Braveheart Bio (NASDAQ:BRVE) presented one-year data from a small study of its experimental heart drug BHB-1893 and outlined plans for a fixed-dose trial, as the company advances the treatment through late-stage development for hypertrophic cardiomyopathy.
The data, presented with partner Hengrui at the American Heart Association Scientific Sessions 2026, came from an open-label extension of a Phase II study in patients with obstructive hypertrophic cardiomyopathy, or HCM. All 42 patients in the original study chose to enter the 52-week extension, which did not have a placebo group.
Chief Executive Officer Travis Murdoch said the company plans to start a separate Phase IIb fixed-dose study, called TRUEHEART-HCM, in the first half of 2027 after receiving supportive feedback from the Food and Drug Administration on the approach. Braveheart is also conducting or initiating Phase III studies in both obstructive and non-obstructive forms of the disease.
One-year results and safety
HCM is an inherited heart disease characterized by thickened heart muscle. In the obstructive form, that thickening can impede blood flow out of the heart. BHB-1893 is a cardiac myosin inhibitor, a class of drugs intended to address the heart’s excessive contraction.
In the extension study, patients started on 40 milligrams of BHB-1893 twice daily, with opportunities to increase the dose at weeks one and two. Before titration, 67% had a provoked outflow tract gradient below 30 millimeters of mercury at week one, a threshold the company defined as a complete gradient response. At the same point, 83% had a resting gradient below that level, Murdoch said.
At 52 weeks, patients showed an average improvement of 2.7 milliliters per kilogram per minute in peak oxygen consumption, a measure of exercise capacity, from the start of the extension. A patient-reported symptom score improved by 17.3 points, and 60% of patients were classified as having no limitation during ordinary physical activity, Murdoch said.
There were no severe or serious adverse events while patients were receiving treatment in the extension, according to Murdoch. He said two patients in the study had left ventricular ejection fraction readings below 50%, a measure monitored because drugs in the class can reduce the heart’s pumping function.
Murdoch also pointed to changes in cardiac measurements after treatment was withdrawn as potential evidence of disease modification. He said outflow gradients and the heart-failure biomarker NT-proBNP rebounded less after 52 weeks of treatment than after the earlier treatment period. The company characterized the finding as a potential signal, rather than confirmation of a lasting effect.
Non-obstructive data and development plans
Dr. Anjali Owens, an associate professor of medicine at the University of Pennsylvania’s Perelman School of Medicine, reviewed previously presented results from an 84-patient, placebo-controlled Phase II study in non-obstructive HCM. Patients received a low dose, a high dose or placebo for 12 weeks.
Owens said the high-dose group had reductions from baseline of 68% in NT-proBNP and 60% in troponin, a marker of cardiac injury. She also described improvements in measures of heart relaxation and structure. Four participants had a temporary ejection fraction decline below 50%; all returned to at least 50% after dose reduction, and none discontinued because of low ejection fraction, she said.
Braveheart’s planned TRUEHEART study will evaluate a fixed 40-milligram twice-daily dose without titration visits in symptomatic obstructive HCM patients receiving standard care. Murdoch said the approach could help expand access in community cardiology practices, while patients needing a higher dose might be referred to specialized centers.
The company has initiated LIONHEART-HCM, a Phase III study comparing BHB-1893 with the beta blocker metoprolol and allowing one dose increase at week four. It is also initiating the placebo-controlled NOBLEHEART-HCM Phase III study in non-obstructive HCM.
Hengrui’s fully enrolled Phase III study is expected to produce top-line results in the first half of 2027, Murdoch said. Braveheart also expects an interim LIONHEART analysis and longer-term non-obstructive study data in 2027. The company said its cash runway extends into 2029.
About Braveheart Bio (NASDAQ:BRVE)
We are a clinical‑stage biopharmaceutical company focused on developing therapies for patients with hypertrophic cardiomyopathy (“HCM”) and other serious cardiovascular diseases. Our lead product candidate, BHB‑1893, is a next-generation oral small‑molecule cardiac myosin inhibitor (“CMI”), that we are developing for the treatment of obstructive HCM (“oHCM”) and non‑obstructive HCM (“nHCM”). Our goal is to improve the treatment options for these patients by enhancing speed of onset, depth of gradient response, systolic safety, and reversibility, and reducing prescribing complexity.
