IDEAYA Biosciences (IDYA) Jefferies 2024 Global Healthcare Conference summary

Lead program and clinical progress

• Darovosertib in neoadjuvant uveal melanoma shows high rates of tumor shrinkage and eye preservation, with over half of patients experiencing at least 50% tumor reduction and three-quarters avoiding enucleation.

• Phase II trial is expanding to include both large and small/mid-sized tumors, with neoadjuvant therapy duration extended up to 12 months and adjuvant therapy for another year, potentially treating patients for up to two years.

• Data package from phase II will inform FDA discussions in the second half of the year, focusing on endpoints like tumor response, eye preservation, and vision outcomes.

• Plans include using novel response criteria for localized uveal melanoma, with imaging modalities tailored to ocular tumors rather than standard RECIST.

• Regulatory strategy aims for accelerated approval via single-arm phase II, with a randomized phase III for full approval, targeting high-risk blindness patients.

Market opportunity and commercial outlook

• Pre-metastatic uveal melanoma market is estimated to be at least 10 times larger than the metastatic setting, with annual incidence of 8,000-10,000 patients and a total prevalence of about 100,000.

• Duration of therapy in pre-metastatic patients could reach two years, further expanding commercial potential.

• The "watch and wait" patient population represents a significant untapped segment for neoadjuvant therapy.

Pipeline and strategic partnerships

• MAT2A inhibitor program is advancing in both monotherapy and combination studies, with focus on bladder and non-small cell lung cancer, showing promising responses.

• Key collaborations with Amgen (PRMT5 combo) and Gilead (Trodelvy combo) are ongoing, with the PRMT5 combination expected to deliver deep and durable responses in MTAP-deficient tumors.

• The MAT2A/PRMT5 combination is highlighted as a strategic priority, with more updates expected.