AI Drug Discovery for Pharma and Biotech

Drug discovery

3

drugs

With orphan designations

Overview

Zollinger-Ellison syndrome (ZES) is caused by gastrin-secreting neuroendocrine tumors (gastrinomas), leading to gastric acid hypersecretion, refractory peptic ulcers, and chronic diarrhea [1][6][8]. Approximately 20-30% of cases occur in multiple endocrine neoplasia type 1 (MEN1) patients [4][6]. Diagnosis is confirmed by elevated fasting gastrin levels and secretin stimulation testing [10].

Population

  • Annual incidence: 0.1–3 cases per million [4][6]

  • Peak onset: Ages 30–50 (sporadic cases), earlier in MEN1 [6][7]

  • Sex distribution: Male predominance in sporadic cases (1.5–2:1) [5][9], slight female predominance overall (1.3:1) [2]

Burden

  • Median diagnostic delay: 6–9 years [8][14]

  • Complications: GI bleeding (25%), perforation, and metastatic spread (60–90% pancreatic gastrinomas) [6][7][9]

  • Survival: 10-year survival >90% with controlled acid secretion and localized disease; 30% with liver metastases [9][11]

Diagnosis requires vigilance in patients with refractory ulcers, chronic diarrhea, or MEN1 features [6][8].

Therapies

  • Acid suppression: High-dose proton pump inhibitors (PPIs) lifelong [6][10]

  • Surgery: Curative resection for localized sporadic tumors [9][10]; not recommended for MEN1-associated multifocal gastrinomas [9]

  • Advanced disease: Targeted therapy (everolimus/sunitinib), chemotherapy, or peptide receptor radionuclide therapy (PRRT) for metastases [1][9]

Categories: rare endocrine diseases, rare gastroenterological diseases, rare neoplastic diseases, rare transplant-related disorders

Research Papers

901 drug discovery papers about Zollinger-Ellison syndrome, with 2 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

901 drug discovery papers about Zollinger-Ellison syndrome, with 2 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

2026-05-06 | Formulation and Characterization of Enteric Coated Tablet Made by Combination of Pantoprazole and Omeprazole

Pantoprazole and omeprazole are proton pump inhibitors (PPIs) widely used in the treatment of acid-related gastrointestinal disorders such as peptic ulcer, GERD, and Zollinger–Ellison syndrome. Both drugs are highly unstable in acidic environments and undergo rapid degradation in the stomach. Therefore, the present study focuses on the formulation and characterization of enteric-coated tablets containing a combination of pantoprazole and omeprazole to protect them from gastric degradation and ensure targeted intestinal release. The tablets were prepared using direct compression/wet granulation techniques followed by enteric coating using polymers such as cellulose acetate phthalate and Eudragit L100. Preformulation studies including compatibility (FTIR), flow properties, and physicochemical parameters were evaluated. Post-compression parameters such as hardness, friability, weight variation, and drug content were assessed. In vitro dissolution studies showed minimal drug release in acidic pH (1.2) and maximum release in phosphate buffer (pH 6.8). Stability studies indicated that the formulation remained stable under accelerated conditions. The study concludes that enteric-coated combination tablets provide effective protection and improved bioavailability of both drugs.

Open article ↗



2026-03-20 | Zollinger-Ellison Syndrome: A Narrative Review of Clinical Presentation, Pathogenesis, Diagnosis and Modern Management Approaches

Zollinger-Ellison Syndrome (ZES) is rare disorder which is caused by gastrin-secreting neuroendocrine tumours known as gastrinomas, primarily located in the duodenum or pancreas. These tumours further result in excessive production of gastric acid, leading to recurrent, treatment-resistant peptic ulcers, chronic diarrhoea, and gastroesophageal reflux. Clinical features presented in patient include abdominal pain, weight loss, anaemia, and prominent gastric folds. Diagnosis of ZES is based upon elevated Fasting Serum Gastrin (FSG) levels, low gastric pH, and confirmatory secretin stimulation tests. Imaging methods such as Ga-68 DOTATATE PET-CT and Endoscopic Ultrasound (EUS) help in localisation of tumour, while Multiple Endocrine Neoplasia type 1 (MEN1)-associated cases require additional endocrine screening. Management in such cases involves high-dose Proton Pump Inhibitors (PPI) for suppression of acid and surgical resection is useful for localised tumours. Advanced and metastatic cases may prove helpful from Somatostatin Analogs (SSA) PPI or Peptide Receptor Radionuclide Therapy (PRRT). Novel agents like sunitinib and everolimus control tumour. Early diagnosis, multidisciplinary treatment can thus improve clinical outcomes and quality of life in patients with ZES. The narrative review is inclusive of clinical presentation, pathogenesis, diagnostic, and contemporary management and emerging approaches of ZES.

Open article ↗



2026-02-23 | Rendezvous-assisted endoscopic retrograde pancreatography using a dual-wire balloon technique for stenotic pancreaticojejunostomy in post-Whipple anatomy.

Pancreatic duct (PD) stones and a stenotic pancreaticojejunal anastomosis (PJA) can make ERCP highly challenging, especially in post-Whipple anatomy. EUS-assisted rendezvous (RV) may serve as a salvage approach, but severe stenoses can prevent guidewire passage. We describe a novel dual-wire balloon technique to achieve PD drainage in this setting. A 67-year-old man with Zollinger-Ellison syndrome and previous Whipple surgery presented with recurrent pancreatitis. Imaging revealed PD stones impacted at the PJA. Multiple enteroscopy-assisted ERCPs and an initial EUS-assisted RV failed. On repeat EUS-assisted RV, a 22-gauge needle was used to puncture the PD, and a 0.018-inch guidewire was advanced antegrade into the jejunum. Standard devices could not achieve dual-wire access. A therapeutic gastroscope was introduced, and a 5.5F balloon catheter was used to dilate the anastomosis. Removing the wire stiffener allowed a second 0.021-inch wire to be placed alongside the first. This approach enabled retrograde cannulation, deep wire access beyond the rendezvous wire through a stenotic PJA precluding larger dual-wire devices, and stent placement. The patient was discharged without adverse events, with stone extraction planned at follow-up. This case demonstrates the utility of a dual-wire balloon technique for EUS-guided PD drainage in severe PJA stenosis.

Open article ↗



2026-05-06 | Formulation and Characterization of Enteric Coated Tablet Made by Combination of Pantoprazole and Omeprazole

Pantoprazole and omeprazole are proton pump inhibitors (PPIs) widely used in the treatment of acid-related gastrointestinal disorders such as peptic ulcer, GERD, and Zollinger–Ellison syndrome. Both drugs are highly unstable in acidic environments and undergo rapid degradation in the stomach. Therefore, the present study focuses on the formulation and characterization of enteric-coated tablets containing a combination of pantoprazole and omeprazole to protect them from gastric degradation and ensure targeted intestinal release. The tablets were prepared using direct compression/wet granulation techniques followed by enteric coating using polymers such as cellulose acetate phthalate and Eudragit L100. Preformulation studies including compatibility (FTIR), flow properties, and physicochemical parameters were evaluated. Post-compression parameters such as hardness, friability, weight variation, and drug content were assessed. In vitro dissolution studies showed minimal drug release in acidic pH (1.2) and maximum release in phosphate buffer (pH 6.8). Stability studies indicated that the formulation remained stable under accelerated conditions. The study concludes that enteric-coated combination tablets provide effective protection and improved bioavailability of both drugs.

Open article ↗



2026-03-20 | Zollinger-Ellison Syndrome: A Narrative Review of Clinical Presentation, Pathogenesis, Diagnosis and Modern Management Approaches

Zollinger-Ellison Syndrome (ZES) is rare disorder which is caused by gastrin-secreting neuroendocrine tumours known as gastrinomas, primarily located in the duodenum or pancreas. These tumours further result in excessive production of gastric acid, leading to recurrent, treatment-resistant peptic ulcers, chronic diarrhoea, and gastroesophageal reflux. Clinical features presented in patient include abdominal pain, weight loss, anaemia, and prominent gastric folds. Diagnosis of ZES is based upon elevated Fasting Serum Gastrin (FSG) levels, low gastric pH, and confirmatory secretin stimulation tests. Imaging methods such as Ga-68 DOTATATE PET-CT and Endoscopic Ultrasound (EUS) help in localisation of tumour, while Multiple Endocrine Neoplasia type 1 (MEN1)-associated cases require additional endocrine screening. Management in such cases involves high-dose Proton Pump Inhibitors (PPI) for suppression of acid and surgical resection is useful for localised tumours. Advanced and metastatic cases may prove helpful from Somatostatin Analogs (SSA) PPI or Peptide Receptor Radionuclide Therapy (PRRT). Novel agents like sunitinib and everolimus control tumour. Early diagnosis, multidisciplinary treatment can thus improve clinical outcomes and quality of life in patients with ZES. The narrative review is inclusive of clinical presentation, pathogenesis, diagnostic, and contemporary management and emerging approaches of ZES.

Open article ↗



2026-02-23 | Rendezvous-assisted endoscopic retrograde pancreatography using a dual-wire balloon technique for stenotic pancreaticojejunostomy in post-Whipple anatomy.

Pancreatic duct (PD) stones and a stenotic pancreaticojejunal anastomosis (PJA) can make ERCP highly challenging, especially in post-Whipple anatomy. EUS-assisted rendezvous (RV) may serve as a salvage approach, but severe stenoses can prevent guidewire passage. We describe a novel dual-wire balloon technique to achieve PD drainage in this setting. A 67-year-old man with Zollinger-Ellison syndrome and previous Whipple surgery presented with recurrent pancreatitis. Imaging revealed PD stones impacted at the PJA. Multiple enteroscopy-assisted ERCPs and an initial EUS-assisted RV failed. On repeat EUS-assisted RV, a 22-gauge needle was used to puncture the PD, and a 0.018-inch guidewire was advanced antegrade into the jejunum. Standard devices could not achieve dual-wire access. A therapeutic gastroscope was introduced, and a 5.5F balloon catheter was used to dilate the anastomosis. Removing the wire stiffener allowed a second 0.021-inch wire to be placed alongside the first. This approach enabled retrograde cannulation, deep wire access beyond the rendezvous wire through a stenotic PJA precluding larger dual-wire devices, and stent placement. The patient was discharged without adverse events, with stone extraction planned at follow-up. This case demonstrates the utility of a dual-wire balloon technique for EUS-guided PD drainage in severe PJA stenosis.

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

3 orphan drug designations for Zollinger-Ellison syndrome, including 2 approved therapies.

3 orphan drug designations for Zollinger-Ellison syndrome, including 2 approved therapies.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

omeprazole-lansoprazole with buffer

small molecules

FDA

2015-02-10

Effexus Pharmaceuticals, LLC

Synthetic porcine secretin [Secreflo]

peptides

FDA

1999-06-18

2002-04-04

ChiRhoClin, Inc.

Synthetic human secretin [Chirostim]

peptides

FDA

1999-06-16

2004-04-09

ChiRhoClin, 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.