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AcuraStem and Takeda Form Licensing Agreement to Progress PIKFYVE Therapeutics

Tuesday, September 26, 2023

AcuraStem, a pioneering biotechnology firm dedicated to revolutionizing the development of treatments for neurodegenerative disorders, has announced a significant licensing agreement with Takeda. This partnership is intended to advance AcuraStem's targeted therapeutics focused on PIKFYVE, including AS-202, an innovative antisense oligonucleotide (ASO) developed for the treatment of Amyotrophic Lateral Sclerosis (ALS).

PIKFYVE is a novel therapeutic target with potential implications for ALS and other TDP-43 proteinopathy conditions such as Frontotemporal Dementia (FTD). AcuraStem's therapeutic strategy for PIKFYVE centers on combatting neurodegeneration by removing toxic protein aggregates and preserving the healthy function of neurons.

It was further developed utilizing AcuraStem's iNeuroRx® disease modeling platform, as documented in scientific publications (Shi Y et al Nat Med 2018, Hung S-T et al Cell 2023). AcuraStem's researchers have demonstrated that suppressing PIKFYVE through ASO treatment can restore motor function, reduce neurodegeneration, and enhance survival in multiple in vivo models of both ALS and FTD.

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

Sam Alworth, CEO of AcuraStem, expressed the company's commitment to bringing promising therapeutics to patients swiftly and expressed excitement about partnering with Takeda, leveraging their clinical development and commercialization capabilities.

AcuraStem will oversee specific activities related to advancing AS-202 IND enabling studies and assessing potential backup ASOs, while Takeda will take responsibility for all other development aspects, including clinical development, regulatory affairs, and global commercialization.

Sarah Sheikh, Head of Neuroscience Therapeutic Area Unit at Takeda, highlighted the potential of AS-202 to address unmet needs in neurological diseases like ALS, emphasizing its unique dual mechanism of action.

AcuraStem, co-founded by Alworth, Ichida, Dr. Paul August, and Dr. Qing Liu in 2016, remains dedicated to improving its patient-centered iNeuroRx® technology platform, exploring more effective therapeutic approaches across various neurodegenerative indications, and rapidly advancing therapeutic programs, including SYF2 and UNC13A programs for ALS and FTD.

Dr. Justin Ichida expressed satisfaction with the partnership and the progress made in targeting PIKFYVE as a therapeutic approach for ALS, acknowledging the contributions of philanthropic and government agencies in supporting this research.

Destum Partners served as the transaction advisor to AcuraStem, and Foley Hoag provided legal counsel to AcuraStem.

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