Ultragenyx DTX301 Gene Therapy Update

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Update on DTX301 Gene Therapy Clinical Study for Ornithine Transcarbamylase (OTC) Deficiency

Nine people with OTC deficiency are enrolled & have received gene therapy at 3 different dose levels in the Ultragenyx DTX301 study to date. Another group of patients has been identified for participation in the study, but due to the COVID-19 pandemic and the closure of the institutions, they are waiting to receive the dose of gene therapy. We expect results from this group in the second half of 2020, unless there are further delays related to COVID-19. Ultragenyx is also planning the Phase 3 gene therapy study, which is expected to begin enrolling pediatric and adult patients with OTC in the first half of 2021.

In addition, Ultragenyx is planning to start an observational study (no treatment administered) with ~30 people who are 18 years of age & older with neonatal, late onset OTC or asymptomatic carriers. This study will last 2 years, with a total of 5 study visits, one every 6 months at 10 clinics across the USA. The study will collect information on rate of ureagenesis (how well each person’s urea cycle works) along with neurocognitive tests, patient-reported questionnaires and medical history.

Lastly, Ultragenyx is working with Endpoints Outcome who is interviewing individuals with OTC 18 years and older, as well as caregivers of & individuals with OTC between the ages of 8-17, to discuss their experiences. The interview will be approximately 90 minutes long and will be conducted by telephone or video with audio recording. Eligible participants 18 years or older will be paid $150 and eligible participants ages 8 to 17 will be paid $75 at the end of the interview. Email OTC@endpointoutcomes.com to participate.

Please contact patientadvocacy@ultragenyx.com with any questions.

MRCM-DTX301-00033
June 2020

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