During normal gestation, the developing fetus is reliant on certain growth factors from the maternal serum; full term babies can produce these growth factors on their own. In preterm infants (born before 31 weeks of gestation), the early separation from the maternal circulation results in a loss of specific growth factors, such as insulin-like growth factor 1 (IGF-1), that are believed to result in lifelong complications, including ROP.
This acquisition allows Shire HGT to enter a new therapeutic area - neonatology - while maintaining its focus on developing novel therapies for the treatment of rare diseases with high unmet medical need. With the acquisition of Premacure, Shire HGT will continue the ongoing Phase II study, the primary goal of which is to restore the IGF-1 levels in the preterm infant to those found during normal in utero development.
"ROP is a devastating eye disorder that can severely impact preterm infants for the rest of their lives," said Flemming Ornskov, MD, CEO Designate, Shire. "This investigational protein has the potential to provide a first-in-class treatment that may minimize the development and impact of complications arising from ROP. We will build on the work that Premacure has done and will apply Shire’s proven ability in developing protein replacement therapies for rare disorders to bring this much needed therapy to the market."
"The acquisition of Premacure by Shire further underscores the potential to change the long-term outlook for preterm infants with ROP and their families," said Jan Borg, founding CEO of Premacure. "We are excited that this program will become part of the innovative pipeline at Shire and believe that their experience and resources may accelerate the development of a product that seeks to prevent some of the devastating long-term consequences of ROP."
Premacure AB is a private company launched in 2006 by entrepreneurs and internationally recognized clinicians in the area of neonatology.
Retinopathy of Prematurity (ROP) is a potentially blinding eye disorder that primarily affects premature infants weighing less than 2 pounds (about 1kg) who are born before 31 weeks of gestation (a full term pregnancy has a gestation of 38–42 weeks). The smaller a baby is at birth, the more likely that baby is to develop ROP. This disorder, which usually develops in both eyes, is one of the most common causes of visual loss in childhood and can lead to lifelong vision impairment and blindness.
In the US and EU, there are approximately 87,000 and 54,000 premature infants (born) born annually. Each year approximately 14,000–16,000 preterm infants in the US are affected by some degree of ROP. In 1,100–1,500 of these infants the ROP is severe enough to require medical treatment, and consequently 400–600 infants become legally blind.
About Insulin-Like Growth Factor 1
IGF-1 is primarily produced by the liver and supports normal childhood growth and development. Administration of IGF-1 to premature infants, as a substitute for the maternal and endogenous source, allows for the establishment of a physiological level of the hormone comparable to that present in utero. Such a treatment is believed to promote normal development and thereby prevent the development of complications of ROP.
Premacure initiated the clinical development of the preventative treatment with a formulation of recombinant human IGF-1 combined with a recombinant version of its naturally occurring binding protein, insulin-like growth factor-1 binding protein-3 (IFGBP3).
A Phase I clinical trial was conducted and results showed that the levels of IGF-1 were increased to within physiological levels and that administration of the investigational protein to preterm infants is generally well tolerated. A Phase II, safety and efficacy multi-centre clinical trial has started in Sweden and is on-going.
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