PTC Therapeutics Inc at Bank of America Global Healthcare Conference Transcript

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

Question: Tazeen Ahmad - BofA Securities - Analyst : So I've been starting off all the conversations with macro questions. I'll ask you in the order of most recently occurred macro events. Can you talk about the executive order that was announced yesterday to the extent that you can?

Question: Tazeen Ahmad - BofA Securities - Analyst : Okay. So just to clarify, for PKU, are you expecting to price this at a narrow range?

Question: Tazeen Ahmad - BofA Securities - Analyst : Okay. And then can you talk to us about tariffs in general?

Question: Tazeen Ahmad - BofA Securities - Analyst : Okay. And then lastly, on your interactions with FDA.

Question: Tazeen Ahmad - BofA Securities - Analyst : Okay. When we spoke on some a few weeks back, there was one thing that you had mentioned about said all these things about hasn't really changed with FDA. But in relation to AdComs, you had made a comment along the lines of potentially the folks who arrange for advisory committee meetings might have been somewhat impacted by changes. Is that something that you've been able to get color on?

Question: Tazeen Ahmad - BofA Securities - Analyst : Okay. So let's talk about Translarna as it relates to Europe. So it's a drug that doesn't exist but yet exists -- is what I'd like to say. So you continue to record sales, how are those sales occurring now?

Question: Tazeen Ahmad - BofA Securities - Analyst : I mean it could be higher.

Question: Tazeen Ahmad - BofA Securities - Analyst : Okay. So you talked about PKU, and that would have a large commercial contribution, both dollar-wise in terms of how it's reimbursed commercial payers. DMD on the other hand, does have a significant Medicaid component, right?

Question: Tazeen Ahmad - BofA Securities - Analyst : So then if approved, does that complicate the US launch as it relates to the first question we started off with on --

Question: Tazeen Ahmad - BofA Securities - Analyst : Okay. Got it. So then how should we be thinking about expectations you've talked about the strength of the Translarna data. We -- I go back with Translarna in the very beginning --

Question: Tazeen Ahmad - BofA Securities - Analyst : And so if this makes it this last attempt to get it approved, does get it approved, how should we be thinking about investments of resources from PTC into this launch?

Question: Tazeen Ahmad - BofA Securities - Analyst : And what about the comments that that Dr. McCarrey has made about continuing to focus on rare disease. Do you find that encouraging for Translarna's prospects as well.

Question: Tazeen Ahmad - BofA Securities - Analyst : Okay. So let's move on to the PKU launch, which is a much more traditional launch for the company. Can you just remind us on where you are with that?

Question: Tazeen Ahmad - BofA Securities - Analyst : Now can you talk to us about the competitive landscape as in like what do patients currently take and we'll talk about some potential advantages.

Question: Tazeen Ahmad - BofA Securities - Analyst : And why is that?

Question: Tazeen Ahmad - BofA Securities - Analyst : Yeah. So as you describe it, this seems sort of like a layup in terms of launch. So but nothing over right? So what is it that you're focusing in on? Are you trying to switch patients from the two drugs that you just mentioned initially or are you trying to get to those, let's say, 85% of patients that are not on any --

Question: Tazeen Ahmad - BofA Securities - Analyst : So most recently, you talked about just general ideas about pricing. You talked about pricing at a premium to Palynziq. So to me, that was a little bit on the new side relative to what you've been talking about historically. So I guess, what's changed that gives you the confidence that, that kind of price premium is justified and would be reimbursed?

Question: Tazeen Ahmad - BofA Securities - Analyst : So if you do the math, I mean you only need like low double-digit percent penetration into this population to be not that far away from $1 billion and so.

Question: Tazeen Ahmad - BofA Securities - Analyst : And how are you thinking about -- what is the IP estate for this as well?

Question: Tazeen Ahmad - BofA Securities - Analyst : And how has the population split US and ex US Is it equal?

Question: Tazeen Ahmad - BofA Securities - Analyst : Okay. Are you going to be ready to launch on day 1 after approval?

Question: Tazeen Ahmad - BofA Securities - Analyst : Okay. So maybe let's move on to PTC518, which which actually a lot of people say, if you're excited about PTC, this is the reason to be excited. So would you agree with that comment?

Question: Tazeen Ahmad - BofA Securities - Analyst : Can you maybe just talk to us about -- there are several companies that are trying to do treatments for Huntington's and it's kind of been a graveyard, it's a challenging disease to really drug. So can you talk to us about the data that you've presented so far? And the most recent data set, do you think that there was did the market not appreciate certain -- to a certain level, what you were going to show versus what they expected you to show? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 13, 2025 / 5:00PM, PTCT.OQ - PTC Therapeutics Inc at Bank of America Global Healthcare Conference

Question: Tazeen Ahmad - BofA Securities - Analyst : Yeah. You brought up Novartis that's your partner in this program. I guess given the timing of when that partnership was announced, some had thought that was based on maybe Novartis feeling a sense of confidence, if you will, on an accelerated path being a highly likely scenario. Novartis was -- where was that in the mix outside of them looking at the science and trying to understand the product. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 13, 2025 / 5:00PM, PTCT.OQ - PTC Therapeutics Inc at Bank of America Global Healthcare Conference

Question: Tazeen Ahmad - BofA Securities - Analyst : Okay. And so how do you just to wrap things up, think about the competitive landscape for HD? If you have to take a traditional path to approval versus accelerated, how does that change where this product could be relative to the competition?