Longeveron Announces Constructive Type C Meeting with U.S. FDA Ahead of Data Readout for ELPIS II Phase 2b Clinical Trial Evaluating Treatment for Hypoplastic Left Heart Syndrome (HLHS)
Rhea-AI Summary
Longeveron (NASDAQ: LGVN) held a Type C meeting with the U.S. FDA; the Agency said right ventricle ejection fraction (RVEF) is not an appropriate primary endpoint for the ELPIS II Phase 2b trial for hypoplastic left heart syndrome. Top-line results are anticipated in August 2026. The FDA will not agree a new primary endpoint while the trial remains ongoing because of an interim NIH-mandated analysis, and therefore no longer describes ELPIS II as pivotal. The FDA said objective measures (all-cause mortality, transplant-free survival, cardiac transplantation, well-defined MACE) could be informative. Longeveron will submit a Sponsor Statistical Analysis Plan proposing a composite primary endpoint and remains optimistic about a possible BLA path after readout.
Positive
- Top-line ELPIS II results expected in August 2026
- Trial is capturing objective endpoints: mortality, transplant-free survival, MACE
- FDA agreed to meet again after study completion to discuss results
Negative
- FDA concluded RVEF is not an appropriate primary efficacy endpoint
- FDA will not agree to a new primary endpoint while trial is ongoing
- FDA no longer refers to ELPIS II as pivotal
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 +17.1% during that session. Argus tracked a trough of -15.0% from its starting point during tracking. Our momentum scanner triggered 7 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 | Frailty Phase 2b data | Positive | -5.6% | Published Phase 2b results showing improved 6MWT and biomarker signals in frailty. |
| Jun 24 | HLHS Phase 2b enrollment | Positive | +7.8% | Completion of enrollment in pivotal ELPIS II HLHS trial and multiple FDA designations. |
| Mar 11 | Alzheimer’s Phase 2a data | Positive | +5.4% | Nature Medicine publication of positive CLEAR MIND Phase 2a Alzheimer’s results. |
| Oct 29 | Alzheimer’s data presentation | Positive | +0.5% | CTAD24 presentation linking MMP14 inhibition with improved Alzheimer’s outcomes. |
| Oct 14 | Alzheimer’s CTAD selection | Positive | +8.4% | Lomecel-B Alzheimer’s data selected for late-breaking CTAD24 poster presentation. |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
For clinical trial news, LGVN has mostly traded higher on positive updates, with one notable selloff despite favorable Phase 2b frailty results.
Over the past several clinical updates, Longeveron has repeatedly highlighted progress for laromestrocel across frailty, Alzheimer’s disease, and HLHS. Positive Phase 2a and 2b data in frailty and Alzheimer’s were published in high-profile journals and presented at CTAD, while the pivotal ELPIS II HLHS trial completed enrollment of 40 pediatric patients across 12 institutions. Today’s FDA Type C feedback sits within this ongoing effort to translate these clinical programs into a potential Biologics License Application for HLHS.
Key Terms
u.s. food and drug administration (fda) regulatory
phase 2b medical
hypoplastic left heart syndrome (hlhs) medical
right ventricle ejection fraction (rvef) medical
major adverse cardiac events (mace) medical
biologics license application (bla) regulatory
statistical analysis plan (sap) technical
AI-generated analysis. How Rhea-AI works. Not financial advice.
MIAMI, May 08, 2026 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN), a clinical stage regenerative medicine biotechnology company developing cellular therapies for life-threatening and chronic aging-related conditions, today announced that a constructive Type C meeting with the U.S. Food and Drug Administration (FDA or Agency) was held in late March, with the FDA providing their meeting summary in late April, to discuss the ongoing development of laromestrocel (LOMECEL-B®), a proprietary, scalable, allogeneic, investigational cellular therapy currently being evaluated in a Phase 2b clinical trial (ELPIS II) for hypoplastic left heart syndrome (HLHS). Top-line results from the randomized, controlled Phase 2b ELPIS II clinical trial are anticipated in August 2026.
In the Type C meeting, the FDA acknowledged that HLHS is a rare disease associated with significant morbidity and mortality with a high unmet medical need for safe and effective therapies, but also asserted that the primary endpoint of right ventricle ejection fraction (RVEF) in the ELPIS II trial is not an appropriate endpoint to demonstrate efficacy. While Longeveron agreed with the FDA regarding the insufficiency of RVEF as the primary endpoint, and was prepared to discuss other potentially appropriate endpoints sufficient to demonstrate efficacy, the FDA indicated that given the interim analysis mandated and conducted by the National Institute of Health (NIH) during the trial (to which the Company was and remains blinded), a new primary endpoint could not be agreed to while the trial is still ongoing. Without an agreed upon primary endpoint sufficient for efficacy, the FDA no longer refers to the ELPIS II trial as pivotal, as had been specifically discussed in the Company’s Type C meeting in 2024. Nevertheless, the FDA expressly agreed that it is willing to meet with Longeveron again when the ongoing ELPIS II study is completed to discuss the study results and align on a potential path forward. The FDA further indicated that only the most objective measures, including, all-cause mortality, cardiac transplant-free survival, event of cardiac transplantation, and well-defined major adverse cardiac events (MACE), could be informative of efficacy in ELPIS II, and in that regard, the Company is capturing all of these measures in ELPIS II along with some additional key measures to support an efficacy determination. The Company intends to submit to the FDA a Sponsor Statistical Analysis Plan (SAP) for ELPIS II with a composite primary endpoint and secondary endpoints for the FDA’s review and approval, and remains optimistic that the trial results and other available evidence will be sufficient to support filing a Biologics License Application (BLA) following the readout of top-line results of the ELPIS II data.
About ELPIS II
ELPIS II is a multicenter, randomized, controlled Phase 2b clinical trial evaluating laromestrocel as a potential adjunct therapy in infants with HLHS undergoing Stage II surgical palliation. The clinical trial enrolled 40 pediatric patients at twelve premiere infant and children’s treatment institutions across the country. ELPIS II is being conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) through grants from the NIH.
The laromestorcel HLHS program has received three FDA designations: Orphan Drug designation, Fast Track designation and Rare Pediatric Disease designation. Under the Rare Pediatric Disease designation, if Longeveron were to receive FDA marketing approval for laromestrocel for HLHS, the Company could be eligible to receive a Priority Review Voucher.
About Hypoplastic Left Heart Syndrome (HLHS)
HLHS is a congenital birth defect in which the left ventricle (one of the pumping chambers of the heart) is either severely underdeveloped or missing. As a consequence, infants born with this condition have severely diminished systemic blood flow, which requires them to undergo a complex, three stage heart reconstruction surgery process over the course of the first 5 years of their lives. Although surgical advances have enabled survival into adulthood for some patients, early mortality remains substantial in this population, due to right ventricular failure. As such, there remains an important unmet medical need to improve right ventricular function in these patients in order to support both short-term and long-term outcomes.
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, 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, the ability of our clinical trials, including our ELPIS II trial for HLHS, to demonstrate safety and efficacy of our product candidates, and other positive results; the willingness of the FDA to deem the ELPIS II trial as pivotal following-completion of the ELPIS II trial or to otherwise reach alignment with the Company on a potential path toward regulatory approval; receipt of FDA approval, following review, of the Company’s Sponsor Statistical Analysis Plan (SAP) for ELPIS II; receipt of trial results and other available evidence sufficient to support the Company filing a BLA following the readout of top-line results of the ELPIS II data; the timing and focus of our ongoing and future preclinical studies and clinical trials, and the reporting of data from those studies and trials; 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 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, 2025, filed with the Securities and Exchange Commission on March 17, 2026, 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
