Ann: Trofinetide successful in Phase 2 trial in Fragile X, page-92

hottod,

Nice analysis, and it highlights that finding a "perfect fit" peer is a difficult (to near impossible).

You said:

"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.

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.

In fact, the average time from Phase II to launch for orphan drugs is 3.9 years, compared with 5.4 years for non-orphan drugs. This period is potentially significantly shortened further due to Fast Track designation. More so, once a compound has been granted orphan designation, the odds for approval are high (82%) compared to traditional drugs (35%).

You said:

"However, it is probably also worth noting that it took the company 20 years from founding, 15 years from listing to get to this point!"

If you use the same lens on Neuren you would note 10 years from the January 2005 listing to today!  I am agreeable that the total raised by Lexicon is signifcant however as you point out with multiple molecules they've adopted portfolio theory to land on their two seemingly valuable ones, compared to Neuren's more "all-in" approach which if NNZ-2566 and NNZ2591 are successful then we should expect a better ROI (input d$ wise).

Lastly, here is another potential peer for you to run -- Applied Genetic Technologies Corp. A handful of products at the phase 1 and 2 stage for three orphan diseases - Alpha-1 Antitrypsin Deficiency (Alpha 1), X-linked Retinoschisis (XLRS) and Achromatopsia (ACHM). Market cap US$308m or A$440m equating to a NEU equivalent share price of A$0.26.  I recognise that this will again highlight my firtst point i.e. the difficulty of finding a perfect fit peer however I trust my analysis yesterday covers additional points (http://hotcopper.com.au/threads/ann...in-fragile-x.2656328/page-88?post_id=16604327) as to why I believe we should be trading at the mid to high point relative to Neuren's developmental-stage peers.

Cheers,