PTC Therapeutics Inc Q3 2023 Earnings Call Transcript

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

Question: Kristen Brianne Kluska - Cantor Fitzgerald & Co., Research Division - Analyst : First, just wanted to congratulate you 4on the Royalty Pharma deal. And I had a question about Translarna. Can you talk about why you're only pursuing re-examination of the conditional authorization? I guess, what advantages do you see going this way versus both? And assuming that you do get the green light, how are things going to be moving forward? Will it be kind of similar, like what we've seen over the last 8 years when they've given you the green light, or will you be required to conduct any type of work in the background?

Question: Kristen Brianne Kluska - Cantor Fitzgerald & Co., Research Division - Analyst : And just second part of that, I would imagine since you had the announcement that the community was probably pretty upset, especially the ones that have been on the therapy long-term. So, wondering if there's anything you can collect from them, anecdotes, stories, et cetera, and if they can be of any help ahead of this upcoming meeting and decision.

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : On the sepiapterin NDA filing timeline, I'm just curious, what is the latter end of your guidance 3Q '24? I'm curious, can you confirm whether the study has started? And it just seems like a sort of extreme upper bound of this timeline if this study is all that's needed. And then on the AADC, could you just put a little more detail on why FDA thinks the comparability data is still not sufficient? And then what are you sort of hoping or what will be discussed at the pre-BLA meeting in December?