Kura Oncology (KURA) Study Update summary

Key study results and regulatory progress

• KOMET-001 phase II trial in relapsed/refractory NPM1-mutant AML achieved its primary endpoint of CR/CRh, with a favorable safety profile and statistically significant results, consistent with prior reports.

• Ziftomenib is the first investigational menin inhibitor granted Breakthrough Therapy Designation for R/R NPM1-mutant AML, facilitating regulatory alignment.

• NDA submission for ziftomenib in relapsed/refractory NPM1-mutant AML is planned for Q2 2025, with top-line and full results to be presented at a major medical meeting in the same quarter.

• End-of-phase I FDA meetings resulted in alignment on design and endpoints for two phase III frontline trials under the KOMET-017 protocol, supporting both accelerated and full approval pathways.

• FDA feedback endorsed the use of MRD-negative CR and CR as primary endpoints for accelerated approval in the two KOMET-017 trials.

Study design and endpoints

• KOMET-017 will include two independent, randomized, placebo-controlled phase III trials for intensive and non-intensive chemotherapy settings, starting in 2H 2025.

• The KOMET-017 NIC trial will evaluate ziftomenib with venetoclax and azacitidine in newly diagnosed NPM1-mutant AML patients unfit for intensive chemotherapy, using CR and overall survival as dual primary endpoints.

• The KOMET-017 IC trial will assess ziftomenib with intensive chemotherapy in newly diagnosed NPM1-mutant and KMT2A-rearranged AML, using MRD-negative CR and event-free survival as dual primary endpoints.

• FDA agreed to allow modified venetoclax dosing in the NIC trial, reflecting real-world practice and aiming to reduce myelosuppression.

• MRD negativity is used as a novel surrogate endpoint for accelerated approval in the intensive setting, leveraging the unique NPM1 mutation as a marker.

Market opportunity and commercial plans

• Relapsed/refractory NPM1-mutant AML represents a significant unmet need, with fewer than 10% of patients surviving five years.

• The U.S. market for relapsed/refractory NPM1-mutant AML is estimated at $350–$400 million annually, with peak sales for ziftomenib projected up to $3 billion in broader frontline settings and >$7 billion/year in AML overall.

• Preparations for commercialization are underway in partnership with Kyowa Kirin, with Kura retaining U.S. rights and global development leadership.

• Ziftomenib's safety and efficacy profile is expected to support sustained therapy and efficient market entry.

• $785.3 million in pro forma cash as of September 30, 2024, including $330 million upfront from Kyowa Kirin, supports development and commercialization plans.