PTC Therapeutics Inc at JPMorgan Healthcare Conference Transcript

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

Question: Eric Joseph - JPMorgan - Analyst : We have some time for questions. And so if you have any, just raise your hand, we'll get a mic over to you. But just picking up on sepiapterin and PKU with the approval and approaching approval decision and approaching launch. You commented on your pricing expectation here, anticipating payer support for a price point, a premium to Palynziq. Can you just talk a little bit about sort of the -- just the feedback from payers, a little bit sort of the value proposition that you're taking to payers that gives you confidence in being able to support that premium price point they anticipate?

Question: Eric Joseph - JPMorgan - Analyst : And once in the market, I guess, how to think about where new starts on drug will sort of originate? Is there sort of a low-hanging fruit opportunity that you expect to target first?

Question: Eric Joseph - JPMorgan - Analyst : Question over here? Unidentified Participant Yeah. I just wanted to ask about your thoughts on the accelerated approval using HTT as a surrogate end point and how you're thinking about that relative to the FDA's views on, for example, uniQure's therapy? Thank you.

Question: Eric Joseph - JPMorgan - Analyst : I guess in the analysis, you're proposing to the agency that would help support the relationship between mutant Huntington lowering and clinical scales. I guess, are there certain particular clinical scales at the agency wants you to focus on demonstrated improvement and relate those to mutant Huntington lowering? Just maybe a little bit of a sense of the analysis that you're going in with leading up to the additional 12-month readout later or this half?

Question: Eric Joseph - JPMorgan - Analyst : And you had the opportunity to look at Huntington lowering beyond 12 months in PIVOT-HD trial? You'll have some cohort of patients people. So I guess, there's two kind of buckets of longitude and the follow-up that you'll get, right?

Question: Eric Joseph - JPMorgan - Analyst : So just coming back to PKU, I guess -- sorry, you're not alone in the space, right? Obviously, there's a well-known competitor that you alluded to earlier that also plans to sort of seize the growth opportunity through label expansion initiatives and so forth. I mean, does -- how receptive are clinicians today to potentially seeing Palynziq move into the younger adolescent population? Does that kind of present as a risk factor to your launch?

Question: Eric Joseph - JPMorgan - Analyst : And where would you say sort of sepiapterin awareness is across the PKU treating community?

Question: Eric Joseph - JPMorgan - Analyst : And maybe just -- I think we have time for one or two more questions. One on vatiquinone in Friedreich's ataxia. Just the scope of the approval that is kind of on the table with the recent filing, I believe -- it's -- would support accelerated approval, if I have that correct, I guess? And if that's the case, how to think about the confirmatory study would look like with that agent?

Question: Eric Joseph - JPMorgan - Analyst : So with two -- potentially two product launches in the second half, I guess, how to think about the sizing of the organization on the sales side, MSL sizing, and so forth to support both launches? Obviously, you have a presence currently in DMD. I guess how much expansion is needed to support these two upcoming launches?