Theratechnologies Inc T.TH

RE:RE:RE:RE:RE:Lowering expectations on efficacy???

[qwerty22]

Wino is right one indication or two doesn't make a huge difference. I'm not sure a large Pharma would do things much different. The really different path is if the program went indication agnostic with the trial. You can talk about SORT1 as agnostic in the hypothetical but in practical terms it becomes more complicated to implement.To justify that you'd be either be looking at a very large trials to prove the agnostic approach was valid or have worked up a validate companion diagnostic to focus in on sort+ responders IMO. That's less practical as a first approval for anybody to do. I'm not concerned about THTX's capabilities, I think the practical program has to go after focused goals and if they workout then expand to wider applications. In my view that would be the approach of anybody who owned this.

SPCEO1 wrote: If the phase 1b trial shows efficacy in several tumor types, it is hard for me to see how TH stays independent as they would likely quickly attract some offers they could not refuse. I ahve to wonder if the FDA in situations like this, where the cancer fighting implications could be enormous, tells several big pharma companies on the sly they would prefer if they took the research out of the hands of the tiny company and into their own as it is just too important to entrust to a smaller company. I wonder if that is what happened to IMMO?
 

Wino115 wrote: I'm an optimist too, but we're all just speculating. If they see things like what they saw in pre-clinical and draw comparisons, I think that would get loads of drool on the street.  In other company trials, even just the anecdotal on a few patients, if very strong, excites investors.  So guess we'll just see. Anything showing POC I think would be validating and exciting news.  The more, the better, but I think the market would take any signal positively and finally get SORT1 platform on the radar for new investors.  Frankly, it would be pretty eye opening just to say our PDC was safe when we delivered 4x the normal docetaxel dose into solid tumors across 4-5 cancer types.  That's pretty astounding news in and of itself when you think of the implications those that know oncology would surmise.

It was confusing to me on how they would move forward after the basket trial too.  At first he emphasized the fast track was predicated on sort1 expression being wide in many tumors, so was "tumor agnostic".  Then he said was very specific in saying they would look at the basket trial results and if they see high efficacy in one, they'll pick that "for accelerated development".  I take that to mean they will look to take advantage of the benefits of fast-track for one they see the highest probabaility of success on to move the whole platform into commercialization as fast as possible.  But I don't see that as them stopping from continuing to develop indications for the other tumors. Given the relatively low cost of Phase 2 cancer trials, I've seen companies similar in size do multiple tumor trials.  I can't see them stopping those indications, just that they will either do them at normal approval pace or push to accelerate after the first indication. 

From past posts it was told to us that you can't actually get a drug registered for something broad like "all solid tumors overexpressing sortilin receptor" so they'll have to eventually do trials for each indication.  But he didn't rule out that they could do more than one type of tumor in Phase 2.  I'm thinking if they see a few with very high efficacy, they may accelerate those together with multiple trials.  

qwerty22 wrote:

What you are saying here is right, and I know it's right, but I'm hoping for more. I guess I'm seeing his words indicating that they will get their PoC but not much more than that. But the optimist in me was hoping for something extra that went a little beyond that. I guess I'm not seeing anything extra being represented in his words. So yes things are on track for part2 but I'm not seeing a release to Wall St that gets people drooling.

The other thing I thought sounded a little limited was the couple of times he talked about moving forward with one indication. So that's totally my expectation and always has been. Not knowing the data it's reasonable to expect the path forward to be just in the strongest indication. But if I was sitting on data that showed shrinking tumours in more than one indication I might say that but quickly pivot to the wider applicability of SORT1 tech. I guess I'm picking up limited expectations there also.

Here's how I heard things in cancer. When he was talking more speculatively about things off in the more distant future he was effusive, things like China deals and dosing and combo trials. When he was down to more immediate practical things like PRs to Wall St or were the ongoing trial might go then expectations were much more limited. It all just suggests the data is on track to deliver PoC but not over-delivering anything else which might just be the reality of it being a 1a trial.
 

 

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.