RESEARCH TRIANGLE PARK – A drug being developed by Cary-based Rescindo Therapeutics for treating a rare developmental disorder in children has received two special designations from the U.S. Food and Drug Administration that will help the company advance the potential therapy.
The drug, called RSC-57, was granted Orphan Drug Designation and Rare Pediatric Disease Designation for the treatment of patients with Kabuki syndrome.
“The decision validated the urgency and need for treatment of this devastating disease for which there are no FDA-approved therapies,” said Athanasios (Thanos) Maroglou, Ph.D., co-founder, president and chief executive officer of Rescindo. “With RSC-57 being a repurposed drug with a large established safety profile, we hope to proceed with clinical trials rapidly after the completion of our pre-IND package and guidance from the FDA.”
Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity.
Rare Pediatric Disease Designation is granted by the FDA in the case of serious or life-threatening diseases affecting fewer than 200,000 people in the United States, primarily those 18 years old and younger. The sponsor of a drug with this designation may, upon marketing approval, qualify for a priority review of a subsequent marketing application.
Kabuki syndrome (KS), typically diagnosed in early childhood, is a rare genetic disorder marked by intellectual disability, muscle weakness, distinctive facial features, short stature, immune deficiency and malformations of the cardiac, renal, gastrointestinal and/or skeletal systems.
The global prevalence of KS is estimated at one in 32,000 births, although its incidence is likely greater given the acceleration of new cases identified by genome sequencing as that technology has become widely available, according to Rescindo. KS has no known cure.
In addition to Kabuki syndrome, the company has a research program for Bardet-Biedl syndrome, a rare genetic disorder with variable symptoms that can include retinal degeneration, obesity, reduced kidney function and extra digits on the hands or feet.
Rescindo, a 2015 spin-out of Duke University, is focused on developing treatments for rare genetic diseases. Its technology platform combines genetics, molecular biology and high-throughput use of model organisms including zebrafish and mice to identify suppressors of genetic disorders.
Over $70 million has been invested in developing the technology, and more than 260 papers have been published in scientific journals, according to Duke’s Office of Licensing and Ventures. Rescindo has an exclusive technology license from Duke.
The technology was developed by a research team led by Nicholas Katsanis, Ph.D., at Duke’s Center for Human Disease Modeling. Katsanis, Rescindo’s co-founder and chief scientific officer, is the Jean and George W. Brumley Jr., M.D., Professor of Developmental Biology at Duke’s School of Medicine.
Co-founder Maroglou is also a familiar face in the Research Triangle’s life science community. Prior to his role at Rescindo, he held executive positions at several companies in the area including Cato Research, Cato BioVentures, Chimerix, Ercole Biotech and Paradigm Genetics.
Rescindo has received two research grants from the National Institutes of Health and has signed research collaboration agreements with two undisclosed biotech and pharmaceutical companies.
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