BiotechTV’s Post

Transcript

First of all, it's Carla Rock. We eat, breathe and sleep science. So we're all about to science. And to your point, absolutely our target, the growth factors that we're focused on is a family of growth factors called. TGF beta superfamily that are involved in growth and differentiation across a number of cellular processes including controlling muscle growth as well as controlling response, for example to immune stimulus. So we were in a number of places. The strategy which was the foundation of the company as nature tries to protect and control is that we target the precursor or pro form of these growth factors before they're activated. And that was really the foundation of the company, that structural insight. And by doing that we blocked the growth factor before it's activated and are very focused and selective on how we manage to control the growth factor. Just to take a step back, very exciting time for us as we're waiting for the phase three data. This will really catapult us into potential. Being a commercial stage company. But we did have a proof of concept study that we studied in combination for example, with nursing, nursing and we're able to show with that is that we can't increase on approved regulatory functional scale the motor function, we get improvement on that. And so that allowed us to design the phase three trial taking the best of the phase two design. So very good randomized trial on the background of the standard of care therapy. The primary endpoint is the same one we used in the proof of concept study, it's regulatory accepted endpoint. Discussed the protocol both with the FDA and in the European Medicine Agency. So a lot of good work going into the trial, a lot of enthusiasm to recruit for this study. And now we're on the threshold of coming close to reporting on the data because of our foundation. We've been interested in myostatin for years. We went into spinal muscular atrophy because that was our first opportunity. But the role that muscle plays in metabolism and in healthy weight management was the reason we entered into this space. And to your point. The current therapies for weight loss are excellent. Weight loss is profound, but unfortunately with the muscle loss as well, it leads to issues perhaps with managing glucose or not maintaining healthy weight styles. So our view is we can enhance and preserve, augment the profile, but importantly where we started because we targeted so specifically and so selectively that we can do this without bringing additional risk to the combination over the risk benefit ratios. We we feel really good about it. We've got a program that we're testing that now in clinic. And can you describe what the development path would be for that? Like for example, Azure developing it. Would you want or need to partner with one of the GLP companies? Or can you just kind of test this with a GLP and if your hypothesis works, get it approved and sell it is one piece of a combination by yourself? Like what would be the path forward? Well, for an area like SMA we feel like. They can commercialize that ourselves because it's orphan indication and we know the patients and the community and the centers. Obesity of course, is a general medicine therapeutic area that would be pretty big for a company our size to tackle. So our thinking around it is we can do a proof of concept to show that our strategy works, but optimally it would be a partner program that we would look for someone with the potential therapy that we could enhance their profile. So it will ultimately be a partner program. But right now, what we can control is to demonstrate that our therapy works against the concept of preserving lean muscle mass.