Vosoritide one year phase 2 results of the third cohort to be presented in the next ASHG 2016 meeting

The developers of vosoritide (BMN-111), the first potential therapy for achondroplasia, will present further results of the ongoing phase 2 study during the next American Society of Human Genetics (ASHG) Meeting to be held in Vancouver in October.

This is a link to a description of the phase 2 study at ClinicalTrials.gov:  

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia (ACH).

In summary, according with the investigators, vosoritide has been showing safety and evidence of efficacy to allow its continuing development as a therapy for achondroplasia.

Main results provided:

• "After 12 months at 15 μg/kg/day, Cohort 3 subjects had 1.9 ± 2.0 cm/year (46%) increase in mean AGV from baseline (p=0.02)."

In easier words, the first number, 1.9, is the mean increase in growth velocity from baseline (day one) and the second number, 2.0 is the standard deviation, showing the growth velocity pace range among the participants of the study. The wide range can be explained by the small number of participants. The smaller the number of participants the wider any variation in measurements can be.

The 46% increase in growth velocity reported now is lower than the figure (50%) provided in a previous report of the first six months of therapy.

• "Twelve subjects escalated to 15µg/kg/day for ≥6 months had an increase of 2.3 ± 1.9 cm/year."

It is interesting to learn that the participants who were in the lower dose cohorts (3.75mcg/Kg/day and 7.5mcg/Kg/day) had a further increase in their growth velocity (65%). 

To read the complete abstract of the results that will be presented at the upcoming  ASHG meeting in Vancouver on next October, follow this link. 

Noteworthy, reviewing previous press releases from Biomarin about BMN-111/vosoritide and achondroplasia I found out that most of the data provided in the above mentioned abstract was already published in April 20th, here. 

Since there is no real new data disclosed (at least in the meeting abstract), the attention now falls over the results of the ongoing fourth cohort, which is using a higher dose of vosoritide (30mcg/Kg/day) and the planned phase 3 study.