Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
MLN0264 · 1 trial · 1 indication
DLT was defined as any of the following Adverse Events (AEs) that occur and are considered by the investigator to be related to therapy. 1. Grade 4 neutropenia (Absolute Neutrophil Count \< 500 cells/mm\^3). 2. Grade 3 or greater neutropenia with fever and/or infection. 3. Grade 4 thrombocytopenia (platelets \< 25,000/mm\^3). 4. Grade 3 or greater thrombocytopenia with clinically meaningful bleeding at any time. 5. Grade 3 or greater nausea and/or emesis that occurs despite of prophylaxis. 6. Grade 3 or greater diarrhea that occurs despite supportive care. 7. Any other Grade 3 or greater non-hematological toxicity other than Grade 3 fatigue or Grade 3 Alopecia. 8. Inability to start the next cycle of therapy due to treatment delay of more than 2 weeks because of lack of recovery. 9. Other MLN0264-related non-hematologic toxicities Grade 2 or greater requiring discontinuation of therapy.
An Adverse Event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (eg, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A Serious Adverse Event (SAE) was any experience that suggests a significant hazard, contraindication, side effect or precaution that: results in death, is life-threatening, required in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect or is medically significant. A treatment-emergent adverse event (TEAE) is defined as an adverse event with an onset that occurs after receiving study drug.
MTD of MLN0264 was determined. Decisions regarding dose escalation were made based on any DLT that occurred during the first cycle of treatment.
Maximum observed serum concentration (Cmax) is the peak serum concentration of a drug after administration, obtained directly from the serum concentration-time curve. Cmax is reported for the 1.8 mg/kg dose, which is the MTD, where there is adequate data to provide robust parameter information reliably.
Maximum observed plasma concentration (Cmax) is the peak plasma concentration of a drug after administration, obtained directly from the plasma concentration-time curve. Cmax is reported for the 1.8 mg/kg dose, which is the MTD, where there is adequate data to provide robust parameter information reliably.
Area under the drug concentration versus time curve from time 0 to Day 21. AUC0-21 is reported for the 1.8 mg/kg dose (the MTD), where there is adequate data to provide robust parameter information reliably.
Area under the plasma drug concentration versus time curve from time 0 to Day 21. AUC0-21 is reported for the 1.8 mg/kg dose (the MTD), where there is adequate data to provide robust parameter information reliably.
| Arm | Type | Description |
|---|---|---|
| MLN0264 | EXPERIMENTAL | MLN0264 starting dose 0.3 mg/kg escalated until Maximum Tolerated Dose (MTD) was determined, 30-minute infusion, on Day 1 of each 21-Day treatment cycle. |
| Name | Type | Description |
|---|---|---|
| MLN0264 | DRUG | MLN0264 30-minute infusion on Day 1 of each treatment cycle |
Inclusion Criteria: * Voluntary consent form * Diagnosis of GI malignancy with a GCC protein expressing tumor * Male or female patients 18 years or older with measurable disease as defined by Response Evaluation Criteria in Solid Tumors (RECIST) * Female patients who are post menopausal, surgically...
MLN0264 is an investigational small molecule being studied for advanced gastrointestinal malignancies, specifically tumors that express guanylyl cyclase C. It has been evaluated in adult patients with these advanced cancers. It is not an approved therapy and remains in clinical development under Takeda Pharmaceutical Company Limited.
MLN0264 is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. Takeda sponsored the clinical research conducted with the agent in advanced gastrointestinal malignancies. The program is an investigational oncology asset within Takeda's pipeline.
MLN0264 is in Phase 1 development. The single clinical trial conducted with the drug was a Phase 1 study, and it has been completed. The drug is investigational and has not been approved by the FDA for any indication.
MLN0264 was studied in one Phase 1 clinical trial, NCT01577758, titled a Phase 1 Study of MLN0264 in Adult Patients With Advanced Gastrointestinal Malignancies Expressing Guanylyl Cyclase C. The trial enrolled 41 patients, was conducted in the United States, and has been completed.
The MLN0264 Phase 1 trial, NCT01577758, was an uncontrolled, open-label study. It was not randomized and not double blinded. It enrolled 41 adult patients aged 18 years and older with advanced gastrointestinal malignancies expressing guanylyl cyclase C, and it has been completed.
The MLN0264 Phase 1 trial, NCT01577758, enrolled patients with advanced gastrointestinal malignancies expressing guanylyl cyclase C, so tumor expression of that marker was part of the study population definition. The trial was not described as a biomarker-selected design in the registry data.