A couple of years ago, when new Shire CEO Flemming Ornskov was selling a bright future to investors, he carefully singled out SHP607 as one of the company’s top experimental drugs, on track to earn more than $500 million in annual sales by 2020.
But he may have to revisit that estimate after reporting this morning that the protein replacement therapy failed the primary endpoint of reducing the severity of retinopathy of prematurity.
SHP607 is a combination of an insulin-like growth factor 1 (IGF-1) and a binding protein, IGF binding protein-3 (IGFBP-3). Among babies born prematurely, there’s a sharp drop in IGF-1 that threatens their eyes and other organs.
But while Shire missed on its primary goal, the big biotech says that it hit on a slate of secondary goals, pointing the way to a revised Phase III development program.
The (mostly) bad news followed a big bump for Shire’s shares $SHPG yesterday, when the Lexington, MA-based research arm for the company reported that it registered promising data for their new ADHD drug. That should take some of the sting out of today’s announcement.
Shire – which recently expanded its pipeline with the Baxalta buyout – says that it still has plenty of reasons to be optimistic about 607’s future.
The secondaries for 607, with some caveats about which of the 121 infants responded, showed:
- A 53% reduction in the incidence of severe bronchopulmonary dysplasia (BPD), defined by oxygen challenge testing, in all assessed patients that received SHP607, as compared to untreated infants.
- An 89% reduction in those who achieved the prespecified target drug exposure, based on serum concentrations of IGF-1, as compared to untreated infants.
- 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.
- 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.
“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, in a statement. “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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