AI Drug Discovery for Pharma and Biotech

Drug discovery

6

drugs

With orphan designations

Overview

Polymyositis (PM) is a rare idiopathic inflammatory myopathy characterized by autoimmune-mediated skeletal muscle inflammation, leading to progressive symmetric proximal muscle weakness. Symptoms include difficulty climbing stairs, rising from seated positions, dysphagia, and fatigue. Systemic complications may involve interstitial lung disease, cardiomyopathy, or arthritis. Diagnosis requires clinical evaluation, elevated muscle enzymes (e.g., CK), electromyography, MRI, and muscle biopsy [1][5][13].

Population

  • Prevalence: 5–22 cases per 100,000 individuals globally, with peak incidence in adults aged 50–70 [2][4].

  • Gender: Female predominance (2:1 ratio), higher prevalence in African Americans [17][12].

  • Comorbidities: Associated with malignancies (e.g., lung, breast) and connective tissue diseases (e.g., lupus, scleroderma) [9][11].

Burden

  • Survival: 75% 5-year survival (PM) vs. 63% (dermatomyositis); median survival 11–12 years [2][4].

  • Complications: Respiratory failure, dysphagia-related aspiration, and malignancy-driven mortality [9][17].

  • Healthcare impact: High disability rates, prolonged immunosuppressive therapy, and frequent hospitalizations [9][19].

Therapies

  • First-line: High-dose corticosteroids (prednisone) ± steroid-sparing immunosuppressants (methotrexate, azathioprine) [3][8].

  • Refractory cases: Biologics (rituximab), IV immunoglobulin, or mycophenolate mofetil [3][18].

  • Supportive care: Physical therapy to preserve muscle function and multidisciplinary management for dysphagia/respiratory involvement [3][13].

Categories: rare neurological diseases, rare renal diseases, rare systemic and rheumatological diseases, rare transplant-related disorders

Research Papers

955 drug discovery papers about Polymyositis, with 2 first-in-class and 7 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

955 drug discovery papers about Polymyositis, with 2 first-in-class and 7 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

2026-06-25 | Acthar Gel treatment in patients with rheumatoid arthritis, systemic lupus erythematosus, or dermatomyositis/polymyositis: analysis of physician-reported charts.

Aim: To examine the characteristics, treatment patterns, and physicians' assessments of outcomes for patients with rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), or dermatomyositis/polymyositis (DM/PM) who received Acthar Gel. Materials & methods: This survey-based medical chart review study asked rheumatologists who met specific inclusion criteria to abstract data from patient records covering the period from April 2022 to November 2024. Eligible patients were adults ≥18 years diagnosed with RA, SLE, or DM/PM, treated with Acthar Gel for ≤24 months. An online questionnaire screened and identified contributing physicians and collected anonymized patient data (baseline demographic, medical history, concomitant medications, Acthar Gel treatment history, and physicians' assessment of health status, symptom severity, treatment outcomes with Acthar Gel). Results: The study population comprised 73 patients with RA (average age 50 years; 49 [67%] female; 44 [60%] White/non-Hispanic), 56 with SLE (average age 42 years; 47 [84%] female; 28 [50%] African-American), and 104 with DM/PM (average age 52 years; 69 [66%] female; 62 [60%] White/non-Hispanic). Patients had received Acthar Gel for an average of 9 (RA) or 8 months (SLE, DM/PM), with most receiving treatment at the time of the study. Per physicians' assessment, health status improved in 68 (93%) patients with RA, 50 (89%) patients with SLE, and 100 (96%) patients with DM/PM after starting treatment with Acthar Gel. The most common treatment goals achieved in patients with improved overall health status were improved overall symptoms, pain, physical function, and corticosteroid use in the RA cohort; overall symptoms, pain, corticosteroid use, and fatigue in the SLE cohort; and overall symptoms, strength, physical function, and corticosteroid use in the DM/PM cohort. Conclusion: These findings support the use of Acthar Gel as a potential treatment option for appropriate patients with RA, SLE, or DM/PM.

Open article ↗



2026-06-19 | JAK inhibitors in areas outside of inflammatory arthritis: focus on connective tissue diseases.

Janus kinase (JAK) inhibitors have emerged as a transformative therapeutic class across autoimmune diseases by targeting multiple cytokine networks implicated in inflammation and fibrosis. Beyond inflammatory arthritis, increasing evidence supports their potential efficacy in connective tissue diseases such as idiopathic inflammatory myopathies, systemic sclerosis, primary Sjögren's disease, and systemic lupus erythematosus. Through modulation of type I/II interferon and interleukin signalling, JAK inhibition exerts broad anti-inflammatory and antifibrotic effects, translating into clinical improvements in cutaneous, articular and pulmonary domains. Early clinical trials and real-world data confirm meaningful responses in refractory disease, though randomized evidence remains limited. Overall, JAK inhibitors represent a promising, mechanistically grounded option for systemic autoimmune diseases, with ongoing studies expected to refine selectivity, indications and long-term safety profiles.

Open article ↗



2026-06-08 | Aggressive Nutrition Therapy in a Rehabilitation Hospital for Severe Tachypnoea and Profound Weight Loss With Interstitial Lung Disease: A Case Report.

A 67-year-old male suffering from polymyositis-associated interstitial lung disease (ILD) was transferred to a rehabilitation hospital. Upon admission, the patient presented with severe malnutrition (weight of 32.0 kg; BMI of 12.10 kg/m2; skeletal muscle index of 3.8 kg/m2) and rapid shallow breathing with pronounced exercise-induced tachypnoea. The patient underwent physiotherapy, which included belt electrode skeletal muscle electrical stimulation and progressively titrated aerobic/resistance training. Following the initial stepwise optimisation for transient poor intake, an aggressive nutrition therapy was started. At discharge, the patient gained 4.6 kg in weight, with an increase in skeletal muscle mass from 13.9 to 15.5 kg. The skeletal muscle index had increased to 4.4 kg/m2, representing an increase of 0.6 kg/m2. Furthermore, the duration of continuous aerobic exercise had increased to 30 min, representing a 27-min increase. This case may present a pragmatic strategy for the implementation of aggressive nutrition therapy during rehabilitation for tachypnoeic ILD.

Open article ↗



2026-06-25 | Acthar Gel treatment in patients with rheumatoid arthritis, systemic lupus erythematosus, or dermatomyositis/polymyositis: analysis of physician-reported charts.

Aim: To examine the characteristics, treatment patterns, and physicians' assessments of outcomes for patients with rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), or dermatomyositis/polymyositis (DM/PM) who received Acthar Gel. Materials & methods: This survey-based medical chart review study asked rheumatologists who met specific inclusion criteria to abstract data from patient records covering the period from April 2022 to November 2024. Eligible patients were adults ≥18 years diagnosed with RA, SLE, or DM/PM, treated with Acthar Gel for ≤24 months. An online questionnaire screened and identified contributing physicians and collected anonymized patient data (baseline demographic, medical history, concomitant medications, Acthar Gel treatment history, and physicians' assessment of health status, symptom severity, treatment outcomes with Acthar Gel). Results: The study population comprised 73 patients with RA (average age 50 years; 49 [67%] female; 44 [60%] White/non-Hispanic), 56 with SLE (average age 42 years; 47 [84%] female; 28 [50%] African-American), and 104 with DM/PM (average age 52 years; 69 [66%] female; 62 [60%] White/non-Hispanic). Patients had received Acthar Gel for an average of 9 (RA) or 8 months (SLE, DM/PM), with most receiving treatment at the time of the study. Per physicians' assessment, health status improved in 68 (93%) patients with RA, 50 (89%) patients with SLE, and 100 (96%) patients with DM/PM after starting treatment with Acthar Gel. The most common treatment goals achieved in patients with improved overall health status were improved overall symptoms, pain, physical function, and corticosteroid use in the RA cohort; overall symptoms, pain, corticosteroid use, and fatigue in the SLE cohort; and overall symptoms, strength, physical function, and corticosteroid use in the DM/PM cohort. Conclusion: These findings support the use of Acthar Gel as a potential treatment option for appropriate patients with RA, SLE, or DM/PM.

Open article ↗



2026-06-19 | JAK inhibitors in areas outside of inflammatory arthritis: focus on connective tissue diseases.

Janus kinase (JAK) inhibitors have emerged as a transformative therapeutic class across autoimmune diseases by targeting multiple cytokine networks implicated in inflammation and fibrosis. Beyond inflammatory arthritis, increasing evidence supports their potential efficacy in connective tissue diseases such as idiopathic inflammatory myopathies, systemic sclerosis, primary Sjögren's disease, and systemic lupus erythematosus. Through modulation of type I/II interferon and interleukin signalling, JAK inhibition exerts broad anti-inflammatory and antifibrotic effects, translating into clinical improvements in cutaneous, articular and pulmonary domains. Early clinical trials and real-world data confirm meaningful responses in refractory disease, though randomized evidence remains limited. Overall, JAK inhibitors represent a promising, mechanistically grounded option for systemic autoimmune diseases, with ongoing studies expected to refine selectivity, indications and long-term safety profiles.

Open article ↗



2026-06-08 | Aggressive Nutrition Therapy in a Rehabilitation Hospital for Severe Tachypnoea and Profound Weight Loss With Interstitial Lung Disease: A Case Report.

A 67-year-old male suffering from polymyositis-associated interstitial lung disease (ILD) was transferred to a rehabilitation hospital. Upon admission, the patient presented with severe malnutrition (weight of 32.0 kg; BMI of 12.10 kg/m2; skeletal muscle index of 3.8 kg/m2) and rapid shallow breathing with pronounced exercise-induced tachypnoea. The patient underwent physiotherapy, which included belt electrode skeletal muscle electrical stimulation and progressively titrated aerobic/resistance training. Following the initial stepwise optimisation for transient poor intake, an aggressive nutrition therapy was started. At discharge, the patient gained 4.6 kg in weight, with an increase in skeletal muscle mass from 13.9 to 15.5 kg. The skeletal muscle index had increased to 4.4 kg/m2, representing an increase of 0.6 kg/m2. Furthermore, the duration of continuous aerobic exercise had increased to 30 min, representing a 27-min increase. This case may present a pragmatic strategy for the implementation of aggressive nutrition therapy during rehabilitation for tachypnoeic ILD.

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

6 orphan drug designations for Polymyositis.

6 orphan drug designations for Polymyositis.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

Glucagon like peptide-1 (GLP-1) Elastin like peptide (ELP)-120 fusion protein

proteins

FDA

2021-08-31

ImmunoForge Co., Ltd.

(2S,3R)-N-[(2S)-3-(cyclopent-1-en-1-yl)-1-[(2R)-2-methyloxiran-2-yl]-1-oxopropan-2-yl]-3-hydroxy-3-(4-methoxyphenyl)-2-[(2S)-2-[2-(morpholin-4-yl)acetamido]propanamido]propanamide maleate

small molecules

FDA

2020-10-21

Kezar Life Sciences, Inc.

Siponimod [BAF312]

small molecules

EMA

2014-11-19

Novartis Europharm Limited

Siponimod

small molecules

FDA

2013-11-26

Novartis Pharmaceuticals Corporation

Human normal immunoglobulin [Gammagen]

antibodies

EMA

2003-10-24

[INACTIVE] Orfagen

Immune globulin intravenous (human)

antibodies

FDA

1992-10-13

Immuno Clinical Research Corp.

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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.