Longeveron (NASDAQ: LGVN) Reports 100% 5-Year Survival In Trial For Rare Pediatric Disease HLHS (Compared To 80% Survival In Historical Trials) To Market

Advancements in healthcare and medicine have helped contribute to the reduction in child mortality rates, plummeting from 185 per 1,000 births a century ago to just 7 per 1,000 births in 2020. However, some serious pediatric conditions continue to pose a threat. One is hypoplastic left heart syndrome (HLHS), a rare but dangerous heart defect where the child’s left ventricle is severely underdeveloped. 

Hypoplastic left heart syndrome (HLHS) impacts approximately 1 in 3,800 newborns (or about 1,025 live births in the United States annually). Children born with this condition have a significant risk of death stemming from the failure of their right heart ventricle. The case of T.J. Olsen, son of former NFL star Greg Olsen, who was born with HLHS, illustrates the gravity of this condition. Despite undergoing four intricate heart surgeries, T.J.'s condition continued to deteriorate, resulting in congestive heart failure. Ultimately, TJ had to have a life-saving heart transplant at 8 years of age – highlighting the severity of HLHS. 

Longeveron LGVN, a clinical-stage biopharmaceutical company focusing on age-related and life-threatening conditions, is developing Lomecel-B™, its leading drug candidate, to try to improve outcomes for children like T.J. who suffer from HLHS. Lomecel-B™is a cellular medicine known as a medicinal signaling cell (MSC) that can be derived from the bone marrow of healthy adults. Administering Lomecel-B™ directly into the heart tissue of the right ventricle during the 2nd heart surgery that HLHS patients undergo may have the potential to boost right ventricular function. If effective, this treatment could result in enhanced overall outcomes and potentially lessen the need for future heart transplants in the HLHS population.

100% Survival Rate For Phase 1 Trial

Longeveron is advancing its phase 2 trial (ELPIS II), studying 38 HLHS patients. The primary outcome measure is safety, and efficacy endpoints include the change in right ventricular ejection fraction (RVEF) at 12 months post-treatment. The trial will also assess several secondary endpoints, including changes in right ventricular function and morphology, changes in clinical outcomes and quality of life as well as blood biomarkers and safety.

This follows the long-term success of its phase 1 trial (ELPIS I). Longeveron recently released new data showing that among the 10 patients in the ELPIS I study, none experienced serious adverse events (SAEs), none of them needed heart transplants, and the group experienced a 100% 5-year survival rate, as compared with approximately 80 survival rate over the same period of time in historical trials. 

Sunjay Kaushal, M.D. Ph.D., Principal Investigator of the ELPIS I trial, commented on the results, “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-B™ as a much needed therapeutic innovation for this patient group.”

Position In Market

Though there are only approximately 1,000 babies born with this condition every year, there are opportunities available for drug development addressing smaller/rare conditions that  aren’t always available for drugs targeting conditions affecting larger populations: 

1. The development cost for these studies is significantly reduced as the population size – which can drive the cost of a drug trial – is much lower for rare conditions like HLHS than for more common chronic diseases. 
2. Longeveron may be able to leverage the benefits of the Orphan Drug Designation, Fast-Track designation, and Rare Pediatric Designation Longeveron has received from the Food & Drug Administration (FDA) for the development and request for approval of Lomecel-B™ in the treatment of HLHS, including potentially being able to take advantage of a priority review voucher often associated with these designations. Notably, Serepta recently sold its priority review voucher for $102 million.
3. Longeveron has the advantage of in-house manufacturing capabilities, including its state-of-the-art cGMP facility based in Miami. Because the market size is relatively small, Longeveron anticipates it can produce and distribute its own off-the-shelf Lomebel-B™ product without needing to scale up production beyond its current capacity for the potential HLHS market size.

Other companies involved in treating pediatric diseases include Moderna MRNA and Amgen AMGN.

Want to read more about how Longeveron is taking on HLHS? Check out its website. 

Other References:

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.

Featured photo by Carlo Navarro on Unsplash.

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