Vosoritide Proceeding to Phase 3
On the 11th January 2016, at the 34th annual J.P. Morgan Healthcare Conference, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, did the following presentation:
Recently secured "Freedom to Operate" worldwide
Next Steps:
1. Pivotal Phase 3 Study
Health authority discussions on study design and potential timelines
Anticipate Phase 3 start in 4Q16 (expected to start between October and December 2016)
2. Ancillary Studies
Enrolled 4th Cohort in Phase 2 study with 30 µg/kg/QD dose
Initiate a younger than 5 years study to explore earlier treatment option (finally, younger children will be included)
3. Program update at R&D Day April 2016 including
1 year data with 15 µg/kg/QD dose
At the Biotech Watcher, you can find the following table:
| BMN 111 (Vosoritide) - CNP Analog | |
| Achondroplasia | |
| Phase IIa Pediatrics Proof of Concept Study Program Update - 3rd Cohort (15 µg/mg) 1-Year Results, 4th Cohort (30 µg/mg) Safety Data, 5th Cohort (60 µg/mg) Launch |
April 2016 |
| Phase III Pivotal Study (15 µg/mg) Launch |
Last trimester-2016 |
| Phase II young children (under 5 years) Launch |
First Trimester-2016* |
Biotech Watcher's opinion:
"Early results indicate strong promise. The company is testing higher dose cohorts in the ongoing trial. BioMarin intends to launch a pivotal trial in 2016. Expect key update on results at April 2016 R&D Day"
