Braveheart Bio (BRVE) Investor update summary

Program and opportunity

• BHB-1893 is a next-generation cardiac myosin inhibitor with rapid onset, predictable PK and a shallow LVEF exposure response; oHCM and nHCM represent ~700K–960K U.S. patients.

• Cardiac myosin inhibition is clinically validated; nHCM has no approved therapy. Global development includes Phase 3 programs in both HCM forms, an oHCM fixed-dose Phase 2b study and a planned long-term extension.

oHCM clinical data

• In the 42-patient, 52-week Phase 2 OLE, 90% achieved Valsalva LVOT gradient <30 mmHg during treatment; 67% met the threshold by Week 1 before titration, and 83% had resting gradient <30 mmHg then.

• At Week 52, peak pVO₂ improved 2.7 mL/kg/min; 70% gained ≥1.5 and 50% ≥3.0 mL/kg/min. VE/VCO₂ slope improved 4.1.

• KCCQ-CSS increased 17.3 points, with 45% gaining ≥20 points; 60% reached NYHA Class I. Resting and Valsalva gradients and NT-proBNP showed significantly less rebound after washout, while maximal LV wall thickness continued to improve.

• More than 50 patient-years of exposure included two patients with LVEF <50%; no on-treatment severe or serious adverse events were reported. At Week 52, 57.1% remained on 40 mg BID and 19.0% on 60 mg BID.

nHCM clinical data

• The 12-week placebo-controlled Phase 2 enrolled 84 symptomatic adults; high-dose treatment used 20-40-60 mg BID.

• High-dose BHB-1893 reduced NT-proBNP 68% and troponin 60% from baseline; 42% of patients at 60 mg had NT-proBNP below 125 pg/mL.

• Echocardiographic measures improved, including septal and lateral e′ by 2.0 and 2.6 cm/s, LAVI by 7.1 mL/m², LV mass index by 17.4 g/m² and LV wall thickness by 3.3 mm.

• Four patients (7%) had transient LVEF <50% (46%–49%); all recovered to ≥50% after dose reduction, with no low-LVEF discontinuations.