TraceyMcC Northern Ireland
DianaR London
Andleo South East
annagg South West
S007CLA North East
Rhodri23 Wales
This is the latest update from Biomarin with regard to Utrophin.
In the first quarter of 2009, BioMarin recorded impairment loss on investments of $5.9 million, comprised of $1.4 million related to its equity investment in Summit Corporation plc and $4.5 million related to its equity investment in La Jolla Pharmaceutical Company. The remaining investments of $0.2 million in Summit Corporation and $1.8 million in La Jolla Pharmaceutical are reflected on the balance sheet as of March 31, 2009. Since both of these companies have announced that they do not have sufficient resources to fund operations for the next twelve months, the guidance below reflects the assumption that the remaining investments in both companies will be written down to zero by the end of the year.
BMN-195 – Utrophin upregulator for Duchenne Muscular Dystrophy: BioMarin is completing reformulation work and toxicology studies and expects to initiate a Phase I trial by the first quarter of 2010. BMN-195 is an orally available small molecule which may upregulate utrophin, a potential replacement for the missing dystrophin protein in DMD patients.
Anticipated Upcoming Milestones
2Q09: Results from PEG-PAL Phase I trial
2Q09: Initiation of PEG-PAL Phase II trial
3Q09: Decision on strategy for BMN-103 for Pompe Disease
3Q09: Decision on 6R-BH4 cardiovascular program
1Q10: Initiation of Phase I trial for BMN-195 for DMD
1H10: Results from Phase I/II trial for GALNS for MPS IVA
Mid-2010: Results from PEG-PAL Phase II trial
2H10: Initiation of pivotal Phase III trial for GALNS for MPS IVA
2H10: Availability of blood Phe monitor
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