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Also known as Varegacestat Reference Formulation, AL102
Varegacestat · 6 trials · 5 indications
Evaluation of the safety and tolerability of AL102 in subjects with progressing desmoid tumors as defined by as defined by the frequency and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
PFS as defined as the time from randomization until the date of assessment of progression as assessed by BICR based on Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 or death by any cause
AUC0-t varegacestat and metabolite
AUC0-t for varegacestat administered with and without itraconazole.
| Arm | Type | Description |
|---|---|---|
| Part A Main Study 1.2 mg daily | EXPERIMENTAL | AL102 1.2 mg |
| Part A Main Study 2 mg Intermittent | EXPERIMENTAL | AL102 2 mg |
| Part A Main Study 4 mg Intermittent | EXPERIMENTAL | AL102 4 mg |
| Part B AL102 | EXPERIMENTAL | AL102, recommended dose regimen from Part A, 1.2 mg daily |
| Part B Placebo | PLACEBO_COMPARATOR | Placebo to match recommended dose regimen from Part A |
| Open Label Extension | EXPERIMENTAL | AL102, recommended dose regimen from Part A, 1.2 mg daily |
| Reference Formulation, then Test Formulation | EXPERIMENTAL | - |
| Test Formulation, then Reference Formulation | EXPERIMENTAL | - |
| Treatment Arm | EXPERIMENTAL | Single oral dose of varegacestat administered on study day 1 |
| Drug: varegacestat | EXPERIMENTAL | Single oral dose of varegacestat administered on study Day 1 |
| Varegacestat | EXPERIMENTAL | - |
| Placebo | PLACEBO_COMPARATOR | - |
| Moxifloxacin | ACTIVE_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| AL102 | DRUG | AL102 is an inhibitor of gamma secretase-mediated Notch signaling. |
| Placebo | OTHER | Placebo to match AL102 |
| Varegacestat Reference Formulation | DRUG | Oral administration |
| Varegacestat Test Formulation | DRUG | Oral administration |
| varegacestat | DRUG | experimental intervention |
| Moxifloxacin 400 mg | DRUG | Single oral dose of moxifloxacin |
| Itraconazole (200 mg) | DRUG | Intervention to evaluate DDI with experimental intervention |
Inclusion Criteria Part A: 1. At least 18 years of age (inclusive) at the time of signing the informed consent form (ICF). 2. Histologically confirmed desmoid tumor (aggressive fibromatosis) by local pathologist (prior to informed consent). 3. Disease progression, assessed locally by the investigat...
Varegacestat is an investigational small molecule developed by Immunome, Inc. (ticker IMNM). It is being studied in the gastrointestinal therapeutic area and is currently in Phase 1 clinical development. Its specific disease indications have not been disclosed. Varegacestat is also referred to as Varegacestat Reference Formulation.
Varegacestat targets gamma-secretase, an enzyme belonging to the -stat target class. Gamma-secretase is a multi-subunit protease complex involved in cleaving transmembrane proteins. By targeting this enzyme, Varegacestat acts as a small molecule inhibitor of gamma-secretase activity.
Varegacestat is being developed by Immunome, Inc., which trades on the Nasdaq under the ticker symbol IMNM. Immunome is the sponsor of the clinical program evaluating Varegacestat in Phase 1 studies.
Varegacestat is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. Five Phase 1 trials have been conducted or are ongoing, with one currently active and four completed, enrolling a total of 115 participants.
Varegacestat has been evaluated in five Phase 1 trials. These include NCT07610005, a recruiting relative bioavailability and bioequivalence study; NCT06855264, a completed cardiac liability study; NCT06845852, a completed mass balance study; and NCT06841315, a completed hepatic impairment study. All were conducted in the United States.
Yes. Varegacestat Reference Formulation is an alternative name for Varegacestat. Both names refer to the same investigational small molecule developed by Immunome, Inc. for the gastrointestinal therapeutic area.