CAMBRIDGE (MASSACHUSETTS) (dpa-AFX) - Vertex Pharmaceuticals Inc. (VRTX)
announced Monday that inaxaplin (VX-147) has advanced into the Phase 3 portion
of the global Phase 2/3 pivotal clinical trial in APOL1-mediated kidney disease
(AMKD), where a 45 mg once daily oral dose will be compared to placebo, on top
of standard of care.

The clinical trial is designed to assess the impact of inaxaplin on kidney
function and proteinuria for people living with proteinuric kidney disease
mediated by two variants in the APOL1 gene, known as AMKD.

In addition, the trial has been expanded to include adolescents with AMKD ages
10 to 17 years.

Previously reported Phase 2a proof-of-concept data demonstrated that inaxaplin
led to a statistically significant and clinically meaningful mean reduction in
the urine protein to creatinine ratio (UPCR) of 47.6% at 13 weeks of treatment
compared to baseline, providing the first clinical evidence that an oral small
molecule APOL1 inhibitor can decrease proteinuria in people with AMKD.

An Independent Data Monitoring Committee (IDMC) recommended the selection of a
single inaxaplin dose of 45 mg once daily in the Phase 3 portion of the Phase
2/3 study. The IDMC also recommended enrolling adolescents with AMKD ages 10 to
17 years in the Phase 3 portion of the study.

The U.S. Food and Drug Administration (FDA) has granted inaxaplin Rare Pediatric
Disease Designation (RPD) and Breakthrough Therapy Designation (BTD) for
APOL1-mediated focal segmental glomerulosclerosis (FSGS).

The European Medicines Agency (EMA) has also granted inaxaplin Priority
Medicines (PRIME) and Orphan Drug designations for AMKD.



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