A rare yet early-stage win for Galapagos and Gilead in scleroderma

Of all the rheumatic disorders, diffuse cutaneous systemic sclerosis (dcSSc) has one of the worst prognoses, and unhappily for sufferers options are limited. Immunosuppressants are used but more effective agents are desperately needed; Corbus’s lenabasum crashed out in phase III only this week. Thus a hit in a phase IIa trial with Gilead and Galapagos’s autotaxin inhibitor ziritaxestat is welcome, if early-stage, progress. The primary endpoint, change on a score that measures skin thickness, mRSS, was hit with statistical significance, although only just (p=0.0411); with only 33 patients enrolled, this hint of efficacy still needs confirming in larger studies. Two serious adverse events in the ziritaxestat arm also mute enthusiasm, though the patients recovered and chose to participate in a long-term extension trial, along with the vast majority of other enrollees. A quick scan of other projects being trialled yields a short list, and the fact that much work is government-funded speaks to the challenge here. Seattle Genetics’ Adcetris, Glaxosmithkline’s Benlysta, and Roche’s Rituxan and Esbriet are some of the more novel agents that are being investigated by academics. Trials funded purely by industry are much rarer.

Searching for solutions for diffuse scleroderma active industry sponsored trials
Product  Mechanism  Company  NCT ID
Phase III       
Lenabasum (failed) CB2 agonist Corbus Pharmaceuticals NCT03398837
Phase II/I      
AVID200 TGF beta inhibitor Forbius NCT03831438
Tepezza  IGF-1R antibody Horizon Therapeutics NCT04478994
Vasculan  PG receptor agonist; TXA2 receptor antagonist Cumberland Pharmaceuticals NCT02682511
KD025 ROCK2 inhibitor Kadmon NCT03919799
Ziritaxestat (GLPG-1690) Autotaxin inhibitor Galapagos NCT03976648
Privigen + Hizentra Immunoglobulin CSL NCT04137224
Privigen Immunoglobulin CSL NCT04138485
EHP-101 CB2 agonist; PPAR-gamma agonist Emerald Health Sciences NCT04166552
MT-7117 (dersimelagon) Melanocortin type 1 receptor agonist Mitsubishi Chemical NCT04440592
Source: EvaluatePharma. 

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