Transition Bio and Voyager Therapeutics have entered into a drug discovery collaboration and licence option agreement. The companies will collaborate to discover and develop novel, selective small molecules for people suffering from Amyotrophic Lateral Sclerosis (ALS) and frontotemporal dementia (FTD) with TDP-43 pathology. TDP-43 pathology is common in many neurodegenerative diseases, including over 90 percent of ALS cases and up to 45 percent of FTD cases.

Under the terms of the partnership, Transition Bio is responsible for the discovery and optimisation of small molecules targeting TDP-43 until nomination of a development candidate, upon which Voyager will have an option to license the worldwide exclusive rights to develop and commercialise the programme. Transition Bio has received a single-digit million-dollar upfront payment and is eligible to receive potential research, development, commercial and net sales milestone payments totaling up to USD 500M. The company is also eligible for high single-digit to low double-digit royalties on net sales.

“This collaboration with Transition Bio fits into Voyager’s vision of building a multi-modality neurotherapeutic pipeline that matches the optimal modality to each target. Historically, TDP-43 has been difficult to address therapeutically because of the complexity of targeting toxic forms of the protein without impacting the non-toxic forms that are necessary to the cell. Transition Bio’s molecular condensate technology uniquely identifies small molecules that aim to precisely correct the mislocalisation of TDP-43 without abolishing its important functional activity,” said Alfred W Sandrock, Jr, MD, PhD, President and Chief Executive Officer (CEO), Voyager and member of the Transition Bio Board of Directors.

Adding to it, G Kelly Martin, Executive Chairman, Transition Bio, also said, “By working closely with the Voyager team, we will be able to leverage a world-leading translational team with deep expertise in ALS and FTD. We look forward to advancing this programme together to achieve our shared goal of transforming the lives of patients with these devastating diseases.”