Full Press Release Details
Longeveron Announces U.S. FDA Approval
of IND Application for a Phase 2 Pivotal Registration Study
Evaluating Laromestrocel as a Treatment of Pediatric Dilated Cardiomyopathy
July 8, 2025 -- 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 approved the Investigational New Drug (IND) application for its stem cell therapy laromestrocel as a potential treatment for pediatric
dilated cardiomyopathy (DCM). The accepted IND application provides for moving directly to a single Phase 2 pivotal registration clinical
"We are gratified to advance laromestrocel
into clinical development for pediatric dilated cardiomyopathy, a rare, progressive, life-threatening disease with no current effective
treatment," said Nataliya Agafonova, M.D., Chief Medical Officer of Longeveron. "With nearly 40% of children with DCM requiring
a heart transplant or dying within two years of diagnosis, there is a tremendous need for novel approaches to treat this pediatric cardiovascular
disease. Our development program in DCM reinforces Longeveron's commitment to developing innovative stem cell therapies for rare
diseases, particularly for cardiovascular conditions, where we believe laromestrocel may have significant potential to improve patients'
Laromestrocel is a proprietary, scalable, allogeneic,
investigational cellular therapy derived from mesenchymal stem cells (MSCs) with 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
"The FDA approval of this laromestrocel
IND is an important milestone for pediatric cardiovascular treatment," said Barry Byrne, M.D., Ph.D, Associate Chair of Pediatrics
and Director of the Powell Gene Therapy Center at the University of Florida. "Current treatment for DCM focuses on managing symptoms,
improving heart function, and preventing complications rather than addressing the underlying cause or causes. Many therapeutic agents
with known efficacy in adults lack the same evidence in children. Longeveron's innovative stem cell therapy approach, with the possibility
for stem cells to repair damaged heart tissue, is a potential groundbreaking development in the treatment of children with cardiovascular
DCM occurs when the muscles in one of more of
the heart chambers become enlarged or stretched (dilated). The other chambers of the heart need to work harder to compensate for the affected
chambers, and may also become dilated and enlarged. As the condition progresses, the heart becomes weaker, and it becomes more difficult
to pump blood through the body. This can lead to congestive heart failure causing a build-up of fluid in the lungs, liver, abdomen, and
lower legs. In the majority of cases, the exact cause of DCM cannot be determined (idiopathic cardiomyopathy).
DCM is the most common form of cardiomyopathy
in children. About 50 to 60 percent of all pediatric cardiomyopathy cases are diagnosed as dilated. According to the Pediatric Cardiomyopathy
Registry, DCM is reportedly more common in boys than girls. Although all age groups are affected, studies show that DCM is more common
in infants (before age 1) than in older children.
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 laromestrocel
(Lomecel-B ), an allogeneic mesenchymal stem cell (MSC) therapy product isolated from the bone marrow of young, healthy adult donors.
Laromestrocel 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. Laromestrocel 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
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are not historical facts are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation
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materially from those expressed or implied in any forward-looking statements in this release include, but are not limited to, our cash
position and need to raise additional capital, the difficulties we may face in obtaining access to capital, and the dilutive impact it
may have on our investors; our financial performance, and ability to continue as a going concern; the period over which we estimate our
existing cash and cash equivalents will be sufficient to fund our future operating expenses and capital expenditure requirements; the
ability of our clinical trials to demonstrate safety and efficacy of our product candidates, and other positive results; the timing and
focus of our ongoing and future preclinical studies and clinical trials, and the reporting of data from those studies and trials; the
size of the market opportunity for certain of our product candidates, including our estimates of the number of patients who suffer from
the diseases we are targeting; our ability to scale production and commercialize the product candidate for certain indications; the success
of competing therapies that are or may become available; the beneficial characteristics, safety, efficacy and therapeutic effects of our
product candidates; our ability to obtain and maintain regulatory approval of our product candidates in the U.S. and other jurisdictions;
our plans relating to the further development of our product candidates, including additional disease states or indications we may pursue;
our plans and ability to obtain or protect intellectual property rights, including extensions of existing patent terms where available
and our ability to avoid infringing the intellectual property rights of others; the need to hire additional personnel and our ability
to attract and retain such personnel; and our estimates regarding expenses, future revenue, capital requirements and needs for additional
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, 2024, filed with the Securities
and Exchange Commission on February 28, 2025, its Quarterly Reports on Form 10-Q, and its Current Reports on Form 8-K. The Company operates
in highly competitive and rapidly changing environment; therefore, new factors may arise, and it is not possible for the Company's
management to predict all such factors that may arise nor assess the impact of such factors or the extent to which any individual factor
or combination thereof, may cause results to differ materially from those contained in any forward-looking statements. The forward-looking
statements contained in this press release are made as of the date of this press release based on information available as of the date
of this press release, are inherently uncertain, 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.
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