Gastroschisis is a rare, life-threatening, and debilitating birth abnormality in late preterm infants where the infant is born with externalized abdominal organs, affecting 2,000 US infants per year.
Gastroschisis represents an area of significant unmet medical need requiring operative intervention to reposition the herniated organs into the abdominal cavity. Post-operative complications arise, such as impaired gut motility and delayed ability to tolerate oral feeding with a detrimental impact on intestinal maturation.
Management of gastroschisis is largely aimed at overcoming the significant morbidity related to the impaired gut function and consequent feeding intolerance necessitating the prolonged intensive care treatment and risks associated with parenteral nutrition and central line usage. Infants suffering from gastroschisis suffer from risks of growth retardation, sepsis, NEC, and in-hospital mortality. It is common for neonates born with gastroschisis to have a significantly extended hospital stay causing a significant burden to the healthcare system, in the US alone estimated at $200-$300M (20-30 days at $5K per day for 2,000 infants).
Treatment with the active bacteria used in IBP-1016, Lactobacillus reuteri, is known to have four interactive mechanisms of action that can alleviate complications associated with gastroschisis all of which contribute to a healthy gut and an improvement in feeding tolerance:
IBP-1016 helps to establish a healthy microbiome that is often disrupted in patients with gastroschisis.
IBP-1016 is shown to enhance gut motility and thus benefits infants with feeding intolerance by increasing bowel movement.
Experimental data demonstrates that gastroschisis is associated with poor intestinal maturity and integrity, while IBP-1016 has been shown to be beneficial in the development of healthy intestinal mucosa.
There is an observed increase in intestinal inflammation in patients with gastroschisis which can be tackled and reduced through the use of IBP-1016 which has immunomodulatory effects.
IBP-1016 targets an infant population adjacent to IBP-9414, as IBP-9414 would target patients of <1500 g birth weight and a gestational age of 23-32 weeks, while IBP-1016 targets late preterm or term infants of ~2500 g birth weight and a gestational age of ~35 weeks.
IBP-1016 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.