Reneo Pharmaceuticals Receives Fast Track Designation from the FDA for Mavodelpar (REN001) in a Genotype of Long-Chain Fatty Acid Oxidation Disorder (LC-FAOD)
January 31 2023 - 7:35AM
Reneo Pharmaceuticals, Inc. (Nasdaq: RPHM), a clinical-stage
pharmaceutical company focused on the development and
commercialization of therapies for patients with rare genetic
mitochondrial diseases, today announced that the U.S. Food and Drug
Administration (FDA) has granted mavodelpar (REN001) Fast Track
designation for long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD)
deficiency, one of the predominant genotypes in patients with
long-chain fatty acid oxidation disorder (LC-FAOD).
“Receiving this Fast Track designation for a second indication
continues to solidify mavodelpar as a potential therapeutic option
for patients with rare mitochondrial disease,” said Gregory J.
Flesher, President and Chief Executive Officer of Reneo
Pharmaceuticals. “We appreciate the continued collaboration with
the FDA and look forward to their partnership in advancing the
LC-FAOD program which will include patients with LCHAD as well as
other genotypes.”
The FDA’s Fast Track regulatory process is designed to
facilitate and expedite the development of investigational
treatments that demonstrate a potential to address unmet medical
needs in serious or life-threatening conditions. Programs with FDA
Fast Track designation can benefit from early and frequent
communication with the FDA in addition to a rolling submission of
the marketing application.
The company recently completed a natural history study (FORWARD)
and an open label study evaluating mavodelpar in patients with
LC-FAOD that included multiple genotypes, including the LCHAD
genotype. Mavodelpar is also being evaluated in a pivotal clinical
trial (STRIDE study) in patients with primary mitochondrial
myopathies (PMM) with mitochondrial DNA (mtDNA) defects. The
company expects topline data from the STRIDE study in the fourth
quarter of this year.
About Reneo Pharmaceuticals
Reneo is a clinical-stage pharmaceutical company focused on the
development and commercialization of therapies for patients with
rare genetic mitochondrial diseases, which are often associated
with the inability of mitochondria to produce adenosine
triphosphate (ATP). Our lead product candidate, mavodelpar
(REN001), is a potent and selective agonist of the peroxisome
proliferator-activated receptor delta (PPARδ). Mavodelpar has been
shown to increase transcription of genes involved in mitochondrial
function and increase fatty acid oxidation and may increase
production of new mitochondria. For additional information, please
see reneopharma.com.
About Mavodelpar
Mavodelpar (REN001) is a potent and selective peroxisome
proliferator-activated receptor delta (PPARδ) agonist currently in
clinical development for two rare genetic mitochondrial diseases
that typically present with myopathy and have high unmet medical
needs: primary mitochondrial myopathies (PMM) and long-chain fatty
acid oxidation disorders (LC-FAOD). For additional information,
please see clinicaltrials.gov.
About PMM
PMM are a group of rare, genetic metabolic disorders caused by
mutations or deletions in the mitochondrial DNA (mtDNA) or nuclear
DNA (nDNA). These genetic alterations hamper the ability of
mitochondria to generate energy from nutrient sources, resulting in
energy deficits that are most pronounced in tissues with high
energy demand such as muscle, brain, and heart. The symptoms of PMM
include muscle weakness, exercise intolerance, movement disorder,
deafness, blindness, and droopy eyelids among others. The prognosis
for these disorders ranges in severity from progressive weakness to
death.
About LC-FAOD
LC-FAOD are a group of rare, genetic metabolic disorders caused
by mutations or deletions in the nuclear DNA (nDNA). These genetic
alterations prevent the body from breaking down long-chain fatty
acids during metabolism. The most severe cases of LC-FAOD are
diagnosed within the first few days or weeks of life. Young
patients often present with a severe energy deficit that results in
lethargy, liver dysfunction, hypoglycemia, encephalopathy, and high
risk for sudden death. Older patients usually present with muscle
weakness, exercise intolerance, muscle aches, or rhabdomyolysis
which can damage the heart and kidneys and cause permanent
disability or even death.
Forward-Looking Statements
Statements contained in this press release regarding matters
that are not historical facts are “forward-looking statements”
within the meaning of the Private Securities Litigation Reform Act
of 1995. Such forward-looking statements include statements
regarding, among other things, the potential development,
registration, and commercialization of mavodelpar (REN001),
results, conduct, progress, plans and timing of Reneo’s clinical
studies, completion of enrollment of clinical trials, presentation
of data from clinical trials, meetings with regulatory agencies and
the regulatory approval path for REN001. Because such statements
are subject to risks and uncertainties, actual results may differ
materially from those expressed or implied by such forward-looking
statements. Words such as “plans,” “will,” “believes,”
“anticipates,” “expects,” “intends,” “goal,” “potential” and
similar expressions are intended to identify forward-looking
statements. These forward-looking statements are based upon Reneo’s
current expectations and involve assumptions that may never
materialize or may prove to be incorrect. Actual results could
differ materially from those anticipated in such forward-looking
statements as a result of various risks and uncertainties, which
include, without limitation, risks and uncertainties associated
with Reneo’s business in general, and the other risks described in
Reneo’s filings with the Securities and Exchange Commission. All
forward-looking statements contained in this press release speak
only as of the date on which they were made. Reneo undertakes no
obligation to update such statements to reflect events that occur
or circumstances that exist after the date on which they were made,
except as required by law.
Contacts:
Danielle SpanglerInvestor RelationsReneo Pharmaceuticals,
Inc.dspangler@reneopharma.com
David Melamed, Ph.D.Media InquiriesRusso Partners,
LLCdavid.melamed@russopartnersllc.com
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