Longeveron® Announces Positive Type C Meeting with U.S. FDA Regarding Pathway to BLA for Lomecel-B™ in Hypoplastic Left Heart Syndrome (HLHS)

Rhea-AI Summary

Longeveron (NASDAQ: LGVN) announced a positive Type C meeting with the FDA regarding the pathway to BLA for Lomecel-B™ in Hypoplastic Left Heart Syndrome (HLHS). Key points include:

• The ongoing Phase 2b clinical trial (ELPIS II) may be deemed pivotal for BLA submission
• Alignment reached on primary and secondary endpoints for ELPIS II
• Agreement on Chemistry, Manufacturing and Controls (CMC) and Potency Assay plans
• ELPIS II builds on positive results from ELPIS I, which showed 100% transplant-free survival up to five years
• Lomecel-B™ has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations

The company views this as a significant step towards potential approval of Lomecel-B™ as an HLHS adjunct therapy.

Positive

• FDA alignment on ELPIS II as potentially pivotal for BLA submission
• Agreement on primary and secondary endpoints for ELPIS II
• Potential for accelerated approval pathway
• Positive results from ELPIS I with 100% transplant-free survival up to five years
• Multiple FDA designations (Orphan Drug, Fast Track, Rare Pediatric Disease) for Lomecel-B™
• Potential eligibility for Priority Review Voucher upon FDA approval

Negative

• Conditional requirements still to be met for ELPIS II to be deemed pivotal
• Need to submit prespecified Statistical Analysis Plan and CMC readiness plan for FDA review

Insights

Biotechnology Analyst

This is a significant development for Longeveron's Lomecel-B™ program. The FDA's agreement that the ELPIS II trial could be pivotal for a BLA submission is a major milestone. It potentially accelerates the path to market for Lomecel-B™ in HLHS treatment, reducing time and costs associated with additional clinical trials.

The alignment on endpoints and CMC requirements provides clarity on regulatory expectations, which is important for biotech companies. The 100% transplant-free survival rate observed in ELPIS I is remarkably promising, especially compared to the 20% historical mortality rate. However, investors should note that Phase 2b results are still pending and success is not guaranteed.

The multiple FDA designations (Orphan Drug, Fast Track, Rare Pediatric Disease) highlight the unmet medical need in HLHS and could provide significant market advantages if approved, including extended market exclusivity and a potential Priority Review Voucher, which could be valuable for future pipeline development or monetization.

Financial Analyst

This news is likely to have a positive impact on Longeveron's stock in the short term. The potential for ELPIS II to serve as a pivotal trial for BLA submission significantly de-risks the development pathway for Lomecel-B™ in HLHS. This could lead to faster time-to-market and reduced R&D expenses, potentially improving the company's financial outlook.

Investors should consider the market potential for HLHS treatment. While it's a rare condition, orphan drugs often command premium pricing. The possibility of obtaining a Priority Review Voucher is also financially significant, as these have been sold for $100 million to $350 million in recent years.

However, it's important to note that Longeveron is still a clinical-stage company with no approved products. The success of ELPIS II and subsequent regulatory approval are not guaranteed. Investors should carefully consider the company's cash position and burn rate to ensure it can fund the trial to completion and potential commercialization.

Pediatric Cardiologist

The potential of Lomecel-B™ in HLHS treatment is extremely promising from a medical perspective. HLHS is a severe congenital heart defect with treatment options and high mortality rates. The 100% transplant-free survival rate observed in ELPIS I, if replicated in ELPIS II, would represent a paradigm shift in HLHS management.

The cellular therapy approach of Lomecel-B™ is innovative, potentially addressing the underlying cause rather than just managing symptoms. This could lead to improved long-term outcomes and quality of life for HLHS patients. The collaboration with NHLBI adds credibility to the research.

However, it's important to note that Phase 2b results are still pending. While the FDA's feedback is encouraging, the efficacy and safety profile in a larger patient population need to be demonstrated. The medical community will be closely watching for data on factors such as ventricular function, growth and long-term complications. If successful, Lomecel-B™ could become a critical component of HLHS treatment protocols.

• On-going Phase 2b clinical trial (ELPIS II) deemed pivotal and, if positive, acceptable for Biological License Application (BLA) submission for potential full traditional approval
• Alignment with FDA on ELPIS II primary and secondary endpoints
• Alignment with FDA on CMC and Potency Assay plan and requirements
  

MIAMI, Sept. 03, 2024 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN), a clinical stage regenerative medicine biotechnology company developing cellular therapies for life-threatening and chronic aging-related conditions, today announced the positive Type C meeting with the U.S. Food and Drug Administration (FDA), supporting the advancement of Lomecel-B™, a proprietary, scalable, allogeneic, investigational cellular therapy currently being evaluated in a Phase 2b clinical trial (ELPIS II) for hypoplastic left heart syndrome (HLHS).

The Company and the FDA reached foundational alignment on the primary endpoint and secondary endpoints for ELPIS II. The FDA confirmed that, with several conditional requirements to be accomplished, ELPIS II may be deemed pivotal, and, if positive, acceptable for Biological License Application (BLA) submission for full traditional approval. Among other items, the Company will need to submit its prespecified Statistical Analysis Plan (SAP) and Chemistry, Manufacturing and Controls (CMC) readiness plan, including Lomecel-B™ stability and comparability data, to the FDA for prior review.

“We are pleased to have alignment with FDA on the development pathway for our Lomecel-B™ development program in HLHS, which has a devastating impact on patients and their families,” said Wa’el Hashad, Chief Executive Officer of Longeveron. “While we have a lot of work yet to do, the potential for ELPIS II to serve as the foundation for a BLA submission significantly reduces the time to reach submission and potential approval of Lomecel-B™ as an HLHS adjunct therapy.”

ELPIS II builds on the positive clinical results of ELPIS I, in which children in the trial experienced 100% transplant-free survival up to five years of age after receiving Lomecel-B™ compared to an approximate 20% mortality rate observed from historical control data.

ELPIS II is being conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) through grants from the National Institutes of Health (NIH).

The Lomecel-B™ HLHS program has received three FDA designations: Orphan Drug designation, Fast Track designation and Rare Pediatric Disease designation. Under the Rare Pediatric Disease designation, if Longeveron were to receive FDA marketing approval for Lomecel-B™ for HLHS, the Company could be eligible to receive a Priority Review Voucher.

About Lomecel-B™
Lomecel-B™ is an allogeneic, investigational product made from specialized cells isolated from the bone marrow of young healthy adult donors. These specialized cells, known as medicinal signaling cells (MSCs), are essential to the endogenous biological repair mechanism. MSCs have shown high promise for cardiac regenerative therapy (Kaushal and Wehman 2015; Wehman et al. 2016). They also have been shown to respond to sites of injury or disease and secrete bioactive factors that are anti-inflammatory and regenerative. Longeveron believes that Lomecel-B™ may have multiple potential mechanisms of action that may lead to anti-inflammatory, pro-vascular regenerative responses, and therefore may have broad application for a range of rare and aging related diseases.

About Hypoplastic Left Heart Syndrome (HLHS)
HLHS is a rare congenital heart defect that effects approximately 1,000 infants per year in the U.S. Infants 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. The current treatment requires infants to undergo a complex three-stage heart reconstruction surgery process over the first five years of their life. 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.

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. Lomecel-B™ development programs have received five distinct and important U.S. FDA designations: for the HLHS program - Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation; and, for the AD program - Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation. 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 an effective treatment for hypoplastic left heart syndrome or achieve U.S. FDA approval under the Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation programs, or any approval. 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, market and other conditions, our limited operating history and lack of products approved for commercial sale; 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 allogeneic, cell-based therapies for Aging-related Frailty, Alzheimer’s Disease, or other aging-related conditions, or 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; intellectual property rights do not necessarily address all potential threats to our competitive advantage; 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; the volatility of price of our Class A common stock; we could lose our listing on the Nasdaq Capital Market; 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 and Media Contact:
Derek Cole
Investor Relations Advisory Solutions
derek.cole@iradvisory.com

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/cfb4911d-2b0b-4c52-b2fa-b6982947155d

FAQ

What did the FDA meeting reveal about Longeveron's Lomecel-B™ for HLHS?

The FDA meeting revealed that Longeveron's ongoing Phase 2b clinical trial (ELPIS II) for Lomecel-B™ in HLHS may be deemed pivotal and, if positive, acceptable for BLA submission for potential full traditional approval, subject to certain conditions.

What are the key FDA designations for Longeveron's Lomecel-B™ HLHS program?

Longeveron's Lomecel-B™ HLHS program has received three FDA designations: Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation.

What were the results of the ELPIS I trial for Lomecel-B™ in HLHS patients?

In the ELPIS I trial, children who received Lomecel-B™ experienced 100% transplant-free survival up to five years of age, compared to an approximate 20% mortality rate observed from historical control data.

What is the potential benefit for Longeveron (LGVN) if Lomecel-B™ receives FDA approval for HLHS?

If Longeveron (LGVN) receives FDA marketing approval for Lomecel-B™ for HLHS, the company could be eligible to receive a Priority Review Voucher under the Rare Pediatric Disease designation.