Insitro and BMS Team Up to Pave the Way for New ALS and FTD Treatments

Daphne Koller and Mary Rozenman Insitro

Insitro founder and CEO Daphne Koller (left) and CFO Mary Rozenman (right). Photo courtesy of Insitro.

San Francisco-based Insitro announced today that it has entered a five-year discovery collaboration agreement with Bristol Myers Squibb to discover and develop novel therapies for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

Through this collaboration, Insitro will utilize its proprietary platform, Insitro Human (ISH), to create induced pluripotent stem cell (iPSC) derived disease models for both diseases. This platform applies machine learning, human genetics and functional genomics to create predictive in vitro models. ISH can potentially provide insight into how these diseases progress within patients. Bristol Myers Squibb will have the option to select from targets identified by Insitro to advance through clinical development and commercialization.

“Neurodegenerative disorders like ALS and FTD have historically been a challenging therapeutic area, with no disease modifying treatments today. We are excited to partner with Bristol Myers Squibb and its world-class neuroscience leaders, who share our vision of leveraging human genetics, machine learning, and high-throughput biology and chemistry in order to identify and provide new treatments for patients suffering from these devastating diseases,” said Daphne Koller, founder and chief executive officer of Insitro. “Since founding Insitro just over two years ago, we have demonstrated our capabilities in building predictive models to discover novel targets and patient segments. We have also developed new approaches to machine-learning-enabled therapeutics design, which we look forward to deploying to discover treatments for novel targets emerging from this collaboration.”

Insitro is set to receive $50 million as an upfront payment, and it will be eligible to receive an additional $20 million in near term operational milestones.

“We believe that machine learning and data generated by novel experimental platforms offer the opportunity to rethink how we discover and design novel medicines,” said Richard Hargreaves, Ph.D., senior vice president, head of neuroscience TRC research and early development, Bristol Myers Squibb. “There is an unmet medical need for therapies to treat ALS and FTD and 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.”

Insitro recently strengthened its machine learning-based drug discovery capabilities through the acquisition of Haystack Sciences back on Oct. 22. Haystack focuses on synthesizing, breeding and analyzing large, diverse combinatorial chemical libraries encoded by unique DNA sequences called DNA-encoded libraries (DELs). Insitro intends on leveraging the DEL technology to collect massive small molecule data.

“We are thrilled to have the Haystack team join Insitro,” Koller said at the time of the announcement. “For the past two years, Insitro has been building a company focused on the creation of predictive cell-based models of disease in order to enable the discovery of novel targets and evaluate the benefits of new or existing molecules in genetically defined patient segments. This acquisition enables us to expand our capabilities to the area of therapeutic design and advances us towards our goal of leveraging machine learning across the entire process of designing and developing better medicines for patients.”

Haystack’s platform combines several elements, including the capability to synthesize small molecule collections. With these advantages, Insitro will be better equipped to develop multi-dimensional predictive models for small molecule design.

“I am excited by the opportunity to join a company with such a uniquely open and collaborative culture and to work with and learn from colleagues in data science, machine learning, automation and cell biology,” said Richard E. Watts, co-founder and chief executive officer of Haystack Sciences. “The capabilities enabled by joining our efforts are considerably greater than the sum of the parts, and I look forward to helping build core drug discovery efforts at Insitro.”

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