Thursday, June 24, 2021
Home Latest Pharma-News Novartis announces Phase II data for iptacopan in rare kidney disease C3G

Novartis announces Phase II data for iptacopan in rare kidney disease C3G

Jun 07, 2021: “Novartis announced positive new interim Phase II data showing investigational iptacopan (LNP023) – a first-in-class, oral, targeted factor B inhibitor – improved estimated glomerular filtration rate (eGFR) slope and stabilized kidney function in patients with C3 glomerulopathy (C3G) treated with iptacopan.

The data were presented at the 58th ERA-EDTA Congress held virtually from June 5–8, 2021.

- Advertisement -

The new interim analysis data from the open-label Phase II study (NCT03832114) in patients with C3G showed twice-daily 200mg iptacopan stabilized kidney function, as measured by change in eGFR slope: a key measure of kidney clearance function that estimates the rate of blood passing through and being filtered by the kidneys.

The kidney function of 12 patients treated with iptacopan for 12 weeks was compared with their historical kidney function data for the two-year period before they entered the study (or since diagnosis was made where this was less than two years).

Furthermore, seven C3G patients who received iptacopan for up to 25 weeks in a long-term extension study (NCT03955445) showed ongoing eGFR stabilization, suggesting extended iptacopan treatment may prolong the time to, or even potentially prevent, the development of kidney failure.

Related News:

About the study
NCT03832114 is a Phase II, open-label, two cohort, non-randomized study evaluating the efficacy, safety and pharmacokinetics of iptacopan in patients with C3 glomerulopathy (C3G) (Cohort A) and patients who have undergone kidney transplantation and have subsequent C3G recurrence in the transplanted organ (Cohort B).

The aim of the analysis presented at the 2021 ERA-EDTA Congress was to determine whether iptacopan treatment altered eGFR slope in Cohort A.

On completion of the study, all patients had the option to receive ongoing iptacopan in a long-term extension study (NCT03955445)1.

It has the potential to become the first targeted therapy to delay progression to dialysis in C3G.

About iptacopan
Iptacopan is an investigational, first-in-class, orally administered factor B inhibitor of the alternative complement pathway, targeting one of the key drivers of these diseases.

Discovered at the Novartis Institutes for BioMedical Research, iptacopan is currently in development for a number of CDRDs where significant unmet needs exist, including C3G, IgA nephropathy (IgAN), atypical hemolytic uremic syndrome (aHUS), and membranous nephropathy (MN), as well as the blood disorder PNH.

While Novartis has a 35-year history in kidney transplantation treatments, iptacopan is the first treatment in the nephrology pipeline addressing CDRDs.

Our aim is to transform treatment by targeting one of the key drivers of these rare and often progressive diseases and, in doing so, potentially extend dialysis-free life for people with CDRDs.


Please enter your comment!
Please enter your name here

12 − six =

Most Popular

Merck Announces Phase 3 KEYNOTE-826 Trial Met Dual Primary Endpoints of OS and PFS

June 22, 2021: Merck known as MSD outside the United States and Canada announced that the pivotal Phase 3 KEYNOTE-826 trial investigating...

EU approve Koselugo for children with neurofibromatosis & plexiform neurofibromas

June 22, 2021: "AstraZenecaand MSD's Koselugo (selumetinib) has been granted conditional approval in the EU for the treatment of symptomatic, inoperable plexiform neurofibromas (PN)...

FDA Approves First Oral Blood Thinning Medication for Children

June 21, 2021: The U.S. FDA approved Pradaxa (dabigatran etexilate) oral pellets to treat children 3 months to less than 12 years...

Sanofi and Translate Bio initiate Phase 1 clinical trial of mRNA influenza vaccine

JUNE 22, 2021: "Sanofi Pasteur, the vaccines global business unit of Sanofi and Translate Bio, a clinical-stage messenger RNA (mRNA) therapeutics company, have...