PTC Therapeutics, Inc.

Thank you for standing by, and welcome to the PTC Therapeutics Third Quarter 2023 Financial Resorts Conference Call. At this time, all participants are on a listen-only mode. After the speaker's presentations, there will be a question-and-answer session. To ask a question at that time, please press star 11 on your telephone. Please be advised today's call is being recorded. I will now turn the conference to your host, Jane Hanlon, Associate Director of Investor Relations. Please go ahead.

Good afternoon, and thank you for joining us today to discuss PCC Therapeutics' third quarter 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 the results of operations. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent quarterly report on Form 10-Q and annual report on Form 10-K filed with the Securities and 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 earning release. With that, Let me pass the call over to our CEO, Matthew Klein. Matt?

Thank you, Jane. Good afternoon, and thank you all for joining the call. I'm pleased to share our third quarter results and outlook for the remainder of the year, including an update on our development programs. I will begin with the recent announcement of our agreement with Royalty Pharma to monetize up to $1.5 billion of the Abrisdy royalty stream. This non-dilutive financing provides PTC the capital to support planned operations and allowed us to retire the Blackstone debt obligations. In addition, the deal structure includes flexibility for accessing additional capital over the next two years. Notably, PTC maintains its rights to the remaining $250 million of milestones related to the RISD global net sales. The royalty financing deal, along with the operating expense reductions announced in September, put PTC on very strong financial footing as we continue to focus our resources on our differentiated high potential R&D programs and robust global commercial infrastructure. Now I'll turn to our third quarter results. We had another solid quarter with total revenue of $197 million, which keeps us on target for meeting our 2023 total revenue guidance of $940 million to $1 billion. Our DMV franchise revenue in the quarter totaled $136 million. This strong performance allows us to update our 2023 DMV revenue guidance to between $565 million and $595 million from between $545 million and $575 million. Eric and Kylie will provide additional details on our commercial performance shortly. I'd like to now provide an update on recent regulatory activities for several of our programs. Let me begin with an update on Transline. Following the negative opinion from the CHMP on the conversion of the conditional marketing authorization to full marketing authorization and on the renewal of the conditional authorization, CHMP gave us the option to request reexamination of both opinions or only one opinion. We decided to pursue reexamination of the negative opinion on renewal of the conditional authorization only. As such, the reexamination process will focus solely on the allowance of continued conditional authorization of TransLarna in Europe. We remain optimistic that we can address key concerns raised by the CHMP on the evidence of benefit in the TransLarna clinical trials, as well as concerns raised on the methodological robustness of the STRIDE data analyses. As previously discussed, in accordance with EMA guidelines, we expect the opinion from the reexamination procedure in late January with adoption of that opinion by the European Commission 67 days later. The U.S., a Type C meeting with FDA to discuss a potential path to NDA resubmission, is scheduled for this quarter. Turning to CpEuterin, we held a pre-NDA meeting in the third quarter with FDA to discuss the NDA submission. At the meeting, FDA stated that the CpEuterin clinical safety and efficacy data supported NDA submission for the treatment of pediatric and adult PKU patients. However, they requested that we complete a 26-week non-clinical mouse study to assess potential carcinogenicity risk of sepia-tarian prior to submission. This non-clinical study was initially not required when sepia-tarian was acquired from CENSA as the NDA submission was planned under the Section 505B2 pathway. With our decision to file under the 505B1 pathway, the 26-week study is considered a required NDA component if needed to inform labeling. we will continue to discuss with FDA the potential to submit the mouse study results during the NDA review process. We now expect NDA submission to occur no later than the third quarter of 2024. This submission could occur in the second quarter if FDA allows submission of the nonclinical study report during the review process. For the EU, we expect to submit a marketing authorization application to EMA in the first half of 2024. The delay in NDA submission in no way mitigates the strength of the affinity data. Given the highly meaningful clinical effects observed in the trial, as well as the continued evidence of providing phenylalanine tolerance benefits to the full spectrum of PKU patients in the long-term open-label extension study, we remain incredibly enthusiastic about the potential billion-dollar-plus global commercial opportunity for C. peterin. Moving to the PTC518 Huntington's disease program, Enrollment is ongoing in the PIVOT-HD study for both the Stage 2 and early Stage 3 cohorts. We expect the next data update to occur in the first half of 2024. This update will include 12-month data on the initial group of subjects on whom we reported data in June of this year. Regarding the status of the trial in the United States, we had a Type A meeting with FDA to discuss the clinical safety data needed to enable resumption of enrollment of the PIVOT-HD trial at U.S. study sites. At the meeting, FDA stated that the existing three months of safety data could support 12-week dosing at 5 milligrams and 10 milligram dose levels, and that six months of clinical safety data demonstrating a similar favorable safety profile could support 12-month dosing in the PIVOT-HD trial. This is very good news, as it suggests that the safety data being generated from PIVOT-HD should be sufficient to lift a partial hold in the United States. Turning to vitiquinone, the data in the MOVE-FA study demonstrated vitiquinone's treatment benefit across several disease endpoints, including favorable effects on the upright stability subscale of the mFARS assessment, which is predictive of time to loss of ambulation. We had a type C written response only meeting with FDA in the third quarter to determine whether the data from MOVE-FA would be sufficient to support an NDA for accelerated approval. In the written response, the FDA stated that while they see the value of upright stability as a clinically meaningful endpoint, they believe the confirmatory study would likely be needed to support NDA submission. As this was a written response only, and we believe we can address the concerns raised by the FDA, we have requested a follow-up live meeting. In parallel, we are participating in a scientific advice procedure with the EMA to determine if the MOVE-FA data could support a conditional marketing authorization application in the EU. We expect to have the outcome of this procedure in the first quarter of 2024. Turning to Upstaza, we had an informal meeting with FDA in the third quarter, at which time they said that the data we have provided to support comparability between the clinical drug product and the intended commercial drug product were still not sufficient. However, in that meeting, the FDA said that the available data from the clinical study in the United States assessing the safety of the drug delivery cannula could be used to support a BLA for accelerated approval based on biomarker data demonstrating a treatment-related increase in de novo dopamine production. FDA suggested that we conduct a pre-BLA meeting to review the contents of the planned BLA. This meeting has been scheduled for December, and pending the outcome, we expect to submit the BLA shortly thereafter. Let me conclude by saying I'm incredibly proud of our team's continued ability to execute on all fronts. The recent Royalty Pharma financing deal, along with our operating expense reductions, position PTC as strongly as possible for future growth as we realize the potential of our many promising programs. I will now turn the call over to Eric and Kylie to discuss our strong commercial performance in the quarter. Eric.