AcuraStem, a biotechnology company pioneering treatments for neurodegenerative diseases, entered into a license agreement with Takeda to develop and commercialize AcuraStem’s PIKFYVE targeted therapeutics including AS-202, an innovative antisense oligonucleotide (ASO) for the treatment of Amyotrophic Lateral Sclerosis (ALS).

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.

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.

Takeda will receive an exclusive, worldwide license to AcuraStem’s PIKFYVE program. AcuraStem will receive an upfront payment and milestones totaling up to approximately $580 million if all clinical, regulatory, and commercial milestones are achieved, plus royalties on potential sales of any commercial products resulting from this license.

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.

“Our mission at AcuraStem is to get our promising therapeutics to the patients who need them as quickly as possible,” said Sam Alworth, CEO of AcuraStem. “We are delighted to have secured a partner in Takeda who has the world class clinical development and commercial capabilities to deliver on our mission.”

“We aim to develop transformative treatments for some of society’s most debilitating neurological diseases including ALS,” said Sarah Sheikh, head, neuroscience therapeutic area unit at Takeda. “Whilst recent treatment advances have brought new hope to some patients, there remains a significant unmet need. 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. We look forward to building on AcuraStem’s work to progress this exciting science into development and to patients.”