Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE) Q1 2022 Earnings Conference Call May 5, 2022 5:00 PM ET

Company Participants

Joshua Higa - Director, IR & Corporate Communications

Emil Kakkis - President, CEO & Director

Erik Harris - EVP & Chief Commercial Officer

Mardi Dier - EVP & CFO

Camille Bedrosian - Chief Medical Officer & EVP

Conference Call Participants

Huidong Wang - Barclays Bank

Yigal Nochomovitz - Citigroup

Tazeen Ahmad - Bank of America Merrill Lynch

Joel Beatty - Robert W. Baird & Co.

Maurice Raycroft - Jefferies

Joon So Lee - Truist Securities

Brendan Smith - Cowen and Company

Dae Gon Ha - Stifel, Nicolaus & Company

Jeff Hung - Morgan Stanley

Joori Park - SVB Leerink

Laura Chico - Wedbush Securities

Operator

Good day, and thank you for standing by. Welcome to the Ultragenyx First Quarter 2021 Financial Results and Corporate Update Conference Call. [Operator Instructions].

I'd like to hand the conference over to your speaker today, Joshua Higa.

Joshua Higa

Good afternoon, and welcome to the Ultragenyx Pharmaceutical financial results and corporate update conference call for the first quarter of 2022. We have issued press release detailing our financial results, which you can find on our website at ultragenyx.com.

I am Joshua Higa, Director of Investor Relations. Joining me on this call are Emil Kakkis, Chief Executive Officer and President; Erik Harris, Chief Commercial Officer; Mardi Dier, Chief Financial Officer; and Camille Bedrosian, Chief Medical Officer.

I would like to remind everyone that during today's call, we will be making forward-looking statements. These statements are subject 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.

Emil Kakkis

Thanks, Josh, and good afternoon, everyone. In the first quarter, we continued to make progress across our diverse clinical and commercial programs. In the late-stage clinical pipeline, we're now enrolling patients in 3 registrational studies with the fourth to initiate later this year. This includes the 3 gene therapy programs for glycogen store disease Type 1a, Wilson disease and ornithine transcarbamylase deficiency as well as our anti-sclerostin antibody for osteogenesis imperfecta, the GTX-102 study for Angelman syndrome is progressing well, and we remain confident in the program. The results we shared in 2020 from the original 5 patients led us to reimagine what is possible for patients with Angelman syndrome. Given the importance of this program, we've looked at ways -- various ways to accelerate this program. And recently, we amended our agreement with genetics to allow us an additional option to acquire them at an earlier time point based on interim data.