PTC Therapeutics, Inc. Wells Fargo 21st Annual Healthcare Conference

Yeah, absolutely. Look, I think we're incredibly excited for the company's position today. We're exactly where we want to be. As we planned over the past couple of years to really transform the company, into the diversified, valuable, rare disease company we believe it could be. The Upstaza launch, we're now one year in, is off to a tremendous start. We remain exactly on the trajectory we believe we need to be on to get to that $2 billion-plus revenue potential. We're doing this in the context of a year where we believe that we can reach the important cash flow breakeven milestone, which really sets us up for future profitability. We have over $2 billion in cash in the bank.

The R&D portfolio is coming along quite well with the votoplam HD program, the Friedreich's ataxia program, with the recently acquired Fabry gene therapy program, as well as great progress from our splicing and inflammation platforms. We believe we are in a really strong position in terms of current revenue, future revenue growth, and a very strong financial position, which gives us incredible optionality, not only to pursue activities with our current assets, but also continue to look at business development opportunities that can bring short and medium-term top-line growth.

Yeah. I would say across the board, we are incredibly excited about the performance to date. Now we are about a year into the launch globally. First, the fact that it is a global launch and that we were able to launch in the U.S. and Europe and Japan within about 6 months of each other, which is pretty remarkable to be able to do that. I think most importantly is the incredibly positive feedback we are getting in terms of patient experience. When we started talking a lot about SUFIANCE, we said this is a very unique opportunity. This is a drug that has a dual mechanism of action. It is oral, it is well-tolerated, and it is able to provide potential important benefits for the full spectrum of PKU patients.

While there have been previous approved therapies, there still remained a significant unmet need for the vast majority of the 17,000 patients in the U.S. and 58,000 patients in the sort of the global markets where we think we could commercialize. What we have seen so far, again, has been positive. One, we have seen a number of patients return to clinic. If you go back a year from now, we had this conversation and everyone was saying, "Well, the launch is going to start with individuals who are on KUVAN, and they are going to get switched, and that is going to be the lion's share, the early part of the launch." Not the case. In fact, what we saw was the opposite. We saw an interest in getting individuals, kids and adults, who are not on a therapy onto a therapy.

We've seen individuals who've been remote to the clinics, therapy-naive adults who many believed would be very hard to get on drug. We ourselves believed this would be a goal for the latter part of the launch. They've come in and they've made up at this point about 25%-30% of the patients on drug. That's really, really good. A number of patients who've tried and failed are coming back in. We're hearing about the benefits, not only in terms of phenylalanine lowering, which is important in and of itself, but hearing great stories, seeing case reports and case series coming out of individuals being able to liberalize their diet, enjoy foods they never were able to enjoy before. Some folks being able to be completely liberalized.

This includes not only individuals with what are known as BH4 responsive mutations, but also those with classical and more severe patients. So in a nutshell, in this year, we've been able to access not only all, as you mentioned, 100% of the centers of excellence in the U.S., but every key market segment. Therapy-naive patients, tried and failed patients, switch patients from existing therapies, patients with non-BH4 responsive mutations, patients with classical PKU. I think that breadth at this stage of a rare disease launch is very impressive. What we're seeing now, I think we've said we probably have over 2,000 prescription patient start forms in the U.S. That leaves about 15,000 more or roughly.

We think we have a very long way to go, and to have established this foothold in every segment tells us the work now is to just go deeper and deeper into every segment rather than have to sort of create new ones. That's why we're so bullish on the opportunity.

Yeah. Look, there's 103 centers of excellence or so maybe different at different centers. I'd say by and large, there was a preference early in the launch for prescribers to get individuals who don't have a therapy to try a therapy. The idea was, okay, I may have a group of patients on KUVAN, branded or generic. The data show that 100% of them should do better on SUFIANCE, but let them be for now. Let's start with those who don't have a therapy. In a way, that makes a lot of sense, right? Now we're starting to see, I think, and hear more and more of prescribers doing the work now to switch patients.

We commented a little bit on this dynamic at Q2 earnings when we said, we're seeing a little bit longer time to fill, because clearly now with payer policies in place, which have been very favorable, very flexible, but nonetheless, when you're going to switch someone from a less expensive drug to a more expensive drug, it's going to be a little bit more back and forth. As you know, that back and forth takes time from when you get a first request to fill to when ultimately getting that filled. I think what makes us so excited here is there's probably still about 2,500 patients on KUVAN in the country, branded or generic. As we said, we expect all of those patients to do very well on SUFIANCE.

I'm not saying 100% of them are going to get switched, but a lot of the prescribers said they expect over time that the vast majority of them would get switched. It's going to take a little bit of time, but we know when that switch occurs, going from one once a day well-tolerated therapy to another, that just simply provides greater benefit. That reads strong motivation for switching, both in terms of patients wanting it and prescribers wanting it. Two, adherence, right? You've already proven yourself adherent on a drug that's once a day in oral, now you're going to go to another drug that's once a day oral, but delivers more benefit.