Bristol Myers bags FDA tumor-agnostic nod for Augtyro to challenge Bayer, Roche

A Bristol Myers Squibb drug has got an FDA approval to treat tumors with a specific biomarker regardless of their locations in the body.

The drug, Augtyro, has won the FDA’s accelerated approval in solid tumors that have an NTRK gene fusion, Bristol Myers said Thursday. The tyrosine kinase inhibitor (TKI) can be used in patients 12 years of age and older, but the tumors must have been previously treated or have no satisfactory alternative therapy.

This tumor-agnostic indication is familiar to the oncology community. Back in 2018, Bayer’s Vitrakvi became the first targeted therapy to get its initial FDA go-ahead for cancers based on a genetic biomarker rather than where the tumor originated, and the indication was NTRK gene fusion. Then Roche’s Rozlytrek followed with the same label addition in 2019.

BMS got Augtyro from its $4.1 billion acquisition of Turning Point Therapeutics in 2022, and then in November 2023 pushed the drug across the FDA finish line in ROS1-positive non-small cell lung cancer, where it’s also competing with Roche’s Rozlytrek.

In NTRK mutation-positive solid tumors, Augtyro differentiates itself as the only option that has been studied both in patients who had not tried a TKI before and in TKI-experienced patients, Bristol Myers said.

In the phase 1/2 TRIDENT-1 trial, Augtyro triggered a response in 58% of 40 TKI-naïve patients, including 15% who had complete responses. Of those who responded, 83% were still in response after one year.

Among 48 TKI-pretreated patients, Augtyro’s overall response rate was 50%, which included no complete response. At one year, 42% of responding patients were still in remission. The median duration of response was 9.9 months.

Across the two subgroups, the TRIDENT-1 trial enrolled patients with 15 different types of cancer, including NSCLC, thyroid cancer, salivary gland cancer, glioblastoma, colorectal cancer and others.

With the accelerated approval, BMS will need to provide additional data to confirm Augtyro’s benefits. To convert the nod into a full approval, the FDA is asking for tumor response and duration of response data from “a sufficient number of patients” across tumor types, with all responding patients being followed for at least 12 months from the onset of response or until disease progression, according to the agency.

The FDA’s request for Augtyro is similar to those for Vitrakvi and Rozlytrek, neither of which has converted their accelerated approvals.

Although a tumor-agnostic label covers tumors at different locations, NTRK gene fusions are rare. Last year, Rozlytrek only got Roche 86 million Swiss francs ($96 million) in sales across its two indications. Bayer didn’t specify Vitrakvi’s sales in its annual report.

Augtyro marked Bristol Myers Squibb’s initial expansion into targeted therapies from an immunotherapy-focused oncology portfolio. The New Jersey pharma went deeper into targeted therapies with the acquisition of Mirati Therapeutics, which brought with it the KRAS inhibitor Krazati.