Shots:

• PTC Therapeutics to receive $650M as upfront in cash in exchange for ~43% of the risdiplam royalties- up to a specified amount while PTC maintains the majority of the risdiplam royalty stream and retains all economics associated with up to ~$400M in remaining regulatory and commercial milestones
• The collaboration enables PTC to further develop and expand its innovative rare disorder portfolio- including its validated splicing- Bio-e and gene therapy platform
• Risdiplam is an investigational SMN2 splicing modifier for SMA- its NDA is under the US FDA’s Priority Review with anticipated PDUFA date as Aug 24- 2020. The company is planning to submit MAA to the EMA as well as filings in other international markets

  Ref: PTC Therapeutics | Image: RTTNews

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