Aptose Biosciences Inc.

Pipeline

Product	Mechanism	Ownership	Indication	Preclinical	Clinical Proof-of-Concept	Pivotal
APTO-253	MYC	Aptose: WW	AML & MDS	complete AML & MDS Preclinical Phase complete	in progress Clinical Proof-of-Concept Phase in progress	not started Pivotal Phase not started

The Story Behind APTO-253

APTO-253 is a first-in-Class MYC Inhibitor currently in a phase 1b clinical trial for the treatment of patients with AML and MDS.

Tremendous Interest in Targeting MYC

MYC is a transcription factor that regulates cell growth, proliferation, differentiation, and apoptosis, and overexpression amplifies new sets of genes to promote oncogenesis. The MYC oncogene is overexpressed in hematologic cancers, including both myeloid and lymphoid tumors, and is typically associated with poor survival. Additionally, MYC is dysregulated or overexpressed in several solid tumors, including ovarian, breast, colorectal, pancreatic and gastric cancers.

APTO-253 as a Small Molecule MYC Inhibitor

APTO-253 does not target directly the MYC protein, but rather binds directly and selectively to a regulatory motif in the promoter of the MYC oncogene and dramatically down-regulates expression of the MYC protein in tumor cells, leading to apoptotic cell death. Thus, APTO-253 may serve as a safe and effective MYC inhibitor for MYC-driven or MYC-dependent tumors that combines well with other anti-tumor agents.

Clinical Development

Phase 1b Clinical Study of APTO-253

The Phase 1b, multicenter, open-label dose-escalation clinical trial of APTO-253 is designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamic responses and establish the recommended phase 2 dose of APTO-253 as a single agent. APTO-253 is being administered once weekly, over a 28-day cycle. The dose-escalation portion of the study is expected to enroll up to 20 patients with relapsed or refractory AML and high-risk MDS, and is designed to then transition, as appropriate, to single-agent expansion cohorts in AML and MDS, followed by combination studies.

We have completed dosing of the first four cohorts in our Phase 1b trial with APTO-253, with only one patient required in each of the first two cohorts and three patients in subsequent cohorts. The clinical experience to date has shown continued favorable safety and tolerability in heavily pretreated patients with relapsed or refractory AML and MDS, and continued dose-related exposure, with sustained active drug species in the 2-3µM range at the fourth dose level of 100mg/m2. Biomarker data from AML and MDS patients have demonstrated reductions in MYC gene expression in their peripheral blood cells. We are currently dosing patients at the fifth dose level of 150mg/m2.

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Posters & Presentations

Our robust library includes numerous publications, including presentations, posters, and other media that provide additional information on our drug candidates, pre-clinical and clinical studies.

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