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FDA Grants Orphan Drug Designation to RASRx’s Duchenne Muscular Dystrophy Drug

NEWPORT BEACH, Calif.–(BUSINESS WIRE)–RASRx announced today that the United States Food and Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) for its compound RASRx1902 for the treatment of Duchenne Muscular Dystrophy. RASRx1902 is an oral therapy that has shown positive effects on muscle function in animal models of Duchenne. In these models, the RASRx1902 has improved muscle strength and regeneration while decreasing muscle inflammation, degeneration, and necrosis.

Through this ODD, RASRx is eligible for financial incentives that can stimulate investment in this program and expedite its regulatory timeline. In concert with CureDuchenne, the company is now transitioning to toxicology studies to advance RASRx1902 towards human clinical trials for the treatment of Duchenne.

RASRx’s founders worked with the USC Stevens Center for Innovation, the technology transfer office for the University of Southern California, to exclusively license RASRx1902. Funding by CureDuchenne Ventures and a collaborative U.S. Department of Defense research grant with USC has accelerated the preclinical development of this program. “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.

Duchenne is a fatal genetic disease that causes muscles to degenerate. It impacts approximately 1 in 3,500 boys. Those with Duchenne are usually diagnosed by age 5, lose their ability to walk by 12 and most don’t survive their mid-20s. There are limited approved treatments and no cure for Duchenne.

“We are passionate about making an impact in areas of unmet needs like Duchenne Muscular Dystrophy,” said Kathleen Rodgers, a co-founder of RASRx. “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. 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.”

About RASRx

RASRx is developing and commercializing technology licensed from the University of Southern California. The founders of the company are USC researchers with extensive drug development expertise having taken projects through late clinical stage development. RASRx has innovated a leading-edge, systems biology approach to treating the complex muscle pathology of Duchenne muscular dystrophy.

About CureDuchenne

CureDuchenne is the leading nonprofit focused on funding research to find a cure for Duchenne muscular dystrophy, a disease that affects more than 300,000 boys worldwide. CureDuchenne’s mission is to extend and improve the lives of those affected by Duchenne. With support from CureDuchenne, nine research projects have advanced to human clinical trials. CureDuchenne provides the Duchenne community with resources on the best standard of care through its CureDuchenne Cares program. For more information, please visit CureDuchenne.org and follow us on Facebook, Twitter, Instagram and YouTube.

About the USC Stevens Center for Innovation

The USC Stevens Center for Innovation is the technology transfer office for the University of Southern California and a university-wide resource for USC innovators in the Office of the Provost. The mission at the USC Stevens Center for Innovation is to maximize the translation of USC research into products for public benefit through licensing, collaborations, and the promotion of entrepreneurship and innovation. For more information, visit http://stevens.usc.edu.

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