INSM Insmed Inc

Insmed Incorporated (NASDAQ: INSM) provided an update on its ongoing pivotal clinical trial of iPlex(TM) (rhIGF-1/rhIGFBP-3) (Mecasermin rinfibate) for the treatment of children with growth failure who suffer from Severe Primary IGF-1 (insulin-like growth factor-1) deficiency (Primary IGFD). Some of these data were recently featured in a podium presentation at the European Society of Pediatric Endocrinology/Lawson Wilkins Pediatric Endocrine Society's 7th Joint Meeting. The data demonstrated that as a once-daily IGF-1 therapy, iPlex safely and significantly increased height velocity, the primary endpoint of the study, in children with short stature due to Severe Primary IGF-1 deficiency. Cecelia Camacho-Hubner, M.D., of St. Bartholemew's Hospital, London, United Kingdom commented, "Treatment with twice daily injections of rhIGF-I and once-daily injections of rhIGF-I/rhIGFBP-3 complex are effective in promoting linear growth in children with Severe Primary IGF-1 deficiency. Patients with Severe Primary IGF-1 deficiency are susceptible to spontaneous hypoglycemia; therefore it is important to monitor blood glucose levels before initiating any hormonal treatment. It is our opinion that the complex has demonstrated a superior safety profile in children with Severe Primary IGF-1 deficiency, especially regarding the number of hypoglycemic events and the severity of those events. This is most likely due to the modulating effects of IGFBP-3. Once a day injections with rhIGF-I/rhIGFBP-3 has been associated with very good compliance in our patients". Data Summary: Efficacy- -- All patients were pre-pubertal and identified as having Severe Primary IGF-1 deficiency (including subjects with GH receptor deficiency and GH gene deletion). The patients were characterized with severe short stature, low blood levels of IGF-1, and normal to elevated growth hormone levels. -- All patients received once-daily injections of iPlex at doses of 0.5 - 2.0 mg/kg, with the goal of restoring and maintaining IGF-1 blood levels in the normal range. -- 25 of the initial 29 patients enrolled in the study were evaluated for the primary efficacy endpoint: change in annualized height velocity (growth rate) after 6 months of treatment. Additional analyses were performed looking at longer-term treatment. -- The average pre-treatment height velocity in these 25 children was 3.0 centimeters per year (1.2 inches per year). Increases in height velocity during treatment were related to both the iPlex dose received and the IGF-1 blood levels achieved. In a subset of patients (n=16) treated at a low fixed dose (up to 1.0 mg/kg), a better height velocity was observed in those whose IGF-1 blood levels reached a normal target range than in those with persistently low IGF-1 levels on this dose (8.3 vs 5.6 cm/yr at Month 12, respectively). The overall increase in height velocity in this subset of patients was highly statistically significant (p