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PRTO:
PRTO:
PRTO:
Re: treatment of RCF vs BCF
PRTO:
ABIO / Blade:
Bristow is a legit name in CV. The bucindolol program may have a chance, but I'm generally negative on these subgroup-driven trials.
Also keep in mind that unlike other beta-blockers, bucindolol failed to improve survival versus placebo. This may be reflective of the patient populations they studied, or of the fact that bucindolol is a slightly weaker blocker than the others.
Nonetheless, this type of thing isn't my cup of tea.
TRIL:
TRIL:
TRIL:
TRIL:
Blade:
Blade, re: CYAD:
I think the cardio program is nuclear winter. It was garbage from the start, and will continue to fail as long as they pursue it.
I've no opinion on their NK program. However, do note I'm rather certain they picked this up as a back-up program due to low or zero confidence in their cardio program. In that regard, it's not clear to me that there is any expertise in-house to develop and oncology product.
SRPT:
MYOK:
Yes, I've read that article.
MYOK:
MYOK:
MYOK:
ZIOP:
MYOK:
ZIOP:
On/Off/Rheostat:
I've absolutely no doubt you can do it in preclinical.
But i think the ability to control the in vivo setting in the preclinical model is key. In the clinical setting, the variables increase and so does the complexity.
Disease burden, amount of CAR-T that is manufactured and injected, and the persistence of the CAR-T in the circulation all become variables that will have to be juggled.
Great that people are working on it, but at this stage I don't see it being a meaningful differentiator for one company over another.
I guess I'm just a curmudgeon on this one.
CAR-T on/off
CAR-T / Off and On:
ZIOP:
If you're talking about Ad-RTS-hIL-12 specifically, perhaps it is not coming up because it's not a CAR-T approach?
CAR-T etc...
MYOK:
They're basically doing a version of what CYTK did, but this time they're trying to lower work done by the heart (which I prefer) rather than increase it (as CYTK did).
Worrying thing is that there is lots of overlap between the CYTK and MYOK people, so perhaps some intellectual recycling is going on. The freshest perspective is likely the Seidmans who accrue much of the genetic background data for the inherited cardiomyopathies. Problem with these cardiomyopathies is that many of them impact proteins that don't have catalytic activity, and are therefore not readily drug-able.
I'm interested in this field but see no reason personally to dive into MYOK right now.
SRPT:
Pretty amazing that it has come to this. Here is a drug that ostensibly produces less than 0.9% of normal dystrophin, in a manner that is statistically significant only if you set some of the baseline values at 0 rather than the lower limit of detection.
The fact that we're even discussing these data as part of the basis for approval is a testament to the lobbying efforts of the families.
With this upcoming analysis, I think they're supposed to be analyzing before and after samples from the same participant. If they do a paired analysis, minuscule "increases" in dystrophin are likely to be stat sig.
SRPT:
I'm thinking this drug gets approved. Too many concessions are being made by the FDA.
- Company runs trial that is clearly not sufficient for AA.
- Company doesn't have a complete sample set from said trial and therefore can't do a proper analysis of their surrogate
- To make case for AA, company dips into samples from an ongoing trial to serve as controls for the completed trial (also acknowledging, at the time, that 3 patient samples from ongoing trial had already been practically used up and therefore unavailable for analysis)
- Now company advised to provide surrogate analysis from 13 before / after samples from the currently running trial in order to satisfy AA
Basically the FDA was not able to conclude anything from the studies submitted for AA... yet the door is still open for approval.
From a biotech investment standpoint, hopefully investors don't take the wrong lesson. This isn't how the drug approval process works, and if they bet on similar treatment for other companies, their SRPT profits will disappear pretty quickly.
PI3K:
TGTX:
I don't have too much of an opinion on the drug insofar as efficacy. However, the rallying cry behind this drug (on twitter) is centered on its putative improved AE profile versus competitors. Although this may be true (i think the data can be debated), i know at least one competitor who says they've identified the area of PI3K targeting drugs they believe is responsible for the transaminase elevations seen in the class. Preliminary clinical data from this new candidate drug appears to support their claim.
Therefore, I don't think the AE profile of the TGTX drug remains a unique advantage for them. In that regard, their lead in development time may be the one remaining advantage. We will see how well they execute.
TRVN:
Sad to see this. I had expressed doubts about it due to the subtlety of the proposed mechanism, but would have liked to see a hint or two of efficacy.
What is the next milestone for the pain drug?
Apologies! I'm still sensitive about TELK I guess... eom.
SRPT:
SRPT: