AI Drug Discovery for Pharma and Biotech

Drug discovery

14

drugs

With orphan designations

Overview

Necrotizing enterocolitis (NEC) is a life-threatening gastrointestinal emergency most common in preterm infants, characterized by intestinal inflammation, mucosal injury, and necrosis. Pathogenesis involves intestinal ischemia, dysbiosis, and enteral feeding in immature gut mucosa. Mortality approaches 20–30%, with survivors at risk for short bowel syndrome, neurodevelopmental delays, and intestinal strictures [1][5][6].

Population

  • Primarily affects preterm infants (90% of cases), especially very low birth weight (VLBW) neonates (<1,500 g), with 7–15% incidence in NICUs [7][8].

  • Term infants account for ~9% of cases, often with comorbidities (congenital heart disease, perinatal asphyxia) [4][13].

Burden

  • Mortality rates reach 50% in surgical cases; survivors face 30–50% risk of long-term neurodevelopmental/GI sequelae [1][6].

  • Annual U.S. hospitalization costs exceed $1 billion, with incremental post-discharge costs up to $18,274 per infant [13].

  • Leading cause of infant mortality among GI disorders, contributing to 10.2 deaths per 100,000 live births [12][13].

Therapies

  • Medical: NPO status, gastric decompression, IV antibiotics (ampicillin + aminoglycoside ± anaerobic coverage), and parenteral nutrition [1][6].

  • Surgical: Laparotomy with bowel resection for perforation or necrosis (required in 25–40% of cases) [1][6].

  • Emerging: Probiotics, stem cell therapy (BM-MSCs/AF-MSCs), and IL-22 supplementation show experimental promise [5][15].

Categories: rare gastroenterological diseases, rare transplant-related disorders

Research Papers

3,697 drug discovery papers related to Necrotizing enterocolitis, with 6 first-in-class and 4 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

3,697 drug discovery papers related to Necrotizing enterocolitis, with 6 first-in-class and 4 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

2026-07-06 | Prenatal corticosteroid use improves the severity and complications of necrotizing enterocolitis in preterm infants: a retrospective multicenter clinical study in China.

Our study is a retrospective multicenter observational cohort study to investigate effect of the use of antenatal corticosteroids (ACS) in preterm infants on the severity of necrotizing enterocolitis (NEC) and its associated complications. We collected clinical data from 443 preterm infants with gestational age (GA) of less than 37 weeks who were diagnosed with NEC in four hospitals across various provinces in China, covering the period from June 2020 to June 2024. According to whether they received a full course of prenatal corticosteroid treatment in the week before delivery, infants were divided into the exposed group and the unexposed group. A total of 213 preterm infants (48.08%) had received ACS therapy. When compared to the non-exposed group, the severity of NEC in the exposed group demonstrated a statistically significant difference (p = 0.005). Comparative analysis revealed that ethnicity, chorioamnionitis, antenatal steroid use, antenatal antibiotic use, premature rupture of membranes, gestational age, age at onset, respiratory support mode at onset, presence of intracranial hemorrhage before onset, postnatal occurrence of hemodynamically significant patent ductus arteriosus (hsPDA), absolute white blood cell count at onset, absolute platelet count at onset, and serum creatinine (SCr) levels during NEC (all p < 0.05) were identified as risk factors influencing NEC severity. In the univariate regression analysis, ACS therapy was identified as a significant protective factor against the occurrence of hsPDA (OR = 0.612, CI [0.385-0.974]), bronchopulmonary dysplasia (BPD) (OR = 0.611, CI [0.377-0.989]), and the need for surgical intervention (OR = 0.609, CI [0.384-0.967]). After adjusting for multiple confounding factors, ACS still demonstrated a protective effect against NEC severity (OR = 0.401, CI [0.257-0.672]), while chorioamnionitis (OR = 3.586, CI [1.571-8.185]), invasive respiratory support prior to onset (OR = 3.045, CI [1.464-6.330]), prenatal antibiotic use (OR = 3.752, CI [1.700-8.277]), and partially hydrolyzed formula feeding (OR = 3.500, CI [1.372-8.945]) were identified as significant risk factors for NEC severity. Therefore, ACS can reduce the severity of NEC and lower the incidence of hsPDA, BPD, and the necessity for surgical in preterm infants.

Open article ↗



2026-07-01 | The radiographic bubbly fecal pattern of intestinal pneumatosis in newborns revisited.

A bubbly fecal pattern on abdominal radiographs of neonates was described in the 1980s as a potential sign of intestinal pneumatosis, particularly in preterm infants during the first 2 weeks of life. This sign has not been systematically re-evaluated or correlated with sonographic findings. To assess whether, and up to which age, a radiographic bubbly fecal pattern represents intestinal pneumatosis, using bowel ultrasound as the reference standard. Infants aged 0-97 days who underwent abdominal radiography and bowel ultrasound for suspected ischemic enterocolitis in the neonatal intensive care unit were retrospectively included between January 2012 and December 2023. Radiography and ultrasound had to be performed within 24 h of each other. Imaging findings were analyzed per patient and per imaging episode. A total of 191 infants (82 female) were included, resulting in 402 paired examinations. Median age at imaging was 18 days. A bubbly fecal pattern was identified on 212 radiographs in 119 infants, with sonographic intestinal pneumatosis present in 172 cases (81.1%). Positive predictive values were consistently high, reaching 80.7% overall, while specificity and negative predictive values remained low across all age groups. A negative ultrasound in the presence of a bubbly fecal pattern was associated with a low surgical rate (6.5%). Sonographic pneumatosis was present in 90% of infants who underwent surgery. In infants evaluated for suspected necrotizing/ischemic enterocolitis, a radiographic bubbly fecal pattern is a relevant imaging sign associated with intestinal pneumatosis, particularly during the first 8 weeks of life.

Open article ↗



2026-07-06 | Prenatal corticosteroid use improves the severity and complications of necrotizing enterocolitis in preterm infants: a retrospective multicenter clinical study in China.

Our study is a retrospective multicenter observational cohort study to investigate effect of the use of antenatal corticosteroids (ACS) in preterm infants on the severity of necrotizing enterocolitis (NEC) and its associated complications. We collected clinical data from 443 preterm infants with gestational age (GA) of less than 37 weeks who were diagnosed with NEC in four hospitals across various provinces in China, covering the period from June 2020 to June 2024. According to whether they received a full course of prenatal corticosteroid treatment in the week before delivery, infants were divided into the exposed group and the unexposed group. A total of 213 preterm infants (48.08%) had received ACS therapy. When compared to the non-exposed group, the severity of NEC in the exposed group demonstrated a statistically significant difference (p = 0.005). Comparative analysis revealed that ethnicity, chorioamnionitis, antenatal steroid use, antenatal antibiotic use, premature rupture of membranes, gestational age, age at onset, respiratory support mode at onset, presence of intracranial hemorrhage before onset, postnatal occurrence of hemodynamically significant patent ductus arteriosus (hsPDA), absolute white blood cell count at onset, absolute platelet count at onset, and serum creatinine (SCr) levels during NEC (all p < 0.05) were identified as risk factors influencing NEC severity. In the univariate regression analysis, ACS therapy was identified as a significant protective factor against the occurrence of hsPDA (OR = 0.612, CI [0.385-0.974]), bronchopulmonary dysplasia (BPD) (OR = 0.611, CI [0.377-0.989]), and the need for surgical intervention (OR = 0.609, CI [0.384-0.967]). After adjusting for multiple confounding factors, ACS still demonstrated a protective effect against NEC severity (OR = 0.401, CI [0.257-0.672]), while chorioamnionitis (OR = 3.586, CI [1.571-8.185]), invasive respiratory support prior to onset (OR = 3.045, CI [1.464-6.330]), prenatal antibiotic use (OR = 3.752, CI [1.700-8.277]), and partially hydrolyzed formula feeding (OR = 3.500, CI [1.372-8.945]) were identified as significant risk factors for NEC severity. Therefore, ACS can reduce the severity of NEC and lower the incidence of hsPDA, BPD, and the necessity for surgical in preterm infants.

Open article ↗



2026-07-01 | The radiographic bubbly fecal pattern of intestinal pneumatosis in newborns revisited.

A bubbly fecal pattern on abdominal radiographs of neonates was described in the 1980s as a potential sign of intestinal pneumatosis, particularly in preterm infants during the first 2 weeks of life. This sign has not been systematically re-evaluated or correlated with sonographic findings. To assess whether, and up to which age, a radiographic bubbly fecal pattern represents intestinal pneumatosis, using bowel ultrasound as the reference standard. Infants aged 0-97 days who underwent abdominal radiography and bowel ultrasound for suspected ischemic enterocolitis in the neonatal intensive care unit were retrospectively included between January 2012 and December 2023. Radiography and ultrasound had to be performed within 24 h of each other. Imaging findings were analyzed per patient and per imaging episode. A total of 191 infants (82 female) were included, resulting in 402 paired examinations. Median age at imaging was 18 days. A bubbly fecal pattern was identified on 212 radiographs in 119 infants, with sonographic intestinal pneumatosis present in 172 cases (81.1%). Positive predictive values were consistently high, reaching 80.7% overall, while specificity and negative predictive values remained low across all age groups. A negative ultrasound in the presence of a bubbly fecal pattern was associated with a low surgical rate (6.5%). Sonographic pneumatosis was present in 90% of infants who underwent surgery. In infants evaluated for suspected necrotizing/ischemic enterocolitis, a radiographic bubbly fecal pattern is a relevant imaging sign associated with intestinal pneumatosis, particularly during the first 8 weeks of life.

Open article ↗



Access all drug discovery articles and probability of success in trials forecasts:

Access all drug discovery articles and probability of success in trials forecasts:

Drug Discovery Landscape

14 orphan drug designations for Necrotizing enterocolitis.

14 orphan drug designations for Necrotizing enterocolitis.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

bifidobacterium longum subsp. infantis

other

FDA

2025-05-06

Infinant Health, Inc.

Amnion-derived Cellular Cytokine Solution

cell therapies

FDA

2022-04-26

Noveome Biotherapeutics, Inc.

Human Placental Extract

other

FDA

2020-05-28

Plakous Therapeutics, Inc.

inter-Alpha-Inhibitor-Proteins (IaIp) (Human)

proteins

FDA

2018-02-13

ProThera Biologics, Inc.

melatonin

small molecules

FDA

2017-01-25

WORPHMED Srl

Melatonin

small molecules

EMA

2016-08-01

Worphmed Srl

Lactobacillus acidophilus and Bifidobacterium animalis subsp. lactis

combination

FDA

2015-03-24

Leadiant Biosciences, Inc.

bovine lactoferrin

proteins

FDA

2015-02-23

Metrodora Therapeutics, LLC

Lactobacillus reuteri

other

EMA

2015-02-12

Infant Bacterial Therapeutics AB

Lactobacillus acidophilus and Bifidobacterium bifidum

other

EMA

2013-12-18

Laboratorio Farmaceutico S.I.T. s.r.l.

L. reuteri

other

FDA

2013-08-01

Infant Bacterial Therapeutics

Heparin-binding epidermal growth factor-like growth factor (HB-EGF), amino acids 74-148

proteins

EMA

2006-10-31

Dr Michael Moore

Heparin-binding epidermal growth factor-like growth factor

proteins

FDA

2006-09-18

Trillium Therapeutics, Inc.

Bacitracin

FDA

1984-03-13

A. L. Laboratories, Inc.

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New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.