Ziftomenib Drug Asset Due Diligence Report 2026

This Ziftomenib Drug Asset Due Diligence Report was built with PatSnap Life Sciences MCP workflows. Drug & Asset MCP establishes identity, ownership and stage; Clinical Trials MCP checks design, endpoints and readouts; Company & Deal Intelligence MCP reconstructs transaction precedent. Explore the MCP servers used in this report.

Decision date: 15 July 2026. Currency fields are presented in US$ millions as returned by the deal dataset. This is a screening memorandum, not legal, medical, patent or investment advice.

Approved

Highest phase

Registered trials

Result records

Matched deals

Executive recommendation: GO

Decision memo

Advance diligence: development maturity and available evidence support continued investment, subject to indication-specific safety, IP, and commercial gates.

The central underwriting question is whether Ziftomenib can convert its Small molecule drug profile and MLL1 x menin biology into clinically meaningful differentiation while preserving an investable safety, IP and commercial position.

1. Asset identity and development status

Asset	Ziftomenib (query alias: ziftomenib)
Modality / target	Small molecule drug; MLL1 x menin; MLL1 inhibitors, menin inhibitors
Highest global status	Approved
Originator	Kura Oncology, Inc.
Active developers	Kura Oncology, Inc., Kura Ltd.Sti., Kyowa Kirin Co., Ltd.

The MCP disease footprint includes Acute myeloid leukemia with mutated NPM1, Acute Myeloid Leukemia, Acute myeloid leukaemia with 11q23 abnormality. The highest-phase flag is a useful orientation point, but the licensing case depends on indication-level evidence and rights, not the global label alone.

2. Clinical program: design and endpoint audit

Registry	Phase	Status	Enrollment	Lead primary endpoint
NCT07623616	Phase 2	Recruiting	6	CR+CRh rate
JPRN-jRCT2031250550	Phase 2	募集中	6	CR+CRh割合
NCT07355335	Phase 1	Not yet recruiting	24	Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

The trial set should be diligenced for randomization, comparator relevance, endpoint hierarchy, analysis population, multiplicity, geographic mix and readout timing. For early-stage studies, safety and dose selection can be as decision-critical as response rate.

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3. Clinical readouts: what is known

REGISTRATIONAL PHASE 3 STUDIES OF ZIFTOMENIB IN COMBINATION WITH NONINTENSIVE OR INTENSIVE CHEMOTHERAPY FOR NEWLY DIAGNOSED NPM1‑M OR KMT2A-R ACUTE MYELOID LEUKEMIA (AML): THE KOMET-017 TRIAL

Phase 3; n=351; evaluation: Positive. Reported fields: CR = 73.0 %

Ziftomenib in combination with venetoclax and azacitidine in newly diagnosed NPM1-m acute myeloid leukemia: Phase 1b results from KOMET-007

Phase 1; n=39; evaluation: Positive. Reported fields: Treatment-emergent AEs(Grade ≥3) = 74.0 %

Ziftomenib in Relapsed or Refractory <i>NPM1</i> -Mutated AML

Phase 1/2; n=92; evaluation: Positive. Reported fields: CR/CRh = 22.0 % ( 14 - 32)

These fields are structured evidence signals, not a substitute for statistical review. The next diligence pass should reconcile denominators, confidence intervals, follow-up, censoring, dose cohorts and treatment-emergent toxicity against the original abstract, registry and protocol.

4. Market and competitive position

Ziftomenib addresses Acute myeloid leukemia with mutated NPM1, Acute Myeloid Leukemia, Acute myeloid leukaemia with 11q23 abnormality. Commercial attractiveness rests on addressable biomarker-positive patients, treatment-line placement, duration, administration burden, pricing and displacement of entrenched standards. The modality—Small molecule drug—must demonstrate a benefit large enough to offset class-specific safety and operational costs.

The strongest market-validation signal in this screen is partner behavior: 2 matched transaction record(s) indicate that sophisticated counterparties have assigned strategic value to the asset or its rights. That does not establish net present value; probability of success, remaining R&D spend, royalties, cost sharing and territorial scope still need modeling.

5. Transaction precedent and economics

Date	Transaction	Phase at deal	Disclosed economics
2024-11-20	Kura Oncology and Kyowa Kirin Announce Global Strategic Collaboration to Develop and Commercialize Ziftomenib in Acute Leukemias	NDA/BLA	US$330.0M upfront; US$1,161.0M milestones
2021-12-13	MD Anderson Cancer Center will assess the combined effectiveness of Kura's KO-539 and venetoclax in AML models.	Phase 2	Financial terms not disclosed

Headline values are not directly comparable. Diligence should normalize upfront cash, equity, development and sales milestones, tiered royalties, opt-in mechanics, cost sharing, change-of-control clauses and geography.

6. IP and freedom-to-operate screen

The milestone feed surfaced a patent-application signal described as “Menin inhibitors for the treatment of myeloproliferative neoplasms”. The milestone feed surfaced a patent-application signal described as “Process of making a menin-MLL inhibitor”. The milestone feed surfaced a patent-application signal described as “Methods of treating acute leukemias with a menin inhibitor in a combination therapy”.

The claim chart should separately test composition or sequence coverage, formulation and dosing, indication and biomarker claims, combinations, manufacturing know-how, prosecution history, term extensions and third-party blocking rights. Confirm that licensed patents, data and know-how track every granted territory and field.

7. Principal risks and diligence gates

Therapeutic index across KRAS/BRAF mutation subgroups
On-target toxicity and adaptive MAPK resistance
Lack of matched efficacy results in the current MCP result set

Cross-functional diligence should also test CMC comparability, supply chain, pharmacovigilance, regulatory correspondence, data integrity, investigator concentration, partner obligations and change-of-control restrictions.

8. Final go/no-go memorandum

GO