There's every possibility Steve could be aiming for an Accelerated trial for PTX-100. Thanks for positng.
Steve has only made reference to the co. pursuing a Registrational trial, though. There is a difference between the two (both highly advantageous) but from what I have read, there is no reason why Phase 2 trials can't achieve both Accelerated and Registrational status.
He has also recently referenced a "go or no go" scenario which allows early decision-making whether to continue a trial based on early efficacy results. That is scenario is something I came across in context of Acellerated Approval where surrogate endpoints can mitigate spending heaps of dollars on trials that don't succeed. The use of surrogate enpoints is becoming more and more commonplace. Biomarkers are becoming great tools in expediting clinical trials.
With the safety results, the ORR and CRR in the Phase 1b trial being what they are, and coupled with Orphan Drug designation, it would be hard to think that Steve wouldn't be shooting for the moon. He knows that his job is on the line too. For that reason alone, he'd be working his toosh off!
We have likely notched up more than enough proof of clinical activity and duration of response in patients with CTCL to progress it to Phase 2 (under either or both pathways). My take is that Key Opinion Leaders (and PTX) are keen to see if similar robust data for PTCL can be gathered. The only way we can do that is to get Phase 2 underway with strong recruitment of patients with PTCL.
I suspect that AI will feature prominently in our trial designs and various aspects including in recruitment (whether PTX is building its own IP around this is another matter) but is bound to be a significant piece of the puzzle we are not privy to yet.
PTX Price at posting:
4.2¢ Sentiment: Buy Disclosure: Held
