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Longeveron® Announces Completion of Successful Investigator Meeting for On-going Phase 2b Clinical Trial Evaluating Lomecel-B™ as a Potential Treatment for HLHS, a Rare Pediatric Cardiac Disease

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Longeveron announces the completion of an investigator meeting for its ELPIS II Phase 2b clinical trial evaluating Lomecel-B™ as a treatment for Hypoplastic Left Heart Syndrome (HLHS), a rare pediatric heart defect. The trial is on track to complete enrollment of 38 patients by the end of 2024. Multiple nationally recognized children's treatment centers are participating. Phase 1 results showed a 100% transplant-free survival rate up to five years, compared to a 20% historical mortality rate. The trial aims to further evaluate the safety and efficacy of Lomecel-B™.

Positive
  • ELPIS II Phase 2b clinical trial on track to complete enrollment by end of 2024.
  • Multiple nationally recognized children’s treatment centers participating in ELPIS II.
  • Phase 1 trial showed 100% transplant-free survival up to five years.
  • Lomecel-B™ shows potential as a new treatment option for HLHS.
  • ELPIS II trial supported by grants from the National Institutes of Health (NIH).
Negative
  • Current HLHS treatment has only 50-60% survival rate to adolescence.
  • Potential risks in clinical trial outcomes not meeting Phase 1 success.
  • No guarantee that Phase 2b will replicate Phase 1 results.
  • Enrollment completion delayed until the end of 2024, which extends timelines.
  • The investigational nature of Lomecel-B™ implies regulatory and market uncertainties.

The promising outcomes from the ELPIS I Phase 1 clinical trial, where children showed a 100% transplant-free survival rate up to five years, present a significant advancement for Longeveron. This is a meaningful comparison against the 20% mortality rate seen in historical control data. Such results solidify the potential efficacy of Lomecel-B™ in treating Hypoplastic Left Heart Syndrome (HLHS), a severe congenital defect. The ongoing Phase 2b clinical trial is important for validating these initial findings.

It's important to note that HLHS typically results in a 50-60% survival rate to adolescence. The need for improved treatments is evident. The participation of nationally recognized healthcare institutions in ELPIS II further strengthens its credibility, potentially speeding up the regulatory approval process if results remain positive.

For investors, the success of Lomecel-B™ could mean a significant breakthrough, ensuring a strong foothold for Longeveron in the regenerative medicine market. However, keep in mind the inherent risks in clinical trials, including potential safety concerns or inefficacy in larger populations.

Longeveron's collaboration with renowned institutions and the National Institutes of Health (NIH) grants add substantial credibility to their research efforts. The ongoing Phase 2b trial, if successful, could lead to Lomecel-B™ becoming an essential therapy for HLHS, a relatively untapped market given the rarity of the condition. This could translate to a unique position in the pediatric cardiology market, offering substantial revenue growth opportunities.

Investors should note the timeline; with enrollment completion by the end of 2024, marketable results may not appear until mid to late 2025. This means short-term financial impact is minimal, while long-term gains could be significant. Key risks involve potential delays or negative trial outcomes, which could adversely affect stock performance.

Given the high survival rates observed in the Phase 1 trial and the involvement of multiple reputable entities, the company's prospects in this niche market look favorable. However, patience is necessary when dealing with biotech stocks due to the lengthy nature of clinical trials and regulatory approvals.

  • With current HLHS treatment, only 50-60% of infants survive to adolescence
  • In ELPIS I Phase 1 clinical trial, children experienced 100% transplant-free survival up to five years of age after receiving Lomecel-B™ compared to approximate 20% mortality rate observed from historical control data
  • ELPIS II Phase 2b clinical trial on track for completing enrollment by end of 2024
  • Multiple nationally recognized children’s treatment centers participating in ELPIS II

MIAMI, June 11, 2024 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN), a clinical stage regenerative medicine biotechnology company developing cellular therapies for rare, life-threatening and chronic aging-related conditions, today announced the successful completion of an investigator meeting for ELPIS II, the on-going Phase 2b clinical trial evaluating Lomecel-B™ as a potential adjunct treatment for Hypoplastic Left Heart Syndrome (HLHS), a rare pediatric congenital heart birth defect. The investigator meeting was organized to discuss the progress to date and operational implementation of the clinical trial, inviting the principal investigator and site staff from premiere infant and children’s treatment institutions across the country. ELPIS II is on track to complete enrollment by the end of 2024.

Healthcare institutions participating in the ELPIS II Phase 2 clinical trial include:

Three additional nationally recognized pediatric cardiothoracic institutions participated in the investigator meeting as part of their preparation for participating as investigative sites in ELPIS II.

“While we have made significant advances in diagnosing HLHS before a baby is born, we don’t yet know the cause, and current HLHS treatment requires infants to undergo a complex three-stage surgical reconstruction over the first 3-4 years of their life,” said Ram Kumar Subramanyan, division chief of Pediatric Cardiothoracic Surgery at Children’s Nebraska, and a Principal Investigator in ELPIS II. “Even with this comprehensive treatment, only 50% to 60% of infants survive to adolescence due to right ventricular failure. There is clearly an important unmet medical need to improve right ventricular function in these infants to positively impact both short- and long-term patient outcomes. Lomecel-B’s™ Phase 1 clinical data is highly encouraging and suggests it has the potential to be a new, additive treatment option for physicians and their patients dealing with HLHS. The community is very much looking forward to the results of ELPIS II.”

“We believe the data generated to date in HLHS support potential for Lomecel-B™ as a regenerative medical therapy to help these infants,” said Nataliya Agafonova, MD, Chief Medical Officer of Longeveron. “In the ELPIS I Phase 1 clinical trial, participating infants experienced 100% transplant-free survival up to five years of age after receiving Lomecel-B™ compared to approximate 20% mortality rate observed from historical control data. ELPIS II is designed to further our understanding of Lomecel-B’s™ safety and efficacy in this patient population. With the collaboration of our outstanding investigative sites, we remain on track to complete enrollment of the clinical trial by the end of this year.”

Longeveron’s lead investigational therapeutic candidate is Lomecel-B™, a proprietary, scalable, allogeneic, adult human bone marrow-derived medicinal signaling cell therapy. ELPIS II is enrolling 38 pediatric patients, with enrollment completion anticipated by year end 2024. The clinical trial is being conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) through grants from the National Institutes of Health (NIH). ELPIS II builds on the positive clinical results of ELPIS I.

About Hypoplastic Left Heart Syndrome (HLHS)

HLHS is a congenital birth defect in which the left ventricle (one of the pumping chambers of the heart) is either severely underdeveloped or missing. As a consequence, infants born with this condition have severely diminished systemic blood flow, which requires them to undergo a complex, three stage heart reconstruction surgery process over the course of the first 5 years of their lives. While these infants can now live into adulthood, early mortality is still extremely high in this population due to right ventricle failure, which is not meant for the increased load demanded for systemic circulation (blood circulation throughout the body). As such, there is an important unmet medical need to improve right ventricular function in these patients to improve both short-term and long-term outcomes.

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 potential 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 pursuing three pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer’s disease, and Aging-related Frailty. For more information, visit www.longeveron.com or follow Longeveron on LinkedIn, X, and Instagram.

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 and include, but are not limited to, the potential for Lomecel-B™ to be a beneficial treatment for patients with HLHS. 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, adverse global conditions, including macroeconomic uncertainty; inability to raise additional capital necessary to continue as a going concern; our history of losses and inability to achieve profitability going forward; the absence of FDA-approved allogenic, cell-based therapies for HLHS or other cardiac-related indications; ethical and other concerns surrounding the use of stem cell therapy or human tissue; our exposure to product liability claims arising from the use of our product candidates or future products in individuals, for which we may not be able to obtain adequate product liability insurance; the adequacy of our trade secret and patent position to protect our product candidates and their uses: others could compete against us more directly, which could harm our business and have a material adverse effect on our business, financial condition, and results of operations; if certain license agreements are terminated, our ability to continue clinical trials and commercially market products could be adversely affected; the inability to protect the confidentiality of our proprietary information, trade secrets, and know-how; third-party claims of intellectual property infringement may prevent or delay our product development efforts; the inability to successfully develop and commercialize our product candidates and obtain the necessary regulatory approvals; we cannot market and sell our product candidates in the U.S. or in other countries if we fail to obtain the necessary regulatory approvals; final marketing approval of our product candidates by the FDA or other regulatory authorities for commercial use may be delayed, limited, or denied, any of which could adversely affect our ability to generate operating revenues; we may not be able to secure and maintain research institutions to conduct our clinical trials; ongoing healthcare legislative and regulatory reform measures may have a material adverse effect on our business and results of operations; if we receive regulatory approval of Lomecel-B™ or any of our other product candidates, we will be subject to ongoing regulatory requirements and continued regulatory review, which may result in significant additional expense; being subject to penalties if we fail to comply with regulatory requirements or experience unanticipated problems with our therapeutic candidates; reliance on third parties to conduct certain aspects of our preclinical studies and clinical trials; interim, “topline” and preliminary data from our clinical trials that we announce or publish from time to time may change as more data become available and are subject to audit and verification procedures that could result in material changes in the final data; provisions in our certificate of incorporation and bylaws and Delaware law might discourage, delay or prevent a change in control of our company or changes in our management and, therefore, depress the market price of our Class A common stock; we have never commercialized a product candidate before and may lack the necessary expertise, personnel and resources to successfully commercialize any products on our own or together with suitable collaborators; and in order to successfully implement our plans and strategies, we will need to grow our organization, and we may experience difficulties in managing this growth. 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, 2023, filed with the Securities and Exchange Commission on February 27, 2024, as amended by the Annual Report on Form 10-K/A filed March 11, 2024, its Quarterly Reports on Form 10-Q, and its Current Reports on Form 8-K. 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:
Derek Cole
Investor Relations Advisory Solutions
derek.cole@iradvisory.com


FAQ

What is Longeveron’s ELPIS II clinical trial?

ELPIS II is a Phase 2b clinical trial by Longeveron evaluating Lomecel-B™ as a treatment for Hypoplastic Left Heart Syndrome (HLHS).

When is the ELPIS II trial expected to complete enrollment?

The ELPIS II trial is expected to complete enrollment by the end of 2024.

What were the results of the ELPIS I Phase 1 clinical trial?

The ELPIS I Phase 1 trial showed a 100% transplant-free survival rate up to five years for infants who received Lomecel-B™.

Which institutions are participating in the ELPIS II trial?

Institutions include Children's Hospital of Los Angeles, Children's Healthcare of Atlanta, Ann & Robert H. Lurie Children's Hospital of Chicago, and several others.

What is the potential benefit of Lomecel-B™ for HLHS?

Lomecel-B™ may improve right ventricular function and patient outcomes in infants with HLHS, addressing an unmet medical need.

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