PTC Therapeutics Inc Q2 2024 Earnings Call Transcript

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

Question: Kluska Kristen - Cantor Fitzgerald - Analyst : Hi, everyone. Good afternoon. Congrats on a great quarter and thanks for taking my question. The first one I had was on Huntington's disease. Obviously, looking at the field, we've seen a lot of data recently. But one thing that stood out is your approach seems to be one of the only ones where you're not seeing an increase in NFL through the therapy. Obviously, it's a prognostic biomarker of neurodegeneration, but other drugs have been shown to make it worse. So a question we've been getting is do we have a sense of how dangerous it is when NFL increases via drug targeting? And then second, is this a really important consideration for physicians on safety?

Question: Kluska Kristen - Cantor Fitzgerald - Analyst : Thanks for that, Matt. And then if I may ask a second question just on PKU. I think when we think about rare disease launches, the uptake curve is always different depending on this disease. So how would you think about this one for maybe, call it, the first two years? You already have a really good sense of where the patients are that you've identified that either don't respond or don't take standard of care. Thank you again.

Question: Kluska Kristen - Cantor Fitzgerald - Analyst : Thanks, everyone.

Question: Eric Joseph - JPMorgan - Analyst : Thank you and good evening. A couple of questions from us. First, on top-line guidance. I'm wondering if you can unpack that a little bit for what's anticipated in terms of net product sales marketed by PTC. Secondly, on PTC518 in Huntington's, wondering if you could give us a sense of when do you hope to have a meeting with FDA for a Type B or Type C meeting on a potential registration path. And also, wondering how you think about the merits of breakthrough therapy designation for this indication and whether that's something you intend to pursue with the compound. And then finally, on vatiquinone, what's left to get over the line for that submission in Friedreich ataxia? I believe data from the open-label extension portion of MOVE-FA was part of that view. Have you reviewed additional follow-up from that study? Is that something you plan to share with the Street? Thanks very much.

Question: Eric Joseph - JPMorgan - Analyst : Okay, great. Thanks for taking the questions.

Question: Kelly Shi - Jefferies - Analyst : Hi. Thank you for taking my questions. So curious on the regulatory front of Translarna in Europe. Could you share what's the most current status of Translarna and what are the timeline of next steps if you have a new update -- newer update after last call? And also in the US, any other color you would be able to share on the discussion with the regulatory agency after the NDA submission to FDA? And how are you thinking about the risk of US approval?

Question: Kelly Shi - Jefferies - Analyst : Thank you very much.

Question: Brooke Schuster - William Blair & Company - Analyst : Hi. This is Brooke Schuster on for Sami. We were wondering if you could provide some more color you expect for the upcoming ALS trial readout, like the bar for efficiency, and if you expect this readout to be at a medical conference or a company event.

Question: Joseph Thome - TD Cowen - Analyst : Hi there. Good afternoon. Congrats on the progress and thank you for taking our questions. Maybe the first one, just a follow-up on the ALS trial. I guess do you believe there's been any change in what the FDA wants to see for a pivotal package for ALS given the experience with the Amylyx compound? And then maybe second, on Huntington's, I know we're waiting on more data in the first half of next year, the full data set from PIVOT. Is there anything in that data set that you'd like to see before launching a Phase 3?

Question: Joseph Thome - TD Cowen - Analyst : Perfect. Thank you very much.

Question: Gena Wang - Barclays - Analyst : Thank you. I have two questions. First, you will have several launches underway. Should we see a meaningful increase in SG&A in 2025 and beyond? And second question is regarding the vatiquinone in Friedreich ataxia. So the NDA in late 2024 and regarding the natural history data, when do you plan to share that data with investors?

Question: David Lebowitz - Citi - Analyst : Thank you very much for taking my question. Given the operating spend, we'll have to start accounting for potential launches of additional products, namely PKU. Could you run us through what your assumptions would be and expectations, especially given those the convert that's due next year?

Question: David Lebowitz - Citi - Analyst : Thank you.