Vertex Pharmaceuticals Inc. announced on Monday the initiation of the phase 3 part of their global clinical trial for inaxaplin (VX-147), advancing it from phase 2. The drug is being tested for its effectiveness in treating APOL1-mediated kidney disease (AMKD) at a single daily oral dosage of 45mg. This dosage will be evaluated alongside the placebo, both being provided in addition to the standard care.
The primary goal of the clinical trial is to determine the effect of inaxaplin on kidney function and proteinuria in individuals who have proteinuric kidney disease that is mediated by two variants of the APOL1 gene, a condition known as AMKD.
The scope of the trial has widened to include adolescent patients aged between 10 and 17 years who are suffering from the same condition.
Inaxaplin showed promising results in previous phase 2a proof-of-concept data. It reported a significant and clinically meaningful average reduction of 47.6% over 13 weeks in the urine protein to creatinine ratio (UPCR). This reveals preliminary evidence that an oral small molecule APOL1 inhibitor, such as inaxaplin, could be instrumental in reducing proteinuria in patients with AMKD.
The Phase 3 phase of the study was guided by the recommendations from an Independent Data Monitoring Committee (IDMC), which suggested using a single 45mg daily dosage of inaxaplin. The committee also endorsed the inclusion of adolescent AMKD patients aged 10 to 17 in the study.
In addition, the U.S. Food and Drug Administration (FDA) has bestowed upon inaxaplin the labels of Rare Pediatric Disease Designation (RPD) and Breakthrough Therapy Designation (BTD) for APOL1-mediated focal segmental glomerulosclerosis (FSGS).
The European Medicines Agency (EMA) also granted inaxaplin Priority Medicines (PRIME) and Orphan Drug designations for the treatment of AMKD.
