$25 Million Milestone Reached in Collaboration with Bristol Myers Squibb Unlocks First Novel ALS Target
Key Highlights
- insitro earns $25 million milestone payment from Bristol Myers Squibb for selecting first AI-discovered novel ALS target
- AI-enabled platform integrates disease models, patient-derived cells, and machine learning to uncover genetic modifiers of ALS
- First target now advancing into drug discovery, with potential for additional targets and over $2 billion in future milestone payments
insitro and Bristol Myers Squibb Achieve Major ALS Discovery Milestone
insitro, a machine learning-driven drug discovery company, announced it has received $25 million from Bristol Myers Squibb following key achievements in their collaboration on ALS (amyotrophic lateral sclerosis). The payment recognizes both scientific discovery milestones and the selection of the first novel ALS target identified through insitro’s AI platform.
ALS is a fatal neurodegenerative disease with limited treatment options. To address this challenge, insitro built a next-generation discovery engine powered by three core innovations:
- Over 200 engineered and patient-derived ALS cell lines reflecting both genetic and sporadic forms of ALS
- High-content imaging pipelines optimized for machine learning to uncover cellular disease mechanisms
- POSH (Pooled Optical Screening in Human cells), a proprietary technology enabling high-throughput screening of genetic modifiers in motor neurons
This integrated platform revealed several previously unknown genes that, when inhibited, reversed ALS-like phenotypes at the RNA and protein levels. The selected target is now being advanced by Bristol Myers Squibb into the next stage of drug discovery, with additional targets under exploration.
A Platform for High-Conviction Targets in ALS
Daphne Koller, Ph.D., CEO and founder of insitro, emphasized the importance of the achievement:
“ALS is one of the most devastating and complex diseases. Our ability to identify validated, high-confidence targets is a testament to the strength of our ML-enabled disease modeling and the partnership with Bristol Myers Squibb, who believed in this vision from the beginning.”
Richard Hargreaves, SVP and Head of Neuroscience Research at Bristol Myers Squibb, added:
“We’re committed to pioneering new approaches in neuroscience. insitro’s unique platform aligns with our goal of developing treatments rooted in human biology.”
Future Outlook and Strategic Potential
The partnership will continue with Bristol Myers Squibb progressing the lead target into clinical development. The company retains options for additional target nominations.
The original 2020 agreement provided insitro with a $50 million upfront payment and access to over $2 billion in potential milestone payments, plus royalties on future sales.
About insitro
insitro is transforming drug discovery through the integration of machine learning, human biology, and high-throughput cellular data. By applying AI to patient-derived data, insitro uncovers validated genetic targets to drive precision medicine across neurodegeneration, metabolic disease, and oncology. The company has raised over $700 million and is building a platform-first drug development model.
Website: https://insitro.com
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