October 9, 2023
Topline data results from the STRIDE study are expected in December 2023
IRVINE, Calif., Oct. 09, 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, announced last patient last visit (LPLV) in the pivotal STRIDE study of mavodelpar in PMM. Topline data results from the STRIDE study are expected in December 2023.
“This marks a major milestone for mavodelpar and the company. This achievement reached in the STRIDE program underscores the significant progress toward our corporate mission to bring therapies to patients with rare genetic mitochondrial diseases. We anticipate sharing topline results from the STRIDE clinical trial in December 2023,” said Gregory J. Flesher, Chief Executive Officer. “In addition, we are encouraged by the participation in the ongoing STRIDE AHEAD study, which has enrolled over 85% of eligible patients.”
Ashley Hall, Chief Development Officer, added “We anticipate completing the final steps in the STRIDE clinical process in the coming months. Subsequently, we plan to share the results of data analysis with the United States Food and Drug Administration (FDA) in the first quarter of 2024. We expect that the STRIDE and STRIDE AHEAD studies will form the basis of a New Drug Application (NDA) to the FDA which is planned for submission in the first half of 2024 and thereafter to additional regulatory agencies.”
The STRIDE study is a global, randomized, double-blind, placebo-controlled pivotal Phase 2b trial of mavodelpar in adult patients with PMM due to mitochondrial DNA (mtDNA) defects. The study is designed to investigate the efficacy and safety of 100 mg mavodelpar administered once-daily over a 24-week period. The primary efficacy endpoint of the trial is the change from baseline in the distance walked during the 12-minute walk test (12MWT) at week 24. Secondary endpoints include changes from baseline in PROMIS® Short Form Fatigue 13a, Modified Fatigue Impact Scale (MFIS), Patient Global Impression of Change (PGIC), Patient Global Impression of Severity (PGIS), 30 Second Sit-To-Stand (30STS) Test, Brief Pain Inventory (BPI), 36-Item Health Survey (SF-36), Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP), and Pedometer Step Count.
About STRIDE AHEAD
The STRIDE AHEAD study is an open-label extension (OLE) trial outside of the United States in patients with PMM due to mtDNA defects who participated in STRIDE or the mavodelpar Phase 1b study. The study is designed to evaluate the long-term safety and tolerability of 100 mg mavodelpar administered once-daily over a 24-month period. STRIDE AHEAD was amended to allow enrollment of treatment naïve patients with PMM due to nuclear DNA (nDNA) defects.
PMM are a group of rare, genetic metabolic disorders caused by mutations or deletions in the mtDNA or 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.
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: PMM and long-chain fatty acid oxidation disorder. For additional information, please see clinicaltrials.gov.
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, increase fatty acid oxidation, and may increase production of new mitochondria. For additional information, please see reneopharma.com.
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, the timing of topline data from the STRIDE study, the timing of the final steps in the clinical process for the STRIDE study and for sharing the results of data analysis with the FDA, the prospects of the STRIDE AHEAD study, and the potential filing and timing of an NDA to the FDA and thereafter to additional regulatory agencies. 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.
Reneo Pharmaceuticals, Inc.
Matthew Purcell, Pharm.D.
Russo Partners, LLC