Ultragenyx Pharmaceutical Inc.

Good afternoon and welcome to the Ultragenix second quarter financial results conference call. At this time, all participants are on listen-only mode. At the end of the prepared remarks, you will have the opportunity to ask a question during the Q&A portion of the call. It is now my pleasure to turn today's call over to Joshua Higa, Executive Director and Head of Investor Relations. Thank you.

We issued a press release detailing our financial results, which you can find on our website at ultragenix.com. Joining me on this call are Emil Kakas, Chief Executive Officer and President, Eric Harris, Chief Commercial Officer, Marty Deer, Chief Financial Officer, and Camille Bedrosian, 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, and good afternoon, everyone. We're now six months into the year, and across the company, we continue to make meaningful progress against our goals. The commercial team delivered another solid quarter of revenue growth as they commercialized our products across the globe. We acquired the late-stage product UX111 from MPS3A, or San Felipe Syndrome, an MPS disease area for which we have extensive experience. In July, we bolstered our cash position with a substantial royalty financing that also enabled us to acquire genetics and gain full control of our important Angelman program. These activities, along with progress across all of our early and late stage clinical programs, put us in good strong position over the coming years for exceptional value creation. I want to touch on a couple of pipeline updates before turning the call over to the other leadership team members to provide more detail on the quarter. Starting with GTX102 for Angelman syndrome, we reached a seminal moment in this program with the acquisition of genetics and now a full control over the GTX102 program. As we said on our call last week, we are all in on Angelman syndrome with the exceptional science regarding the target region and with the excellent data we've seen to date in Phase 1-2 study. It's rare to see a significant improvement in development function as we have seen recently. This is something I haven't seen in 30 years of drug development. Last week, we shared data that demonstrated our ability to safely dose patients with GTX1 and 2 at up to 10 milligram doses. We observed meaningful clinical changes in a group of children that are severely impacted by the disease due to the deletion of the maternal EB3A gene region. This severe mutation always predicts a very slow rate of learning on natural history, as Dr. Barry Kravis, one of the principal investigators on the study, said on our call last week. These early clinical responses across multiple domains and multiple clinical measures have been independently confirmed by clinicians, therapists, and patient families. We observed statistically significant improvements for some of these children and expect to further enhance these effects by increasing loading doses and providing more time in the study. One specific measure I would like to highlight is the Bayley Scales Infant Development, or Bayley. I'd like to remind you that Bayley is a standardized measure used to diagnose developmental delay in childhood. It is administered in the clinic by a trained therapist, not the investigator. And Bayley is often used in clinical trials to assess cognitive language and motor development in children. In this study, we're using the latest version of Bayley, which allows for evaluation of children with delays who exceed the Bayley age limit of neurotypical children. The extensive historical data with this measure allows the ability to set statistics and infant thresholds for improvement when evaluating individual patient results and showing they're different from error or variation. We know from natural history that scores in this measure do not meaningfully change for patients with Angelman syndrome, particularly those with deletion-type mutations. Receptive and expressive communication subscores are an important part of Bayley, particularly for patients with Angelman syndrome who lack communication skills. Across the patients in cohort four and five, when treated for a minimum of 128 days, seven of nine children showed a statistically significant improvement in either Bayley receptive... Operator, it seems like we might have lost Emil's audio connection.