• 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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