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MLN0264

Phase 2

Advanced Gastrointestinal Malignancies | Small molecule | Gastrointestinal |Takeda Pharmaceutical Company Limited|Trials Updated: Sep 7, 2016

MLN0264 development status

Highest phase Phase 2 (NCT02202785)
Phase scored for TAKPhase 1
Registered trials 4 across 1 sponsor since Jul 2014

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment41

FDA Designations

No designations recorded

MLN0264 clinical trials

MLN0264 · 1 trial · 1 indication

Phase 1 1
NCT01577758Phase 1 Study of MLN0264 in Adult Patients With Advanced Gastrointestinal Malignancies Expressing Guanylyl Cyclase CAdvanced Gastrointestinal Malignancies
COMPLETED41 Analytics
PHASE1COMPLETED
Phase 1 Study of MLN0264 in Adult Patients With Advanced Gastrointestinal Malignancies Expressing Guanylyl Cyclase C
Advanced Gastrointestinal MalignanciesUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Dose-Limiting Toxicities (DLTs)
From the time informed consent is signed through 30 days after the last dose of study drug, approximately 9 months

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.

Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
From the time informed consent is signed through 30 days after the last dose of study drug, approximately 9 months

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.

Maximum Tolerated Dose (MTD) of MLN0264
Every 3 weeks until MTD is established, approximately 9 months

MTD of MLN0264 was determined. Decisions regarding dose escalation were made based on any DLT that occurred during the first cycle of treatment.

Cmax: Maximum Observed Serum Concentration for MLN0264
Cycle 1: Day 1 pre-dose to Day 21 post-dose

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.

Cmax: Maximum Observed Plasma Concentration for Monomethyl Auristatin E (MMAE)
Cycle 1: Day 1 pre-dose to Day 21 post-dose

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.

AUC0-21 Days: Area Under the Curve Day 0 to Day 21 for MLN0246
Cycle 1: Day 1 pre-dose to 21 Days post-dose

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.

AUC0-21 Days: Area Under the Curve Day 0 to Day 21 for MMAE
Cycle 1: Day 1 pre-dose to 21 Days post-dose

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.

Secondary Endpoints

Best Overall Response
At the completion of every second cycle up to 12 cycles (approximately 9 months). Each cycle is a 21 days cycle
Number of Participants With Antitherapeutic Antibodies (ATA)
Day 1 of every 21 days cycle and at End of study (EOS) approximately 9 months
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
MLN0264EXPERIMENTALMLN0264 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.

Interventions

NameTypeDescription
MLN0264DRUGMLN0264 30-minute infusion on Day 1 of each treatment cycle
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

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...

Countries:United States
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Frequently asked questions about MLN0264

What is MLN0264 used for?

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.

Who is developing MLN0264?

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.

What phase is MLN0264 in?

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.

What clinical trial was conducted with MLN0264?

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.

Was the MLN0264 trial randomized or blinded?

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.

Were patients selected for MLN0264 based on a biomarker?

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.