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Hemab Therapeutics Holdings (COAG): ISTH Updates: HMB-002’s Encouraging Efficacy, Reveal of HMB-003, +VGA039’s Updates
研报英文原文证据摘录
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
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