AI Drug Discovery for Pharma and Biotech

Drug discovery

18

drugs

With orphan designations

Overview

Autoimmune Hemolytic Anemia (AIHA) is a rare immune disorder characterized by autoantibody-mediated destruction of red blood cells, leading to anemia. It is classified as warm AIHA (IgG antibodies active at 37°C) or cold AIHA (IgM antibodies reactive at lower temperatures). Symptoms range from fatigue, pallor, and jaundice to severe complications like thrombosis or organ failure. Diagnosis relies on direct antiglobulin (Coombs) testing, with subtyping guiding management [1][6][11].

Population

  • Annual incidence: 1–3/100,000 [1][11]; prevalence ~1 in 8,000 [9][13].

  • Predominantly affects females (60%) and individuals >40 years [7][11][15].

  • Secondary AIHA (49% of cases) links to hematologic malignancies, autoimmune diseases (e.g., SLE), or infections [2][7][11].

Burden

  • One-year mortality: 17.9% (primary AIHA) to 28.4% (secondary AIHA) [2][15].

  • High healthcare utilization: 48% of severe cases require hospitalization; 55% need transfusions [4][2][15].

  • Long-term risks: Cardiovascular events, chronic immunosuppression-related infections, and reduced quality of life [2][15][16].

Therapies

  • First-line: Corticosteroids (prednisone) with response rates of 70–85% [8][12][14].

  • Refractory/relapsed cases: Rituximab (overall response: 75–90%), splenectomy (long-term remission in ~66%), or immunosuppressants (azathioprine, cyclosporine) [8][12][14].

  • Cold AIHA: Cold avoidance, rituximab, or complement inhibitors (e.g., sutimlimab under investigation) [6][14][16].

Categories: rare hematological diseases

Research Papers

1,473 drug discovery papers about Autoimmune hemolytic anemia, with 2 first-in-class and 22 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

1,473 drug discovery papers about Autoimmune hemolytic anemia, with 2 first-in-class and 22 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

2026-07-08 | Sutimlimab for cold agglutinin disease: an updated perspective from approval to real-world clinical treatment.

Sutimlimab, a classical complement pathway (CP) inhibitor, was approved in 2022 in the US, EU, and Japan for the treatment of cold agglutinin disease (CAD), a rare form of autoimmune hemolytic anemia (AIHA) characterized by CP‑mediated extravascular hemolysis and circulatory symptoms related to IgM‑mediated red blood cell (RBC) agglutination. This review reexamines the clinical trial data and compares those findings with published real‑world experience (RWE), providing clinicians with efficacy and safety data that extend beyond the clinical trial experience in this rare AIHA. The first-in-human trials, the two seminal clinical trials (CARDINAL and CADENZA), and the published post‑marketing RWE are presented. Literature searches for CAD and sutimlimab were conducted in PubMed and in abstracts from ASH and EHA from 2016 to present. All articles and abstracts regarding sutimlimab and CAD were included. Long-term data from clinical trials and published RWE support the safety and efficacy of sutimlimab. Targeting the CP as primary therapy for CAD offers a unique, targeted management strategy that minimizes exposure to immunosuppressive regimens. The rapid onset of sutimlimab's activity provides a potentially lifesaving treatment option in situations where immediate control of hemolysis is critical. Opportunities remain to increase our understanding of the role of complement inhibition combined with immunosuppressive therapy in CAD. The impact of complement inhibition on morbidity and mortality in CAD remains to be determined.

Open article ↗



2026-07-06 | Immune cell-derived membrane nanovesicles: A promethean fire for autoimmune disease therapy through immune cell mimicry.

Autoimmune diseases (AIDs) constitute a heterogeneous group of disorders characterized by immune dysregulation, loss of self-tolerance, and chronic inflammation, which leads to tissue damage and organ dysfunction. Current therapies for AIDs are often limited by their lack of specificity, systemic side effects, and insufficient restoration of immune tolerance. Recent advances in nanotechnology and bioengineering have introduced immune and associated cell-derived membrane vesicles (IACMVs) as a promising therapeutic platform. Derived from macrophages, dendritic cells, neutrophils, platelets, or red blood cells, IACMVs inherit key surface proteins and receptors from their parent cells, conferring endogenous biocompatibility, inflammation-specific targeting, and intrinsic immunomodulatory capabilities. These vesicles can be engineered to carry therapeutic cargoes (e.g., peptide inhibitors, nucleic acids) or modified with surface ligands to enhance disease-site specificity, making them versatile tools for specific immunomodulation. This review provides a comprehensive overview of IACMVs, focusing on their preparation techniques, functional mechanisms, and therapeutic applications in prototypical AIDs such as rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), inflammatory bowel disease (IBD), autoimmune hemolytic anemia (AIHA), type 1 diabetes (T1D), multiple sclerosis (MS), and autoimmune myocarditis (AM). We highlight translational challenges, including production scalability, membrane integrity, immunogenicity, and cargo-loading efficiency, that must be addressed to advance clinical translation. Finally, we discuss future directions for optimizing IACMVs as next-generation, safe, and targeted immunotherapeutic platforms for AIDs.

Open article ↗



2026-06-30 | Autoimmune Hemolytic Anemia Associated With Leptospirosis: A Rare Complication

Introduction Autoimmune hemolytic anemia (AIHA) is rare but potentially life-threatening complication of leptospirosis, a zoonotic infection caused by the Leptospira species. Autoimmune hemolytic anemia occurs when the immune system targets red blood cells, resulting in hemolysis and anemia. The relationship between leptospirosis and AIHA is uncommon and often under-recognized, which can delay diagnosis and management. This case highlights the importance of considering hematologic complications in leptospirosis. Case Presentation A 23-year-old male presented with low-grade fever, productive cough, shortness of breath, jaundice, and pallor for 15 days. On examination, he was pale and icteric with bilateral chest crepitations. Laboratory studies showed severe anemia (hemoglobin 5.5 g/dL), elevated lactate dehydrogenase (1027 U/L), hyperbilirubinemia, thrombocytopenia, and peripheral smear evidence of hemolysis. A direct Coombs’ test was strongly positive. Leptospira immunoglobulin M serology confirmed the diagnosis. The patient was managed with broad-spectrum antibiotics (piperacillin–tazobactam and doxycycline) and high-dose corticosteroids (1 mg/kg/day). He improved clinically with stabilization of hemoglobin and a resolution of symptoms. Conclusion This case demonstrates a rare immune-mediated manifestation of leptospirosis presenting as AIHA without hepatic or renal failure. Clinicians should maintain a high index of suspicion for hematologic complications in leptospirosis. Early recognition and timely initiation of corticosteroid therapy alongside antibiotics can improve patient outcomes.

Open article ↗



2026-07-08 | Sutimlimab for cold agglutinin disease: an updated perspective from approval to real-world clinical treatment.

Sutimlimab, a classical complement pathway (CP) inhibitor, was approved in 2022 in the US, EU, and Japan for the treatment of cold agglutinin disease (CAD), a rare form of autoimmune hemolytic anemia (AIHA) characterized by CP‑mediated extravascular hemolysis and circulatory symptoms related to IgM‑mediated red blood cell (RBC) agglutination. This review reexamines the clinical trial data and compares those findings with published real‑world experience (RWE), providing clinicians with efficacy and safety data that extend beyond the clinical trial experience in this rare AIHA. The first-in-human trials, the two seminal clinical trials (CARDINAL and CADENZA), and the published post‑marketing RWE are presented. Literature searches for CAD and sutimlimab were conducted in PubMed and in abstracts from ASH and EHA from 2016 to present. All articles and abstracts regarding sutimlimab and CAD were included. Long-term data from clinical trials and published RWE support the safety and efficacy of sutimlimab. Targeting the CP as primary therapy for CAD offers a unique, targeted management strategy that minimizes exposure to immunosuppressive regimens. The rapid onset of sutimlimab's activity provides a potentially lifesaving treatment option in situations where immediate control of hemolysis is critical. Opportunities remain to increase our understanding of the role of complement inhibition combined with immunosuppressive therapy in CAD. The impact of complement inhibition on morbidity and mortality in CAD remains to be determined.

Open article ↗



2026-07-06 | Immune cell-derived membrane nanovesicles: A promethean fire for autoimmune disease therapy through immune cell mimicry.

Autoimmune diseases (AIDs) constitute a heterogeneous group of disorders characterized by immune dysregulation, loss of self-tolerance, and chronic inflammation, which leads to tissue damage and organ dysfunction. Current therapies for AIDs are often limited by their lack of specificity, systemic side effects, and insufficient restoration of immune tolerance. Recent advances in nanotechnology and bioengineering have introduced immune and associated cell-derived membrane vesicles (IACMVs) as a promising therapeutic platform. Derived from macrophages, dendritic cells, neutrophils, platelets, or red blood cells, IACMVs inherit key surface proteins and receptors from their parent cells, conferring endogenous biocompatibility, inflammation-specific targeting, and intrinsic immunomodulatory capabilities. These vesicles can be engineered to carry therapeutic cargoes (e.g., peptide inhibitors, nucleic acids) or modified with surface ligands to enhance disease-site specificity, making them versatile tools for specific immunomodulation. This review provides a comprehensive overview of IACMVs, focusing on their preparation techniques, functional mechanisms, and therapeutic applications in prototypical AIDs such as rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), inflammatory bowel disease (IBD), autoimmune hemolytic anemia (AIHA), type 1 diabetes (T1D), multiple sclerosis (MS), and autoimmune myocarditis (AM). We highlight translational challenges, including production scalability, membrane integrity, immunogenicity, and cargo-loading efficiency, that must be addressed to advance clinical translation. Finally, we discuss future directions for optimizing IACMVs as next-generation, safe, and targeted immunotherapeutic platforms for AIDs.

Open article ↗



2026-06-30 | Autoimmune Hemolytic Anemia Associated With Leptospirosis: A Rare Complication

Introduction Autoimmune hemolytic anemia (AIHA) is rare but potentially life-threatening complication of leptospirosis, a zoonotic infection caused by the Leptospira species. Autoimmune hemolytic anemia occurs when the immune system targets red blood cells, resulting in hemolysis and anemia. The relationship between leptospirosis and AIHA is uncommon and often under-recognized, which can delay diagnosis and management. This case highlights the importance of considering hematologic complications in leptospirosis. Case Presentation A 23-year-old male presented with low-grade fever, productive cough, shortness of breath, jaundice, and pallor for 15 days. On examination, he was pale and icteric with bilateral chest crepitations. Laboratory studies showed severe anemia (hemoglobin 5.5 g/dL), elevated lactate dehydrogenase (1027 U/L), hyperbilirubinemia, thrombocytopenia, and peripheral smear evidence of hemolysis. A direct Coombs’ test was strongly positive. Leptospira immunoglobulin M serology confirmed the diagnosis. The patient was managed with broad-spectrum antibiotics (piperacillin–tazobactam and doxycycline) and high-dose corticosteroids (1 mg/kg/day). He improved clinically with stabilization of hemoglobin and a resolution of symptoms. Conclusion This case demonstrates a rare immune-mediated manifestation of leptospirosis presenting as AIHA without hepatic or renal failure. Clinicians should maintain a high index of suspicion for hematologic complications in leptospirosis. Early recognition and timely initiation of corticosteroid therapy alongside antibiotics can improve patient outcomes.

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

18 orphan drug designations for Autoimmune hemolytic anemia, including 2 approved therapies.

18 orphan drug designations for Autoimmune hemolytic anemia, including 2 approved therapies.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

rituximab

antibodies

FDA

2025-07-18

ODDIFACT SAS

Bispecific humanized IgG4 BCMA-directed CD3 T cell engager antibody

antibodies

FDA

2025-06-25

Lakefront Biotherapeutics West LLC

rilzabrutinib

small molecules

FDA

2025-02-20

Sanofi US Services Inc.

Rilzabrutinib

small molecules

EMA

2025-01-16

Sanofi B.V.

rituximab

antibodies

FDA

2023-02-07

Mabion S.A.

a humanized IgG4 monoclonal antibody, produced in CHO cells, that binds to and inhibits the activated form of the classical complement pathway (CP) specific serine protease, C1s

antibodies

FDA

2023-01-12

Bioverativ USA Inc

Parsaclisib

small molecules

EMA

2022-07-18

Incyte Biosciences Distribution B.V.

Humanised IgG4 monoclonal antibody against active complement component 1, subcomponent s

antibodies

EMA

2022-07-18

Sanofi B.V.

rituximab

antibodies

FDA

2020-11-12

Taxon Therapeutics Ltd.

parsaclisib

small molecules

FDA

2020-07-28

Incyte Corporation

nipocalimab

antibodies

FDA

2019-12-05

Janssen Research & Development, LLC

Recombinant complement-specific multimerized human IgG1 Fc

proteins

FDA

2019-10-21

Gliknik, Inc.

pegcetacoplan

peptides

FDA

2019-02-01

Apellis Pharmaceuticals, Inc.

fostamatinib disodium

small molecules

FDA

2018-01-31

Rigel Pharmaceuticals, INc.

sutimlimab-jome [Enjaymo]

antibodies

FDA

2016-07-27

2022-02-04

Recordati Rare Diseases Inc.

Humanised IgG4 monoclonal antibody against total complement component 1, subcomponent s [Enjaymo]

antibodies

EMA

2016-02-17

2022-11-23

Recordati Rare Diseases

Revimmune

antibodies

FDA

2011-02-18

Accentia Biopharmaceuticals

Epoetin alpha

FDA

1989-03-07

R. W. Johnson Pharmaceutical Research Institute

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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.