PTC Therapeutics Inc at Barclays Global Healthcare Conference Transcript

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

Question: Gena Wang - Barclays - Analyst : Thank you. I wanted to make a comment, the Huntington program so far, I think we haven't seen any one can surpass that deal term. It's so favorable to PTCT. Really congratulations on that deal. And then I know this is one of very important pipeline asset, and that's why maybe Novartis are willing to pay so much money for the programs. So with the midyear update, I wanted to maybe give a little bit layout exactly -- I know you've mentioned in the past about 150 patients. Maybe lay out exactly where you will be presenting the data and what should we expect? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 11, 2025 / 4:30PM, PTCT.OQ - PTC Therapeutics Inc at Barclays Global Healthcare Conference

Question: Gena Wang - Barclays - Analyst : So I did a quick calculation. If we -- and you did mention in the past, like it will be evenly split between Stage II and Stage III and each will be three arms. So when we calculate roughly, each arm is about 25 patients.

Question: Gena Wang - Barclays - Analyst : 10 milligram, yes. So now when we look at the last update, I know each arm is about 10 patients, and you did show dose response and the -- certainly in the blood lowering and then also the magnitude like a 43% in the CSF. And then -- and you do see the longer follow-up, you do see the further improvement in terms of protein lowering.

Question: Gena Wang - Barclays - Analyst : And then should we -- and then of course, you do -- you also did share a lot of the functional measurement, right? Some has a more clear trend, some a little bit noisy because given a small number of patients. So now we have, say, more than doubling of each arm of the patient, like should we expect the similar trend largely persist with this more comprehensive data update?

Question: Gena Wang - Barclays - Analyst : So I know you will not be able to comment on the actual data, but in terms of pattern difference, what kind of a functional measurement you think it could be different between Stage II and Stage III?

Question: Gena Wang - Barclays - Analyst : So what about the other functional measurements? Any major differences between the two classes?

Question: Gena Wang - Barclays - Analyst : Okay. Very good. And then you likely will share either a separate or group.

Question: Gena Wang - Barclays - Analyst : Okay. Good. And do you see all these data real time?

Question: Gena Wang - Barclays - Analyst : Okay. Does any clinical team in your company see the data, blinded data?

Question: Gena Wang - Barclays - Analyst : So the same -- the whole company --

Question: Gena Wang - Barclays - Analyst : Do not see any data?

Question: Gena Wang - Barclays - Analyst : Okay. Good. And then unblinding and then do the analysis, like how long would that take?

Question: Gena Wang - Barclays - Analyst : Okay. Because we are almost at the end of 1Q, so you only have few more months.

Question: Gena Wang - Barclays - Analyst : Okay. Good. Maybe the neurofilament as a biomarker, how important that is?

Question: Gena Wang - Barclays - Analyst : With the data update, will these two also be presented?

Question: Gena Wang - Barclays - Analyst : And the FDA communication, like what kind of data do you think that will satisfy their comments on, I forgot the exact --

Question: Gena Wang - Barclays - Analyst : Yes, associations between the Huntington protein lowering and the function.

Question: Gena Wang - Barclays - Analyst : So do you think that -- I totally agree with you. The minimum one, you have to show dose response in terms of Huntington protein lowering, right, and then ideally both in the blood and the CSF. So do you think the agency also will be looking for the correlation? I know they use the word association, but would they also wanted to see the dose response in terms of a functional measurement. And which are the -- I'm pretty sure you will share all the functional measurement and which one you think most importantly likely would they care about like say, in the future, if you do the confirmatory study, that could be the primary endpoint. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 11, 2025 / 4:30PM, PTCT.OQ - PTC Therapeutics Inc at Barclays Global Healthcare Conference

Question: Gena Wang - Barclays - Analyst : So for the 2Q update, let's say, three scenarios, maybe even more than three, some gray area. So one is a very clear association. Like what would you define as a clear data that you think FDA will be happy or have an accelerated approval path there?

Question: Gena Wang - Barclays - Analyst : I see. So you think the last year data that should be sufficient?

Question: Gena Wang - Barclays - Analyst : So when you talk to the FDA in December, and did they say if you can repeat this data, you have a path forward? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 11, 2025 / 4:30PM, PTCT.OQ - PTC Therapeutics Inc at Barclays Global Healthcare Conference

Question: Gena Wang - Barclays - Analyst : Okay. And is it fair to say 10 milligram will be the going-forward dose if we saw similar data sets?

Question: Gena Wang - Barclays - Analyst : So wouldn't that be likely would benefit from accelerated approval because functional data would take us forever, right?

Question: Gena Wang - Barclays - Analyst : Okay. Great. Okay. Now switch gears. You still have tons of other pipeline assets. We have a few more minutes. I wanted to ask about the vatiquinone in the Friedreich's ataxia. So I think I was informed that you guys will not have the 74 letter. So that's already FDA community with you guys already?

Question: Gena Wang - Barclays - Analyst : Okay. So the mid-cycle review basically.

Question: Gena Wang - Barclays - Analyst : And then I've given my rough calculation, that should be April time frame.

Question: Gena Wang - Barclays - Analyst : Right. Okay. Good. And then -- so at what point when you don't hear from FDA, hey, you will not have -- they never talk about AdCom, that you know that, okay, you will not have AdCom.

Question: Gena Wang - Barclays - Analyst : So do you think that you have a better chance with AdCom or without AdCom?

Question: Gena Wang - Barclays - Analyst : Maybe based on your prior experience, at what point you will start to discuss with the FDA about the label?

Question: Gena Wang - Barclays - Analyst : Okay. Good. We still have a few more minutes. I wanted to discuss about sepiapterin PKU. So maybe launch strategy and then also maybe the thoughts on the pricing and then also the patient population, where you think it would be the low-hanging fruit you can start?

Question: Gena Wang - Barclays - Analyst : Okay. I'll ask the last question to Pierre regarding the revenue guidance and how much assumption for the DME franchise versus sepiapterin?

Question: Gena Wang - Barclays - Analyst : Should we consider like a real revenue contribution will start 4Q for sepiapterin?

Question: Gena Wang - Barclays - Analyst : Great. We are running a little bit over time, but we have lots of great discussions, and then we look forward to the 2Q data update.

Question: Gena Wang - Barclays - Analyst : Thank you, guys. Thank you.