Programs

Our lead candidate, REN001, is a selective peroxisome proliferator-activated receptor delta (PPAR) agonist that has been shown to increase transcription of genes involved in mitochondrial function and increase fatty acid oxidation (FOD), and may also increase production of new mitochondria.

REN001 Overview

We are developing REN001 to modulate genes critical to metabolism and generation of ATP, which is the primary source of energy for cellular processes.

We believe REN001 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.

One Approach, Multiple Diseases

REN001 is being developed in three rare diseases primary mitochondrial myopathies (PMM), long-chain fatty acid oxidation disorders (LC-FOAD) and glycogen storage disease V (McArdle disease).

We plan to broadly investigate the potential of REN001 in other rare diseases, such as Duchenne’s muscular dystrophy and Alport’s syndrome.

Pipeline

Clinical Trials

We have initiated a global Phase 2b clinical trial of REN001 in patients with PMM and expect to start enrolling patients in the first half of 2021 (STRIDE study). STRIDE is a randomized, double-blind, placebo-controlled, multi-center clinical trial designed to investigate the efficacy and safety of REN001 in patients with PMM.

Reneo is currently enrolling an open-label Phase 1b clinical trial in adult patients with LC-FAOD. The primary objective of the trial is to evaluate the safety and tolerability of REN001 in the LC-FAOD patient population, and we will also explore multiple clinical outcomes.
We are also conducting a non-interventional, international study in adult patients with LC-FAOD to better understand disease characteristics of patients (FORWARD study). We plan to evaluate patients prospectively with exercise tests and symptom questionnaires.
Reneo is currently enrolling an open-label Phase 1b clinical trial in adult patients with McArdle disease. The primary objective of the trial is to evaluate the safety and tolerability of REN001 in the McArdle patient population, and we will also explore multiple clinical outcomes such as muscle symptoms, physical function, and work productivity.
We completed a Phase1b clinical trial of REN001 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 REN001, and we also included measures of clinical outcome such as exercise tests and symptom questionnaires. REN001 was generally well tolerated with no drug related serious adverse events (SAE) observed. Improvements were observed in exercise tests and symptoms questionnaires.

Expanded Access Policy

Reneo is committed to developing promising new therapies to address the unmet medical needs of patients suffering from rare and seriously debilitating diseases. We currently have a late-stage clinical trial planned for the treatment of Primary Mitochondrial Myopathies (PMM) and early-stage studies for Glycogen Storage Disorder V (GSDV, also known as McArdle Disease) and Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD). Our goal is to provide access to our medicines at the appropriate time and in a manner that is most beneficial to the relevant patient population. 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 for our clinical trials and may wish to access our products through expanded access. However, at this time, we do not have the resources available to offer expanded access use of our investigational medicines. 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 enrolling.


Reneo may revise this expanded access policy at any time. 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.