Longeveron Announces New Long-Term Survival Data from ELPIS I Trial of Lomecel-B™ for Hypoplastic Left Heart Syndrome

- Children in the ELPIS I trial had 100% survival up to 5 years of age after receiving Lomecel-B^TM compared to 20% mortality rate observed from historical control data -

- Data reinforce potential survival benefit of Lomecel-B^TM for patients with hypoplastic left heart syndrome (HLHS) -

- Enrollment ongoing in Phase 2a ELPIS II trial -

- Lomecel-B^TM has Rare Pediatric Disease Designation, Fast Track Designation, and Orphan Drug Designation for HLHS Indication -

MIAMI, May 09, 2023 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN) ("Longeveron" or "Company"), a clinical stage biotechnology company developing regenerative medicines for unmet medical needs, today announced new long-term follow-up data from the Company’s ELPIS I trial of Lomecel-B^TM for patients with hypoplastic left heart syndrome (HLHS).

The data showed that 100% of the 10 patients who participated in the ELPIS I trial survived and remained heart transplant-free for up to 5 years of age after receiving Lomecel-B^TM during their Stage 2 surgery. To-date All 10 patients enrolled in the study have been monitored for at least 3.5 years after treatment with Lomecel-B^TM. All patients are more than 4 years of age, with 2 being 5 years of age, and additional long-term follow-up is ongoing in the ELPIS I participants. Historical results have shown that children with HLHS have approximately 20% mortality by 5 years.

In addition, about three years after the Stage 2 surgery, most of these patients undergo the third palliative surgery, which is the last of the standard sequence of surgeries that comprises the current standard of care for HLHS. To-date, 5 of 5 of the eligible patients from the ELPIS I trial have already undergone this third surgery.

The Company expects to present these updated data at a scientific conference later in 2023.

“We are pleased to share additional long-term follow-up data demonstrating the continued survival of the ELPIS I trial participants,” said Joshua M. Hare, M.D., co-founder of Longeveron, Chief Science Officer, and Chairman of the Board of Directors. “Previously, we disclosed the participants had 100% survival rate for up to 3.5 years post-administration of Lomecel-B^TM. These data represent an additional 2 years of follow-up data, reinforcing our enthusiasm for Lomecel-B^TM in this indication, and providing support for the ongoing ELPIS II study. As we continue to monitor these patients from ELPIS I, we will provide additional updates. There are currently no FDA-approved treatments for HLHS, and we hope Lomecel-B^TM can significantly improve the treatment landscape for patients with HLHS.”

“These results are encouraging, as patients with HLHS have progressive time-dependent increases in mortality and need for transplantation,” added Sunjay Kaushal, M.D., Ph.D., Division Head of Cardiovascular-Thoracic Surgery at Lurie Children’s and principal investigator of the ELPIS I and ELPIS II studies. “Historical data collected by the National Heart, Lung, and Blood Institute has shown that more than 15% of HLHS patients either required a heart transplant or died from their illness 12 months after having undergone Stage 2 surgery. Additional historical data from the SVR trial (The NHLBI-sponsored Single Ventricle Reconstruction (SVR) Trial is the largest HLHS trial to date, having enrolled over 500 patients) shows that patients undergoing Stage 2 surgery have approximately 15% mortality by year 3 after surgery, which increases to approximately 20 % mortality by 5 years.^1,2,3 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^TM as a much needed therapeutic innovation for this patient group.”

The ELPIS I trial was open-label, Phase 1b study designed to evaluate the safety of Lomecel-B^TM in patients with HLHS. Patients underwent the Glenn Procedure (an open heart surgery) at approximately 4-5 months of age. The results from the ELPIS I trial, which were previously reported, showed that the study met its primary safety endpoint, and that all patients were alive, transplant-free, and maintained their expected rate of growth one year after treatment.

Longeveron is currently enrolling patients at 7 sites in the ELPIS II trial, a 38-patient, randomized (1:1) blinded, controlled Phase 2a clinical trial designed to evaluate the safety and efficacy of intramyocardial injection of Lomecel-B^TM in infants with HLHS undergoing the Glenn Procedure. Lomecel-B^TM for HLHS has received Fast Track, Rare Pediatric Disease, and Orphan Drug Designations from the U.S. Food and Drug Administration (FDA).

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 (Stage I – III) to reconfigure the right ventricle to to provide all cardiac output. These three surgeries must be performed within the first five years of life. The Stage I surgery (the Norwood procedure) is conducted within 2 weeks of birth and the Stage II (the Glenn procedure) is typically conducted by 4-5 months of age. A Stage III surgery (the Fontan procedure) is typically 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 15 years of age.

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 in the U.S., Japan and other jurisdictions; the Company’s plans relating to the further development of its product candidates, including additional disease states or indications it may pursue; 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 expenditure 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, 2022, filed with the SEC on March 14, 2023. 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.

Investor Contact:

Stern IR, Inc.
212-698-8700
longeveron@sternir.com

Source: Longeveron Inc

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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.