IDEAYA Biosciences reported that its Phase 2/3 OptimUM‑02 trial met its primary endpoint, showing a statistically significant improvement in median progression‑free survival (PFS) for the combination of darovasertib and crizotinib versus investigator‑choice therapy in first‑line HLA‑A*02:01‑negative metastatic uveal melanoma.
The randomized, open‑label study enrolled 313 patients and was designed to evaluate the safety and efficacy of the PKC inhibitor darovasertib in combination with the c‑MET inhibitor crizotinib. The patient population comprised individuals with metastatic uveal melanoma who were negative for the HLA‑A*02:01 allele, a group for which no systemic therapy has yet been approved.
At the data cut‑off of January 23 2026, median PFS was 6.9 months in the combination arm compared with 3.1 months for investigator‑choice therapy, representing a 58 % reduction in the risk of disease progression. The overall response rate was 37.1 % versus 5.8 % in the control arm, and five complete responses were observed in the combination group while none were seen in the control group.
Safety was consistent with the known profiles of both agents, with a manageable overall safety profile and the most common Grade 3 or higher adverse events being diarrhea, syncope, and hypotension. Overall survival data are not yet mature, but an early trend toward improvement has been noted. The results build on the single‑arm OptimUM‑01 trial, which reported a median PFS of 7.0 months and a median overall survival of 21.1 months in a similar patient cohort.
The company plans to submit a New Drug Application for accelerated approval in the second half of 2026, and full data from the trial will be presented at a major medical conference later in 2026. The announcement was well received by investors, reflecting confidence in the drug’s potential to address a high‑unmet need in a rare cancer.
CEO Yujiro Hata said, 'OptimUM‑02 is the first randomized study to demonstrate a statistically significant and clinically meaningful benefit in PFS in the clinical setting of first‑line HLA‑A02:01‑negative metastatic uveal melanoma.' Dr. Meredith McKean of the Sarah Cannon Research Institute added, 'Metastatic uveal melanoma is an area of high unmet medical need with poor prognosis and short overall survival, and there are currently no approved therapies for HLA‑A02:01‑negative mUM patients. The data from the OptimUM‑02 study provides potential practice‑changing results for the treatment of first‑line metastatic uveal melanoma.'
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