- Ultragenyx Pharmaceutical (NASDAQ:RARE) is unchanged premarket in response to its announcement of positive preliminary data from a Phase 1/2 clinical trial evaluating gene therapy DTX301 in patients with ornithine transcarbamylase (OTC) deficiency, an inherited disorder characterized by the accumulation of ammonia in the body. OTC, an enzyme, plays a key role in the urea cycle (breakdown and removal of nitrogen).
- The company says patient #1 in the lowest-dose cohort has experienced increased levels of ureagenesis (formation of urea) through 24 weeks, has discontinued other medications and continues to do well.
- The other two patients in the cohort, followed for 20 weeks and 12 weeks, respectively, have not shown clinically meaningful changes in urea formulation rate.
- On the safety front, no infusion-related adverse events and no serious adverse events have been reported. Two patients experienced mild elevations in an enzyme called alanine aminotransferase (ALT), a biomarker for liver damage, that were effectively managed with corticosteroids.
- The Data Monitoring Committee has signed off on the advancement to a second higher-dose cohort. Top-line data should be available in H2.
- Previously: Ultragenyx completes dosing in first cohort of Phase 1/2 study of DTX301 in OTC deficiency (Nov. 16, 2017)
- Now read: Ultragenyx Pharmaceutical: Buy The Offering
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