Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Refametinib (BAY86-9766)
Refametinib · 3 trials · 2 indications
Parameters are laboratory parameters, vital signs, ECG parameters, cardiac function, and parameters of ophthalmologic examinations
| Arm | Type | Description |
|---|---|---|
| Refametinib and Sorafenib (Nexavar) | EXPERIMENTAL | In Cycle 1 reduced sorafenib dose (600 mg daily; 200 mg in the morning + 400 mg in the evening) is administered, which is escalated to the standard dose in Cycle 2, if no Hand-foot skin reaction (HFSR), fatigue, or gastrointestinal (GI) toxicities of grade 2 or higher occur. For the purposes of data recording, the treatment period will be divided into 3-week cycles. Patients will continue on treatment until at least one of the following occurs (main criteria): Progressive Disease (PD) {PD as defined by mRECIST criteria or clinical progression \[e.g. Eastern Cooperative Oncology Group performance status (ECOG PS) of ≥3\], treatment may be continued past radiological progression, provided the patient derives clinical benefit as judged by the treating physician.}, Death, Unacceptable toxicity, Subject withdraws consent, Treating physician determines discontinuation of treatment is in the subject's best interest, Substantial non-compliance with the protocol |
| Refametinib (BAY86-9766) | EXPERIMENTAL | For purposes of data recording, the treatment period will be divided into 3-week cycles. Patients will continue on treatment until at least one of the following occurs (main criteria): Death Unacceptable toxicity Subject withdraws consent Substantial non-compliance with the protocol Treating physician determines discontinuation of treatment is in the subject's best interest. Radiological progression as determined by RECIST (Version 1.1) or mRECIST criteria or clinical progression (e.g. Eastern Cooperative Oncology group performance status - ECOG PS ≥3) patients may continue to receive study treatment if identified as having continued clinical benefit as judged by the treating physician. |
| Refametinib (BAY86-9766)+ Gemcitabine | EXPERIMENTAL | Single dose of BAY86-9766 on Cycle 1 Day -17; twice daily dosing every day starting on day -14, start dose 50mg bid ( 30mg or 20mg are possible based on adverse events need) in addition with Gemcitabine intravenous on day 1,8 and 15 1000mg/m2 |
| Name | Type | Description |
|---|---|---|
| Refametinib (BAY86-9766) | DRUG | Patients will receive refametinib 50 mg (2x20 mg + 1x10mg capsules or 50 mg tablets) bid |
| Sorafenib (BAY43-9006) | DRUG | Patients will receive sorafenib 400 mg (2 x 200 mg tablets) bid. |
| Gemcitabine | DRUG | - |
Inclusion Criteria: Eligibility criteria for RAS mutation testing * Unresectable or metastatic HCC, confirmed either by histology or clinically according to the American Association for the Study of Liver Disease (AASLD) criteria for cirrhotic patients. For non-cirrhotic patients, histological con...
Refametinib is an investigational oral small molecule studied in hepatocellular carcinoma, a form of liver cancer, and in other neoplasms. It has been evaluated specifically in patients whose tumors carry RAS mutations, a biomarker-selected population. It is not an approved therapy and remains in clinical development.
Refametinib targets MAP2K1 and MAP2K2, also known as MEK1 and MEK2. It is a small molecule inhibitor of these kinases, which sit in the RAS/RAF/MEK/ERK signaling pathway. Because the trials enrolled RAS mutant hepatocellular carcinoma, the target class is an inhibitor of MAP2K1 and MAP2K2.
Refametinib is developed by Bayer AG, which trades over the counter in the United States under the ticker BAYRY. Bayer sponsored the clinical program evaluating the drug in RAS mutant hepatocellular carcinoma and in combination with gemcitabine in Asian patients with neoplasms.
Refametinib is in Phase 2 development. Two Phase 2 trials in RAS mutant hepatocellular carcinoma have been completed, and one Phase 1 trial of Refametinib plus gemcitabine in Asian patients has also been completed. No active trials are ongoing, and the drug is not approved.
Refametinib has been studied in three completed trials: NCT01915602, which combined Refametinib with sorafenib in RAS mutant hepatocellular carcinoma; NCT01915589, which tested Refametinib in RAS mutant hepatocellular carcinoma; and NCT01764828, a Phase 1 study of BAY86-9766 plus gemcitabine in Asian patients with neoplasms.
Yes, Refametinib is also known as BAY86-9766. The two names refer to the same investigational compound. Bayer used the code BAY86-9766 in early study titles, including the Phase 1 trial NCT01764828, while later hepatocellular carcinoma trials used the name Refametinib.