PTC Therapeutics, Inc.

Good day and thank you for standing by. Welcome to PTC fourth quarter 2023 financial results. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 11 on your telephone. You will then hear a message advising your hand is raised. To withdraw your question, press star 11 again. Please note that today's conference is being recorded I would now like to pass the call over to the Senior Director of Investor Relations, Ron Aldrich.

Good afternoon, and thank you for joining us today to discuss PTC Therapeutics' fourth quarter and full year 2023 corporate update and financial results. I'm joined today by our Chief Executive Officer, Dr. Matthew Klein, our Chief Business Officer, Eric Powles, our Chief Commercial Officer, Kylie O'Keefe, and our Chief Financial Officer, Pierre Gravier. Today's call will include forward-looking statements based on our current expectations. Please take a moment to review the slide posted on our investor relations website in conjunction with the call, which contains our forward-looking statements. Our actual results could materially differ from these forward-looking statements, as such statements are subject to risks that can materially and adversely affect our business and results of operations. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent annual report on Form 10-K filed with the Securities Exchange Commission, as well as the company's other SEC filings. We will disclose certain non-GAAP information during this call. Information regarding our use of GAAP to non-GAAP financial measures and a reconciliation of GAAP to non-GAAP are available in today's earnings release. With that, let me pass the call over to our CEO, Matthew Klein. Matt?

Thank you, Ron. Good afternoon, and thank you all for joining today's call. I'm pleased to share our fourth quarter and full year 2023 results and to provide an update on the progress of our pipeline programs as we move into what will be an exciting 2024 with a number of potential significant milestones. As we closed out 2023, we had another solid quarter of commercial performance, with total fourth quarter revenue of $307 million and full year 2023 revenue of $938 million, representing 34% growth over 2022. Our DMV franchise revenue totaled $143 million in the quarter and $611 million for the full year. Eric and Kylie will provide additional detail on our commercial performance shortly. Our revenue performance reflects the continued outstanding work of our global customer-facing team, and our successful efforts in geographic expansion. With this infrastructure and track record of execution, I remain confident in our team's ability to succeed with our next product launches, including sepia tarant for the treatment of PKU. Most of you are aware, in January, we received the negative opinion from the CHMP on the continued conditional marketing authorization for Translarna in the EU. This opinion is expected to be ratified by the EC in late March or early April. Importantly, Translarna remains on the market until ratification occurs. In addition, we continue to commercialize Translarna in several regions outside of the EU where independent authorizations exist. While we are, of course, disappointed in the CHMP opinion and the potential impact it has on patients in Europe, PTC is well positioned to withstand this outcome. As I have previously emphasized, Through our efforts to focus our R&D portfolio, right-size the organization, and strengthen our balance sheet, we have a solid foundation for building the company forward and continuing to deliver on our goal of discovering, developing, and commercializing transformative therapies for patients. As we look forward to 2024, we have a number of important potential regulatory and clinical milestones for a number of our programs. I will begin with our CP Taran program for the treatment of children and adults with PKU. We remain on schedule to submit the MAA for CpAterin to the EMA in the first quarter of this year and to submit the NDA in the United States no later than the third quarter of this year. As we continue to collect data from the open-label extension study following the Phase III affinity trial, we continue to see durability of CpAterin treatment effect and the ability of patients on CpAterin to tolerate increases in dietary protein intake beyond the recommended daily allowance. This liberalization of diet is incredibly meaningful to patients and further supports the potential of sepia tarant to fill the persistent unmet medical need of the majority of the 58,000 PKU patients worldwide. Moving to our vaticanone program for Friedreich's ataxia, we had a type C meeting with the FDA earlier this quarter. Based on discussions with FDA, we now have a path to potential NDA filing based on the placebo-controlled results of the MOVE-FA study in combination with long-term open-label extension data currently being collected. The open-label data will be compared to a natural history population from the robust Friedreich Ataxia patient registry, using analyses similar to those provided to FDA by REATA as part of the Slyclaris NDA. Based on the time needed to collect sufficient long-term open-label data, we expect to be able to submit an NDA in late 2024. We are very excited about the potential of the tiquidone to fill the significant remaining unmet need for pediatric and adolescent FAA patients. In other regulatory updates, we remain on track to submit the BLA for abstainment to the FDA in March and are also scheduled to meet with FDA in March to discuss the contents of a potential NDA resubmission for transwana. Turning to our ongoing clinical trials, We expect to share interim 12-month results from the PIPHD trial of PTC518 in HD patients in the second quarter of this year from the initial cohort of subjects on whom we reported data last summer. The 12-month results will include additional safety and tolerability data, as well as biomarker data, including CSF Huntington protein levels, NFL levels in the blood and in the CSF, and volumetric changes on MRI. We will also be sharing data from the clinical outcome measures collected as part of the study. Finally, we expect to share top line results from the Cardinals Registration Directed Trial of Eutraloxistat in ALS patients in the fourth quarter of this year. In closing, we are well positioned for an exciting 2024. We have the team, the capital, and the strategy that position us to execute on the many impactful opportunities that lie ahead. I will now turn the call over to Eric and Kylie to discuss our commercial performance.