Kura Oncology (KURA) investor relations material

Key clinical data and trial results

• Ziftomenib combined with azacitidine and venetoclax achieved 86% composite complete remission and 73% complete remission in newly diagnosed NPM1-mutated AML, with 68% MRD negativity and median time to MRD negativity of 9.4 weeks.

• In relapsed/refractory NPM1-mutated AML, the triplet showed a 65% overall response rate and 48% composite complete remission, with median duration of response of 39.9–40 weeks and median overall survival of 54.9 weeks.

• In relapsed/refractory KMT2A-rearranged AML, the combination achieved a 41% overall response rate and 28% composite complete remission, with median overall survival of 21.1 weeks.

• Several patients proceeded to hematopoietic stem cell transplant and ziftomenib maintenance therapy.

• Clinical activity was observed in both venetoclax-naïve and -exposed patients, with higher response rates in venetoclax-naïve subgroups.

Safety and tolerability

• The combination was well tolerated, with a safety profile consistent with venetoclax/azacitidine alone and manageable adverse events.

• Low rates of ziftomenib-related myelosuppression; neutrophil and platelet recovery times were similar to standard therapy.

• Differentiation syndrome occurred in up to 26% of relapsed/refractory NPM1-m AML patients, with 13% grade 3 and two fatal cases; most cases were managed with protocol mitigation.

• QTc interval prolongation was reported in up to 12% of patients, with 8% grade 3; one case each of grade 2 differentiation syndrome and grade 3 QTc prolongation in newly diagnosed cohort were managed without discontinuation.

• Permanent discontinuation due to adverse reactions occurred in 21% of patients; serious adverse reactions in 79%, most commonly infection, febrile neutropenia, and differentiation syndrome.

Clinical development and future strategy

• The registrational phase 3 KOMET-017 trial is underway, enrolling a broad frontline AML patient pool and evaluating ziftomenib in both non-intensive and intensive chemotherapy settings.

• Additional studies are ongoing with combinations involving FLT3 inhibitors, FLAG-IDA, and LDAC, aiming to broaden clinical utility and support future guideline submissions.

• Data generation in various combinations is prioritized to provide robust evidence for real-world adoption and regulatory discussions.

• Ziftomenib's development program spans multiple AML subtypes, including NPM1-m, FLT3-m, and KMT2A-r.

• KOMZIFTI (ziftomenib) is FDA-approved for relapsed/refractory AML with susceptible NPM1 mutation and is commercially available in the US.