AI Drug Discovery for Pharma and Biotech

Drug discovery

4

drugs

With orphan designations

Overview

Granulomatosis with polyangiitis (GPA) is a rare ANCA-associated vasculitis characterized by necrotizing inflammation of small-to-medium vessels, granuloma formation, and frequent involvement of the upper/lower respiratory tracts and kidneys [1][4][19]. Diagnosis relies on clinical presentation, ANCA serology (primarily PR3-ANCA), and histopathology [10][19]. Untreated mortality exceeds 80%, but modern regimens reduce this to ~10% [4][19].

Population

  • Incidence: 3-14.4 cases/million annually in Europe, 12.8/million in U.S. working-age adults [2][12][16]

  • Demographics: Peak onset 40-65 years; slight male predominance (1.2:1). More common in Caucasians (~90% of cases) [2][7][19]

  • Pediatric: Rare (1.8/million incidence), higher hematologic complications vs adults [12][19]

Burden

  • Morbidity: 35-44% relapse rate; chronic kidney disease in 20-25%, hearing loss in 22-42% [4][6][12]

  • Mortality: 10% at 5 years (vs 80% untreated), often from infections or cardiovascular events [4][19]

  • Economic: Annual costs ~$78,000/patient with relapses vs $27,000 in remission [9]. Pediatric patients incur 2-3× higher hospitalization rates [12].

Therapies

  • Induction: High-dose glucocorticoids + rituximab (preferred) or cyclophosphamide for severe disease [1][3][19]

  • Maintenance: Rituximab monotherapy or methotrexate/azathioprine for 12-24 months [1][19]

  • Emerging: Avacopan (complement C5a inhibitor) reduces steroid exposure [1], plasma exchange reserved for refractory cases [1][19]

Categories: 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

1,991 drug discovery papers about Granulomatosis with polyangiitis, with 2 first-in-class and 2 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

1,991 drug discovery papers about Granulomatosis with polyangiitis, with 2 first-in-class and 2 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 | Necrotizing Scleritis in Granulomatosis With Polyangiitis: A Clinical Challenge for an Ophthalmologists.

Granulomatosis with polyangiitis (GPA) is a granulomatous disease with multisystem involvement, frequently with ocular manifestations. It can lead to ocular morbidity due to tissue melting and necrosis. A 35-year-old male presented with a painless nodular lesion in the left eye, along with gradual diminution of vision and redness for 2 months. Eight months ago, he was diagnosed with GPA and left eye anterior uveitis. He was treated with topical steroids, topical cycloplegic, pulse cyclophosphamide, oral prednisolone, and azathioprine. At presentation, best corrected visual acuity in the right eye was 6/6 and 1/60 in the left. Left eye showed scleral thinning and necrosis in the superonasal quadrant with +1 cells in the anterior chamber. Intraocular pressure was 10 mmHg. Funduscopy examination showed exudative retinal detachment with hyperemic disc. Since refractory to topical and systemic immunomodulators, he underwent corneoscleral patch graft in the left eye for the progressive scleral thinning along with injection rituximab. Significant improvement was seen at 1 month with corneoscleral patch graft in situ, decreased inflammation with formed anterior chamber depth. However, at 2 months follow-up, the left eye showed worsening of ocular symptoms with progressive scleral thinning, necrosis, and graft lysis with total retinal detachment. GPA-associated necrotizing scleritis is challenging. A collaborative timely management with aggressive compliant immunosuppressive therapy is a necessity to avoid ocular morbidity.

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 ↗



2026-07-11 | 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 | Necrotizing Scleritis in Granulomatosis With Polyangiitis: A Clinical Challenge for an Ophthalmologists.

Granulomatosis with polyangiitis (GPA) is a granulomatous disease with multisystem involvement, frequently with ocular manifestations. It can lead to ocular morbidity due to tissue melting and necrosis. A 35-year-old male presented with a painless nodular lesion in the left eye, along with gradual diminution of vision and redness for 2 months. Eight months ago, he was diagnosed with GPA and left eye anterior uveitis. He was treated with topical steroids, topical cycloplegic, pulse cyclophosphamide, oral prednisolone, and azathioprine. At presentation, best corrected visual acuity in the right eye was 6/6 and 1/60 in the left. Left eye showed scleral thinning and necrosis in the superonasal quadrant with +1 cells in the anterior chamber. Intraocular pressure was 10 mmHg. Funduscopy examination showed exudative retinal detachment with hyperemic disc. Since refractory to topical and systemic immunomodulators, he underwent corneoscleral patch graft in the left eye for the progressive scleral thinning along with injection rituximab. Significant improvement was seen at 1 month with corneoscleral patch graft in situ, decreased inflammation with formed anterior chamber depth. However, at 2 months follow-up, the left eye showed worsening of ocular symptoms with progressive scleral thinning, necrosis, and graft lysis with total retinal detachment. GPA-associated necrotizing scleritis is challenging. A collaborative timely management with aggressive compliant immunosuppressive therapy is a necessity to avoid ocular morbidity.

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 ↗



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Drug Discovery Landscape

4 orphan drug designations for Granulomatosis with polyangiitis, including 1 approved therapy.

4 orphan drug designations for Granulomatosis with polyangiitis, including 1 approved therapy.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

(2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide [Tavneos]

small molecules

EMA

2014-11-19

2022-01-19

Vifor Fresenius Medical Care Renal Pharma France

gusperimus trihydrochloride

small molecules

FDA

2011-06-29

Nordic Group B.V.

Gusperimus trihydrochloride

small molecules

EMA

2001-03-29

Nordic Group B.V.

Etanercept

proteins

FDA

1999-04-06

Immunex Corporation

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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.