PTC Therapeutics, Inc. (NASDAQ:PTCT) Q2 2023 Earnings Call Transcript

Page 10 of 13

And of course that in and itself does so much to preset the market. It increased both patient and physician desire to get that therapy. So Kylie, do you want to talk a little bit about preparation for the U.S.?

Kylie O’Keefe: Yes. Absolutely Joe. We are engaging with U.S. payers and we do this well ahead of launch to understand what are the types of queries that they have and what are some of the roadblocks that might be put in front of us. And we obviously especially with AADC need to do education around what is the disease the high morbidity and mortality rates the lack of standard of care, or any disease-modifying therapies out there available and obviously budget impact. And all of that work happens ahead of approval and the U.S. team has been actively engaged in that process to date. As Matt said, with a strong data package and transformative data not just in the short-term, but also in durability, which is one of the questions that a lot of payers have put in front of other companies with gene therapies.

How well does your treatment perform not only in the short-term, but also in the long-term? We feel very, very confident around the data package that we have for Upstaza. And the value proposition in totality has been very well received by U.S. payers and there has been a high willingness to pay.

Matthew Klein: And Joe on your second question regarding — we’ll learn a lot more about the portfolio in the coming months. And obviously, we’ve done a lot of work in our own strategic prioritization. Obviously, the decision to discontinue the preclinical gene therapy programs, as they move more towards appropriate strategy and focus and obviously BD can be an important part of that. Pierre, do you want to add some more color on funding and BD opportunities?

Pierre Gravier: Absolutely. So as Matt said the team and the company is well aligned about our capital and our strategy to leverage our expertise and being opportunistic on BD. As it comes to Blackstone, we’re very proud to have partnered with such a strong group with life science expertise and we will always evaluate our funding options as it comes to be there on our capital structure.

Joseph Thome: Great. Thank you very much.

Operator: Thank you. And one moment for our next question. And for our next question we have Gena Wang from Barclays. Your line is now open.

Unidentified Analyst : Hi. It’s Tony on for Gena. Going back to the Huntington’s program, could you just add some more color potentially on what kind of threshold you might be looking for in terms of CSF protein? I know you talked about 30% to 50% range, but is there kind of a specific cutoff you would be specifically looking for?

Matthew Klein: Yes. Tony, thank you very much for the question. Obviously, there’s been a lot of discussion about CSF Huntington protein, what it means and how do we measure it and many things like that. Look we’re in a position, where there’s many important biomarkers of disease that we’re measuring in the study. We’re measuring CSF protein. We’re also measuring NfL, which obviously has been shown in neurodegenerative disease to be a very important marker obviously of safety in terms of potential nerve injury, which I mentioned we’ve not see any sign of thus far in the PIVOT-HD trial. But also efficacy. We can look to first an accelerated approval as an indicator that there’s broad interest including from regulatory authorities to look at NfL as an important marker of efficacy in neurodegenerative disorders that are characterized by neuronal injury and neuronal loss.

Page 10 of 13