LGVN - HLHS Is A Rare And Serious Congenital Heart Disease With A 33% Neonatal Death Rate — Could Longeveron’s Lomecel-B Improve Outcomes?
(NewsDirect)
By David Willey,Benzinga
Longeveron (NASDAQ: LGVN) announced the results of itsphase 1 trial for infants with a rare and serious congenital heartdisease (CHD), known as hypoplastic left heart syndrome. The Company,which is now conducting its phase 2a trial, is a clinical-stagebiotechnology firm working to develop treatments for rare diseases andage-related and life-threatening conditions.
CHDs arethe most common congenital disease , affecting around 40,000babies born in the U.S. every year. While many CHDs can be medicallymanaged, 25% are much more serious and often require immediatesurgical intervention. As a result, CHDs are a leading cause of deathfor infants. An increase in critical CHDs is powering growth in thenewborn screening market, whichis expected to reach $3 billion in 2031 , growing at a compoundannual growth rate (CAGR) of 11.2% from 2021 to 2031.
Longeveron is working todevelop a treatment for one of the most severe CHDs, hypoplastic leftheart syndrome (HLHS). HLHS is a very serious heart defect in whichthe left side of the heart is underdeveloped and unable to providenormal blood circulation through the heart. HLHS accounts for 2-3% ofall CHDs, with around 1 in every 3,800 US babies being born with HLHSevery year. HLHS is considered a rare pediatric disease, andLongeveron has received a Rare Pediatric Disease Designation from theFDA for Lomecel-B. Children with HLHS require 3 different open-heartsurgeries to allow survival, but even with these surgeries, many ofthe children will go on to need a heart transplant.
Despite the increasing sophistication ofthese surgeries, the neonataldeath rate for HLHS is 33%, and the 5-year survival rate isonly 50-60%. HLHS treatments are also expensive, costing the averageneonatal hospital stay over $280,000.
Longeveron CompletesPhase 1, Starts Phase 2
Longeveron is focusing on HLHS with its cell-based therapy, the drug candidate,Lomecel-B ™. This drug, which is made from medicinalsignaling cells (MSCs), will be a potential combinational therapydelivered via intracardiac injection during the second neonatalopen-heart surgery. The goal of adding Lomecel-B™ is to boost thefunction of the single ventricle, with the ultimate goal of reducingthe need for transplantation and possibly increasing survival.
The Company concludedits open-label phase 1 trial on 10 patients, aged four to five months,and followed these children for a period ranging from 3.5-5 years. Theone-year trial’sresults were published in the European Heart JournalOpen , and revealed that all the patients had respondedwell to the procedure and were transplant-free one year after thetreatment. None of the babies experienced a pre-defined safetyendpoint, and the procedure was well tolerated.
“This data supportsour hope that Lomecel-BTM has the potential to alter the treatmentlandscape for patients with HLHS, a rare and life-threateningcongenital heart disease,” said Wa’el Hashad, Longeveron’s ChiefExecutive Officer. “Notably, the procedure appeared to be generallysafe and well-tolerated in the babies undergoing heart surgery in thefirst 6 months of life. The study provisionally shows suggestions ofpossible improvement in measures of cardiac function, findings thatare presently being tested in our ongoing controlled and randomizedPhase 2 study.”
Dr. Joshua Hare, who co-authored the European Heart article, is the co-founder of Longeveron and is an established leaderin the field of cardiac treatment. He recently publisheda second article on HLHS , which described the current statusof treatments for HLHS and outlined the future direction of the field.The article identifies Lomecel-B™ as the first-in-class MSC therapyto reach the trial stage.
Longeveron is moving forward with its phase 2 trialin partnership with the National Heart, Lung, and Blood Institute andthe trial is being conducted at 8 leading pediatric cardiac surgicalprograms. This randomized, blind, controlled study with 38 patientswill evaluate Lomecel-B’s™ intracardiac injection for both safetyand efficacy. Future positive results could position Longeveron as aleader in the field, with the hope of saving the lives of many infantsthrough this novel therapy.
Keep up to date with Longeveron’scell-based therapies on its website www.longeveron.com .
We are a clinical-stage biotechnologycompany developing cellular therapies for aging-related andlife-threatening conditions.Our lead investigational product isLomecel-B™, which is derived from culture-expanded medicinalsignaling cells (MSCs) that are sourced from bone marrow of younghealthy adult donors. We believe that by using the same cells thatpromote tissue repair, organ maintenance, and immune system function,we can develop safe and effective therapies for some of the mostdifficult diseases and conditions associated withaging.
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