IRVINE, CA, USA I July 28, 2021 I 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.
SOURCE: Reneo Pharmaceuticals
Post Views: 132
IRVINE, CA, USA I July 28, 2021 I 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.
SOURCE: Reneo Pharmaceuticals
Post Views: 132
