Endpoints NewsOriginal article
BioMarin discontinues rare disease drug; CSL and Arcturus split up
Phase 3negativeBMN401Negative
AI Analysis
Summary
BioMarin discontinued development of BMN401, an enzyme replacement therapy for ENPP1 deficiency, following mixed Phase 3 results. The discontinuation also marks a split between CSL and Arcturus in their partnership with BioMarin.
Clinical Trial Data
Phase
3
Primary Endpoint
Not Met
Outcome Details
BioMarin discontinued development of BMN401 following mixed Phase 3 results
Importance:6/10
Sentiment:
-0.90
rare_diseasedrug_discontinuationPhase_3_failureenzyme_replacement_therapypartnership_dissolution
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Published by Endpoints News on August 7, 2026 3:08 PM