance but other sponsor-defined measures are positive, the company would pursue available options to seek a biologics license application, or BLA, path. She cited the FDA’s stated willingness to exercise regulatory flexibility in the rare pediatric disease.
The company also plans a long-term extension study intended to follow HLHS patients through age 10. Agafonova said Longeveron has discussed the proposed study with the FDA and is addressing agency questions while undertaking feasibility work. Some ELPIS II participants began the study about five years ago, she said, and the company expects to have up to five years of survival data for certain patients at the upcoming readout.
Manufacturing and Other Pipeline Plans
Willard said Longeveron has made progress in chemistry, manufacturing and controls work and is working with a provider to transfer manufacturing. He said the company did not see manufacturing as an impediment to pursuing a BLA if the clinical data and FDA feedback support that path.
The company’s investigational new drug application for laromestrocel in pediatric dilated cardiomyopathy became effective in July 2025. Agafonova said Longeveron continues planning for a potential 2027 start of a Phase II registrational trial in that indication. She noted that nearly 40% of children with pediatric dilated cardiomyopathy require a heart transplant or die within two years of diagnosis.
Co-founder, Chief Science Officer and Executive Chairman Dr. Joshua Hare said laromestrocel has potential anti-inflammatory, pro-vascular and pro-regenerative mechanisms. He added that the company holds 52 issued patents and has more than 60 patent applications pending worldwide, along with five FDA expedited designations including RMAT, Fast Track, Orphan Drug and Rare Pediatric Disease designations.
XPRIZE Healthspan Recognition
Longeveron also discussed its selection as a finalist in the XPRIZE Healthspan competition based on published clinical trial results showing that laromestrocel increased six-minute walk distance in patients with age-related frailty.
Willard said the company believes it is the only publicly traded finalist in the competition. The seven-year, $101 million competition selected finalists from more than 600 applicants in 58 countries. Milestone II awardees receive $1 million to advance their therapeutic approaches, while the grand prize could award up to $81 million.
Willard said Longeveron intends to seek a partnership in longevity before the final XPRIZE outcome. The competition’s final phase includes coordinated clinical trials through 2029.
Second-Quarter Financial Results
For the quarter ended June 30, 2026, Longeveron reported revenue of $0.3 million, down $29,000, or 10%, from the prior-year period. Chief Financial Officer Marie Washburn attributed the decline primarily to the absence of contract manufacturing revenue.
- General and administrative expenses rose 23% to $3.2 million, driven primarily by a $0.4 million increase in legal spending and a $0.2 million increase in personnel costs.
- Research and development expenses increased 7% to $3.2 million, reflecting higher clinical trial expenses associated with the approaching ELPIS II data release.
- Net loss widened to $6.1 million from $5 million in the second quarter of 2025.
- Cash and cash equivalents totaled $10.1 million as of June 30, 2026.
Washburn said Longeveron expects its existing cash and cash equivalents to fund operating expenses and capital expenditures into the fourth quarter of 2026, based on its current operating budget.
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.
