Takeda Inks $580M Deal to Co-Develop AcuraStem’s PIKFYVE-Targeting Antisense Oligonucleotide Therapy for ALS

▎WuXi

Edited by Kant Content Team

Recently, AcuraStem Incorporated announced that it has reached a licensing agreement with Takeda worth up to $580 million in total. The two parties will jointly develop and commercialize the company’s PIKFYVE-targeted therapies, including the innovative antisense oligonucleotide (ASO) therapy AS-202 for the treatment of amyotrophic lateral sclerosis (ALS).

PIKFYVE is a new therapeutic target for ALS and may also be applied to treat TDP-43 proteinopathies, such as frontotemporal dementia (FTD).AcuraStem Focuses on Developing Therapies Targeting PIKFYVE, Mainly Addressing Neurodegeneration by Clearing Toxic Protein Aggregates and Preserving Healthy Neuronal Function. AcuraStem Identified the Clinical Candidate AS-202 Through Screening on Its Patient-Based iNeuroRx Technology Platform.AS-202 is an ASO therapy administered via intrathecal injection, demonstrating potent inhibition of PIKFYVE in the central nervous system.

According to the terms of the agreement,Takeda to Receive Global Exclusive License for AcuraStem’s PIKFYVE Program. If all future clinical, regulatory, and commercial milestones are achieved within the term of the agreement, AcuraStem could receive upfront and milestone payments totaling up to approximately $580 million.AcuraStem will be responsible for assisting in advancing AS-202 into clinical trials, while Takeda will handle all other development activities, including clinical development, regulatory affairs, and global commercialization.

AcuraStem was co-founded in 2016 by Mr. Sam Alworth, Dr. Justin Ichida, Dr. Paul August, and Dr. Qing Liu. It is dedicated to continuously improving its patient-based iNeuroRx technology platform, discovering effective therapies for various neurodegenerative diseases, and rapidly advancing its treatment programs to patients, including its current SYF2 and UNC13A programs for the treatment of ALS and FTD.

References:

[1] AcuraStem Enters into a License Agreement with Takeda toAdvance PIKFYVE Therapeutics. Retrieved September 26, 2023 from https://acurastem.com/blog/acurastem-enters-license-agreement-takeda-advance-pikfyve-therapeutics