PTC Therapeutics Inc at UBS Global Healthcare Conference Transcript

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

Question: Ellie Merle - UBS - Analyst : Absolutely, and congratulations.

Question: Ellie Merle - UBS - Analyst : Maybe just in the near term, how do we think about the opportunity? How many patients are currently diagnosed?

Question: Ellie Merle - UBS - Analyst : Yeah. Absolutely. What's the latest going rate for PRV?

Question: Ellie Merle - UBS - Analyst : Helpful context.

Question: Ellie Merle - UBS - Analyst : Absolutely. Well, taking a step back, you have a lot of programs, both commercial and hopefully soon to be commercial. Can you provide an overview across the portfolio?

Question: Ellie Merle - UBS - Analyst : Yeah, absolutely. It's a huge year ahead for you guys, exciting. Maybe starting with PKU and the commercial opportunity, assuming an approval and potential launch in the second half of the year, how should we think about the cadence of uptake? I mean there's all the patients who tried [Kuvan] that didn't respond. Could there be a bolus?

Question: Ellie Merle - UBS - Analyst : Absolutely. And I know you've spoken about it could potentially be $1 billion in peak sales even just in the US But can we break that down into segments like even if you only penetrated, say, the patients who failed Kuvan, like what proportion would that make up? And then if you have, say, the switches from Kuvan and then probably the much larger is how do you bring all the people into care that are not currently actively managed?

Question: Ellie Merle - UBS - Analyst : Yeah, absolutely. And in terms of competition from Palynziq, and I understand these are very different profiles and probably patient populations as well. But as Palynziq potentially moving into the younger ages in the coming years, how do you think that affects your opportunity? And maybe just broadly, like -- I mean, you kind of alluded to it, but how you're thinking about like the opportunity in younger versus older?

Question: Ellie Merle - UBS - Analyst : Makes sense. And maybe pivoting to Huntington's on the clinical side of things --

Question: Ellie Merle - UBS - Analyst : Okay. Yeah. which is mindful that you are soon meeting with the FDA and could potentially establish a pathway to have a biomarker for accelerated approval, which would be massive for the states. What are the reasons why the FDA might accept mutant Huntington's and your level of confidence in the ability to have a biomarker? Because here, we know it's connected to the disease, but also we understand that there's wild-type huntingtin's protein as well that's being lowered.

Question: Ellie Merle - UBS - Analyst : Absolutely. And just on the safety front, how do you think about the maybe long-term implications of lowering the wild-type protein as well?

Question: Ellie Merle - UBS - Analyst : That's exciting. When will we learn about what you hear from the FDA? 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 13, 2024 / 10:45PM, PTCT.OQ - PTC Therapeutics Inc at UBS Global Healthcare Conference

Question: Ellie Merle - UBS - Analyst : And in terms of the update next year in PIVOT-HD, but not pivoting away from HD in the conversation right now, sorry. What should we be looking for there in terms of validating mutant Huntington as a biomarker? Because you mentioned like there's a potential scenario where the FDA says, okay, potentially, we use this as a biomarker, but maybe we want to see more data correlating it with these clinical outcomes. So what do we look for in that data update?

Question: Ellie Merle - UBS - Analyst : Makes sense. And I could ask many more questions on that, but we'll move to Friedreich's ataxia. What drives your confidence in approval?

Question: Ellie Merle - UBS - Analyst : And ahead of the filing, can you share some color from the recent FDA meetings?

Question: Ellie Merle - UBS - Analyst : And how do you think about the commercial opportunity, particularly in the context of the competitive landscape?

Question: Ellie Merle - UBS - Analyst : Makes sense. And heading into your ALS data, how are you thinking about that? 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 13, 2024 / 10:45PM, PTCT.OQ - PTC Therapeutics Inc at UBS Global Healthcare Conference

Question: Ellie Merle - UBS - Analyst : I got it. Maybe just coming off the back of earnings, you alluded to the commercial performance across the board was stronger than I think a lot of us anticipated. Translarna and Emflaza for similar or different reasons, I guess, are kind of challenging to model. Can you walk us through how to think about this cash flow stream from here?

Question: Ellie Merle - UBS - Analyst : Great. Well, I think we're out of time, but thank you so much for joining us.