PTC Therapeutics, Inc. Q2 2026 Earnings Call

Ladies and gentlemen, thank you for standing by. Welcome to PTC Therapeutics second quarter 2020 Earnings conference call. All participants are in listen only mode. After the presentation, there will be a question and answer session. Today's conference is being recorded. I would now 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 second quarter 2026 corporate update and financial results. I'm joined today by our Chief Executive Officer, Doctor Matthew Klein. Our Chief Business officer, Eric Powell, 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 GAAP to non-GAAP financial measures and Reconciliation of GAAP to non-GAAP are available in today's earnings release. I will now pass the call over to our CEO, Doctor Matthew Klein.

Thank you all for joining today. I'm excited to share the results of another quarter of outstanding execution across the company Starting with revenue, we achieved another record quarter with total revenue of $361 million, including $239 million of product revenue, driven by continued strong global supply and sales based on. This performance, we are raising our full year 2026 product revenue guidance to $850 million to $950 million. And now expect total revenue of 1.18 billion to 1.28 billion. In addition, with our continued effective management of expenses, we remain in position to potentially reach the milestone of being cash flow breakeven in 2026. I'll be. Giving an update on the supply and global launch momentum remains strong in the quarter with continued steady growth in the US and accelerating growth internationally. Second quarter global revenue was 151 million, up 21% quarter over quarter, with the majority coming from the US and increasing contributions internationally And as of June 30th, we had 1647 commercial patients on supply globally. We. To see broad uptake across all patient segments and age groups worldwide. There remains strong underlying demand for supply and our teams continue to effectively execute in us. We now have received prescriptions from 100% of the Centers of Excellence, as well as prescriptions from other centers, not previously designated as Centers of Excellence.

This breadth of penetration, this early in the launch is impressive, and we will now work to continue to penetrate deeper into these centers. We continue to see demand across disease, severities and treatment histories, including treatment naive patients, prior therapy failures, and those switching from existing treatments. Adherence rates remain strong, and we continue to hear reports. Not only of meaningful reductions in phenylalanine and increasing evidence of diet liberalization, but also of benefits on mood, cognition, and quality of life. These dynamics of strong demand and reported patient benefit reinforce our confidence in the long term commercial opportunity. In international markets. Momentum for science continues to build Commercial sales in Japan began to ramp in Q2 following our first commercial sale in late March. We started expanded access programs in a number of European countries and have seen positive progress towards future revenue in Latin America, including Brazil, where Sufyan's recently received the highest classification for therapeutic benefit from the country's drug market. Regulation chamber. As we have previously shared, we expect international revenue to increasingly contribute in late 2026 and into 2027 as additional markets come on board Overall, based on the continued strong launch momentum and the persistent strong underlying demand, we remain confident in the 2 billion plus global commercial opportunity for suppliers.

Turning to the Huntington's disease program. In April, we reported positive top line results from the 24 month interim analysis of the pivot HD. Long term extension study At month 24, VOD plan, demonstrated dose dependent slowing of disease progression on Q Hdrs, including an average slowing of 52% relative to natural history in the ten milligrams stage, two cohort Importantly, we continue to observe a favorable safety profile across doses and disease stages These data support the potential for plan to provide long term, meaningful clinical benefit for individuals affected by Huntington's disease. We also shared that Novartis has initiated enrollment in the global phase three invest HD study. Which is being conducted and funded by Novartis. The study is expected to enroll approximately 770 individuals with early symptomatic disease. Randomized 3 to 2 to receive ten milligrams or placebo, and the study includes an interim analysis. While the phase three study remains the base case for voter plan approval. The Novartis and PTC teams are finalizing a plan to engage with FDA in the second half of 2026 to discuss the 24 month results and potential accelerated paths for the Friedreich's ataxia program. Based on discussions with FDA, we have finalized a study protocol for the open label trial with a natural history comparator arm to support NDA resubmission.

We plan to initiate this study. Prove f a in the third quarter of this year. The trial will enroll approximately 120 individuals ages 7 to 21, and the study primary endpoint is the change in MFR from baseline to month 24. The study design reflects the key learnings from our prior development work and has understandably generated enthusiasm within the FA community, particularly because it offers patients the opportunity to access therapy without the uncertainty of receiving placebo. We believe the study design, coupled with our experience in FA therapy, clinical development, meaningfully enhances the probability of success of the proof study and the potential to deliver an important new therapy for children and adults affected by FA Shifting to our other clinical pipeline programs, we have initiated the phase one study of PTC six one two. Hour oral Nlrp3 inhibitor and have completed several of the single and multiple ascending dose treatment cohorts. Notably, this healthy volunteer study will enroll a cohort of individuals with obesity and cardiovascular disease, which we expect will provide an early view of pkpd effect. As we have previously shared, PTC 6.2 is differentiated from other Nlrp3 inhibitors in terms of potency, selectivity and chemical structure.

Later in the third quarter of this year, we expect to initiate a phase two, a study of PTC. Eight four for our next generation dose inhibitor, PTC eight. 44 is highly selective for the Doe enzyme and benchmarks favorably to other DHO inhibitors in preclinical potency. Models The PTC 844 study will be a 12 week PK PD study in which we will assess treatment effect on biomarkers related to T cell and B cell immunity. The results of this study will help inform the ultimate target indications for PTC. 844. We have also made a number of advances in our splicing platform. We recently selected a development candidate for our Msh3 splicing program PTC 303. Msh3 is increasingly recognized as an important target for triplet repeat expansion diseases, including Huntington's disease and myotonic dystrophy, as we have discussed the targeting, Msh3 provides a complementary approach to HCT lowering in addressing the key pathological causes of Huntington's disease. The MSC program also further reinforces PTC leadership in the discovery and development of oral small molecule splicing therapies. PTC 303 is fully owned by PTC, and we expect it to be in the clinic in 2027. Overall, the company remains in a very strong financial position In June, we strategically managed the 2026 Convertible Note liability with refinancing of the majority of the existing notes at a 0% coupon and 40% conversion premium to the stock's closing price at the time of the deal.

We closed the quarter with over $2.2 billion in cash and remain in position to achieve cash flow break even in 2026. In some. Summary. I am proud of our team's outstanding performance in the first half of 2026 as we continue to make significant progress across the business. I will now turn the call over to Eric to provide a commercial update, including additional details on this appliance launch Eric.

Thanks. Matt. In the second quarter, our commercial team continued the strong global launch of defiance driving solid product revenue and reinforcing confidence in our growth trajectory through 2026 and beyond. Launch momentum remains strong, supported by sustained contributions in the US and increasing contributions from international markets And now we have 1647 patients on commercial therapy globally on science. In the second quarter. Science revenue was 151 million, representing 21% growth over the first quarter of 2026. In the U.S.. Launch execution remains strong, with steady demand. As Matt noted, all US Centers of excellence are now prescribing science with adoption spanning the full spectrum of disease severity. All ages, including classical patients and a broad mix of treatment backgrounds, including therapy, naive adults. Prior treatment failures, and an increasing proportion of switches from existing therapies. We are pleased to see persistent strong demand and to hear reports of the meaningful impacts of science is providing children and adults on diet liberalization, as well as mood cognitive improvements and improvements in quality of life with our. Growing base of commercial PKU patients being treated with science in the US. Our customer facing teams are focused on rapidly filling new prescriptions and successfully managing Reauthorizations refills and providing exceptional patient support.

Our customer facing teams are dedicated to work closely with healthcare providers and the patient community to facilitate access to science. These. Efforts will help support continued momentum and growth as we move further into the launch. Turning to our patient advocacy support, we had a strong presence at the National PCPs Association conference in Chicago earlier this month, where we focused on connecting with patients and caregivers and understanding their unmet needs. At this meeting, we presented data from the Amplify study, demonstrating the ability of defiance to reduce blood. PHE significantly more than Bh4, as well as new data on patients switching to science in both pediatric and adult PKU populations. We also held several engagement events with patient advocacy groups, including a symposium emphasizing the extensive real world data and examples of patients who. In addition to seeing important reductions in blood PHE levels, have also experienced significant improvement in their day to day quality of life on treatment. Internationally, launch momentum continues to build in Japan. The early launch results are exceeding our internal expectations, with rapid uptake across many Japanese centers of excellence. We have secured pricing in Japan on par with the US and importantly, this price is locked in for ten years.

As we observed in the early stages of the US launch, we are seeing broad uptake in Japan across disease severities and age groups, as well as treatment histories. In Germany, we continue to see strong momentum in the quarter, especially with an increase in newly prescribed adult and naive PKU patients. As our pricing and reimbursement discussions are continuing in the country. In other European markets, we are leveraging early access and named patient programs effectively and are seeing accelerated demand in France. Italy and Spain. In other regions, including Latin America and the Middle East, we are similarly leveraging early access programs as we grow the global footprint and see important contributions to future commercial patients. While the US. US remains an important near-term growth driver, we expect global revenue to become an increasingly meaningful contributor in the second half of this year, and well into 2027. As we. Discussed, there is a large addressable PKU population globally in markets where innovative treatments are reimbursed and we expect to bring. Suffice to as many of these countries as quickly as possible. Reinforcing our confidence in achieving a multibillion dollar peak revenue potential. Looking ahead to our key event this summer, our.

Team is preparing for a strong showing at the SS. I'm meeting in Helsinki next month. This being. The largest global metabolism meeting of the year. We have 17 oral and poster presentations planned that continue to showcase evidence of treatment benefit across the broad range of individuals with PKU. Based on new analyses of long term trial data, as well as reports of real world evidence Many of these presentations will highlight the positive impact that science has had on PKU patients in countries around the world. With lower PHE levels, leading to better diet, neurocognitive and quality of life outcomes for these patients. Importantly, we plan to present new data demonstrating that treatment with syphilis responders led to a substantial portion of participants achieving complete normalization of blood PHE levels of 120 micromoles per milliliter in a rapid time frame, including those PKU patients who are classical and bh4 non-responsive. This impressive real world data demonstrated that achieving PHE level normalization is an important key treatment goal for all potential PKU patients. Now, turning to our mature brands. In the second quarter, we continued to generate meaningful revenue despite challenges in the DMD franchise. For. Translarna we had.

Continued sales in Europe and Latin America, as well as a government order from Russia. In. The US Emflaza continues to see new prescriptions and. Despite generic pressure, we see ongoing brand loyalty attributable in part to our programs and