RARE Daily

Takeda Licenses ALS Therapy from AcuraStem for up to $580 Million

September 26, 2023

Rare Daily Staff

Takeda has entered into a license agreement with AcuraStem to develop and commercialize the company’s targeted therapeutics including AS-202, an antisense oligonucleotide for the treatment of the rare, neurodegenerative condition amyotrophic lateral sclerosis.

Under the terms of the agreement, Takeda will receive an exclusive, worldwide license to AcuraStem’s PIKFYVE program. AcuraStem will receive an upfront and milestone payments totaling up to approximately $580 million if all future clinical, regulatory, and commercial milestones are achieved during the term of the agreement plus tiered royalties on potential net sales of any commercial products resulting from this license.

AS-202 is an intrathecally delivered ASO that potently suppresses PIKFYVE in the central nervous system and addresses neurodegeneration by expelling toxic protein aggregates and restoring healthy neuronal function in in vivo preclinical models. PIKFYVE is a novel therapeutic target for ALS and may also be relevant for treating additional TDP-43 proteinopathies such as frontotemporal dementia (FTD). AcuraStem’s therapeutic strategy for PIKFYVE focuses on addressing neurodegeneration by expelling toxic protein aggregates and protecting healthy neuronal function.

This novel therapeutic mechanism was initially discovered at the lab of AcuraStem’s co-founder, Justin Ichida using patient-derived disease models. AcuraStem exclusively licensed it from the USC Stevens Center for Innovation. The AcuraStem researchers further demonstrated that ASO-mediated suppression of PIKFYVE can restore motor function, reduce neurodegeneration, and improve survival in multiple in vivo models of both ALS and FTD.

AcuraStem will be responsible for certain activities to help advance AS-202 IND enabling studies and characterizing potential backup ASOs. Takeda will be responsible for all other development activities including clinical development, regulatory affairs, and global commercialization.

“We aim to develop transformative treatments for some of society’s most debilitating neurological diseases including ALS. Whilst recent treatment advances have brought new hope to some patients, there remains a significant unmet need,” said Sarah Sheikh, head of neuroscience therapeutic area unit at Takeda. “We believe AS-202 has the potential to address this unmet need through its unique dual mechanism of action, which addresses TDP-43 aggregation and improves TDP-43 function, the pathological hallmark of ALS and other TDP-43 proteinopathies including certain forms of dementia.”

Photo: Sarah Sheikh, head of neuroscience therapeutic area unit at Takeda


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