Beyond Achondroplasia

Growing together with Clara

BMN-111 – 6 key tidbits

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Comments by Mark Schoenebaum, Biotech/Pharma analyst since 2000 MD, Johns Hopkins School of Medicine, 2000 BA, Indiana University, 1995

6 key tidbits:

 1)      Phase 3 could enroll 50-100 patients and could be ~1 year in duration. Patient natural history could serve as control (likely no need for a placebo arm)

2)      Growth velocity improvement was consistent across gender and age. Response to treatment over time appears constant according to cGMP levels (biomarker for effect)

3)      Plan to enroll a 4th dose cohort at 30 microgram/kg starting later this summer and plan to initiate a study in children aged 0-5 years early next year

4)      BMRN has composition of matter patents in the US that extend to 2030 (extendable to 2033) and similar IP in other countries. BMRN is trying to license conflicting IP (methods, not composition of matter) in US/Japan that expires in 2022

5)      Any effect on secondary endpoints (e.g. bone proportionality) may take years – younger patients may take less time to see benefit

6)      Detailed data will be presented at a future medical meeting later this year (possibly in the fall)

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