Reneo Pharmaceuticals Receives Fast Track Designation from the FDA for Mavodelpar (REN001) in a Genotype of Long-Chain Fatty Acid Oxidation Disorder (LC-FAOD)

IRVINE, Calif., Jan. 31, 2023 (GLOBE NEWSWIRE) — 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 Spangler
Investor Relations
Reneo Pharmaceuticals, Inc.
dspangler@reneopharma.com

David Melamed, Ph.D.
Media Inquiries
Russo Partners, LLC
david.melamed@russopartnersllc.com