New research partnership aims to accelerate new drug for fatal childhood muscle disease

A prominent disease research foundation has invested in RASRx, a startup company formed by USC researchers working to develop a novel therapy for Duchenne, a rare and deadly form of muscular dystrophy.

The financial support by CureDuchenne Ventures, the venture funding arm of CureDuchenne, will help to fund ongoing research on the drug development for this disease. But it’s also important because the investment is part of a partnership that teams RASRx with experts at CureDuchenne who have significant fundraising experience and have navigated the government regulatory process specifically for novel rare disease treatments.

“It’s a very unique opportunity,” said Kathleen Rodgers, Ph.D., Co-Founder of RASRx and Associate Professor in the Titus Family Department of Clinical Pharmacy and Pharmaceutical Economics & Policy in the USC School of Pharmacy. “This is a very synergistic collaboration. The people, connections and understanding of the disease are more valuable than the investment funds alone.”

RASRx and CureDuchenne are working together to streamline the development of a Duchenne drug to reach patients as soon as possible. In addition to helping with the regulatory and fundraising side, CureDuchenne’s support will enable additional research studies that will produce more targeted data to help the therapy through the regulatory process, said Rodgers.

Duchenne is a progressive disease that causes muscles to degenerate. It affects approximately 1 of every 3,500 male births. Those born with the disease typically do not live past age 25 and are wheelchair bound during their lives. Duchenne is caused by a genetic mutation on the X chromosome and impairs the body from producing a protein called dystrophin, which is crucial to all muscles including for normal heart function. Heart failure is the leading cause of death for those with Duchenne.

RASRx’s project is an oral therapy that has shown positive effects on muscle function in a mouse model of the disease. The therapy has demonstrated improved muscle strength and regeneration, with decreased muscle inflammation and fibrosis. It also received support by a U.S. Department of Defense research grant awarded through USC. RASRx’s founders worked with the USC Stevens Center for Innovation to obtain a license for the USC research to develop it within their company.

“We felt RASRx had a very viable project and a very good team that would give them reason for success,” said Debra Miller, president of CureDuchenne Ventures and founder of CureDuchenne.

“The opportunity here is to have a new class of medications for the Duchenne boys…we at CureDuchenne are very excited,” added Jak Knowles, Vice President, Medical and Scientific Affairs at CureDuchenne who will join the RASRx Board of Directors.

The Newport Beach-based foundation CureDuchenne was formed by Paul and Debra Miller in 2003 after their son Hawken, who is now a first-year student at the Annenberg School for Communication and Journalism at USC, was diagnosed with Duchenne as a child.

Debra Miller, who learned at a scientific conference about the USC research project on Duchenne, said she was excited about the idea of having CureDuchenne support researchers at her son’s college.

“I knew he was going to go to USC when we heard about this, and I was really excited about it,” said Debra Miller. “Knowing he was going to USC and that we would be funding a project that would be helping him and the other boys with Duchenne was really special.”

The amount of CureDuchenne’s investment in RASRx is being kept private but includes milestones, which once achieved could lead to additional funding from the foundation. CureDuchenne, which uses a scientific advisory team to carefully select companies to invest in, has previously invested in Prosensa (now BioMarin Pharmaceutical) and MyoTherix. The foundation also made an early grant to Sarepta Therapeutics, all for the acceleration of cures and treatments for the disease.

–By Peijean Tsai, USC Stevens Center for Innovation, published April 6, 2016. Photo: Light micrograph of longitudinally sectioned skeletal muscle fibers by Rollroboter is licensed under CC BY-SA 3.0.