Hemab Therapeutics (COAG) investor relations material

Company vision and pipeline overview

• Focused on developing a franchise for blood coagulation disorders, with multiple clinical assets in rare bleeding diseases.

• Lead program, sutacimig, completed phase I/II in Glanzmann's thrombasthenia and is set to enter phase III in the second half of the year.

• Ongoing phase II study in Factor VII deficiency, with initial data expected in 2026 or early 2027.

• HMB-002 in phase I/II for von Willebrand disease, with multiple ascending dose efficacy data anticipated in 2026 or early 2027.

• HMB-003 data to be presented at ISTH in July, with further pipeline assets in development.

Clinical data and regulatory alignment

• Sutacimig showed up to 87% reduction in annualized treated bleed rate and 100% reduction in life-threatening bleeds in high-risk patients.

• Robust safety and tolerability profile established, with risk mitigation strategies for thrombotic events based on phase I/II learnings.

• Regulatory authorities have endorsed the transition to pivotal phase III, with open-label extension data supporting sustained efficacy and safety.

• Breakthrough Therapy designation enables ongoing dialogue with FDA; alignment achieved on endpoints, study design, and statistical analysis.

• Final phase III design (single-arm vs. randomized) and dosing regimen under discussion, with global patient enrollment planned.

Study design and operational plans

• Phase III will include a run-in period of up to six months to prospectively collect baseline bleed rates.

• Target enrollment is 75-100 patients, with global recruitment across the US, Europe, Gulf States, and Japan.

• Enrollment rates expected to be similar to hemophilia trials, with no major differences between single-arm and randomized designs.

• Safety mitigation focuses on dose selection and investigator education rather than extensive exclusion criteria.