-- Full results published in European Heart Journal Open --
-- Study met primary safety endpoint --
-- All patients alive, transplant-free, and maintained expected rate of growth one year after treatment --
-- Data supports ongoing clinical development of Lomecel-BTM for HLHS, currently being evaluated in a Phase 2a trial --
MIAMI, Jan. 19, 2023 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN) ("Longeveron" or "Company"), a clinical stage biotechnology company developing regenerative medicines for unmet medical needs, today announced the publication of the full results from the Company’s ELPIS I trial of Lomecel-BTM for hypoplastic left heart syndrome (HLHS) in the European Heart Journal Open. In the uncontrolled open-label Phase 1b study entitled “Intramyocardial cell-based therapy with Lomecel-B during bidirectional cavopulmonary anastomosis for hypoplastic left heart syndrome: The ELPIS phase I trial,” patients (n=10) underwent the Glenn Procedure (an open heart surgery) at approximately 4-5 months old, and received intracardiac injections of Lomecel-BTM.
“We are pleased that the data from the ELPIS I trial have been published in European Heart Journal Open,” said Chris Min, M.D., Ph.D., Interim Chief Executive Officer and Chief Medical Officer. “This data supports our conviction that Lomecel-BTM has the potential to alter the treatment landscape for patients with HLHS, a rare and life-threatening congenital heart disease. 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.”
The ELPIS I study was conducted by a consortium of leading pediatric cardiac surgeons led by Sunjay Kaushal, M.D., Ph.D., currently Division Head of Cardiovascular-Thoracic Surgery at Lurie Children’s and principal investigator of the ELPIS I study. Dr. Kaushal said, “On behalf of the families affected with this devastating condition and my surgical colleagues, we are thrilled to publish these results. We are hopeful we may be able to add this therapy to the armamentarium for managing babies with HLHS.”
The findings of the study included:
- None of the children experienced the prespecified safety endpoint over one year of follow-up. The procedure was generally well-tolerated in all ten study participants.
- The babies maintained their weight-for-age z-score after the surgery, and all were alive and transplant-free one year following the procedure.
- Several indices of right ventricular (RV) function, notably the tricuspid regurgitation fraction, showed suggestions of either improvement or prevention of deterioration over one-year following the surgery.
- In a sub-study of 6 babies, a blood test showed evidence of the persistence and activity of Lomecel-BTM one week after injection.
Longeveron is currently enrolling patients in the ELPIS II trial, a 38-patient, randomized (1:1), blinded, controlled Phase 2a clinical trial intended to evaluate the safety and efficacy of intramyocardial injection of Lomecel-BTM in infants with HLHS who are undergoing the Glenn Procedure. This ongoing trial is funded by a grant from the National Heart, Lung, and Blood Institute. Lomecel-BTM for HLHS has received Fast Track, Rare Pediatric Disease, and Orphan Drug Designations from the U.S. Food and Drug Administration (FDA).
The manuscript may be found at:
https://academic.oup.com/ehjopen/advance-article/doi/10.1093/ehjopen/oead002/6984935.
About Hypoplastic Left Heart Syndrome
Hypoplastic left heart syndrome (HLHS) is a rare and life-threatening congenital heart defect affecting approximately 1,000 babies per year. Infants born with HLHS have an underdeveloped or absent left ventricle. Current standard-of-care is comprised of three reconstructive heart surgeries to reposition the right ventricle to cardiac function. These three surgeries must be performed within the first five years of life. The first surgery (the Norwood procedure) is conducted within 2 weeks of birth and the second (the Glenn procedure) is conducted by 4-5 months of age. A final third operation is performed when the children are 4-5 years of age. Without surgical treatment, the condition is always fatal, and even with reconstructive surgical reconstruction, patients often die or require heart transplantation by age 15.
About Longeveron Inc.
Longeveron is a clinical stage biotechnology company developing regenerative medicines to address unmet medical needs. The Company’s lead investigational product is Lomecel-B™, an allogeneic medicinal signaling cell (MSC) therapy product isolated from the bone marrow of young, healthy adult donors. Lomecel-B™ has multiple mechanisms of action encompassing pro-vascular, pro-regenerative, anti-inflammatory, and tissue repair and healing effects with broad potential applications across a spectrum of disease areas. Longeveron is currently advancing Lomecel-B™ through clinical trials in three indications: hypoplastic left heart syndrome (HLHS), Alzheimer’s Disease, and Aging-Related Frailty. Additional information about the Company is available at www.longeveron.com.
Forward-Looking Statements
Certain statements in this press release that are not historical facts are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, which reflect management's current expectations, assumptions, and estimates of future operations, 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. Factors that could cause actual results to differ materially from those expressed or implied in any forward-looking statements in this release include, but are not limited to, statements about the ability of Longeveron’s clinical trials to demonstrate safety and efficacy of the Company’s product candidates, and other positive results; the timing and focus of the Company’s ongoing and future preclinical studies and clinical trials and the reporting of data from those studies and trials; the size of the market opportunity for the Company’s product candidates, including its estimates of the number of patients who suffer from the diseases being targeted; the success of competing therapies that are or may become available; the beneficial characteristics, safety, efficacy and therapeutic effects of the Company’s product candidates; the Company’s ability to obtain and maintain regulatory approval of its product candidates; the Company’s plans relating to the further development of its product candidates, including additional disease states or indications it may pursue; existing regulations and regulatory developments in the U.S., Japan and other jurisdictions; the Company’s plans and ability to obtain or protect intellectual property rights, including extensions of existing patent terms where available and its ability to avoid infringing the intellectual property rights of others; the need to hire additional personnel and the Company’s ability to attract and retain such personnel; the Company’s estimates regarding expenses, future revenue, capital requirements and needs for additional financing; the Company’s need to raise additional capital, and the difficulties it may face in obtaining access to capital, and the dilutive impact it may have on its investors; the Company’s financial performance, and the period over which it estimates its existing cash and cash equivalents will be sufficient to fund its future operating expenses and capital expenditures requirements. 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 Securities and Exchange Commission, including Longeveron’s Annual Report on Form 10-K for the year ended December 31, 2021, filed with the SEC on March 11, 2022, and the Company’s Quarterly Reports on Form 10-Q. 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.
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Source: Longeveron Inc
