Hemab Therapeutics Holdings (COAG): ISTH Updates: HMB-002’s Encouraging Efficacy, Reveal of HMB-003, +VGA039’s Updates

Research evidence excerpt

Hemab Therapeutics Holdings (COAG): ISTH Updates: HMB-002’s Encouraging Efficacy, Reveal of HMB-003, +VGA039’s Updates

Goldman Sachs Hemab Therapeutics Holdings (COAG)

Exhibit 3: VIVID2 showed substantial ABR reduction in 3 patients with historical ABR>50 but worse effect in patient with

lower ABR

SC Dose of Baseline FVIII

Cohort Type/sub-type Weight (kg) ABR before VGA039 ABR after VGA039 Change in ABR (%)

VGA039 activity

F 4.5 mg/kg Type 3 5 IU/dL 100 54.8 6.4 -88%

Type 1 + Mild

F 4.5 mg/kg 9 IU/dL 72 5 6.4 +28%

hemophila A

F 4.5 mg/kg Type 2M 23 IU/dL 46.7 104 25.6 -75%

F 4.5 mg/kg Type 3 2 IU/dL 163.7 771 112.9 -85%

Source: Company data, Goldman Sachs Global Investment Research

HMB-002 is well tolerated, no serious TEAEs

HMB-002 was generally well tolerated. 53.8% patients experienced TEAEs (n=1 or 7.7%

had Gr.3+ TEAE). Most TEAEs were mild to moderate in severity, no serious TEAEs

occurred, no events were considered related to HMB-002, and no patient discontinued

due to TEAE. There were no thromboembolic events, no injection site reactions, no

thrombocytopenia, and no hypersensitivity reactions.

HMB-003 announced as plasmin inhibitor designed to reduce bleeding across

multiple settings beginning with heavy menstrual bleeding. HMB-003 is a novel

fatty-acid-conjugated peptide antifibrinolytic, designed to stabilize clots and reduce

bleeding across multiple settings. In preclinical studies, HMB-003 showed potent and

selective plasmin inhibition. Following a single subcutaneous dose in minipigs, HMB-003

achieved peak plasma levels within hours and sustained antifibrinolytic activity for ~1

week, supporting the potential for cycle-matched dosing in heavy menstrual bleeding.

VGA039’s updated Ph. 1/2 efficacy results continue to look encouraging, but may