SMID Biotech & Gene Editing 🧬 Hype welcome, receipts mandatory. Primary sources. Long some of what I post. Opinions, not 💵 advice.
Joined November 2011
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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 trailer. The filing is the movie.
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.
🤖 Made with AI
REGAL hit death number 80. Now comes the unblinding.
$SLS's Phase 3 tests galinpepimut-S as AML maintenance in second remission; overall survival is the primary endpoint. Topline is promised this quarter.
An event count says nothing about efficacy. What HR would matter here?
Same pill, twice the survival in one subgroup.
$ZNTL's WEE1 pill azenosertib, platinum-resistant ovarian cancer (ESMO): median OS 15.9 mo if Cyclin E1-positive vs 7.5 mo if negative. ORR 37.2%.
Single arm; 43% needed dose cuts. Is Cyclin E1 predictive, or just prognostic?
ALT $ZNTL azenosertib DENALI Cyclin E1 OS charcoal card
7 of 26 needed zero surgeries for a year. That earned the first FDA approval for a DNA medicine.
$INO's VOCERYQ (INO-3107), accelerated approval Oct 30 for adult RRP, an HPV airway disease. Single arm, 32 treated. Confirmatory protocol due by end of 2026. Durable past year one?
$31M more Attruby than last quarter. BridgeBio now frames $25M to $30M a quarter as the near term pace.
$BBIO US Attruby sales hit $253.2M in Q3. The small $11.6M operating loss leaned on a single $75M Bayer milestone.
How steep can the climb stay in a crowded ATTR-CM market?
When I saw the Attruby numbers last night, I thought that this drug is doing best in the very competitive aTTR market, but I also think the intense competition is hurting everyone in this space overall. I do not think Attruby will be as big as many think. I always have my numbers lower than everyone else. With that said, I am a buyer on weakness as it has 3 more drugs going commercial which have far less competition.
One trial, two jobs, if FDA's feedback holds.
$WVE says FDA aligned on a 2-year registrational study of RNA editor WVE-006 in AATD, with a 1-year biomarker interim for possible accelerated approval. Runway into Q3 2028.
Blood AAT isn't lung function. Is the interim enough?
Add bevacizumab and response rose from 37% to 59%, if a cross-trial read holds.
$CORT's relacorilant + nab-paclitaxel + bev in platinum-resistant ovarian (BELLA, n=95): median PFS 7.2 mo vs 5.4 in ROSELLA without bev. Bev-naive ORR 66%.
Single-arm Part A, no control.
ALT $CORT relacorilant BELLA ovarian navy card
Ten of 24 responded after anti-PD-1 had already failed.
$NBTX's JNJ-1900 in pretreated melanoma (ESMO): one nanoparticle shot, SBRT, then a PD-1 drug. 4 complete responses, median OS 17.8 mo.
Phase 1, single arm. How much is the radiation alone doing?
Plaque fell. Decline didn't.
$ABOS's oligomer antibody sabirnetug missed its Phase 2 primary endpoint in early Alzheimer's at both doses (n=542, 18 months). CDR-SB missed too. ARIA edema in ~10%.
Acumen is ending the program and exploring options.
Wrong target, or wrong dose?
Source, Acumen press release (Oct 30, 2026): globenewswire.com/news-relea…
Two years on first-line therapy, kidney function barely moved.
$TVTX's FILSPARI in newly found IgA nephropathy (SPARTAN, ASN): 75% hit complete proteinuria remission at some point; eGFR down 1.75 by wk 110.
Only 12 patients, open-label, no control arm: a signal, not proof.
ALT $TVTX FILSPARI SPARTAN IgA nephropathy forest card
From 2.5 hours to 15 before blood sugar crashed, after one infusion.
$BEAM's base editor BEAM-301 in GSDIa (ESGCT): 2 of 3 at 40 mg finished a 15-hour fast and stopped cornstarch. The third didn't improve.
n=6, open-label, 3 Grade 3 infusion reactions. Can 60 mg close the gap?
1/🚨WOW! @BeamTx has presented initial clinical data from its ongoing Phase 1/2 clinical trial evaluating $BEAM - 301 - a liver-targeting lipid-nanoparticle (LNP) formulation of adenine base editing (ABE) editors designed to correct the R83C mutation, as a “one and done” treatment for patients with glycogen storage disease type Ia (GSDIa). The most significant finding in today’s clinical data was that in the 40 mg cohort, two patients have achieved 15 hours of controlled fasting, improvements in glucose control and no longer required starch supplementation following a single administration of BEAM-301. These results strengthen BEAM-301’s potential as a “one and done” Gene Editing treatment for GSDIa patients by replacing the current intensive daily management which is currently required for treating this disease. Another strong indication of Base Editing’s potential as a meaningful therapeutic modality.
ALT WOW! BeamTx has presented initial clinical data from its ongoing Phase 1/2 clinical trial evaluating BEAM - 301 - a liver-targeting lipid-nanoparticle (LNP) formulation of adenine base editing (ABE) editors designed to correct the R83C mutation, as a “one and done” treatment for patients with glycogen storage disease type Ia (GSDIa). The most significant finding in today’s clinical data was that in the 40 mg cohort, two patients have achieved 15 hours of controlled fasting, improvements in glucose control and no longer required starch supplementation following a single administration of BEAM-301. These results strengthen BEAM-301’s potential as a “one and done” Gene Editing treatment for GSDIa patients by replacing the current intensive daily management which is currently required for treating this disease. Another strong indication of Base Editing’s potential as a meaningful therapeutic modality.
Biotech can sit flat for years, then move in a day.
$GMAB was $28.29 when I flagged it in Jan 2024 for a 3 year hold list. It closed $57.29 on Oct 28, +103%.
Not advice, just an observation. Which pipeline asset matters most now?
59.1% vs 24.6%. Same trial, sliced after the fact.
$IOVA's one-time TIL lifileucel, 2L nonsquamous lung cancer: 17 of 69 responded. KRAS wild-type, HER2 wild-type ever-smokers: 13 of 22.
Retrospective cut. POSITILITY tests it prospectively from November. Does the signal hold?
$IOVA Reports Updated IOV-LUN-202 Results and Adds Precision TIL Strategy for Second-Line mNSCLC
24.6% Objective Response Rate (ORR) in 69 Patients with Non-Squamous mNSCLC
ir.iovance.com/news-releases…
Oral VK2735 Phase 3 is two 560-patient studies (SUNRISE-1/2), 68 weeks, three doses. Q3 prints after the close today; the Oct raise was ~$548M net.
How many Phase 3 quarters does that buy before more dilution?