Royalty Pharma Acquires Royalty Interest in Risdiplam From PTC Therapeutics

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NEW YORK, July 20, 2020 (GLOBE NEWSWIRE) — Royalty Pharma plc (Nasdaq: RPRX) announced today an agreement with PTC Therapeutics, Inc. (Nasdaq: PTCT) to acquire a portion of PTC’s royalty interest in risdiplam for a one-time payment of $650 million.

Risdiplam, to be marketed by Roche, is an investigational, orally administered survival motor neuron-2 (SMN2) splicing modifier for the treatment of spinal muscular atrophy (SMA). SMA is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. It is the most common genetic cause of infant mortality and one of the most common rare diseases, affecting approximately one in 11,000 babies.

Pablo Legorreta, Royalty Pharma’s Founder and Chief Executive Officer, 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 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., Chief Executive Officer 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.”

The risdiplam 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 for submission 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.

Relative to Royalty Pharma’s existing business, the risdiplam royalty acquisition is expected to have minimal impact on its Adjusted cash receipts and Adjusted cash flow in 2020 and 2021, with growing accretion beyond 2021 resulting in enhanced long-term growth. Royalty Pharma is purchasing approximately 43% of the royalty up to a specified amount. PTC has previously disclosed that it is entitled to tiered royalties ranging from 8% to 16% on worldwide net product sales of risdiplam. 

Wilmer Hale acted as legal advisor to PTC Therapeutics on the transaction. Goodwin Procter, Dechert and Maiwald acted as legal advisors to Royalty Pharma.

About Royalty Pharma plc 

Founded in 1996, Royalty Pharma is the largest buyer of biopharmaceutical royalties and a leading funder of innovation across the biopharmaceutical industry, collaborating with innovators from academic institutions, research hospitals and not-for-profits through small and mid-cap biotechnology companies to leading global pharmaceutical companies. Royalty Pharma has assembled a portfolio of royalties which entitles it to payments based directly on the top-line sales of many of the industry’s leading therapies. Royalty Pharma funds innovation in the biopharmaceutical industry both directly and indirectly – directly when it partners with companies to co-fund late-stage clinical trials and new product launches in exchange for future royalties, and indirectly when it acquires existing royalties from the original innovators. Royalty Pharma’s current portfolio includes royalties on more than 45 commercial products, including AbbVie and J&J’s Imbruvica, Astellas and Pfizer’s Xtandi, Biogen’s Tysabri, Gilead’s HIV franchise, Merck’s Januvia, Novartis’ Promacta, and Vertex’s Kalydeco, Symdeko and Trikafta, and four development-stage product candidates. For more information, visit

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Source: RP Management, LLC

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