Given the complete lack of treatment for the rare diseases you listed and the general consensus that PSP is a "cleaner" tauopathy than AD, it makes sense to focus on the that first. However, the challenge then becomes a commercial one as much as a scientific/clinical one; how would you ultimately reconcile rare disease pricing of the same drug for rare disease (six figures plus) with high-prevalence potential (in AD)?
Still that may be a high quality problem to own. Let's see what the presentation brings.
Good question, and I think Ionis already ran the experiment. Olezarsen priced for FCS first, then sHTG behind it. Same molecule, orphan number anchoring going in, broad indication arriving later at a different net.
So PSP first, Alzheimer's later, is a path the field has walked.
Given the complete lack of treatment for the rare diseases you listed and the general consensus that PSP is a "cleaner" tauopathy than AD, it makes sense to focus on the that first. However, the challenge then becomes a commercial one as much as a scientific/clinical one; how would you ultimately reconcile rare disease pricing of the same drug for rare disease (six figures plus) with high-prevalence potential (in AD)?
Still that may be a high quality problem to own. Let's see what the presentation brings.
Good question, and I think Ionis already ran the experiment. Olezarsen priced for FCS first, then sHTG behind it. Same molecule, orphan number anchoring going in, broad indication arriving later at a different net.
So PSP first, Alzheimer's later, is a path the field has walked.
Agreed it's a high quality problem to own.