Takeda Puts Nearly $600M on the Line for AcuraStem’s ALS Program

Takeda US_iStock, hapabapa

Pictured: Takeda office in Cambridge, Massachusetts/iStock, hapabapa

Takeda on Monday inked a development and commercialization agreement with California-based biotech AcuraStem to target amyotrophic lateral sclerosis and other neurodegenerative diseases.

Under the terms of the deal, Takeda will pledge upfront and milestone payments of up to approximately $580 million if the collaboration meets all future clinical, regulatory and commercial milestones. AcuraStem will also be eligible to receive tiered royalties on potential net sales of any product that emerges from the partnership.

In return, the Japanese multinational pharma will gain access to AcuraStem’s PIKFYVE program, which includes AS-202, an investigational antisense oligonucleotide (ASO) and the biotech’s most mature candidate. Takeda will have exclusive worldwide license to AS-202 as well as to AcuraStem’s other PIKFYVE-directed assets.

AcuraStem will take charge of certain studies to enable an Investigational New Drug application for AS-202, while Takeda will be responsible for all development activities, including clinical studies, regulatory efforts and commercialization, according to the announcement.

PIKFYVE is an emerging therapeutic target in amyotrophic lateral sclerosis (ALS), according to AcuraStem’s website. The company’s studies have shown that using an ASO therapeutic to inhibit the gene that encodes for the kinase helps neurons discard toxic proteins and pathologic aggregates. In turn, this restores healthy neuron function and prevents neurodegeneration.

This mechanism of action also points to the potential of AcuraStem’s PIKFYVE approach to frontotemporal dementia (FTD) and other diseases caused by the misfolding, and eventual clumping, of the protein TDP-43.

In many pre-clinical models of ALS and FTD, using PIKFYVE-targeted ASOs, such as AS-202, can reverse the pathological protein deposition in neurons, according to AcuraStem.

In a statement, Sarah Sheikh, head of Takeda’s Neuroscience Therapeutic Area Unit, said that “through its unique dual mechanism of action, which addresses TDP-43 aggregation and improves TDP-43 function,” AS-202 has the potential to address the existing unmet need among patients with “debilitating neurological diseases.”

Takeda has also inked other high-value deals this year. In July 2023, the company signed a strategic discovery collaboration and licensing agreement with British biotech F-star Therapeutics to discover next-generation and multi-specific antibodies for cancer targets. Though the complete financial details of the deal were not revealed, the companies said that F-star could receive up to $1 billion in milestones.

In January, Takeda entered into another potential billion-dollar deal with Hutchmed to develop fruquintinib, a VEGF inhibitor being trialed for colorectal cancer.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. He can be reached at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

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