TuHURA Biosciences (HURA) Canaccord Genuity's 46th Annual Growth Conference summary

Strategic focus and pipeline overview

• Focused on overcoming primary and acquired resistance to cancer immunotherapies, with three distinct technologies in development.

• Lead program is a phase III trial for frontline Merkel cell carcinoma, with enrollment completion expected in the second half of next year and top-line data by late next year or early 2028.

• Special Protocol Assessment agreement with the FDA for the phase III trial, eliminating the need for a confirmatory trial.

• Second asset is a VISTA-inhibiting antibody targeting molecularly defined subsets of AML, with phase I/II trials starting soon.

• Novel immune-targeting antibody-drug conjugates in preclinical development, with proof of concept data expected later this year.

Clinical trial design and regulatory engagement

• Phase III Merkel cell carcinoma trial uses a randomized, placebo-controlled design with IFx plus pembrolizumab versus pembrolizumab alone.

• Key secondary endpoint is progression-free survival, enabling accelerated approval without a separate confirmatory trial.

• FDA collaboration under Project Front Runner initiative encouraged moving IFx into the frontline setting to maximize patient benefit.

• SPA with the FDA provides regulatory clarity and risk mitigation for the pivotal trial.

• Orphan drug designation expected for IFx in Merkel cell carcinoma and VISTA in AML.

Scientific rationale and preliminary results

• IFx technology uses plasmid DNA encoding a bacterial protein to activate innate and adaptive immune responses against tumors.

• Early clinical data in Merkel cell carcinoma showed durable responses in patients previously resistant to checkpoint inhibitors.

• VISTA-inhibiting antibody targets a key checkpoint highly expressed in NPM1 and FLT3-mutated AML, addressing a major unmet need.

• Initial phase I/II AML trial will focus on patients failing menin inhibitors, with morphologic leukemia-free state as a key endpoint.

• ADC platform targets delta opioid receptor on immunosuppressive cells, with potential applications in both cancer and autoimmune diseases.