Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q3 2020 Earnings Conference Call - Final Transcript

Oct 27, 2020 • 05:00 pm ET

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Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q3 2020 Earnings Conference Call - Final Transcript

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Presentation
Executive
Emil D. Kakkis

now the Angelman program and the business development team brought in a new gene therapy program for Duchenne on top of the gene therapy technology partnership announced with Daiichi Sankyo in March. We've been firing on all cylinders and will provide updates on all of this progress on the call today.

Starting with our commercial programs which provide a stable source of growth underpins the rest of our development. Crysvita continues to perform strongly and is now bolstered by the additional tumor-induced osteomalacia indication. In the first quarter of launch, Dojolvi is off to a strong start in its first quarter, which is encouraging. We received start-forms from a large number of prescribers for patients in our trials, but also those naive to Dojolvi. This speaks to the unmet need for those living with LC-FAOD and to the strong relationships that we have with physicians and the major treatment centers.

Moving to our strategic collaboration with Solid Biosciences. Last week, we announced that we've been developing a new gene therapy for the treatment of Duchenne Muscular Dystrophy or DMD. While there are other Duchenne programs that are far more advanced, this was an unique opportunity to combine Solid's technology with our technology and two like-minded companies to create a next-generation gene therapy for this disease. We will combine Solid's best-in-class microdystrophin with its potential ability to enhance blood flow to working muscles with our novel capsid which has an excellent immune profile and is produced by high-quality 2000 liter HeLa cell -- producer cell manufacturing process. We believe this could be a differentiated therapy for Duchenne that we will seek to achieve global accessibility. We look forward to collaborating with Solid to bring a new treatment forward rapidly and we'll continue to update you on the progress coming out of the collaboration in the coming quarters.

Moving to Angelman. Yesterday, we announced positive interim data from the ongoing Phase 1/2 study of the investigational product GTX-102 in Angelman syndrome, which is a part of the collaboration with GeneTx Biotherapeutics. GTX-102 is the first antisense oligonucleotide program for Angelman to reach the clinic and is based on the extraordinary science from Dr. Scott Dindot supported by GeneTx. In our update, all five patients of the first patients treated in the study have shown substantial improvements in the Clinical Global Impression scale or CGI tailored for some of the key domains of Angelman with a mean change score of 2.4. The positive clinical effects were supported by other endpoints and remarkable reports of change is provided by the caregivers of patients treated with GTX-102. These include two patients going from being non-verbal their entire lives with difficulty communicating to now using multiple words, while others are beginning to use sign, gestures, and augmentative communication devices for the first time. Some patients adopted independent capabilities such as using a fork to feed themselves for the first time. Others are learning to swim on their own and other reports includes ability to follow commands,