Seeing a lot of folks trying to handicap $SRPT “base biz”/exon skipping valuation That “base biz” could potentially evaporate in approx a year when the ESSENCE confirmatory study for Vyondys/Amondys reads out (like all of SRPT drugs, it’ll likely fail as well)

$UCB.BB $MLTX Looks like the FDA has decided they’ll slap a suicidal ideation warning if a drug acts through IL-17F Siliq and Bimi with SIB on the label, whereas Cosentyx (IL-17A) did not despite similar EAIR rate Notes for jefferies and Citi below citing the IL-17F connection

Stock will do what it will do, but this $KRYS Vyjuvek launch is objectively one of the most underwhelming ones I’ve seen, for a disease clearly as severe as DEB is Only 284 *start forms*, ~18 weeks since approval. 121 in q2 and so 163 in q3 by my math . Comp to likes of RETA

I think you're (inadvertently) making a strong bear case for $KRYS. If this is a wax-wane/acute type treatment (presumably due to cost?) then there's severe downside to this launch, since this valuation seems to be pricing in full 52 vials/year for the apparently tiny population

In patients w/ cardiovascular disease & overweight or obesity but no diabetes, semaglutide was superior to placebo in lowering the risk of major adverse cardiovascular events at a mean follow-up of 39.8 mos. Full SELECT trial results: nej.md/465pj91 #AHA23 AHA Science

$MOR fail. Company has been on record saying need both endpoints to hit stat sig for approval. TSS50 p value = 0.216

$MOR - Please no more totality of data [evidence] BS.. It's not in FDA regulatory language - Ha!

Killer Qs from JPM: Are p values adjusted for multiplicity? NO COMMENT Did FDA suggest absolute TSS as an approvable endpoint? NO COMMENT Do you need to wait for PFS & OS? NO COMMENT $MOR

Does anybody actually seriously believe these efficacy scenario bars $EYPT is setting?

Sounds about right. Lower bound for non-inferiority is -4.5 letters. Looking at typical wAMD Ph3 trials below, ~300 patients per arm (KOD recent trial similar n), a 1-1.5 letter difference already gets the lower bound CI to ~4.5+. So if $EYPT shows up in a ph2 with 2 letters

$REPL with the unique achievement of a worse disease control rate (OR + SD) for the RP1 combo vs the control arm. Implications for survival, in case anyone is still holding out hope

Commercially Repugnant Science Project

I was wrong. The 50mg, 100mg BID and 200mg all have HbA1c reductions of 0.4-0.5%. This calls into question the 100mg data, and likely means the drug isn’t doing anything investors.biomeafusion.com/news-releases/…

HaploBMT with posttransplant cyclophosphamide for sickle cell disease. Bombs dropped against gene therapy during late breaking #ASH23 and during QnA. Access and affordability is key!

Historic approval, one and done cure for SCD /s (Note h/t Guggenheim)

$ARGX announces fail in PV BP under review given comparable biology Illustrative note from Cowen 2024 best ideas list gives perspective on sellside model % of Vyvgart peak sales from these indications (~30%+)

$KRYS citizen petition to prevent Chiesi's Filsuvez approval denied. Filsuvez approved yday with broad label for DEB and JEB, no HCP required. Will be interesting to see how this tiny market gets split between Chiesi $KRYS and potentially $ABEO