Reneo Pharmaceuticals Announces First Patient Dosed in the STRIDE Study

Rhea-AI Summary

Reneo Pharmaceuticals (NASDAQ: RPHM) announced that the first patient has been dosed in its STRIDE study, aimed at evaluating REN001 for primary mitochondrial myopathies (PMM). This Phase 2b trial will include approximately 200 adult patients and assess efficacy through a 12-minute walk test after 24 weeks. REN001 has received Orphan Drug and Fast Track designations from the FDA. PMM affects 1 in 5,000 individuals globally, causing significant energy production deficits. The company plans to further develop REN001 for additional mitochondrial dysfunction-related diseases.

Positive

• First patient dosed in the STRIDE clinical trial for REN001.
• REN001 has received FDA Orphan Drug and Fast Track designations.
• Study involves approximately 200 adult patients, indicating strong trial scale.

Negative

• No approved treatment options currently available for PMM, indicating high competition and risk.
• Forward-looking statements include uncertainty about clinical trial outcomes.

IRVINE, Calif., July 28, 2021 (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 first patient has been dosed in the STRIDE study. The trial is designed to evaluate the efficacy and safety of REN001 for the treatment of patients with primary mitochondrial myopathies (PMM).

STRIDE is a global, double-blind, randomized, placebo-controlled Phase 2b trial designed to assess the efficacy and safety of 100 mg REN001 administered orally, once-daily for 24 weeks. Approximately 200 adult patients with PMM caused by alterations in mitochondrial DNA are expected to be enrolled into STRIDE. The primary efficacy endpoint is the change from baseline in the distance walked during a 12-minute walk test after 24 weeks of treatment. Secondary efficacy endpoints include changes from baseline in the Modified Fatigue Impact Scale, Patient Global Impression of Change scale, and other patient-reported outcomes.

“We are truly excited about achieving this important milestone,” said Lynn Purkins, Ph.D., Senior Vice President of Clinical Operations. “Patients with PMM have genetic alterations that hamper the ability of their mitochondria to generate energy from nutrient sources, affecting multiple organ systems. It is a debilitating condition, for which there are unfortunately no approved treatment options. As we continue to advance REN001 in this patient population, we look forward to completing enrollment of the STRIDE study and sharing the results,” concluded Dr. Purkins.

REN001 is a potent and selective agonist of the peroxisome proliferator-activated receptor delta (PPARδ) receptor. The company has received Orphan Drug and Fast Track designations for REN001 from the U.S. Food and Drug Administration for PMM. In addition to PMM, the company is also developing REN001 for the treatment of other diseases that result from mitochondrial dysfunction or impaired energy production, including long-chain fatty acid oxidation disorders (LC-FAOD) and glycogen storage disorder type V (McArdle disease).

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). Reneo is developing REN001 to modulate genes critical to metabolism and generation of ATP, which is the primary source of energy for cellular processes. REN001 has been shown to increase transcription of genes involved in mitochondrial function and increase fatty acid oxidation and may increase production of new mitochondria.

About PMM
PMM are a group of disorders that affect roughly 1 in 5,000 people worldwide. PMM are caused by genetic mutations in the mitochondrial or nuclear DNA that reduce the ability of mitochondria to produce energy from nutrient sources. This energy deficit particularly affects tissues and organs with high energy demand such as muscle, brain, and heart. The symptoms of PMM include muscle weakness or exercise intolerance, movement disorder, deafness, blindness, and droopy eyelids among others. The prognosis for these disorders ranges in severity from progressive weakness to 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 enrollment, conduct, progress, timing, and results of Reneo’s clinical trials, 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.

Contact:

Joyce Allaire
Managing Director
LifeSci Advisors, LLC
jallaire@lifesciadvisors.com

Vinny Jindal
Chief Financial Officer
Reneo Pharmaceuticals, Inc.
investors@reneopharma.com

FAQ

What is the STRIDE study by Reneo Pharmaceuticals (RPHM)?

The STRIDE study is a Phase 2b clinical trial evaluating the efficacy and safety of REN001 in patients with primary mitochondrial myopathies (PMM).

What are the primary endpoints of the STRIDE study?

The primary efficacy endpoint is the change in distance walked during a 12-minute walk test after 24 weeks of treatment.

What designations has REN001 received from the FDA?

REN001 has received Orphan Drug and Fast Track designations from the FDA for treating primary mitochondrial myopathies.

How many patients will be enrolled in the STRIDE trial?

Approximately 200 adult patients with PMM are expected to be enrolled in the STRIDE study.

What does primary mitochondrial myopathy (PMM) affect?

PMM affects energy production in cells, particularly impacting tissues with high energy demands, such as the muscles and brain.