Braveheart Bio, Inc. Common Stock 12th Annual Cantor Fitzgerald Global Healthcare Conference

Sure. Yeah. No. It's a little over a year ago that I joined as the first employee at Braveheart. For me, I, at the time, was at Biogen and just had recently sold my previous business, HI-Bio, to Biogen, joined Biogen, and was enjoying myself actually launching some phase III studies and working with Chris Viehbacher, who now ends up being our chairman. There was emerging data from this program, which ultimately was presented last year at ESC. For me, the initial dataset that I found quite compelling was really around the rapidity and depth of gradient reduction in patients with obstructive hypertrophic cardiomyopathy in that initial phase I-B data. That was really the data for me that got me to get excited in the asset, join, and really start the company build with a great team.

Yeah. I think it's a really unique time for hypertrophic cardiomyopathy patients. We're seeing data emerge that really supports not only in the obstructive form of the disease, where now there's two approved agents, but also emerging data in the non-obstructive form of the disease where there have been no approvals. Over the last year, we've seen both external catalysts across the industry, and we've also had some really promising data emerge for our lead program, BHB-1893, in both obstructive and non-obstructive HCM. I think it's a great moment in the industry, and we're really focused on executing now on global phase III studies.

Sure. I would just see the opportunities slightly differently. I think they're both large opportunities. From an epidemiologic perspective, in previous years, it was thought that the obstructive form of the disease is actually larger than the non-obstructive form. I think what we're starting to see now as therapies make their way through development, is that in fact, there are probably more patients with a non-obstructive form of the disease than was originally perceived. We're seeing some new epidemiologic data emerge that the end market there is large, and probably as large as the obstructive form.

From the perspective of how we think about BHB-1893 sitting in those markets in a future state, I think there are different aspects of differentiation and potential differentiation that would drive, at least in our market research, adoption of a next-generation agent in obstructive, really around potential for differentiation on efficacy, safety, and/or ease of use. In the non-obstructive form of the disease, obviously there's a lot of white space. But we think that an agent that has a potential to be disease modifying from the perspective of structural improvements as well as showing improvements in feel and function would be a really optimal TPP.

Sure. We have a 12-week placebo-controlled study that was run by our partner, Hengrui, in the non-obstructive form of the disease, 28 patients per arm. I think the data were very encouraging and highly directional across metrics. I would include a number of key metrics. We saw rapid deep reduction in biomarkers, including NT-proBNP and troponin. We saw improvements in measures of diastolic function that were quite significant. So there's metrics on echo of cardiac relaxation, including e' prime, septal and lateral e' prime, that actually moved from abnormal values to normal values, paired with decreases in measures of really the back pressure of left atrial volume index, as an example. So those echocardiographic parameters and then also measures really of the disease, where we saw decreases in wall thickness, as an example.

When we take those metrics and look at them, they really suggest that there is a potential for reverse remodeling, and we think that's very exciting. We saw highly directional data as well in terms of KCCQ and peak VO2 and dose effects there that we think are highly directional and suggest a potential for real efficacy in this disease. Interestingly, when we did our market research, we did pretty extensive market research with ClearView Healthcare Partners. One of the things that came out of that was that these improvements in cardiac structure really are quite important to physicians as they think about treatment options. If you could have an agent that replicates that in a global phase III program, that would be an important part of how physicians think about treatment decisions.

Because, as you know, we want our patients to feel and function better, but ultimately, this is a disease of cardiac structure. So being able to show improvements in structure is an important part, I think, of the narrative.