Arbutus Biopharma (ABUS) Jefferies 2024 Global Healthcare Conference summary

Strategic vision and clinical focus

• Aims to achieve a functional cure for hepatitis B using combination therapies of direct-acting antivirals and immunologics, targeting a cure rate of at least 20% with finite treatment duration.

• Lead asset imdusiran (AB-729), a GalNAc-conjugated siRNA, is in multiple phase IIa trials (IMPROVE I, II, III) with various combinations, including interferon, VTP-300 vaccine, and durvalumab.

• AB-101, an oral PD-L1 inhibitor, is in phase I development, with plans to combine it with imdusiran in future studies.

• Strategic partnership with Qilu Pharmaceutical in China includes $40M upfront and $50M equity investment, expanding reach in the Asia Pacific region.

• Strong cash position and ongoing patent litigation regarding LNP technology with Moderna and Pfizer, with a trial set for April 2025.

Clinical trial updates and data highlights

• IMPROVE I phase IIa data showed 33% of patients achieved undetectable HBsAg and HBV DNA at end of treatment, with sustained S antigen loss and high HBV S antibodies.

• Combination of imdusiran and VTP-300 vaccine demonstrated sustained S antigen reduction, with additional benefit from nivolumab in a third cohort.

• IMPROVE-3 is evaluating imdusiran with durvalumab, targeting immune exhaustion via the PD-1/PD-L1 axis.

• AB-101 demonstrated safety, receptor occupancy, and T cell function reconstitution in preclinical and early clinical studies.

• Key opinion leaders consider functional cure rates above 15% clinically meaningful; further data expected by year-end.

Market and scientific rationale

• Over 250 million people globally are affected by HBV, with significant prevalence in Asia Pacific due to limited vaccine access and mother-to-child transmission.

• Combination therapy strategy aims to reduce HBV DNA, lower S antigen, and boost immune response for a finite, more tolerable treatment.

• Imdusiran offers dosing every 8 weeks, robust S antigen declines, and immune reactivation, providing a competitive advantage.

• Small molecule PD-L1 inhibitor AB-101 is designed to avoid immune-related adverse events seen with antibody therapies.