IDEAYA Biosciences (IDYA) RBC Inaugural Virtual Ophthalmology Conference summary

Key program updates and milestones

• Lead candidate darovasertib is in a registrational study for metastatic uveal melanoma, with a first readout expected before year-end, potentially enabling accelerated approval based on median progression-free survival.

• Breakthrough Therapy Designation was recently granted for darovasertib in the neoadjuvant setting, making it only the second oncology drug to receive this in neoadjuvant use.

• Six clinical programs are active, including MTAP deletion (IDE397), topo ADC, PARG, DLL3, Werner Helicase, Pol Theta, PRMT5, and KAT6, with multiple data readouts and IND filings anticipated through 2025.

• Data from the DLL3 program is expected at a medical conference in 2025, and Werner Helicase will have an oral presentation at AACR, marking the first clinical data for this high-value target.

• The company’s R&D engine leverages computational and structural biology, focusing on first-in-class opportunities and rational drug combinations to address tumor heterogeneity.

Darovasertib in uveal melanoma: clinical impact and strategy

• Darovasertib targets the protein kinase C pathway, a key driver in uveal melanoma, and is being evaluated across the patient journey, from primary to metastatic disease.

• Combination with crizotinib addresses both PKC and MET signaling, showing profound activity in metastatic settings, including both HLA-A2 positive and negative patients.

• Phase 2 data show a 40% response rate and over seven months progression-free survival, significantly outperforming current standards of care.

• The registrational trial uses a seamless phase 2/3 design, with accelerated approval possible based on PFS and full approval contingent on OS data.

• The trial includes both HLA-A2 negative and positive patients, with strategies to support broad label expansion and guideline inclusion.

Neoadjuvant and vision-preserving advances

• Breakthrough Therapy Designation was granted for darovasertib in neoadjuvant uveal melanoma, based on data showing significant tumor shrinkage and eye preservation.

• About 60% of eyes were saved in enucleation patients, and nearly universal tumor shrinkage was observed in plaque brachytherapy patients, with evidence of radiation reduction and vision preservation.

• Upcoming data presentations will further detail these outcomes, with mid-year and year-end updates planned at ophthalmologic and scientific conferences.

• Real-world feedback highlights reversal of retinal detachment and vision improvement, even in patients with severe baseline impairment.