Novartis bets $2.2B on Arrowhead’s RNA brain drug to crack Parkinson’s code

Novartis is licensing a preclinical RNA drug from Arrowhead Pharmaceuticals for $200M upfront — and potentially $2B+ more — aiming to target alpha-synuclein, a key protein linked to Parkinson’s and other neurodegenerative diseases.

Why it matters: Parkinson’s has resisted treatment for decades. Novartis sees potential in Arrowhead’s RNA interference tech to succeed where other major players have failed.

Backstory: Novartis’ prior alpha-synuclein therapy, minzasolmin (partnered with UCB), failed in Phase 2 trials. Arrowhead’s TRiM tech delivers siRNA subcutaneously to reach deep brain regions, showing promising preclinical results.

Zoom in: Arrowhead’s pipeline also includes a near-commercial drug for a rare fat metabolism disorder, with FDA approval expected by mid-November. Arrowhead has other RNA deals with Takeda, Amgen, Sarepta, and GSK, but is newer to the CNS space.

Big picture: If Arrowhead’s TRiM platform succeeds in the CNS, it could redefine how RNA-based medicines are delivered to the brain, potentially unlocking new treatments for other neurodegenerative diseases.

What’s next: Novartis will lead human testing and commercialization while Arrowhead handles the early-stage research. Novartis can also license additional targets beyond Arrowhead’s current pipeline. Arrowhead shares jumped over 9% after the announcement