• Multicenter, double-blinded, controlled study designed to evaluate safety & efficacy of Lomecel-B intramyocardial injection in infants with HLHS

• Funded by the National Heart, Lung and Blood Institute (Grant number 1UG3HL148318 and 1U24HL148316) in collaboration with Longeveron

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 first patient has been enrolled and treated in the Phase 2b clinical study evaluating Lomecel-B intraventricular injection in infants with Hypoplastic Left Heart Syndrome (HLHS). Lomecel-B is an investigational allogeneic, bone marrow-derived medicinal signaling cell (MSC) product manufactured in Longeveron’s cell processing facility in Miami, Florida.

Longeveron’ s HLHS program has transitioned to Phase 2, with a randomized, double-blind, controlled trial titled: Evaluation of Lomecel-B™ Injection in Patients with Hypoplastic Left Heart Syndrome: A Phase IIb Clinical Trial. (ELPIS II). The trial is being funded by a grant from the National Institute of Health’s National Heart, Lung and Blood Institute (NHLBI), 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. With a target enrollment of 38 infants, the trial will be enrolling in 7 children’s hospitals in major metropolitan centers located throughout the U.S. For more information regarding the trial design and location of clinical sites please visit www.elpistrial.org hosted by the University of Texas Health Sciences Center which serves as the data coordinating center.

“After demonstrating safety and feasibility in the successfully completed Phase 1 ELPIS trial, we are now embarking upon the next phase of development, which importantly includes a control arm for comparison against babies treated with Lomecel-B injection,” remarked Dr. Kaushal. “Our hypothesis is that transplanted Lomecel-B medicinal signaling cells into the right ventricle as an adjunct to HLHS surgery will improve right ventricle structure and performance compared with surgery alone, potentially leading to a clinical benefit. We feel that this trial is a crucial step forward for developing cell-based therapy as an adjunct for surgery in HLHS.”

About Hypoplastic Left Heart Syndrome

HLHS is a rare congenital heart defect that effects approximately 1,000 babies per year in the U.S. Babies with HLHS are born with an underdeveloped left ventricle, which creates a life-threatening condition due to the heart’s inability to pump adequate amounts of blood throughout the body. Children must undergo a complex three-staged reconstructive surgery. Even with the advent of surgical intervention, babies with the condition still have a high rate of needing heart transplant, and is associated with high mortality. In the Phase 1 trial, the primary safety endpoint was met, with no major adverse cardiac events reported.  The cell injections also did not cause infections attributable to the study product. Additional clinical safety and efficacy results from the Phase 1 trial are currently being analyzed.

In HLHS patients, the right ventricle is subject to chronic pressure overload due to the lack of left ventricle, and thus becomes dysfunctional, leading to heart failure. It is believed that the primary therapeutic benefit of MSCs comes from the secretion of bioactive molecules that promote neovascularization, favorable remodeling, and activation of endogenous stem cells and cardiomyocytes, ultimately leading to improvement in overall structure, function, and durability. The goal of Lomecel-B injection is reduced myocardial fibrosis, increased angiogenesis, and increased cardiomyocyte and endothelial cell proliferation.

About Longeveron Inc.

Longeveron is a clinical stage biotechnology company developing cellular therapies for specific aging-related and life-threatening conditions. The Company’s lead investigational product is the LOMECEL-B™ cell-based therapy product (“Lomecel-B”), which is derived from culture-expanded medicinal signaling cells (MSCs) that are sourced from bone marrow of young, healthy adult donors. Longeveron 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. Longeveron is currently sponsoring Phase 1 and 2 clinical trials in the following indications: Aging Frailty, Alzheimer’s disease, the Metabolic Syndrome, Acute Respiratory Distress Syndrome (ARDS), and hypoplastic left heart syndrome (HLHS). The Company’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. Additional information about the Company is available at www.longeveron.com.

Forward-Looking and Other Statements

Certain statements in this press release that are not historical facts are forward-looking statements that reflect management's current expectations, assumptions, and estimates of future performance and economic conditions, and involve risks and uncertainties that could cause actual results to differ materially from those anticipated by the statements made herein. Forward-looking statements are generally identifiable by the use of forward-looking terminology such as "believe," "expects," "may," "looks to," "will," "should," "plan," "intend," "on condition," "target," "see," "potential," "estimates," "preliminary," or "anticipates" or the negative thereof or comparable terminology, or by discussion of strategy or goals or other future events, circumstances, or effects. Moreover, forward-looking statements in this release include, but are not limited to, statements about the ability of our clinical trials to demonstrate safety and efficacy of our product candidates, and other positive results; the timing and focus of our ongoing and future preclinical studies and clinical trials; the size of the market opportunity for our product candidates, the beneficial characteristics, safety, efficacy and therapeutic effects of our product candidates; our ability to obtain and maintain regulatory approval of our product candidates, our plans and ability to obtain or protect intellectual property rights, including extensions of existing patent terms where available and our ability to avoid infringing the intellectual property rights of others. Further information relating to factors that may impact the Company's results and forward-looking statements are disclosed in the Company's filings with the SEC. The forward-looking statements contained in this press release are made as of the date of this press release, and the Company disclaims any intention or obligation, other than imposed by law, to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.

Research referred to in this press release is being supported by the National Heart, Lung, And Blood Institute of the National Institutes of Health under Award Number UG3HL148318. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

Investor Contact:

Brendan Payne
Stern Investor Relations
Office Direct: 212-698-8695 |Office Main: 212-362-1200
This email address is being protected from spambots. You need JavaScript enabled to view it. | www.sternir.com

Source: Longeveron Inc
Source: LGVN