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VX-147 · 3 trials · 3 indications
| Arm | Type | Description |
|---|---|---|
| Part A (Phase 2/3): VX-147 | EXPERIMENTAL | Participants will be randomized to receive different doses of VX-147. Participants will receive the selected dose of VX-147 for atleast 96 weeks. |
| Part A (Phase 2/3): Placebo | PLACEBO_COMPARATOR | Participants will receive placebo matched to VX-147. |
| Part B (Phase 3): VX-147 | EXPERIMENTAL | Participants who complete Part A will receive VX-147 for an additional 96 weeks. |
| VX-147 | EXPERIMENTAL | All participants received VX-147 at a dosage of 15 mg once daily (qd) for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included participants with urine protein to creatinine ratio (UPCR) approximately greater than or equal to (≥) 3 g/g (± 10%) and less than (\<) 10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included participants with UPCR approximately ≥0.8 g/g (± 10%) and \<2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2. |
| Sequence A: VX-147 | EXPERIMENTAL | Participants will receive VX-147 reference tablet under fasted condition in Treatment Period 1, then VX-147 test tablet under fed condition in Treatment Period 2, and VX-147 test tablet under fasted condition in Treatment Period 3. A washout period of 5 days will be maintained between 3 treatment periods. |
| Sequence B: VX-147 | EXPERIMENTAL | Participants will receive VX-147 test tablet under fed condition in Treatment Period 1, then VX-147 test tablet under fasted condition in Treatment Period 2, and VX-147 reference tablet under fasted condition in Treatment Period 3. A washout period of 5 days will be maintained between 3 treatment periods. |
| Sequence C: VX-147 | EXPERIMENTAL | Participants will receive VX-147 test tablet under fasted condition in Treatment Period 1, then VX-147 reference tablet under fasted condition in Treatment Period 2, and VX-147 test tablet under fed condition in Treatment Period 3. A washout period of 5 days will be maintained between 3 treatment periods. |
| Name | Type | Description |
|---|---|---|
| VX-147 | DRUG | Tablets for oral administration. |
| Placebo | DRUG | Tablets for oral administration. |
Key Inclusion Criteria: Part A: * APOL1 genotype of G1/G1, G2/G2, or G1/G2 * Proteinuric kidney disease Part B: \- Completion of Treatment Period in Part A and no permanent discontinuation of study drug. Key Exclusion Criteria: Part A: * Solid organ or bone marrow transplant * Uncontrolled hy...
VX-147 is an investigational small molecule being developed by Vertex Pharmaceuticals for APOL1-mediated proteinuric kidney diseases, including focal segmental glomerulosclerosis (FSGS). It is designed to target the APOL1 gene. VX-147 is currently in clinical development and is not yet approved by the FDA.
VX-147 targets APOL1, a gene implicated in certain forms of kidney disease. By targeting APOL1, VX-147 is being studied as a potential treatment for APOL1-mediated proteinuric kidney diseases, including focal segmental glomerulosclerosis (FSGS). The drug is an investigational small molecule developed by Vertex Pharmaceuticals.
VX-147 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker VRTX. The company is conducting clinical trials to evaluate VX-147 as a potential treatment for APOL1-mediated proteinuric kidney diseases, including focal segmental glomerulosclerosis (FSGS).
VX-147 is in Phase 1 clinical development, though it has also been studied in later-phase trials. A Phase 2a study (NCT04340362) has been completed, and a Phase 2/3 adaptive study (NCT05312879) is currently recruiting participants. VX-147 is investigational and has not been approved by the FDA.
VX-147 has been studied in several clinical trials. A Phase 2a study (NCT04340362) in adults with APOL1-mediated FSGS is completed. A Phase 2/3 adaptive study (NCT05312879) in adult and pediatric participants with APOL1-mediated proteinuric kidney disease is recruiting. A Phase 1 bioavailability study (NCT05955872) is also completed.
VX-147 is also known as inaxaplin, an investigational small molecule targeting APOL1 for the treatment of APOL1-mediated proteinuric kidney diseases, including focal segmental glomerulosclerosis (FSGS). Vertex Pharmaceuticals is developing inaxaplin under the code name VX-147.