New drug for systemic sclerosis! Gilead/Galapagos Selective ATX Inhibitor Ziritaxestat Concept Verification Phase 2 study was successful!

September 14, 2020 // -- Belgian biotech company Galapagos recently announced positive top-line results for the treatment of diffuse skin systemic sclerosis (dcSSc) patients in phase 2 NOVESA clinical trials.
NOVESA was a double-blind, placebo-controlled Phase 2a proof-of-concept trial that assessed the efficacy, safety and tolerance of ziritaxestat in 33 patients with dcSSc.
patients recruited for the trial were women in their 50s (70%) with an average course of 1.9 years.
patients received background immunosuppressants during the study.
94% of patients (31 out of 33) who completed the NOVESA trial continued to have long-term open label expansion trials.
data show that ziritaxestat reached the main endpoint of the study: in week 24 of treatment, the improved Rodnan skin score (mRSS) in the ziritaxestat treatment group was statistically significant compared to the baseline (-8.3 vs -5.7).
test, ziritaxestat was generally well-to-do.
no deaths were reported in the trial.
2 patients who took ziritaxestat had severe adverse events, while 1 patient in the placebo group had severe adverse events.
patients in the Ziritaxestat treatment group have made a full recovery and are still participating in long-term expansion trials.
results of the NOVESA trial will be presented at a future medical conference. Dr Walid Abi-Saab, chief medical officer of
Galapagos, said: "Following promising activity in the IDF Phase 2 FLORA trial, we are pleased to see that ziritaxestat has achieved a statistically significant improvement in msSS for diffuse SSc (the main endpoint of the NOVESA study).
note that this is our first study in SSc and that the effects on the skin in background therapy with immunosuppressants are difficult to measure, and we are pleased with the results reported today.
we will now further analyze NOVESA's data to determine the next steps for SSc, a seriously unsealed medical need.
"DcSSc is a serious autoimmune disease with one of the highest mortality rates from rheumic disease, and no drugs have been approved to treat it."
in the United States and Europe, systemic sclerosis (SSc) affects about 124,000 people, of whom women make up the majority.
ziritaxestat chemical structure (Photo: medchemexpress.cn) ziritaxestat is a small molecule, selective autotaxin, ATX inhibitor developed in collaboration with Gilead Sciences as part of a global collaboration between Galagos and Gilead.
ATX is the main enzyme that produces hemolytic phospholipid acid (LPA).
LPA is a well-known fibrosis- and inflammatory lipid that works through at least six G-protein coupled lipolys.
Galapagos used its proprietary target discovery platform to identify ATX targets and developed ziritaxestat molecules as inhibitors for that target.
, which has been granted U.S. and European Union Orphan Drug Qualification (ODD) for idiotrex fibrosis (IPF) and SSc, is currently being studied in the IPF's Global Phase 3 Project (ISABELA) and the treatment of SSc is undergoing ANVESA expansion trials.
() original source: Primary endpoint achieved with ziritaxestat in NOVESA trial in systemic sclerosis patients.