HLHS Is A Rare And Serious Congenital Heart Disease With A 33% Neonatal Death Rate — Could Longeveron's Lomecel-B Improve Outcomes?

Longeveron LGVN announced the results of its phase 1 trial for infants with a rare and serious congenital heart disease (CHD), known as hypoplastic left heart syndrome. The Company, which is now conducting its phase 2a trial, is a clinical-stage biotechnology firm working to develop treatments for rare diseases and age-related and life-threatening conditions. 

CHDs are the most common congenital disease, affecting around 40,000 babies born in the U.S. every year. While many CHDs can be medically managed, 25% are much more serious and often require immediate surgical intervention. As a result, CHDs are a leading cause of death for infants. An increase in critical CHDs is powering growth in the newborn screening market, which is expected to reach $3 billion in 2031, growing at a compound annual growth rate (CAGR) of 11.2% from 2021 to 2031. 

Longeveron is working to develop a treatment for one of the most severe CHDs, hypoplastic left heart syndrome (HLHS). HLHS is a very serious heart defect in which the left side of the heart is underdeveloped and unable to provide normal blood circulation through the heart. HLHS accounts for 2-3% of all CHDs, with around 1 in every 3,800 US babies being born with HLHS every year. HLHS is considered a rare pediatric disease, and Longeveron has received a Rare Pediatric Disease Designation from the FDA for Lomecel-B.  Children with HLHS require 3 different open-heart surgeries to allow survival, but even with these surgeries, many of the children will go on to need a heart transplant.

Despite the increasing sophistication of these surgeries, the neonatal death rate for HLHS is 33%, and the 5-year survival rate is only 50-60%. HLHS treatments are also expensive, costing the average neonatal hospital stay over $280,000.

Longeveron Completes Phase 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 medicinal signaling cells (MSCs), will be a potential combinational therapy delivered via intracardiac injection during the second neonatal open-heart surgery. The goal of adding Lomecel-B™ is to boost the function of the single ventricle, with the ultimate goal of reducing the need for transplantation and possibly increasing survival.

The Company concluded its 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. The one-year trial’s results were published in the European Heart Journal Open, and revealed that all the patients had responded well to the procedure and were transplant-free one year after the treatment. None of the babies experienced a pre-defined safety endpoint, and the procedure was well tolerated.

“This data supports our hope that Lomecel-BTM has the potential to alter the treatment landscape for patients with HLHS, a rare and life-threatening congenital heart disease,” said Wa’el Hashad, Longeveron’s Chief Executive Officer. “Notably, the procedure appeared to be generally safe and well-tolerated in the babies undergoing heart surgery in the first 6 months of life. The study provisionally shows suggestions of possible improvement in measures of cardiac function, findings that are presently being tested in our ongoing controlled and randomized Phase 2 study.”

Dr. Joshua Hare, who co-authored the European Heart article, is the co-founder of Longeveron and is an established leader in the field of cardiac treatment. He recently published a second article on HLHS, which described the current status of treatments for HLHS and outlined the future direction of the field. The article identifies Lomecel-B™ as the first-in-class MSC therapy to reach the trial stage. 

Longeveron is moving forward with its phase 2 trial in partnership with the National Heart, Lung, and Blood Institute and the trial is being conducted at 8 leading pediatric cardiac surgical programs. This randomized, blind, controlled study with 38 patients will evaluate Lomecel-B’s™ intracardiac injection for both safety and efficacy. Future positive results could position Longeveron as a leader in the field, with the hope of saving the lives of many infants through this novel therapy.

Keep up to date with Longeveron’s cell-based therapies on its website www.longeveron.com.

Featured photo by Aditya Romansa on Unsplash.

This post contains sponsored content. This content is for informational purposes only and not intended to be investing advice