RARE - Ultragenyx Pharmaceutical Inc. (RARE) Q1 2024 Earnings Call Transcript
2024-05-02 20:28:09 ET
Ultragenyx Pharmaceutical, Inc. (RARE)
Q1 2024 Earnings Conference Call
May 02, 2024, 17:00 PM ET
Company Participants
Joshua Higa - Vice President of Investor Relations
Emil Kakkis - Chief Executive Officer & President
Erik Harris - Chief Commercial Officer
Howard Horn - Chief Financial Officer
Eric Crombez - Chief Medical Officer
Conference Call Participants
Yigal Nochomovitz - Citigroup
Anupam Rama – JPMorgan
Tazeen Ahmad - Bank of America Securities
Gena Wang – Barclays
Dae Gon Ha – Stifel
Kristen Kluska - Cantor Fitzgerald
Maurice Raycroft – Jefferies
Jeffery Hung - Morgan Stanley
Joon Lee - Truist
Joseph Schwartz - Leerink Partners
Brendan Smith - TD Cowen
Lidia Rachkova - Goldman Sachs
Jack Allen – Baird
Luca Issi - RBC Capital
Presentation
Operator
Good afternoon, and welcome to the Ultragenyx First Quarter 2024 Financial Results Conference Call. [Operator Instructions] It is now my pleasure to turn the call to Joshua Higa, Vice President of Investor Relations. You may begin.
Joshua Higa
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, Erik Harris, Chief Commercial Officer; Howard Horn, Chief Financial 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.
Emil Kakkis
Thanks, Josh, and good afternoon, everyone. This is the year that we're harvesting the exciting results of multiple years of focused execution across our key clinical programs, and we've shared a lot of meaningful data already this year. At the World Symposium meeting in February, we presented positive biomarker and long-term cognition data from our UX-111 gene therapy in Sanfilippo syndrome. The data showed the treatment resulted in rapid and sustained reduction of CSF heparan sulfate and that this was correlated with improved long term cognitive development.
We also participated in a workshop on the heparan sulfate biomarker hosted by the Reagan-Udall Foundation. This workshop brought together FDA representatives, patient advocates, scientists and industry leaders to discuss the overwhelming body of data supporting the use of CSF heparan sulfate as a biomarker to enable accelerated approval in neuronopathic MPS diseases.
The support of Peter Marks and the FDA in recognizing this biomarker as a surrogate endpoint to support accelerated approval would be a profound benefit for the MPS communities and companies working on these diseases and really to all companies working on gene therapies or other type precision medicines.
Shifting to setrusumab, just this week, we announced that we've completed enrollment in our Phase II/III Orbit study and our Phase III COSMIC study in osteogenesis imperfecta. The Phase II data presented late last year was clearly compelling for the study investigators that led to accelerated interest and enrollment in the program. Two weeks ago, we announced strong positive interim data from the Phase I/II study of GTX-102 in Angelman syndrome. The interim data we shared confirmed in a larger body of data that GTX-102 can fundamentally change the development trajectory of Angelman patients. Importantly, the magnitude of the effect across all domains and expansion cohorts was found to be similar or greater than what we observed previously with the dose escalation cohorts....
Ultragenyx Pharmaceutical, Inc. (RARE) Q1 2024 Earnings Call Transcript