@A_May_MD@SylvainGariel The panic really started when the dunce KOL on $ABVX call said the cancer number and mix was not a problem for him, but maybe the FDA would call an adcom because the MoA was new. I think he felt he sounded scholarly and objective. He was not a stock market wiz for sure.
@a_a_free nice list. Feel $FBRX unlikely to ‘fail’, per se, in vitiligo. It’s signal-seeking study and much shorter than TEVA’, but adjusting for time its potential should become clear. Mgmt talking down trial is one reason for price volatility, but could be a case of over managing street
@Biohazard3737 long believer in “the trip is the treatment” for psychedelics at least of the 5-ht2a axis (NMDA?). Can’t imagine roll back of centuries of writing. But equally interested to understand if the hallucinations can be dialed out significantly (eg Glilgamesh). https://t.co/P5GuiEkqi2
@AppleHelix Calypo’s MAbs 2017 paper was for me a starting point into the IL15 space. Very good work especially after AMGN 714 development almost derailed the field
https://t.co/hOJqVMHSLi
@A_May_MD@oakazaki There’s a creeping narrative, at least for $ORKA and $SYRE, that by delivering higher, prolonged, drug exposures they may deliver greater efficacy -that ABBV and TAK left “efficacy on the table”. There are some stray empirical findings of this but the mechanism is not clear.
@AB1001_disciple@peter_mantas yea but value peace of mind. But on this specific point the Roche HD trial suggests pbo response vanish by year 2, so longer natural history needs regulatory deliberation, while acknowledging residual bias (if one believes in flexibility; + nfl and high vs low dose findings)
@AB1001_disciple@peter_mantas Probably because many glad to see this crazy period over, and tired. Arguments about evidentiary standards always good, but institutions can’t survive ad hoc personality-driven process, ill-defined regulatory frameworks, and poor leadership
@BiotechAnalysst Whoever this official it would make sense for the FDA to review the entire AMT-130 program before arriving at such a judgement (not the top line 1-yr CT that Makary blanketedly judged). Would then be more palatable and less arbitrary.
$QURE
@BiotechObserver all that we have been seeing -across rare diseases including amt130 & bitopertin recently- isn’t random noise, its a clear articulation of views that the AA pathway itself, as it stands, is not very valid (although an argument that its way overused can have merit)
@BiotechObserver $QURE for me is (yet) another example of technically defensible FDA positions but misses the philosophy of AA and reg flexibility. Demanding something like the 1-yr control to be met is a circular evidentiary standard that makes AA structurally impossible for many rare diseases
@BrianSkorney@BentheFidler@RxRegA@peterbachmd abs. It’s ad hoc regulatory science unmoored from policy and philosophy, whatever the individual decisions. Then a FDA commissioner points to disgruntled investors without regard to any coherent policy around how or why (or not) investment might play a role in shaping innovation.
@Biohazard3737 It’s been impossible to get my tech investor guys understand the biology bottleneck for AI in drug discovery. They see the world in software terms with “scaled” emergent solution that will ‘disrupt’ the industry. Last couple of weeks though they’re running in for bios to buy.
@Biohazard3737 A couple of other Abs and contracts with Novartis and Amgen, but GB is still stuck protein engineering stage, with ‘me-too betters’. My guys on tech side keep lecturing me on AI-biotech disruption (words like “singularities and non linear” abound); and Gen B is my fave rebuttal
@Prof_Oak_ yea but the $RAPT slides at JPM were pretty good. It included a highly dense page on the commercial opportunity. At the time I wondered why show such a slide, despite the solid accompanying narrative - but, of course, they had made it earlier for GSK!
@doepke_michel been great to see $IONS strategy maturing. If there’s any major hiccup on ‘horizon’ it’s the Lp(A) outcomes in 1H. Real chance it doesn’t cross 15% RR in pop >70mg/dl with improving standard of care/time delay. Strong believer in Lpa but trial exigencies may not fully capture it?
@LaminarRed follow up to where above base case can be wrong - the defn of materiality in this context. This is beyond my scope but it might well be that unless they're an actual offer on table a clear nmpi window exists for Sofinivva to file a 144. Anyone with a view?
@LaminarRed my 2-bits: if LLY did meet French authorities that process is dead. Now, there cannot be any active process (eg BD teams in data room), giving a nmpi window for the VC to file 144. Therefore no deal in near term; $ABVX action postponed to q2 data