Takeda Pharmaceuticals struck a deal with AcuraStem to purchase its amyotrophic lateral sclerosis (ALS) treatment program on Monday.

Per terms of the transaction, the Japanese pharma giant will develop and commercialize AcuraStems’ PIKFYVE-targeted therapeutics, including AS-202, an antisense oligonucleotide (ASO) for treating ALS.

The California-based biotech released promising data from preclinical studies related to its AS-202 program in late 2021, finding that it was a potent PIKFYVE suppressor with “no significant off-target effects” in human cells and was well-tolerated in rodent models at high doses. 

The deal could be worth nearly $600 million in upfront and milestone payments for AcuraStem if all clinical, regulatory and commercial milestones are achieved in addition to tiered royalties on potential net sales of any commercial products resulting from the license.

AcuraStem will be responsible for certain activities to advance AS-202’s enabling studies for submitting an Investigational New Drug application with the Food and Drug Administration while Takeda will be responsible for all other development activities.

In a press release announcing the deal, Takeda said it sees potential for AS-202 to tackle TDP-43 aggregation and boost TDP-43 function, which it called the “pathological hallmark” of ALS and certain forms of dementia.

“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,” stated Sarah Sheikh, BM BCh, MSc, MRCP, head of the neuroscience therapeutic area unit at Takeda.

Additionally, AcuraStem CEO Sam Alworth, MS, MBA said the company is eager to work with Takeda to pursue clinical development and commercial capabilities for the sake of patients in need.

The ALS treatment space has taken off in 2023 thanks to two medications receiving FDA approval within the past year.

In April, the FDA approved Qalsody (tofersen), an investigational drug developed by Biogen and Ionis Pharmaceuticals, to treat SOD1-mutation-mediated ALS, a rare genetic form of the disease.

Prior to the Qalsody greenlight, Amylyx Pharmaceuticals’ Relyvrio was approved by the FDA in 2022. In March, the pharma reported that Relyvrio had brought in full-product revenue of $22.2 million.