AI Drug Discovery for Pharma and Biotech

Drug discovery

2

drugs

With orphan designations

Overview

B-lymphoblastic leukemia/lymphoma (B-ALL/LBL) with recurrent genetic abnormalities is an aggressive hematologic malignancy characterized by specific cytogenetic and molecular alterations that dictate prognosis and therapeutic approaches. These include high-risk variants like BCR::ABL1 (Philadelphia chromosome) and KMT2A rearrangements, as well as favorable-risk abnormalities such as ETV6::RUNX1 fusion. Diagnosis requires immunophenotyping and genetic profiling to guide risk stratification [1][2][5].

Population

  • Bimodal age distribution: peak in children <6 years (75% of cases) and adults >65 years [1][6]

  • Accounts for 25% of adult ALL cases and 20-30% of pediatric relapsed/refractory ALL [5][9]

  • Higher incidence in Hispanics and Native Americans with CRLF2 abnormalities [5][10]

Burden

  • 5-year survival: >90% in children vs 40% in adults [10][14]

  • Accounts for 1.2% of lymphoid neoplasms with 1.6 cases/100,000 annually [1][14]

  • Adult mortality exceeds 80% in relapsed cases despite novel therapies [7][15]

Therapies

  • Targeted therapies: Tyrosine kinase inhibitors (e.g., imatinib) for BCR::ABL1+ cases; menin inhibitors for KMT2A-rearranged disease [3][7][15]

  • Immunotherapy: CD19-directed CAR T-cells (tisagenlecleucel) and blinatumomab for relapsed/refractory cases [7][11]

  • Transplant: Allogeneic HSCT recommended for high-risk genetic profiles (e.g., hypodiploidy, KMT2A rearrangements) [4][16]

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

Research Papers

249 drug discovery papers about B-lymphoblastic leukemia/lymphoma with recurrent genetic abnormality, with 2 first-in-class and 3 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

249 drug discovery papers about B-lymphoblastic leukemia/lymphoma with recurrent genetic abnormality, with 2 first-in-class and 3 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

2024-04-02 | Calaspargase Pegol (Asparlas)

CADTH reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class.

The assessments inform non-binding recommendations that help guide the reimbursement decisions of Canada's federal, provincial, and territorial governments, with the exception of Quebec.

This review assesses calaspargase pegol (Asparlas), 3,750 units/5 mL (750 units/mL), concentrate for solution for IV infusion

Indication : As a component of a multiagent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) in pediatric and young adult patients aged 1 year to 21 years


Open article ↗



2024-03-21 | Different Levels of Therapeutic Strategies to Recover the Microbiome to Prevent/Delay Acute Lymphoblastic Leukemia (ALL) or Arrest Its Progression in Children

Alterations in the composition and diversity, metabolism, and products of the microbiome, and of the gut microbiome (GM), have been shown to be closely associated with the onset and progression of many human diseases, including cancer (i.e. hematological neoplasms). Acute lymphoblastic leukemia (ALL) is the most common form of childhood malignancy. Affected cases present typical alterations of GM, followed by inflammation, which contribute to its progression, response to therapy, as well as possible relapses. Recent evidence also reports that GM influences the development and functions of the newborn&#039;s hematopoietic system through the process of developmental programming during fetal life, as well as its susceptibility to the onset of onco-hematological pathologies, namely ALL. Furthermore, in children with ALL, GM has been found to vary in composition, variety, and functions during the clinical stages of ALL, and such variation may influence and predict the complications and prognosis of ALL after chemotherapy treatment or stem cell hematopoietic transplant. Here, we suggest some therapeutic strategies, which can be applied at two levels of intervention to recover the microbiome, and consequently prevent/delay ALL or arrest its progression.

Open article ↗



2024-02-21 | Biological Markers of High-Risk Childhood Acute Lymphoblastic Leukemia

Childhood acute lymphoblastic leukemia (ALL) has witnessed substantial improvements in prognosis; however, a subset of patients classified as high-risk continues to face higher rates of relapse and increased mortality. While the National Cancer Institute (NCI) criteria have traditionally guided risk stratification based on initial clinical information, recent advances highlight the pivotal role of biological markers in shaping the prognosis of childhood ALL. This review delves into the emerging understanding of high-risk childhood ALL, focusing on molecular, cytogenetic, and immunophenotypic markers. These markers not only contribute to unraveling the underlying mechanisms of the disease, but also shed light on specific clinical patterns that dictate prognosis. The paradigm shift in treatment strategies, exemplified by the success of tyrosine kinase inhibitors in Philadelphia chromosome-positive leukemia, underscores the importance of recognizing and targeting precise risk factors. Through a comprehensive exploration of high-risk childhood ALL characteristics, this review aims to enhance our comprehension of the disease, offering insights into its molecular landscape and clinical intricacies in the hope of contributing to future targeted and tailored therapies.

Open article ↗



2024-04-02 | Calaspargase Pegol (Asparlas)

CADTH reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class.

The assessments inform non-binding recommendations that help guide the reimbursement decisions of Canada's federal, provincial, and territorial governments, with the exception of Quebec.

This review assesses calaspargase pegol (Asparlas), 3,750 units/5 mL (750 units/mL), concentrate for solution for IV infusion

Indication : As a component of a multiagent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) in pediatric and young adult patients aged 1 year to 21 years


Open article ↗



2024-03-21 | Different Levels of Therapeutic Strategies to Recover the Microbiome to Prevent/Delay Acute Lymphoblastic Leukemia (ALL) or Arrest Its Progression in Children

Alterations in the composition and diversity, metabolism, and products of the microbiome, and of the gut microbiome (GM), have been shown to be closely associated with the onset and progression of many human diseases, including cancer (i.e. hematological neoplasms). Acute lymphoblastic leukemia (ALL) is the most common form of childhood malignancy. Affected cases present typical alterations of GM, followed by inflammation, which contribute to its progression, response to therapy, as well as possible relapses. Recent evidence also reports that GM influences the development and functions of the newborn&#039;s hematopoietic system through the process of developmental programming during fetal life, as well as its susceptibility to the onset of onco-hematological pathologies, namely ALL. Furthermore, in children with ALL, GM has been found to vary in composition, variety, and functions during the clinical stages of ALL, and such variation may influence and predict the complications and prognosis of ALL after chemotherapy treatment or stem cell hematopoietic transplant. Here, we suggest some therapeutic strategies, which can be applied at two levels of intervention to recover the microbiome, and consequently prevent/delay ALL or arrest its progression.

Open article ↗



2024-02-21 | Biological Markers of High-Risk Childhood Acute Lymphoblastic Leukemia

Childhood acute lymphoblastic leukemia (ALL) has witnessed substantial improvements in prognosis; however, a subset of patients classified as high-risk continues to face higher rates of relapse and increased mortality. While the National Cancer Institute (NCI) criteria have traditionally guided risk stratification based on initial clinical information, recent advances highlight the pivotal role of biological markers in shaping the prognosis of childhood ALL. This review delves into the emerging understanding of high-risk childhood ALL, focusing on molecular, cytogenetic, and immunophenotypic markers. These markers not only contribute to unraveling the underlying mechanisms of the disease, but also shed light on specific clinical patterns that dictate prognosis. The paradigm shift in treatment strategies, exemplified by the success of tyrosine kinase inhibitors in Philadelphia chromosome-positive leukemia, underscores the importance of recognizing and targeting precise risk factors. Through a comprehensive exploration of high-risk childhood ALL characteristics, this review aims to enhance our comprehension of the disease, offering insights into its molecular landscape and clinical intricacies in the hope of contributing to future targeted and tailored therapies.

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

2 orphan drug designations for B-lymphoblastic leukemia/lymphoma with recurrent genetic abnormality, including 1 approved therapy.

2 orphan drug designations for B-lymphoblastic leukemia/lymphoma with recurrent genetic abnormality, including 1 approved therapy.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

Surovatamig

antibodies

EMA

2025-11-21

AstraZeneca AB

Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 [Kymriah]

cell therapies

EMA

2014-04-29

2018-08-27

Novartis Europharm Limited

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.

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.