Recently, I had valid information that BioMarin announced that the measurement study is now open for recruitment again for the multicenter study. However, BioMarin didn´t change the age requirement, so children still need to be 4.5 years or older.
The person responsible for this information, working in one of the clinical trial centers, said they have not received much information about the results of the ongoing Phase 2 study, but the fact that BioMarin wants to enroll more children in the measurement study is an indication that they are pleased with the Phase 2 data that has been analyzed so far.
From another source, I received the information that the first 3 cohorts of phase 2 study have been reportedly fully enrolled and that there have been no adverse reactions to BMN-111 to date.
At BioMarin´s R&D day held in December 2014, BioMarin reported that in the original phase 2 protocol there were 5 cohorts, but the FDA stipulated that cohorts 4 and 5 could not be enrolled until safety assessments had been made of cohorts 1-3. Probably, this new enrolment is an indication of positive and safe results in cohorts 1-3.
A special thanks to the ones that gave these informations.