Scholar Rock Holding Corporation (SRRK) Presents at Goldman Sachs 47th Annual Global Healthcare Conference 2026 Transcript

Unknown Analyst

Maybe to start, if you could provide us an overview of your company for those who may not be familiar, including the updates that we’ll see within the next year.

David Hallal
CEO & Chairman of the Board

Great. Thanks, Tommy. It’s great to be here once again at the 47th Goldman Sachs Healthcare Conference. David Hallal, Chairman and CEO of Scholar Rock, which is a Cambridge-based biotech company that has been focused on myostatin biology, and we’re very proud that we are the world leaders in myostatin biology. This is obviously a target that was identified in 1997.

The first anti-myostatin inhibitor went into the clinic in 2004, 2005. And really, over the period of these last 20-some years, there have been multiple myostatin inhibitors that have failed in clinical development. We stand alone with our unique technology platform and approach to myostatin biology as the first and only company that has successfully developed a myostatin inhibition program through mid- and late-stage development with a successful Phase III trial with our lead asset, which is apitegromab for children and adults living with SMA.

As Tommy has asked, what does the next 12, 18 months look like for our company? We’re super excited having hit a statistically significant and clinically meaningful benefit in motor function with the highest gold standard measure of the Hammersmith Motor Function Scale in SMA for children and adults living with SMA to now be on file with both