pharmaphorum December 2, 2024
Novartis has made a dramatic return to the Huntington’s disease therapy stage by licensing a candidate from PTC Therapeutics for a whopping $1 billion upfront.
The deal – which has a potential total value of up to $2.9 billion – gives the Swiss pharma group rights to PTC518, PTC’s oral mRNA splicing modifier, which is aiming to become the first disease-modifying therapy for Huntington’s.
Novartis’ move comes after it abandoned the development of its own Huntington’s candidate branaplam last year, which had reached phase 2b development, after concluding that the drug’s risks were likely to outweigh any potential clinical benefit.
PTC518 is designed to reduce levels of the mutated huntingtin protein that leads to injury and death of neurons, which...
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