IDEAYA Biosciences Eyes Busy 2026 as Darovasertib Advances Toward FDA Filing

Key Points

• Darovasertib, IDEAYA’s lead drug, posted strong Phase I/II results in first-line HLA-A2 negative metastatic uveal melanoma, including median progression-free survival of 6.9 months versus 3.1 months for control. The company has received FDA RTOR support and is preparing for an NDA submission focused on accelerated approval.
• IDEAYA plans a major 2026 regulatory and clinical calendar, with additional ASCO data, an interim overall survival analysis expected next year, and further readouts across its uveal melanoma program. The company is also advancing HLA-A2 positive data that could support broader label or guideline inclusion.
• Beyond darovasertib, IDEAYA is building out a broader oncology pipeline, including DLL3 ADC, MTAP, and KAT6/7 programs, with multiple data updates and registrational studies expected. The company is also preparing commercially, including hiring sales leadership and supply-chain readiness ahead of a possible launch.

IDEAYA Biosciences NASDAQ: IDYA Chief Executive Yujiro Hata said the precision oncology company is preparing for a busy 2026, led by regulatory and clinical milestones for darovasertib in metastatic uveal melanoma and multiple data readouts across its pipeline.

Speaking at a Stifel-hosted event, Hata said darovasertib, IDEAYA’s lead program, recently produced positive top-line results in the Phase I/II OptimUM-02 study in first-line HLA-A2 negative metastatic uveal melanoma. He said the company also received Real-Time Oncology Review, or RTOR, from the U.S. Food and Drug Administration to support an expedited New Drug Application submission process.

Hata said IDEAYA is preparing to submit its pre-submission module 1 and will present additional data in an ASCO late-breaker oral presentation on Monday, June 1. That update is expected to include more detailed efficacy and safety information, including waterfall plots and safety tables.

Darovasertib Data Show Progression-Free Survival Benefit

Hata characterized HLA-A2 negative metastatic uveal melanoma as an area of “extraordinary unmet need,” noting that there is currently no FDA-approved therapy for this population. He said patients are commonly treated with ipilimumab plus nivolumab, PD-1 therapy alone or sometimes chemotherapy.

In the randomized study, Hata said darovasertib showed median progression-free survival of 6.9 months in the treatment arm compared with 3.1 months in the control arm. He said that corresponded to a hazard ratio of 0.42 and a p-value of less than 0.0001.

Hata also said the control arm had a single-digit response rate, while the darovasertib arm showed a response rate approaching 40%. He noted that response rate under central review was higher than investigator scoring, which he described as uncommon.

At ASCO, Hata said IDEAYA plans to provide additional detail on response rates by central review and investigator assessment, as well as progression-free survival by investigator scoring. He said the company also plans to show treatment and control arm waterfall plots and subset analyses. Safety tables are also expected, with Hata saying the safety profile has been consistent with prior reports and with crizotinib’s historical profile.

Regulatory Strategy Focused on First-Line HLA-A2 Negative Population

Hata said IDEAYA’s initial regulatory focus is on first-line HLA-A2 negative metastatic uveal melanoma. The company is seeking accelerated approval based on progression-free survival, with overall survival intended to support full approval later.

He said the company has observed a preliminary trend in overall survival based on a January data cut, though follow-up was just over 10 months. Hata said the interim overall survival analysis is currently projected for the middle of next year, and IDEAYA does not expect to refresh overall survival data at ASCO.

Beyond the HLA-A2 negative population, Hata said IDEAYA is generating a large single-arm dataset in HLA-A2 positive patients. The company expects to present data on about 100 patients in the second half of the year at a major medical conference, including response rate, progression-free survival and survival data in the frontline and all-comer population. Hata said that data will be submitted as part of the NDA, though he described label inclusion for that population as upside rather than the base case.

For HLA-A2 positive patients, Hata said IDEAYA’s base case strategy is to pursue compendia and NCCN guideline inclusion, potentially on an HLA-agnostic basis.

Company Sees Broader Opportunity in Uveal Melanoma

Hata said IDEAYA estimates the global annual incidence of metastatic uveal melanoma at roughly 4,000 to 5,000 patients, largely driven by the U.S. and Europe. In the pre-metastatic setting, he said the pool is about 10,000 to 12,000 patients. Based on clinical screening, he said IDEAYA estimates about two-thirds of patients are HLA-A2 negative and one-third are HLA-A2 positive.

IDEAYA is also studying darovasertib in neoadjuvant and adjuvant settings. Hata said there are no approved therapies in either setting for uveal melanoma and noted that darovasertib has received breakthrough therapy designation in the neoadjuvant setting.

For the neoadjuvant Phase III program, Hata said the study includes enucleation and plaque brachytherapy cohorts. The enucleation cohort’s primary endpoint is eye preservation, and he said IDEAYA is currently “well above” the lower bound of 10% it aims to exceed. The plaque therapy cohort uses a 15-letter best-corrected visual acuity test, with the company seeking a 20% improvement in the treatment arm compared with control.

Hata also said IDEAYA is preparing to launch an adjuvant Phase III study with Servier following a positive Type B meeting with the FDA. The randomized trial will compare the darovasertib-crizotinib combination against observation, with relapse-free survival as the superiority endpoint. Hata said the study is expected to include about 450 patients, with no HLA restriction and one year of treatment.

Commercial Preparation Underway

As IDEAYA prepares for a potential transition to a commercial-stage company, Hata said the organization is hiring and recently brought on a vice president and head of sales. He said the company is building its sales organization and working on supply chain and commercial supply readiness.

Hata said IDEAYA’s partnership with Servier allows the company to focus execution on a U.S. launch. He added that launching in a first-line setting with no approved competing agent would be an unusual opportunity for a company.

Pipeline Updates Include DLL3 ADC, MTAP and KAT6/7 Programs

Hata highlighted IDE849, IDEAYA’s Phase I/II DLL3 Topo ADC, as the next pipeline asset to watch. He said IDEAYA expects two clinical data updates by year-end, including data from partner Hengrui Pharma. The Hengrui dataset is expected to include more than 100 patients, mostly in small cell lung cancer, as well as the first data in neuroendocrine carcinoma. Hata said Hengrui is planning to start a Phase III registrational study this year, and IDEAYA also plans to start a registrational study.

In MTAP-deleted cancers, Hata said IDEAYA believes it has a first-in-class and best-in-class MTA-cooperative PRMT5 inhibitor, with dose escalation progressing well and combination studies expected to start shortly. He also pointed to IDE397, a MAT2A inhibitor in Phase II, and said the company is focused on combinations, including PRMT5 and MAT2A in non-small cell lung cancer and potential KRAS-related combinations in pancreatic cancer.

Hata also discussed IDE574, IDEAYA’s Phase I KAT6/7 inhibitor, saying the company views dual KAT6 and KAT7 inhibition as biologically important because of bypass mechanisms observed with KAT6 inhibition alone. He said dose escalation is going well and highlighted potential applications in breast cancer, colorectal cancer, prostate cancer and other indications.

About IDEAYA Biosciences NASDAQ: IDYA

IDEAYA Biosciences is a clinical-stage precision oncology company dedicated to the discovery and development of novel therapies that exploit synthetic lethality in cancer cells. By targeting key DNA damage response pathways, the company aims to selectively kill tumor cells exhibiting specific genetic vulnerabilities while sparing healthy tissue. IDEAYA's pipeline includes small-molecule inhibitors designed to address underserved tumor types, and its lead programs are advancing through Phase 1 and Phase 2 clinical trials in multiple oncology indications.

Central to IDEAYA's approach is its Modular Approach to Precision (MAP) platform, which integrates proprietary genomic and functional screening technologies to identify critical cancer-specific dependencies.

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