Novartis to Disburse Up to .9 Billion in Huntington’s Agreement With PTC

Novartis and PTC Therapeutics announced a global licensing agreement on Monday aimed at advancing PTC’s Huntington’s disease treatment, PTC518.

Under the deal’s terms, Novartis will provide an upfront payment of billion and has the potential to invest up to .9 billion linked to various developmental, regulatory, and sales milestones. PTC will secure a 40% profit share in the United States, while Novartis retains 60%. They will also exchange double-digit tiered royalties on sales outside the U.S. If regulatory approvals and other standard conditions are met, the parties anticipate finalizing the agreement by Q1 2025.

Following the announcement, PTC’s stock surged approximately 20% prior to Monday’s trading session, as reported by Seeking Alpha.

PTC518 is a small molecule drug designed for oral administration that effectively penetrates the brain. It functions by reducing the levels of the mutant huntingtin protein to combat neuronal death and brain damage associated with Huntington’s disease (HD). In September 2024, the FDA awarded PTC518 a Fast Track designation for HD.

The drug is currently in Phase II trials, specifically the PIVOT-HD study. An interim report from June 2024 indicated a “dose-dependent lowering” of the mutant huntingtin protein in blood and cerebrospinal fluid over 12 months, although specific figures were not disclosed. Positive trends were noted in various clinical assessments, such as the Total Motor Score.

According to the deal, PTC will handle the completion of PIVOT-HD, expected in the first half of 2025, after which Novartis will assume responsibility for the development, manufacturing, and completion of PTC518.

Novartis has shown sustained interest in Huntington’s disease treatments. In March 2022, they identified that the small molecule candidate branaplam could lower the levels of mutant huntingtin and shifted its focus towards HD instead of spinal muscular atrophy. Branaplam modifies huntingtin mRNA, facilitating its degradation and leading to lower huntingtin protein expression.

However, plans for branaplam were halted due to adverse side effects detected shortly after, with the company officially discontinuing the drug for HD in February 2023.

Earlier in the year, Novartis entered into a potentially lucrative .3 billion deal with Voyager Therapeutics, focusing on its candidates for Huntington’s and spinal muscular atrophy. The partnership aims to utilize Voyager’s TRACER Capsid Discovery Platform to deliver gene therapy directly into the brain.

For PTC, this licensing agreement arrives shortly after the company discontinued utreloxastat due to unsatisfactory Phase II results in amyotrophic lateral sclerosis.