Additional Information
Additional Relevant Details
- Top-line SHP607 study results in extremely premature infants showed no impact on primary endpoint of reducing the severity of Retinopathy of Prematurity
- Top-line analysis of secondary endpoints showed clinically relevant effects on severe complications related to lung and brain damage
- These data support further development of SHP607 in preterm infants; Shire plans to meet with regulatory authorities to discuss clinical path forward for phase 3 clinical program focusing on several complications of prematurity
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Clinical Data
The Phase 2 study included 121 extremely premature infants (born between 23 weeks and 27 weeks +6 days) randomized at birth to either SHP607 or standard neonatal care, and treated continuously until an equivalent gestational age of 30 weeks. IGF-1 is a growth factor that plays a major role in the development of the growing fetus in the uterus. It is supplied by the mother until about 30 weeks of gestation when the fetus begins producing the growth factor on its own. Levels of IGF-1 dramatically decrease in infants born extremely premature (before 28 weeks of gestation), thereby increasing the risk for complications related to the lungs, brain, eyes, and other organs.The Phase 2 top-line data showed a 53% reduction in the incidence of severe BPD, as defined by oxygen challenge testing, in all assessed patients that received SHP607, as compared to untreated infants; and an 89% reduction in those who achieved the prespecified target drug exposure, based on serum concentrations of IGF-1, as compared to untreated infants. The data also showed a 44% reduction in the incidence of severe IVH (Grade III and IV on centrally read ultrasounds) in all assessed patients that received SHP607, as compared to untreated infants; and a 64% reduction in those who achieved the prespecified target drug exposure based on serum concentrations of IGF-1, as compared to untreated infants. The secondary endpoint of time to discharge from neonatal intensive care was not met.Approximately 28,000 infants in the U.S. are born extremely premature – before 28 weeks of gestation. The overall death rate (16%) in the Phase 2 trial was consistent with mortality rates in this fragile population. There were more deaths in the treatment arm (20%) as compared to untreated babies (12%); however, no deaths were considered related to treatment. There were no serious adverse events related to the investigational medicinal product.Research suggests that 60% of extremely premature infants experience one or more severe complications related to prematurity, which include: IVH (grade ≥3); BPD; or ROP. Severe complications can have life-long implications for the developing infant.“This is the first controlled clinical trial to confirm the crucial role of IGF-1 in maturation of extremely preterm children,” said Professor Neil Marlow of the University College London Hospitals, UK, and one of the clinical trial investigators. “The reduction in BPD and IVH, as the two most important morbidities suffered by these children, are welcoming and a first in neonatal medicine. It will be important to confirm these findings in additional clinical studies.”
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