USA — Vertex Pharmaceuticals, a leading American biopharmaceutical company headquartered in Boston, Massachusetts, has announced a significant stride in its efforts to combat kidney diseases.

The company revealed its plans to progress its promising experimental drug, inaxaplin, into Phase 3 testing and to extend enrollment to include adolescents aged between 10 and 17.

The forthcoming clinical trial aims to evaluate the efficacy of inaxaplin in managing kidney function and proteinuria among individuals afflicted with proteinuric kidney disease due to two variants in the APOL1 gene, commonly referred to as APOL1-mediated kidney disease (AMKD).

Phase 3 trials will scrutinize Vertex’s medication inaxaplin, also known as VX-147, as a daily oral treatment for kidney ailments stemming from mutations in the APOL1 gene, operating by inhibiting proteins encoded this  gene.

These gene variants, prevalent among individuals of African descent, are associated with a heightened risk of proteinuric kidney disease and hastened progression in affected individuals.

Vertex anticipates conducting an interim analysis after 48 weeks, with potentially positive outcomes prompting an accelerated approval request to the Food and Drug Administration (FDA).

The final study analysis is scheduled following a minimum of two years’ worth of data collection from participants.

This development marks Vertex’s strategic expansion beyond its acclaimed cystic fibrosis treatments, as the company previously ventured into addressing APOL1-mediated kidney disease several years ago.

A preliminary study in 2021 demonstrated that treatment with inaxaplin could reduce urine protein levels in individuals with focal segmental glomerulosclerosis (FSGS), a manifestation of kidney disease characterized by kidney scarring impeding blood filtration, thereby elevating protein levels in urine.

Encouraged by these findings, Vertex advanced inaxaplin into a Phase 2/3 trial.

Results from the initial Phase 2 segment were published last year in The New England Journal of Medicine, corroborating a decrease in proteinuria.

The Phase 3 segment of the trial will enroll a diverse range of individuals with proteinuric APOL1-mediated kidney disease (AMKD).

Dr. Carmen Bozic, Executive Vice President of Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex, emphasized the promising outcomes observed in the Phase 2a proof-of-concept study, underscoring inaxaplin’s potential to address the underlying cause of APOL1-mediated kidney disease.

Moreover, Dr. Glenn M. Chertow, Professor of Medicine at Stanford University School of Medicine and Chair of Vertex’s APOL1 Program Steering Committee, emphasized the urgent need for effective therapies for AMKD, stressing the absence of approved disease-specific treatments.

Dr. Chertow expressed optimism regarding inaxaplin’s potential to revolutionize AMKD care and improve patient outcomes.

Presently, there are no disease-specific therapies for AMKD, with treatment primarily consisting of standard care for chronic kidney disease.

Vertex disclosed that over 200 trial sites are operational in the United States and Europe.

Inaxaplin has garnered Rare Pediatric Disease Designation (RPD) and Breakthrough Therapy Designation (BTD) from the FDA for APOL1-mediated focal segmental glomerulosclerosis (FSGS), while the European Medicines Agency (EMA) bestowed Priority Medicines (PRIME) and Orphan Drug designations for AMKD.

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