The collaboration is looking to develop and commercialise several antisense oligonucleotides to treat genetically-defined neurological diseases, including Huntington’s disease, amyotrophic lateral sclerosis (ALS) – commonly referred to as Lou Gehrig’s disease –frontotemporal dementia (FTD) and spinocerebellar ataxia type 3 (SCA3).
Japanese pharma firm Takeda will pay an initial $110m (€89m) to Wave and buy $60m of shares in the Massachusetts-based company as part of the agreement. Takeda will also fund at least $60m of Wave research over a four-year period.
Antisense oligonucleotides are short, single strands of DNA or RNA molecules. Rather than modulating the activity of already-formed proteins, antisense oligonucleotides act before proteins are produced at the level of messenger RNA in the cell, thus opening up new opportunities for therapeutic intervention.
“Wave’s expertise in optimizing oligonucleotides offers a complementary approach to programs that Takeda is currently pursuing for neurological disorders, maximizing our potential for success, and their pipeline and focus are closely aligned with our own,” Daniel Curran, head of Takeda’s Center for External Innovation said.
Wave’s lead oligonucleotide candidates are WVE-120101 and WVE-120102, which selectively target the mutant allele of the huntingtin (HTT) gene. They are currently in Phase 1b/2a clinical trials.
Meanwhile WVE-3972-01 is set to be evaluated in clinical studies for the treatment of ALS and FTD by the end of 2018.