RazorSharpDNA 🧬

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.

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?

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?

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.

Strong response, heavy side-effect bill. $XNCR's XmAb819 in pretreated kidney cancer (ESMO): 45.5% confirmed response at the chosen 400 mcg/kg dose, n=11. Grade 3+ related AEs: 72.1% across expansion doses, led by AST/ALT. Is n=11 enough to pick a Phase 3 dose?

$318M before any patient data. $LYCA, Nobel laureate Carolyn Bertozzi's protein-degrader biotech, priced an upsized Nasdaq IPO. Its two lead drugs (IgE in allergy, Graves' autoantibodies) are in Phase 1; data in 2027. Platform premium before patient data. 2027 reads will test it.

HR 0.29 on progression. HR 0.43 on survival. $GMAB's ADC Rina-S beat chemo in platinum-resistant ovarian cancer (Phase 3 RAINFOL-02, n=584), whatever the FRα level or prior Elahere. All p<0.0001. Open-label PFS can flatter. OS can't, and it held.

Two rejections. Neither about the data. FDA accepted $UNCY's refiled NDA for oxylanthanum carbonate, a dialysis phosphate binder. PDUFA by March 25, 2027. The last CRL cited the old contract plant; the refile uses a new one. Third try lucky, or are plant problems ever fixed?