- Ultragenyx Pharmaceutical Inc RARE and GeneTx Biotherapeutics LLC announced interim data from the Phase 1/2 study in pediatric patients with a genetically confirmed diagnosis of full maternal UBE3A gene deletion.
- Interim results demonstrate a meaningful improvement in clinical disease and an acceptable safety profile.
- 7/9 patients showed improvement on the disease scale from Baseline in at least 3 of 5 domains and the overall score.
- Six patients exhibited a decrease in severity in at least 2 of 5 domains and the overall score.
- Also See: Ultragenyx Sells One-Third Of Future North American Crysvita Royalties For $500M.
- There have been no treatment-related serious adverse events of any type nor adverse events related to lower extremity weakness observed in these patients.
- Cerebrospinal fluid (CSF) protein levels have remained stable.
- These interim data support a protocol amendment to the Phase 1/2 study approved in the U.K. and Canada to initiate additional new cohorts of patients at higher monthly loading doses.
- The study has begun enrollment and has dosed the first patient in these new cohorts.
- Ultragenyx also announced it has exercised its option to acquire GeneTx and has closed on the acquisition for an upfront payment of $75.0 million plus future milestone and royalty payments.
- Price Action: RARE shares closed 5.76% lower at $61.02 on Monday.
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