AI Drug Discovery for Pharma and Biotech

Drug discovery

5

drugs

With orphan designations

Overview

Eosinophilic granulomatosis with polyangiitis (EGPA) is a rare ANCA-associated vasculitis characterized by eosinophilic inflammation, necrotizing granulomas, and small-to-medium vessel vasculitis. It typically progresses through three phases: allergic (asthma, sinusitis), eosinophilic (organ infiltration), and vasculitic (neuropathy, purpura). ANCA positivity occurs in 30–40% of cases, often linked to glomerulonephritis. Multisystem involvement includes respiratory (95% asthma), cardiac (leading cause of death), and gastrointestinal systems [1][6][10][11].

Population

  • Incidence: 1.07–4.0 cases per million person-years globally; UK incidence stable at 2.3–4.0 (2006–2019) [2][5][7].

  • Prevalence: Increased from 22.7 to 45.6 per million in the UK (2005–2019); mean age at diagnosis 57 years, slight female predominance (51%) [2][10][18].

Burden

  • Morbidity: 35% relapse within 5 years; 26% develop new vasculitic manifestations (e.g., mononeuritis multiplex, cardiomyopathy) [5][18].

  • Healthcare: 19% require EGPA-related hospitalization (median 11 days); 39% receive ≥5 OCS prescriptions/year [2][9][10].

  • Comorbidities: Asthma (80.6%), nasal polyps (32.1%), and cardiac involvement (up to 50% mortality if untreated) [1][2][15].

Therapies

  • First-line: High-dose corticosteroids (≥47% OCS dependence post-diagnosis) ± immunosuppressants (cyclophosphamide, azathioprine) [3][9][15].

  • Biologics: Mepolizumab (anti-IL-5) for refractory/relapsing disease; rituximab (anti-CD20) in ANCA-positive cases [8][13][17].

  • Relapse management: Chronic low-dose corticosteroids; monitoring for hypogammaglobulinemia with biologics [3][8][13].

Categories: rare cardiac diseases, rare circulatory system diseases, rare neurological diseases, rare renal diseases, rare respiratory diseases, rare systemic and rheumatological diseases, rare systemic or rheumatologic diseases of childhood, rare transplant-related disorders

Research Papers

1,313 drug discovery papers about Eosinophilic granulomatosis with polyangiitis, with 2 first-in-class and 12 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

1,313 drug discovery papers about Eosinophilic granulomatosis with polyangiitis, with 2 first-in-class and 12 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

2026-07-11 | Eosinophil diversity and function: emerging insights into subtypes and therapeutic targets.

Eosinophils are multifunctional polymorphonuclear granulocytes garnering attention for their roles in disease and maintaining physiological homeostasis. Historically, these immune cells were known to mediate allergic responses and defend against parasitic infections. Recent evidence further links eosinophils to several eosinophil-associated disorders, including eosinophilic esophagitis, eosinophilic granulomatosis with polyangiitis, and hypereosinophilic syndrome; however, their pathogenic contribution appears to be disease-specific and remains incompletely defined in some contexts. The advances in immunology and molecular biology have yielded targeted biologic therapies against eosinophils, significantly improving outcomes for patients with eosinophilic disorders. Concurrently, research reveals that eosinophils are a heterogeneous cell population comprising distinct subtypes that differ in surface protein expression, tissue localization, and functional properties. Specifically, resident eosinophils (rEos) reside in tissues for local maintenance and immune regulation, whereas inflammatory eosinophils (iEos) are rapidly recruited during immune responses to drive inflammation. Initially characterized in murine models, similar eosinophil heterogeneity has since been confirmed in human blood and tissues. Recognizing distinct eosinophil subsets is crucial for understanding their contributions to disease progression. This review highlights recent discoveries in eosinophil diversity and explores therapeutic implications of selectively targeting specific eosinophil subsets in clinical practice.

Open article ↗



2026-07-02 | Case Report: Benralizumab combined with a steroid-sparing strategy in a case of severe eosinophilic granulomatosis with polyangiitis.

Eosinophilic granulomatosis with polyangiitis (EGPA) is a rare anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis characterized by eosinophilic inflammation and necrotizing vasculitis. Benralizumab, an anti-IL-5Rα monoclonal antibody that directly targets eosinophils, was approved for EGPA in China in December 2025. We report an early real-world experience of benralizumab combined with corticosteroids in a patient with severe ANCA-negative EGPA. A 73-year-old male with a history of childhood asthma, chronic rhinosinusitis, CKD stage 4 presented with recurrent pulmonary infiltrates, progressive interstitial lung disease, severe eosinophilia (peak 12.90×109/L), diffuse skin lesion and new-onset arrhythmia. After systematic differential diagnosis, EGPA was diagnosed according to the 2022 ACR/EULAR criteria (score 7). BALF and blood testing detected nucleic acids of Pneumocystis jirovecii and CMV. He was treated with methylprednisolone 40 mg/day and subcutaneous benralizumab 30 mg every 4 weeks. Concurrently, empirical preemptive anti-infective therapy (caspofungin for Pneumocystis jirovecii, ganciclovir for CMV) was initiated. At 4-week follow-up, prednisone was tapered to 30 mg/day; dyspnea and rash markedly improved, eosinophils decreased to 0, IgE fell from 1,860 to 519 IU/mL, chest CT showed significant resolution of infiltrates, and pulmonary function improved from severe to mild restrictive impairment. At 3 months, prednisone was further tapered to 25 mg/day in combination with benralizumab, and the patient remained stable without disease relapse or acute infection. This case represents an early real-world application of benralizumab in a high-risk ANCA-negative EGPA patient following its approval in China. The findings suggest that benralizumab, used as an adjunctive steroid-sparing agent, may facilitate rapid corticosteroid tapering in selected vulnerable patients with infection risk. Long-term follow-up and studies are needed to validate these findings.

Open article ↗



2026-06-30 | Comparative real-world effectiveness and safety of benralizumab and two mepolizumab dosing regimens in eosinophilic granulomatosis with polyangiitis: a 24-month prospective single-center cohort study.

Long-term prospective real-world data on anti-IL-5 and anti-IL-5 receptor biologics in eosinophilic granulomatosis with polyangiitis (EGPA) are limited. In this 24-month prospective single-center observational study, 66 adults with EGPA received benralizumab 30 mg (every 4 weeks for the first three doses, then every 8 weeks), mepolizumab 300 mg every 4 weeks or mepolizumab 100 mg every 4 weeks. Remission was defined as BVASv3 = 0 with prednisone ≤5 mg/day. Glucocorticoid (GC)-free status was defined as no oral GCs. The primary analysis was conducted in patients starting biologics as first-line. To address confounding by indication, we performed multivariable logistic regression and multinomial propensity score inverse probability weighting (IPTW). Analyses were repeated in an all-lines dataset (81 treatment lines) using generalized estimating equations (GEE) clustered by patient. Clinical outcomes improved over the 24-month follow-up across all regimens. By 24 months, remission was 73.7% (14/19) with benralizumab, 81.0% (17/21) with mepolizumab 300 mg, and 50.0% (6/12) with mepolizumab 100 mg, without statistically significant differences between regimens. GC-free status increased over time, reaching 63.2% (12/19) with benralizumab, 85.7% (18/21) with mepolizumab 300 mg, and 58.3% (7/12) with mepolizumab 100 mg at 24 months, with no significant between-group differences. Adjusted and propensity-based sensitivity analyses yielded consistent conclusions. Eosinophils decreased markedly, with near-complete suppression with benralizumab. Pulmonary function indices showed improvement during follow-up, with the clearest within-group improvement observed in the benralizumab group. Treatment persistence at 24 months varied across regimens, although no statistically significant difference was observed (log-rank p=0.222). Discontinuations were mainly driven by inadequate control of ENT or respiratory manifestations. In all-lines GEE analyses, regimen effects on remission and GC-free status were broadly consistent with the primary analysis. Adverse events were uncommon and no serious events were reported. Anti-IL-5/IL-5R biologics were effective and well tolerated over 24 months in EGPA, with substantial GC sparing effect. The observational design and potential confounding by indication limit comparative effectiveness inference. These findings support individualized biologic selection in EGPA.

Open article ↗



2026-07-11 | Eosinophil diversity and function: emerging insights into subtypes and therapeutic targets.

Eosinophils are multifunctional polymorphonuclear granulocytes garnering attention for their roles in disease and maintaining physiological homeostasis. Historically, these immune cells were known to mediate allergic responses and defend against parasitic infections. Recent evidence further links eosinophils to several eosinophil-associated disorders, including eosinophilic esophagitis, eosinophilic granulomatosis with polyangiitis, and hypereosinophilic syndrome; however, their pathogenic contribution appears to be disease-specific and remains incompletely defined in some contexts. The advances in immunology and molecular biology have yielded targeted biologic therapies against eosinophils, significantly improving outcomes for patients with eosinophilic disorders. Concurrently, research reveals that eosinophils are a heterogeneous cell population comprising distinct subtypes that differ in surface protein expression, tissue localization, and functional properties. Specifically, resident eosinophils (rEos) reside in tissues for local maintenance and immune regulation, whereas inflammatory eosinophils (iEos) are rapidly recruited during immune responses to drive inflammation. Initially characterized in murine models, similar eosinophil heterogeneity has since been confirmed in human blood and tissues. Recognizing distinct eosinophil subsets is crucial for understanding their contributions to disease progression. This review highlights recent discoveries in eosinophil diversity and explores therapeutic implications of selectively targeting specific eosinophil subsets in clinical practice.

Open article ↗



2026-07-02 | Case Report: Benralizumab combined with a steroid-sparing strategy in a case of severe eosinophilic granulomatosis with polyangiitis.

Eosinophilic granulomatosis with polyangiitis (EGPA) is a rare anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis characterized by eosinophilic inflammation and necrotizing vasculitis. Benralizumab, an anti-IL-5Rα monoclonal antibody that directly targets eosinophils, was approved for EGPA in China in December 2025. We report an early real-world experience of benralizumab combined with corticosteroids in a patient with severe ANCA-negative EGPA. A 73-year-old male with a history of childhood asthma, chronic rhinosinusitis, CKD stage 4 presented with recurrent pulmonary infiltrates, progressive interstitial lung disease, severe eosinophilia (peak 12.90×109/L), diffuse skin lesion and new-onset arrhythmia. After systematic differential diagnosis, EGPA was diagnosed according to the 2022 ACR/EULAR criteria (score 7). BALF and blood testing detected nucleic acids of Pneumocystis jirovecii and CMV. He was treated with methylprednisolone 40 mg/day and subcutaneous benralizumab 30 mg every 4 weeks. Concurrently, empirical preemptive anti-infective therapy (caspofungin for Pneumocystis jirovecii, ganciclovir for CMV) was initiated. At 4-week follow-up, prednisone was tapered to 30 mg/day; dyspnea and rash markedly improved, eosinophils decreased to 0, IgE fell from 1,860 to 519 IU/mL, chest CT showed significant resolution of infiltrates, and pulmonary function improved from severe to mild restrictive impairment. At 3 months, prednisone was further tapered to 25 mg/day in combination with benralizumab, and the patient remained stable without disease relapse or acute infection. This case represents an early real-world application of benralizumab in a high-risk ANCA-negative EGPA patient following its approval in China. The findings suggest that benralizumab, used as an adjunctive steroid-sparing agent, may facilitate rapid corticosteroid tapering in selected vulnerable patients with infection risk. Long-term follow-up and studies are needed to validate these findings.

Open article ↗



2026-06-30 | Comparative real-world effectiveness and safety of benralizumab and two mepolizumab dosing regimens in eosinophilic granulomatosis with polyangiitis: a 24-month prospective single-center cohort study.

Long-term prospective real-world data on anti-IL-5 and anti-IL-5 receptor biologics in eosinophilic granulomatosis with polyangiitis (EGPA) are limited. In this 24-month prospective single-center observational study, 66 adults with EGPA received benralizumab 30 mg (every 4 weeks for the first three doses, then every 8 weeks), mepolizumab 300 mg every 4 weeks or mepolizumab 100 mg every 4 weeks. Remission was defined as BVASv3 = 0 with prednisone ≤5 mg/day. Glucocorticoid (GC)-free status was defined as no oral GCs. The primary analysis was conducted in patients starting biologics as first-line. To address confounding by indication, we performed multivariable logistic regression and multinomial propensity score inverse probability weighting (IPTW). Analyses were repeated in an all-lines dataset (81 treatment lines) using generalized estimating equations (GEE) clustered by patient. Clinical outcomes improved over the 24-month follow-up across all regimens. By 24 months, remission was 73.7% (14/19) with benralizumab, 81.0% (17/21) with mepolizumab 300 mg, and 50.0% (6/12) with mepolizumab 100 mg, without statistically significant differences between regimens. GC-free status increased over time, reaching 63.2% (12/19) with benralizumab, 85.7% (18/21) with mepolizumab 300 mg, and 58.3% (7/12) with mepolizumab 100 mg at 24 months, with no significant between-group differences. Adjusted and propensity-based sensitivity analyses yielded consistent conclusions. Eosinophils decreased markedly, with near-complete suppression with benralizumab. Pulmonary function indices showed improvement during follow-up, with the clearest within-group improvement observed in the benralizumab group. Treatment persistence at 24 months varied across regimens, although no statistically significant difference was observed (log-rank p=0.222). Discontinuations were mainly driven by inadequate control of ENT or respiratory manifestations. In all-lines GEE analyses, regimen effects on remission and GC-free status were broadly consistent with the primary analysis. Adverse events were uncommon and no serious events were reported. Anti-IL-5/IL-5R biologics were effective and well tolerated over 24 months in EGPA, with substantial GC sparing effect. The observational design and potential confounding by indication limit comparative effectiveness inference. These findings support individualized biologic selection in EGPA.

Open article ↗



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Drug Discovery Landscape

5 orphan drug designations for Eosinophilic granulomatosis with polyangiitis, including 2 approved therapies.

5 orphan drug designations for Eosinophilic granulomatosis with polyangiitis, including 2 approved therapies.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

small molecule, selective Janus kinase (JAK) 1 inhibitor

small molecules

FDA

2025-04-14

NS Pharma, Inc.

Methyl-(1-{[6-{[(1S)-1-cyclopropylethyl]amino}-2-(pyrazolo[5,1-b][1,3]thiazol-7-yl)-pyrimidin-4-yl]carbonyl}piperidin-4-yl)carbamate mono(4-methylbenzenesulfonate)

antibodies

EMA

2024-01-12

Syneos Health Netherlands B.V.

benralizumab [Fasenra]

antibodies

FDA

2018-11-21

2024-09-17

AstraZeneca Pharmaceuticals LP

Mepolizumab

antibodies

EMA

2013-03-12

Glaxosmithkline (Ireland) Limited

mepolizumab [NUCALA]

antibodies

FDA

2011-07-14

2017-12-12

GlaxoSmithKline LLC

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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.

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.