AI Drug Discovery for Pharma and Biotech

Drug discovery

5

drugs

With orphan designations

Overview

Angioimmunoblastic T-cell lymphoma (AITL) is an aggressive peripheral T-cell lymphoma derived from follicular helper T-cells, characterized by systemic symptoms, immune dysregulation, and frequent advanced-stage presentation at diagnosis. Histopathology reveals polymorphic infiltrates, proliferative vasculature, and CD10/CXCL13-positive malignant T-cells. Despite CHOP-based induction and autologous stem cell transplant consolidation, relapse rates remain high, with a 5-year survival of 30-35% [1][3][11][15].

Population

Median age 65-70 years, male predominance (53-55%), with Hispanics diagnosed 4-5 years earlier than non-Hispanics [2][4][11].

Burden

  • 5-year survival ≤35%, median overall survival 22-24 months [11][13][15].

  • 80-90% present with stage III/IV disease; urban areas show higher incidence but better survival due to healthcare access [4][11][17].

  • High relapse rates (>50%) and increased risk of second malignancies (8% incidence) [4][17].

Therapies

  • First-line: CHOP/CHOEP ± autologous transplant; CD30-positive cases may use brentuximab vedotin combinations [1][3][16].

  • Relapsed/refractory: Epigenetic agents (HDAC inhibitors, hypomethylating agents), PI3K inhibitors, or allogeneic transplant [3][8][15].

  • Emerging options: Lenalidomide-CHOEP trials (#NCT02561273) and targeted immunotherapy [1][3].

Categories: rare hematological diseases, rare neoplastic diseases, rare transplant-related disorders

Research Papers

722 drug discovery papers related to Angioimmunoblastic T-cell lymphoma, with 3 first-in-class and 13 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

722 drug discovery papers related to Angioimmunoblastic T-cell lymphoma, with 3 first-in-class and 13 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

2026-06-25 | Spiegler-Fendt Sarcoid: A Case Report

A 79-year-old male presented with pruritic erythematous papules and plaques on chest and upper back of 2 weeks duration, along with history of bilateral enlarged cervical lymph nodes for 1 month. Skin biopsy showed dense interstitial lymphohistiocytic infiltration and occasional plasma cell infiltration consistent with Spiegler-Fendt Sarcoid (Lymphocytoma Cutis), while lymph node biopsy confirmed concurrent Angioimmunoblastic T-cell Lymphoma (AITL). The patient showed excellent response to methotrexate and narrowband UVB therapy for cutaneous lesions alongside chemotherapy for AITL. This rare case highlights the diagnostic challenges in distinguishing benign pseudolymphoma from malignant lymphoma and emphasizes the importance of comprehensive evaluation in atypical presentations. The concurrent occurrence of these conditions in an elderly male expands the known demographic spectrum of Spiegler-Fendt Sarcoid.

Open article ↗



2026-06-09 | TFH phenotype and response to tucidinostat in relapsed/refractory PTCL: analysis of patients from a phase IIb trial.

T follicular helper (TFH)-derived peripheral T-cell lymphomas (PTCLs) harbor frequent mutations in epigenetic regulators and are sensitive to epigenetic therapies. The histone deacetylase inhibitor tucidinostat demonstrated efficacy in relapsed/refractory PTCL, especially angioimmunoblastic T-cell lymphoma (AITL), in a phase IIb trial; however, the lack of TFH phenotyping has prevented assessment of the predictive value of this phenotype across a broader PTCL spectrum. Therefore, we retrospectively analyzed patients originally diagnosed with AITL or PTCL not otherwise specified (NOS) based on 2008 World Health Organization criteria who participated in the aforementioned trial. TFH phenotype was defined as the expression of ≥ 2 TFH markers. Among the 23 evaluable patients, the TFH group (n = 17) included six with AITL and 11 with PTCL-NOS exhibiting TFH features, and the non-TFH group included six patients with PTCL-NOS. The objective response rate was numerically higher in the TFH group (12/17, 70.6%) than in the non-TFH group (2/6, 33.3%; P = 0.162). Three patients with the TFH phenotype maintained a progression-free survival exceeding 3 years. These findings highlight the potential long-term benefit of tucidinostat in patients with TFH-derived PTCL, supporting the distinct susceptibility of this subtype to epigenetic modulation.

Open article ↗



2026-05-28 | Association of facility type with overall survival in older adults with angioimmunoblastic T-cell lymphoma: A National Cancer Database analysis.

e19074 Background: Angioimmunoblastic T cell lymphoma (AITL) is a distinct and aggressive subtype of peripheral T cell lymphoma characterized by immune dysregulation and frequent presentation with advanced stage disease. Predominantly affects older adults and is associated with inferior survival outcomes compared with many B cell lymphomas, despite advances in clinical practice. Although therapeutic advances and supportive care strategies have improved outcomes across lymphoma subtypes, patients aged ≥75 y remain underrepresented in clinical trials, resulting in limited real world data to guide management in this population. Advanced age is often accompanied by higher comorbidity burden and barriers to specialized care that may influence outcomes independently of disease biology. The impact of treatment facility type on outcomes in elderly patients with AITL is not well defined. Methods: We conducted a retrospective analysis of patients aged ≥75 y diagnosed with AITL in the US between 2004-2022 using the National Cancer Database. Demographic, socioeconomic, clinical, treatment, and survival characteristics were compared between patients treated at Academic Cancer Programs (ACP) and Community Cancer Programs (CCP). Kaplan-Meier and Cox regression analyses were used to compare overall survival (OS) between the two cohorts. Variables used for adjustment included age, ethnicity, insurance status, distance from hospital, and Charlson-Deyo comorbidity score. Results: A total of 2,117 patients aged ≥75 y with AITL were identified including 1,112 treated at ACP and 1,005 treated at CCP. Median age was 80 y in both cohorts, and most patients presented with advanced stage. The majority were insured through Medicare, and baseline comorbidity burden was substantial and similar across treatment settings. Patients treated at ACP were more likely to receive treatment, while a high proportion of patients treated at CCP (45%) had no treatment given or had unknown treatment status. Time from diagnosis to initiation of chemotherapy was significantly shorter at ACP compared with CCP.OS favored patients treated at ACP, with higher two year (37% vs 30%), five year (24% vs 17%), and ten year (11% vs 5%) survival compared with CCP. Conclusions: In this national cohort of patients aged ≥75 y with AITL,OS differed significantly by treatment facility type, with a statistically significant and clinically meaningful survival advantage observed among patients treated at ACP. These findings underscore persistent disparities in treatment delivery and early mortality in this vulnerable population and support efforts to strengthen referral pathways to ACP or develop structured partnerships between CCP and ACP. Such strategies may facilitate timely access to specialized care, multidisciplinary expertise, and clinical trials for older adults with aggressive T-cell lymphomas.

Open article ↗



2026-06-25 | Spiegler-Fendt Sarcoid: A Case Report

A 79-year-old male presented with pruritic erythematous papules and plaques on chest and upper back of 2 weeks duration, along with history of bilateral enlarged cervical lymph nodes for 1 month. Skin biopsy showed dense interstitial lymphohistiocytic infiltration and occasional plasma cell infiltration consistent with Spiegler-Fendt Sarcoid (Lymphocytoma Cutis), while lymph node biopsy confirmed concurrent Angioimmunoblastic T-cell Lymphoma (AITL). The patient showed excellent response to methotrexate and narrowband UVB therapy for cutaneous lesions alongside chemotherapy for AITL. This rare case highlights the diagnostic challenges in distinguishing benign pseudolymphoma from malignant lymphoma and emphasizes the importance of comprehensive evaluation in atypical presentations. The concurrent occurrence of these conditions in an elderly male expands the known demographic spectrum of Spiegler-Fendt Sarcoid.

Open article ↗



2026-06-09 | TFH phenotype and response to tucidinostat in relapsed/refractory PTCL: analysis of patients from a phase IIb trial.

T follicular helper (TFH)-derived peripheral T-cell lymphomas (PTCLs) harbor frequent mutations in epigenetic regulators and are sensitive to epigenetic therapies. The histone deacetylase inhibitor tucidinostat demonstrated efficacy in relapsed/refractory PTCL, especially angioimmunoblastic T-cell lymphoma (AITL), in a phase IIb trial; however, the lack of TFH phenotyping has prevented assessment of the predictive value of this phenotype across a broader PTCL spectrum. Therefore, we retrospectively analyzed patients originally diagnosed with AITL or PTCL not otherwise specified (NOS) based on 2008 World Health Organization criteria who participated in the aforementioned trial. TFH phenotype was defined as the expression of ≥ 2 TFH markers. Among the 23 evaluable patients, the TFH group (n = 17) included six with AITL and 11 with PTCL-NOS exhibiting TFH features, and the non-TFH group included six patients with PTCL-NOS. The objective response rate was numerically higher in the TFH group (12/17, 70.6%) than in the non-TFH group (2/6, 33.3%; P = 0.162). Three patients with the TFH phenotype maintained a progression-free survival exceeding 3 years. These findings highlight the potential long-term benefit of tucidinostat in patients with TFH-derived PTCL, supporting the distinct susceptibility of this subtype to epigenetic modulation.

Open article ↗



2026-05-28 | Association of facility type with overall survival in older adults with angioimmunoblastic T-cell lymphoma: A National Cancer Database analysis.

e19074 Background: Angioimmunoblastic T cell lymphoma (AITL) is a distinct and aggressive subtype of peripheral T cell lymphoma characterized by immune dysregulation and frequent presentation with advanced stage disease. Predominantly affects older adults and is associated with inferior survival outcomes compared with many B cell lymphomas, despite advances in clinical practice. Although therapeutic advances and supportive care strategies have improved outcomes across lymphoma subtypes, patients aged ≥75 y remain underrepresented in clinical trials, resulting in limited real world data to guide management in this population. Advanced age is often accompanied by higher comorbidity burden and barriers to specialized care that may influence outcomes independently of disease biology. The impact of treatment facility type on outcomes in elderly patients with AITL is not well defined. Methods: We conducted a retrospective analysis of patients aged ≥75 y diagnosed with AITL in the US between 2004-2022 using the National Cancer Database. Demographic, socioeconomic, clinical, treatment, and survival characteristics were compared between patients treated at Academic Cancer Programs (ACP) and Community Cancer Programs (CCP). Kaplan-Meier and Cox regression analyses were used to compare overall survival (OS) between the two cohorts. Variables used for adjustment included age, ethnicity, insurance status, distance from hospital, and Charlson-Deyo comorbidity score. Results: A total of 2,117 patients aged ≥75 y with AITL were identified including 1,112 treated at ACP and 1,005 treated at CCP. Median age was 80 y in both cohorts, and most patients presented with advanced stage. The majority were insured through Medicare, and baseline comorbidity burden was substantial and similar across treatment settings. Patients treated at ACP were more likely to receive treatment, while a high proportion of patients treated at CCP (45%) had no treatment given or had unknown treatment status. Time from diagnosis to initiation of chemotherapy was significantly shorter at ACP compared with CCP.OS favored patients treated at ACP, with higher two year (37% vs 30%), five year (24% vs 17%), and ten year (11% vs 5%) survival compared with CCP. Conclusions: In this national cohort of patients aged ≥75 y with AITL,OS differed significantly by treatment facility type, with a statistically significant and clinically meaningful survival advantage observed among patients treated at ACP. These findings underscore persistent disparities in treatment delivery and early mortality in this vulnerable population and support efforts to strengthen referral pathways to ACP or develop structured partnerships between CCP and ACP. Such strategies may facilitate timely access to specialized care, multidisciplinary expertise, and clinical trials for older adults with aggressive T-cell lymphomas.

Open article ↗



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Drug Discovery Landscape

5 orphan drug designations for Angioimmunoblastic T-cell lymphoma.

5 orphan drug designations for Angioimmunoblastic T-cell lymphoma.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

allogeneic chimeric antigen receptor (CAR) T-cell therapy targeting CD70

cell therapies

FDA

2025-09-12

Nanjing Miracle Biotechnology Co., Ltd.

imiquimod-ferulic acid

small molecules

FDA

2025-05-25

Celista Pharmaceuticals LLC

4-[[(1S)-1-(4, 8-dichloro-1-oxo-2-phenyl-3-isoquinolyl) ethyl] amino]-8H-pyrido[2,3-d] pyrimidin-5-one]

small molecules

FDA

2024-01-03

Boryung Co., Ltd.

nanatinostat and valganciclovir

small molecules

FDA

2019-03-15

Viracta Therapeutics, Inc.

brentuximab vedotin

antibodies

FDA

2013-09-13

Seattle Genetics, Inc.

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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.