SOBI’s Kiobrina pivotal Ph III study fails to meet primary endpoint

Stockholm-based Swedish Orphan Biovitrum (STO: SOBI) has released disappointing top-line results from a Phase III clinical trial of its enzyme therapy Kiobrina (bucelipase-alfa; rhBSSL – recombinant human Bile Salt Stimulated Lipase).

The primary endpoint of the study - growth velocity measured after four weeks of treatment with rhBSSL - was not met. No statistically significant improvement in growth velocity was demonstrated in preterm infants treated with rhBSSL compared to placebo.

Top-line results from the multicenter Phase III European trial of rhBSSL, called the LAIF study (Lipase Added to Infant Feeding), showed that in 410 infants born before 32 weeks of gestational age, the mean growth velocity did not differ when treated with rhBSSL (16.8g/kg/day) compared to placebo (16.6g/kg/day). The estimated difference in growth velocity was 0.21g/kg/day (95% CI (-0.40; 0.83), p=0.49). The growth velocity was comparable between groups throughout the treatment period, and the median enteral feeding volume was 2.7mL/kg/day lower in the rhBSSL group. 98% of infants completed the four week treatment period.