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

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Sami Corwin: Great. Thanks. And then given you have about $330 million in cash, how are you guys kind of thinking about capital deployment in terms of either focusing on your commercial franchises and products that will be going through regulatory submissions in the near term versus your earlier stage research pipeline?

Matthew Klein: Yes. I think as we’ve talked about Sami, we are well capitalized to take us to the next — take us through this year and get us to the PKU launch. We’ve talked a lot about having the infrastructure in place to launch PKU and other products. We also have a robust discovery and development infrastructure. So all the pieces are there and so we’re well positioned and well capitalized to move forward the programs and prepare for the launches that we expect to occur in the next one to two years.

Sami Corwin: Great. Thank you.

Operator: And thank you, and one moment for our next question. And our next question comes from Kristen Kluska from Cantor Fitzgerald. Your line is now open.

Kristen Kluska: Hi, everyone. Thanks for taking my questions and welcome Pierre. First just wanted to ask what the main questions will be at PKU pre-NDA meeting and essentially what feedback you’re looking for here.

Matthew Klein: Hey. Sure Kristen. Thanks for the question. So obviously the pre-NDA meeting is often focused around the structure of the NDA. How the components are put together how we do integrated safety analyses and created efficacy analyses. And then going through the checklist of are we in line with the terms of what need from a clinical standpoint efficacy standpoint safety database non-tox package CMC package. So it’s a fairly standard approach to pre-NDA meeting. Obviously, we are quite gratified to have the meeting granted. And our expectation is that we’ll be able to align with the agency and move forward with the submission in the fourth quarter.

Kristen Kluska: Okay. Thanks for that. And we’ve often talked to you about the synergies with the splicing platform, especially now that you’re progressing along with Huntington’s disease. But maybe just kind of wanted to ask a question from the sense of how much overlap you think there is with the neurologist community, in particular the adult community with the experience with Evrysdi given the launch has been pretty substantial here. And then, if you have any initial feedback that you’ve heard from some leaders in the space based off your early data?

Matthew Klein: Yes absolutely. I can give a little bit of color and then I’ll turn it over to Kylie for a bit more detail on this. Obviously SMA, just in general, it was both pediatric and adult but Huntington is obviously going to be mostly adult to we’re able to initiate and complete our work in juveniles. But I would say there is broad recognition in the scientific physician as well as the patient communities, importantly about the power of the splicing platform. And obviously, we talked a lot about Evrysdi being able to provide us with a blueprint of how to successfully discover, optimize, develop an oral compound — oral splicing compound for a whole brain disease such as SMA and now with Huntington disease. And I would say in many ways the Evrysdi experience has also set a path for us as well as we think about getting this out and doing clinical trials in both the patient and physician communities because of the recognition of the power of splicing platform and the ability to deliver an effective oral small molecule that is not only safe but be able to deliver meaningful results.

Kylie, I don’t know if you want to provide any more color on how we’re thinking about it and working with the physicians.

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