PTC Therapeutics, Inc., a globally recognized biopharmaceutical firm, has entered into a worldwide license and collaboration agreement with Novartis Pharmaceuticals Corporation, a division of Novartis AG. This partnership centers on PTC518, a program designed to innovate treatment for Huntington’s disease, along with related compounds.
The agreement stipulates that PTC will receive a $1.0 billion upfront payment and may earn up to $1.9 billion through various developmental, regulatory, and sales milestones. Additionally, PTC is entitled to share profits in the US and receive double-digit tiered royalties on sales outside the US.
“PTC518 is poised as the foremost oral therapy aiming to modify the course of Huntington’s disease, and the economic terms of this deal reflect the treatment’s potential,” commented Matthew B. Klein, M.D., CEO of PTC Therapeutics. “This partnership merges our proficiency in small molecule splicing solutions with Novartis’s prowess in developing and marketing neuroscience products globally. We’re thrilled to work alongside Novartis to expedite the advancement of PTC518, offering hope to numerous Huntington’s disease sufferers worldwide with a treatment focused on safety and efficacy. The proceeds will bolster our splicing platform while supporting commercial and development activities.”
Vas Narasimhan, CEO of Novartis, added, “Huntington’s disease is a dire, hereditary ailment. This collaboration with PTC aims to enhance our pipeline in neuroscience, underscoring our strategic dedication to exploring transformative interventions for neurodegenerative conditions that desperately require innovative solutions. We are eager to leverage our expertise in neurodegenerative disorders and Huntington’s disease to advance this promising oral therapy for the Huntington’s community.”
Originating from PTC’s verified splicing technology, PTC518 is undergoing evaluation in the ongoing Phase 2 PIVOT-HD trial. Results from June 2024 have shown that treatment with PTC518 leads to sustained, dose-dependent reductions in mutant huntingtin protein levels in blood and cerebrospinal fluid (CSF), alongside preliminary evidence of dose-dependent clinical benefits over 12 months. Moreover, PTC518 has consistently shown a favorable safety profile.
Novartis will oversee the development, manufacture, and marketing of PTC518 following the conclusion of PIVOT-HD’s current placebo-controlled segment, slated for completion in the first half of 2025.
The firms will equally share US profits and losses, with PTC receiving 40% and Novartis 60%.
Closing of the transaction is contingent on customary conditions, including regulatory approval, with expectations for closure in the first quarter of 2025.
Huntington’s disease is a hereditary central nervous system disorder caused by a gene defect leading to production of a toxic mutant huntingtin protein. Symptoms, varying widely per individual, predominantly affect brain function, causing abnormal movements, as well as speech, swallowing, and walking difficulties, in addition to cognitive, behavioral, and motor issues.
Currently, no cures or drugs exist to delay or halt disease progress, though some treatments target specific symptoms.
PTC has pioneered mRNA splicing technology to identify small molecules that target high unmet medical needs and developed Evrysdi (risdiplam), the first approved splicing modifier. Leveraging this platform, PTC has expanded across therapeutic areas such as neurodegenerative diseases, oncology, and metabolism with its advanced PTSeek platform for accelerated drug discovery.
PTC stands as a leader in developing clinically distinguished therapies that enhance the lives of individuals facing rare health challenges.