Kura Oncology and Kyowa Kirin Report Combination Data for KOMZIFTI(TM) (Ziftomenib) with Venetoclax and Azacitidine in Newly Diagnosed and Relapsed/Refractory AML

Executive Summary

• Kura Oncology announced robust efficacy and a favorable safety profile for KOMZIFTI (ziftomenib) combined with venetoclax + azacitidine in both newly diagnosed, chemotherapy‑ineligible AML and relapsed/refractory AML harboring NPM1 mutations or KMT2A rearrangements.
• In newly diagnosed NPM1‑mutated AML, 86% achieved composite complete remission (CRc) with 68% of responders attaining molecular MRD negativity; median duration of response and overall survival remain unreached.
• In relapsed/refractory disease, overall response rates were 65% (NPM1‑m) and 41% (KMT2A‑r), rising to 83% and 70% respectively in venetoclax‑naïve sub‑populations; median OS was 54.9 weeks (NPM1‑m) and 21.1 weeks (KMT2A‑r).

Key Details

• Study: Ongoing Phase 1a/1b KOMET‑007 (NCT05735184) – data cutoff 24 Sep 2025.
• Newly Diagnosed NPM1‑m AML (n=40, 37 evaluable):
• CRc 86%; CR 73%.
• 68% of CRc responders achieved MRD negativity by central NGS.
• Median follow‑up 26.1 weeks; median CR and OS not reached.
• 5 patients proceeded to HSCT (3 received post‑transplant ziftomenib maintenance).
• Relapsed/Refractory AML (n=83, 80 evaluable):
• NPM1‑m cohort (48 pts): ORR 65%; CRc 48% (median CRc duration 39.9 wks).
  • Venetoclax‑naïve: ORR 83%, CRc 70%.
  • Median OS 54.9 weeks (95 % CI 32.0–NE).
  • 14 patients underwent HSCT (5 entered maintenance, 5 pending).
• KMT2A‑r cohort (25 pts): ORR 41%; CRc 28% (median CRc duration 12.4 wks).
  • Venetoclax‑naïve: ORR 70%, CRc 60%.
  • Median OS 21.1 weeks (95 % CI 12.4–64.9).
  • 2 patients received HSCT and continued on maintenance.
• Safety:
• Triplet regimen well tolerated; myelosuppression rates low, recovery times comparable to venetoclax/azacitidine alone.
• Grade 2 differentiation syndrome (1 case) and grade 3 QTc prolongation (1 case) resolved without discontinuation.
• No new ziftomenib‑related QTc events in R/R cohorts.
• Regulatory Status: KOMZIFTI is FDA‑approved for relapsed/refractory AML with a susceptible NPM1 mutation and commercially available in the U.S.
• Future Plans: Ongoing registrational trials (KOMET‑017) in front‑line AML (both intensive‑chemo eligible and ineligible).
• Investor Communication: Virtual investor webcast scheduled for 12:30 p.m. ET / 9:30 a.m. PT on the day of release; slides from ASH presentations posted on Kura’s website.

Notable Quotes

• “These compelling data reinforce our conviction that ziftomenib has the potential to become a foundational, best‑in‑class menin inhibitor for patients with AML,” – Mollie Leoni, M.D., Chief Medical Officer, Kura Oncology.

Materiality: Material – Positive (significant new efficacy and safety data supporting expanded use of an FDA‑approved therapy).