Longeveron Announces Phase 2b ELPIS II Trial Of Laromestrocel In Hypoplastic Left Heart Syndrome Misses Primary Efficacy Endpoint; Initiates Cost-Optimization Measures To Maximize Shareholder Value

The top line results showed that the ELPIS II trial did not meet its primary endpoint of improvement in right ventricular ejection fraction (RVEF) at month 12, but there were exploratory clinical endpoints that the Company is continuing to analyze and plans to discuss with the FDA to determine a potential path forward in HLHSLaromestrocel has maintained what Longeveron believes is a favorable safety and tolerability profile consistent with prior clinical trials, now with 644 patients treated across Longeveron's multiple clinical programs, and has shown potential efficacy in clinical studies across multiple indications and has received 5 designations from FDALaromestrocel has shown positive initial results in Aging-related Frailty, including publication of clinical trial results in Cell Stem Cell and selection as a Finalist awardee in the XPRIZE Healthspan global competitionThe Company has initiated a process of optimizing cost containment and will explore all options with the goal of maximizing shareholder value MIAMI, Sept. 16, 2026 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ:LGVN), a clinical-stage biotechnology company developing cellular therapies for life-threatening, rare pediatric and chronic aging-related conditions, today announced topline results from ELPIS II, its Phase 2b clinical trial evaluating its investigational stem cell therapy laromestrocel as an adjunct to Stage 2 palliative surgery in 40 infants with hypoplastic left heart syndrome (HLHS). The ELPIS II trial did not meet the primary efficacy endpoint of change from baseline in right ventricular ejection fraction (RVEF) at Month 12. In the intent-to-treat (ITT) population, the least-squares mean difference between treatment groups was −0.7 percentage points (95% CI: −7.3 to 5.9; p=0.8336).Initial exploratory clinical outcomes in the as-treated analyses include:Over a 12-month period, there were no deaths in patients that received laromestrocel, compared to one patient in the control group, in the as-treated groupOver a long-term follow up of transplant-free survival (up to five years across all patients), the laromestrocel arm had one event (out of 17 patients) vs. two events (out of 21 patients) in the standard-of-care armHospitalization burden was similar between armsAdjudicated Major Adverse Cardiovascular Events (MACE) were approximately 31% fewer in the laromestrocel arm (12 events in the treated arm vs. 19 events in the untreated arm, respectively). However, the negative binomial analysis was not statistically significant. A sponsor-defined exploratory hierarchical composite endpoint consisting of all-cause mortality and duration of inpatient hospitalization was not statistically significant in the ITT population.Laromestrocel demonstrated a safety profile generally consistent with prior clinical trials, and no new safety signals were identified in the study.The Company is conducting additional analyses of the complete dataset and intends to discuss the results with the U.S. Food and Drug Administration (FDA) to determine potential next steps for the HLHS development program. FDA previously indicated its willingness to meet with the Company following completion of the study to discuss the results and potential paths forward.