PTC Therapeutics Inc. has announced an agreement to monetize a portion of the risdiplam royalty stream for $650 million from Royalty Pharma plc. The capital from the collaboration will enable PTC to further develop and expand its rare disorder portfolio, particularly its validated splicing, Bio-e and gene therapy platforms.
 
Risdiplam is an investigational survival motor neuron 2 (SMN2) splicing modifier for SMA and is an orally administered liquid. It is designed to increase and sustain SMN protein levels both throughout the central nervous system and in peripheral tissues of the body.
 
“The discovery, development and expected commercialization of risdiplam exemplifies how PTC’s strengths in novel scientific approaches to diseases with high unmet needs can generate value for the benefit of all of our stakeholders,” said Stuart W. Peltz, Ph.D., CEO of PTC Therapeutics. “Today’s announcement of our strategic partnership with Royalty Pharma brings forward significant, non-dilutive capital to drive further innovation and growth across our robust and diverse rare disorder portfolio.”
 
Under the terms of the royalty purchase agreement, PTC Therapeutics will receive $650 million in upfront cash from Royalty Pharma in return for approximately 43% of the risdiplam royalties, up to a specified amount. PTC Therapeutics maintains the majority of the risdiplam royalty stream and retains all economics associated with up to approximately $400 million in remaining regulatory and sales milestones.
 
Pablo Legorreta, Royalty Pharma’s founder and CEO, stated, “Risdiplam is consistent with our focus on high value, differentiated therapeutics addressing diseases with high unmet medical need. We recognize the value and importance of an oral therapy for the treatment of all types of SMA. We are delighted to partner with PTC and to help fund their innovative pipeline of treatments for rare diseases.”
 
The risdiplam New Drug Application (NDA) for the treatment of Types 1, 2 and 3 spinal muscular atrophy (SMA) is under Priority Review by the U.S Food and Drug Administration, with a PDUFA date of August 24, 2020. A Marketing Authorization Application (MAA) is planned to be submitted to the European Medicines Agency (EMA), as well as filings in other international markets. The risdiplam SMA program is a collaboration between PTC, the SMA Foundation, and Roche.