Immunome Announces EMA Validation of Marketing Authorization Application for Varegacestat for the Treatment of Adults with Desmoid Tumors

The application advances to European review following a Phase 3 trial that met its primary and all key secondary endpoints.

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• The Marketing Authorization Application (MAA) is supported by positive Phase 3 RINGSIDE results demonstrating statistically significant improvements across the primary and all key secondary endpoints
• If approved, varegacestat could provide a meaningful new oral treatment option for adults with desmoid tumors in the European Union (EU)

BOTHELL, Wash.--(BUSINESS WIRE)-- Immunome, Inc. (Nasdaq: IMNM), a clinical-stage targeted oncology company committed to developing first-in-class and best-in-class targeted cancer therapies, today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for varegacestat, an investigational, oral, once-daily gamma secretase inhibitor (GSI), for the treatment of adults with desmoid tumors. Validation confirms that the application is complete, and the EMA will initiate review under the centralized marketing authorization procedure.

“The EMA's validation of our MAA for varegacestat is an important step in our efforts to bring this treatment to patients with desmoid tumors globally,” said Clay Siegall, Ph.D., President and Chief Executive Officer of Immunome. “The application is supported by the robust efficacy and safety data generated from the Phase 3 RINGSIDE trial. There is a strong need for new treatment options for desmoid tumors, and we look forward to the potential to make varegacestat available to patients living with this challenging disease.”

The MAA is supported by results from the global, randomized, double-blind, placebo-controlled Phase 3 RINGSIDE trial evaluating varegacestat in adults with progressing desmoid tumors. Detailed efficacy and safety data from the study were presented at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting. The trial met its primary endpoint and achieved all key secondary endpoints, with key findings including:

• The registrational trial met its primary endpoint of improving progression-free survival vs. placebo, with a statistically significant and clinically meaningful 84% reduction in the risk of disease progression or death (hazard ratio = 0.16, p<0.0001).
• The trial also met all key secondary endpoints, including achieving an objective response rate of 56% vs. 9% with placebo (p<0.0001), as assessed by blinded independent central review.
• Varegacestat demonstrated statistically significant improvement in worst pain intensity at week 12, with a clinically significant difference observed as early as the first evaluation at week 4.
• In an exploratory analysis, varegacestat showed a median best change in tumor volume of -83% vs. +11% with placebo, as assessed by blinded independent central review.
• Varegacestat was generally well tolerated with a manageable safety profile, consistent with the gamma secretase inhibitor class. The most common adverse events for participants in the treatment arm were diarrhea (82%), fatigue (44%), rash (43%), nausea (35%) and cough (34%). Most (95%) events were grade 1 or 2.

MAA validation follows acceptance by the U.S. Food and Drug Administration (FDA) of the New Drug Application (NDA) for varegacestat for the treatment of adults with desmoid tumors. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 28, 2027.

About the RINGSIDE Trial

The global, randomized, double-blind, placebo-controlled Phase 3 RINGSIDE trial (ClinicalTrials.gov Identifier: NCT04871282) evaluated the efficacy and safety of varegacestat in patients with progressing desmoid tumors. A total of 156 patients were randomized to receive varegacestat 1.2 mg daily or placebo until disease progression or death, representing the largest randomized study in this population. The primary endpoint of the trial was progression-free survival as assessed by blinded independent central review. Statistically controlled key secondary endpoints were confirmed ORR using RECIST v1.1 and change in tumor volume at week 24, both determined by blinded independent central review, as well as change in pain intensity at week 12 as determined using a patient-reported outcome instrument. Additional secondary endpoints included duration of response, best reduction in tumor volume, patient-reported outcomes, and safety and tolerability. RINGSIDE includes an open-label extension phase, which is ongoing.

About Desmoid Tumors

Desmoid tumors (also known as aggressive fibromatosis or desmoid-type fibromatosis) are aggressive non-metastatic soft tissue tumors that are prone to recurrence. Approximately 1,000-1,650 people are diagnosed with desmoid tumors each year in the United States, and there are approximately 10,000-11,000 actively managed patients. Those affected face debilitating pain, deformity and, in some cases, life-threatening organ damage. The chronic pain and physical limitations associated with desmoid tumors lead to a high clinical burden and impaired quality of life. Although desmoid tumors are not considered cancerous, they often require systemic treatment to prevent permanent disability and alleviate disease burden.

About Varegacestat

Varegacestat is an investigational, oral, once-daily gamma secretase inhibitor. In December 2025, Immunome reported positive topline results for the Phase 3 RINGSIDE trial of varegacestat in adults with progressing desmoid tumors. Immunome’s NDA for varegacestat was accepted by the FDA in July 2026 (PDUFA target action date of April 28, 2027) and its Marketing Authorization Application was validated by the European Medicines Agency in October 2026.

About Immunome, Inc.

Immunome is a clinical-stage targeted oncology company committed to developing first-in-class and best-in-class targeted cancer therapies. We are advancing an innovative portfolio of therapeutics, supported by a leadership team with deep experience in the design, development, and commercialization of cutting-edge therapies, including antibody-drug conjugates (ADCs). Our pipeline includes varegacestat, an investigational, oral, once-daily gamma secretase inhibitor for which an NDA has been accepted by the U.S. FDA; IM-1021, a clinical-stage ROR1 ADC; IM-1617, a clinical-stage solid tumor ADC; IM-3050, a clinical-stage FAP-targeted radiotherapy; and IM-1340, an IND-cleared solid tumor ADC. We are also advancing a broad portfolio of early-stage ADCs pursuing undisclosed solid tumor targets. For more information, visit www.immunome.com.

Cautionary Statement Regarding Forward-Looking Statements

Statements in this press release that are not purely historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. We use words such as “could,” “if,” “committed to,” “look forward,” “potential to,” and similar expressions to identify these forward-looking statements. These forward-looking statements include statements regarding the potential of varegacestat, including its therapeutic benefits, its ability to provide a meaningful new oral treatment option and regulatory outcomes; the potential availability of varegacestat to patients; and other statements regarding management’s intentions, plans, beliefs, expectations or forecasts for the future.

These forward-looking statements are based on Immunome’s current expectations and involve assumptions that may never materialize or may prove to be incorrect; consequently, actual results may differ materially from those expressed or implied due to a number of factors, including risks related to the EMA’s review of the MAA for varegacestat, such as potential delays or failure to obtain regulatory approval; risks related to the safety profile of varegacestat; risks related to the labeling and commercialization of varegacestat, if approved; Immunome’s ability to grow and advance its pipeline and execute on its business plan; and other risks and uncertainties included under the caption “Risk Factors” in Immunome’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, filed with the Securities and Exchange Commission on August 11, 2026.

These documents can also be accessed on Immunome’s website at www.immunome.com by clicking on the link “Financials” under the “Investors” tab. The forward-looking statements included in this press release are made only as of the date of this press release. Except as required by law, Immunome assumes no obligation and does not intend to update any forward-looking statements included in this press release.

View source version on businesswire.com: https://www.businesswire.com/news/home/20261030925417/en/

Investor Contact:
Max Rosett
Chief Financial Officer, Immunome
investors@immunome.com

Source: Immunome, Inc.

Key Terms

marketing authorization application regulatory

A marketing authorization application is a formal request submitted to a government regulator asking permission to sell a prescription medicine or medical product in a country or region. Think of it like asking for a business license after showing evidence the product is safe and works; investors care because approval determines whether the product can generate sales, how soon revenue starts, and how much regulatory risk and uncertainty remains.

gamma secretase inhibitor medical

A gamma secretase inhibitor is a type of drug that blocks an enzyme (gamma secretase) which acts like a pair of molecular scissors cutting larger proteins into smaller, sometimes harmful, fragments. Investors watch these drugs because they aim to reduce production of sticky protein fragments implicated in diseases such as Alzheimer’s and certain cancers; their success or failure in clinical trials, safety profile, and regulatory decisions can strongly affect a biotech company's value.

progression-free survival medical

Progression-free survival is the length of time during and after a treatment that a patient's disease does not get worse, measured from the start of treatment until the disease shows measurable signs of progression or the patient dies. Investors care because longer progression-free survival in clinical trials often signals that a drug is effective, improving chances of regulatory approval, market adoption, and revenue potential—think of it as a stopwatch showing how long a therapy can keep the illness at bay.

objective response rate medical

The objective response rate (ORR) is the percentage of patients in a clinical trial whose tumors measurably shrink or disappear according to preset rules. Investors use it as a quick, objective signal of a drug’s ability to produce a clear treatment effect—like counting how many plants visibly respond after applying a new fertilizer—and higher ORR can improve odds of regulatory approval, commercial success, and company valuation.

hazard ratio medical

A hazard ratio is a way scientists compare the chance of something happening over time between two groups, like patients taking different medicines. If the ratio is high, it means one group is more likely to experience the event sooner or more often, which helps determine how effective a treatment is or how risky a situation might be.