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INAXAPLIN

 People with homozygosity or compound heterozygosity  for G1 and G2 risk alleles are at high risk of developing multiple kidney diseases. The leading hypothesis is that podocyte-intrinsic G1 or G2 APOL1 creates pores in the cell membrane leading to focal segmental glomerulosclerosis (FSGS), focal global glomerulosclerosis (FGGS), HIV-associated nephropathy (HIVAN), and lupus nephritis. 


Inaxaplin is a four-ringed small molecule that inhibits APOL1 protein-mediated induction of pore formation in podocytes, thereby stopping aberrant channel-mediated ion flux.

Wavy Abstract Background

Enroll in this clinical study

Medical Information

617-341-6777

medicalinfo@vrtx.com

How is Inaxaplin being studied in APOL1 kidney disease?

In a recently published proof-of-concept single-group, open-label, phase 2a clinical study, targeted inhibition of APOL1 channel function with Inaxaplin showed reduced proteinuria in 12 out of 13 participants with two APOL1 variants and focal segmental glomerulosclerosis and was safe and well tolerated without side effects leading to treatment discontinuation. In an ongoing Phase 2/3 adaptive, double-blind, placebo-controlled study to evaluate the efficacy and safety of Inaxaplin, 66 participants will be randomized to receive different doses of Inaxaplin vs matched placebo control. In phase 3 study 400 participants will receive Inaxaplin with the dose to be based on the outcome of Phase 2.


The primary outcome of measure is percent change from baseline in UPCR at week 48, eGFR slope assessed at the Week 48 and at study completion.

Eligibility Criteria

  1. Age 12 Years to 65 Years

  2. APOL1 genotype of G1/G1, G2/G2, or G1/G2

  3. Proteinuric kidney disease

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