Exelixis licenses AI-designed cancer drug from Insilico Medicine

By Jonathan Smith

- Last updated on GMT

© Getty Images
© Getty Images

Related tags Small molecule Cancer Oncology

Insilico Medicine is due to receive $80 million upfront plus potential milestone payments as Exelixis gains global rights to develop and commercialize the Hong Kong firm’s small molecule cancer treatment.

According to the company release, Insilico expects to receive the upfront payment from Exelixis in late 2023. The milestone payments depend on future development, commercial and sales goals, and Insilico can expect tiered royalties on potential sales of the drug.

The licensed candidate, ISM3091, is designed to be a potentially best-in-class blocker of the protein Ubiquitin Specific Peptidase 1 (USP1), which helps cells repair damage to their DNA. Healthy cells would be spared by the drug because they have other ways to repair their DNA. However, tumor cells with a mutation in their DNA repair machinery such as BRCA can be killed by this treatment: an approach known as synthetic lethality.

Insilico designed ISM3091 with the help of its artificial intelligence (AI)-guided drug discovery technology, which scans multi-omics datasets for potential drug targets and identifies molecules that can block them.

According to Insilico, ISM3091 was effective at killing tumor cells in preclinical studies, including when combined with existing synthetic lethality drugs called PARP inhibitors. The drug may be able to tackle a range of tumors with a mutation in BRCA, including ovarian, prostate and breast cancer. Earlier this year, the company cleared a way to clinical trials of the drug with the U.S. Food and Drug Administration (FDA).

“We believe preclinical data on ISM3091’s potent anti-tumor activity, tolerability, and pharmacokinetics set the compound apart from competing USP1 inhibitors and make it an important addition to Exelixis’ growing clinical-stage pipeline,” said Dana Aftab, Exelixis’ executive vice president, discovery and translational research and chief scientific officer, in a public statement.

Insilico now has several clinical-stage candidates in its pipeline, with the lead program in phase 2 development for the treatment of idiopathic pulmonary fibrosis (IPF). The company is also working with Fosun Pharma to develop an immunotherapy drug for cancer in phase 1 testing.

In 2021, the startup raised a huge Series C round worth $255 million to finance the development of its technology. The company then followed up in 2022 with a $95 million Series D round and a subsequent research collaboration deal with Sanofi worth up to $1.2 billion.

This deal is one of many happening with players in AI-driven drug discovery. Recent examples include AstraZeneca’s Alexion teaming up with Verge Genomics and Johnson & Johnson’s company Janssen tapping into Intellomx.

Related topics Clinical Development Drug Delivery

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