In May 2012, we entered into an agreement with Premacure pursuant to which we granted to Premacure an exclusive, worldwide license to develop manufacture and commercialize IGF-1, with its natural binding protein, IGFBP-3, for the prevention and treatment of complications of preterm birth (the "Premacure License Agreement")
“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."
Shire said it now “expects to begin discussions with regulatory authorities about a Phase III clinical program focusing on clinically relevant complications of prematurity,” which may see it re-jig its endpoints in the next stage of testing.
Analysts at Bernstein were cautiously optimistic. In their initial thoughts on the data, the firm said in a note to clients that Shire was overall able to provide premature babies with a “broad-effect growth factor they were missing (which usually supplied by the mother) and measure all potential outcomes.” While noting the company’s trial failures, it added that Shire was still “able to influence other conditions associated with very premature birth.”
But the analysts believe that, while still a risk, the negative results may not be as bad as it first seems. “This is the first test of IGF-1 in pre-term babies and the trial was 'hypothesis generating'--put the drug in and see what you can improve (one could argue that if you measure enough things, something will come up). However, it should not be too surprising IGF-1 has beneficial effects in pre-term babies and there appears to be a correlation between achieving blood level and outcomes.”
Bernstein expects the drug to go into a “high risk p3,” but are still touting it as a blockbuster drug. A similar sentiment was shared by Leerink, who said that the program “remains viable.”