Longeveron Granted
Orphan Drug Designation by FDA for Lomecel-B to Treat Infants with
Hypoplastic Left Heart Syndrome (HLHS)
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HLHS is a rare
congenital heart defect that affects approximately 1,000 babies in
the U.S. per year
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Orphan Drug
Designation confers certain benefits and may result in seven year
market exclusivity upon approval for this indication if all
statutory and regulatory requirements are met
-
Supplements
Rare Pediatric Disease Designation recently granted by
FDA
Miami, Florida—December
6, 2021-- InvestorsHub NewsWire
-- Longeveron Inc. (NASDAQ: LGVN) ("Longeveron" or "Company"), a clinical
stage biotechnology company developing cellular therapies for
chronic aging-related and certain life-threatening conditions,
announced today that the U.S. Food and Drug Administration (FDA)
has granted Orphan Drug Designation (ODD) for Lomecel-B for the
treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and
life-threatening congenital heart defect in infants.
ODD is intended to assist
and encourage companies to develop safe and effective therapies for
the treatment of rare diseases or conditions. ODD positions
Longeveron to be able to potentially leverage a range of financial
and regulatory benefits, including government grants for conducting
clinical trials, waiver of FDA user fees for the potential
submission of a marketing application, and certain tax
credits.
Receiving ODD may also
result in the product receiving seven years market exclusivity upon
approval for use in the rare disease or condition for which the
product was designated if all of the statutory and regulatory
requirements are met.
"Adding to the Rare Pediatric Disease (RPD) designation
already granted to Lomecel-B for treatment of HLHS, the FDA's
decision to grant ODD to Lomecel-B for this indication indicates
the ongoing and unmet need for new therapies to treat infants with
HLHS," said Geoff Green, Chief Executive Officer at Longeveron.
"Building on results from our completed Phase 1 safety-focused
trial, we believe Lomecel-B has potential to improve outcomes for
these severely impacted infants by way of repairing cardiac tissue
and improving ventricular function. The combination of both RPD and
ODD allows us to potentially move more efficiently through clinical
development and regulatory review, and Lomecel-B may be eligible
for a period of marketing exclusivity upon approval for this
indication."
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 1 clinical study of Lomecel-B in HLHS
patients. When cardiac surgeons injected Lomecel-B directly into
the babies' hearts at the time of the second 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 the babies 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. Longeveron is currently enrolling ELPIS II, a
38-subject, Phase 2 randomized, double-blind, controlled clinical
trial examining the effect of Lomecel-B in HLHS affected infants.
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.
"We are gratified to receive both rare pediatric disease and
orphan drug designations from FDA for this clinical trial program,"
stated Sunjay Kaushal, M.D, Ph.D. study Principal Investigator and
Division Head of Cardiovascular Thoracic Surgery at Lurie
Children's Hospital of Chicago. "This is a severe condition and a
significant unmet medical need, and these important designations
will be vital to facilitating a potentially more rapid development
program. My colleagues around the country and I are extremely
excited about the progress of the current trial, and we look
forward to seeing this program move through the regulatory and
clinical pathway."
About Longeveron Inc.
Longeveron is a clinical stage biotechnology company
developing cellular therapies for specific aging-related and
certain 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