Kura Oncology (KURA) Status update summary

Clinical trial results and efficacy

• Ziftomenib combined with 7+3 chemotherapy in newly diagnosed AML showed composite remission rates of 96% for NPM1-mutated and 90% for KMT2A-rearranged patients, with high MRD negativity rates (85% local, 79% central for NPM1-m) and durable responses at 12 months.

• Median overall survival was not reached in either cohort; 12-month OS was 94% for NPM1-m and 71% for KMT2A-r, both exceeding historical benchmarks.

• Median follow-up was 17.6 months for NPM1-m and 11 months for KMT2A-r, with most patients still on study or in follow-up.

• In relapsed/refractory NPM1-mutant AML, ziftomenib plus venetoclax/azacitidine showed 48% ORR in venetoclax-experienced and 87% ORR in ven-naïve patients, with deep, durable responses.

• Ziftomenib did not delay neutrophil or platelet recovery; time to count recovery was around 28 days, indicating no additive myelosuppression.

Safety and tolerability

• No new or unexpected adverse events were observed; main side effects were consistent with intensive chemotherapy, such as cytopenias and febrile neutropenia.

• Differentiation syndrome and QTc prolongation were rare, manageable, and did not require permanent discontinuation; no Grade 4 events reported.

• No evidence of additive myelosuppression or increased induction mortality; 60-day mortality was 2% for NPM1-m and 4% for KMT2A-r.

• Patients tolerated maintenance ziftomenib well, with minimal chronic toxicity and no need for dose adjustments.

• Ziftomenib did not delay neutrophil or platelet recovery.

Regulatory and commercial outlook

• Ziftomenib (KOMZIFTI) is FDA-approved as monotherapy for relapsed/refractory NPM1-mutated AML; combination use is investigational.

• KOMET-017 Phase 3 trials are designed for global regulatory credibility, with central MRD testing as a co-primary endpoint for accelerated approval.

• Robust launch and early momentum in the relapsed/refractory setting, with a U.S. market opportunity estimated at $350–400 million, potentially expanding to $7 billion if frontline use is established.

• The company is focused on expanding ziftomenib use across the AML treatment continuum, aiming to benefit up to 50% of AML patients.

• Ongoing studies will further evaluate ziftomenib in combination with venetoclax/azacitidine and in maintenance settings.