Immunome Clears Key Regulatory Milestone as Lead Drug Goes Under FDA Review

Immunome Inc. (NASDAQ:IMNM) said the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application for varegacestat, an investigational oral, once-daily gamma secretase inhibitor, for the treatment of adults with desmoid tumors.

The agency assigned a Prescription Drug User Fee Act target action date of April 28, 2027.

The NDA is supported by data from the Phase 3 RINGSIDE trial, which evaluated varegacestat in patients with progressing desmoid tumors.

Trial Met Primary And Secondary Endpoints

According to the company, the registrational study achieved its primary endpoint by significantly improving progression-free survival compared with placebo.

Patients treated with varegacestat experienced an 84% reduction in the risk of disease progression or death versus placebo, corresponding to a hazard ratio of 0.16.

The trial also met all key secondary endpoints. Varegacestat delivered an objective response rate of 56%, compared with 9% for placebo, based on assessments by a blinded independent central review.

Pain And Tumor Volume Findings

Immunome said the treatment also demonstrated a statistically significant improvement in worst pain intensity by week 12. A clinically meaningful difference was observed as early as the first assessment at week 4.

In an exploratory analysis, patients receiving varegacestat recorded a median best change in tumor volume of negative 83%, compared with a positive 11% change in the placebo group.

Safety Profile

The company said varegacestat was generally well tolerated and demonstrated a manageable safety profile that was consistent with the gamma secretase inhibitor class.

Desmoid tumors are aggressive non-metastatic soft tissue tumors that are prone to recurrence. In the U.S., approximately 1,000-1,650 people are diagnosed with desmoid tumors each year, with approximately 10,000-11,000 actively managed patients.

In June, Immunome dosed the first patient in the Phase 1, first-in-human trial of IM-1617, a potential first-in-class ADC directed at an undisclosed solid tumor target and incorporating HC74, Immunome’s TOP1 inhibitor payload.

The Phase 1 trial is designed to determine the safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity of IM-1617.

The study is expected to include participants with advanced solid tumors, including colorectal cancer, non-small cell lung cancer, and breast cancer.

IMNM Price Action: Immunome shares were down 1.07% at $24.13 at the time of publication on Wednesday, according to Benzinga Pro data.

Image via Shutterstock/ Aunt Spray