PTC Therapeutics Inc Global Agreement with Novartis for PTC518 HD Program Transcript

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

Question: Tiago Fauth - Wells Fargo Securities, LLC - Analyst : Great. Thank you so much for taking the call and congrats on the deal. Just two for me real quick. Just wondering what sort of data was available for the parties that were part of the process? Was it just the June 2024 update or something else since? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 02, 2024 / 1:30PM, PTCT.OQ - PTC Therapeutics Inc Global Agreement with Novartis for PTC518 HD Program And you did mention that Novartis is going to be involved in regulatory interactions upcoming. But I'm assuming the deal is not conditional in any way, shape or form on the outcome of that. Is that correct? Thank you.

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Hi. Good morning. Congrats on the great deal. And I think bigger picture, a testament to your team with now to [$1 billion] upfront cash deals with this platform in the last 13 months. So maybe just on that note, you said that you're going to use some of the cash to look at this platform in more detail with the success you've seen from Evrysdi as well as 518 now, how are you thinking about looking at potential other strategies with this platform?

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Thank you so much. And then obviously you guys are pros at meeting with the FDA, especially this year with all of the approvals and ongoing applications. But just given Novartis' willingness to work with you on this pathway, curious if they gave you any good insights or suggestions as it relates to the upcoming Type C meeting? Will they physically be in attendance with you at this meeting? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 02, 2024 / 1:30PM, PTCT.OQ - PTC Therapeutics Inc Global Agreement with Novartis for PTC518 HD Program

Question: Jeff Hung - Morgan Stanley - Analyst : Congratulations and thanks for taking my questions. How do the $1.9 billion in milestones split out between development, regulatory and sales? And then for the tiered double-digit royalties on ex-US net sales, how many tiers are there? And how are those tiers triggered? Like are they triggered by cumulative sales or do they reset on an annual basis? Thanks.

Question: Brian Abrahams - RBC Capital Markets - Analyst : Hey, guys. Good morning. Congrats on the deal. Thanks for taking my question. Can you talk a little bit more about like your expected next steps beyond PIVOT-HD? I guess what Novartis will be running when they take over development of the program. And then I guess along those lines, how will this development decisions get made in the future? You mentioned PTC still going to have an important role. Can you talk a little bit about, I guess, the types of committees that will be involved there and how future data disclosures will be handled as well? Thanks.

Question: Ellie Merle - UBS - Analyst : Hey, guys. Congratulations on the deal. I just curious in terms of the timing. I guess what's the latest -- excuse me, in terms of when you plan to meet with the FDA around accelerated approval or pathway and Huntington's -- I guess, did that meeting happened already? I guess just given the timing of the deal here, what's your latest in terms of like your expectations around the likelihood of an accelerated approval pathway with Huntington's as a surrogate. And then I have a follow up question.

Question: Ellie Merle - UBS - Analyst : Great. And just a follow-up to Brian's question, just if you can give us more detail around your conversations with Novartis. I guess, what the latest you're thinking in terms of what a Phase 3 design could look like. I mean, whether that's for confirmatory or for initial approval? Just any more details on those conversations and the Phase 3 thinking there. Thanks.

Question: Joel Beatty - Robert W. Baird & Co. Incorporated - Analyst : Thanks. Congrats on the deal. The first question is what do you hope to see in the PIVOT-HD readout coming in the first half of next year. And then second question is what are your plans for this additional capacity you've got with the $1 billion upfront today and then as well as the recent sale of the voucher.

Question: Joseph Thome - TD Cowen - Analyst : Hi there. Good morning. Congrats on the deal and thank you for taking my questions. Maybe first one, even going back to the prior management team, the company had indicated that it did want to kind of keep whole rights to PTC518 given the experience with the plan. Maybe can you talk a little bit about just what changed in the management thought process there? And then second, do you have any follow-on compounds for Huntington's that you're working on the splicing platform? Are you limited in what you can pursue there and does Novartis have access to these follow-on compounds if they exist as well? Thank you. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 02, 2024 / 1:30PM, PTCT.OQ - PTC Therapeutics Inc Global Agreement with Novartis for PTC518 HD Program

Question: Gena Wang - Barclays - Analyst : Thank you. Also add my congrats. That's a great deal. So I have two questions. One, is it fair to say that the base assumption for the Huntington program from Novartis side to moving forward is based on the Phase 3 functional approval path. And also, did the Novartis see like additional three more months data since last June, I think someone already asked these questions but just wanted to ask again. And then regarding the deal term, the royalty, double digit royalty, is it fair to assume like a low-teen to mid-20s range? Like what we usually see in other deals and regarding the profit sharing in the US [40:60]. Will you need to share also the development cost? For example, if moving forward post PIVOT-HD, if there is a Phase 3 study ongoing, were you need to share also 40% there?

Question: Sami Corwin - William Blair & Company, L.L.C. - Analyst : Hi there. Thanks for taking my questions and congrats on the deal. I guess what are your thoughts about the best-case scenario would be for the pivotal trial design? And do you think it'll take multiple interactions with FDA to have clarity on what that design might look like? And do you think it's possible that you would have a insight into what the trial design would look like before the data update in 2025? Thank you.

Question: Sami Corwin - William Blair & Company, L.L.C. - Analyst : Got you. And does the $1 billion upfront payment change your estimated timeline becoming cash flow breakeven at all?

Question: Paul Choi - Goldman Sachs & Co. LLC - Analyst : Hi. Thank you. Good morning. And let me add my congratulations on the deal nicely done. My first question is with regard to ex-US development and I think the European Clinical Trials Registry is currently only listing the 5 milligram and the 20 milligram doses. Can you maybe just comment on the status of the 10 milligram cohort? And what's going on with regard to international development? And my second question is for your plan to update on the PIVOT-HD trial next year, can you just confirm that the 20 mg dose cohort is fully enrolled and that you'll have both Huntington biomarker data and TMS clinical data endpoints when you report that update? Thank you very much for taking our questions.

Question: Danielle Brill - Raymond James Financial, Inc. - Analyst : Hey. Good morning. Thanks so much for the question and let me extend my congrats as well on a great deal here. Just a quick one, Matt, you said the deal is highly competitive in you're prepared to mark. So I'm curious if you could elaborate on how many parties were interested. Thank you.

Question: Tazeen Ahmad - BofA Securities, Inc. - Analyst : Hi. Good morning. Thanks for taking my question and also congrats from me on the deal terms. I was just curious, Matt, as to how you're thinking about timelines. You've talked about the specific reasons why you're excited about Novartis coming on board. And it seems like a lot of that is with regards to the potential for commercial opportunity. How are you thinking about the potential for narrow timelines? Let's say that you don't get an accelerated path to approval and you do have to do a traditional Phase 3 relative to the timelines you were thinking about if you were running the study by yourself versus now with Novartis involved, can you give us a sense on whether or not that timeline is now accelerated as well even for a traditional approval? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 02, 2024 / 1:30PM, PTCT.OQ - PTC Therapeutics Inc Global Agreement with Novartis for PTC518 HD Program And then secondly, I just want to clarify based on your PRV sale and based on the cash received from Novartis, do you expect to need to do any financings going forward? Thanks.