PTC Therapeutics Inc Q4 2024 Earnings Call Transcript

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

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : I have 2. The first one is on PKU. So ultimately, the understanding out there is that diet liberalization is something that could impact patient treatment, both for naive patients as well as those on current other therapies. Do you have a sense of what the bar or the minimum that these patients would want to see in terms of liberalizing their diet to at least try sepiapterin?

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Appreciate that. We'll definitely be interested in that presentation next month. And then on Friedreich's ataxia, I think a lot of people aren't necessarily giving this credit yet. So I'm hoping, can you give us a sense of how you're thinking about the market opportunity? You laid out specifically the patient populations that you could target, but how should we be thinking about peak sales, anything around a cadence of launch or anything that would be helpful for us as we think about modeling that?

Question: Eric Joseph - JPMorgan - Analyst : Congrats on the quarter progress. Three questions, I'll try to work in quickly. First, what kind of visibility do you have on the Translarna review process in the U.S. Do you anticipate a formal PDUFA date in place ahead of a decision? Second, just as it relates to your top line guidance consistent where it was in January, I guess, how does that accommodate or factor in Translarna maintaining authorization in the EU? To some extent, do you think you should be thinking raising or trending kind of off the lower end of guidance? And then thirdly, as it relates to [Votoplam] and the PIVOT-HD update coming next quarter, I guess, how should investors be kind of framing their expectations when looking at measures of functional benefit given the slightly different mix in patients, including Stage 3 disease versus the initial 12 months readout last year?

Question: Eric Joseph - JPMorgan - Analyst : Great. One quick follow-up, if I could, on PIVOT-HD. I wonder whether there is the potential for longer follow-up from the set of interim -- set of patients reported in the interim last June. That is to say, upwards of 24 months follow-up, particularly when it comes to huntingtin decline in the CSF in those patients. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 27, 2025 / 9:30PM, PTCT.OQ - Q4 2024 PTC Therapeutics Inc Earnings Call

Question: Kelly Shi - Jefferies - Analyst : Congrats on the quarter. I also have a couple for the PIVOT-HD trial. Firstly, from the 12 months to 24 months, what kind of improvement do you expect on total motor score and UHDRS and maybe also the TFC, the total functional capacity? And also, can you help us on the -- regarding the Stage 2 and Stage 3 split for this pivotal trial? Is this relatively similar to the last interim update mid of last year?

Question: Tiago Fauth - Wells Fargo Securities, LLC - Analyst : I just have a follow-up on PKU. Kind of one of the pushbacks we generally get from investors is that the low treatment rate for those patients. So I'm curious if you were to break down the $1 billion potential that you see in the US, part of that is an assumption on premium pricing, which you've kind of made clear. But how much of that is also the assumption on potential conversion of existing patients on therapy versus bringing patients "back" to medical treatment. I'm curious how much is conversion, how much is expansion and how to think about that $1 billion opportunity.

Question: Brian Abrahams - RBC Capital Markets - Analyst : This is Joe on for Brian. On PKU, what is your sense on how the payers will potentially think about utilization management for those who are currently taking treatments and are looking to switch? And what about for those who are not on any treatments with prior experience and other therapies?

Question: Eliana Merle - UBS Equities - Analyst : Another couple on the PKU launch. I guess how should we think about the size of the target prescriber base? And is it centralized or dispersed? And in terms of the population, I know you mentioned this a little bit in terms of Tiago's question, like the longer-term. But in the near-term, as you approach a commercial launch, who's the population that you think could be the most rapid adopters? And I guess, how often do these patients see their physicians? And then just last one quickly. Just on the pricing, did I hear correctly you're talking about a potential premium pricing to Palynziq. Just want to clarify if I heard that correctly and your latest commentary on pricing potentially in PKU.

Question: Gena Wang - Barclays - Analyst : I have 2 questions. One is regarding vatiquinone Friedreich's ataxia. Did FDA confirm that there will be no AdCom? And my second question is regarding the PIVOT-HD data in second quarter. Since this -- based on your FDA feedback, this will be important data set to show the correlation between huntingtin lowering and the clinical measurement. So how would you define there is a correlation? I think, for example, you have 3 cohorts, placebo, 5 milligram, 10 milligram, do you need to see the dose response among all these 3 cohorts in terms of protein knockdown and also the correlation to the functional measurement. And I think you did mention 2 different important measurements based on the Stage 2 and Stage 3. And how would you look across different measurements, which will be the most important measurements for the FDA based on your feedback?

Question: Gena Wang - Barclays - Analyst : If I can may just ask a quick question regarding the protein lowering, did FDA care more about the CSF huntingtin protein lowering or blood huntingtin protein lowering?

Question: Joel Beatty - Robert W. Baird & Co., Inc. - Analyst : This is a follow-up to a question a couple of questions ago. For PKU, you mentioned there's a long list of patients at centers with a bolus of patients waiting. Could you help us think through the cadence of when those patients could start on therapy of sepiapterin and maybe what the rate-limiting factors are for that?

Question: Samantha Corwin - William Blair & Company - Analyst : I was pleasantly surprised to hear that you still have new patient starts on Translarna in Europe. And I guess I was curious if you expect that trend to continue into Q2 or how you're kind of expecting like what the current status of Translarna is in Europe? And then how are you thinking about the timing of additional meetings with FDA regarding Huntington's? And when do you expect to have alignment on a registrational path going forward there? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 27, 2025 / 9:30PM, PTCT.OQ - Q4 2024 PTC Therapeutics Inc Earnings Call

Question: Samantha Corwin - William Blair & Company - Analyst : And will Novartis be taking the lead on those regulatory interactions now? Or will it be a joint effort?

Question: Peyton Bohnsack - TD Cowen - Analyst : This is Peyton on for Joe, and congratulations on all the progress in 2024. I guess you mentioned looking into exploring business development opportunities in the prepared remarks. How likely is this to occur in 2025? And is this contingent on all 3 therapies being approved this year? And then building off that, how do you plan to balance that with the internal pipeline? And do you plan to bring anything else from the internal pipeline into the clinic this year? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 27, 2025 / 9:30PM, PTCT.OQ - Q4 2024 PTC Therapeutics Inc Earnings Call

Question: Khalil Fenina - Goldman Sachs - Analyst : This is Khalil calling in for Paul. I guess I'll ask about your capital position. So I see that you guys have a very strong cash position right now. And if you take out the asset impairment, it looks like you guys are close to breakeven. I guess the Translarna worries notwithstanding, I'm curious to hear what your capital deployment plans are for the remainder of the year.

Question: Tazeen Ahmad - BofA Global Research - Analyst : When we talk about splicing modulators in HD, can you maybe talk a little bit how your program could be differentiated from others that are also trying in HD? For example, Skyhawk has an early-stage program, I think it's 0515. And then I have a few questions on Translarna. So how should we be thinking about the cadence of the launch if approved in the U.S.? Would the launches of the PMOs from Sarepta be good markers of examples on how to think about the pace of uptake among patients? How easy will it be for you to find the patients, the nonsense mutation patients, here in the U.S.? And then lastly, on Translarna, how are you thinking about the competitive landscape with the introduction of gene therapy, which does seem to be having a good initial uptake?

Question: David Lebowitz - Citi - Analyst : In terms of your meeting with the FDA on Huntington's in December, did they provide a framework for which clinical endpoints they want to see and what they want to see from them in an analysis?

Question: David Lebowitz - Citi - Analyst : And then towards that end, did they want to see some of that data with respect to the prior update, or they just want to wait until after 2Q?