SMID Biotech & Gene Editing 馃К Hype welcome, receipts mandatory. Primary sources. Long some of what I post. Opinions, not 馃挼 advice.
Joined November 2011
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One drug: 0 of 14 major responses. Add a second: 4 of 14 colon tumors had no viable cancer at surgery.
$AGEN's botensilimab plus balstilimab, given before surgery in MSS colon cancer. No combo patient relapsed at 19.2 months median.
14 per arm, not randomized. Will it hold?
$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?
