PTC Therapeutics Inc at TD Cowen Healthcare Conference Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Joe Thome - TD Securities LLC - Analyst : Perfect. Maybe we'll start with the commercial franchise and then dive into the pipeline. But the company did release revenue guidance of between $600 million to $800 million for fiscal year '25. Maybe can you go into a little bit of detail about what that assumes on the top end and the low end, kind of what factors you'd be looking for this year?

Question: Joe Thome - TD Securities LLC - Analyst : Perfect. And maybe can you touch a little bit about Emflaza? What are you seeing? Obviously, generics have been able to launch, but the brand has been pretty sticky, and that's what we're hearing from our KOLs as well. Kind of what are you seeing in the commercial setting?

Question: Joe Thome - TD Securities LLC - Analyst : Perfect. And maybe jumping over to Translarna. I guess what are your expectations for next steps in -- as it relates to Europe? It seems like physicians are trying to find mechanisms to keep patients on therapy that they are at least already on. Kind of what are your expectations for what you'll hear next and how that kind of feeds to the Street?

Question: Joe Thome - TD Securities LLC - Analyst : Perfect. And maybe on Translarna in the US, as you indicated, they accepted the filing. We don't have a specific PDUFA date, but can you kind of qualify your interactions with the FDA? Kind of what gives you confidence that the application that you submitted will hopefully eventually be successful?

Question: Joe Thome - TD Securities LLC - Analyst : Perfect. And maybe the last in-house revenue-generating commercial product. We do have the approval for the gene therapy for AADC. Obviously, we've seen gene therapy launches go a couple of different ways, but it seems like when there's a rare CNS condition and you have an efficacious gene therapy, they tend to go a little bit better. I guess, can you kind of tell us how this launch is going? And how can you make sure that it's more Zolgensma like than maybe some of these other ones that are --

Question: Joe Thome - TD Securities LLC - Analyst : Perfect. Maybe moving on to some of the pipeline opportunities. We'll start with sepiapterin in PKU. You have the EU opinion slated for the second quarter and then the July 29 PDUFA data this year. Maybe can you touch a little bit on your launch strategy? How can you emphasize some of the differentiated data that you've seen versus maybe what's seen with Kuvan?

Question: Joe Thome - TD Securities LLC - Analyst : And maybe can you talk a little bit about the commercial sales force efforts? Are you going to be able to sort of essentially completely use, obviously, the EU force that's been in place for Translarna and obviously, US with Emflaza, Will you have to build out any additional members?

Question: Joe Thome - TD Securities LLC - Analyst : Perfect. And we can always come back at some other questions for this, but obviously, a lot else to get through. Most recently, the FDA did accept the vatiquinone NDA for Friedreich's ataxia for priority review. Can you kind of just walk us through maybe what are the remaining questions for the application? I know the day 74 letter should be coming out at some point soon. Can you kind of walk us through what you'd be looking at that, if anything?

Question: Joe Thome - TD Securities LLC - Analyst : Perfect. And obviously, the application is going to be for both children and adults, I suppose. But as you indicated, SKYCLARYS isn't yet approved for the pediatric population. So kind of how are you going to tailor maybe your initial commercial approach to that? Will you initially target maybe those that treat more pediatric patients or go broadly? How are you thinking about capturing the best -- as best, as quickly as you can?

Question: Joe Thome - TD Securities LLC - Analyst : And we've heard from our physicians that it would make sense mechanistically to in adults, combine SKYCLARYS potentially with vatiquinone. I guess, do you feel the same way? And do you think that would be reimbursed given the unmet need?

Question: Joe Thome - TD Securities LLC - Analyst : And if you're successful with the FDA, do you think this would also be successful in Europe? Kind of what are the status of your interactions ex-US?

Question: Joe Thome - TD Securities LLC - Analyst : And maybe jumping over to PTC518, and we'll start maybe with the partnership first and then kind of dive into the individual data. But obviously, the partnership did a great job at kind of helping the balance sheet and kind of funding for going forward. Maybe what made them the right partner and why was now maybe the right time to partner 518?

Question: Joe Thome - TD Securities LLC - Analyst : Perfect. And in Huntington's readouts, there's a lot to look at. So obviously, you have the Huntington protein reduction, you have NFL, you have TFC, TMS, UHDRS. There's a lot of different things going on here. I guess maybe can you hit the highlights of what you've seen so far with 518 and maybe on the backdrop of that, what is most important to look at?

Question: Joe Thome - TD Securities LLC - Analyst : And maybe we're going to see some additional data from the trial in the second quarter here with 100 additional patients. Maybe can you set the stage for what investor expectations should kind of roughly be. So that we don't get too far ahead of ourselves, but also obviously, you want to see efficacy. Should stats versus placebo be expected? Or would that be great upside? What do you need to show, I guess?

Question: Joe Thome - TD Securities LLC - Analyst : Yes. That was my next question is if you could put that a little bit in the context with what have you talked to the FDA about in reference to accelerated approval here? And are they clear on kind of how you can correlate the biomarker to function? Or is it going to take some conversations?

Question: Joe Thome - TD Securities LLC - Analyst : Perfect. And maybe as it relates to the Novartis partnership, obviously, they will take on the expense for running the Phase III program as part of the deal. What will be the next step after this Q2 readout? How quickly do you think you could advance to a Phase III? And how involved is PTC in sort of that process?

Question: Joe Thome - TD Securities LLC - Analyst : Perfect. And obviously, you've been very successful with the splicing platform with Evrysdi and now obviously, PTC518. What's next for the splicing platform?

Question: Joe Thome - TD Securities LLC - Analyst : Perfect. And you indicated you now have over $2 billion in cash after the Novartis upfront. How far does that get you in terms of your current operating plan? And kind of what's -- what are kind of the pushes and pulls there?

Question: Joe Thome - TD Securities LLC - Analyst : Perfect. Awesome. With that, we are out of time. So great execution over the past year, and we look forward to more. Thank you for joining us.