Wino115 wrote: Are you saying that because he reemphasized that 1a is focused on MTD primarily? I think he's probably right to do this and we know given it's all-comers, last option patients, relatively short treatment period, small group, etc... that it's not a significant cohort for capturing true efficacy. I think we've all only hoped for a few signs that may back up some of the pre-clinical work seen. If you just see that tad in combination with showing very high safety profile, then you are in very good shape going in to the extension portion. So I'm not sure he's really saying anything other than the 1a mantra and what we've come to expect. Maybe not an n=1, but not too far off that is my guess. It will be an anecdote(s), but still a very useful one given the MOA and what is understood from the clinic. It would be a great foundation for going in to 1b and pushing for the acceleration with FDA.
I picked up a few new tidbits.
1. Definietly laid out their thinking more strongly on Phase 2. Definitely will be picking one cancer to focus on where efficacy is highest and moving as quickly as possible believing they'll get accellerated approval status on that one. The others will move along, but one will go warp speed. This is good and would put at least one indication along that Trodelvy path of moving very, very quickly into Phase 2 and maybe not even the need for a full Phase 3 like IMMU did.
2. Partner in China (very specifically) for cancer. Why so specific and we hadn't heard that before. Perhaps one of the "alternative" scenarios for NASH. Get a large China pharma on board for distrubution partner there based off the Phase 1 data and an upfront payment helps move NASH program in to gear. You basically have to partner for China distrubution.
3. Exploring immunology in lab and diagnostic testing for SORT1. We sort of knew that but he spoke in the present tense about immunology so sounds like they're working on that already in the lab. Weekly dosing seems to be recurring theme; leads one to believeTo be expected.
4. Specifically mentioned "companies involved in cardio-vascular" as partners for NASH. Not sure if he meant that or was a slip of the tongue. Also, needing a sales force in US and Europe for NASH - so once again, the type of partner that may pay upfront just for distrubution agreement and let THTX do the development. Clearly a wide net being cast by their deal bankers.
5. Market size -- keeping expectations realistic in saying "tens of thousands", but I think he was saying that in regard to whatever it is they choose for that first, accelerated approval they would go for, not the overall size because he also mentioned adding other payloads and the 5 cancers in extension trial. Not super clear answer and somethiing they need to refine going forward.
Questions from Cantor were good. Hopefully they pick them up once we get data going. All in, informative and PL tryinig to get a bit of excitement going around things while being factual so it doesn't come back to bite him. The THTX way. I'm ok with that given we're only 8-10 weeks from knowing what's really going on with dosage portion.
qwerty22 wrote: Not strong.
Also. He's clearly not prep'd to talk on market opportunity.