Raymond James Says PTC Therapeutics' CHMP Backing Is 'Under Exceptional Circumstances' What's Next

  • Last week, PTC Therapeutics Inc's PTCT Upstaza (PTC-AADC; AAV2-based DDC gene therapy) received a CHMP opinion for approval under exceptional circumstances, says Raymond James. It maintains Outperform rating on the stock.
  • The CHMP recommended Upstaza for AADC deficiency (AADCD) patients aged 18 months and older with a severe phenotype.
  • According to the analysts, with the exceptional circumstances designation, PTCT will be required to provide registry study data to characterize long-term safety/efficacy further (since unable to conduct a placebo-controlled trial with so few patients). 
  • Little color was provided on price point/patient numbers. The management did indicate that they have identified over 300 patients across the globe and anticipate ~$20 million - 40 million in Upstaza revenues in 2022
  • Raymond James believes the AADCD opportunity is relatively modest but views the recommended approval of an AAV-based therapy (especially one directly administered into the brain) as a positive step for the broader gene therapy space.
  • Price Action: PTCT shares are down 2.55% at $31.38 during the market session on the last check Tuesday.
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