Totus Medicines is targeting a common cancer mutation in a new Phase I trial with its sights set on an accelerated approval.

Totus CEO Neil Dhawan

On 13 April, the Emeryville, California-based Totus announced the first patient was dosed in a 241-patient Phase I trial targeting solid tumours with the PI3Kα mutation. Built into the study is an accelerated approval strategy that allows any successful cohort to expand into a potentially registrational study, CEO Neil Dhawan, PhD, told Clinical Trials Arena.

The Phase I study (NCT05683418) tests TOS-358 as a monotherapy in cohorts of patients with a range of tumour types, including breast cancer, colorectal cancer, and non-small cell lung cancer. If a specific cohort shows initial efficacy, Totus will expand that cohort into a single-arm, open-label pivotal trial that could ultimately garner accelerated approval, Dhawan said. This is a similar strategy to how Amgen approached the accelerated approval of its cancer drug Krazati (adagrasib), he added.

Totus presented preclinical data showing TOS-358 is a covalent inhibitor of PI3Kα, at the recently concluded American Association for Cancer Research (AACR) Annual Meeting held April 14–19. PI3Kα mutations are the root cause of around 15% of all cancers.

Totus drug discovery platform

Totus is one of many pharma companies leveraging artificial intelligence (AI) to accelerate the drug discovery process. Dahwan said the Totus Accel Platform, which employs cell-based screening and AI machine learning, led to the discovery of TOS-348 in only around four months.

“Finding a highly effective drug is like winning the lottery,” Dhawan said. “If you can buy 1,000 times the number of tickets—or screen 1,000 times as many therapeutic targets—you’re going to win a lot more and a lot faster.”

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Overall, experts say the surge in AI-based drug discovery is only beginning, with its peak likely years away. Meanwhile, regulators have begun to more closely scrutinize the increasingly prominent technology.