Clinical Results Summary
A clinical study to learn about the plasma levels of quizartinib in
healthy people compared to people with liver disease
Protocol number: AC220-016

Thank You!

Daiichi Sankyo, Inc., the sponsor of this study, would like to thank the participants who
took part in this study for quizartinib, also known as AC220. Each participant helped toadvance medical research and knowledge for people affected with acute myeloid
leukemia. Their contribution to medicine and healthcare is greatly appreciated.

Important note: This summary only shows the results of a single study. Other studies may have differentfindings. Researchers and health authorities look at the results of many studies to understand which
treatments work and how they work. It takes a lot of people in many studies around the world to advance
medical science and healthcare.
Do not use the results of this study to make health decisions. Please talk to a doctor before changing any
treatment you are taking or if you have any questions about these study results.

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What was the main purpose of this study?
Acute myeloid leukemia (AML)
Quizartinib is an investigational drug being tested for the treatment of acute myeloid leukemia, or AML.
AML is a cancer of the blood and bone marrow. The bone marrow is found in the center of most bones, where
new healthy blood cells are made. AML starts in the bone marrow and prevents it from making normal bloodcells. The abnormal (cancer) cells build up in the bone marrow, so there are fewer healthy blood cells. Thesecancerous cells can also enter the blood stream and circulate in the blood, and go to different parts of thebody.
Before a new drug can be given to patients, the researchers developing it perform many research studies toshow that the drug is safe and effective. The first step in studying a new drug is to test it in healthy people.
This means people without any serious health problems. This study involved healthy participants.
In the studies done so far, researchers learned that the liver plays an important role in removing quizartinibfrom the body. Therefore in this study, researchers wanted to compare the plasma* levels of quizartinib andits breakdown product AC886 in people with liver disease with the plasma levels in healthy people. This will
enable researchers to decide what dose of quizartinib should be given to people with liver disease.
* Plasma is the fluid part of the blood. It contains different components of the blood that are necessary for life andhealth such as hormones, proteins, etc.

Treatment given in this study

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Main purpose of this study
The main question the researchers wanted to answer in this study was:Other purpose of this study
Researchers also wanted to answer the following question:•What side effects could the participants develop during the study?

There were some additional questions that researchers wanted to answer but these are not discussed in thissummary.

How long was this study?
An individual participant could have been in this study for
30 days.
The study started in November 2014 and was completed asplanned in March 2015. A study report was created. Thissummary is based on that report.

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Who was in this study?
This study included 30 participants from UnitedStates.
Participants could take part in this study if they:•were aged 18 to 70 years of age•

had previously been diagnosed with liverdisease, and were assessed as having mild or
moderate liver disease as measured by ascoring system*ORwere healthy, as assessed by the study doctor•

were willing to eat high-potassium foods, such as bananas, at least 24 hours before taking quizartinib
on Day 1 of the study•

used effective birth control methods during the study or were unable to have children.

* The Child-Pugh scoring system, or CPS, was used to find out whether participants had mild or moderate liver diseaseby using 5 clinical measures (symptoms & signs). A score of 1, 2, or 3 was given to each measure, with 3 being the mostsevere. The scores were added for all 5 measures. Out of a maximum score of 15, only participants with scores of 5 to 6
(mild liver disease), or 7 to 9 (moderate liver disease) were included in the study.

What happened during this study?
This was a Phase 1 study. Phase 1 studies are done to find out how a new study treatment works in a small
number of participants. This Phase 1 study helped researchers understand what happens to quizartinib in thebody, and if there are any side effects.
This study was “open label”. This means that both the researchers and the participants knew which treatment
was given to which participants.
Participants were screened to find out if they could take part in the study. All participants received 30 mg
quizartinib tablets on only one day in the study (Day 1), on an empty stomach. Participants continued fastingfor 4 hours after taking the quizartinib tablets.

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There were 3 groups of participants:•

8 participants with mild liver disease,•

8 participants with moderate liver disease, and•

14 healthy participants

The healthy participants were split into 2 subgroups and matched for age, gender and weight with the groupsof participants with mild and/or moderate liver disease. These 2 subgroups of healthy participants served ascontrol groups for the 2 groups of participants with mild or moderate liver disease. Within these control
groups, 2 participants had appropriate age, gender and weight measurements which allowed them to serve ascontrols for both groups of participants with mild and/or moderate liver disease. Therefore although there
were only 14 healthy participants in the study, this meant there were 8 participants in each of the matchedhealthy contol groups.

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What were the key results of this study?
Key results from this study are shown for the total group of participants as average results. This summary does
not show the results from each individual participant. An individual participant’s results could be different
from the total group of participants. A full list of the questions the researchers wanted to answer and adetailed presentation of the results can be found on the websites listed at the end of this summary.

The total plasma levels of quizartinib and AC886 measured over time were similar in participants with mild or
moderate liver disease, when compared with levels in matched healthy participants.
This means that liver impairment disease did not have much effect on the total plasma levels of quizartiniband AC886.

How was this measured?
Researchers took blood samples from participants at defined time points and calculated the total levels ofquizartinib and AC886 in participants’ plasma over time, in ng·h/ mL*.
*This means the total levels of quizartinib and AC886 in nanograms (one thousand-millionth of a gram) found in eachmilliliter of plasma over time.

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What side effects did the participants develop during the study?
Side effects are medical problems (this may range from something mild such as feeling tired or something
more severe like a severe infection or other medical problem) that happened during the study, which theresearchers thought could be related to the treatments in the study.. This section provides a summary of side
effects related to quizartinib.
Side effects are considered serious if they cause death, are life-threatening, cause disability, cause lasting
problems, cause birth defects, or require hospitalization. Sometimes participants stop taking study treatment
because of side effects.
Side effects other than those related to quizartinib study treatment are not reported here. For more
information on medical problems, please visit the websites listed at the end of this summary.
There were no deaths or serious side effects reported during the study that were related to quizartinib. Noneof the participants in the study stopped quizartinib early because of side effects.

How many participants had side effects?
The side effects experienced by participants in any group are reported below:

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* One participant with mild liver disease had 2 different side effects.

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How was this study useful for patients and researchers?
This study helped researchers to compare the plasma levels of quizartinib and AC886 in healthy people frompeople with liver disease.
Findings from this study may be used in other studies. Other studies for quizartinib are ongoing.
Please remember, this summary only shows the results of a single study. Other studies may have different
findings. Please talk to a doctor before changing any treatment you are taking or if you have any questions
about these study results.

Where can I learn more about this study?
If you were a study participant and have questions about the results of this study, please speak with thedoctor or staff at your study site.
Full study title:
A Phase 1, Open-label, Single-dose, Parallelgroup Study to Evaluate the Pharmacokinetics ofQuizartinib and its Active Metabolite, AC886, in
Subjects with Mild or Moderate Hepatic
Impairment Compared to Healthy Subjects

Sponsor: Daiichi Sankyo, Inc.
Sponsor contact information:
211 Mount Airy Road, Basking Ridge, NJ 07920
Email: CTRInfo@dsi.com
Phone number: 1-908-992-6640
Date of this summary: 15 February 2022

This summary was prepared by Kinapse Ltd, a Syneos Health® company.

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