PTC Therapeutics Inc Q3 2024 Earnings Call Transcript

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

Question: Rick Bienkowski - Cantor Fitzgerald - Analyst : Hi. This is Rick on for Kristen. Thanks for taking our question. We got two for you. So first of all, thanks for the update on the Type C meeting in Huntingtin's. Do you have a sense for when you could update the community on any clarity that might come out of the meeting? Is there any chance that we'll learn more about a potential path forward here? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 07, 2024 / 9:30PM, PTCT.OQ - Q3 2024 PTC Therapeutics Inc Earnings Call

Question: Rick Bienkowski - Cantor Fitzgerald - Analyst : Excellent. And maybe just one more if we have time. We've seen a lot of regulatory flexibility in the rare disease space recently, so given the FDA resubmission for Translarna, can you talk about how you think the FDA might sort of weigh the STRIDE registry data and Study 041 that were submitted and just kind of looking at this new evidence given sort of how things have been going in terms of regulatory flexibility? Thanks.

Question: Eric Joseph - JPMorgan - Analyst : Good evening. Thanks for taking the questions. I have one on the CardinALS trial. We noticed recently that you updated the or changed modified the primary endpoint to use a participant ranked combined assessment of ALSFRS and also survival. Can you talk about what some of the motivations were behind changing to that endpoint and what that impact might be from a statistical powering perspective? And I guess I think you've also now over enrolled the study. Was that meant to accommodate the change in endpoint or would you in fact now be sort of slightly overpowered versus prior expectations? Thank you.

Question: Kelly Shi - Jefferies - Analyst : Thank you for taking my questions. Given the revenue bid for this quarter, could you help us to set up an expectation for Q4, maybe also into 2025, what would be the dynamic DMD franchise also versus relatives from Evrysdi? Thank you.

Question: Michael Riad - Morgan Stanley - Analyst : Hi. This is Michael Riad on for Jeff Hung. Thank you for taking our question and congrats on all the progress and all the programs. Ahead of the CardinALS top line in 4Q, do you plan to share like baseline characteristics, maybe like time since diagnosis or can you comment on maybe what percent of patients are going into the OLE?

Question: Michael Riad - Morgan Stanley - Analyst : Okay, thank you. And then maybe for the long-term FA extension studies of vatiquinone. Why do you think you start to see the meaningful separation more in the extension studies? Was it like a rapid impact on mFARS expected, or could you perhaps like add some more color on how like vatiquinone is like influencing the trajectory there?

Question: Brian Abrahams - RBC Capital Markets - Analyst : Hey, good evening. Thanks for taking my questions. I guess one on sepiapterin and one on Translarna. Just for sepiapterin, I'm curious if you could maybe talk a little bit more about the reactions from providers, patients and payers to the Lancet data and in particular maybe elaborating on some of the payer research and communication that's given you confidence in the potential for the premium pricing. And then on Translarna, just wondering if you could have any sense of the potential timelines for the next review steps there, both in the US and Europe and whether you think we might hear some updates in 2025. Thanks. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 07, 2024 / 9:30PM, PTCT.OQ - Q3 2024 PTC Therapeutics Inc Earnings Call

Question: Ellie Merle - UBS - Analyst : Hey, guys. Thanks for taking the question. Can you talk a little bit more about the potential path forward and potential range of outcomes in terms of the feedback that you could specifically get from this Type C meeting? And if the FDA is not supportive of the use of Huntingtin lowering as a surrogate, I guess, what would the potential range of outcomes from there be in terms of what the clinical endpoint study and path forward for registration could look like? And I guess in that scenario, where FDA is not supportive of the use of a surrogate, how are you thinking about your strategy around whether you would run a full Phase 3 on your own or look for a potential partner? Thanks.

Question: Ellie Merle - UBS - Analyst : Great, thanks. And just a quick follow-up. I think you mentioned that you had requested two Type C meetings with the FDA, but that the FDA asked to combine the two into a single meeting. Maybe this is just logistical, but can you share maybe any context around this or maybe what this might suggest? Yeah, thanks.

Question: Joel Beatty - Baird - Analyst : Thanks. For Translarna, what percent of the current ex-US sales would not be affected by any potential European Commission decision? And also how long could those sales potentially last into the future?

Question: Joel Beatty - Baird - Analyst : And how long would exclusivity be in those other regions?

Question: Joel Beatty - Baird - Analyst : And then one last question for Friedreich ataxia in the US. Do you expect AdCom? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 07, 2024 / 9:30PM, PTCT.OQ - Q3 2024 PTC Therapeutics Inc Earnings Call

Question: Sami Corwin - William Blair - Analyst : Hey, there. Congrats on the quarter and thanks for taking my questions. As you look ahead for the launch of sepiapterin, are you envisioning specific patients to be early adopters and what are your assumptions in terms of the percentage of patients that may go on the drug, who would be classical. And based on your conversations with payers, have they kind of disclosed that many of their covered patients would have to step through generic Kuvan? And then I have a follow-up.

Question: Sami Corwin - William Blair - Analyst : Great. Thank you. And I think the team has pointed out the $1 billion opportunity in PKU before and that was with the assumption of modest penetration. So I guess what are your internal assumptions for penetration to reach that $1 billion opportunity in the US?

Question: Peyton Bohnsack - TD Cowen - Analyst : Hi. This is Peyton on for Joe and congrats on another strong quarter and thanks for taking our questions. I guess on the vatiquinone application for December, what's outstanding for the submission and then I know the FDA requested certain stats natural history when you were designing the analysis. Have you sent that to the FDA? Have they reviewed it and given any feedback? And then I've got a follow-up. Thanks.

Question: Peyton Bohnsack - TD Cowen - Analyst : Oh, I was asking whether or not after the readout came in October, the FDA has reviewed that or you've sent that to the FDA and if they've given any feedback on it. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 07, 2024 / 9:30PM, PTCT.OQ - Q3 2024 PTC Therapeutics Inc Earnings Call

Question: Peyton Bohnsack - TD Cowen - Analyst : Great. That makes a lot of sense. And then, I guess really quickly pivoting to Upstaza. Have all the necessary inspections been done for the manufacturing facility and then assuming approval next week, how quickly could you launch a therapy and how is patient identification going on?

Question: Paul Choi - Goldman Sachs - Analyst : Hi. Good afternoon and congratulations on the quarter. Thank you for taking our questions. I want to maybe just follow up on with regard to vatiquinone payer discussions here. Can you maybe just comment on what payer feedback has been and just sort of how you may be potentially sort of thinking about pricing relative to Skyclarys here for the adult population. And secondly the cash position remains very healthy here. And Matt, I just wanted to sort of ask what your appetite is currently for potential external business development versus funding the upcoming multiple launches here in '25. Thank you very much.

Question: Gena Wang - Barclays - Analyst : Thank you. I have a few quick questions. The first one regarding sepiapterin, PDUFA date July 29, 2025, just want to confirm that FDA confirmed there will be no ad account. Second question is regarding the Translarna, you mentioned that right now the 67 days in EU. So can you walk us through like say after 67 days, if the decision is unfavorable, what will be the next steps and how long would that take the process and then when we really will see the actual impact on the revenue in Europe and related questions or maybe unrelated Brazil, I know you are under independent assessment there. They have an independent approval process back in April. Any update there regarding the renewal process there? Lastly, very quickly regarding AADC next week you will get approval hopefully keep our fingers crossed. How ready are you regarding the manufacturing? How many doses you already stocked? And how quick you think you can get ready to how many patients you already identified in the US? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 07, 2024 / 9:30PM, PTCT.OQ - Q3 2024 PTC Therapeutics Inc Earnings Call

Question: Danielle Brill - Raymond James - Analyst : Great. Thank you so much for the question. I just have a quick clarification from an earlier question. I'm curious why the Translarna NDA submission is technically a resubmission under protest. Why wouldn't it be a new NDA since you now have new data to review from Study 041. Thanks so much. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 07, 2024 / 9:30PM, PTCT.OQ - Q3 2024 PTC Therapeutics Inc Earnings Call

Question: Joseph Schwartz - Leerink Partners - Analyst : Hi. Thanks so much. I have a question about vatiquinone and one on utreloxastat. So in FA, we've heard that while some patients think that they've benefited, more patients seem to not be blown away by Skyclarys' efficacy. So we're wondering how that might influence how patients might feel about vatiquinone, which is following its footsteps and works further downstream. How do you think this market is evolving now in terms of patient demand and retention for new treatment options as well as their potential to switch or try a combo with vatiquinone?

Question: Joseph Schwartz - Leerink Partners - Analyst : Okay. Thank you. And then for targeting ferroptosis in ALS, can you give us some context for what you're trying to achieve mechanistically? Is there a particular threshold you're looking to meet based on any preclinical or natural history data that supports your therapeutic hypothesis? And have you done anything in the CardinALS study to select patients that might best respond? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 07, 2024 / 9:30PM, PTCT.OQ - Q3 2024 PTC Therapeutics Inc Earnings Call