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Use of Ultra-high Field MRI in Small Rodent Models of Polycystic Kidney Disease for In Vivo Phenotyping and Drug Monitoring

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Inaxaplin for Proteinuric Kidney Disease in Persons with Two APOL1 Variants.

Ogo Egbuna¹, Brandon Zimmerman¹, George Manos¹

¹From Vertex Pharmaceuticals (O.E., B.Z., G.M., A.F., M.C.C., L.A.D., M.E.B., D.A.), and Beth Israel Deaconess Medical Center, Harvard Medical School (D.J.F., M.R.P.) - both in Boston; King's College London, London (K.B.); Icahn School of Medicine at Mount Sinai, New York (K.C.); Necker Hospital, Assistance Publique-Hôpitaux de Paris, Université Paris Cité, Paris (B.K.); Duke University, Durham (L.B.), and the University of North Carolina at Chapel Hill, Chapel Hill (R.J.F.) - both in North Carolina; the University of Michigan, Ann Arbor (D.S.G.); Georgetown University Hospital, Washington, DC (M.S.L.); University of Kansas School of Medicine, Kansas City (A.O.); and Stanford University School of Medicine, Palo Alto, CA (G.M.C.).

The New England Journal of Medicine

|March 15, 2023

Summary

This summary is machine-generated.

Inaxaplin effectively reduced proteinuria in individuals with two APOL1 variants and focal segmental glomerulosclerosis. This small-molecule compound shows promise for treating APOL1-mediated kidney disease.

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Area of Science:

Nephrology
Pharmacology
Genetics

Background:

Individuals with toxic gain-of-function variants in apolipoprotein L1 (APOL1) face increased risk of rapidly progressive, proteinuric nephropathy.
Currently, effective therapies for proteinuric kidney disease in individuals with two APOL1 variants (G1 or G2) are limited.

Purpose of the Study:

To evaluate the efficacy of inaxaplin, a small-molecule compound, in inhibiting APOL1 channel function and reducing proteinuria.
To assess the safety and efficacy of inaxaplin in a phase 2a clinical study for patients with APOL1-mediated kidney disease.

Main Methods:

In vitro studies using tetracycline-inducible APOL1 HEK293 cells to assess inaxaplin's inhibition of APOL1 channel function.
Preclinical assessment in an APOL1 G2-homologous transgenic mouse model to evaluate inaxaplin's effect on proteinuria.
A single-group, open-label, phase 2a clinical study administering inaxaplin to participants with two APOL1 variants, focal segmental glomerulosclerosis, and proteinuria.

Main Results:

In preclinical studies, inaxaplin inhibited APOL1 channel function and reduced proteinuria in a mouse model.
In the phase 2a study, 13 participants meeting adherence criteria showed a mean 47.6% reduction in proteinuria at week 13.
Adverse events were mild to moderate and did not lead to study discontinuation.

Conclusions:

Targeted inhibition of APOL1 channel function with inaxaplin demonstrated a reduction in proteinuria in patients with APOL1 variants and focal segmental glomerulosclerosis.
Inaxaplin represents a potential therapeutic strategy for APOL1-mediated kidney disease.