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Also known as IXP, Inaxaplin
VX-147 · 4 trials · 3 indications
| Arm | Type | Description |
|---|---|---|
| IXP (Inaxaplin) | EXPERIMENTAL | Participants with proteinuric APOL1-mediated kidney disease (AMKD) with or without the comorbidity of Type 2 diabetes mellitus (T2DM) will receive a single dose of IXP once daily, for 13 weeks. |
| 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 |
|---|---|---|
| Inaxaplin | DRUG | Tablets for Oral Administration. |
| VX-147 | DRUG | Tablets for oral administration. |
| Placebo | DRUG | Tablets for oral administration. |
Key Inclusion Criteria: * Participant has an APOL1 genotype of G1/G1, G2/G2, or G1/G2 obtained with a Vertex designated investigational clinical study assay * Estimated Glomerular Filtration Rate (eGFR) of greater than or equal to (≥) 25 milliliter per minute (mL/min)/1.73m\^2 at screening Key Exc...
VX-147 is an investigational small molecule being developed for APOL1-mediated proteinuric kidney disease, including focal segmental glomerulosclerosis (FSGS). It is designed for patients whose kidney disease is driven by variants in the APOL1 gene. VX-147 is in Phase 2 clinical development and is not approved for any use.
VX-147 targets APOL1, a gene whose variants are linked to kidney disease. By acting on APOL1, the small molecule is being developed to address APOL1-mediated proteinuric kidney disease and focal segmental glomerulosclerosis. The target class is a gene, and the approach focuses on the genetic driver of these kidney conditions.
VX-147 is being developed by Vertex Pharmaceuticals Incorporated, which trades on Nasdaq under the ticker VRTX. Vertex is advancing the small molecule in nephrology, with a focus on APOL1-mediated proteinuric kidney disease and focal segmental glomerulosclerosis.
VX-147 is in Phase 2 clinical development. It is investigational and is being studied in APOL1-mediated proteinuric kidney disease and focal segmental glomerulosclerosis. The program includes a Phase 2/3 adaptive study that is active but no longer recruiting, along with earlier-phase studies that have been completed.
VX-147 has been studied in three registered trials. NCT05312879 is a Phase 2/3 adaptive study in adults and children with APOL1-mediated proteinuric kidney disease, with 522 participants, and is active but not recruiting. NCT04340362 was a completed Phase 2a study in adults with APOL1-mediated FSGS, and NCT05955872 was a completed Phase 1 tablet bioavailability study.