Bristol Myers Squibb Grants $25M for insitro’s Milestone in ALS Target Discovery

insitro has secured $25 million in milestone payments from Bristol Myers Squibb for reaching discovery milestones and identifying the first novel genetic target for amyotrophic lateral sclerosis. This achievement builds on a five-year collaboration agreement established in 2020, focused on leveraging machine learning (ML) and experimental platforms to discover transformative therapies for ALS and frontotemporal dementia (FTD).

The collaboration, which began with a $50 million upfront payment to insitro, applies the company’s proprietary insitro Human platform. This platform uses ML, human genetics, and functional genomics to create predictive in vitro models that provide insights into disease progression, identify patient segments, and discover novel targets. Bristol Myers Squibb oversees clinical development and commercialization, with potential payments to insitro exceeding $2 billion across discovery, development, regulatory, and commercial milestones, plus royalties on net product sales.

ALS is a neurodegenerative disease characterized by progressive nerve cell degeneration, leading to impairments in movement, speech, and breathing. Life expectancy post-diagnosis is just 3-5 years. To tackle the complexities of ALS biology, insitro scaled its ML platform, which features engineered and patient-derived ALS cell lines, high-content imaging capabilities, and a proprietary pooled optical screening technology (POSH). These tools enabled the discovery of novel genetic targets that reverse pathological markers in ALS patient cell models.

The first validated target is now advancing into drug discovery, while additional targets are under study.

Daphne Koller, Ph.D., CEO of insitro:

“Neurodegenerative disorders like ALS and FTD have historically been a challenging therapeutic area, with no disease-modifying treatments today... Our scientists uncovered meaningful insights and surfaced potentially viable targets that we hope will lead to meaningful treatments for patients in need.”

Richard Hargreaves, Senior Vice President at BMS:

“There is an unmet medical need for therapies to treat ALS and FTD. We are excited by the prospect of working with insitro’s team towards our shared goal of identifying transformative treatments for patients with these devastating diseases.”

Topics: AI & Digital