Policy & Regulation
Epirium Bio Announces Starts Phase 1 Clinical Trial of EPM-01 in Becker Muscular Dystrophy
13 August 2020 - - The first participants have been enrolled in US-based biopharmaceutical company Epirium Bio Inc's Phase 1 clinical trial of EPM-01 in Becker muscular dystrophy, the company said.

EPM-01 is a novel, oral, synthetic compound that has demonstrated compelling proof-of-concept in multiple preclinical and clinical studies of mitochondrial dysfunction.

The Phase 1 study will be conducted by investigators at three leading academic centers in the United States, including University of California, Davis, and Washington University School of Medicine, St. Louis.

Additionally, the company announced that the European Commission granted orphan designation for EPM-01 as a potential treatment for Becker muscular dystrophy, a rare, genetic, neuromuscular disorder that affects approximately 10,000 people in the European Union and 8,000 people in the United States.

EPM-01 was previously granted orphan drug designation by the US Food and Drug Administration for the treatment of Becker and Duchenne muscular dystrophy.


The Phase 1 open-label, dose-escalation study is evaluating the safety, preliminary clinical efficacy and potential biomarkers of three doses of EPM-01 in participants with Becker or Becker-like muscular dystrophy.

The study is expected to enroll approximately 20 male participants, ages 16 to 59, who are ambulatory and have confirmed mutations of the dystrophin gene.

After the six-month dose-escalation period, participants will continue treatment at the highest tolerated dose for an additional six months with a total one-year follow-up duration after dose initiation.

Orphan designation in the EU is granted by the EC based on a positive opinion issued by the European Medicines Agency Committee for Orphan Medicinal Products.

To qualify, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient non-clinical or clinical data to suggest the investigational medicine may produce clinically relevant outcomes.

Orphan designation provides companies with benefits and incentives including clinical protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralised marketing authorisation procedure valid in all EU member states, reduced regulatory fees, and 10 years of market exclusivity.

Becker muscular dystrophy is a rare, genetic, degenerative disease that causes progressive muscle weakening and loss, primarily affecting skeletal and cardiac muscles.

The disease mainly affects boys and usually start to manifest between age 10 and 15. It is estimated to affect between one in 18,000 and one in 30,000 male births globally.

Becker muscular dystrophy is caused by mutations in the gene responsible for the production of dystrophin, a protein that forms an important component of muscle fibers. As patients do not have enough working dystrophin, muscle fibers gradually break down, leading to muscle weakness.

Becker muscular dystrophy causes long-term disability and is life-threatening because of its effects on the heart and the muscles used to breathe. No therapies are currently approved for Becker muscular dystrophy in the United States or the EU.

EPM-01 is a synthetic compound that appears to affect multiple physiologic pathways including cellular energetics and muscle regeneration through key regulatory steps.

Reduced levels of abnormally functioning dystrophin lead to a vicious cycle of harmful processes in the muscles of patients with Becker muscular dystrophy, including calcium dysregulation, mitochondrial dysfunction, inflammation and ultimately loss of functioning muscle tissue to fibrosis.

Based on its mechanism of action, EPM-01 is expected to address multiple steps in this destructive cycle to break or slow its progression, potentially leading to clinical and functional benefit in patients with severe mitochondrial disorders.
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