Immunome is seeking US approval for its gamma secretase inhibitor (GSI) for the treatment of desmoid tumours after the drug demonstrated its efficacy and tolerability in a pivotal trial.
Top line results from the global Phase III RINGSIDE study (NCT04871282) reveal that varegacestat met its primary endpoint – offering significant improvements to progression-free survival (PFS). During the study, varegacestat slashed the risk of disease progression or death by 84% versus placebo.
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The oral therapy also triggered a confirmed objective response rate (ORR) of 56% in patients who received the study drug, with 9% of patients in the placebo arm experiencing the same outcome.
This trumps the ORR demonstrated by Ogsiveo (nirogacestat), which is the only US Food and Drug Administration (FDA) approved therapy for desmoid tumours. In Ogsiveo’s pivotal trial, the drug exhibited an ORR of 41% versus 8% in the placebo group.
Alongside its capacity to stimulate desmoid tumour responses, treatment with varegacestat, dosed at 1.2mg once daily, also prompted an 83% decline in tumour volume. This effect was not mirrored in the placebo group, which experienced an 11% tumour volume growth.
Varegacestat also offered statistically significant improvements in worst pain intensity experienced by patients, suggesting the ameliorative effect the drug may have on quality of life (QoL).
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By GlobalDataThe candidate was generally well tolerated during the study, with a majority of patients experiencing mild-to-moderate treatment-emergent adverse events (TEAEs). The most common occurrences were diarrhoea, fatigue and rash, which were experienced by 82%, 44% and 43% of patients on the varegacestat treatment arm, respectively.
Immunome acquired the rights to varegacestat in 2024 through a $50m deal with Ayala Pharmaceuticals, which saw Immunome claim the GSI alongside another mid-stage asset for adenoid cystic carcinoma.
Following the positive results of this trial, Immunome plans to submit a new drug application (NDA) for varegacestat to the FDA in Q2 2026. If varegacestat were to secure FDA approval, it would be the second drug approved in this indication.
Varegacestat’s potential market entry appears to have garnered investor interest. Immunome’s stock value rose by more than 15% from $19.57 at market close on 12 December to $22.64 at the same time on 15 December following the debut of the RINGSIDE topline results.
Vargacestat’s future on the desmoid tumour market
Following the positive results of the RINGSIDE trial, Immunome’s CEO Clay Siegall touted varegacestat’s potential as a “best-in-class” therapy for desmoid tumours.
If the drug were to gain FDA approval, it would have a slight dosing edge over Ogsiveo, which requires twice-daily administration.
According to Mrinal Gounder, RINGSIDE’s primary investigator and sarcoma medical oncologist at the Memorial Sloan Kettering Cancer Center, the findings of this study both “elevate the role of GSIs” and confirm varegacestat’s potential as the new standard of care (SoC) treatment for desmoid tumours.
Currently, analysts at GlobalData forecast that varegacestat will launch in 2027, bringing in $55m during this year. In 2031, the drug is forecast to generate $650m in sales for Immunome.
Meanwhile, Ogsiveo is forecast to become a blockbuster seller in 2031, with the drug expected to produce $1.06bn in sales for Merck KGaA during the year.
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Immunome could also see varegacestat reach the market in two haematological malignancies, as the company is currently assessing the drug’s potential in relapsed/refractory multiple myeloma (r/r MM) and r/r T-cell acute lymphoblastic leukaemia (T-ALL).
