Longeveron® Announces World Health Organization Approval of “laromestrocel” as International Non-proprietary Name for Stem Cell Therapy Lomecel-B™

Rhea-AI Summary

Longeveron (NASDAQ: LGVN) announced that its cellular therapy Lomecel-B™ has received the International Non-proprietary Name (INN) 'laromestrocel' from the World Health Organization. Lomecel-B™ is being evaluated for Alzheimer's disease and hypoplastic left heart syndrome (HLHS), a rare pediatric disease.

The therapy has received multiple FDA designations, including Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designation for mild Alzheimer's disease, as well as Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation for HLHS treatment. The company has reported positive Phase 2a data in mild Alzheimer's disease and is nearing completion of enrollment for its Phase 2b HLHS clinical trial.

Positive

• Received WHO's International Non-proprietary Name (INN) approval, advancing commercialization prospects
• Holds multiple FDA designations including RMAT, Fast Track, and Orphan Drug status
• Reported positive Phase 2a results in Alzheimer's disease
• Phase 2b HLHS trial enrollment nearing completion

Negative

• None.

Insights

Biotechnology Research Analyst

The WHO's approval of 'laromestrocel' as the International Non-proprietary Name for Lomecel-B™ marks a important advancement in Longeveron's commercialization strategy. This milestone carries significant implications beyond mere nomenclature - it represents international recognition and standardization of the therapy's identity, facilitating global regulatory processes and market access preparations.

The dual-indication strategy targeting both Alzheimer's disease and hypoplastic left heart syndrome (HLHS) positions Longeveron uniquely in the regenerative medicine space. The Alzheimer's market, valued at over $3.5 billion globally, presents a substantial commercial opportunity, while the HLHS indication, though serving a smaller patient population, benefits from accelerated regulatory pathways and potential premium pricing as an orphan drug.

The accumulation of regulatory designations - RMAT and Fast Track for Alzheimer's, plus Orphan Drug and Rare Pediatric Disease designations for HLHS - demonstrates strong regulatory support and potential for expedited development. These designations typically reduce development timelines by 30-50% and increase the probability of approval.

With Phase 2a data in Alzheimer's showing positive results and Phase 2b HLHS trial nearing full enrollment, Longeveron is approaching critical value-inflection points. The INN approval particularly strengthens the company's position as it prepares for potential late-stage development and commercialization, providing a standardized identifier that will be important for global marketing applications, medical literature, and healthcare provider adoption.

• Cellular therapy Lomecel-B™ has been granted an International Non-proprietary Name (INN) of “laromestrocel” on the INN List issued by the World Health Organization (WHO)
• Lomecel-B™ is a proprietary, scalable, allogeneic cellular therapy being evaluated in Alzheimer’s disease and hypoplastic left heart syndrome (HLHS), a rare pediatric disease and orphan indication
• Lomecel-B™ first cellular therapeutic candidate to receive U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) designation for Alzheimer’s disease
  

MIAMI, Feb. 18, 2025 (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 that the International Nonproprietary Names (INN) Expert Committee of the World Health Organization approved “laromestrocel” for the non-proprietary name of the Company’s cellular therapy Lomecel-B™. Commonly referred to as a generic name, each INN is a unique name used to identify pharmaceutical substances or active pharmaceutical ingredients. Lomecel-B™ is a proprietary, scalable, allogeneic cellular therapy being evaluated in Alzheimer’s disease and hypoplastic left heart syndrome (HLHS), a rare pediatric disease and orphan indication.

“Approval of the name laromestrocel is an important step in the development and potential commercialization of Lomecel-B^TM,” said Wael Hashad, Chief Executive Officer of Longeveron. “Following on our positive Phase 2a data in mild Alzheimer’s disease and the upcoming completion of enrollment for our on-going Phase 2b clinical trial in HLHS, receiving this INN marks yet another important milestone in the advancement of Lomecel-B^TM as a potential therapeutic for patients in need of new treatment options.”

The U.S. FDA has granted Lomecel-B™ Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation for the treatment of mild Alzheimer’s disease, and Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation for the treatment of HLHS.

About Lomecel-B™ (laromestrocel)
Lomecel-B™ is a living cell 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 our endogenous biological repair mechanism. MSCs have been shown to perform a number of complex functions in the body, including the formation of new tissue. They also have been shown to respond to sites of injury or disease and secrete bioactive factors that are immunomodulatory and regenerative. We believe 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 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^TM development programs have received five distinct and important 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 anticipated use of proceeds from recent offerings. 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 allogenic, 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 the price of our Class A common stock; 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 is the new WHO-approved name for Longeveron's (LGVN) Lomecel-B therapy?

The World Health Organization has approved 'laromestrocel' as the International Non-proprietary Name (INN) for Longeveron's Lomecel-B cellular therapy.

What medical conditions is LGVN's Lomecel-B being developed to treat?

Lomecel-B is being developed to treat mild Alzheimer's disease and hypoplastic left heart syndrome (HLHS), a rare pediatric disease.

What FDA designations has LGVN's Lomecel-B received for Alzheimer's treatment?

For Alzheimer's treatment, Lomecel-B has received FDA's Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation.

What is the current clinical trial status of LGVN's Lomecel-B?

Lomecel-B has shown positive Phase 2a data in mild Alzheimer's disease, and its Phase 2b clinical trial for HLHS is approaching completion of enrollment.

What regulatory designations has LGVN's Lomecel-B received for HLHS treatment?

For HLHS treatment, Lomecel-B has received FDA's Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation.