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Disease

Acute promyelocytic leukemia

Late-stage therapeutic development
Also known as AML M3, AML with t(15;17)(q22;q12), AML with t(15;17)(q22;q12);(PML/RARalpha) and variants, APL+22 more

AML M3, AML with t(15;17)(q22;q12), AML with t(15;17)(q22;q12);(PML/RARalpha) and variants, APL, APML, APML - acute promyelocytic leukaemia, APML - acute promyelocytic leukemia, FAB M3, acute myeloblastic leukaemia 3, acute myeloblastic leukaemia type 3, acute myeloblastic leukemia 3, acute myeloblastic leukemia type 3, acute myeloid leukaemia M3, acute myeloid leukaemia with t(15;17)(q22;q12);(PML/RARalpha) and variants, acute myeloid leukemia M3, acute myeloid leukemia with t(15;17)(q22;q12);(PML/RARalpha) and variants, acute promyelocytic leukaemia with PML-rara, acute promyelocytic leukaemia with t(15;17)(q22;q12); PML-rara, acute promyelocytic leukaemia with t(15;17)(q22;q12); PML/rara, acute promyelocytic leukemia with PML-rara, acute promyelocytic leukemia with t(15;17)(q22;q12); PML-rara, acute promyelocytic leukemia with t(15;17)(q22;q12); PML/rara, leukemia, acute promyelocytic, somatic, promyelocytic leukaemia, promyelocytic leukemia, leukemia, acute promyelocytic.

19
Clinical trials
12
Associated genes
5
Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Low
Key developments
  • 2 clinical trials expected to report results, the earliest in Q1 2027.
Upcoming Milestones
Clinical Milestones5View
Results posted 1
Results expected 2
Recently completed 2
Activity timeline5

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Arsenic trioxideapproved

Approval — Arsenic trioxide medac is indicated for induction of remission, and consolidation in adul… (2020)

Gemtuzumab ozogamicinapproved

Approval — Mylotarg is indicated for combination therapy with daunorubicin (DNR) and cytarabine (Ara… (2018)

Clinical trials

16 sponsors · 1 new · 0 completed in the last 12 months (net +1)

The current development programme across all trial phases.

Clinical programme
19
All trials
6
Active
8
Late-stage
6
Completed
Late-stage studies

A Pivotal Open-label Phase 3 Clinical Study Evaluating the Efficacy and Safety of QTX-2101 in Combination With All-trans Retinoic Acid in Newly Diagnosed, Low-risk Acute Promyelocytic Leukemia

Phase 3 · Recruiting · Quetzal Therapeutics

A Phase III Study for Patients With Newly Diagnosed Acute Promyelocytic Leukemia (APL) Using Arsenic Trioxide and All-Trans Retinoic Acid

Phase 3 · Active, not recruiting · Children's Oncology Group

Risk Adapted Treatment of Newly Diagnosed Childhood Acute Promyelocytic Leukemia (APL) Using Arsenic Trioxide (Trisenox® ) During Consolidation

Phase 3 · Completed · Children's Oncology Group

A Multicenter and Randomized Prospective Study for Improving the Outcome of Childhood Acute Promyeloid Leukemia

Phase 4 · Completed · South China Children's Leukemia Group

Phase III Randomized Study of Concurrent Tretinoin and Chemotherapy With or Without Arsenic Trioxide (AS2O3) (NSC # 706363) as Initial Consolidation Therapy Followed by Maintenance Therapy With Intermittent Tretinoin Versus Intermittent Tretinoin Plus Mercaptopurine and Methotrexate for Patients With Untreated Acute Promyelocytic Leukemia

Phase 3 · Completed · National Cancer Institute (NCI)

A Randomized Phase III Study to Compare Arsenic Trioxide (ATO) Combined to ATRA and Idarubicin Versus Standard ATRA and Anthracyclines-based Chemotherapy (AIDA Regimen) for Patients With Newly Diagnosed, High-risk Acute Promyelocytic Leukemia

Phase 3 · Completed · Technische Universität Dresden
Recruiting

A Pivotal Open-label Phase 3 Clinical Study Evaluating the Efficacy and Safety of QTX-2101 in Combination With All-trans Retinoic Acid in Newly Diagnosed, Low-risk Acute Promyelocytic Leukemia

Phase 3 · Recruiting · Quetzal Therapeutics

Combination of Oral Arsenic With ATRA and Minimal-Dose Chemotherapy for Newly Diagnosed Patients With Acute Promyelocytic Leukemia: a Study by the International Consortium on APL

Phase 2 · Recruiting · Instituto do Cancer do Estado de São Paulo

Phase II Study of Treatment of Acute Promyelocytic Leukemia (APL) With ATRA, Arsenic Trioxide and Gemtuzumab Ozogamicin (GO)

Phase 2 · Recruiting · M.D. Anderson Cancer Center

Treatment Study for Children and Adolescents With Acute Promyelocytic Leukemia

Phase 2 · Recruiting · Associazione Italiana Ematologia Oncologia Pediatrica

Frontline Oral Arsenic Trioxide-based Induction in Newly Diagnosed Acute Promyelocytic Leukaemia

Phase 2 · Recruiting · The University of Hong Kong
Recently completed

A Randomized Phase III Study to Compare Arsenic Trioxide (ATO) Combined to ATRA and Idarubicin Versus Standard ATRA and Anthracyclines-based Chemotherapy (AIDA Regimen) for Patients With Newly Diagnosed, High-risk Acute Promyelocytic Leukemia

Phase 3 · Completed · Technische Universität Dresden

A Multicenter and Randomized Prospective Study for Improving the Outcome of Childhood Acute Promyeloid Leukemia

Phase 4 · Completed · South China Children's Leukemia Group

Risk Adapted Treatment of Newly Diagnosed Childhood Acute Promyelocytic Leukemia (APL) Using Arsenic Trioxide (Trisenox® ) During Consolidation

Phase 3 · Completed · Children's Oncology Group

APL-R2007: Treatment of Relapsed Promyelocytic Leukemia With Arsenic Trioxide (ATO)

Phase 4 · Completed · PETHEMA Foundation

Treatment of Newly Diagnosed Patients With Acute Promyelocytic Leukemia (PETHEMA LPA 2005): Remission Induction With ATRA + Idarubicin. Risk-adapted Consolidation With ATRA and Anthracycline-based Chemotherapy (Idarubicin/Mitoxantrone) With Addition of Ara-C for High-risk Patients. Maintenance Therapy With ATRA + Low Dose Chemotherapy (Methotrexate + Mercaptopurine).

Phase 4 · Completed · PETHEMA Foundation

Phase III Randomized Study of Concurrent Tretinoin and Chemotherapy With or Without Arsenic Trioxide (AS2O3) (NSC # 706363) as Initial Consolidation Therapy Followed by Maintenance Therapy With Intermittent Tretinoin Versus Intermittent Tretinoin Plus Mercaptopurine and Methotrexate for Patients With Untreated Acute Promyelocytic Leukemia

Phase 3 · Completed · National Cancer Institute (NCI)

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2020emaApproval — Arsenic trioxide· Arsenic trioxide medac is indicated for induction of remission, and consolidation in adult patients with: Newly diagnosed low-to-intermediate risk acute promyelocytic leukaemia (APL) (white blood cell count, ? 10 x 10³/?l) in combination with all-trans-retinoic acid (ATRA) Relapsed/refractory APL (previous treatment should have included a retinoid and chemotherapy) characterised by the presence of the t(15;17) translocation and/or the presence of the pro-myelocytic leukaemia/retinoic-acid-receptor-alpha (PML/RAR?) gene. The response rate of other acute myelogenous leukaemia subtypes to arsenic trioxide has not been examined. source ↗
2019emaApproval — Arsenic trioxide· Arsenic trioxide is indicated for induction of remission, and consolidation in adult patients with: Newly diagnosed low-to-intermediate risk acute promyelocytic leukaemia (APL) (white blood cell count, ? 10 x 103/?l) in combination with all-trans-retinoic acid (ATRA) Relapsed/refractory acute promyelocytic leukaemia (APL)(Previous treatment should have included a retinoid and chemotherapy) characterised by the presence of the t(15;17) translocation and/or the presence of the promyelocytic leukaemia/retinoic-acid-receptor-alpha (PML/RAR-alpha) gene.The response rate of other acute myelogenous leukaemia subtypes to arsenic trioxide has not been examined. source ↗
2018emaApproval — Gemtuzumab ozogamicin· Mylotarg is indicated for combination therapy with daunorubicin (DNR) and cytarabine (AraC) for the treatment of patients age 15 years and above with previously untreated, de novo CD33-positive acute myeloid leukaemia (AML), except acute promyelocytic leukaemia (APL). source ↗
2002emaApproval — Arsenic trioxide· Trisenox is indicated for induction of remission, and consolidation in adult patients with: Newly diagnosed low-to-intermediate risk acute promyelocytic leukaemia (APL) (white blood cell count, ? 10 x 103/µl) in combination with all?trans?retinoic acid (ATRA) Relapsed/refractory acute promyelocytic leukaemia (APL) (previous treatment should have included a retinoid and chemotherapy) characterised by the presence of the t(15;17) translocation and/or the presence of the Pro-Myelocytic Leukaemia/Retinoic-Acid-Receptor-alpha (PML/RAR-alpha) gene. The response rate of other acute myelogenous leukaemia subtypes to arsenic trioxide has not been examined. source ↗
Safety updates
2025emaMarket withdrawal — Arsenic trioxide· Arsenic trioxide Mylan is indicated for induction of remission, and consolidation in adult patients with:- Newly diagnosed low to intermediate risk acute promyelocytic leukaemia (APL) (white blood cell count, ? 10 x 103/?l) in combination with all trans retinoic acid (ATRA)- Relapsed/refractory acute promyelocytic leukaemia (APL) (Previous treatment should have included a retinoid and chemotherapy)characterised by the presence of the t(15;17) translocation and/or the presence of the promyelocytic leukaemia/retinoic acid receptor alpha (PML/RAR alpha) gene. The response rate of other acute myelogenous leukaemia subtypes to arsenic trioxide has not beenexamined. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

RARA0.61RARG0.60RARB0.59TXNRD10.59CD330.47IFIT30.08YOD10.08IRF90.08LAT20.07SUMO20.07ANXA80.04UBE2L60.04

Disease biology

5 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Retinoic acid receptor alphavia RARA0.61Retinoic acid receptor gammavia RARG0.60Retinoic acid receptor betavia RARB0.59Thioredoxin reductase 1, cytoplasmicvia TXNRD10.59Myeloid cell surface antigen CD33via CD330.47

Reference

Authoritative identity, definition & identifiers.

Synonyms

AML M3, AML with t(15;17)(q22;q12), AML with t(15;17)(q22;q12);(PML/RARalpha) and variants, APL, APML, APML - acute promyelocytic leukaemia, APML - acute promyelocytic leukemia, FAB M3, acute myeloblastic leukaemia 3, acute myeloblastic leukaemia type 3, acute myeloblastic leukemia 3, acute myeloblastic leukemia type 3, acute myeloid leukaemia M3, acute myeloid leukaemia with t(15;17)(q22;q12);(PML/RARalpha) and variants, acute myeloid leukemia M3, acute myeloid leukemia with t(15;17)(q22;q12);(PML/RARalpha) and variants, acute promyelocytic leukaemia with PML-rara, acute promyelocytic leukaemia with t(15;17)(q22;q12); PML-rara, acute promyelocytic leukaemia with t(15;17)(q22;q12); PML/rara, acute promyelocytic leukemia with PML-rara, acute promyelocytic leukemia with t(15;17)(q22;q12); PML-rara, acute promyelocytic leukemia with t(15;17)(q22;q12); PML/rara, leukemia, acute promyelocytic, somatic, promyelocytic leukaemia, promyelocytic leukemia, leukemia, acute promyelocytic

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.