Longeveron® Announces U.S. FDA Grants Fast Track Designation for Lomecel-B™ for the Treatment of Mild Alzheimer’s Disease
Longeveron (NASDAQ: LGVN) has received Fast Track designation from the FDA for Lomecel-B™, its investigational cellular therapy for mild Alzheimer's Disease. This follows the recent Regenerative Medicine Advanced Therapy (RMAT) designation granted on July 9, 2024. The Fast Track status aims to expedite development and review of Lomecel-B™, recognizing its potential to address unmet medical needs in Alzheimer's treatment.
Lomecel-B™ demonstrated promising results in the CLEAR MIND Phase 2a clinical trial, showing an overall slowing/prevention of disease worsening compared to placebo. Full results from this trial will be presented at the upcoming Alzheimer's Association International Conference® (AAIC) in July 2024, including a Featured Research Session oral presentation and a poster presentation.
- FDA granted Fast Track designation for Lomecel-B™ in mild Alzheimer's Disease treatment
- Received Regenerative Medicine Advanced Therapy (RMAT) designation on July 9, 2024
- Lomecel-B™ showed overall slowing/prevention of disease worsening in Phase 2a trial
- Phase 2a results selected for Featured Research Session at AAIC 2024
- Potential for expedited development and regulatory review timelines
- None.
Insights
The FDA's Fast Track designation for Lomecel-B™ in treating mild Alzheimer’s Disease signifies a substantial step forward in the therapeutic landscape for neurodegenerative diseases. This designation is designed to expedite the development and review processes for treatments that address serious conditions with unmet needs. Consequently, this could significantly reduce the timeline for Lomecel-B™ to reach the market, providing earlier access to patients and potentially setting a new standard for Alzheimer’s treatment.
From a clinical perspective, the Fast Track status, alongside the previously granted RMAT designation, reflects promising preliminary results from the Phase 2a trial, which showed an overall slowing or prevention of disease progression. This is critical, as current treatments are limited in their efficacy at halting the progression of Alzheimer’s disease.
In the short term, this designation can bolster investor confidence in Longeveron’s research and development capabilities, potentially leading to a positive market response. Long-term, if subsequent trials continue to demonstrate positive outcomes, Lomecel-B™ could significantly alter Alzheimer’s treatment paradigms and capture a substantial market share in a field desperate for effective therapies.
Industry-specific terms:Fast Track designation: A process designed by the FDA to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Regenerative Medicine Advanced Therapy (RMAT) designation: A special status granted by the FDA to expedite the development and review of regenerative medicine therapies intended to treat serious conditions.
The announcement of FDA's Fast Track designation for Lomecel-B™ is a pivotal development for Longeveron. This designation often leads to reduced regulatory uncertainty and faster time-to-market, key elements that can significantly influence a company's financial health. The market typically responds favorably to such news and we can anticipate a potential rise in Longeveron’s stock price due to this announcement.
Notably, Longeveron is also progressing with other clinical indications for Lomecel-B™, such as Aging-related Frailty and hypoplastic left heart syndrome (HLHS), which broadens their potential revenue streams. If successful, Lomecel-B™ could diversify the company's portfolio and mitigate risks associated with reliance on a single therapeutic area.
In the short-term, investors might see an uptick in stock volatility as the market digests the news. Over the long-term, if subsequent trial phases yield positive results, Longeveron could secure substantial market share and revenue growth, enhancing shareholder value.
Key data to consider: Stock Ticker: NASDAQ: LGVN. Phase 2a trial results: Demonstrated significant slowing/prevention of disease progression.
– FDA designation enables expedited clinical development and regulatory review timelines for Lomecel-B™ –
– Second designation received for Lomecel-B™ for the treatment of mild Alzheimer’s Disease after Regenerative Medicine Advanced Therapeutics (RMAT) Designation announced on July 9, 2024 –
– Phase 2a Lomecel-B™ data in mild Alzheimer’s Disease selected for Featured Research Session oral presentation and poster presentation at upcoming Alzheimer’s Association International Conference® (AAIC) –
MIAMI, July 17, 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 that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Lomecel-B™ for the treatment of mild Alzheimer’s Disease. Lomecel-B™ is a proprietary, scalable, allogeneic, investigational cellular therapy being evaluated across multiple indications, including Alzheimer’s Disease (Phase 2a completed), Aging-related Frailty (Phase 2b completed) and hypoplastic left heart syndrome (HLHS) (Phase 2b ongoing).
“Fast Track designation is another important milestone for Longeveron and Lomecel-B™, which, along with the recent granting of Regenerative Medicine Advanced Therapy (RMAT) designation, recognizes the critical need to quickly advance novel, safe and effective investigational treatments for Alzheimer’s Disease, which has a devastating impact on patients and their families,” said Wa’el Hashad, Chief Executive Officer of Longeveron. “Alzheimer’s Disease is a neurodegenerative disorder that leads to progressive memory loss and death, and its tremendous impact on our aging population is exacerbated by a lack of therapeutic options that slow disease progression or improve cognitive function. We believe that Lomecel-B™, which demonstrated an overall slowing/prevention of disease worsening compared to placebo in the CLEAR MIND Phase 2a clinical trial, has the potential to become an important treatment option for physicians and patients, and we look forward to sharing the latest data with the Alzheimer’s Disease research and patient communities at AAIC 2024.”
The FDA’s Fast Track designation process is designed to facilitate the development and expedite the review of new therapies intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Investigational therapies that receive Fast Track designation may be eligible for Priority Review if relevant criteria are met at the time of application submission to FDA, and rolling FDA review.
The Fast Track designation granted to the Lomecel-B™ Alzheimer’s Disease program is the fifth special regulatory designation received for Lomecel-B™. The Lomecel-B™ Alzheimer’s Disease program has also been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA, while the Lomecel-B™ HLHS program has received three FDA designations: Orphan Drug designation, Fast Track designation and Rare Pediatric Disease designation.
Upcoming Presentation at the Alzheimer’s Association International Conference® (AAIC)
The Fast Track designation and continued clinical development of Lomecel-B™ are based upon positive topline data reported from the Phase 2a CLEAR MIND clinical trial. Full results from the CLEAR MIND clinical trial evaluating Lomecel-B™ in mild Alzheimer’s Disease have been selected for a Featured Research Session oral presentation and poster presentation at AAIC, taking place July 28 – August 1, 2024, in Philadelphia and online. Longeveron Co-Founder, Chief Scientific Officer and Chairman Joshua M. Hare, MD, FACC, FAHA, was invited to chair this Featured Research Session.
Details of the presentations at AAIC are as follows:
Oral Presentation
Date: Sunday, July 28, 2024
Time: 4:15 pm – 5:45pm EDT
Session: Featured Research Session, 1-32-FRS-B
Title: Results from a Phase 2a Proof-of-Concept Double-Blind, Randomized, Placebo-Controlled Trial of Lomecel-B™ in Mild Alzheimer’s Disease Dementia
Poster Presentation
Date: Tuesday, July 30, 2024
Time: 7:30am – 4:15pm EDT
Session: In-Person Posters Tuesday; Drug Development
Topic: MRI Imaging results in the CLEAR MIND phase 2a clinical trial of Lomecel-B™
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 investigational 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 separate and distinct FDA designations: for the HLHS program - Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation; and, for the Alzheimer’s Disease 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 mild Alzheimer’s Disease or achieve U.S. FDA approval under the Fast Track designation program. 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 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 Contact:
Derek Cole
Investor Relations Advisory Solutions
derek.cole@iradvisory.com
Media Contact:
Andrea Cohen
Sam Brown Inc. Healthcare Communications
andreacohen@sambrown.com
(917) 209-7163
A photo accompanying this announcement is available at:
https://www.globenewswire.com/NewsRoom/AttachmentNg/cfb4911d-2b0b-4c52-b2fa-b6982947155d
FAQ
What is the latest FDA designation received by Longeveron (LGVN) for Lomecel-B™?
What were the results of the CLEAR MIND Phase 2a clinical trial for Lomecel-B™ in Alzheimer's Disease?
When and where will Longeveron (LGVN) present the full results of the CLEAR MIND Phase 2a trial?
How many special regulatory designations has Lomecel-B™ received from the FDA?
