Occam's Toothbrush

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Occam's Toothbrush

Occam's Toothbrush

@occams_tooth

Biotech Operator | Not investment advice

Katılım Kasım 2024
310 Takip Edilen482 Takipçiler
Occam's Toothbrush
Occam's Toothbrush@occams_tooth·
@cremieuxrecueil Don’t have an issue with the target, but why are you doing AAV gene therapy for this? Seems like an awful idea given the safety track record of viral vectors
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Crémieux
Crémieux@cremieuxrecueil·
I'm proud to announce a trial! And YOU -- yes, you! -- might be able to get in it. At the end of this thread, you'll find a link to sign up. The trial is for a gene therapy for HUGE MUSCLES🧵 (like this cow)
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Occam's Toothbrush
Occam's Toothbrush@occams_tooth·
Crazy that $TAK has a market cap of $50B, about 20% LESS than what they paid to acquire Shire in 2019. CEO has a tough job bringing them back to growth. Sad to see layoffs but given their performance over the last few years, tough choices are probably warranted
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Occam's Toothbrush
Occam's Toothbrush@occams_tooth·
@avidresearch My guess is they will wait for clinical data from their AATD program. They announced FDA IND clearance for that program in Jan. So, probably 2H 2026
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Samuel Hume
Samuel Hume@DrSamuelBHume·
The number of public biotech companies has been dropping since 2022 (with a peak of almost 900) -- now it looks like it's now stabilizing, at ~667
Samuel Hume tweet media
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Occam's Toothbrush
Occam's Toothbrush@occams_tooth·
Good NYT article on the ALS drug Qalsody from $BIIB and $IONS , and example of accelerated approval done right: At initial approval, results had not shown it worked better than a placebo. Approval was based on biomarker; functional data now compelling nytimes.com/2026/05/06/wel…
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Occam's Toothbrush
Occam's Toothbrush@occams_tooth·
In some ways, $GENB is a Fairmount style play from Flagship. Lead asset is a half-life extended TSLP with 2x yearly dosing vs monthly for SOC. But mkt cap less than half of similar Fairmount cos ( $APGE $SYRE ) Is this a Flagship v Fairmount premium or related to the target?
Occam's Toothbrush tweet media
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Occam's Toothbrush
Occam's Toothbrush@occams_tooth·
$ARWR BD continues to deliver. Second time this particular asset has been partnered. Was in a $JNJ collab; returned in 2023 and now re-partnered for additional economics with $MDGL
ArrowheadPharma@ArrowheadPharma

We’ve entered a strategic partnership with @MadrigalPharma to advance an RNAi therapeutic for MASH, combining the precision of Arrowhead’s TRiM™ platform with Madrigal’s liver disease expertise. Read the full announcement: bit.ly/42SoWza

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Occam's Toothbrush
Occam's Toothbrush@occams_tooth·
One of the better takes on biotwitter. Sad to see so much investment going to incremental innovation for clinically validated targets. I don’t know $ORKA well, and think some Fairmount plays (eg $APGE) may show clinical differentiation, but agree with the broader sentiment.
Adam May@A_May_MD

Well over a billion dollars raised for $ORKA’s decade+ late psoriasis me-too drugs. I can’t emphasize enough the fact there are ZERO psoriasis patients under my care that we cannot control with existing options. 1-2 shots per year instead of 4 is not meaningful innovation, period. Over a billion dollars going to this instead of other medical innovation. Psoriasis is just about the *only* condition in my field where we flat out don’t need something new, and these funds are just stuffing cash down ORKA’s throat to get their performance locks the easy way instead of investing somewhere that could actually benefit someone. As long as nobody sells for a while they’ll all make money, even if these stupid companies can’t get an exit in the long run. The investing system *should* be set up such that it doesn’t reward this nonsense - so far it isn’t. I know there are a lot of smart people involved in this who I otherwise like and respect, but I won’t mince words (hopefully they’ll at least respect me for that): This entire YTE situation is a pathetic use of the incentive system that’s supposed to ultimately help patients by creating medical innovation. As investors we are nothing but parasites if we aren’t taking risks to profit off of innovation and financial engineering our way to success instead. The only way this YTE situation works in the long run from these current valuations is if they are able to exploit a completely broken payor system by forcing doctors to abandon perfectly good generic drugs that the system could cheaply afford. I think this whole thing is gross.

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Occam's Toothbrush
Occam's Toothbrush@occams_tooth·
$NTLA HAE P3 results likely better than top line numbers suggest for 2 reasons: 1. HAE attacks are self reported. Once pts knew they got drug, attack-free rate drops to ~0 2. Efficacy improves over time Long term data will likely show ~100% attack free rate.
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Occam's Toothbrush
Occam's Toothbrush@occams_tooth·
$LLY making a move on Kelonia. The second in vivo CART play for $LLY after Orna (different approach : lenti vs RNA) Most US Based in vivo CART biotechs now snapped up: - $BMY Orbital $1.5B - $ABBV capstan $2.1B - $GILD interius $350M - $LLY Orna $2.4B wsj.com/tech/biotech/e…
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Occam's Toothbrush
Occam's Toothbrush@occams_tooth·
@ladanuzhna The tau failures were antibodies, which cannot penetrate cell bodies and target extracellular tau. But the tau tangles that are thought to drive the pathology are intracellular. The excitement is now about oligo therapies that are targeting those intracellular tangles
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lada
lada@ladanuzhna·
No other therapeutic area has much wishful thinking as AD. Drug class can literally fail across dozen+ different trials, and people would still be looking for a trial design where it might work. Was true for beta-amyloid, now clearly becoming true for tau.
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Occam's Toothbrush
Occam's Toothbrush@occams_tooth·
@GeneInvesting Yeah exactly. And if $LLY is putting forth unsolicited offers, they are probably targeting companies before value inflection points
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Gene Investing w/Anthony 🧬
Gene Investing w/Anthony 🧬@GeneInvesting·
Comments from $LLY head of business development…. I was always under the impression that a deal offered has to be proposed to the board/shareholders but it doesn’t if they don’t formally offer it I guess. And Big Pharma doesn’t do hostile takeovers that often. Basically explains why so many obvious deals aren’t getting done.
Gene Investing w/Anthony 🧬 tweet mediaGene Investing w/Anthony 🧬 tweet media
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