AI Drug Discovery for Pharma and Biotech

Drug discovery

30

drugs

With orphan designations

Overview

Precursor B-cell acute lymphoblastic leukemia (B-ALL) is an aggressive hematologic malignancy characterized by uncontrolled proliferation of immature B-cell lymphoblasts in bone marrow and blood. It accounts for 75–80% of pediatric ALL cases and 75% of adult ALL cases [1][6]. Prognosis depends on age, cytogenetics (e.g., BCR-ABL1, ETV6-RUNX1), and treatment response, with 5-year survival rates exceeding 90% in children but ≤60% in adults [12][16]. Modern protocols combine chemotherapy, targeted therapies (e.g., blinatumomab, TKIs), and allogeneic hematopoietic cell transplantation (allo-HCT) for high-risk or relapsed cases [3][8].

Population

  • Median diagnosis age: 17 years; 53% occur in patients <20 years [7][12]

  • Ethnic disparities: Higher incidence in Hispanics and American Indian/Alaska Natives [7]

  • Poorer outcomes in adolescents/adults (5-year survival: 74% for ages 15–19 vs. 43% for ages 40–59) [12][17]

Burden

  • Financial toxicity: Treatment costs in India exceed 7× average monthly income [9]

  • Late effects: 40–60% of survivors develop cancer-related cognitive impairment [5]

  • Healthcare disparities: African Americans and low-income groups face 20–30% lower survival rates [12]

Therapies

  • First-line: Pediatric/adolescent regimens (BFM-based protocols) yield 85–95% complete remission [3][12]

  • Relapsed/refractory: Immunotherapy (blinatumomab/inotuzumab) followed by allo-HCT doubles survival vs standard chemotherapy [3][14]

  • Emerging approaches: CAR-T cell therapy for multi-relapsed cases and MRD-directed therapies [13][18]

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

Research Papers

3,112 drug discovery papers related to Precursor B-cell acute lymphoblastic leukemia, with 3 first-in-class and 23 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

3,112 drug discovery papers related to Precursor B-cell acute lymphoblastic leukemia, with 3 first-in-class and 23 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

2026-07-11 | Clinical features and initial treatment outcomes of pediatric B-cell acute lymphoblastic leukemia with P2RY8::CRLF2 fusion positivity: a case series.

P2RY8::CRLF2 fusion is a recurrent CRLF2 (cytokine receptor-like factor 2) rearrangement in pediatric B-cell acute lymphoblastic leukemia (B-ALL) and is frequently associated with Philadelphia chromosome-like features and kinase pathway activation. However, clinical data on Chinese children with this lesion remain limited, and its early response under contemporary risk-adapted chemotherapy is not fully defined. We report a single-center case series to describe the clinical, molecular, and early treatment characteristics of this rare subgroup. Seven consecutive children with newly diagnosed P2RY8::CRLF2 fusion-positive B-ALL treated between September 2017 and July 2024 were retrospectively reviewed. The median age at diagnosis was 3 years and 11 months, and the median initial white blood cell (WBC) count was 25.65×109/L. 
All patients had precursor B-cell immunophenotype. Concurrent genetic abnormalities included RAS pathway mutations in three patients, PAX5 fusions in two, and a JAK mutation in one. Two patients were treated according to the CCCG-ALL-2015 protocol and five according to the CCCG-ALL-2020 protocol. One patient received ruxolitinib during induction, and another patient with poor early response received ruxolitinib plus blinatumomab. Five patients achieved minimal residual disease (MRD) negativity on day 19, and six achieved MRD <0.01% with disappearance of the fusion transcript by day 46; the remaining patient became MRD-negative after salvage immunotherapy. After a median follow-up of 32 months, all patients remained in continuous complete remission (CR), although three were still receiving therapy. Pediatric P2RY8::CRLF2 fusion-positive B-ALL may show molecular heterogeneity, including recurrent RAS pathway lesions. Favorable early responses were observed under contemporary intensified chemotherapy with individualized targeted or immunotherapeutic interventions. Because of the small sample size, single-center design, and limited follow-up, these findings should be interpreted cautiously and require validation in multicenter cohorts.

Open article ↗



2026-07-07 | [Prognostic significance of IKZF1 alterations in acute B-lymphoblastic leukemia and the choice of transplantation strategy].

Gene alterations in Ikaros family zinc finger protein 1 (IKZF1) are common genetic abnormalities in B-cell acute lymphoblastic leukemia (B-ALL) and are closely associated with an increased risk of relapse and poor prognosis. In this study, the biological functions of the IKZF1 gene and the clinical significance of its various mutation types were systematically elaborated. Accordingly, a comprehensive clinical decision-making framework spanning from diagnosis to post-transplantation management was constructed. This framework provides an in-depth analysis of the pivotal role of immunotherapy as a "bridge" to transplantation, innovatively proposes an "MRD clearance kinetics" -guided model for deciding on transplantation modalities (allogeneic versus autologous), and defines proactive post-transplantation intervention strategies based on MRD monitoring. Furthermore, this review aims to provide a clear and actionable blueprint for individualized therapy for B-ALL patients with IKZF1 alterations.

Open article ↗



2026-07-07 | Secondary B-Cell Acute Lymphoblastic Leukemia Following Multiple Myeloma Treatment.

Secondary B-cell acute lymphoblastic leukemia (B-ALL) following multiple myeloma (MM) is rare. Our case presents a patient with non-secretory MM, using bortezomib-based induction, autologous transplantation, and sequential Lenalidomide maintenance treatment and persistent complete remission (CR) for about 5 years. Then the patient got secondary B-ALL. Autologous stem cell transplant with bortezomib therapy has achieved CR of patients with MM, but also has an increased risk of secondary B-ALL.

Open article ↗



2026-07-11 | Clinical features and initial treatment outcomes of pediatric B-cell acute lymphoblastic leukemia with P2RY8::CRLF2 fusion positivity: a case series.

P2RY8::CRLF2 fusion is a recurrent CRLF2 (cytokine receptor-like factor 2) rearrangement in pediatric B-cell acute lymphoblastic leukemia (B-ALL) and is frequently associated with Philadelphia chromosome-like features and kinase pathway activation. However, clinical data on Chinese children with this lesion remain limited, and its early response under contemporary risk-adapted chemotherapy is not fully defined. We report a single-center case series to describe the clinical, molecular, and early treatment characteristics of this rare subgroup. Seven consecutive children with newly diagnosed P2RY8::CRLF2 fusion-positive B-ALL treated between September 2017 and July 2024 were retrospectively reviewed. The median age at diagnosis was 3 years and 11 months, and the median initial white blood cell (WBC) count was 25.65×109/L. 
All patients had precursor B-cell immunophenotype. Concurrent genetic abnormalities included RAS pathway mutations in three patients, PAX5 fusions in two, and a JAK mutation in one. Two patients were treated according to the CCCG-ALL-2015 protocol and five according to the CCCG-ALL-2020 protocol. One patient received ruxolitinib during induction, and another patient with poor early response received ruxolitinib plus blinatumomab. Five patients achieved minimal residual disease (MRD) negativity on day 19, and six achieved MRD <0.01% with disappearance of the fusion transcript by day 46; the remaining patient became MRD-negative after salvage immunotherapy. After a median follow-up of 32 months, all patients remained in continuous complete remission (CR), although three were still receiving therapy. Pediatric P2RY8::CRLF2 fusion-positive B-ALL may show molecular heterogeneity, including recurrent RAS pathway lesions. Favorable early responses were observed under contemporary intensified chemotherapy with individualized targeted or immunotherapeutic interventions. Because of the small sample size, single-center design, and limited follow-up, these findings should be interpreted cautiously and require validation in multicenter cohorts.

Open article ↗



2026-07-07 | [Prognostic significance of IKZF1 alterations in acute B-lymphoblastic leukemia and the choice of transplantation strategy].

Gene alterations in Ikaros family zinc finger protein 1 (IKZF1) are common genetic abnormalities in B-cell acute lymphoblastic leukemia (B-ALL) and are closely associated with an increased risk of relapse and poor prognosis. In this study, the biological functions of the IKZF1 gene and the clinical significance of its various mutation types were systematically elaborated. Accordingly, a comprehensive clinical decision-making framework spanning from diagnosis to post-transplantation management was constructed. This framework provides an in-depth analysis of the pivotal role of immunotherapy as a "bridge" to transplantation, innovatively proposes an "MRD clearance kinetics" -guided model for deciding on transplantation modalities (allogeneic versus autologous), and defines proactive post-transplantation intervention strategies based on MRD monitoring. Furthermore, this review aims to provide a clear and actionable blueprint for individualized therapy for B-ALL patients with IKZF1 alterations.

Open article ↗



2026-07-07 | Secondary B-Cell Acute Lymphoblastic Leukemia Following Multiple Myeloma Treatment.

Secondary B-cell acute lymphoblastic leukemia (B-ALL) following multiple myeloma (MM) is rare. Our case presents a patient with non-secretory MM, using bortezomib-based induction, autologous transplantation, and sequential Lenalidomide maintenance treatment and persistent complete remission (CR) for about 5 years. Then the patient got secondary B-ALL. Autologous stem cell transplant with bortezomib therapy has achieved CR of patients with MM, but also has an increased risk of secondary B-ALL.

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

30 orphan drug designations for Precursor B-cell acute lymphoblastic leukemia, including 4 approved therapies.

30 orphan drug designations for Precursor B-cell acute lymphoblastic leukemia, including 4 approved therapies.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

enzomenib

small molecules

FDA

2026-07-19

Sumitomo Pharma America, Inc.

cell therapy product derived from an allogeneic hematopoietic stem cell donor and expressed anti-CD19 chimeric antigen receptor, T cell receptor of invariant natural killer T cells, and interleukin-15

cell therapies

FDA

2026-04-16

Shanghai Allovanta Biotechnology Co., Ltd.

blinatumomab

antibodies

FDA

2025-12-12

Amgen Inc.

surovatamig

antibodies

FDA

2025-11-09

AstraZeneca Pharmaceuticals LP

universal chimeric antigen receptor T cells (UCAR-T) targeting CD7

cell therapies

FDA

2025-11-04

Bioheng Therapeutics US LLC

chimeric antigen receptor T cells targeting CD19 & CD22

cell therapies

FDA

2025-08-19

Shenzhen Pregene Biopharma Co., Ltd.

Allogeneic peripheral blood-derived T-cells, fratricide-resistant, transduced with a lentivirus vector expressing a chimeric antigen receptor against CD7

cell therapies

EMA

2025-07-18

Yes Pharmaceutical Development Services GmbH

small molecule cytidine triphosphate synthase 1 (CTPS1) inhibitor

small molecules

FDA

2025-05-02

Step Pharma

adeno-associated viral serotype rh74 vector encoding an anti-CD19-anti-CD3 bispecific protein

gene therapies

FDA

2024-09-13

Vironexis Biotherapeutics, Inc.

Anti-CD19 Chimeric Antigen Receptor T-Cell Injection

cell therapies

FDA

2022-01-13

Juventas Cell Therapy Ltd.

Axicabtagene ciloleucel

cell therapies

EMA

2015-11-11

Kite Pharma EU B.V.

Allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2

cell therapies

EMA

2015-08-10

Fate Therapeutics Limited

Spanlecortemlocel

cell therapies

EMA

2014-12-16

Novartis Europharm Limited

Pinometostat

small molecules

EMA

2014-01-16

Voisin Consulting Life Sciences

Moxetumomab pasudotox

proteins

EMA

2013-07-17

AstraZeneca AB

Inotuzumab ozogamicin [Besponsa]

antibodies

EMA

2013-06-07

2017-07-03

Pfizer Europe MA EEIG

inotuzumab ozogamicin [Besponsa]

antibodies

FDA

2013-03-25

2024-03-06

Wyeth Pharmaceuticals, LLC

inotuzumab ozogamicin

antibodies

FDA

2013-03-25

2017-08-17

Wyeth Pharmaceuticals, LLC

inotuzumab ozogamicin [Besponsa]

antibodies

FDA

2013-03-25

2024-03-06

Wyeth Pharmaceuticals, LLC

Ponatinib hydrochloride [Iclusig]

small molecules

EMA

2010-02-02

Incyte Biosciences Distribution B.V.

Mercaptopurine [Xaluprine]

small molecules

EMA

2009-04-30

Nova Laboratories Ireland Limited

Allogeneic umbilical cord blood cells-derived CD133+ cells ex vivo expanded

cell therapies

EMA

2009-02-27

Regulatory Resources Group Limited

Vincristine sulfate

small molecules

EMA

2008-07-08

Pharma Gateway AB

Dasatinib [Sprycel]

small molecules

EMA

2005-12-23

[INACTIVE] Bristol-Myers Squibb Pharma EEIG

Imatinib mesilate [Glivec]

small molecules

EMA

2005-08-26

Novartis Europharm Limited

Nelarabine [Atriance]

small molecules

EMA

2005-06-16

Novartis Europharm Limited

Asparaginase [Spectrila]

proteins

EMA

2005-01-26

[INACTIVE] Medac Gesellschaft fuer klinische Spezialpraeparate mbH

Plitidepsin [Aplidin]

small molecules

EMA

2003-07-09

Pharma Mar S.A.

Clofarabine [Evoltra]

small molecules

EMA

2002-02-05

[INACTIVE] Sanofi B.V.

Ricin (blocked) conjugated murine mca (anti-b4)

antibodies

FDA

1991-01-24

ImmunoGen, 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.

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.