Phase 3 ready rare disease company focused on MCT8 deficiency (a.k.a. Allan-Herndon Dudley Syndrome). Currently no approved therapies exist for AHDS. Our lead Program - SRW101 received Orphan Drug (ODD) in US & EU and Rare Pediatric Disease (RPD) designations with a potential to receive Priority Review Voucher (PRV) upon NDA approval. Potential market size based on 5,000-10,000 MCT8 deficient patients in target population is around $2B. Program was developed by the world-renowned physician scientists on the MCT8 deficiency from Univ of Miami and University of Chicago {Roy Weiss, MD, PhD (CMO) and Nobel Prize nominee Sam Refetoff MD (CSO)}.
Launched the company in 2020 and currently working with academia in completing necessary non-clinical studies for launching the Phase III pivotal registration study.
Currently SRW101 is undergoing an emergency use clinical trial at University of Miami for treating pregnant mothers carrying male fetus with MCT8 deficiency under the pre-natal IND.
Launched the company in 2020 and currently working with academia in completing necessary non-clinical studies for launching the Phase III pivotal registration study.
Currently SRW101 is undergoing an emergency use clinical trial at University of Miami for treating pregnant mothers carrying male fetus with MCT8 deficiency under the pre-natal IND.
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