RE:RE:My take so far...I think Ionis is focused elsewhere and has a very lengthy pipeline and 5 existing drugs. Doesn't seem likely but who knows?
On these results, decent but not powered enough to reach any conclusions - just more data to add. It does look like they understand the safety issues better with lower dosage more frequently being tolerable. Now, how high can you go is the open question. Second is that the actual delivery mechanism and the secondary and tertiary MOAs seem to be showing in the marker analysis, which is good. In other words, their theory around how the toxin gets in and what it does was not counter-acted --it appears to be doing some of that around the stem cells and vasculogenic mimicry.
They really needed more time to give more doses, this was rather short. Needed more patients, etc...
I seem to recall they had the option to add 4-5 more people in the last leg and had the money for that budgeted. Is that what they're saying around doing slightly higher dosage with another small group of 4-5? Hard to really tell form the wording, or is that what they will seek initial financing and. partnering for --something small like that, get to know the partner, then depending on results, move to a proper Phase 2 to get efficacy optimized. This is novel --first of it's kind PDC, unique biomarker, lots of data around what it's doing. I have to think someone will want to try to see if they can boost efficacy. It may not be one of the big drug companies, and it may even be one of the larger overseas names who has an ADC franchise or oncology. The announcement of a partner will be the big even and hopefully they find one or find a financing partner - either one. They've moved the ball a fair way down the field and have a ton of data now. I would like to see them collate all this, publish it, do another 5 patients with the higher dose (if FDA allows) and add that to the mix. It's also too bad they couldn't keep those patients on dosage for another few weeks and see if they shrink >30% (Maybe they are?). But the point made above, that these are patients near death that tried everything and it failed, yet this has shown SD with some better results, is still a very good start for a new oncology drug.