Highlight

1.Huge drug market for AML and clear MOA

1) High morbidity and mortality: AML accounts for ~30% of leukemia’s and ~40% of leukemia-related deaths.

2) The frequency of FLT3 mutations in AML patients is highest (25-30%). FLT3-ITD is a common mutation (75% FLT3 mutation) and correlates with a poor prognosis and highest risk of relapse found in all AML patients.

3) FLT3, a RTK, is normally expressed on immature hematopoietic cells and has functions of developing stem cells and immune systems.

4) The asset is a potent, orally available small molecule TKI against FLT3-ITD and its downstream signal phosphorylation (AKT, ERK,STAT5) to treating AML.

2.A selective inhibition, showing low nanomolar inhibition of FLT3.

3.Excellent anti-tumor effect in vivo.

1) Asset was able to induce complete regression of tumors at 4.5 mpk QD, with 172% TGI.

2) Significant efficacy in prolongation of survival in MOLM-13 systemic xenograft model. The ILS of the low dose of this asset is 56%, which is superior to that of the high dose of sorafenib.

3) Additive effects in the combination cytarabine or CDK4/6i.

4.Excellent clinical trial date: good safety, well-tolerated, lower dose but better effect.

For R/R AML (≥ 2L) pts with FLT3-ITD mutation in 40 mg dose asset, CRc=57.1%, CR=10.7%;

However, for R/R AML pts with FLT3 mutation in 120 mg dose Gilteritinib, CRc=54.3%, CR=14.2%.

Research progress：

Phase 3 trial is on-going;

Good safety profile, well-tolerated both in preclinical and clinical studies;

Interim results: CRc 57.1% for FLT3-ITD mutation AML.