Longeveron Inc. (NASDAQ: LGVN) ("Longeveron" or "Company"), a
clinical stage biotechnology company developing cellular therapies
for chronic aging-related and life-threatening conditions,
announced today that the U.S. Food and Drug Administration (FDA)
has granted Rare Pediatric Disease (RPD) designation for Lomecel-B
for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare
and life-threatening congenital heart defect in infants. Lomecel-B,
an investigational allogeneic, bone marrow-derived medicinal
signaling cell (MSC) product, is currently being evaluated in a
Phase 2 trial.
“We are pleased by FDA’s acknowledgment of the
urgent need for a safe and effective treatment for children born
with this rare and devastating congenital heart defect,” stated
Longeveron co-founder and Chief Science Officer Joshua M. Hare,
M.D. “We are encouraged by our Phase 1 clinical data, and the
progress being made in the ongoing Phase 2 trial. Lomecel-B
represents a unique cell therapy approach that could potentially be
administered at the same time as surgery in these critically
impacted infants.”
Approximately 1,000 babies are born with HLHS
each year in the U.S. HLHS babies have an underdeveloped left
ventricle, which impairs the heart’s ability to pump blood
throughout the body. HLHS is often fatal without surgical
intervention, in which three surgical procedures are performed over
the period of about 5 years, to allow the right ventricle to be
configured to pump blood to the body. Longeveron is evaluating the
safety of Lomecel-B injection into the right ventricle during the
second surgery (4 – 6 months of age), and the effect on cardiac
function and other health status endpoints.
Longeveron recently reported clinical results
from its safety-focused Phase I clinical study of Lomecel-B in HLHS
patients. When cardiac surgeons injected Lomecel-B directly into
the babies’ hearts at the time of surgery, the cells were well
tolerated with no major adverse cardiac events and no infections
considered to be related to the investigational treatment.
One hundred percent of infants enrolled in the
Phase 1 trial (n=10) were alive and had not required a transplant
between 2 – 3.5 years post-surgery. Other measurements of the
babies’ health, such as weight gain and growth pattern, matched
that of normal healthy babies.
The FDA may grant RPD designation for diseases
that primarily affect children ages 18 years old or younger, and
fewer than 200,000 individuals in the U.S. Under this program, if
the FDA approves Lomecel-B for the treatment of HLHS, Longeveron
may be eligible to receive a priority review voucher (PRV) if the
application submitted for the product satisfies certain conditions,
and under current law, is approved prior to September 30,
2026. Drug companies receiving a PRV can have any subsequent drug
or biologic application reviewed and a decision made in six months
as opposed to the standard 10 months, potentially accelerating the
time to market, or they can sell the PRV to another company. The
PRV accelerated review is for patients with serious conditions
providing access to a potentially lifesaving or -changing treatment
sooner. For companies, it means they can potentially go to market
with their product more quickly, and potentially begin generating
product revenue.
Infants with HLHS are currently being enrolled
in ELPIS II, a 38-subject, phase 2 randomized, double-blind,
controlled clinical trial. ELPIS II is being funded in part by a
grant from the National Institute of Health’s National Heart, Lung,
and Blood Institute (NHLBI; Grant number 1UG3HL148318), in
collaboration with Longeveron, and is led by Principal Investigator
Sunjay Kaushal, MD, PhD, Division Head, Cardiovascular-Thoracic
Surgery, Ann and Robert H. Lurie Children’s Hospital of Chicago.
Other participating clinical sites currently open for enrollment
are Advocate Children’s Hospital, Park Ridge, IL; Primary
Children’s Hospital/University of Utah, Salt Lake City, UT;
Children’s Hospital Los Angeles, Los Angeles, CA; and Cincinnati
Children’s Hospital Medical Center, Cincinnati, OH.
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 (“Lomecel-B”),
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: Aging Frailty,
Alzheimer’s disease, the Metabolic Syndrome, Acute Respiratory
Distress Syndrome (ARDS), and hypoplastic left heart syndrome
(HLHS). The Company’s mission is to advance Lomecel-B and other
cell-based product candidates into pivotal Phase 3 trials, with the
goal of achieving regulatory approvals, subsequent
commercialization and broad use by the healthcare community.
Additional information about the Company is available at
www.longeveron.com.
Cautionary Note Regarding
Forward-Looking Statements
Certain statements in this press release that
are not historical facts are forward-looking statements that
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. Moreover,
forward-looking statements in this release include, but are not
limited to, statements about 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; the size of the
market opportunity for our product candidates, the beneficial
characteristics, safety, efficacy and therapeutic effects of our
product candidates; our ability to obtain and maintain regulatory
approval of our product candidates, 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. 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 our Quarterly Report on Form 10-Q for the period ended
September 30, 2021. 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.
Contact:Brendan PayneStern Investor
RelationsTel: (212) 362-1200Email: Brendan.payne@sternir.com
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