First Patients Enroll in Phase 1 Trial of EPM-01, Potential Becker MD Therapy

First Patients Enroll in Phase 1 Trial of  EPM-01, Potential Becker MD Therapy
2.7
(3)

A Phase 1 clinical trial of EPM-01 ((+)-epicatechin), a potential oral treatment of Becker muscular dystrophy that targets cellular energy, has enrolled its first patients.

This one-year study of Epirium Bio‘s small molecule therapy is taking place at three U.S. locations: the University of California, Los Angeles, the University of California, Davis, and Washington University School of Medicine in St. Louis.

Enrollment is open to patients, ages 16 to 59, and contact information can be found here.

The company also announced that the European Commission (EC) granted EPM-01 an orphan designation as a potential Becker muscular dystrophy treatment. The U.S. Food and Drug Administration had previously awarded orphan drug status to EPM-01, under the name of EB 002. This U.S. designation covers both Duchenne and Becker muscular dystrophies.

Orphan designation provides certain benefits, such as financial incentives for clinical development and commercialization, fee exemptions, and market exclusivity of seven years in the U.S. and 10 years in the EU.

“The initiation of enrollment in our Phase 1 study and the EC designation of orphan drug status for EPM-01 in Becker muscular dystrophy are both important milestones for this development program,” Ransi Somaratne, MD, chief medical officer of Epirium, said in a press release.

“Patients living with Becker muscular dystrophy are in need of dedicated therapies, as there are no approved treatments for this inherited disorder. We look forward to the results of our Phase 1 trial, which will help us select the optimal dose of EPM-01 for a pivotal clinical study in this patient population,” Somaratne added.

EPM-01 is a synthetic compound intended to act upon multiple cellular pathways, including cellular energetics and muscle regeneration. Specifically, the therapy is a flavonol, a type of flavonoid molecule normally found in food with antioxidant and anti-inflammatory effects.

According to Epirium, treatment with EPM-01 may slow or stop disease progression by addressing processes such as mitochondrial dysfunction, inflammation, and loss of muscle tissue. (Mitochondria are the cell compartments responsible for the production of energy.)

The Phase 1 study (NCT04386304) will evaluate the safety, preliminary efficacy, and potential biomarkers of three ascending doses of EPM-01 in participants with Becker or Becker-like muscular dystrophy.

It expects to enroll up to 20 ambulatory male patients with confirmed mutations in the DMD gene, which cause both Duchenne and Becker.

Once enrolled, participants will undergo a six-month dose-escalation period, after which they will continue treatment at the highest tolerated dose for six more months, for a total of one year. The study is expected to end in December 2021.

“This advancement into Phase 1 enrollment for EPM-01 represents a major step forward in the development of a novel therapy for people living with Becker muscular dystrophy,” said Russ Cox, president and CEO of Epirium. “I expect this study will provide valuable insights to inform the next phases of clinical development and bring us closer to being able to treat patients with this degenerative and life-threatening disease.”

Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
Total Posts: 42
José is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.
×
Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
Latest Posts
  • givinostat
  • Duchenne Action Month
  • SRP-9001

How useful was this post?

Click on a star to rate it!

Average rating 2.7 / 5. Vote count: 3

No votes so far! Be the first to rate this post.

As you found this post useful...

Follow us on social media!

We are sorry that this post was not useful for you!

Let us improve this post!

Tell us how we can improve this post?