Insitro, Bristol Myers extend research into new drug for ALS
Companies identified novel disease target during first phase of collaboration
Bristol Myers Squibb is extending a research collaboration with the artificial intelligence company Insitro to design new medicines against a novel target in amyotrophic lateral sclerosis (ALS).
The target was identified during the previous five-year agreement between the companies, which aimed to discover new molecules that could lead to next-generation therapies for ALS. That collaboration would have ended this month, but it is being extended for one more year to give the companies more time to explore a novel ALS target.
With the new extension, Insitro may receive up to $20 million in new funding and may be eligible for up to $2 billion in discovery, development, regulatory, and commercial milestone payments, in addition to royalty payments if a new therapy for ALS successfully reaches the market.
“Our collaboration with Bristol Myers Squibb has uncovered novel targets with potential to address the underlying biology of ALS,” Daphne Koller, PhD, founder and CEO of Insitro, said in a company press release. “We are now moving into the next phase — turning these discoveries into medicines. Our aim remains unwavering: to deliver truly transformative treatments that enable people with ALS to live longer.”
Insitro’s AI-based platform helps identify ALS drug targets quickly
ALS is caused by the progressive loss of motor neurons, the nerve cells that control voluntary movements. Based on whether an individual has a family history of the disease, it can be classified as familial or sporadic ALS.
The initial phase of the collaboration focused on identifying underlying disease mechanisms that were shared by most ALS cases to help develop therapies that could treat the largest number of patients.
Insitro developed cellular models of ALS derived from induced pluripotent stem cells (iPSCs). These are obtained from a person’s skin or blood cells and reprogrammed back into a stem cell-like state, providing a source of any type of human cell. These iPSCs could either be derived from ALS patients or genetically modified to carry disease-causing mutations.
Using Insitro’s artificial intelligence-based platform, the companies identified “multiple differentiated, high-confidence novel ALS drug targets at record speed,” according to Philip Tagari, Insitro’s chief scientific officer. “These targets are supported by robust evidence, including functional data that demonstrates motor neuron survival and the reversal of multiple downstream markers of ALS pathology.”
Under the agreement, Bristol Myers had the option to select a number of targets identified by Insitro’s platform and advance them through clinical trials. The company exercised that option in December, and selected one target for further testing. This triggered a $25 million milestone payment to Insitro.
While advancing these initial drug candidates, we will continue our efforts to identify additional new targets that have the potential to be disease-modifying.
Now, Insitro’s platform will be used to design new medicines for an identified ALS target. The companies did not disclose, however, whether this target is the same that was selected last year by Bristol Myers.
The platform can generate data from hundreds of millions of drug-target binding. Advanced machine learning models are then used to predict the drugs’ pharmacological properties.
The platform combines computational predictions with cellular experiments and learns every time it drives a cycle of drug design, production, and testing. Thus, AI can guide each round of drug synthesis, with the goal of designing a new therapeutic molecule that is safe and effective.
With this “end-to-end drug design platform, we can translate novel targets into advanced small-molecule leads rapidly, leveraging a differentiated set of capabilities that spans AI-driven modeling, medicinal chemistry, and structural biology,” Koller said. “While advancing these initial drug candidates, we will continue our efforts to identify additional new targets that have the potential to be disease-modifying.”
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