PTC Therapeutics, Inc. Morgan Stanley 24th Annual Global Healthcare Conference

Absolutely. First, Judah, thanks for having us.

Sure. Great to be here.

PTC is a global rare disease company that discovers, develops, and commercializes innovative therapies for diseases of high unmet need. We have a robust commercial portfolio with six marketed products that we deliver to patients around the world, as well as a broad R&D portfolio, including two scientific platforms, including our oral small molecule platform, which has already demonstrated to be quite valuable and well-validated. So far in 2026, we're off to a great start with the continued strong momentum of the Sephience launch that continues in the U.S. and worldwide. We remain on target to achieve the important milestone of being cash flow breakeven this year. So I think that really is an important milestone for the company, and is something that we've been working for over the past couple of years as we transform PTC into a diversified, sustainable, profitable rare disease company.

Yeah, absolutely. Look, we're incredibly excited about the success of SKYCLARYS thus far, and we remain incredibly bullish on the overall opportunity and we're very much on track to reaching that $2 billion-plus level of revenue we've been talking about. I think there have been a number of surprises for everyone, but I'd say first and foremost, what we're not surprised about is the success.

I think this marketplace was quite misunderstood, and there was a belief that since there were two approved therapies that didn't reach that billion-dollar mark, it was hard for a PKU therapy to get there.

That's simply not true. The success is really attributable to a number of important factors.

First, SKYCLARYS, the drug itself. It's a differentiated molecule with a dual mechanism of action that uniquely allows it to have benefit for the full spectrum of PKU patients, regardless of severity. It's classical and non-classical underlying mutation, regardless of age, as well as being oral, convenient, and having a very favorable safety and tolerability profile. That's differentiating. Second is the fact that all the pillars for commercial success were there.

Right. There is newborn screening, there are centers of excellence.

It is a well-aggregated community. All that was missing was a therapy that could address the broad 17,000 patients in the U.S. and 58,000 patients in global markets where we could commercialize. Next is our team. We are an experienced team in terms of delivering rare disease therapies to patients, not only in the U.S., but globally. We have a patient services team that is very well accustomed to helping manage through a competitive landscape, providing necessary patient support. Quite simply, we are very good at executing on commercializing rare disease drugs. So when you put that combination together of the marketplace, the quality of the product, and the execution of our teams, this is what you get.

We were certainly excited to have 17 presentations, to be able to continue to reinforce the differentiated nature of Sephience, as well as to bring forward real-world evidence that is substantiating that Sephience has been able to deliver meaningful benefits, not only in terms of phenylalanine lowering, but diet liberalization, increased protein intake in the full spectrum of patients. I think a couple of things I want to point out. One is we are still early in this launch. I think when we think about all the success we have had to date, we still believe we have a very long way to go just given the size of the population and the market opportunity. The SSIEM and these kinds of conferences are part of the launch playbook.

You basically come in a year into the launch and be able to continue to support product differentiation and help foster uptake for the large number of patients who we believe can benefit from Sephience. I'd say some of the key data points were the fact that people have asked, "Well, we know that your data show that any individual who is on sapropterin and a KUVAN brand or generic has a much better response with Sephience, whether in terms of Phe lowering or diet liberalization. But what about more severe patients in your trial?