Distinctive machine learning platform combines proprietary disease models and screening techniques to reveal genetic insights that generate targets to transform ALS treatment

insitro, a machine learning-enabled drug discovery and development company, today announced it has received $25 million from Bristol Myers Squibb (NYSE: BMY) representing both the achievement of discovery milestones and the selection of the first novel target for amyotrophic lateral sclerosis (ALS) that was identified and validated by insitro. The companies signed a collaboration agreement in 2020 to discover new therapies for this disease that lacks any disease modifying treatment.

ALS is a devastating disease that occurs sporadically. It causes the progressive degeneration of nerve cells in the spinal cord and brain, leading to muscle spasms, impaired speech and gait, and eventual respiratory failure. Life expectancy from diagnosis is three to five years, and all cases are fatal.

To disentangle the complexity of ALS biology, insitro built and scaled a platform, using machine learning (ML), for drug discovery with three distinct proprietary elements, including:

• A collection of more than 200 engineered and patient ALS cell lines representing a comprehensive collection of ALS genetics and sporadic patients, complemented by a unique protocol for rapid, ML-enabled differentiation of motor neurons that model the disease;
• High-content imaging capabilities, fit-for-purpose for machine learning (ML), to identify disease mechanisms; and
• A proprietary, ML-enabled technology for pooled optical screening in human cells (POSH) to uncover genetic modifiers of disease-relevant cellular phenotypes.

Powered by this platform, researchers identified several novel gene targets that rescue functional deficits found in ALS patients. Knockdown of these targets reverted protein and RNA profiles that are found in ALS patient spinal cords, including proven markers of ALS pathology. These effects were also observed in cell lines capturing the genetics of ALS patients. The first credentialed target from the collaboration is now moving into drug discovery, while other targets continue to be studied.

“ALS is a grievous disease that causes immense suffering. Identifying potentially transformative targets with high conviction for such a complex disease is a testament to insitro’s state-of-the-art disease models and our cutting-edge cell-ML platform,” said Daphne Koller, Ph.D., founder and CEO of insitro. “Our team’s hard work and determination has been matched by the quality of our partnership with Bristol Myers Squibb scientists and leaders, who understood and invested in our vision early on. Thanks to both of those factors, our scientists were able to uncover meaningful insights and surface potentially viable targets that we hope will lead to meaningful treatments for patients in need.”

“Bristol Myers Squibb is committed to unlocking new frontiers in neuroscience, based on validated targets linked to causal human biology,” said Richard Hargreaves, Senior Vice President and Head of Bristol Myers Squibb’s Neuroscience Thematic Research Center. “We look forward to working with insitro to advance drug discovery for this novel target, as we strive to tackle some of the most pressing areas of unmet patient needs.”

With the selection of the first candidate target, the parties will continue to work closely to advance therapeutics targeted to this high value target and pathway. Bristol Myers Squibb will progress the program through clinical development and maintains the option for the selection of additional targets.

Under the terms of the collaboration agreement, insitro received a $50 million upfront payment and is eligible to receive up to more than $2 billion in further development, regulatory and commercial milestones in addition to royalty payments on net product sales.

About insitro

insitro is a machine learning-enabled drug discovery and development company creating a new approach for target and drug discovery. We integrate multimodal data from human cohorts and cellular models with the power of AI and machine learning. insitro is uncovering genetic targets and new therapeutic hypotheses, leveraging human and cell data to increase the probability of success. These insights provide the starting point for discovering new molecules, which we either build with our in-house, AI-enabled drug discovery platforms or with partners that extend our impact. With more than $700 million in capital raised to date, insitro is building a "pipeline through platform" with a focus on metabolic disease, neurodegeneration and oncology. Approaching the clinic, insitro aims to deploy its AI models to run smaller, better powered trials, enrolling the patients who can benefit most. Learn more at insitro.com

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