Ann: Trofinetide successful in Phase 2 trial in Fragile X, page-95

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Re length of Phase 3 trials, you suggested that orphan drugs got to market sooner because their clinical trials were shorter

…the time from Phase II to market is significantly shorter due to shorter and smaller clinical trials.

According to Evaluate Pharm, with respect to Phase 3 trials, that’s not so. Why the seeming discrepancy? I’m not sure, although I note that Evaluate Pharma’s analysis is based on more recent data than the source you provided.

I do agree with you that the odds for approval are higher for orphan drugs than for non-orphan drugs.

Finally, re Applied Genetics Technologies Corp:

Applied Genetics Technologies (AGT) has a current market cap of US$291million – a bit over AU$400 million, so roughly double Neuren’s market cap. Its pipeline is not as deep as NEU’s, but it is broader.

At first glance it would appear that Neuren is way undervalued when compared with AGT.
However, it should be noted that AGT has total current assets of US$173million (AU$241m) including US$140 million (AU$195 million) in cash v. Neuren’s AU$18 million approx.

Also, as well as the value of its products in development, AGT has value in its proprietary viral vector gene delivery platform and GMP-compliant, commercial-scale, vector-generating platform.

Perhaps most importantly, AGT has a licensing deal in place with Biogen, announced earlier this year. The headline value was $1+ billion and Biogen paid an upfront of $124 million. According to AGT, under the terms of this deal, Biogen will pay for substantially all of the related program development costs.

With all this taken into account, it’s not so clear to me that Neuren is way undervalued compared with AGT.