AcuraStem Enters Into License Agreement With Takeda to Advance PIKFYVE Therapeutics

AcuraStem recently announced it has 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.

This novel therapeutic mechanism was initially discovered at the lab of AcuraStem’s co-founder, Justin Ichida, PhD, using patient-derived disease models, is exclusively licensed to AcuraStem by the USC Stevens Center for Innovation, and was further developed on AcuraStem’s iNeuroRx disease modeling platform (Shi Y et al Nat Med 2018, Hung S-T et al Cell 2023). 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.

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.

“Our mission at AcuraStem is to get our promising therapeutics to the patients who need them as quickly as possible,” said Sam Alworth, MS, MBA, 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.”

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, BM BCh, MSc, MRCP, Head, 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. We look forward to building on AcuraStem’s work to progress this exciting science into development and to patients.”

AcuraStem, co-founded by Alworth, Ichida, Paul August, PhD, and Qing Liu, PhD, in 2016, remains committed to continually improving its patient-based iNeuroRx technology platform, discovering ever more effective therapeutic approaches across neurodegenerative indications, and rapidly advancing its therapeutic programs to patients including its existing SYF2 and UNC13A programs for ALS and FTD.

“I’m very pleased that AcuraStem is partnering with Takeda around their PIKFYVE program,” said Justin Ichida, PhD. “We originally identified PIKFYVE as a novel therapeutic target for ALS using nerve cells derived from patient stem cells, and this illustrates the power of using this modern approach. I am grateful for the funding and help we received from philanthropic and government agencies that enabled us to better understand the therapeutic potential of targeting PIKFYVE. AcuraStem has done an outstanding job of generating best-in-class molecules to target PIKFYVE in humans, and I am excited to see this move forward.”

AcuraStem identified clinical candidate AS-202 through screening on its iNeuroRx® patient-based technology platform. 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.

AcuraStem is a patient-based biotechnology company pioneering treatments for neurodegenerative diseases including sporadic ALS and FTD. AcuraStem’s best-in-class disease modeling platform, iNeuroRx, enables the discovery of innovative, effective, and broadly acting treatments. The team’s strong expertise in ASO technology provides for the rapid advancement of treatments to the clinic. AcuraStem’s research is funded in part by support from the Alzheimer’s Drug Discovery Foundation, Harrington Discovery Institute, Alzheimer’s Association, Rainwater Charitable Foundation, Department of Defense and the National Institute of Neurological Disorders and Stroke. For more information, visit acurastem.com.