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Welcome to the receipts desk 🧬
SMID biotech & gene editing. Numbers first, caveat second — and only with receipts.
House rules:
1. Hype is welcome. Receipts are mandatory. FDA letters, 8-Ks, trial registries. Not "people familiar with the matter."
2. A press release is the traimovie.
3. "Up to $2B" is not $2B. Milestones are wishes with a spreadsheet attached.
4. When I'm wrong, I'll say it here. No quiet deletes.
5. Sarcasm is free. Facts aren't negotiable.
Moonshot calls are fine. Moonshot calls with zero receipts get roasted.
Follow if you want the numbers and the caveat.
SMID biotech & gene editing. Numbers first, caveat second — and only with receipts.
House rules:
1. Hype is welcome. Receipts are mandatory. FDA letters, 8-Ks, trial registries. Not "people familiar with the matter."
2. A press release is the traimovie.
3. "Up to $2B" is not $2B. Milestones are wishes with a spreadsheet attached.
4. When I'm wrong, I'll say it here. No quiet deletes.
5. Sarcasm is free. Facts aren't negotiable.
Moonshot calls are fine. Moonshot calls with zero receipts get roasted.
Follow if you want the numbers and the caveat.
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1/ $CRSP CTX310 Phase 1b at AHA: one IV infusion cut ANGPTL3 65% and triglycerides 63% (mean, Day 30) in 10 people with severe hypertriglyceridemia. Median baseline TG was above 1,000 mg/dL. Thread 🧵
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5/ Caveats: n=10, open label, mean values (no range in the release), only 7 past 90 days. Does a deep 30 day TG drop hold at a year in this population, and does it cut pancreatitis? Nobody knows yet 🤷♂️
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47% median polyp cut in 12 weeks. 85% still below baseline 12 weeks off drug.
$RXRX REC-4881 in FAP (TUPELO Phase 2): 13 of 15 cut total burden; 6 of 8 with >5 mm polyps improved. Registrational start aimed 2H27.
Single arm, small n. Can oral MEK inhibition retire FAP surgery?
$RXRX REC-4881 in FAP (TUPELO Phase 2): 13 of 15 cut total burden; 6 of 8 with >5 mm polyps improved. Registrational start aimed 2H27.
Single arm, small n. Can oral MEK inhibition retire FAP surgery?
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44% slowing on cUHDRS at 48 months vs external control (p=0.144).
$QURE AMT-130 high dose (n=12): TFC slowed 61% (nominal p=0.008). BLA Priority Review, PDUFA by May 1 2027. Cash $768.4M into 2030.
External control, not randomized. Does TFC hold in confirmatory?
$QURE AMT-130 high dose (n=12): TFC slowed 61% (nominal p=0.008). BLA Priority Review, PDUFA by May 1 2027. Cash $768.4M into 2030.
External control, not randomized. Does TFC hold in confirmatory?
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3.8 points on MADRS at week 6. That gap now anchors a completed psilocybin NDA.
$CMPS COMP360 in treatment resistant depression: COMP006 (n=581) two 25 mg doses vs 1 mg, p<0.001. COMP005 (n=258) one dose vs placebo: 3.6.
A psychedelic is hard to blind. Does the gap hold?
$CMPS COMP360 in treatment resistant depression: COMP006 (n=581) two 25 mg doses vs 1 mg, p<0.001. COMP005 (n=258) one dose vs placebo: 3.6.
A psychedelic is hard to blind. Does the gap hold?
$CMPS Compass Pathways completes submission of NDA to FDA for COMP360 psilocybin for treatment-resistant depression
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Sources: Compass NDA completion release (Nov 9) morningstar.com/news/business-…
Phase 3 data deck s204.q4cdn.com/927483861/file…
Phase 3 data deck s204.q4cdn.com/927483861/file…
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17.8 mmHg off systolic BP at week 48, and 56% of patients got under 130.
$MLYS lorundrostat Transform-HTN extension at AHA (n=504 efficacy). Hyperkalemia needing a dose change: 5.1%. PDUFA by Dec 22.
Open label, no placebo at week 48. Does it hold in real practice?
$MLYS lorundrostat Transform-HTN extension at AHA (n=504 efficacy). Hyperkalemia needing a dose change: 5.1%. PDUFA by Dec 22.
Open label, no placebo at week 48. Does it hold in real practice?
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Source: Mineralys release, Nov 8 2026 (AHA late breaker; 632 in safety set, 92% completed 48 weeks)
globenewswire.com/news-release/2…
globenewswire.com/news-release/2…
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$NTLA MAGNITUDE-2 ATTR-PN enrollment finished in Q3, over 60 patients. Lonvo-z PDUFA by Mar 10 2027. Cash $614.8M into 2028.
Still not approved. How far is MAGNITUDE CM enrollment?
Still not approved. How far is MAGNITUDE CM enrollment?
1/🚨WOW! Intellia announced the completion of enrollment in its MAGNITUDE-2 Phase 3 clinical trial aimed to evaluate nex-z in patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). Nex-z - formerly known as $NTLA - 2001, is Intellia’s In Vivo andidate designed to inactivate the TTR gene in the liver, thus preventing the production of transthyretin (TTR) protein which is the root cause for hereditary transthyretin amyloidosis.
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A single shot, at least two years of LDL control. In monkeys.
$SCTX STX-1150 epigenetically silences PCSK9: up to 90% PCSK9 and 68% LDL drops in NHPs, DNA left intact. Phase 1 in high LDL adults is open.
No human efficacy yet. Will patients match monkeys?
$SCTX STX-1150 epigenetically silences PCSK9: up to 90% PCSK9 and 68% LDL drops in NHPs, DNA left intact. Phase 1 in high LDL adults is open.
No human efficacy yet. Will patients match monkeys?
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AHA durability: globenewswire.com/news-release/2…
Earlier EAS reduction figures: scribetx.com/newsroom/scrib…
Earlier EAS reduction figures: scribetx.com/newsroom/scrib…
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1/ One RNAi molecule, two genes silenced. $ARWR brought three lipid datasets to AHA today.
ARO-DIMER-PA hits PCSK9 and APOC3 at once, a first in the clinic. Phase 1/2a, mixed hyperlipidemia, single doses: LDL down up to 65%, triglycerides down up to 78% from baseline.
ARO-DIMER-PA hits PCSK9 and APOC3 at once, a first in the clinic. Phase 1/2a, mixed hyperlipidemia, single doses: LDL down up to 65%, triglycerides down up to 78% from baseline.
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4/ Caveats: DIMER is small and early (21 on drug, 10 on placebo), changes are from baseline and not placebo adjusted, and there are no outcomes data. The FCS extension had no placebo arm.
One shot for two targets, or two drugs dosed separately?
One shot for two targets, or two drugs dosed separately?
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