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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$182M in one quarter, a year into launch.
$PTCT's oral PKU drug Sephience grew 20% vs Q2, with 2,000+ patients on it. Product sales guidance is now $960M to $990M. Next: a Q4 BLA for ST-920, a one dose Fabry gene therapy.
Ex-US was only $37M. How much room is left abroad?
ALT $PTCT Sephience Q3 2026 sales card: $182M quarter, 20% growth vs Q2, 2,000+ patients, guidance $960M to $990M
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
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
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
FDA questioned the pathway the day it took the file.
$INO's INO-3107 for HPV airway growths (RRP), PDUFA by Oct 30: single arm, 32 adults, 28% surgery-free in year 1. Filed only for accelerated approval, with Papzimeos already approved.
What counts as "meaningful benefit" now?
ALT INO INO-3107 PDUFA card: 28% surgery-free year 1, accelerated pathway
Stopped early, unblinded anyway.
$AARD's Phase 3 HERO in Prader-Willi hunger ended at 67 of 90 patients; all efficacy is exploratory. ARD-101 cut HQ-CT "modestly" vs placebo by week 2, then scores slid back off drug.
FDA lifted the hold. Can a lower dose keep the signal?
ALT AARD Phase 3 HERO card: stopped at 67 of 90, exploratory efficacy
38% in immunologic remission by week 12.
$CLYM's CD19 antibody budoprutug in membranous nephropathy (ASN Kidney Week): 5 of 13 at 200 mg; anti-PLA2R antibodies down 70% on average. No related serious AEs.
Proteinuria lags antibodies. Do higher doses move it?
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.
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