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Cellular Therapy Co. Earns Rare Pediatric Disease Designation for Infant Congenital Heart Treatment

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Shares of Longeveron Inc. traded 50% higher after the company reported that the U.S. FDA approved its Lomecel-B for Rare Pediatric Disease Designation to treat a rare and life-threatening defect in infants called Hypoplastic Left Heart Syndrome.

Clinical-stage biotechnology company Longeveron Inc. (LGVN:NASDAQ), which is focused on developing cellular therapies for chronic aging-related and life-threatening conditions, yesterday announced 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."

Longeveron stated that "Lomecel-B is an investigational allogeneic, bone marrow-derived medicinal signaling cell (MSC) product, derived from culture-expanded medicinal signaling cells (MSCs) isolated from the donated bone marrow of young healthy adults."

The company's Co-Founder and Chief Science Officer Joshua M. Hare, M.D. remarked, "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…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."

The firm noted that every year about 1,000 babies are born with HLHS in the U.S. Infants born with this condition have an underdeveloped left ventricle, which effects the heart's ability to pump and circulate blood throughout the body. The company advised that without surgical intervention HLHS is often fatal. Typically, this involves a series of three separate surgical procedures that are conducted over a five-year period. The primary objective of these procedures is to a re-configure the right ventricle allowing it to pump blood to the body.

Longeveron explained that "it 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."

The company indicated that it recently reported results from its Phase I Lomecel-B clinical trial in infant patients diagnosed with HLHS. The Phase 1 study was primarily focused on verifying the safety of Lomecel-B. The firm stated that during the trial, Lomecel-B was injected directly into the babies' hearts by cardiac surgeons at the time of surgery. The results from the study showed that the cells were well tolerated and that no major adverse cardiac events or infections were recorded.

Longeveron commented that all ten of the infants enrolled in the Phase 1 study were alive and did not require a heart transplant between two to three and a half years following surgery. The firm added that the heath of the babies in the clinical trial was comparable to other normal healthy babies in terms of growth pattern and weight gain.

The firm pointed out that "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." The company advised that if the FDA approves Lomecel-B for the treatment of HLHS under the RPD program, it may be eligible to receive a priority review voucher (PRV) if specific conditions are met. The PRV provides drug companies with expedited drug or biologic application review in as little as six months versus the standard ten months.

The company mentioned that a Phase 2 ELPIS II clinical trial is now underway for Hypoplastic Left Heart Syndrome. The firm indicated that the randomized, double-blind study calls for enrollment of thirty-eight infant patient subjects with HLHS. The study, in collaboration with Longeveron, is being partly funded by a grant from the National Institute of Health's National Heart, Lung, and Blood Institute. The lead investigator in the study was identified as Sunjay Kaushal, M.D., Ph.D., Division Head, Cardiovascular-Thoracic Surgery, Ann and Robert H. Lurie Children's Hospital of Chicago.

Longeveron is a clinical-stage biotechnology company based in Miami, Fla. that is engaged in the development of cellular therapies for treating aging-related and life-threatening conditions. The firm is striving to create cellular therapies that are derived from cells within the body in order to repair, regenerate and restore tissue at the site of damage, inflammation and disease.

The company listed that its lead investigational cell-based therapy product, LOMECEL-B™, is sourced from bone marrow of young, healthy adult donors and is derived from culture-expanded medicinal signaling cells (MSCs). Longeveron claimed that "it believes that by using the same cells that promote tissue repair, organ maintenance, and immune system function, it can develop safe and effective therapies for some of the most difficult disorders associated with the aging process and other medical disorders."

In addition to its ongoing efforts in HLDS studies, Longeveron is also presently supporting and conducting Phase 1 and 2 clinical trials for Acute Respiratory Distress Syndrome, Aging Frailty, Alzheimer's disease and Metabolic Syndrome. The company wrote that "it's mission is to advance Lomecel-B and other cell-based product candidates into pivotal Phase 3 trials, with the goal of achieving regulatory approvals, subsequent commercialization and broad use by the healthcare community."

Longeveron started the day on Friday with a market cap of around $131.4 million with approximately 19.1 million shares outstanding and a short interest of about 8%. LGVN shares opened 70% higher at $11.81 (+$4.94, +71.91%) over Thursday's $6.87 closing price and reached a new 52-week high price Friday morning of $14.75. The stock traded between $8.95 and $14.75 per share and closed Friday at $10.03 (+$3.16, +46.00%).

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