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Extended Follow Up Data Show Continued, Transplant Free Survival of All Patients
Results Support Continued Investigation of Lomecel-BTM 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-BTM 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-BTM 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-BTM. 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-BTM 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-BTM 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-BTM 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-BTM as a much-needed therapeutic innovation for this patient group."
Lomecel-BTM 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-BTM 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.
