Rare Disease Report: RASRx is the Latest Duchenne Drug To Get Orphan Status From the FDA

Thanks to Rare Disease Report for this piece.

University of Southern California (USC) start-up RASRx hopes to develop a drug to treat Duchenne muscular dystrophy (DMD). Today, the company announced their lead compound RASRx1902 has been granted an Orphan Drug Designation from the United States Food and Drug Administration (FDA).
 
DMD is a progressive muscle disorder caused by the lack of functional dystrophin protein. DMD patients lose the ability to walk as early as age 10, and experience life-threatening lung and heart complications in their late teens and 20s.

There are an estimated 15,000 DMD patients in the United States but the population has many subsets based on mutations of the dystrophin gene. Drugs, such as the recently approved Exondyx-51 (eteplirsen), are only effective in a subset of DMD patients.

While details of the drug are limited, RASRx1902 has improved muscle strength and regeneration in patients while decreasing muscle inflammation, degeneration, and necrosis. At present, it is not clear what the drug’s mechanism of action is, just that it was developed using a systems biology approach and should be effective in all DMD patients, independent of their gene mutation.
 
Via news release, Kathleen Rodgers, PhD, associate professor at the School of Pharmacy at USC and co-founder of RASRx said, “This lead program at RASRx takes a mutation-independent approach to halting, and possibly reversing, the degenerative progression of Duchenne. Through this approach, we hope to bring relief to patients with Duchenne, as well as other muscular dystrophies.”
 
The RASRx1902 studies are being partially funded by research grants at USC in addition to an investment from CureDuchenne Ventures, an investment off-shoot of the advocacy group CureDuchenne. Toxicology studies are currently being planned for RASRx1902 before advancing into human clinical trials.
 
The collaboration between USC, the biotech company, venture capital, and advocacy is an interesting one and all parties seem thrilled be part of it.
 
“Innovation is a core part of USC’s culture and we are proud to support Dr. Kathleen Rodgers and RASRx to facilitate the technology transfer of RASRx1902 for DMD,” said Michael Arciero, J.D. Director of Technology Commercialization and New Venture, USC Stevens Center for Innovation.
 
Dr Rodgers added, “We have been benefitted immensely from CureDuchenne’s knowledge of the disease and familiarity with the needs of the patients. This collaboration has been invaluable as we move forward in the development of this promising treatment.”
 

CureDuchenne Ventures, led by Debra Miller, first learned of RASRx at a scientific conference. Her son Hawken has DMD and is attending USC. “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,” said Miller.