BioBoyScout @BioBoyScout
Robert Toczycki, JD/MBA. Patent attorney & independent biotech analyst. Author of the BioBoyScout RNAi white papers. Builder of biotech valuation tools. Chicago bioboyscout.com Joined February 2015-
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$ARWR $PRQR $WVE
New white paper out: Edit the Copy, Not the Original. DNA Editing vs. RNA Editing vs. Gene Silencing. $LLY is right in the middle of it all. $PRQR $WVE $ARWR bioboyscout.com/p/edit-the-cop…
New white paper out: Edit the Copy, Not the Original. DNA Editing vs. RNA Editing vs. Gene Silencing. $LLY is right in the middle of it all. $PRQR $WVE $ARWR bioboyscout.com/p/edit-the-cop…
@BikeRieder @BioMan944918473 @elonmusk @ArrowheadPharma Musk-level first principles at $ARWR: build a reusable platform, then point it at target after target. Liver, lung, muscle, adipose, subQ CNS; a new cell type every ~18 months. The reusable rocket, applied to RNAi.
@vedel_j @ValueForAll1 Easiest call on the board. The target's validated up and down this chart. So the race isn't hitting INHBE, it's whose platform delivers it cleanest and most durably. I already know which bar that is. It's at the top. $ARWR
The INHBE race just reset. $WVE's monotherapy thesis cracked in March. $ARWR's EASL data this morning closed the gap, and then some. Combo data with tirzepatide is the piece that matters. Arrowhead has it. Wave hasn't even started its add-on trial. bioboyscout.com/p/the-inhbe-re…
5/5 Why test the combo with Lilly's drug specifically? $LLY owns the GLP-1s. They don't own a twice-yearly, liver-targeted asset that boosts MASH outcomes without GI side effects. ARO-INHBE fills that hole. The endgame just got closer. $ARWR
4/5 And here's what kills the GLP-1 monopoly: ARO-INHBE causes ZERO appetite suppression. Zero nausea bump. Zero GI misery. Patients lose visceral fat without feeling sick, on a twice-yearly shot. That's the holy grail GLP-1s can never deliver alone.
1/5 $ARWR just changed the obesity game at EASL. ARO-INHBE isn't competing with Mounjaro. It's making it work better, and unlocking a second $50B+ market while doing it. Two shots a year and it makes the best GLP-1s even better.
@RNAiAnalyst Respect the personal preference, but FDA doesn't approve drugs based on your choice; I would go the safer and just as effective route. Approval is based on population benefit-risk against alternatives. Willingness to accept doesn't change the framework, the label, or the TAM.
1/ Lilly's VERVE-102 readout is being framed as a paradigm shift. It isn't. It's a Phase 1b mechanism proof in 35 patients that the FDA's own gene-editing framework will keep in a narrow lane. Quick thread on what this actually means for $LLY and $ALNY.
In chess terms, the Fab choice was a hypermodern move. $ARWR made the move. Years of execution turned it into a moat. Broad patent protection now secures the best subcutaneous Fab targeting CNS for ANY kind of payload through 2045, and Arrowhead owns it. bioboyscout.com/p/the-fab-choi…
@biotechious Framework is in force today, reaffirmed in the April 2026 NGS draft. The bull case requires FDA reversing its position on alternatives for a target where PCSK9 mAbs and inclisiran already deliver the same LDL reduction reversibly. That's the unpredictable bet, not mine.
@cremieuxrecueil x.com/BioBoyScout/st…
1/ Lilly's VERVE-102 readout is being framed as a paradigm shift. It isn't. It's a Phase 1b mechanism proof in 35 patients that the FDA's own gene-editing framework will keep in a narrow lane. Quick thread on what this actually means for $LLY and $ALNY.
@afshineemrani x.com/BioBoyScout/st…
1/ Lilly's VERVE-102 readout is being framed as a paradigm shift. It isn't. It's a Phase 1b mechanism proof in 35 patients that the FDA's own gene-editing framework will keep in a narrow lane. Quick thread on what this actually means for $LLY and $ALNY.
13/13 Net: real mechanism. Improved safety vs. VERVE-101. Credible path to a narrow refractory-FH label. NOT a paradigm shift. NOT a Leqvio killer. NOT priced anywhere near reality in $LLY's implied modeling. $ALNY
12/ "Permanent" is a feature only when reversible options fail. For PCSK9 — the easiest possible gene editing case — they haven't failed. They've succeeded across statins, mAbs, and siRNA. The case for irreversibility here is weaker after this readout, not stronger.
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