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Joined August 2009
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$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
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 Skyclarys which had 500 start forms BEFORE the drug was approved. Arguably a more questionable efficacy profile with skyckarys notwithstanding. Or ACAD Daybue, which has 800 patients ON the drug already, approved only ~1 month before KRYS Vyjuvek
Can’t imagine any parent won’t be rushing to fill out a simple ~2 minute start form while their kid’s skin is practically melting off and how unfortunately tortuous DEB is. Is the population smaller than what was imagined or is there something else going on?
ir.krystalbio.com/news-relea…
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Chiesi mamaged to get $AMYT's Filsuvez approved in the US in 2 types of EB
Chiesi Global Rare Diseases Receives FDA Approval for FILSUVEZ (birch triterpenes) topical gel for the Treatment of Epidermolysis Bullosa globenewswire.com/news-relea…
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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!
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/n…
Digging into the $BMEA data. On first pass I agree with the general view coming out of ADA that the data are quite remarkable, with the durability of a short course of therapy and off-treatment effect pretty much unheard of for the commercial diabetes drugs, which of course are monster commercial blockbusters. Obviously early and I’ll have more once I dig further, but there’s not too many new, potentially disease modifying smid plays in diabetes.
Any credible critiques of the data, now that the old bear concerns (safety, durability, rbc and Hb effects) have been quietened? Genuinely curious.
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 or greater difference then logic would dictate they'd have to run massive ph3s to hit non-inferiority. Not to mention the typical degradation of effect from ph2 to ph3.
Not sure how they are characterizing -1.5 to -2.9 letters as a "great" outcome given this fact pattern which argues the exact opposite
This quoted post is unavailable.
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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
$MOR
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$MOR - Please no more totality of data [evidence] BS.. It's not in FDA regulatory language - Ha!
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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 @AHAScience
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
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 Skyclarys which had 500 start forms BEFORE the drug was approved. Arguably a more questionable efficacy profile with skyckarys notwithstanding. Or ACAD Daybue, which has 800 patients ON the drug already, approved only ~1 month before KRYS Vyjuvek
Can’t imagine any parent won’t be rushing to fill out a simple ~2 minute start form while their kid’s skin is practically melting off and how unfortunately tortuous DEB is. Is the population smaller than what was imagined or is there something else going on?
ir.krystalbio.com/news-relea…
$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 and how the warning will impact usage
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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)
classic.clinicaltrials.gov/c…
Of course, this is SRPT so it’s a valid question if they’ll actually ever announce the results