AcuraStem, a biotechnology company focused on developing treatments for neurodegenerative diseases, has entered into a licensing agreement with Takeda to collaborate on the development and commercialization of AcuraStem's PIKFYVE targeted therapeutics, including AS-202, an antisense oligonucleotide (ASO) designed for the treatment of Amyotrophic Lateral Sclerosis (ALS).

PIKFYVE is a novel therapeutic target for ALS and may also have relevance in treating other TDP-43 proteinopathies like Frontotemporal Dementia (FTD). AcuraStem's therapeutic approach to PIKFYVE aims to combat neurodegeneration by eliminating toxic protein aggregates and safeguarding healthy neuronal function.

This innovative therapeutic mechanism was initially discovered by Dr. Justin Ichida, one of AcuraStem's co-founders, using patient-derived disease models. It is exclusively licensed to AcuraStem by the USC Stevens Center for Innovation and has been further developed on AcuraStem's iNeuroRx® disease modeling platform. Researchers at AcuraStem have shown 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.

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

AcuraStem will be responsible for specific activities to advance AS-202, such as IND-enabling studies and characterizing potential backup ASOs. Meanwhile, Takeda will handle all other development aspects, including clinical development, regulatory affairs, and global commercialization.

This partnership reflects AcuraStem's mission to bring promising therapeutics to patients as efficiently as possible. Takeda's extensive clinical development and commercialization capabilities make them a valuable partner in advancing AcuraStem's work in developing treatments for ALS and other neurodegenerative diseases.

Sarah Sheikh, Head of the Neuroscience Therapeutic Area Unit at Takeda, noted that AS-202 holds potential as a transformative treatment for ALS and other TDP-43 proteinopathies. These conditions have significant unmet needs, and AS-202's dual mechanism of action, addressing TDP-43 aggregation and improving TDP-43 function, could offer promising solutions. Takeda is excited to collaborate with AcuraStem to advance this science into development and ultimately to patients.

AcuraStem is dedicated to continually improving its patient-based iNeuroRx® technology platform and advancing therapeutic programs for neurodegenerative diseases, including existing programs for ALS and FTD. Justin Ichida, PhD, who identified PIKFYVE as a therapeutic target for ALS, expressed his satisfaction with the partnership and AcuraStem's progress in developing molecules to target PIKFYVE in humans. He emphasized the power of using modern approaches and acknowledged the support received from philanthropic and government agencies in understanding the therapeutic potential of targeting PIKFYVE.

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