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| Statement |
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| As we discussed, we'll be submitting the MAA for sepiapterin the first quarter, and that infrastructure that we've built will be well-positioned for a successful -- what we're planning in the successful launch of sepiapterin in Europe |
| We're able to reassure both the patients meeting the physician on that you can develop a drug for Huntington lowering that is safe and well-tolerated and has a necessary biodistribution and pharmacodynamic effect necessary for therapeutic benefit |
| 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 team is well-positioned to continue to execute across all our commercial products and across all geographies, together with building out the foundation for sepiapterin for success in 2024 and beyond |
| With this infrastructure and track record of execution, I remain confident in our team's ability to succeed with our next product launches, including sepiapterin for the treatment of PKU |
| 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 |
| In conclusion, the strong fourth quarter rounds out what was a strong 2023 for our commercial team |
| I'm very proud of the team's efforts and determination in these last few months as they have worked tirelessly to ensure that every single Translarna patient in Europe continues to receive treatment until the ratification of the CHMP opinion |
| So, after that discussion, it was agreed that we have the ability to potentially submit an NDA based on MOVE-FA along with confirmatory evidence from the long-term open-label extension portion of MOVE-FA comparing those data to [Indiscernible] data very much like Reata did to its SKYCLARYS NDA, that we're combining solid, reliable, strong evidence of clinical benefit in the MOVE-FA study along with confirmatory evidence from the open-label portion of that study |
| We are very excited about the potential of vatiquinone to fill the significant remaining unmet need for pediatric and adolescent FA patients |
| In closing, we are well-positioned for an exciting 2024 |
| I'm proud to say that we have partnered with -- since the beginning of the clinical development, in particular and ongoing many years back, and we've been incredibly grateful for their partnership, not only in helping us understand how to best design trials, their work in establishing a patient registry that will obviously be very important in us putting together a data package for NDA |
| Our global customer-facing team has delivered yet another strong quarter, continuing the significant momentum we have built |
| In addition, we have heard from patient advocacy groups around the world, the PKU patients are excited as they've been waiting for a therapy that combines efficacy through both Phe reduction and improved fee tolerance with tolerability |
| and EU and also new markets where we have invested in geographic expansion in Latin America, the Middle East, North Africa, and the Commonwealth of Independent States, where there has been a robust year-over-year growth |
| Feedback from the PKU community is extremely positive, and there is a widespread recognition amongst metabolic specialists, geneticists, and dieticians of the potential of sepiapterin to meet the significant unmet needs for many of their PKU patients, not just patients who have been unresponsive to Kuvan, but also those who are fully-controlled on this drug and could potentially do significantly better on sepiapterin as we saw for numerous patients in the APHENITY trial as well as classical PKU patients |
| For the DMD franchise, we closed out the year with a strong fourth quarter for both Translarna and Emflaza, delivering an impressive $143 million in net revenue, which is 25% growth compared to the fourth quarter of 2022, with a strong full year performance of $611 million |
| Our revenue performance reflects the continued outstanding work of our global customer-facing teams and our successful efforts in geographic expansion |
| So, that's obviously very encouraging because as we shared last June, there's very strong evidence of safety and tolerability thus far with no evidence of NFL spikes steam and also no treatment-related serious diverse events |
| The strong balance sheet provides PTC with the resources to execute on our strategy and to achieve our milestones over the next several years, including the anticipated sepiapterin launch |
| Patient identification is robust and the number of patients on treatment continues to grow across the region, including first-time revenue in new countries |
| We closed out the year with robust growth for both Tegsedi and Waylivra, more than doubling our revenue in the region |
| We continue to support DMD patients and their caregivers with outstanding service from our PTC Cares team by enhancing customer experience, providing easier access to treatment, facilitating co-pay assistance, and improving adherence for patients in the U.S |
| And then, of course, the APHENITY study, where we had a number of patients, 27 patients who came in the study on Kuvan we had washed out of it, and then we had a 50%, roughly 50% greater lower phenylalanine [ph] once those patients pushed over to sepiapterin, again, clearly demonstrating that we're able to provide significant greater benefit to these patients |
| And I think that positions us very well to capture significant treatment benefit |
| And I think what we have seen and understand through speaking to both physicians and patients in the community is that there is a strong brand loyalty to Emflaza |
| sepiapterin has novel composition, novel mechanism of action, novel biodistribution, novel pharmacology, and clearly differentiated therapeutic benefit |
| More importantly, we continue to see durability of treatment effect and the ability for patients on sepiapterin to increase their dietary protein intake beyond the recommended daily allowance, while still maintaining control of Phe levels in the long-term EXTENSION study |
| This liberalization of diet is incredibly meaningful to patients and further supports the potential of sepiapterin to fill the persistent unmet medical need of the majority of the 58,000 PKU patients worldwide |
| Statement |
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| Most of you are aware, in January, we received a negative opinion from the CHMP on the continued conditional marketing authorization for Translarna in the EU |
| As we've said in the past, we've obviously had the fact that loss of exclusivity has been on the horizon, and we've been preparing for this |
| 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 |
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