Ultragenyx Pharmaceutical Inc at Goldman Sachs Global Healthcare Conference Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Salveen Richter - Goldman Sachs - Analyst : Emil, perhaps we can start here by just outlining how you think about the strategy for the company on the forward and what we may see from the pipeline over the next 12 months?

Question: Salveen Richter - Goldman Sachs - Analyst : As you mentioned, there's a lot of interest in setrusumab for OI. Help us understand or contextualize the Phase 2 results and -- that we've seen to date and how to think about the read-through to the Phase 3 program.

Question: Salveen Richter - Goldman Sachs - Analyst : Can you just speak to that with regard to the Phase 3 study design, the powering assumptions and your expectations around the two interim analysis, the probability you assigned to it kind of hitting on that first or second one?

Question: Salveen Richter - Goldman Sachs - Analyst : You spoke to how there could be factors just because of how active these individuals become. How does the FDA think about that in the context of quality of life? Because that has to have some value here?

Question: Salveen Richter - Goldman Sachs - Analyst : Could you speak to the launch strategy here or how you think about commercial footprint, recognizing that you have an existing sales force behind Crysvita?

Question: Salveen Richter - Goldman Sachs - Analyst : And how large is this commercial opportunity in your territories?

Question: Salveen Richter - Goldman Sachs - Analyst : Perfect. Let's switch over to Angelman syndrome. You recently shared a data update and initial regulatory updates. How does this data inform your view of efficacy and the next steps here?

Question: Salveen Richter - Goldman Sachs - Analyst : Could you touch on the adverse events that occurred and prior to the protocol amendments and how concerning or not concerning they may be?

Question: Salveen Richter - Goldman Sachs - Analyst : And at your midyear end of Phase 2 meeting with the FDA, what are the items you plan to get on -- gain alignment on? And what would be a best-case outcome for you? And a likely outcome?

Question: Salveen Richter - Goldman Sachs - Analyst : How hard will it be to manage patient variability just given the heterogeneity of this population within a placebo-controlled trial?

Question: Salveen Richter - Goldman Sachs - Analyst : And next here on the gene therapy in Wilson's disease. Can you frame expectations for the Stage 1 data expected in the second half and how you're thinking of validating the efficacy profile.

Question: Salveen Richter - Goldman Sachs - Analyst : And at the same time, you have the GSDI and OTC gene therapy trials progressing here. One, which is heading to a regulatory filing.

Question: Salveen Richter - Goldman Sachs - Analyst : Just a last question here on how you think about business development, but also how you're thinking about the cash runway, recognizing you will likely get (technical difficulty).

Question: Salveen Richter - Goldman Sachs - Analyst : Great. Well, with that, thank you so much, Emil.