Novartis’ investigational oral factor B inhibitor has hit its early target in a phase 3 kidney disorder trial, a success that has the pharma readying to submit a request for fast-track FDA approval next year.
The 9-month data comes from the Swiss pharma’s APPLAUSE-IgAN study, with topline results expected in 2025. The double-blind, placebo-controlled trial is designed to measure the efficacy and safety of twice-daily oral iptacopan among 470 adults with IgA nephropathy (IgAN), a chronic kidney disease also known as Berger’s disease.
The therapy hit the study’s intermediary primary end point, demonstrating statistically significant superiority compared to placebo in proteinuria reduction, according to Novartis, though no figures were provided. The study continues to assess the pill’s ability to slow IgAN progression by measuring estimated glomerular filtration rate slope over two years.
The data is a homerun for Novartis, according to financial analyst group ODDO BHF, who dubbed iptacopan’s performance “faultless” in a Oct. 2 report.
Beyond the potential for becoming the first oral monotherapy for IgAN patients, iptacopan could also become the first targeted IgAN treatment that slows or prevents progression to kidney failure by activating the complement system, according to Novartis.
APPLAUSE-IgAN demonstrated a safety profile consistent with previously reported data from two phase 3 studies for paroxysmal nocturnal hemoglobinuria (PNH). Based on those trials, Novartis has submitted iptacopan to regulators in the rare disease indication and expects a response in 2024.
If approved in PNH, Novartis will give AstraZeneca’s rare disease unit Alexion a run for its money, going up against complement inhibitors Soliris and Ultomiris. Previously, iptacopan out-performed Soliris in a head-to-head PNH trial.
Iptacopan has “pipeline in a pill” potential, according to ODDO BHF analysts. The pill is also being developed to treat several other renal and hematological diseases that currently lack available treatment or unsatisfactory treatments. ODDO BHF anticipates an initial iptacopan launch in 2024 and peak sales of more than $3 billion when considering all indications.