Immunome, Inc. (NASDAQ: IMNM) has dosed the first patient in a Phase 1, first-in-human trial of IM-3050, an investigational radioligand therapy targeting fibroblast activation protein (FAP)-expressing advanced solid tumors. The trial aims to evaluate the safety, tolerability, dosimetry, pharmacokinetics, and preliminary anti-tumor activity of IM-3050. FAP is expressed in 75% of solid tumors, making it a high-potential target for therapy.

The Phase 1 trial is an open-label, multicenter dose escalation and expansion study. The dose escalation portion will evaluate escalating repeated doses of IM-3050 to determine the maximum tolerated dose and/or recommended expansion dose. The expansion portion will further assess safety and tolerability at the recommended dose.

IM-3050 is designed to deliver radioactive lutetium-177 directly to FAP-expressing cells. Emitted beta particles may damage or kill nearby tumor cells through a bystander effect. Immunome is advancing a portfolio of targeted oncology therapies, including antibody-drug conjugates and radioligand therapies.

Key Trial Details

Immunome's pipeline includes varegacestat, IM-1021, IM-1617, and IM-3050. The company is committed to developing first-in-class and best-in-class targeted cancer therapies.

Immunome Inc. announced that the U.S. Food and Drug Administration has accepted its New Drug Application for varegacestat for the treatment of adults with desmoid tumors. The FDA assigned a Prescription Drug User Fee Act target action date of April 28, 2027. Varegacestat is an investigational, oral, once-daily gamma secretase inhibitor.

The acceptance marks a significant step for patients with desmoid tumors, a rare, non-metastatic soft tissue tumor that can cause debilitating pain and functional impairment. Immunome plans to submit a Marketing Authorization Application to the European Medicines Agency by the end of 2026.

RINGSIDE Phase 3 Trial Results

The NDA is supported by data from the Phase 3 RINGSIDE trial, which evaluated varegacestat in patients with progressing desmoid tumors. The global, randomized, double-blind, placebo-controlled trial met its primary endpoint of progression-free survival.

Key findings from the trial include:

The trial demonstrated a statistically significant and clinically meaningful 84% reduction in the risk of disease progression or death compared to placebo. All key secondary endpoints were met, including objective response rate and change in tumor volume. Varegacestat also showed statistically significant improvement in worst pain intensity at week 12.

Safety Profile

Varegacestat was generally well tolerated with a manageable safety profile consistent with the gamma secretase inhibitor class. The most common adverse events in the treatment arm were diarrhea, fatigue, rash, nausea, and cough. The majority of events were grade 1 or 2.

The RINGSIDE trial randomized 156 patients to receive varegacestat 1.2 mg daily or placebo until disease progression or death. An open-label extension phase of the trial is ongoing.