Drug Trials Snapshots: COPIKTRA

HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race and age groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.

LIMITATIONS OF THIS SNAPSHOT:
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your health provider about the risks and benefits of a drug. Refer to the COPIKTRA Package Insert for complete information.

COPIKTRA (duvelisib)
co-PIK-trah
Verastem, Inc
Approval date: September 24, 2018

DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

COPIKTRA is a drug used to treat adult patients with chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), and follicular lymphoma (FL) who have received at least two prior treatments that did not work or are no longer working.

CLL, SLL, and FL are forms of blood cancer.

How is this drug used?

COPIKTRA is taken as one capsule by mouth twice daily for 28-day treatment cycles.

What are the benefits of this drug?

In adult patients with CLL or SLL, COPIKTRA may delay disease worsening. Patients treated with COPIKTRA lived about 16 months without disease worsening (progression) in comparison to patients treated with comparator drug (ofatumumab) and lived about 9 months without disease worsening.

Among 83 patients with FL who were treated with COPIKTRA, 35 patients (42%) had either complete or partial tumor shrinkage (response).

COPIKTRA for treatment of adult patients with FL was approved under FDA’s accelerated approval program, which provides earlier patient access to a new drug while the company continues to conduct clinical trials to confirm that the drug works well.

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Were there any differences in how well the drug worked in clinical trials among sex, race and age?

Sex: COPIKTRA worked similarly in men and women.
Race: The majority of patients were White. Therefore, differences in how well the drug works among races could not be determined.
Age: COPIKTRA worked similarly among patients younger and older than 65 years of age.

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What are the possible side effects?

COPIKTRA may cause serious side effects that can lead to death including infections, diarrhea or inflammation of the intestines, skin reactions, and inflammation of the lungs. Other serious side effects include abnormal liver blood tests and low white blood cell counts (neutropenia).

The most common side effects of COPIKTRA are diarrhea, inflammation of the intestines, low white blood cell count (neutropenia), rash, tiredness, fever, cough, nausea, upper respiratory infection, pneumonia, bone and muscle pain, and low red blood cell count (anemia).

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Were there any differences in side effects among sex, race and age?

Sex: The occurrence of side effects was similar in men and women.
Race: The majority of patients were White. Therefore, differences the occurrence of side effects among races could not be determined.
Age: The occurrence of side effects was similar among patients younger and older than 65 years of age.

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WHO WAS IN THE CLINICAL TRIALS?

Who participated in the clinical trials?

The FDA approved COPIKTRA based on evidence from four clinical trials (Trial 1/NCT02004522, Trial 2/NCT02204982, Trial 3/NCT01476657 Trial 4/NCT02049515) of 442 adult patients with chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), or follicular lymphoma (FL) who received at least two prior treatments that did not work or was no longer working.

Trials were conducted at 92 sites in Australia, Canada, Europe, New Zealand, and the United States.

The population that provided data for side effects of COPIKTRA (safety population) is presented below. Demographics of the patients who provided data for evaluation of benefits (efficacy population) are presented in Table 9, under the MORE INFO section.

Figure 1 summarizes how many men and women were in the clinical trials used to evaluate safety.

Figure 1. Baseline Demographics by Sex

FDA Review

Figure 2 summarizes the percentage of patients by race in the clinical trials used to evaluate safety.

Figure 2. Baseline Demographics by Race

*Other includes Asian, American Indian or Alaska Native, Not Reported

FDA Review

Table 1. Demographics of Trials by Race

Race	Number of Patients	Percentage of Patients
White	407	92%
Black or African American	12	3%
Asian	1	Less than 1%
American Indian or Alaska Native	1	Less than 1%
Other*	15	3%
Not Reported	6	1%

Clinical Trial Data

Figure 3 summarizes the percentage of patients by age in the clinical trials used to evaluate safety.

Figure 3. Baseline Demographics by Age

FDA Review

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How were the trials designed?

The benefit and side effects of COPIKTRA were evaluated in four clinical trials of adult patients 30 – 90 years of age with CLL, SLL, or FL.

Trial 1 enrolled adult patients with chronic lymphocytic leukemia and small lymphocytic lymphoma after at least one prior treatment that did not work or was no longer working. Patients were assigned to receive COPIKTRA twice daily by mouth or ofatumumab for 7 weekly intravenous infusions (injected into the vein) followed by 4 monthly intravenous infusions. Treatment continued until either disease worsened or patients experienced unacceptable toxicity. The benefit of COPIKTRA was assessed based on the length of time that the disease did not get worse, comparing patients in the COPIKTRA and ofatumumab groups.

Trial 2 enrolled adult patients with follicular lymphoma that did not respond to or no longer responded to treatment with rituximab (a drug used to treat CLL or FL), chemotherapy (chemicals used to treat cancer) or radio-immunotherapy (treatment that uses the body’s own immune system to combat cancer). Patients received COPIKTRA twice daily by mouth during a 28-day cycle. Treatment continued until either disease worsened or patients experienced unacceptable toxicity. The benefit of COPIKTRA was evaluated by measuring how many patients had complete or partial tumor shrinkage (response) and how long that response lasted.

Trial 3 enrolled adult patients with advanced blood cancers. Patients received increasing doses of COPIKTRA based on toxicity, over 28-day treatment cycles. Patients in Trial 3 were primarily evaluated for side effects.

Trial 4 enrolled patients from Trial 1 who experienced worsening of disease. Patients who previously received ofatumumab in Trial 1 were assigned to receive COPIKTRA twice daily by mouth until the disease worsened, they discontinued participation in the trial or started another treatment. Patients who previously received COPIKTRA in Trial 1, were assigned to receive ofatumumab as 7 weekly intravenous infusions followed by four monthly intravenous infusions. Patients in Trial 4 were primarily evaluated for side effects.

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GLOSSARY

CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.