Benzinga | Nov 18, 2021 07:30AM EST

EXCLUSIVE: Longeveron Reports FDA Granted Rare Pediatric Disease Designation For Co.'s Lomecel-B For Treatment Of Hypoplastic Left Heart Syndrome

Phase 2 clinical trial underway for Hypoplastic Left Heart

Syndrome, which affects approximately 1,000 babies per

year

Miami, Florida--November 18th, 2021-- Longeveron Inc. (NASDAQ:LGVN)

("Longeveron" or "Company"), a clinical stage biotechnology company developing

cellular therapies for chronic aging-related and life-threatening conditions,

announced today that the U.S. Food and Drug Administration (FDA) has granted

Rare Pediatric Disease (RPD) designation for Lomecel-B for the treatment of

Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital

heart defect in infants. Lomecel-B, an investigational allogeneic, bone marrow-

derived medicinal signaling cell (MSC) product, is currently being evaluated in a

Phase 2 trial.

"We are pleased by FDA's acknowledgment of the urgent need for a safe and

effective treatment for children born with this rare and devastating congenital heart

defect," stated Longeveron co-founder and Chief Science Officer Joshua M. Hare,

M.D. "We are encouraged by our Phase 1 clinical data, and the progress being made

in the ongoing Phase 2 trial. Lomecel-B represents a unique cell therapy approach

that could potentially be administered at the same time as surgery in these critically

impacted infants."

Approximately 1,000 babies are born with HLHS each year in the U.S. HLHS babies

have an underdeveloped left ventricle, which impairs the heart's ability to pump

blood throughout the body. HLHS is often fatal without surgical intervention, in

which three surgical procedures are performed over the period of about 5 years, to

allow the right ventricle to be configured to pump blood to the body. Longeveron is

evaluating the safety of Lomecel-B injection into the right ventricle during the

second surgery (4 -- 6 months of age), and the effect on cardiac function and other

health status endpoints.

Longeveron recently reported clinical results from its safety-focused Phase I clinical

study of Lomecel-B in HLHS patients. When cardiac surgeons injected Lomecel-B

directly into the babies' hearts at the time of surgery, the cells were well tolerated

with no major adverse cardiac events and no infections considered to be related to

the investigational treatment.

One hundred percent of infants enrolled in the Phase 1 trial (n=10) were alive and

had not required a transplant between 2- 3.5 years post-surgery. Other



measurements of the babies' health, such as weight gain and growth pattern,

matched that of normal healthy babies.

The FDA may grant RPD designation for diseases that primarily affect children ages

18 years old or younger, and fewer than 200,000 individuals in the U.S. Under this

program, if the FDA approves Lomecel-B for the treatment of HLHS, Longeveron

may be eligible to receive a priority review voucher (PRV) if the application

submitted for the product satisfies certain conditions, and under current law, is

approved prior to September 30, 2026. Drug companies receiving a PRV can have

any subsequent drug or biologic application reviewed and a decision made in six

months as opposed to the standard 10 months, potentially accelerating the time to

market, or they can sell the PRV to another company. The PRV accelerated review is

for patients with serious conditions providing access to a potentially lifesaving or -

changing treatment sooner. For companies, it means they can potentially go to

market with their product more quickly, and potentially begin generating product

revenue.

Infants with HLHS are currently being enrolled in ELPIS II, a 38-subject, phase 2

randomized, double-blind, controlled clinical trial. ELPIS II is being funded in part by

a grant from the National Institute of Health's National Heart, Lung, and Blood

Institute (NHLBI; Grant number 1UG3HL148318), in collaboration with Longeveron,

and is led by Principal Investigator Sunjay Kaushal, MD, PhD, Division Head,

Cardiovascular-Thoracic Surgery, Ann and Robert H. Lurie Children's Hospital of

Chicago. Other participating clinical sites currently open for enrollment are

Advocate Children's Hospital, Park Ridge, IL; Primary Children's Hospital/University

of Utah, Salt Lake City, UT; Children's Hospital Los Angeles, Los Angeles, CA; and

Cincinnati Children's Hospital Medical Center, Cincinnati, OH.