We are developing mavodelpar to modulate genes critical to metabolism and generation of ATP, which is the primary source of energy for cellular processes.
We believe mavodelpar could benefit patients with genetic mitochondrial myopathies who experience weakness, fatigue, cramping and wasting of muscle due to the mitochondria’s inability to produce adequate levels of ATP.
Mavodelpar is being developed in two rare diseases, primary mitochondrial myopathies (PMM) and long-chain fatty acid oxidation disorders (LC-FAOD).
We continue to explore other diseases where mavodelpar may provide benefit. We intend to establish mavodelpar as the standard of care for multiple rare diseases affecting the mitochondria.
We completed a Phase 1b clinical trial of mavodelpar in patients with PMM and myopathy with mitochondrial DNA (mtDNA) mutations. The primary objective of the trial was to evaluate the safety and tolerability of mavodelpar, and we also included measures of clinical outcome such as exercise tests and symptom questionnaires. Mavodelpar was generally well tolerated with no drug related serious adverse events (SAE) observed. Improvements were observed in exercise tests and symptoms questionnaires.
We are currently enrolling patients into the STRIDE Study. STRIDE is a global, randomized, double-blind, 6-months, placebo-controlled clinical trial designed to assess the efficacy and safety of mavodelpar administered orally once daily to patients with primary mitochondrial myopathies (PMM).
We are also enrolling patients into the STRIDE Ahead study. STRIDE Ahead is a 24-month open-label study of mavodelpar in PMM patients.
A non-interventional, international study in adult patients with LC-FAOD was conducted to better understand the natural history of LC-FAOD and changes in patient function and symptoms over time (FORWARD Study). We evaluated patients prospectively with exercise tests and symptom questionnaires.
Based on the results of the LC-FAOD Phase 1b study, in conjunction with the results of the LC-FAOD non-interventional study, the Company intends to continue development of mavodelpar for patients with LC-FAOD.
Reneo is committed to developing promising new therapies to address the unmet medical needs of patients suffering from rare and seriously debilitating diseases. We are currently enrolling patients in a late-stage clinical trial for the treatment of Primary Mitochondrial Myopathies (PMM) and completed an early-stage study for Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD). Our goal is to provide access to our medicines at the appropriate time and taking into consideration all the potential risks and benefits to the patients. We believe enrollment in our ongoing clinical trials is the safest and most effective way of achieving this goal. We do recognize that some patients will not be eligible to take part in our clinical trials and may wish to access our drugs in development through expanded access. Currently, we do not have the resources available to offer expanded access use of our investigational medicines to patients who have not previously received mavodelpar. We encourage all patients and physicians who are interested in accessing our investigational medicines to visit the clinical trial section of our website to find out about our open studies for enrollment
Reneo may revise this expanded access policy in the future. Additionally, the posting of this policy by Reneo does not serve as a guarantee of access to any specific investigational new drug by any individual patient. If you have any questions, please contact us.