Longeveron Announces Independent Data Monitoring Committee Positive Review and Recommendation to Complete Phase 2b Stem Cell Therapy Clinical Trial in Hypoplastic Left Heart Syndrome (HLHS)
Rhea-AI Summary
Longeveron (NASDAQ: LGVN) reported that an independent Data Monitoring Committee completed its final planned review of the fully enrolled Phase 2b ELPIS II trial of laromestrocel (Lomecel-B) for hypoplastic left heart syndrome (HLHS).
The DMC found no new safety concerns, recommended the study continue as designed, and top-line results are anticipated in August 2026. ELPIS II enrolled 40 pediatric patients at 12 centers and is conducted with NHLBI support. Prior ELPIS I data showed 100% transplant-free survival to age five in treated children. Laromestrocel holds FDA Orphan Drug, Fast Track, and Rare Pediatric Disease designations for HLHS.
Positive
- Independent DMC reported no new safety concerns for ELPIS II HLHS trial
- DMC recommended Phase 2b ELPIS II study continue as designed to completion
- ELPIS II fully enrolled 40 pediatric patients at 12 pediatric cardiac centers
- Top-line ELPIS II results anticipated in August 2026, providing a clear catalyst date
- Prior ELPIS I study reported 100% transplant-free survival to age five in treated children
- Laromestrocel for HLHS holds FDA Orphan Drug, Fast Track, and Rare Pediatric Disease designations
Negative
- None.
News Market Reaction – LGVN
In the May 11 session, LGVN declined 0.86%, reflecting a mild negative market reaction. Argus tracked a peak move of +12.2% during that session. Argus tracked a trough of -7.1% from its starting point during tracking. Our momentum scanner triggered 5 alerts that day, indicating moderate trading interest and price volatility.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Previous Clinical trial Reports
| Date | Event | Sentiment | 24h Move | Catalyst |
|---|---|---|---|---|
| Feb 25 | Phase 2b frailty data | Positive | -5.6% | Published Phase 2b frailty results showing improved 6MWT and biomarker signals. |
| Jun 24 | HLHS enrollment complete | Positive | +7.8% | Completion of pivotal ELPIS II HLHS enrollment with 40 pediatric patients. |
| Mar 11 | Alzheimer’s Phase 2a | Positive | +5.4% | Nature Medicine publication of positive CLEAR MIND Alzheimer’s Phase 2a data. |
| Oct 29 | CTAD24 data poster | Positive | +0.5% | CTAD24 presentation linking MMP14 inhibition to better Alzheimer’s outcomes. |
| Oct 14 | CTAD24 selection | Positive | +8.4% | Selection of Lomecel-B Alzheimer’s data for late-breaking CTAD24 poster. |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Clinical trial news has generally produced positive price reactions for LGVN, with only one notable divergence where strong Phase 2b frailty data coincided with a negative move.
Recent history shows Longeveron repeatedly highlighting laromestrocel across aging and pediatric indications. Publications in Cell Stem Cell and Nature Medicine reported positive Phase 2a/2b outcomes in frailty and Alzheimer’s disease, often followed by gains of 5–8%. A June 2025 update on completing enrollment for the pivotal HLHS Phase 2b trial also saw a strong positive reaction. Today’s DMC review and continuation recommendation for ELPIS II fits this pattern of incremental clinical validation ahead of the planned August 2026 topline readout.
Key Terms
hypoplastic left heart syndrome medical
data monitoring committee medical
orphan drug designation regulatory
fast track designation regulatory
rare pediatric disease designation regulatory
adjunct therapy medical
transplant-free survival medical
pivotal phase 2b medical
AI-generated analysis. How Rhea-AI works. Not financial advice.
- Phase 2b clinical trial (ELPIS II) evaluating laromestrocel as a potential adjunct treatment for HLHS, a rare pediatric disease and orphan-designated indication
- DMC performed a risk-benefit assessment, identified no new safety concerns, and recommended continuation of the study as designed through completion
- This review represents the final planned independent safety assessment prior to completion of the trial
- ELPIS II top-line trial results anticipated in August 2026
MIAMI, May 11, 2026 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing cellular therapy for life-threatening, rare pediatric and chronic aging-related conditions, today announced that the independent Data Monitoring Committee (DMC) has completed its final prespecified data review for the ongoing, fully enrolled, Phase 2b clinical trial (ELPIS II) evaluating laromestrocel (Lomecel-B®) as a potential adjunct therapy for hypoplastic left heart syndrome (HLHS). The DMC based its review on available data for all participating patients enrolled. The DMC performed a risk-benefit assessment, indicated no safety concerns, and approved the study to continue as designed to completion.
“We are grateful to the members of the DMC for their effort and diligence in this review,” said Nataliya Agafonova, M.D., Chief Medical Officer at Longeveron. “Based on the scientific rationale supporting laromestrocel and the clinical data generated to date, we remain encouraged by the potential of our investigational therapy in HLHS. With our Phase 2b clinical trial for HLHS having achieved full enrollment last June, we continue to anticipate top-line trial results in August 2026 and look forward to sharing the data.”
ELPIS II enrolled 40 pediatric patients at 12 leading pediatric cardiac centers across the country. The clinical trial is being conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) through grants from the National Institutes of Health (NIH). ELPIS II builds on findings from ELPIS I study, in which treated children demonstrated
The U.S. Food and Drug Administration (FDA) has granted laromestrocel Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation, for development for HLHS.
About Hypoplastic Left Heart Syndrome (HLHS)
HLHS is a rare congenital heart defect that effects approximately 1,000 infants per year in the U.S. Infants with HLHS are born with an underdeveloped left ventricle, which creates a life-threatening condition due to the heart’s inability to pump adequate amounts of blood throughout the body. The current treatment requires infants to undergo a complex three-stage heart reconstruction surgery process over the first five years of their life. Despite staged surgical palliation, long-term mortality and morbidity remain substantial, with progressive right ventricular dysfunction representing a major contributor to adverse outcomes. There is clearly an important unmet medical need to improve right ventricular function in these infants to positively impact both short- and long-term patient outcomes.
About laromestrocel (Lomecel-B®)
Laromestrocel is a living cell product made from specialized cells isolated from the bone marrow of young healthy adult donors. These specialized cells, known as mesenchymal stem cells (MSCs), are essential to our endogenous biological repair mechanism. MSCs have been shown to perform a number of complex functions in the body and support tissue repair and regenerative signaling pathways. They also have been shown to respond to sites of injury or disease and secrete bioactive factors that are immunomodulatory and regenerative. We believe that laromestrocel MSCs may have multiple potential mechanisms of action that may lead to anti-inflammatory, pro-vascular regenerative responses, and therefore may have broad application for a range of rare and aging related diseases.
About Longeveron Inc.
Longeveron is a clinical stage biotechnology company developing regenerative medicines to address unmet medical needs. The Company’s lead investigational product is laromestrocel (Lomecel-B™), an allogeneic mesenchymal stem cell (MSC) therapy product isolated from the bone marrow of young, healthy adult donors. Laromestrocel has multiple potential mechanisms of action encompassing pro-vascular, pro-regenerative, anti-inflammatory, and tissue repair and healing effects with broad potential applications across a spectrum of disease areas. Longeveron is pursuing four pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer’s disease (AD), Pediatric Dilated Cardiomyopathy (DCM) and Aging-related Frailty. Laromestrocel development programs have received five distinct and important FDA designations: for the HLHS program - Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation; and, for the AD program - Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation. For more information, visit www.longeveron.com or follow Longeveron on LinkedIn, X, and Instagram.
Forward-Looking Statements
Certain statements in this press release that are not historical facts are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, which reflect management’s current expectations, assumptions, and estimates of future operations, performance and economic conditions, and involve known and unknown risks, uncertainties, and other important factors that could cause actual results, performance, or achievements to differ materially from those anticipated, expressed, or implied by the statements made herein. Forward-looking statements are generally identifiable by the use of forward-looking terminology such as “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expects,” “intend,” “looks to,” “may,” “on condition,” “plan,” “potential,” “predict,” “preliminary,” “project,” “see,” “should,” “target,” “will,” “would,” or the negative thereof or comparable terminology, or by discussion of strategy or goals or other future events, circumstances, or effects. Factors that could cause actual results to differ materially from those expressed or implied in any forward-looking statements in this release include, but are not limited to, our cash position and need to raise additional capital, the difficulties we may face in obtaining access to capital, and the dilutive impact it may have on our investors; our financial performance, and ability to continue as a going concern; the period over which we estimate our existing cash and cash equivalents will be sufficient to fund our future operating expenses and capital expenditure requirements; the ability of our clinical trials to demonstrate safety and efficacy of our product candidates, and other positive results; the timing and focus of our ongoing and future preclinical studies and clinical trials, and the reporting of data from those studies and trials; the size of the market opportunity for certain of our product candidates, including our estimates of the number of patients who suffer from the diseases we are targeting; our ability to scale production and commercialize the product candidate for certain indications; the success of competing therapies that are or may become available; the beneficial characteristics, safety, efficacy and therapeutic effects of our product candidates; our ability to obtain and maintain regulatory approval of our product candidates in the U.S. and other jurisdictions; our plans relating to the further development of our product candidates, including additional disease states or indications we may pursue; our plans and ability to obtain or protect intellectual property rights, including extensions of existing patent terms where available and our ability to avoid infringing the intellectual property rights of others; the need to hire additional personnel and our ability to attract and retain such personnel; and our estimates regarding expenses, future revenue, capital requirements and needs for additional financing.
Further information relating to factors that may impact the Company’s results and forward-looking statements are disclosed in the Company’s filings with the Securities and Exchange Commission, including Longeveron’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the Securities and Exchange Commission on February 28, 2025, its Quarterly Reports on Form 10-Q, and its Current Reports on Form 8-K. The Company operates in highly competitive and rapidly changing environment; therefore, new factors may arise, and it is not possible for the Company’s management to predict all such factors that may arise nor assess the impact of such factors or the extent to which any individual factor or combination thereof, may cause results to differ materially from those contained in any forward-looking statements. The forward-looking statements contained in this press release are made as of the date of this press release based on information available as of the date of this press release, are inherently uncertain, and the Company disclaims any intention or obligation, other than imposed by law, to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.
Investor and Media Contact:
Derek Cole
Investor Relations Advisory Solutions
derek.cole@iradvisory.com
A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/e9e96d9c-d6c3-4aec-ba85-36631b520211
