Long-term Survival Data from Longeveron’s ELPIS 1 Trial Presented at the 2023 Scientific Sessions of the American Heart Association

Rhea-AI Summary

Longeveron Inc. (NASDAQ: LGVN NASDAQ: LGVNR) announced that extended long-term follow-up data from the ELPIS I trial of Lomecel-BTM for patients with hypoplastic left heart syndrome (HLHS) is being presented at the annual Scientific Sessions of the American Heart Association. The results show 100% survival and transplant-free status of all 10 patients for up to 5 years after receiving Lomecel-BTM during their Stage II surgery, supporting the ongoing ELPIS II trial. Lomecel-BTM has received Rare Pediatric Disease Designation, Fast Track Designation, and Orphan Drug Designation by the US FDA for the HLHS indication.

Positive

• The ELPIS I trial demonstrated 100% survival and transplant-free status of patients with HLHS up to 5 years post-treatment, showing potential benefits of Lomecel-BTM in this indication.
• The ongoing ELPIS II trial, funded by a grant from the National Institutes of Health, has exceeded its 50% enrollment threshold, showing strong interest and support for further investigation of Lomecel-BTM.

Negative

• None.

Extended Follow Up Data Show Continued, Transplant Free Survival of All Patients

Results Support Continued Investigation of Lomecel-B^TM As Adjunct to Stage II Surgery in HLHS

- Enrollment ongoing in Phase 2 ELPIS II trial -

MIAMI, Nov. 11, 2023 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN NASDAQ: LGVNR) (“Longeveron” or “Company”), a clinical-stage biotechnology company developing cellular therapies for life-threatening and chronic aging-related conditions such as hypoplastic left heart syndrome (HLHS), Alzheimer’s disease and Aging-related Frailty, announced today that extended long-term follow-up data from the Company’s ELPIS I trial of Lomecel-B^TM for patients with hypoplastic left heart syndrome (HLHS) is being presented as a poster at the annual Scientific Sessions of the American Heart Association (AHA), being held in Philadelphia, PA on November 11-13, 2023.

The poster, titled Long-term Transplant-free Survival Is Improved in Hypoplastic Left Heart Syndrome with Cell-based Therapy (Sunjay Kaushal, et al), showed that 100% of the 10 patients who participated in the ELPIS I trial survived and remained heart transplant-free for up to 5 years of age after receiving Lomecel-B^TM during their Stage II surgery. The extended follow-up data on all patients enrolled in the study now includes monitoring for up to 5 years following treatment with Lomecel-B^TM. The average age at the time of the last follow-up visit was 4.5 years, with 2 patients being 5 years of age. Additional long-term follow-up is ongoing in the ELPIS I participants. Historical results from outside studies have shown that children with HLHS have approximately 20% mortality by 5 years.

“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,” said Joshua M. Hare, M.D., Longeveron’s Co-founder, Chief Science Officer, and Chairman 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. There is an unmet need to improve the transplant-free survival for patients with HLHS, and we hope Lomecel-B^TM can significantly improve the treatment landscape for this patient population.”

“These results are encouraging, as patients with HLHS have progressive time-dependent increases in mortality and need for transplantation,” added Sunjay Kaushal, M.D., Ph.D., principal investigator of the ELPIS I study. “Historical data collected by the National Heart, Lung, and Blood Institute 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 II 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 2 surgery have approximately 15% mortality by year 3 after surgery, which increased to approximately 20% mortality by 5 years.^1,2,3 There is a major unmet need among children with HLHS, and today’s data highlighting the 100% survival rate of ELPIS I patients up to 5 years post-treatment underscore the opportunity for Lomecel-B^TM as a much-needed therapeutic innovation for this patient group.”

Lomecel-B^TM has received Rare Pediatric Disease Designation, Fast Track Designation, and Orphan Drug Designation by the US Food and Drug Administration (FDA) for the HLHS indication. The ELPIS I trial (NCT03525418) was an open-label, Phase 1b study 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, 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.

Longeveron is currently enrolling patients at 8 sites in the ELPIS II (NCT04925024) trial, a 38-patient, randomized double-blind, and controlled clinical trial designed to evaluate the safety and efficacy of Lomecel-B in conjunction with reconstructive surgery compared to surgery alone. ELPIS II is being funded by a grant from the National Institute of Health’s National Heart, Lung, and Blood Institute (NHLBI; Grant number 1UG3HL148318), in collaboration with Longeveron.

¹ Newburger et al. Circulation (2018) 137:2246-2253.
² Newburger et al. Circulation (2014) 129:2013-2020.
³ Ohye et al. N Engl J Med (2010) 362:1980-1992.

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 advancing Lomecel-B™ through clinical trials in three indications: hypoplastic left heart syndrome (HLHS), Alzheimer’s disease, and Aging-related Frailty. Additional information about the Company is available at www.longeveron.com.

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. 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 anticipated use of proceeds from the offering, as well as statements about 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; market and other conditions; 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. Further information relating to factors that may impact the Company's results and forward-looking statements are disclosed in the Company’s Quarterly Reports as filed with the Securities and Exchange Commission. 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 Contact
Mike Moyer
LifeSci Advisors
Tel: 617-308-4306
Email: mmoyer@lifesciadvisors.com

FAQ

What is the significance of the ELPIS I trial results for Lomecel-BTM?

The ELPIS I trial results demonstrate 100% survival and transplant-free status of patients with HLHS for up to 5 years post-treatment, indicating potential benefits of Lomecel-BTM in this indication.

What designations has Lomecel-BTM received from the US FDA?

Lomecel-BTM has received Rare Pediatric Disease Designation, Fast Track Designation, and Orphan Drug Designation from the US FDA for the HLHS indication.

What is the status of the ongoing ELPIS II trial?

The ongoing ELPIS II trial, funded by a grant from the National Institutes of Health, has exceeded its 50% enrollment threshold, indicating strong interest and support for further investigation of Lomecel-BTM.