FDA aligns with Longeveron on Alzheimer’s therapy trial

Published 20/03/2025, 14:06
FDA aligns with Longeveron on Alzheimer’s therapy trial

MIAMI - Longeveron Inc. (NASDAQ: LGVN), a biotechnology firm specializing in regenerative medicine with a current market capitalization of $24.63 million, has reached a consensus with the U.S. Food and Drug Administration (FDA) on the pivotal Phase 2/3 clinical trial design for its Alzheimer’s disease treatment, laromestrocel (Lomecel-B™). This agreement, announced today, paves the way for a streamlined development process for the potential therapy. According to InvestingPro data, the company has demonstrated impressive revenue growth of 237% in the last twelve months, reflecting strong commercial progress in its development programs.

The FDA has agreed to consider a Biological License Application (BLA) for laromestrocel based on positive results from the upcoming single, adaptive Phase 2/3 trial. The trial is set to assess the efficacy and safety of the treatment in patients with mild Alzheimer’s disease, focusing on cognitive function and quality of life as primary endpoints.

Laromestrocel, derived from mesenchymal stem cells (MSCs) sourced from young, healthy adult donors, has previously demonstrated a favorable safety profile and potential efficacy in two earlier clinical trials. The Phase 2a CLEAR-MIND study, in particular, indicated a reduction in brain atrophy without amyloid-related imaging abnormalities (ARIA), findings that contributed to the FDA granting laromestrocel Regenerative Medicine Advanced Therapy (RMAT) designation.

The RMAT designation facilitates more frequent interactions with the FDA and provides guidance on drug development. It also opens the door for accelerated approval and priority review, distinguishing laromestrocel as the first cellular therapeutic candidate for Alzheimer’s disease to receive this status.

The initiation of the Phase 2/3 trial is anticipated in the second half of 2026, contingent on securing non-dilutive funding or partnership support. InvestingPro analysis reveals that while the company maintains a healthy current ratio of 7.6 and holds more cash than debt on its balance sheet, it is currently burning through cash rapidly - a crucial consideration for its development timeline. Alzheimer’s disease, characterized by progressive memory loss and cognitive decline, remains a condition with limited treatment options, affecting millions and imposing significant societal and economic burdens.

Longeveron’s Chief Medical Officer, Nataliya Agafonova, expressed optimism about the potential of laromestrocel to address the underlying pathology of Alzheimer’s disease through its anti-inflammatory and regenerative properties.

Investors may note that the advancement of laromestrocel into later-stage clinical testing is a significant milestone for Longeveron. However, the company’s future steps depend on additional funding or partnerships.

This report is based on a press release statement from Longeveron.

In other recent news, Longeveron Inc. reported a notable increase in revenue for 2024, reaching $2.4 million, which marks a 237% rise from the previous year. This surge in revenue was primarily driven by contract manufacturing, contributing $1 million, while the company also managed to reduce its operating expenses by 13%, resulting in a decreased net loss of approximately $16 million. These financial results have been accompanied by significant developments in Longeveron’s Alzheimer’s program, where the Phase 2a clinical trial of laromestrocel (Lomecel-B™) showed promising results in treating mild Alzheimer’s disease. The trial indicated improvements in cognitive function and quality of life, with the therapy being well-tolerated by patients.

Longeveron is preparing for further advancements in its Alzheimer’s and hypoplastic left heart syndrome (HLHS) programs, with plans to complete enrollment for its HLHS Phase 2b trial by the second quarter of 2025. The company anticipates submitting a Biologics License Application (BLA) for its Alzheimer’s program in 2026, which has already received RMAT and Fast Track designations from the FDA. Additionally, Longeveron has secured five FDA designations for its programs, enhancing its regulatory pathway. The company’s recent financial performance and strategic focus on advancing its cellular therapy pipeline have drawn positive attention from investors.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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