IDEAYA Biosciences and Servier announced on Apr. 13 that their investigational drug for metastatic uveal melanoma, darovasertib, achieved significant results in a late-stage clinical trial and is expected to be submitted to the U.S. Food and Drug Administration later this year.
The announcement is important because there are currently no FDA-approved systemic therapies for patients with HLA-A*02:01-negative metastatic uveal melanoma or primary positive uveal melanoma, leaving a gap in treatment options for these patients.
In the Phase 2/3 OptimUM-02 trial, darovasertib combined with crizotinib more than doubled median progression-free survival compared to standard treatments. Patients receiving the combination therapy had a median progression-free survival of 6.9 months versus 3.1 months for those who received other therapies chosen by investigators. The overall response rate was also higher at 37.1% compared to 5.8% in the control group, with five patients experiencing a complete response.
The study enrolled 210 patients with HLA-A*02:01-negative metastatic uveal melanoma treated with the new combination therapy and compared them to an investigator’s choice arm of 103 patients using existing immunotherapies such as Yervoy plus Opdivo or Keytruda. IDEAYA said that darovasertib was well-tolerated and showed a manageable safety profile consistent with previous reports.
Analysts responded positively but cautiously to the news. Truist Securities described darovasertib as having demonstrated "a best-in-class efficacy profile" while projecting around $800 million peak revenue in this indication if approved. Leerink Partners noted that results were "largely in line with most investor expectations," adding that future commercial success will depend on pricing and market share among different patient groups.
Looking ahead, IDEAYA plans to submit a new drug application for darovasertib later this year while also exploring its use in additional patient populations where it may compete against existing drugs like Immunocore’s Kimmtrak.
