To provide some very rough guidance as to possible Phase 3 trial size and cost –
The Phase 3 trial for trofinetide in Rett syndrome had 187 participants.
Soleno’s first Phase 3 trial in PWS had 127 participants.
Acadia’s Phase 3 trial in PWS is enrolling 170 participants.
Harmony Bioscience’s Phase 3 trial in PWS is enrolling 134 participants.
Ultragenyx’s Phase 3 trial in Angelman syndrome will enrol 120 participants.
Note that all these Phase 3 trials are placebo-controlled.
Acadia is currently spending ~US$7m per qtr on its PWS trial and the trial is estimated to run for 10 quarters. Perhaps nudging US$90-100m all up?
Harmony Biosciences is spending US$7-8m per qtr on its PWS trial and is estimated to run for 9 quarters. Possibly similar cost to Acadia to see this through?
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