Ultragenyx Resumes Dosing Antisense Trial In Rare Neurogenetic Disorder

  • The FDA has removed the clinical hold on GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc's RARE GTX-102 trial for Angelman syndrome.
  • The companies can begin dosing naïve patients in the Phase 1/2 study of GTX-102 in pediatric patients. 
  • Ultragenyx paused dosing after patients temporarily lost the ability to walk. All subjects had lower extremity weakness after receiving GTX-102.
  • Under the amended U.S. protocol, eight patients (4 to less than 8 years of age) who were not previously treated with GTX-102 will be enrolled into two groups, an active group, and an age-matched comparator group. 
  • The active group will receive four monthly 2 mg of GTX-102, while the comparator group will have limited assessments at baseline and Day 128. 
  • Patients in the comparator group will then be eligible to receive GTX-102 under the same dosing strategy as the active group.
  • All U.S. patients who have completed the dose-loading phase will then move to a maintenance phase during which they will receive 2 mg of GTX-102 every three months and continue to be monitored for response and safety.
  • GeneTx previously received clearance, under a separate amendment, to begin the Phase 1/2 study in the U.K. and Canada.
  • Under the protocol approved in the U.K. and Canada, approximately 12 patients will be enrolled into two cohorts split by age.
  • The starting doses in Cohorts 4 and 5 will be 3.3 and 5 mg, respectively. Patients will receive three to four monthly doses and will then move to a maintenance phase.
  • Related content: Benzinga's Full FDA Calendar.
  • Price Action: RARE stock is down 2.29% at $92.93 during the market session on the last check Monday.
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Posted In: BiotechNewsHealth CareFDAMoversTrading IdeasGeneralAngelman syndromeBriefsPhase 1 TrialPhase 2 Trial
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