ZETOMIPZOMIB DEMONSTRATES FAVORABLE LONG-TERM SAFETY AND TOLERABILITY PROFILE WITHOUT SIGNS OF IMMUNOSUPPRESSION: RESULTS FROM THE PRESIDIO STUDY AND ITS OPEN-LABEL EXTENSION STUDY IN PATIENTS WITH DERMATOMYOSITIS AND POLYMYOSITIS
Authors: Rohit Aggarwal et al.
Zetomipzomib is a selective inhibitor of the immunoproteasome which reverted the decreased strength and to improved histopathology a myositis mouse model. This RCT evaluated zetomipzomib 45 mg sc weekly for 16 weeks in 25 patients with active PM (n=12) and DM (n=13). Overall, there were clinically meaningful improvements but zetomipzomib did not significantly differentiate from placebo. Zetomipzomib treated patients also showed improvements in secondary efficacy endpoints. In the open label extension, patients generally continued to improve with reduced symptoms and improvement in strength. Treatment-emergent adverse events were generally mild-to-moderate. There were no opportunistic infections.
Zetomipzomib is also under investigation for lupus nephritis and demonstrated a favorable safety and tolerability profile without signs of direct immunosuppression upon prolonged exposure. Given the need for better treatments in PM and DM, larger trials with this mode of action would be desirable.