PTC Therapeutics, Inc. 12th Annual Cantor Fitzgerald Global Healthcare Conference

Yeah, absolutely. Look, I'll say that 2026 has been a great year so far for PTC. We're continuing to build towards being that differentiated rare disease company that we've been setting out to do. SEPHIENCE launch is going incredibly well, and we see no evidence of slowing down and remain confident in meeting our objectives of this being a $2 billion-plus opportunity, and a product that this year will get us, we believe, to the cash flow breakeven milestone and set us up for future profitability. We also are continuing to execute on all fronts across the company, continuing to succeed from a commercial standpoint, remain with a strong balance sheet of over $2 billion in cash.

A number of exciting programs coming through on the R&D front, including our recent planned acquisition of the gene therapy for Fabry, Huntington's disease program progressing well, as well a number of our inflammation programs. So a really exciting time for us as we continue to build towards future success.

There's several factors. First of all, it's a great therapy. It's a highly differentiated oral therapy with a dual mechanism of action that allows it to provide benefits of phenylalanine-lowering, diet liberalization, CNS benefits, a whole host of benefits for the full spectrum of PKU patients. While there are previous approved therapies, the vast majority of patients still needed such a safe, well-tolerated, and efficacious therapy. We saw this opportunity as quite large. Not only in terms of being able to provide a therapy for those who currently on the oral therapy of sapropterin, but those who are therapy naive, those who've tried and failed the oral therapy, those different severity of disease in the full age spectrum. We saw this as the ability to really deliver meaningful therapy to individuals with PKU.

In addition, several of the important pillars for commercial success were already in place. There's newborn screening, there's centers of excellence, there's a well-aggregated patient community, and we have an experienced global customer-facing team that can deliver rare disease therapies to patients. I think when you have a great therapy, a significant unmet need, an identified, well-aggregated population, and a team that knows how to deliver, that really sets you up for success. What we're seeing is just that, an incredibly strong start. You've heard me say time and time again, the theme of the launch thus far has been breadth. Breadth in terms of accessing all the patient segments, including adults who are remote to care and therapy naive, those on existing therapies, the very young newborns up to. We've had patients up to age 80.

Really the full spectrum of patients, and we really see continued growth, sustained growth in the U.S., accelerating growth outside the U.S. as we continue to bring more countries on board. We've pointed out that this really was a global launch where we were able to launch in the U.S., launch in Europe, launch in Japan, all within several months time, and now we're going to go through the work of getting approvals, access, and reimbursement in regions throughout the world as we continue to grow the global business.

Yeah. I never heard of a launch that peaks in year 1 or that all 17,000 patients show up at the clinic and get a drug day 1. That would be a new one, and we didn't expect to see that here. What we've seen is absolutely acceleration in the beginning because there's such a significant pent-up unmet need. What we heard somewhere around November of 2025 in the U.S., a lot of the centers start to say, "Look, we're just going to start seeing patients as they come in every 6 months or every 3 months for their visits to start putting them on therapy." That's the sustained cadence we've seen really since the latter part of 2025 into 2026 and what we expect going forward. Look, there's 17,000 patients in the U.S. We've gotten over 2,000 start forms.