Shire’s protein replacement for use in ROP misses primary endpoint

SHP607 did not meet its primary endpoint of reducing severity of retinopathy of prematurity in a phase 2 clinical trial, Shire announced in a press release. Nor was the secondary endpoint of time to discharge from neonatal intensive care met.

The phase 2 study included 121 extremely premature infants born before 28 weeks’ gestation who were randomized to receive SHP607 or standard neonatal care and were treated until an equivalent gestational age of 30 weeks.

SHP607 is a recombinant human version of the protein complex of insulin-like growth factor 1 (IGF-1) and IGF binding protein-3, which is decreased in extremely premature infants, thus increasing the risk for complications in the lung, brain, eye and other organs, according to the release.

Whereas the protein replacement did not meet its primary endpoint in relation to ROP, it did achieve clinically relevant effects in secondary endpoints related to reducing incidence of severe bronchopulmonary dysplasia (BPD) and of severe intraventricular hemorrhage (IVH). Incidence of severe BPD was reduced by 53% and incidence of severe IVH was reduced by 64% in treated infants compared with untreated infants, according to the release.

“For severe complications related to the lung and brain, there are no approved treatment options, and these data support our commitment to further investigate the potential systemic benefits of SHP607 in this population where the unmet patient need is substantial,” Philip J. Vickers, PhD, head of research and development of Shire, said in the release.