ADDF & FFBW Funded Biotech Agrees to License Agreement with Takeda Pharmaceuticals Focused on PIKfyve Therapeutics

AcuraStem, a California-based biotech firm whose innovative antisense oligonucleotide (ASO) the Foundation For a Better World (FFBW) funded in partnership with the Alzheimer’s Drug Discovery Foundation (ADDF), announced the signing of a licensing deal with Takeda to develop and commercialize its PIKfyve targeted therapeutics. These therapeutics include the ASO, AS-202, AcuraStem’s most mature drug candidate in treating amyotrophic lateral sclerosis (ALS). 

PIKfyve is an emerging treatment target in ALS and other neurodegenerative diseases featuring TDP-43 proteinopathies, like frontotemporal dementia (FTD). When inhibited, PIKfyve activates a protein clearance mechanism that discards toxic proteins. This clearance contributes to the restoration of neuron function and prevents neurodegeneration. 

Takeda, the global biopharmaceutical company based in Japan, pledged upfront and milestone payments of up to approximately $580 million if the partnership meets all future clinical, regulatory and commercial milestones. In exchange, Takeda receives exclusive licensing worldwide to AS-202 and other AcuraStem PIKfyve-targeting assets. 

AcuraStem will spearhead studies to enable an investigational new drug application (IND) for AS-202, while Takeda will lead development activities such as clinical studies, regulatory efforts and eventual commercialization. 

The FFBW is excited about the potential of such a partnership and the benefits of PIKfyve therapeutics.