Ultragenyx Pharmaceutical Inc. 2026 Q2 Earnings Call

Thank you. We have issued a press release detailing our financial results, which you can find on our website at ultragenyx.com. Joining me on this call are Emil Kakkis, Chief Executive Officer and President, Howard Horn, Chief Financial Officer, Erik Harris, Chief Commercial Officer, and Eric Crombez, Chief Medical Officer. I'd like to remind everyone that during today's call, we will be making forward-looking statements. These statements are subject to certain risks and uncertainties, and our actual results may differ materially. Please refer to the risk factors discussed in our latest SEC filings. I'll now turn the call over to Emil.

Thanks, Josh, good afternoon, everyone. In the second quarter, we continued our pattern of strong execution across the development and commercial organizations. The commercial team delivered the highest quarterly revenue in the history of the company, which supports our reaffirmed full-year revenue guidance. As they have done in prior quarters, they continue to find new patients and expand access to Crysvita, DOJOLVI, Evkeeza, and MEPSEVII around the world. The commercial and field teams are also preparing for our first two potential gene therapy launches, which are anticipated in the coming months. If approved, both these therapies would represent first-ever treatments for diseases with significant unmet needs and high urgency to treat. The PDUFA date for DTX-401 for GSD 1A is just a few weeks away.

Patients with GSD 1A have to drink a slurry of cornstarch every few hours around the clock, day and night, knowing that a single missed dose could lead to their death. This is a constant reminder of their disease and the severe consequence of missing a dose. In our phase I/II and phase III studies, patients treated with DTX-401 have been able to significantly reduce the amount and frequency of cornstarch doses to a pre-specified, clinically meaningful degree. More importantly, this gene therapy allows patients to have a more normal glucose metabolism, staying in the normal range a larger fraction of the day. The expression of the missing G6Pase enzyme from the DTX-401 vector should allow their liver to break down glycogen to produce glucose during times of fasting or metabolic stress.

The ability to regulate glucose better has reduced the burden of disease and reduced the potential for rapid decline to dangerous glucose levels. Transitioning now to UX-111. I've been in and around the MPS community for many years, and I've seen so many Sanfilippo patients have to watch helplessly as their children decline and die. Sanfilippo syndrome type A has an urgent need to treat, yet there has been nothing for them. Sanfilippo syndrome is a horrible neurodegenerative disease, and kids are losing brain cells every day. Patients between the age of two and six years progressively lose their cognitive functions. By 10 years old or so, they're often bedridden and tube-fed and in and out of the hospital for many years. They can end up non-responsive for the last five or 10 years and often die as teenagers.

Until now, families have had no option but to watch helplessly as their children decline and die. Based on our clinical data, treatment with UX-111 reduces heparan sulfate in these patients and enables them to stabilize and retain cognitive function compared to natural history. While patients across the age range in the study showed benefits following treatment with UX-111, it's also clear that treating earlier demonstrated better results by protecting their brains before they had lost too much function. With a disease like Sanfilippo syndrome, there's no greater urgency to treat. As long as you wait, the less function you may have. Our development organization under Dr. Krombez has been working with regulatory agencies around the world on BLA submissions for both DTX-401 for GSDIa and UX-111 for Sanfilippo A syndrome. These include preparing the applications, responding to information requests, and supporting facility inspections.

This would be a lot for a single BLA. Our team is working simultaneously with the process for two BLAs that have PDUFA dates a month apart. We remain confident in the work our team is doing to support these applications. Shifting to GTX-102 for Angelman syndrome, where we have the most advanced clinical ASO program. In the Phase III ASPIRE study, the last patient in has had their 48-week visit, and the team is in the process of cleaning and locking the database. For a global study like this, the process could take months, and we expect to unblind and share top-line results with you in September or October timeframe. With three major catalysts on the horizon, the second half of 2026 is poised to be the most significant period in our company's history.

We're positioned to deliver another exciting series of potential firsts for our company, the communities we serve, and the broader field of rare disease medicine, including the first gene therapy approvals for Ultragenyx, the first FDA approvals for two devastating and intractable rare diseases, and the first late-stage clinical data in Angelman syndrome. It's a privilege to continue to lead the future of rare disease medicine, and we believe the combination of continued growth from our current products accelerated by contribution from potential upcoming launches and disciplined expense management gives a clear path to profitability in 2027. I'll now turn the call over to our chief commercial officer, Eric Harris, who'll provide details on the commercial business and launch readiness activities in the second quarter.

Thank you, and good afternoon, everyone. As Emil mentioned, we are pleased with our steady, successful commercial execution, and we continue to see the benefit of our global infrastructure across products and regions. As we described on the last call, underlying demand for our products remains strong, but seasonal factors can cause quarter-to-quarter variability in revenue. In the second quarter, we saw the strong rebound in orders that we were expecting, reinforcing our confidence that we are on track to deliver against our full-year guidance. Starting with Crysvita in North America, where our partner, KKC, is commercializing, we continue to see steady growth in the underlying demand. Eight years post-launch, it's extremely gratifying to continue to see the steady addition of new patients starting this therapy.

In Latin America, Crysvita performance was strong in the second quarter, supported by the combination of continued patient growth and timing of regional orders, including Brazil and Argentina. Approximately 50 patients began commercial therapy in the quarter, bringing the total number of patients on Crysvita to 1,000 in the region. As discussed, we expect that ordering patterns will continue to create some quarter-to-quarter variability, and we are confident that the underlying demand will continue to grow steadily. In Turkey, there are over 100 patients being treated with Crysvita through the named patient program. As has been the case in other countries, there is accelerating demand as HCPs and patients see the benefits this transformative therapy offers. Shifting now to DOJOLVI, the trend of steady growth continues. In North America, our team generated approximately 30 start forms in the quarter, and we now have approximately 675 patients on reimbursed therapy.

In Europe, approximately 300 patients are being treated through named patient or early access programs, with notable growth in the MENA region following marketing authorization approval in Kuwait. We also recently began treating patients with DOJOLVI in Japan, following its listing on the National Health Insurance (NHI) Drug Price List. We are off to a strong start in demand, reinforcing our belief that this country represents another meaningful opportunity for continued growth over time. Evkeeza continues to be an important and growing contributor to our revenue base. In our territories outside of the U.S., we are responsible for commercialization. There are more than 500 patients across 25 countries receiving Evkeeza, as our teams continue to navigate country-by-country reimbursement, support early access, and convert appropriate patients to reimbursed therapy. Beyond the individual product performance, the real strength of this business is the scale, reach, and experience of our global commercial infrastructure.

Rare disease commercialization requires deep market-by-market expertise to include patient finding, reimbursement navigation, and field execution. That is what our global team provides every quarter in giving us high confidence that we will be prepared for the next potential launches. I'll close briefly with a couple of comments on the team's work preparing to successfully launch DTX401 and UX111. Launching two gene therapies in parallel is a big undertaking. We are up to the task and ready to deliver for patients. We are preparing thoughtfully and deliberately to support not only patients and caregivers but clinicians and payers beginning on day one. As we prepare for these potential launches, we are actively working with Qualified Treatment Centers, or QTCs, with contracting and activities progressing as planned.

We believe their strong engagement reflects their sense of urgency for patients and families impacted by GSDIa and Sanfilippo syndrome type A, and their understanding of the clinical potential of our investigational gene therapies. Our market access preparations are also progressing well, informed by extensive education and engagement efforts. Across more than 200 engagements with payers, we've been pleased that they recognize the significant unmet need associated with both diseases and understand the particular urgency to treat in Sanfilippo syndrome. The foundation necessary to support broad and timely access is set. With a focus on minimizing barriers for eligible patients following approval, we've been building toward these potential launches for years. We stand ready to add these products to our existing portfolio and expand our mission of helping patients with rare disease.

I'll turn the call to Howard to share more details on our financial results and guidance.