Longeveron Inc. LGVN, a cell therapy company developing therapies for age-related and life-threatening conditions, reported Thursday final results from a Phase 1 study of its lead therapeutic candidate.
What Happened: Miami, Florida-based Longeveron said the Phase 1 study of Lomecel-B injection in hypoplastic left heart syndrome patients met the primary safety endpoint.
Lomecel-B is an allogeneic, bone marrow-derived medicinal signaling cell therapy product manufactured under current good manufacturing practice in Longeveron's cell processing facility in Miami.
The company noted that no major adverse cardiac events or any treatment-related infections were observed in the first month after the treatment.
Additionally, secondary endpoints measured per protocol pointed toward Lomecel-B injection likely improving patient long-term clinical outcome after surgery, the company added.
The company, however, said the secondary endpoint findings should be treated with caution due to the absence of a control arm that may have given a fair idea of whether the improvement was surgery-related, Lomecel-B-related, or both.
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Longeveron also noted that 100% of infants were alive and heart-transplant free, with follow-up ranging two to 3.5 years after cardiac surgery that included injection with Lomecel-B. This compares favorably to historical control results that indicate only 78% survival free of transplantation following the Glenn procedure, according to the company.
"We are highly motivated to continue our studies of Lomecel-B cell therapy for children with HLHS. If we determine in the future that Lomecel-B reduces the need for heart transplantation in these children, this will represent a major advance in the field, and have a substantial impact on these infants and their families," Dr. Sunjay Kaushal, principal pnvestigator of the study, said in a statement.
Why It's Important: HLHS is a rare congenital heart disease that affects about 1,000 babies per year in the U.S. Babies with HLHS are born with an underdeveloped left ventricle, which impairs the heart's ability to pump blood throughout the body.
The study was partially funded by a grant from the Maryland Stem Cell Research Fund.
Longeveron said the HLHS program has transitioned into a Phase 2 trial. The study is being funded by a grant from the NIH's National Heart, Lung, and Blood Institute.
With a target enrollment of 38 infants, the trial began enrollment in July, and it is anticipated that up to seven children's hospitals will be participating, all in major metropolitan centers located throughout the U.S., Longeveron said.
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