Oryzon Achieves 95% ORR in Phase Ib AML Trial Ahead of Planned Phase III Pivot

With a 95% overall response rate and a planned Phase III trial on the horizon, Oryzon Genomics is approaching a CMC and manufacturing readiness inflection point for iadademstat, its selective LSD1 inhibitor, as Phase Ib data mature toward pivotal-scale requirements. Updated findings from two ongoing trials will be presented in poster sessions at the ASH 68th Annual Meeting, December 12–15, 2026, in New Orleans.

The ALICE-2 trial (NCT06357182) evaluated iadademstat in a triplet combination with azacitidine and venetoclax in newly diagnosed, unfit AML patients. Among 19 efficacy-evaluable patients at the July 2026 data cut, the combination produced a 95% ORR, an 84% composite complete remission (CRc) rate, and a 74% pure CR rate. Twelve-month overall survival reached 76%, with median OS not yet reached across the cohort. In patients carrying ELN2022 intermediate- or adverse-risk disease, 12-month OS rates were 100% and 74%, respectively.

The FRIDA trial contributed topline results for iadademstat combined with gilteritinib in heavily pre-treated, refractory FLT3-mutant AML, a population with limited salvage options. At the dose level selected as the Recommended Phase 2 dose, the CRc rate was 67%. A third abstract covers early-stage findings in myeloproliferative neoplasms, extending the mechanistic rationale for LSD1 inhibition beyond AML into adjacent hematological indications.

For manufacturing and process development teams, the transition from Phase Ib to the planned ALICE-3 Phase III trial will require documented process validation packages, tightened analytical control strategies, and supply-chain continuity planning consistent with ICH Q10 pharmaceutical quality system expectations. Epigenetic inhibitors with narrow therapeutic windows typically demand rigorous impurity profiling and stability data before pivotal-scale batch manufacture can be locked. The completeness of enrollment in ALICE-2 provides a defined patient population and dosing regimen, which sharpens the specification-setting work ahead.

The three accepted ASH abstracts, including the fully enrolled ALICE-2 dataset, will set the evidentiary baseline against which the Phase III protocol and its associated CMC package will be benchmarked.

Source: Oryzon Genomics via company press release, November 4, 2026.