PTC Therapeutics, Inc.

Ladies and gentlemen, thank you for standing by. Welcome to the PTC Therapeutics third quarter 2026 earnings conference call. All participants are on a listen-only mode. After the presentation, there'll be a question and answer session. Today's conference is being recorded. I would like to turn the conference over to Ellen Cavaleri, head of investor relations. Please go ahead.

Good afternoon, and thank you for joining us to discuss PTC Therapeutics third quarter 2026 corporate update and financial results. I am joined today by our Chief Executive Officer, Dr. Matthew Klein, our Chief Business Officer, Eric Pauwels, and our Chief Financial Officer, Pierre Gravier. Today's call will include forward-looking statements based on our current expectations. These statements are subject to certain risks and uncertainties, and actual results may differ materially. Please review the slide posted on our investor relations website in conjunction with the call, which contains information about our forward-looking statements, and our most recent quarterly report on Form 10-Q and annual report on Form 10-K filed with the SEC as well as our other SEC filings for a detailed description of applicable risks and uncertainties that could cause our actual performance and results to differ materially from those expressed or implied in these forward-looking statements. Additionally, we will disclose certain non-GAAP information during this call. Information regarding our use of gap to non-gap financial measures and reconciliation of gap to non-gap are available in today's earnings release. I will now pass the call over to our CEO, Dr. Matthew Klein.

Thank you all for joining us today. I'm pleased to share the results of another quarter of record product revenue and solid execution across the company. In the third quarter, we achieved total revenue of $358 million. including $276 million in product revenue led by continued quarter-over-quarter growth of Sophia in sales in the U.S. and internationally. Based on this performance, we are raising our full-year 2026 product revenue guidance to $960 to $990 million and now expect total revenue of $1.29 to $1.32 billion. This updated guidance represents 64% to 69% in year-over-year product revenue growth. In addition, with our continued effective expense management, we expect to achieve our goal of reaching cash flow breakeven in 2026. Beginning with Safiance, we are now one year into the global launch, which by all measures has been a great success, and all signs point to Safiance being a blockbuster rare disease product. In the third quarter, Worldwide Suppliance Revenue was $182 million, led by consistent growth in the U.S. and continued expansion internationally. And as of September 30, we have over 2,000 active patients on Suppliance worldwide. In the U.S., we continue to see strong underlying demand across all patient segments. Over the course of the third quarter, we saw uptick in branded and generic KuVan switches, with continued penetration into the therapy naive and tried and failed patient segments. Adherence remains strong, reinforcing the meaningful benefits patients are experiencing on therapy, including diet liberalization, cognitive function, and mood improvements. Internationally, we also had continued growth in the quarter, led by the strong launch in Japan, as well as new patients granted access through named patient programs across several regions. As we have discussed, We expect international revenue to continue to grow with acceleration points as we achieve access and reimbursement in additional countries over the course of 2027. In the first year of launch, we built a broad commercial patient base upon which we are well positioned to expand as we move forward into 2027 and beyond. In the U.S., there remains a large number of patients still on branded or generic KUVAN who are candidates for switches. as well as a large number of patients without on any therapy that could benefit from suffiance. Overall, the vast majority of the 17,000 patients in the U.S. have yet to try suffiance. We see strong underlying demand across all patient segments fueled by the differentiated profile of suffiance and the growing real-world evidence of meaningful suffiance benefits. Thus, while we are proud of all we have accomplished over the past year, We believe there is still a long way to go in a launch. Furthermore, our teams have implemented plans to address a number of the key launch learnings that will support continued success in the coming years. Internationally, we expect continued growth in 2027 as we expand into new markets in Europe, Latin America, the Middle East, Russia, and the Commonwealth of Independent States. All geographies where we have experienced teams with a track record of success. Overall, we remain confident in the 2 billion plus peak sales opportunities for suppliers. In the third quarter, we acquired the BLA stage Fabry disease gene therapy asset, ST920. ST920 is a one-time administered AAV gene therapy that provides a humanized copy of the GLA gene, enabling the body to produce sustained physiological levels of the alpha-GalA enzyme deficient in the disease. The data collected to date demonstrate that ST920 significantly increases alpha-GALA activity, decreases the lysogv3 substrate that causes organ damage, and improves renal function and other aspects of disease. In addition, the safety profile has been demonstrated to be favorable, with no serious adverse events related to patotoxicity or other immune reactions. We expect to complete the rolling BLA submission for accelerated approval in Q4 2026 with the potential for approval and commercial launch in 2027. Given the size of the Fabry population, our existing commercial infrastructure and experience working in the Fabry community, as well as a differentiated profile of ST920, we believe this is a potential meaningful global commercial opportunity. Turning to the Vodafone Huntington's Disease Program, Due to FDA scheduling, the Type C meeting to discuss the Vodafone data package and a potential path to accelerated approval is now planned for the first quarter of 2027. Enrollment continues in the Phase 3 InvestHD trial of Vodafone, which is planned to enroll approximately 770 early symptomatic patients. The primary endpoint of the study is change in CUHDRS from baseline up to 36 months with an interim analysis plan for efficacy and futility. Excellent progress on enrollment has been made, with sites now open across the globe. For the reticulodon frigidus taxia program, we initiated the global PROVE-FA trial in the third quarter as expected. PROVE-FA is a single-arm study with a well-matched natural history comparator arm intended to support NDA resubmission. The study design, protocol, and statistical plan have all been reviewed by FDA ahead of a trial initiation. The study is expected to enroll approximately 120 individuals between 7 and 21 years of age, with the primary endpoint being the MFARS disease rating scale. In the third quarter, we also made good progress on our other pipeline programs. We initiated a Phase 2A study of PTC844, our next-generation oral DHO-DH inhibitor. As we shared at our R&D day last December, PTC844 has a differentiated potency of selectivity profile that benchmarks very well against approved DHO-DH inhibitors. The Phase 2A trial is a 12-week PK-PD study being conducted in a cohort of adults with rheumatoid arthritis with the objective of confirming the relationship between dose, DHO-DH inhibition, and favorable effects on markers of B-cell and T-cell immunity. We plan to use the results of this study to help finalize which rare inflammatory disorders we will pursue for PTC844 development. The Phase 1 study of PTC612, our oral NLRP3 inhibitor, is progressing well. We have dosed several SAD and MAD cohorts, including a cohort with inflammation and cardiovascular disease that could provide early evidence of pharmacodynamic effect on a number of biomarkers.