IRVINE, Calif., Aug. 29, 2022 (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 two poster presentations at the International Network for Fatty Acid Oxidation Research and Management (INFORM) annual meeting, being held in Freiburg, Germany August 28-29, 2022. The poster presentations highlight the results of two recently completed clinical trials in patients with long-chain fatty acid oxidation disorders (LC-FAOD), including a successful REN001 Phase 1b study and a non-interventional natural history study.
An open-label study to evaluate the safety and tolerability of 12 weeks of treatment with oral REN001 in subjects with long-chain fatty acid oxidation disorders (LC-FAOD) – Jerry Vockley, et al
In this 12-week, open-label study, REN001 was safe and well tolerated. Of the 24 patients enrolled, 18 patients (75%) reported experiencing an adverse event, the majority reported to be mild or moderate in severity and consistent with the disease state. Improvements in 12-minute walk test distance and physical functioning scores were observed in a subgroup of patients. Investigators concluded that the data were supportive of further evaluation of REN001 in this patient population.
A prospective, multicenter, non-interventional study to investigate the disease characteristics of adult patients with long-chain fatty acid oxidation disorders: The FORWARD study – Melanie Gillingham, et al
The most common historical adverse events reported by patients were rhabdomyolysis (79.3%) and hospitalization (62.1%). In the 4-month, 58 patient study, small improvements in 12-minute walk test distance and physical functioning scores were observed. Investigators concluded that the data provides important information on natural history of FAOD that can be used for the design of future REN001 therapeutic trials.
The poster presentations elaborate on results first announced by the company in July 2022 and highlight the unmet medical need of patients with LC-FAOD. Based on these encouraging data, the company previously announced it will move the program forward.
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, REN001, is a potent and selective agonist of the peroxisome proliferator-activated receptor delta (PPARδ). 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. For additional information, please see reneopharma.com.
About REN001
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 range 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 results, conduct, progress and timing of Reneo’s clinical trials, presentation of data from 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.
Contacts
Joyce Allaire
Managing Director
LifeSci Advisors, LLC
jallaire@lifesciadvisors.com
Danielle Spangler
Investor Relations
Reneo Pharmaceuticals, Inc.
dspangler@reneopharma.com
