Clinical Trials

Multiple clinical trials across Phase 1 and Phase 2 have evaluated pimasertib (AS-703026) as monotherapy and in novel combination regimens for malignancies including advanced solid tumors, acute myeloid leukemia, ovarian cancer, melanoma, non-small cell lung cancer, and RAS/RAF/MAPK-mutated tumors. These studies were sponsored by pharmaceutical entities such as Merck KGaA, EMD Serono, Sanofi, and Day One Biopharmaceuticals alongside academic institutions including M.D. Anderson Cancer Center. Recruitment across all trials has reached either completed or terminated status.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07121829 TERMINATED
Melanoma; Solid Tumor; Pilocytic Astrocytoma; Non Small Cell Lung Cancer; Colorectal Cancer; Pancreatic Cancer; MAP Kinase Family Gene Mutation; RAS Mutation; RAF Mutation; MEK Mutation
Day One Biopharmaceuticals, Inc.
 2022-05-02 PHASE1
NCT04789668 TERMINATED
Anatomic Stage IV Breast Cancer AJCC v8; Clinical Stage IV Cutaneous Melanoma AJCC v8; Hematopoietic and Lymphoid Cell Neoplasm; Hormone Receptor Positive Breast Adenocarcinoma; Metastatic Lung Non-Small Cell Carcinoma; Metastatic Malignant Neoplasm in the Brain; Metastatic Malignant Solid Neoplasm; Metastatic Melanoma; Metastatic Triple-Negative Breast Carcinoma; Pathologic Stage IV Cutaneous Melanoma AJCC v8; Prognostic Stage IV Breast Cancer AJCC v8; Stage IV Lung Cancer AJCC v8; Stage IVA Lung Cancer AJCC v8; Stage IVB Lung Cancer AJCC v8
M.D. Anderson Cancer Center
 2021-01-15 PHASE1; PHASE2
NCT01985191 COMPLETED
Neoplasm Malignant
Sanofi
 2013-11 PHASE1
NCT01693068 COMPLETED
N-Ras Mutated Locally Advanced or Metastasis Malignant Cutaneous Melanoma
EMD Serono
 2012-12-05 PHASE2
NCT01936363 COMPLETED
Ovarian Cancer
EMD Serono
 2013-09-30 PHASE2
NCT01668017 TERMINATED
Advanced Solid Tumors; Hepatocellular Carcinoma
Merck KGaA, Darmstadt, Germany
 2012-09-30 PHASE1
NCT01390818 COMPLETED
Locally Advanced Solid Tumor; Metastatic Solid Tumor; Breast Cancer; Non Small Cell Lung Cancer; Melanoma; Colorectal Cancer
EMD Serono
 2011-05 PHASE1
NCT01713036 COMPLETED
Locally Advanced or Metastatic Solid Tumors
Merck KGaA, Darmstadt, Germany
 2012-11-30 PHASE1
NCT01992874 COMPLETED
Neoplasms
EMD Serono
 2013-11-30 PHASE1
NCT01016483 COMPLETED
Pancreatic Adenocarcinoma
EMD Serono
 2009-11-30 PHASE1; PHASE2
NCT01985191 Completed
Neoplasm Malignant
Sanofi|Merck KGaA Darmstadt Germany
 2013-11 Phase 1
NCT00957580 TERMINATED
Leukemia, Myeloid, Acute; Hematologic Neoplasms
EMD Serono
 2009-09-30 PHASE2
NCT01713036 Completed
Locally Advanced or Metastatic Solid Tumors
Merck KGaA Darmstadt Germany
 2012-11-30 Phase 1
NCT01668017 Terminated
Advanced Solid Tumors|Hepatocellular Carcinoma
Merck KGaA Darmstadt Germany|Merck Serono Co. Ltd. Japan
 2012-09-30 Phase 1
NCT01085331 TERMINATED
Metastatic Colorectal Cancer
EMD Serono
 2010-03 PHASE1; PHASE2
NCT01378377 TERMINATED
Advanced Solid Tumor
EMD Serono
 2011-05-27 PHASE1
NCT01378377 Terminated
Advanced Solid Tumor
EMD Serono
 2011-05-27 Phase 1

(data from https://clinicaltrials.gov, updated on 2025-08-14)

Check the Pimasertib (AS-703026) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Pimasertib is a highly selective, ATP-noncompetitive allosteric inhibitor of MEK1/2 that blocks the phosphorylation and downstream activation of extracellular signal-regulated kinase (ERK) signaling cascades. This pathway blockade induces G0-G1 cell cycle arrest, reduces oncogenic transcription, and suppresses proliferation in mutated or refractory tumor cells, providing therapeutic relevance for treating advanced solid tumors, acute myeloid leukemia, and RAS/RAF/MEK-mutated malignancies evaluated in clinical trials.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.