Braveheart Bio (BRVE) investor relations material

Key clinical insights and data

• Lead asset BHB-1893 showed rapid and deep reduction in biomarkers and improvements in cardiac structure in phase II studies for both obstructive and non-obstructive hypertrophic cardiomyopathy (HCM).

• Phase II data in non-obstructive HCM demonstrated significant improvements in diastolic function, cardiac relaxation, and wall thickness, suggesting potential for reverse remodeling.

• Over 86% of obstructive HCM patients in phase II achieved a complete Valsalva gradient response, with shallow LVEF reduction and tight pharmacokinetics.

• Market research indicates that improvements in cardiac structure are highly valued by physicians for treatment decisions.

• Placebo effects in non-obstructive HCM trials were larger than in obstructive, but high-dose arms showed meaningful clinical effects.

Strategic development plans

• Global phase III studies are underway for both obstructive and non-obstructive HCM, with simplified titration regimens and dual primary endpoints (KCCQ and peak VO2) planned.

• Interim analysis for the obstructive HCM phase III will include 60 patients (2:1 randomization), focusing on gradient, safety, and pharmacokinetics, with Asian patient inclusion to support global relevance.

• Regulatory strategy includes leveraging partner Hengrui's phase III data and interim analysis for accelerated filing, with ongoing FDA discussions.

• The goal is to be second to market in non-obstructive HCM and to expand CMI use beyond centers of excellence by reducing echo monitoring burden.

• Open label extension data for obstructive HCM expected later this year, with focus on long-term exercise capacity improvements.

Market opportunity and business outlook

• Both obstructive and non-obstructive HCM are seen as large orphan market opportunities, with non-obstructive potentially as large as obstructive due to new epidemiologic data.

• Simplified dosing and reduced monitoring could expand CMI adoption, especially in community settings.

• Strong capital position post-IPO enables a standalone path with funding into 2029, though strategic partnerships remain an option.