The STRIDE study did not meet its primary or secondary efficacy endpoint

The company to suspend mavodelpar development and implement a workforce reduction

IRVINE, Calif., Dec. 14, 2023 (GLOBE NEWSWIRE) -- Reneo Pharmaceuticals, Inc. (NASDAQ: RPHM), a pharmaceutical company focused on the development and commercialization of therapies for patients with rare genetic mitochondrial diseases, today announced its pivotal STRIDE study (NCT04535609) of mavodelpar in adult patients with primary mitochondrial myopathies (PMM) did not meet its primary efficacy or secondary efficacy endpoint. The primary efficacy endpoint of the trial was the change from baseline in the distance walked during the 12-minute walk test (12MWT) at week 24. The secondary efficacy endpoint was the change from baseline in the PROMIS® Short Form Fatigue 13a score.

“We are immensely thankful to all the patients, families, and healthcare professionals who participated in the mavodelpar PMM development program,” said Gregory J. Flesher, President and CEO of Reneo Pharmaceuticals. “Although the results of the STRIDE study were negative, the data generated are vitally important to the scientific community and we will make these data available once the final analyses are complete. Finally, I want to thank all Reneo employees for their dedication and tireless contributions to advancing the science of mitochondrial disease.”

The company intends to implement immediate cost savings initiatives, including suspension of the ongoing STRIDE AHEAD study and all other mavodelpar development activities and a workforce reduction of approximately 70%. The company currently has over $100 million in cash, cash equivalents, and short-term investments.

About STRIDE

The STRIDE study was 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 was 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 was the change from baseline in the distance walked during the 12-minute walk test (12MWT) at week 24. Secondary and exploratory endpoints included 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 was an open-label extension (OLE) trial being conducted outside of the United States in adult patients with PMM due to mtDNA defects who participated in the STRIDE study, the mavodelpar Phase 1b study, and mavodelpar-naïve patients with PMM due to nuclear DNA (nDNA) defects. The study was designed to evaluate the long-term safety and tolerability of 100 mg mavodelpar administered once-daily over a 24-month period.

About PMM

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.

About Mavodelpar

Mavodelpar (REN001) is a potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist which has been studied in 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 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). For additional information, please see reneopharma.com.

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 implementation of cost savings initiatives. 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

Matthew Purcell, Pharm.D.
Media Inquiries 
Russo Partners, LLC 
matthew.purcell@russopartnersllc.com