"ROP is a devastating eye disorder that can severely impactpreterm infants for the rest of their lives," Dr. Flemming Ornskov,CEO-designate for Shire, said in a press release. "This investigational proteinhas the potential to provide a first-in-class treatment that may minimize thedevelopment and impact of complications arising from ROP. We will build on thework that Premacure has done and will apply Shire's proven ability indeveloping protein replacement therapies for rare disorders to bring thismuch-needed therapy to the market."
During normal gestation, a fetus receives necessary growthfactors from the maternal serum, and full-term babies can produce said factorson their own. Preterm infants, however, lose specific growth factors such asinsulin-like growth factor 1 (IGF-1), which supports childhood growth anddevelopment, as a result of early separation from their mothers. The lack ofgrowth factors like IGF-1 is linked to lifelong complications such as ROP,bronchopulmonary dysplasia, intraventricular hemorrhage, necrotizingenterocolitis and impaired brain growth, according to Premacure's website, and"chronic complications following premature birth are present in up to 70percent of these neonates."
According to current estimates, roughly 54,000 prematureinfants are born in the European Union each year, and 87,000 in the UnitedStates, and of those numbers, approximately 14,000 to 16,000 preterm infants inthe United States present with some degree of ROP. For anywhere between 1,100and 1,500 of those infants, ROP is severe enough to require medical treatment,and some 400 to 600 infants become legally blind. ROP primarily affects preterminfants born before 31 weeks of gestation, and the smaller an infant is atbirth, the higher its chances of developing ROP, which usually develops in botheyes and is one of the leading causes of childhood vision loss.
At present, only symptomatic treatment exists for ROP.However, administering IGF-1 to premature infants to compensate for the loss ofthe maternal source has been shown to establish a physiological level of thehormone similar to that seen in utero.
Premacure's treatment—which it is developing under the brandname Premiplex—began with a formulation of IGF-1 combined with a recombinantversion of insulin-like growth factor-1 binding protein-3, IGF-1's naturallyoccurring binding protein. In a Phase I clinical trial, the investigationalprotein was shown to be generally well tolerated in preterm infants, and IGF-1levels were increased to within physiological levels. The current multicenterPhase II clinical trial to determine safety and efficacy is ongoing, and ShireHGT will continue the study following the acquisition.
Jessica Cotrone, senior director of corporate communicationsfor Shire HGT, comments that Shire is "always looking to partner or acquirecompanies who have life-altering therapies and are looking for a globaldevelopment and commercial partner. Taking all of this into consideration,Premacure was the right strategic fit for us."
The transaction complements Shire's existing expertise inprotein replacement, she adds, and is "aligned with Shire's focus on the orphandisease space, meeting the needs of the specialist physician and pursuinginnovative treatment options in areas of high unmet need that deliver value forpatients, physicians and the healthcare community." In addition, it allowsShire to expand its footprint into the field of neonatology.
"The acquisition of Premacure byShire further underscores the potential to change the long-term outlook forpreterm infants with ROP and their families," said Jan Borg, founding CEO ofPremacure, in a statement. "We are excited that this program will become partof the innovative pipeline at Shire and believe that their experience andresources may accelerate the development of a product that seeks to prevent someof the devastating long-term consequences of ROP."