- A poster about Longeveron’s long-term follow-up data from its ELPIS I clinical trial was presented at the 2023 Scientific Sessions of the American Heart Association in November 2023
- The poster showed that 100% of the 10 patients enrolled in the study survived and remained heart transplant-free for up to 5 years of age after receiving Lomecel-B(TM) during their Stage II surgery
- The data supports Longeveron’s ongoing ELPIS II study, which has exceeded its 50% enrollment threshold
- The company believes the long-term follow-up data underscore the potential for Lomecel-B(TM) as a much-needed therapeutic innovation for HLHS patients
- Historical results from outside studies have shown that children with HLHS who are undergoing Stage II surgery have approximately 20% mortality by 5 years of age
Longeveron (NASDAQ: LGVN), a clinical-stage biotechnology company developing cellular therapies for aging-related and life-threatening conditions including hypoplastic left heart syndrome (“HLHS”), Alzheimer’s disease and Aging-related Frailty has evaluated – and still is evaluating – its lead investigational therapeutic candidate Lomecel-B(TM), an allogeneic, bone marrow-derived medicinal signaling cell (“MSC”) therapy product, in multiple clinical trials.
One of these trials, titled ELPIS I, was a Phase 1, open-label single-arm study whose final results Longeveron announced in September 2021 (https://ibn.fm/iL6z8). ELPIS I was designed to assess the safety and potential efficacy as of intramyocardial injection of Lomecel-B(TM) administered to 10 infants with HLHS during Stage II surgeries at approximately 4 months of age. The study met the primary safety endpoint: no major adverse cardiac events or any treatment-related infections during the first month post-treatment were reported. Moreover, secondary endpoints measured per protocol suggested Lomecel-B(TM) injection may improve patient long-term clinical outcome post-surgery.
While the positive results permitted the transition to a Phase 2 randomized, double-blind, controlled trial, ELPIS II, with enrollment commencing in July 2021 (https://ibn.fm/HuGcD), investigators elected to extend follow-up of the 10 infants after ELPIS I came to its planned end for a total period of five years. More than two years later, at the annual Scientific Sessions of the American Heart Association (“AHA”) held November 11-13, 2023, in Philadelphia, Pennsylvania, a poster about Longeveron’s extended long-term follow-up data was presented. (Additional long-term follow-up is still ongoing in the ELPIS I children.)
The poster, titled Long-term Transplant-free Survival Is Improved in Hypoplastic Left Heart Syndrome with Cell-based Therapy and authored by Sunjay Kaushal, M.D., Ph.D., the principal investigator of the ELPIS I study, and colleagues, showed that all the 10 children survived and remained heart transplant-free for up to 5 years of age after receiving Lomecel-B(TM) during their Stage II surgery.
The transplant-free survival follow-up on all patients enrolled in the study is ongoing. Moreover, the average age at the time of the last follow-up visit was 4.5 years, with 2 patients being 5 years of follow-up, according to the company.
“Long-term follow-up data from our ELPIS I trial demonstrate the continued survival of the participants, and reinforce potential survival benefit of Lomecel-B(TM) for patients with HLHS,” commented Joshua M. Hare, Longeveron’s Co-Founder, Chief Science Officer, and Chair of the Board of Directors. “These data represent an additional up to 2 years of follow-up data, which point to the potential of Lomecel-B(TM) in this indication and provide support for our ongoing ELPIS II study, which has exceeded its 50% enrollment threshold. We anticipate completing enrollment in this trial in 2024.”
FDA granted Rare Pediatric Disease Designation (“RPD”), Orphan Drug Designation, and Fast Track designation to Longeveron’s Lomecel-B(TM) for treatment of HLHS.
HLHS refers to cardiac malformations characterized by underdevelopment or absence of the left ventricle. Before the 1980s, HLHS was associated with 95% mortality within the first month of life. But in the last two decades of the 20th century, there was remarkable progress in the management of children born with HLHS, thanks to the introduction and successful implementation of operative reconstructive procedures, according to a 2000 study (https://ibn.fm/xo0Cy).
These procedures, which are still used to date, are performed in three stages. The final result is a reconstructed heart in which the right ventricle – which ordinarily pumps deoxygenated (venous) blood to the lungs – is now responsible for pumping oxygenated blood to the body through a reconstructed aorta, with venous blood returning directly to the lungs without going through the heart chambers.
While the operative procedures have helped lower mortality, some patients who have undergone surgeries still succumb to HLHS. According to a 2015 study (https://ibn.fm/r54vE), “The highest mortality for infants with HLHS undergoing surgical intervention is with Stage 1 palliation and the interstage period between Stage I and Stage II surgery, both done in the first year of life.”
Furthermore, Kaushal explained, “Historical data collected by the National Heart, Lung, and Blood Institute (‘NHLBI’) has shown that more than 15% of HLHS patients either required a heart transplant or died from their illness 12 months after having undergone Stage 2 surgery. Additional historical data from the NHLBI-sponsored Single Ventricle Reconstruction (‘SVR’) trial – the largest HLHS trial to date, with enrollment of more than 500 patients – shows that patients undergoing Stage II surgery have approximately 15% mortality by year 3 after surgery, which increased to approximately 20% mortality by 5 years.”
These statistics underscore the high unmet need among children with HLHS. Fortunately, the extended follow-up data, which show the 100% survival rate of ELPIS I patients up to 5 years post-treatment, highlight the opportunity for Lomecel-B(TM) as a much-needed therapeutic innovation for this patient group.
For more information, visit the company’s website at www.Longeveron.com.
NOTE TO INVESTORS: The latest news and updates relating to LGVN are available in the company’s newsroom at http://ibn.fm/LGVN
Certain statements in this document 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. 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 Longeveron’s clinical trials to demonstrate safety and efficacy of the company’s product candidates, and other positive results; the timing and focus of the company’s 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 the company’s product candidates, including its estimates of the number of patients who suffer from the diseases being targeted; the success of competing therapies that are or may become available; the beneficial characteristics, safety, efficacy and therapeutic effects of the company’s product candidates; the company’s ability to obtain and maintain regulatory approval of its product candidates in the U.S., Japan and other jurisdictions; the company’s plans relating to the further development of its product candidates, including additional disease states or indications it may pursue; the company’s plans and ability to obtain or protect intellectual property rights, including extensions of existing patent terms where available and its ability to avoid infringing the intellectual property rights of others; the need to hire additional personnel and the company’s ability to attract and retain such personnel; the company’s estimates regarding expenses, future revenue, capital requirements and needs for additional financing; the company’s need to raise additional capital, and the difficulties it may face in obtaining access to capital, and the dilutive impact it may have on its investors; the company’s financial performance and ability to continue as a going concern, and the period over which it estimates its existing cash and cash equivalents will be sufficient to fund its future operating expenses and capital expenditure requirements. Additionally, Longeveron makes no assurance that any public offering of its securities as described herein will occur at all, or that any such transaction will occur on the timelines, in the manner, or on the terms anticipated due to numerous factors. 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, 2022, filed with the Securities and Exchange Commission on March 14, 2023 and its Quarterly Reports on Form 10-Q filed with the SEC. The forward-looking statements contained in this presentation are made as of the date of this presentation, 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.
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