Longeveron in the NEWS
On May 9, 2023, Longeveron (LGVN) announced new long-term follow-up data from the Company’s ELPIS I trial of Lomecel-BTM for patients with hypoplastic left heart syndrome (HLHS).
What is Hypoplastic Left Heart Syndrome
HLHS is a rare and life-threatening congenital heart defect affecting approximately 1,000 babies per year. Infants born with HLHS have an underdeveloped or absent left ventricle. Current standard-of-care is comprised of three reconstructive heart surgeries (Stage I – III) to reconfigure the right ventricle to provide all cardiac output. These three surgeries must be performed within the first five years of life. The Stage I surgery (the Norwood procedure) is conducted within 2 weeks of birth and the Stage II (the Glenn procedure) is typically conducted by 4-5 months of age. A Stage III surgery (the Fontan procedure) is typically performed when the children are 4-5 years of age.
Without surgical treatment, the condition is always fatal, and even with reconstructive surgical reconstruction, patients often die or require heart transplantation by 15 years of age.
The New Long-Term Follow Up Data Results
The data 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 2 surgery.
To-date All 10 patients enrolled in the study have been monitored for at least 3.5 years after treatment with Lomecel-BTM. All patients are more than 4 years of age, with 2 being 5 years of age, and additional long-term follow-up is ongoing in the ELPIS I participants.
Historical results have shown that children with HLHS have approximately 20% mortality by 5 years. About three years after the Stage 2 surgery, most of these patients undergo the third palliative surgery, which is the last of the standard sequence of surgeries that comprises the current standard of care for HLHS.
To-date, 5 of 5 of the eligible patients from the ELPIS I trial have already undergone this third surgery.The Company expects to present these updated data at a scientific conference later in 2023.
From the Co-Founder of Longeveron and Others
Joshua M. Hare, M.D., co-founder of Longeveron, Chief Science Officer, and Chairman of the Board of Directors, said, “We are pleased to share additional long-term follow-up data demonstrating the continued survival of the ELPIS I trial participants. Previously, we disclosed the participants had 100% survival rate for up to 3.5 years post-administration of Lomecel-BTM. These data represent an additional 2 years of follow-up data, reinforcing our enthusiasm for Lomecel-BTM in this indication, and providing support for the ongoing ELPIS II study. As we continue to monitor these patients from ELPIS I, we will provide additional updates. There are currently no FDA-approved treatments for HLHS, and we hope Lomecel-BTM can significantly improve the treatment landscape for patients with HLHS.”
Sunjay Kaushal, M.D., Ph.D., Division Head of Cardiovascular-Thoracic Surgery at Lurie Children’s and principal investigator of the ELPIS I and ELPIS II studies said, “These results are encouraging, as patients with HLHS have progressive time-dependent increases in mortality and need for transplantation. 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 2 surgery. Additional historical data from the NHLBI-sponsored Single Ventricle Reconstruction (SVR) Trial, which is the largest HLHS trial to date, enrolled over 500 patients). The trial demonstrates that patients undergoing Stage 2 surgery have approximately 15% mortality by year 3 after surgery, which increases to approximately 20 % mortality by 5 years. 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.”
The ELPIS I trial was 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 7 sites in the ELPIS II trial, a 38-patient, randomized (1:1) blinded, controlled Phase 2a clinical trial designed to evaluate the safety and efficacy of intramyoca-rdial injection of Lomecel-BTM in infants with HLHS undergoing the Glenn Procedure.
Lomecel-BTM for HLHS has received Fast Track, Rare Pediatric Disease, and Orphan Drug Designations from the U.S. Food and Drug Administration (FDA).
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 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: HLHS, Alzheimer’s Disease and Aging-Related Frailty.
Additional information about the Company is available at www.longeveron.com.
ELPIS I trial data of Lomecel-BTM for patients with HLHS have encouraging results. It is undoubtedly encouraging when wwe realise that 100% of the patients in the ELPIS I trial have survived and remained heart transplant-free for up to 5 years of age after receiving Lomecel-BTM during their Stage 2 surgery.
We will follow up on the firm’s trial until the promised additional data from the ELPIS 2 trial is provided.
It is important to know that there are no FDA-approved treatments for HLHS,
Satisfactory results from the ongoing ELPIS II study would mean a lot for Longeveron as its treatment for HLHS has been granted a Rare Pediatric Disease Designation, Fast Track Designation and Orphan Drug Designation.
We hope that Lomecel-BTM can significantly improve the treatment landscape for the patients with HLHS.