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ISIS 301012 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Cohort A | EXPERIMENTAL | Loading doses followed by weekly maintenance doses |
| Cohort B | EXPERIMENTAL | Loading doses followed by weekly maintenance doses |
| Cohort C | EXPERIMENTAL | Loading doses followed by weekly maintenance doses |
| Cohort D | EXPERIMENTAL | Loading doses followed by extended weekly maintenance doses |
| Name | Type | Description |
|---|---|---|
| ISIS 301012 | DRUG | 100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36 |
Inclusion Criteria: * Weight ≥ 40 kg for Cohorts A, B, \& C; Weight ≥ 50 kg for Cohort D * Diagnosis of Homozygous Familial Hypercholesterolemia. * Female must be non-pregnant and non-lactating. * On stable lipid lowering therapy for at least 4 weeks. * Lipid values that meet the pre-specified crit...
ISIS 301012 is an investigational small molecule being developed for the treatment of familial hypercholesterolemia, a genetic condition characterized by high cholesterol levels. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
ISIS 301012 is a small molecule, but its specific molecular target has not been disclosed in available information. The drug is being studied for its potential to lower cholesterol in patients with familial hypercholesterolemia, though the exact mechanism of action is not specified.
ISIS 301012 is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with familial hypercholesterolemia.
ISIS 301012 is in Phase 2 clinical development. It is an investigational drug and has not received FDA approval. A Phase 2 clinical trial has been completed, but the drug remains under investigation and is not yet available for commercial use.
ISIS 301012 has one completed clinical trial, registered as NCT00280995. This was a dose-escalating safety study conducted in the United States and Netherlands, enrolling 12 participants with homozygous familial hypercholesterolemia who were already on lipid-lowering therapy. The trial was controlled but not double-blind.