Longeveron® Presents Lomecel-B™ Long-term Transplant-free Survival Data in Hypoplastic Left Heart Syndrome (HLHS) at the Congenital Heart Surgeons’ Society (CHSS) 51st Annual Meeting

Rhea-AI Summary

Longeveron presented long-term survival data for Lomecel-B in Hypoplastic Left Heart Syndrome (HLHS) patients at the CHSS Annual Meeting. The ELPIS I follow-on study showed 100% 5-year survival and no heart transplants needed after Glenn surgery, compared to 83% survival and 5.2% transplant rate in the Single Ventricle Reconstruction Trial. ELPIS I met its primary safety endpoint with 100% survival and transplant-free rate one year post-treatment. The ongoing ELPIS II Phase 2b trial is evaluating Lomecel-B as an adjunct therapy for HLHS versus standard care at twelve institutions.

Positive

• 100% 5-year survival rate post-Glenn procedure with Lomecel-B vs 83% historical data
• 0% heart transplantation rate vs 5.2% historical rate
• Met primary safety endpoint with 100% survival rate
• No Major Adverse Cardiovascular Events reported
• FDA granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations

Negative

• None.

Insights

Medical Research Analyst

The long-term data from ELPIS I trial demonstrates remarkable clinical outcomes for Lomecel-B in HLHS patients. The 100% survival rate and zero transplant requirements over 5 years post-Glenn procedure significantly outperforms historical data showing 83% survival and 5.2% transplant rates. For a devastating condition where only 50-60% of infants survive to adolescence, these results are particularly meaningful.

The clean safety profile, with no MACE events or treatment-related issues, coupled with the FDA's triple designation status (Orphan Drug, Fast Track and Rare Pediatric Disease), positions Lomecel-B favorably for the ongoing Phase 2b ELPIS II trial. The collaboration with NHLBI and involvement of twelve premier pediatric institutions adds credibility to the development program. These results strongly support Lomecel-B's potential as a groundbreaking regenerative therapy for HLHS.

• Oral presentation highlights findings that patients treated with Lomecel-B^TM in ELPIS I, in an open-label follow-on study, experienced 5-year survival after stage 2 Glenn surgery of 100% with none requiring heart transplant; this compared to the largest data set from the Single Ventricle Reconstruction Trial which showed 83% survival, with a 5.2% heart transplantation rate
• ELPIS I met its primary endpoint of safety through 1-year post-treatment, with 100% survival rate, 100% transplant-free and patients maintained expected rate of growth one year after treatment
• Findings support cell based therapy as a potential adjunct to HLHS reconstruction surgery to improve clinical benefits and reduce the need for subsequent heart transplantation
• ELPIS II Phase 2b clinical trial is currently ongoing, further evaluating Lomecel-B^TM as a potential adjunct therapy for HLHS versus standard of care alone

MIAMI, Oct. 27, 2024 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing regenerative medicines, today announced that its positive long-term transplant-free survival data from a multi-year follow-on study to the ELPIS I Phase 1 clinical trial were featured in an oral presentation at the Congenital Heart Surgeons’ Society (CHSS) 51^st Annual Meeting taking place October 27-28, 2024 in Chicago, Illinois.

“HLHS is a devastating disease for patients and their families, so I am impressed and very encouraged to see the continued long-term survival of patients treated with Lomecel-B^TM in the ELPIS I study,” said Sunjay Kaushal, M.D., Ph.D., Professor of Surgery, Cardiovascular and Thoracic Surgery at University of Nevada, Las Vegas. “Even with current comprehensive treatment, only 50% to 60% of infants survive to adolescence due to right ventricular failure and for those that are surviving, parents are strongly seeking to avoid transplant. 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, and stem cell therapy, particularly Lomecel-B^TM, has the potential to play a critical role in treating these patients.”

ELPIS I was an open-label, Phase 1 clinical trial designed to evaluate the safety of Lomecel-B^TM in patients with HLHS. Patients underwent the Glenn Procedure (an open-heart surgery) at approximately 4-5 months of age. The results from the ELPIS I trial, which were previously reported, and published in the European Heart Journal Open in 2023, showed that the study met its primary safety endpoint and that all patients were alive, transplant-free, and maintained their expected rate of growth one year after treatment.

To assess long-term survival benefits, all ELPIS I patients (n=10, 7 males, and 3 females) were followed for five years post-Glenn surgery and treatment with Lomecel-B^TM to evaluate overall survival and transplant-free survival. Outcomes were compared with long-term historical data from patients in the Single Ventricle Reconstruction (SVR) Trial receiving the same shunt type at Stage I (Norwood) operation and underwent Glenn procedures. Key findings include:

• 5-year post-Glenn procedure Kaplan-Meier survival was 100% in patients treated with Lomecel-B™ in ELPIS I, with none requiring heart transplant. This compared to 83% (95% CI= [77.4, 89.3]) transplant-free survival in the SVR trial through 5 years post-Glenn surgery, and a 5.2% (95% CI= [2.0, 8.3]) heart transplantation rate.
• No Major Adverse Cardiovascular Events (MACE) were reported during the study.
• No Lomecel-B^TM related safety issues were reported.
• These findings support the use of Lomecel-B^TM as a potential adjunct to HLHS reconstruction surgery to improve transplant-free survival.

ELPIS II is a Phase 2b randomized clinical trial further evaluating Lomecel-B^TM, compared to standard of care, as a potential adjunct therapy for HLHS. ELPIS II is on-going and enrolling 38 pediatric patients at twelve premiere infant and children’s treatment institutions 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 the positive clinical results of ELPIS I.

The U.S. Food and Drug Administration (FDA) has granted Lomecel-B™ Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation, which allow greater access to the FDA during Lomecel-B^TM’s development for HLHS.

“We believe these results provide further validation of both the safety and therapeutic potential of Lomecel-B™ in the treatment of HLHS and suggest, with further clinical evaluation, that this cellular therapy has the potential to have a positive impact on patients afflicted with this devastating disease,” said Joshua Hare, M.D., Co-founder, Chief Science Officer, and Chairman at Longeveron. “Based on the strength of the ELPIS I data, we are currently conducting the ELPIS II Phase 2b clinical trial evaluating our cellular therapy Lomecel-B^TM as an adjunct therapy for treating HLHS, which, if positive, may serve as the foundation for a BLA submission for potential approval of Lomecel-B^TM.”

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. Even with this comprehensive treatment, only 50% to 60% of infants survive to adolescence due to right ventricular failure. 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 Lomecel-B™
Lomecel-B™ is a living cell product made from specialized cells isolated from the bone marrow of young healthy adult donors. These specialized cells, known as medicinal signaling cells (MSCs), are essential to our endogenous biological repair mechanism. MSCs have been shown to perform a number of complex functions in the body, including the formation of new tissue. 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 Lomecel-B™ 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 Lomecel-B™, an allogeneic medicinal signaling cell (MSC) therapy product isolated from the bone marrow of young, healthy adult donors. Lomecel-B™ 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 currently pursuing three pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer’s disease (AD), and Aging-related Frailty. Lomecel-B^TM development programs have received five distinct and important U.S. 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 risks and uncertainties that could cause actual results to differ materially from those anticipated by the statements made herein. Forward-looking statements are generally identifiable by the use of forward-looking terminology such as “believe,” “expects,” “may,” “looks to,” “will,” “should,” “plan,” “intend,” “on condition,” “target,” “see,” “potential,” “estimates,” “preliminary,” or “anticipates” or the negative thereof or comparable terminology, or by discussion of strategy or goals or other future events, circumstances, or effects and include, but are not limited to, the anticipated use of proceeds from recent offerings. 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, market and other conditions, our limited operating history and lack of products approved for commercial sale; adverse global conditions, including macroeconomic uncertainty; inability to raise additional capital necessary to continue as a going concern; our history of losses and inability to achieve profitability going forward; the absence of FDA-approved allogenic, cell-based therapies for Aging-related Frailty, Alzheimer’s disease, or other aging-related conditions, or for HLHS or other cardiac-related indications; ethical and other concerns surrounding the use of stem cell therapy or human tissue; our exposure to product liability claims arising from the use of our product candidates or future products in individuals, for which we may not be able to obtain adequate product liability insurance; the adequacy of our trade secret and patent position to protect our product candidates and their uses: others could compete against us more directly, which could harm our business and have a material adverse effect on our business, financial condition, and results of operations; if certain license agreements are terminated, our ability to continue clinical trials and commercially market products could be adversely affected; the inability to protect the confidentiality of our proprietary information, trade secrets, and know-how; third-party claims of intellectual property infringement may prevent or delay our product development efforts; intellectual property rights do not necessarily address all potential threats to our competitive advantage; the inability to successfully develop and commercialize our product candidates and obtain the necessary regulatory approvals; we cannot market and sell our product candidates in the U.S. or in other countries if we fail to obtain the necessary regulatory approvals; final marketing approval of our product candidates by the FDA or other regulatory authorities for commercial use may be delayed, limited, or denied, any of which could adversely affect our ability to generate operating revenues; we may not be able to secure and maintain research institutions to conduct our clinical trials; ongoing healthcare legislative and regulatory reform measures may have a material adverse effect on our business and results of operations; if we receive regulatory approval of Lomecel-B™ or any of our other product candidates, we will be subject to ongoing regulatory requirements and continued regulatory review, which may result in significant additional expense; being subject to penalties if we fail to comply with regulatory requirements or experience unanticipated problems with our therapeutic candidates; reliance on third parties to conduct certain aspects of our preclinical studies and clinical trials; interim, “topline” and preliminary data from our clinical trials that we announce or publish from time to time may change as more data become available and are subject to audit and verification procedures that could result in material changes in the final data; the volatility of the price of our Class A common stock; we could lose our listing on the Nasdaq Capital Market; provisions in our certificate of incorporation and bylaws and Delaware law might discourage, delay or prevent a change in control of our company or changes in our management and, therefore, depress the market price of our Class A common stock; we have never commercialized a product candidate before and may lack the necessary expertise, personnel and resources to successfully commercialize any products on our own or together with suitable collaborators; and in order to successfully implement our plans and strategies, we will need to grow our organization, and we may experience difficulties in managing this growth. 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, 2023, filed with the Securities and Exchange Commission on February 27, 2024, as amended by the Annual Report on Form 10-K/A filed March 11, 2024, its Quarterly Reports on Form 10-Q, and its Current Reports on Form 8-K. The forward-looking statements contained in this press release are made as of the date of this press release, 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/f106bde7-972a-4687-a91e-03e924f409fc

FAQ

What were the survival rates for Lomecel-B in the ELPIS I HLHS trial (LGVN)?

The ELPIS I trial showed 100% 5-year survival rate post-Glenn procedure with no heart transplants needed, compared to 83% survival and 5.2% transplant rate in historical data.

What FDA designations has Lomecel-B received for HLHS treatment (LGVN)?

Lomecel-B has received Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation from the FDA for HLHS treatment.

How many patients were included in the ELPIS I trial for HLHS (LGVN)?

The ELPIS I trial included 10 patients (7 males and 3 females) who were followed for five years post-Glenn surgery and Lomecel-B treatment.