Kura Oncology and Kyowa Kirin Announce FDA Approval of KOMZIFTI(TM) (ziftomenib), the First and Only Once-Daily Targeted Therapy for Adults with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

Executive Summary

• The U.S. Food and Drug Administration granted full approval of KOMZIFTI™ (ziftomenib) for adult patients with relapsed or refractory NPM1‑mutated AML who have no satisfactory alternative treatment options.
• Approval is based on the pivotal KOMET-001 trial (112 patients), which showed a 21.4% CR + CRh rate, median duration of response of 5.0 months, and a favorable safety profile with no boxed warning for QTc prolongation or torsades de pointes.
• Kura Oncology will launch the product immediately in the U.S., supported by a patient‑access program (Kura RxKonnect™) and a joint commercialization plan with partner Kyowa Kirin.

Key Details

• Indication: Relapsed/refractory AML with a susceptible NPM1 mutation; no satisfactory alternative therapies.
• Dosage Form: Once‑daily oral menin inhibitor (ziftomenib).
• Clinical Trial Results (KOMET-001, NCT04067336):
• CR + CRh rate: 21.4% (95% CI 14.2–30.2%).
• Median duration of CR + CRh: 5.0 months (95% CI 1.9–8.1).
• Median time to first response: 2.7 months (range 0.9–15).
• 88% of responders achieved response within 6 months of treatment start.
• Safety Highlights:
• No boxed warning for QTc prolongation or torsades de pointes.
• Boxed warning only for differentiation syndrome (DS); DS occurred in 26% of patients, grade 3 in 13%, fatal in 2 cases.
• Most common adverse reactions (≥20%) included elevated liver enzymes, infections, electrolyte abnormalities, diarrhea, nausea, fatigue, and edema.
• Regulatory Milestone: Full FDA approval granted ahead of the PDUFA target action date; no post‑marketing study commitments disclosed in release.
• Commercialization & Access:
• Kura leads U.S. development, regulatory, manufacturing, and commercial strategy; Kyowa Kirin handles non‑U.S. markets.
• Joint U.S. territory commercialization plan to be executed by both companies.
• Patient support program “Kura RxKonnect™” available 8 am–8 pm ET via phone (844‑587‑2777) or online (www.KuraRxKonnect.com).
• Conference Call: Kura will host a call/webcast on November 13, 2025 at 12:30 pm ET / 9:30 am PT; replay available on the company website.
• Future Development: KOMZIFTI is being investigated for front‑line AML with NPM1 mutations, KMT2A translocations, and FLT3 mutations, as well as in combination regimens.

Notable Quotes

“KOMZIFTI combines compelling efficacy, a favorable safety profile, compatibility with concomitant medications, and convenient once‑daily oral administration in a population with few effective treatment options.” – Troy Wilson, Ph.D., J.D., President & CEO, Kura Oncology

“The approval of KOMZIFTI underscores our commitment to advancing precision medicines to address the genetic drivers of disease in hematology and oncology.” – Takeyoshi Yamashita, Ph.D., EVP & CMO, Kyowa Kirin