Yes, the result was not what was expected.
BioMarin’s median level of the factor at year one was 60. At 28 weeks, Pfizer looks to me like that is patient #10, tracking over 60, then you have week 36, patient #11 is not there, leaving it between #7 and #10. 7 is way up there close to 125, while 10 slipped below 50. It appears to me when all hit a year, the 60 achieved by BioMarin will be better because #10 is becoming a median and has fallen under 50.So the only better is hopefully no bleeds. There is also a tremendous amount of variable. Of course, nobody knows what variable was for BMRN. The ALT episodes seem to drop the activity. If there is steroid use, perhaps it can regulate levels of factor avoiding drops, but if one does not need it, since no bleeds or need for replacement, then why bother.The stock sold on unmet expectations, forgetting it is up to Pfizer, to design phase 3, and come up with perhaps even a higher dose and use of steroids to prevent the reduction of factor activity. Also, the lead-in study could answer questions on what characteristics of a patient the treatment would have the most success.
I think, since the AFFINE showed up at the end of April and updated in May, Pfizer/Sangamo knew March results already. The decision to start in July was based on the update we saw Thursday. I think this is positive, as Pfizer can manage the design, and they have trial experience and resources surpassing Sangamo.
I imagine September all are over 52 weeks; likely December ASH will be the forum to discuss it. We will not know what Pfizer is doing in phase 3; nobody knows what is happening to hemophilia B, so I see no difference here.
Do you see anything else come up between now and then? I assumed all trials would be postponed.I also think that ZFN 2.0 is not happening. Ed Rebar was leaving, and I cannot think of any other reason but everyone focusing on Biogen and moving resources away from MPS II. The indication is missing from the corporate deck. I imagine Sangamo is going after diseases with significant prevalence. So cell edit and gene regulation will be the action. I doubt we will see gene therapy either, beyond Fabry. Gene therapy is not ZFN, and the company is ZFN. It makes sense to focus on those. Small genetic diseases are too expensive to address with the budgets of Sangamo.