PTC Therapeutics, Inc. (PTCT) Presents at Citi Annual Global Healthcare Conference 2025 Transcript

Geoffrey Meacham
Citigroup Inc., Research Division

[Audio Gap] My name is Geoff Meacham. I’m the senior biopharma analyst here, and I have Jarwei Fang from my team on here as well. We’re thrilled today to have PTC Therapeutics. With us on stage is Matt Klein, CEO. Matt, welcome. Good to see you.

Matthew Klein
CEO & Director

Thanks Geoff. Great to be here.

Geoffrey Meacham
Citigroup Inc., Research Division

So lots of questions on Sephience and on your R&D Day. But maybe just for those on the webcast, just give us a bit of a quick background or overall, and then we can get right into it.

Matthew Klein
CEO & Director

Yes, absolutely. So PTC is a global biopharmaceutical company. We are focused in rare diseases and other disorders of high unmet need. We have a robust commercial portfolio with 6 products that we market around the globe as well as an innovative R&D pipeline highlighted by our differentiated oral small molecule splicing programs.

2025 has been an incredibly successful year for us, highlighted by the U.S. and EU approval of Sephience, our oral therapy for PKU. As we shared at Q3 earnings, the launch is off to an incredibly strong start. And I’m proud to report we’re seeing continued strong momentum into the fourth quarter. And as we’ve described, this is really a foundational product for PTC that will lead us to cash flow breakeven and beyond in the near future. So really excited to get into the questions and talk about the launch.