Biotech Values

[genisi]

wondering if the nature of the disease where drug is needed most its harder to get into those cells.

In Morquio due to GALNS enzyme deficiency, KS is accumulated in cartilage and macrophages. Bones and joints are severely affected and questions are if ERT would be able to penetrate these tissues and improve the skeletal defects after relatively short treatment of 24 weeks or slow progression in kids whose skeletal already too severely damaged to improve. Makes more sense to me that the improved 6MWD vs. placebo in GALNS trials resulted from reduction in inflammation and better cardiopulmonary function (this is not to say that bone cannot improve over longer period of time).

having a large sample size certainly helped here

Indeed it was the largest ERT trial ever but also enrolling the right patients helped - the entry criteria was patients with a baseline 6MWD of <325m.

Naglazyme didn't hit on 6MW

True, because the primary endpoint in the Naglazyme trial was 12MWD and it hit on that It was in Aldurazyme's registration trial where the 6MWD endpoint was not quite hit, but it showed a positive trend and was approved as we know.