U.S. Food and Drug
Administration Approves Longeveron's Lomecel-B for Rare Pediatric
Disease Designation to Treat Life-Threatening Infant Heart
Condition
Phase 2 clinical trial
underway for Hypoplastic Left Heart Syndrome, which affects
approximately 1,000 babies per year
Miami, Florida—November
18th,
2021 -- InvestorsHub NewsWire -- 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 Payne
Stern Investor Relations
Tel: (212) 362-1200
Email:
Brendan.payne@sternir.com
Source: Longeveron Inc.
Source: LGVN
Source: Longeveron