AI Drug Discovery for Pharma and Biotech

Drug discovery

1

drug

With orphan designation

Overview

The Exstrophy-epispadias complex (EEC) encompasses a spectrum of congenital genitourinary malformations ranging from epispadias to severe cloacal exstrophy. These midline defects involve exposed bladder tissues, pelvic diastasis, and musculoskeletal anomalies, often accompanied by gastrointestinal, spinal, or renal abnormalities [1][5][16]. Surgical reconstruction aims to achieve urinary continence, preserve renal function, and improve quality of life through staged or single-stage repairs [3][18].

Population

  • Prevalence: 1/10,000 live births (overall), with classical bladder exstrophy (1/30,000) and cloacal exstrophy (1/200,000) [1][5]

  • Male predominance (male-to-female ratio 1.4:1 to 2.8:1) [2][7]

  • Increased recurrence risk (1/70 for offspring of affected parents) and associations with IVF [1][12]

Burden

  • Requires median 5-7 surgeries by adulthood, with 17-32% complication rates (e.g., dehiscence, obstruction) [14][18]

  • 60-70% achieve continence with surgery; persistent incontinence correlates with depression/anxiety [4][16]

  • Lifetime costs exceed $2M/patient due to multidisciplinary care needs and lost productivity [9]

Therapies

  • Surgical repair: Staged closure (bladder/pelvis → epispadias repair → bladder neck reconstruction) or single-stage complete primary repair (CPRE) [3][13][18]

  • Adjunctive measures: Pelvic osteotomies, testosterone pre-treatment for penile growth, and post-operative immobilization [14][18]

  • Secondary interventions: Bladder augmentation (30-40% of cases), continent urinary diversions, and management of complications (e.g., fistulas, reflux) [4][14]

Categories: rare abdominal surgical diseases, rare developmental anomalies during embryogenesis, rare genetic diseases, rare renal diseases, rare transplant-related disorders, rare urogenital diseases

Research Papers

572 drug discovery papers about Exstrophy-epispadias complex, with 2 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

572 drug discovery papers about Exstrophy-epispadias complex, with 2 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

2026-01-12 | Multidisciplinary perioperative management of cloacal exstrophy bladder closure: A single institution's approach.

Cloacal Exstrophy (CE), also known as OEIS complex (Omphalocele, Exstrophy of the cloaca, Imperforate anus, and Spinal defects), is the rarest and most severe congenital malformation of the bladder exstrophy-epispadias spectrum, characterized by an open bladder and exposed portions of the large intestine, along with bony anomalies. The objective of this study is to describe the experience of a high-volume exstrophy center's perioperative pathway, with a focus on analgesic use as well as urologic, orthopedic, and dietary interventions, for bladder repair of cloacal exstrophy. An IRB-approved, prospectively-maintained institutional approved Bladder Exstrophy-Epispadias Complex database was reviewed for patients with CE who had undergone bladder closure between 2017 and 2024. Electronic medical records were retrospectively reviewed to examine patient demographics, use of pelvic osteotomy, immobilization status, pediatric intensive care unit (PICU) admission, perioperative analgesia and sedation, nutritional support, drainage tubes, blood transfusion, antibiotic coverage, hospital length of stay, postoperative complications and closure failure. One hundred seventy-six CE and CE-variant patients were identified and 23 underwent bladder closure during this period. The median age at pelvic osteotomy was 1.93 years (IQR, 1.48-2.43, range 1-7). A delay of 14 days (IQR, 10-16) between osteotomy and closure was used in all but two patients. External fixation device and Buck's traction remained for 42 (IQR, 42-47) days. Post-closure, PICU stay was 8 days (IQR, 7-13) with 78 % requiring invasive ventilation for 4.5 days (IQR, 2-13.8). All patients required blood transfusions (2, IQR, 1-3). Enteral feeding resumed by day 11.5 (IQR, 5.8-16.5) for all but one and parenteral nutrition for 21 days (IQR, 13.5-53). Patient-controlled analgesia (PCA) was used in all patients for both pre- and post-closure, morphine most commonly (76 % and 48 %, respectively). Opioid use totaled 45 days (IQR, 40-60). Acetaminophen use was universal and NSAIDs used in 43 % (pre) and 57 % (post), clonidine in 62 % (pre) and 96 % (post). Bladder closure was successful in 87 %, while 13 % required repeat operation. Length of stay averaged 62 days (IQR, 51-86), range 42-191. While our study was limited by a small patient population, this data highlights the complexity and resource-intensive nature of CE bladder closure, emphasizing the need for comprehensive multidisciplinary management at specialized centers. Our 87 % success rate in bladder closure for patients with CE underscores the effectiveness of the multidisciplinary approach described when managing the complex perioperative course of these patients. However, challenges remain including prolonged hospitalization, ventilation requirements and slow reintroduction of enteral nutrition while highlighting the optimization of perioperative strategies of pain, sedation and recovery.

Open article ↗



2025-06-14 | Medium- and long-term results of botulinum toxin injection in patients with bladder augmentation: A retrospective study and literature review.

To determine the medium and long-term results and safety of botulinum toxin treatment in enlarged patients with incontinence secondary to overactive bladder and compared it with literature review. a prospective study was performed analysing the results of 41 procedures performed between 2010 and 2023 in a total of 14 patients with a history of bladder augmentation and clinical or urodynamic findings suggestive of overactive bladder or low compliance. A total of 41 procedures were performed on 14 patients. 71 % of patients received previous oral treatment, the average between the bladder augmentation and the first injection was 20 years. The re-injection rate was 71 % with a mean time between procedures of 19 months. 61 % of the patients presents clinical response. No differences were found in terms of underlying pathology, epidemiological factors, urodynamic parameters or combined vs single bladder patch injections. Differences were found regarding previous use of anticholinergic treatment. Botulinum toxin treatment is safe and effective for patients with cystoplasty and may avoid a major procedure in those patients than show symptomatology or urodynamic findings with overactive bladder or a low accommodation.

Open article ↗



2025-03-31 | Platelet secretions exert anti-inflammatory effects in vitro on neutrophils and uterine epithelia in cattle: a possible role in amplifying the uterine immune network toward pregnancy

Our findings reveal for the first time that platelet secretions directly act on PMNs and BEECs in vitro, thereby assisting the uterine immune network to shift anti-inflammatory environment toward pregnancy. The present study can explain, in part, the successful applications of platelet derivatives in reproductive medicine.

Open article ↗



2026-01-12 | Multidisciplinary perioperative management of cloacal exstrophy bladder closure: A single institution's approach.

Cloacal Exstrophy (CE), also known as OEIS complex (Omphalocele, Exstrophy of the cloaca, Imperforate anus, and Spinal defects), is the rarest and most severe congenital malformation of the bladder exstrophy-epispadias spectrum, characterized by an open bladder and exposed portions of the large intestine, along with bony anomalies. The objective of this study is to describe the experience of a high-volume exstrophy center's perioperative pathway, with a focus on analgesic use as well as urologic, orthopedic, and dietary interventions, for bladder repair of cloacal exstrophy. An IRB-approved, prospectively-maintained institutional approved Bladder Exstrophy-Epispadias Complex database was reviewed for patients with CE who had undergone bladder closure between 2017 and 2024. Electronic medical records were retrospectively reviewed to examine patient demographics, use of pelvic osteotomy, immobilization status, pediatric intensive care unit (PICU) admission, perioperative analgesia and sedation, nutritional support, drainage tubes, blood transfusion, antibiotic coverage, hospital length of stay, postoperative complications and closure failure. One hundred seventy-six CE and CE-variant patients were identified and 23 underwent bladder closure during this period. The median age at pelvic osteotomy was 1.93 years (IQR, 1.48-2.43, range 1-7). A delay of 14 days (IQR, 10-16) between osteotomy and closure was used in all but two patients. External fixation device and Buck's traction remained for 42 (IQR, 42-47) days. Post-closure, PICU stay was 8 days (IQR, 7-13) with 78 % requiring invasive ventilation for 4.5 days (IQR, 2-13.8). All patients required blood transfusions (2, IQR, 1-3). Enteral feeding resumed by day 11.5 (IQR, 5.8-16.5) for all but one and parenteral nutrition for 21 days (IQR, 13.5-53). Patient-controlled analgesia (PCA) was used in all patients for both pre- and post-closure, morphine most commonly (76 % and 48 %, respectively). Opioid use totaled 45 days (IQR, 40-60). Acetaminophen use was universal and NSAIDs used in 43 % (pre) and 57 % (post), clonidine in 62 % (pre) and 96 % (post). Bladder closure was successful in 87 %, while 13 % required repeat operation. Length of stay averaged 62 days (IQR, 51-86), range 42-191. While our study was limited by a small patient population, this data highlights the complexity and resource-intensive nature of CE bladder closure, emphasizing the need for comprehensive multidisciplinary management at specialized centers. Our 87 % success rate in bladder closure for patients with CE underscores the effectiveness of the multidisciplinary approach described when managing the complex perioperative course of these patients. However, challenges remain including prolonged hospitalization, ventilation requirements and slow reintroduction of enteral nutrition while highlighting the optimization of perioperative strategies of pain, sedation and recovery.

Open article ↗



2025-06-14 | Medium- and long-term results of botulinum toxin injection in patients with bladder augmentation: A retrospective study and literature review.

To determine the medium and long-term results and safety of botulinum toxin treatment in enlarged patients with incontinence secondary to overactive bladder and compared it with literature review. a prospective study was performed analysing the results of 41 procedures performed between 2010 and 2023 in a total of 14 patients with a history of bladder augmentation and clinical or urodynamic findings suggestive of overactive bladder or low compliance. A total of 41 procedures were performed on 14 patients. 71 % of patients received previous oral treatment, the average between the bladder augmentation and the first injection was 20 years. The re-injection rate was 71 % with a mean time between procedures of 19 months. 61 % of the patients presents clinical response. No differences were found in terms of underlying pathology, epidemiological factors, urodynamic parameters or combined vs single bladder patch injections. Differences were found regarding previous use of anticholinergic treatment. Botulinum toxin treatment is safe and effective for patients with cystoplasty and may avoid a major procedure in those patients than show symptomatology or urodynamic findings with overactive bladder or a low accommodation.

Open article ↗



2025-03-31 | Platelet secretions exert anti-inflammatory effects in vitro on neutrophils and uterine epithelia in cattle: a possible role in amplifying the uterine immune network toward pregnancy

Our findings reveal for the first time that platelet secretions directly act on PMNs and BEECs in vitro, thereby assisting the uterine immune network to shift anti-inflammatory environment toward pregnancy. The present study can explain, in part, the successful applications of platelet derivatives in reproductive medicine.

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

1 orphan drug designation for Exstrophy-epispadias complex.

1 orphan drug designation for Exstrophy-epispadias complex.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

Primary Human Satellite cell-derived muscle stem cells

cell therapies

FDA

2023-07-13

MyoPax Denmark ApS

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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.