U.S. Food and Drug
Administration (FDA) Grants Fast Track Designation for Longeveron’s
Lomecel-B™ Product for Treatment of Hypoplastic Left Heart Syndrome
(HLHS) in Infants
New
designation may expedite FDA review and potential approval to address
this life-threatening heart condition affecting approximately 1,000
babies per year
MIAMI, Aug. 31, 2022 (GLOBE NEWSWIRE) -- Longeveron
Inc. (NASDAQ: LGVN) ("Longeveron" or "Company"), a clinical stage
biotechnology company developing cellular therapies for chronic
aging-related and life-threatening conditions, today announced the U.S.
Food and Drug Administration (FDA) has granted Fast Track Designation to
Lomecel-B™ for the treatment of Hypoplastic Left Heart
Syndrome (HLHS), a rare and life-threatening congenital heart defect
affecting approximately 1,000 infants per year. Lomecel-B™,
an investigational allogeneic, bone marrow-derived medicinal signaling
cell (MSC) product, is currently in a Phase 2a trial for HLHS.
Fast
Track Designation is intended to facilitate development and expedite
the review of drugs that treat serious conditions and fill an unmet
medical need so a product can potentially be approved and reach patients
more quickly. Fast Track Designation enables the company to have more
frequent interactions with the FDA throughout the drug development
process and allows for eligibility for priority review and accelerated
approval if certain criteria are met, as well as a rolling review. The
Fast Track Designation must continue to be met or FDA can withdraw the
designation. The FDA previously granted Longeveron’s Lomecel-B™ Orphan Drug and Rare Pediatric Disease designations in November of 2021 for HLHS.
“Fast Track Designation represents a significant milestone in our efforts to develop Lomecel-B™
as a treatment for infants with HLHS,” said Chris Min, M.D., Ph.D.,
Longeveron’s Interim Chief Executive Officer and Chief Medical Officer.
“Fast Track Designation underscores the urgent need in HLHS, and we look
forward to continuing to work closely with the FDA to bring this
potential new therapy to infants as expeditiously as possible.”
HLHS
is a rare congenital heart defect that affects approximately 1,000
babies per year in the U.S. Infants born with HLHS have an
underdeveloped or absent left ventricle, which impairs the heart’s
ability to pump adequate amounts of blood throughout the body. Patients
require three reconstructive heart surgeries within the first five years
of life, and many require a heart transplant. Without treatment, the
condition is always fatal. Even with reconstructive surgical
interventions, HLHS is still associated with high mortality. Overall
survival from birth to adolescence is estimated to be 50% to 60%.
Longeveron is currently evaluating Lomecel-B™
in ELPIS II, an ongoing 38-patient, randomized (1:1), blinded,
controlled Phase 2a clinical trial intended to evaluate the safety and
efficacy of intramyocardial (directly into the heart) injection of the
Lomecel-B™ product in infants with HLHS who are
undergoing Stage II reconstructive cardiac surgery. In ELPIS, a Phase 1
open-label trial in ten HLHS patients, Lomecel-B™ was found to be well tolerated with no major adverse cardiac events one-year post-surgery, and/or treatment-related infections.
One
hundred percent of the infants enrolled in the ELPIS Phase 1 trial
(n=10) were alive and had not required a transplant between 2-3.5 years
post-surgery. Normally, approximately 20% of patients require heart
transplants to survive within one year of Stage II reconstructive
surgery. Longeveron recently announced the release of a manuscript
entitled “Intramyocardial
cell-based therapy during bidirectional cavopulmonary anastomosis for
hypoplastic left heart syndrome: The ELPIS phase I trial” on
MedRxiv, a preprint server that posts papers before they are peer
reviewed. The paper will now undergo peer review prior to publication of
the final study report.
About Longeveron Inc.
Longeveron
is a clinical stage biotechnology company developing cellular therapies
for specific aging-related and life-threatening conditions. The
Company’s lead investigational product is the Lomecel-B™ cell-based
therapy product, which is derived from culture-expanded medicinal
signaling cells (MSCs) that are sourced from bone marrow of young,
healthy adult donors. Longeveron believes that by using the same cells
that promote tissue repair, organ maintenance, and immune system
function, it can develop safe and effective therapies for some of the
most difficult disorders associated with the aging process and other
medical disorders. Longeveron is currently sponsoring Phase 1 and 2
clinical trials in the following indications: Alzheimer’s disease,
hypoplastic left heart syndrome (HLHS), Aging Frailty, and Acute
Respiratory Distress Syndrome (ARDS). Additional information about the
Company is available at www.longeveron.com.
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
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. 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, statements about the ability of Longeveron’s clinical trials
to demonstrate safety and efficacy of the Company’s product candidates,
and other positive results; the timing and focus of the Company’s
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 the Company’s product candidates, including its
estimates of the number of patients who suffer from the diseases being
targeted; the success of competing therapies that are or may become
available; the beneficial characteristics, safety, efficacy and
therapeutic effects of the Company’s product candidates; the Company’s
ability to obtain and maintain regulatory approval of its product
candidates; the Company’s plans relating to the further development of
its product candidates, including additional disease states or
indications it may pursue; existing regulations and regulatory
developments in the U.S., Japan and other jurisdictions; the Company’s
plans and ability to obtain or protect intellectual property rights,
including extensions of existing patent terms where available and its
ability to avoid infringing the intellectual property rights of others;
the need to hire additional personnel and the Company’s ability to
attract and retain such personnel; the Company’s estimates regarding
expenses, future revenue, capital requirements and needs for additional
financing; the Company’s need to raise additional capital, and the
difficulties it may face in obtaining access to capital, and the
dilutive impact it may have on its investors; the Company’s financial
performance, and the period over which it estimates its existing cash
and cash equivalents will be sufficient to fund its future operating
expenses and capital expenditures requirements. 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, 2021, filed with the
SEC on March 11, 2022, and the Company’s Quarterly Reports on Form 10-Q
for the periods ended March 31, and June 30, 2022. 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.
BioFlorida is the voice of Florida's life sciences industry, representing 8,600 establishments and research organizations in the BioPharma, MedTech, Digital Health and Health Systems that collectively employ nearly 107,000 Floridians. Source: TEConomy/BIO (released 2022)
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