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Saul's avatar

Good piece. Am mildly surprised that management appears so emphatic regarding their desire to enter AD with Tau knockdown as opposed to say PSP (which is a "purer" Tauopathy, has no approved products and, as a rare disease, would require a much smaller commercial footprint and higher pricing). Still they can easily pivot if necessary.

BioBoyScout's avatar

Thanks, and you're right, PSP is a cleaner tauopathy: no amyloid noise, no approved drugs, orphan pricing. I'd actually expect Arrowhead to go after PSP and CBD in Phase 2, the same way plozasiran entered through FCS, a rare indication first, faster and cheaper to approval, then expand. The drug hits a sequence common to all tau forms, so it works across tauopathies; AD is just where the headline value sits.

Saul's avatar

As I recall, CBD is really awful; rapid decline, no treatment and diagnosis (the patient journey) often takes time. For the sake of argument, if approved for PSP, there would be a lot of off-label usage.