Ann: Trofinetide successful in Phase 2 trial in Fragile X, page-93

• "It's perhaps worth noting that the Phase 2 Proof of Concept results for this drug were announced over 4 years ago; Phase 2b results were announced 3 years ago."
  
  One critical thing Neuren has going for it is their Orphan Drug and Fast Track designations.
  
  Therefore contrary Lexicon's case, based on averages we should see a MUCH faster time from Phase II to market for Neuren.

88888acct, read again and you’ll see I bothered mentioning this precisely because Lexicon’s lead drug (to which I was referring in this statement) is also an orphan drug with FDA Fast Track status.

• By way of background, in the case of orphan drugs the time from Phase II to market is significantly shorter due to shorter and smaller clinical trials and FDA Fast Track designation.

Perhaps. EvaluatePharma analysis (2014) found that, contrary to accepted wisdom, there was no statistically significant difference in Phase 3 trial length between orphan and non-orphan drugs, with a median duration of 2.89 years for orphan drugs and 2.86 years for non-orphan drugs. Median FDA approval times were a bit shorter for orphan drugs -10 months for orphan vs. 13 months for non-orphans, due to orphans tending to receive Priority Review. The most significant difference was in cost – much less for orphans than for non-orphans.

• I am in full agreement with you that we should expect a much better ROI than Lexicon.

• Thanks for the example of Applied Genetics Technologies Corp. I’ll do some research and let you know what I find.