Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q3 2020 Earnings Conference Call - Final Transcript
Oct 27, 2020 • 05:00 pm ET
Yes, sir. [Operator Instructions] Our first question comes from the line of Yaron Werber of Cowen. Your line is open.
Hi guys, this is Brendan on for Yaron. Thanks very much for taking the question and congrats again on the progress. Just a couple of quick ones from us. I guess first on DMD, as you mentioned there are obviously a few players already in motion here and I totally understand it very early in the process. But can you just give us maybe a little bit of a sense of how you kind of plan to be differentiated in your approach maybe mechanistically or if it's really a matter of your capsid versus other ones out there.
And then just really quickly on the data from the gene therapy, prophylactic steroid cohorts. I know you mentioned we could potentially get data from the OTC by year-end. I'm just kind of wondering about timing for the GSD1a patients. Thanks very much.
Emil D. Kakkis
Very good. When I look at the Duchenne program, there are several aspects of differentiation. One is the nNOS-binding microdystrophin from Solid. The second is the capsid we view, which has very excellent immune profile. The third is ability to produce sys [Phonetic] AAV in a mammalian large-scale system, which we've been using now for two programs very successfully and produce a high quality AAV. High quality many features, which are achievable with the producer cell line approach is more difficult to do with transaction approach. We think with those three areas, we are differentiated. Combined that with skills both companies have in this space, I think we have an opportunity to do something special for Duchenne patients and to bring program forward that can become accessible globally, not just in the US. So that's I think how I would answer that question.
On the prophylactic steroid timing, we are dosing patients in both groups. We will have some data on them this year, but neither program does a steroid cohort interfere with the initiation of Phase 3. Both programs were planned to have the prophylactic steroid approach built into the program, but it is helpful to us before we start Phase 3 to have done some tracings and shown how it looks good. So the Phase 3 planning continues for both and prophylactic steroid data, we'll provide some data this year on those patients.
All right, great. Thanks very much.
Thank you. Our next question comes from Chris Raymond of Piper Sandler. Your question please.
Thanks. Also on the DMD program. Emil, there is a hypothesis surrounding gene therapy in DMD where muscles are able to be transfused maybe as efficiently as they would normally because of the damage that's caused by actual disease. I guess -- I'm sure you've looked at this closely, is there a way to address that clinically or do you have any thoughts on that. And then also maybe sort of a question on just execution on the program you have specifically. Can you