PAR. SP, page-192

I think it all comes down to the MPS VI results since we at least will have a placebo group to compare the results against. PAR needs to make sure they determine the power of the study so they can use the correct p-value. Basically, if PAR see a significant fall in GAG levels in the treated group then this should trigger a lot of interest since GAGs can then be used as a surrogate marker, requiring PAR to only perform a single P2 study in order to gain approval. It also means the improvement is over and above ERT, so is additive in its effects. Given that ERT goes for around 80k in Brazil, PAR should be able to request a very significant price.

My guess is PAR will announce a partner in Q1 2024 and this partner will be based in Brazil. Reason being that you need to partner in Brazil in order to get your treatment approved over there. Other partners will depend on the ease of gaining approval in US, EU and AU, or elsewhere. I'm not sure if PAR will need to conduct studies in these countries or if they can use the data they've already generated.