Dewpoint Therapeutics and Mitsubishi Tanabe partner on ALS treatment

Dewpoint Therapeutics and Mitsubishi Tanabe Pharma Corporation (MTPC) have embarked on a strategic research collaboration to develop Dewpoint’s TAR DNA-binding protein 43 (TDP-43) small molecule condensate modulator (c-mod) for treating amyotrophic lateral sclerosis (ALS).

Beginning on December 1, 2024, Dewpoint will receive an initial payment and could potentially earn up to $480 million in milestone payments, contingent on achieving specific research and development goals. Should these milestones be met, Mitsubishi Tanabe will secure an exclusive option to license the program, assuming global responsibilities for clinical development and commercialization. Additionally, Dewpoint will receive tiered royalties based on the net sales of the treatment.

Dewpoint Therapeutics CEO Ameet Nathwani emphasized the importance of this partnership, highlighting the potential of their small molecule condensate modulator to address the underlying pathology of ALS and related neurodegenerative diseases through a unique mechanism of action. The c-mod was discovered and developed using Dewpoint’s innovative condensate-targeted platform. Nathwani pointed out that MTPC’s expertise in ALS, particularly as the developer of one of the few FDA-approved therapies for sporadic ALS, alongside their global clinical capabilities and extensive patient network, positions them as an ideal collaborator.

Dewpoint’s proficiency in condensate biology led to the identification of a small molecule capable of counteracting the mislocalization of TDP-43. This protein plays a critical role in neurodegenerative diseases and is found in over 97% of ALS cases. The company utilizes a high-throughput screening approach to identify a c-mod that can selectively correct TDP-43’s location, thereby restoring its normal function and improving ALS biomarkers in animal models. The treatment functions by addressing condensate dysfunction, known as condensatopathy, which subsequently reinstates TDP-43’s splicing activity and supports neuronal health.

Dewpoint Therapeutics was advised by Gordon Dyal & Co, acting as the exclusive financial advisor during the collaboration agreement.

In a related development, Dewpoint began a strategic partnership with Evotec in November 2023, focusing on advancing its oncology pipeline programs that include condensate modifying therapeutics.