PTC Therapeutics Inks Licensing Pact With Novartis Worth Up To ~$3 Billion For Huntington's Program

On Monday, PTC Therapeutics, Inc. PTCT signed an exclusive global license and collaboration agreement with Novartis AG NVS for its PTC518 Huntington’s disease program, which includes related molecules.

Under the agreement, PTC will receive an upfront payment of $1.0 billion, up to $1.9 billion in development, regulatory and sales milestones, a profit share in the U.S., and double-digit tiered royalties on ex-U.S. sales.

Also Read: FDA Grants Conditional Approval For PTC Therapeutics’ Gene Therapy As First Ever With Direct Administration In Brain

PTC518 was discovered from PTC’s validated splicing platform and is currently being studied in the ongoing Phase 2 PIVOT-HD trial.

Interim results reported in June 2024 showed that PTC518 treatment resulted in durable, dose-dependent reduction in blood and cerebrospinal fluid mutant Huntingtin protein levels as well as early signals of dose-dependent benefit on key clinical measurements at 12 months.

PTC518 continues to demonstrate a favorable safety and tolerability profile.

Novartis will assume responsibility for PTC518’s development, manufacturing, and commercialization following the completion of the ongoing placebo-controlled portion of PIVOT-HD, which is expected to occur in the first half of 2025. 

The companies will share U.S. profits and losses on a 40/60 basis (40% PTC and 60% Novartis). The parties anticipate the agreement will close in the first quarter of 2025.

Last week, PTC Therapeutics’ global Phase 2 placebo-controlled CardinALS study of Utreloxastat in amyotrophic lateral sclerosis patients did not meet its primary endpoint of slowing disease progression on the composite ALSFRS-R and mortality analysis.

Price Action: PTCT stock is up 17.1% at $51.38 at last check Monday.

Read Next:

• Why Is Senti Biosciences Stock Surging Over 400% On Monday?

Photo via Shutterstock.