PTC Therapeutics Inc at Citi Global Healthcare Conference Transcript

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

Question: David Lebowitz - Citi - Analyst : On that. There's been a lot of news lately. We had some questions put together, and I have to reorder everything, given what's occurred. Let me start with 518 and the partnership. Could you, I guess, run us to the impetus of seeking the partnership now at this stage post Phase II, you might be looking to try to submit accelerated, but also there's the confirmatory trial going on what made you do it now and run us to the process that brought you to Novartis?

Question: David Lebowitz - Citi - Analyst : And going back about a year ago, there was -- people who were looking at cash and how that was moving forward, actually 1.5 years ago, and there was restructured a couple of restructurings that occurred and the cash situation is very different now after this partnership. How has that changed just from a strategic level, things for PTC going forward?

Question: David Lebowitz - Citi - Analyst : Now with respect to the -- what's happening with 518 going forward, certainly having meetings with the FDA to decide what the current steps from a regulatory perspective are. And there's the pivotal trial or confirmatory trial underway. What do we expect to learn about -- when could we expect to learn more about what the actual next steps are relative to the program?

Question: David Lebowitz - Citi - Analyst : Now given certainly tominersen and drugs in the past that have gone through the process, previously, the FDA has pushed back on the concept of accelerated approval obviously, this data set is different. How would you compare and contrast what the FDA has done in the past? And what's different about this particular therapy?

Question: David Lebowitz - Citi - Analyst : Of the downstream milestone payments, is there one associated with the full data coming up from the Phase II trial? Is there potentially one associated with if a determination is made at accelerated approval. And go forward, the application, the submission were obviously -- certainly, it will be one based on the approval itself. But --

Question: David Lebowitz - Citi - Analyst : Got it. Got it.

Question: David Lebowitz - Citi - Analyst : And my last question on this, just from a bookkeeping standpoint. The profit share? Just how should we be thinking about in terms of putting it in our models to -- so we're trying to be economically honest?

Question: David Lebowitz - Citi - Analyst : Got it. So let's jump on next, sepiapterin. Tell us about where things stand with sepiapterin, the NDA, there's a there's a PDUFA date and what you're thinking about that?

Question: David Lebowitz - Citi - Analyst : So do you think there's a disconnect with the investment community. They -- on one end they look at Kuvan and they know it's going generic. And on the other end, they see Palynziq, which is in the classical population. And they're just -- they kind of sit back and they're not really sure on either end of the spectrum, what it looks like. There's clearly a sweet spot. But as it drifts further and further into those other ends, they become increasingly tentative.

Question: David Lebowitz - Citi - Analyst : With respect to Kuvan historically, what do you think that the original data for the drug actually said and how much could be attributed to the compliance of therapies to the drug and how their clinical, I guess, the results actually. 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 03, 2024 / 7:30PM, PTCT.OQ - PTC Therapeutics Inc at Citi Global Healthcare Conference

Question: David Lebowitz - Citi - Analyst : That's very helpful. Let's jump over to vatiquinone. There's an NDA coming up before the end of the year, which is also coming up.

Question: David Lebowitz - Citi - Analyst : The trial -- the MOVE-FA trial did not succeed in this primary endpoint, but you saw data within the study, specifically upright stability, subscale of mFARS as the primary endpoint that was that was positive. How would you -- what would you say to the predictive nature of that particular aspect of the endpoint?

Question: David Lebowitz - Citi - Analyst : Now you recently put out a press release, you said that you reached alignment with the FDA on key aspects. And I know that certainly the FDA is the nimbleness of shifting relative to what was a predetermined result objective of a trial to positive findings within the trial the FDA has not always been easy to work with in that regard. What makes you feel confident that the FDA will look at this submission with -- I guess, through the new lens.

Question: David Lebowitz - Citi - Analyst : Now let's jump over to Upstaza or KEBILIDI. Did I get that right?

Question: David Lebowitz - Citi - Analyst : These names always kill me. Biotech names are tough. Tell us about the approval how the launch in Europe and what we can learn from that relative to the United States?

Question: David Lebowitz - Citi - Analyst : Now if we jump over to Translarna, there's 2 ends of this. There's the -- I don't know, Mary go around that's going on right now with Europe and the CHMP. Certainly, what's going to happen next because the CH -- or could the EC once again pass it back for another review causing more of a long-term dance. And then, of course, in the U.S., similar but different to the FDA submission it's -- we'll be going again soon. And how should we think about that?

Question: David Lebowitz - Citi - Analyst : Supposing the EC were to withdraw, have there been any stockpiling or anything of that nature because the patients will still want drug. And so there might be an element of trying to stockpile before any potential withdrawal.

Question: David Lebowitz - Citi - Analyst : Got it. Thank you very much for your time.

Question: David Lebowitz - Citi - Analyst : Appreciate it. Always glad to have 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 03, 2024 / 7:30PM, PTCT.OQ - PTC Therapeutics Inc at Citi Global Healthcare Conference