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In a deal expected to close in Q1 2025, PTC Therapeutics, Inc., a biopharma company, has signed an exclusive global license and collaboration agreement with Novartis Pharmaceuticals Corporation, a subsidiary of Novartis AG, for its PTC518 Huntington’s disease program, which also includes related molecules.1 Huntington’s disease is a genetic disorder of the central nervous system.2
Per the terms of the deal, PTC will receive an upfront payment of $1 billion; there is also the opportunity for the company to make as much as $1.9 billion in development, regulatory, and sales milestones; the agreement not only includes a profit share in the United States, but double-digit tiered royalties on any sales outside the United States as well. Once the on-going placebo-controlled portion of PIVOT-HD is completed by the first half of 2025, Novartis will be responsible for the development, manufacturing and commercialization of PTC518.
The US profits and losses will be split 40/60, 40% PTC and 60% Novartis.
“PTC518 is the leading oral disease-modifying therapy in development for Huntington’s disease and the economics of this agreement are consistent with the promise of this treatment,” noted Matthew B. Klein, MD, CEO of PTC Therapeutics. “This collaboration combines PTC’s expertise in developing small molecule splicing therapies with Novartis’s expertise in global development and commercialization of neuroscience therapies. We are excited to collaborate with Novartis to accelerate the potential of PTC518 for the hundreds of thousands of HD patients worldwide in need of a therapy designed to be well-tolerated and an effective disease-modifying therapy. PTC will use the proceeds of this transaction to expand our splicing platform as well as to support commercial and development portfolio activities.”
Given the severity of this neurodegenerative disease, this partnership also allows Novartis to find new, game-changing ways to tackle Huntington’s, while also boosting its offerings. In this particular case, PTC518 was discovered due to PTC’s validated splicing platform. It’s currently being studied in the Phase II PIVOT-HD trial, with initial June 2024 results indicating that the treatment resulted in “durable, dose-dependent” decrease in blood and cerebrospinal fluid (CSF) mutant Huntingtin protein (HTT) levels; there were also early signs of dose-dependent benefits surrounding various clinical measurements at the 12- month mark. The company also notes that PTC518 continues to show both a promising safety and tolerability profile.3
“Huntington’s disease is a devastating, fatal, familial disease. This agreement with PTC is intended to bolster our neuroscience pipeline and reflects our strategic focus and commitment to explore new and potentially transformative approaches for neurodegenerative diseases with high unmet needs,” commented Vas Narasimhan, CEO of Novartis. “We look forward to building on our expertise in neurodegenerative diseases and experience in HD with the intention to advance this potential first in class oral therapy for the HD community.”
As alluded to previously, PTC uses advanced alternative splicing technology to recognize small molecules that can affect mRNA splicing, which can help treat diseases that have a high unmet need.
References
1. PTC Therapeutics Enters into a Global License and Collaboration Agreement with Novartis for PTC518 Huntington’s Disease Program. PR Newswire. December 2, 2024. Accessed December 3, 2024. https://www.prnewswire.com/news-releases/ptc-therapeutics-enters-into-a-global-license-and-collaboration-agreement-with-novartis-for-ptc518-huntingtons-disease-program-302319374.html
2. World Health Organization, 2020. 8A01.10 Huntington disease. Accessed December 3, 2024. https://icd.who.int/browse11/l-m/en#/http://id.who.int/icd/entity/2132180242
3. Interim PIVOT-HD Results Demonstrate Evidence of Favorable CNS Biomarker and Clinical Effects at Month 12 in Huntington’s Disease Patients. PTC Therapeutics. June 20, 2024. Accessed December 3, 2024. https://ir.ptcbio.com/news-releases/news-release-details/interim-pivot-hd-results-demonstrate-evidence-favorable-cns