Retinopathy of prematurity (ROP) is the leading cause of blindness and visual impairment in children. Around 50-70% of preterm infants weighing less than 1250 grams at birth will be affected by this disease, leading to 20,000 individuals worldwide suffering from blindness or severe visual impairment every year. ROP is characterized as a vasoproliferative disease (ie. abnormal growth of blood vessels in the eye), some cases resolve spontaneously but in others, the abnormal vasoproliferation results in complications. If ROP continues to progress it leads to ischemia in the retina causing retinal neuronal death through scarring in the eye, retinal detachment, an irreversible loss of visual acuity and potentially blindness.
Current treatments rely on early diagnosis from generalized eye screening. However, existing interventions, such as laser therapy and VEGF inhibitor injections aim at limiting disease progression, are invasive and difficult for premature infants to endure. These do not sufficiently address the medical need as they come with their own complications and risks such as loss of peripheral vision, myopia, cataract, and a shrunken non functional eye. Due to increased survival rates of extremely premature infants, cases of ROP are growing significantly, resulting in an increased need for surgical interventions, injections, and follow-ups. Tackling the need for treatment through a preventive approach that reduces disease onset and progression altogether would help solve this problem.
The drug candidate, IBP-1118, is a dipeptide developed under the direction of Dr. Josef Neu, professor at the University of Florida Health, Department of Pediatrics, Division of Neonatology, and Dr. Maria Grant, professor at the University of Florida Health, Department of Endocrinology, Diabetes, and Metabolism. This agent has the potential to prevent the abnormal vascualrization in the retina, thereby promoting healthy retinal development and safeguarding the newborn’s eyesight. The hope is that IBP-1118 will decrease, or even eliminate, the need for invasive procedures for ROP and its progression to blindness by preventing its onset.
IBP-1118 has been granted orphan drug designation by the FDA, providing the company with certain benefits from the US government such as tax reductions and long-term market exclusivity, in exchange for developing the drug. The approval does not change the standard regulatory requirements and processes for obtaining marketing approval for a product. Consequently, all aspects of the development must be investigated, including the clinical safety and efficacy documentation required for market authorization.