AI Drug Discovery for Pharma and Biotech

Drug discovery

2

drugs

With orphan designations

Overview

Idiopathic nephrotic syndrome (INS) is a glomerular disorder characterized by nephrotic-range proteinuria (≥3.5 g/day), hypoalbuminemia, edema, and hyperlipidemia. It is the most common childhood glomerular disease, primarily caused by minimal change disease (MCD) in children and membranous nephropathy or focal segmental glomerulosclerosis (FSGS) in adults. INS often follows a relapsing-remitting course, requiring long-term immunosuppression and supportive care to manage complications like infections, thrombosis, and chronic kidney disease [1][5][12].

Population

  • Children: Peak onset at 2–6 years; incidence of 2–7/100,000/year, with higher rates in Asian (up to 7.1/100,000) and Black (3.5/100,000) populations compared to Caucasians (1.8/100,000) [2][12][17].

  • Adults: Annual incidence of ~3/100,000; FSGS predominates in Black adults, membranous nephropathy in White adults [1][4][7].

  • Sex: Pediatric cases show a 2:1 male-to-female ratio; no gender predominance in adults [7][12].

Burden

  • Clinical: High relapse rates (60–80% in children), thromboembolic risk (8–17%), and progression to CKD/ESRD in steroid-resistant cases [4][6][16].

  • Economic: Annual direct costs exceed $3,000 per patient; caregivers spend >170 hours/year managing dietary and medical needs [4][9].

  • Psychosocial: 15–20% of caregivers report significant psychological distress due to caregiving demands [9][18].

Therapies

  • First-line: Corticosteroids (e.g., prednisone) induce remission in 80–90% of pediatric MCD cases [3][14].

  • Adjunctive: ACE inhibitors/angiotensin receptor blockers reduce proteinuria; diuretics manage edema [1][5][8].

  • Steroid-resistant/dependent cases: Immunosuppressants (cyclophosphamide, calcineurin inhibitors, rituximab) or alkylating agents for frequent relapses [3][13][16].

Categories: rare renal diseases, rare transplant-related disorders

Research Papers

1,589 drug discovery papers related to Idiopathic nephrotic syndrome, with 5 first-in-class and 3 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

1,589 drug discovery papers related to Idiopathic nephrotic syndrome, with 5 first-in-class and 3 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

2026-07-08 | Obinutuzumab as a primary anti-CD20 agent in pediatric frequently relapsing and steroid-dependent nephrotic syndrome.

Idiopathic nephrotic syndrome (INS) is the leading glomerular disease in children, often responding well to steroids. However, frequently relapsing nephrotic syndrome (FRNS) or steroid-dependent nephrotic syndrome (SDNS) require the introduction of steroid-sparing agents to mitigate steroid toxicity. This retrospective, pilot study including 6 patients with FRNS or SDNS assessed the efficacy and safety of obinutuzumab as a first line of anti-CD20 therapy. Using one dose of obinutuzumab, complete remission was achieved in all 6 patients within 11 months. Remission rate was 100% up to 12 months post-injection, and 80% at both 24 and 56 months. Infusion-related reactions occurred in 83% of the patients, primarily mild and transient; one patient required immunoglobulin supplementation but no severe infections were noted. These preliminary results suggest that obinutuzumab is a promising first-line anti-CD20 therapy for FRNS and SDNS in children, offering sustained remission with a good safety profile.

Open article ↗



2026-07-06 | Use of rituximab and mycophenolate in cortic-dependent nephrotic syndrome in pediatrics

introduction: Idiopathic nephrotic syndrome (INS) in children may present frequent relapses or steroid dependence, with a risk of corticosteroid toxicity.In this context, rituximab (RTX) and mycophenolate mofetil (MMF) were used as alternative therapeutic options.Materials and methods: A prospective observational cohort study was conducted in a pediatric nephrology unit in the Autonomous City of Buenos Aires, including 12 patients with steroid-dependent nephrotic syndrome (SDNS) who received sequential treatment with RTX and MMF.results: Of the 12 patients treated with RTX, 4 achieved sustained remission.The remaining 8 had fewer than 2 relapses per year but required potentially toxic steroid doses; therefore, after a mean period of 13 months post-RTX, MMF was added.All patients remained in remission until the end of the study.The cumulative steroid dose (CSD) pre-RTX was 5335 mg/m 2 and post-RTX was 2024 mg/m 2 (p = 0.001).Finally, the mean CSD with MMF was 1378 mg/m 2 (Wilcoxon test: T = 3.5, p = 0.005).In the 10 patients who experienced at least one relapse, the mean CD20 lymphocyte count one month after RTX was 3.7 cells/mm 3 , and at relapse it was 26 cells/mm 3 (CD20-proteinuria correlation coefficient: 0.54).Observed adverse effects were minimal and reversible.conclusion: Sequential therapy with RTX and MMF may be an option for patients with primary steroid-dependent nephrotic syndrome.

Open article ↗



2026-07-06 | Expression of PD-1 on T cells and its predictive value for steroid resistance in children with idiopathic nephrotic syndrome.

Programmed cell death protein 1 (PD-1) plays a pivotal role in regulating T-cell responses. Its expression profile and clinical significance in pediatric idiopathic nephrotic syndrome (INS), particularly in steroid-resistant nephrotic syndrome (SRNS), remain unclear. PD-1 expression on CD3⁺, CD4⁺, and CD8⁺T cells was quantified by flow cytometry in 65 treatment-naive children with idiopathic nephrotic syndrome (INS): steroid-sensitive (SSNS, n = 54), steroid-resistant (SRNS, n = 11), as well as in healthy controls (HC, n = 30). Group differences, clinical correlations and PD-1's predictive value for SRNS were analyzed. Circulating T subsets, including CD3⁺PD-1⁺ T cells, CD4⁺PD-1⁺T cells, and CD8⁺PD-1⁺T were detected with multiparameter flow cytometry. The percentage of CD4⁺PD-1⁺ T cells was significantly higher in SRNS (10.77 ± 4.62%) compared to both SSNS (5.14 ± 1.99%, P = 0.000) and HC (6.36 ± 2.02%, P = 0.028). CD3⁺PD-1⁺ T cells were also elevated in SRNS (8.13 ± 3.12%) versus SSNS (4.67 ± 1.82%, P = 0.001). No significant difference was found in CD8⁺PD-1⁺ T cells. Multivariate logistic regression identified an elevated percentage of CD4⁺PD-1⁺ T cells as an independent risk factor for SRNS (OR = 2.236, P = 0.002). Receiver operating characteristic (ROC) analysis showed that CD4⁺PD-1⁺ T cell cutoff > 7.385% predicted SRNS with an area under the curve (AUC) of 0.891, sensitivity of 81.8%, and specificity of 87.0%. Overexpression of PD-1, predominantly on CD4⁺ T cells, is associated with steroid resistance in pediatric INS. Elevated CD4⁺PD-1⁺ T cells may serve as a predictive biomarker for SRNS, offering a potential tool for early identification and a rationale for exploring PD-1 pathway modulation.

Open article ↗



2026-07-08 | Obinutuzumab as a primary anti-CD20 agent in pediatric frequently relapsing and steroid-dependent nephrotic syndrome.

Idiopathic nephrotic syndrome (INS) is the leading glomerular disease in children, often responding well to steroids. However, frequently relapsing nephrotic syndrome (FRNS) or steroid-dependent nephrotic syndrome (SDNS) require the introduction of steroid-sparing agents to mitigate steroid toxicity. This retrospective, pilot study including 6 patients with FRNS or SDNS assessed the efficacy and safety of obinutuzumab as a first line of anti-CD20 therapy. Using one dose of obinutuzumab, complete remission was achieved in all 6 patients within 11 months. Remission rate was 100% up to 12 months post-injection, and 80% at both 24 and 56 months. Infusion-related reactions occurred in 83% of the patients, primarily mild and transient; one patient required immunoglobulin supplementation but no severe infections were noted. These preliminary results suggest that obinutuzumab is a promising first-line anti-CD20 therapy for FRNS and SDNS in children, offering sustained remission with a good safety profile.

Open article ↗



2026-07-06 | Use of rituximab and mycophenolate in cortic-dependent nephrotic syndrome in pediatrics

introduction: Idiopathic nephrotic syndrome (INS) in children may present frequent relapses or steroid dependence, with a risk of corticosteroid toxicity.In this context, rituximab (RTX) and mycophenolate mofetil (MMF) were used as alternative therapeutic options.Materials and methods: A prospective observational cohort study was conducted in a pediatric nephrology unit in the Autonomous City of Buenos Aires, including 12 patients with steroid-dependent nephrotic syndrome (SDNS) who received sequential treatment with RTX and MMF.results: Of the 12 patients treated with RTX, 4 achieved sustained remission.The remaining 8 had fewer than 2 relapses per year but required potentially toxic steroid doses; therefore, after a mean period of 13 months post-RTX, MMF was added.All patients remained in remission until the end of the study.The cumulative steroid dose (CSD) pre-RTX was 5335 mg/m 2 and post-RTX was 2024 mg/m 2 (p = 0.001).Finally, the mean CSD with MMF was 1378 mg/m 2 (Wilcoxon test: T = 3.5, p = 0.005).In the 10 patients who experienced at least one relapse, the mean CD20 lymphocyte count one month after RTX was 3.7 cells/mm 3 , and at relapse it was 26 cells/mm 3 (CD20-proteinuria correlation coefficient: 0.54).Observed adverse effects were minimal and reversible.conclusion: Sequential therapy with RTX and MMF may be an option for patients with primary steroid-dependent nephrotic syndrome.

Open article ↗



2026-07-06 | Expression of PD-1 on T cells and its predictive value for steroid resistance in children with idiopathic nephrotic syndrome.

Programmed cell death protein 1 (PD-1) plays a pivotal role in regulating T-cell responses. Its expression profile and clinical significance in pediatric idiopathic nephrotic syndrome (INS), particularly in steroid-resistant nephrotic syndrome (SRNS), remain unclear. PD-1 expression on CD3⁺, CD4⁺, and CD8⁺T cells was quantified by flow cytometry in 65 treatment-naive children with idiopathic nephrotic syndrome (INS): steroid-sensitive (SSNS, n = 54), steroid-resistant (SRNS, n = 11), as well as in healthy controls (HC, n = 30). Group differences, clinical correlations and PD-1's predictive value for SRNS were analyzed. Circulating T subsets, including CD3⁺PD-1⁺ T cells, CD4⁺PD-1⁺T cells, and CD8⁺PD-1⁺T were detected with multiparameter flow cytometry. The percentage of CD4⁺PD-1⁺ T cells was significantly higher in SRNS (10.77 ± 4.62%) compared to both SSNS (5.14 ± 1.99%, P = 0.000) and HC (6.36 ± 2.02%, P = 0.028). CD3⁺PD-1⁺ T cells were also elevated in SRNS (8.13 ± 3.12%) versus SSNS (4.67 ± 1.82%, P = 0.001). No significant difference was found in CD8⁺PD-1⁺ T cells. Multivariate logistic regression identified an elevated percentage of CD4⁺PD-1⁺ T cells as an independent risk factor for SRNS (OR = 2.236, P = 0.002). Receiver operating characteristic (ROC) analysis showed that CD4⁺PD-1⁺ T cell cutoff > 7.385% predicted SRNS with an area under the curve (AUC) of 0.891, sensitivity of 81.8%, and specificity of 87.0%. Overexpression of PD-1, predominantly on CD4⁺ T cells, is associated with steroid resistance in pediatric INS. Elevated CD4⁺PD-1⁺ T cells may serve as a predictive biomarker for SRNS, offering a potential tool for early identification and a rationale for exploring PD-1 pathway modulation.

Open article ↗



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Drug Discovery Landscape

2 orphan drug designations for Idiopathic nephrotic syndrome.

2 orphan drug designations for Idiopathic nephrotic syndrome.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

Pioglitazone hydrochloride

small molecules

FDA

2022-07-20

NephKey Therapeutics, Inc.

Levamisol hydrochloride

small molecules

EMA

2005-10-28

ACE Pharmaceuticals BV

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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.