Clinical Results Summary
A clinical study to compare the plasma levels of quizartinib when taken
as tablets or liquid by healthy participantsProtocol number: AC220-014

Thank You!

Daiichi Sankyo, Inc., the sponsor of this study, would like to thank the participants who
took part in this study for quizartinib. Each participant helped to advance medical researchand knowledge for people affected with a type of blood cancer called 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.

What was the main purpose of this study?
Quizartinib, also known as AC220, is an investigational drug that is being tested to treat people with a type ofblood cancer called Acute Myeloid Leukemia, or AML.
AML is a cancer of the blood and the bone marrow. The bone marrow is found in the center of the bones,
where new healthy blood cells are made. AML starts in the bone marrow and prevents it from making normal
blood cells. The abnormal (cancer) cells build up in the bone marrow, so there are fewer healthy blood cells.
These cancerous cells can also enter the blood stream and circulate in the blood, and go to different parts ofthe body.
Quizartinib was first developed in liquid form to be taken by mouth. Researchers have developed tablets forquizartinib. In this study, researchers wanted to compare the levels of quizartinib in the participants’ plasma
when taken as tablet(s) or liquid.
* Plasma is the fluid part of the blood. It contains different components of the blood that are necessary for lifeand health 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:*The body breaks down quizartinib into another product called AC886. AC886 has similar effects in the body toquizartinib.

Other purposes of this study
Other questions researchers wanted to answer in this study were:•

For the tablet form, do the plasma levels of quizartinib and AC886 increase with an increase in dose?

•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?
The study started in June 2012 and ended in September 2012. The
study was completed as planned and a study report was created. Thissummary is based on that report.

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Who was in this study?
This study included 80 participants from the United States.
People could take part in the study if they:•

were healthy individuals between 18 to 55 years ofage,•

were using effective birth control methods during
the study or were unable to have children,•

did not have major surgery within 90 days ofreceiving study drug,•agreeing to consume potassium rich foods such as
bananas, oranges, cantaloupes, and yogurt for
at least 24 hours before starting the study treatment (quizartinib tablet or liquid),•

had a normal weight, and healthy kidneys and liver as confirmed by blood tests.

What happened during this study?
This was a Phase 1 study. Phase 1 studies are done to find out the safety of a new study treatment and how it
works in a small number of participants. This helps researchers understand what happens to the study
treatment in the body, 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 participant.
Participants were screened to find out if they could take part in the study. On the day before receiving thestudy drug, participants were admitted to the study site for an 8-day stay. The researchers randomly assigned
participants to 4 treatment groups using a computer system. This process is called randomization. It meansthat each participant could be assigned to any group and it helps to make sure the groups are distributedfairly.
All participants received a single dose (a tablet or liquid) of quizartinib on an empty stomach and continued
fasting for 4 hours after treatment.

The participants were discharged from the study site on the morning of Day 8. Researchers collected bloodand urine samples from the participants at defined time points. Researchers monitored the participants’
health up until Day 21.

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.

What were the levels of quizartinib and AC886 in the plasma of participants
when quizartinib was taken in tablet form or as a liquid?
The total plasma levels of quizartinib and AC886 measured over time were similar when quizartinib was given
as in tablet form compared to when it was given as a liquid.

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How was this measured?
To answer this question, researchers compared the plasma levels of quizartinib and AC886 between Group 1(60 mg quizartinib liquid) and Group 2 (60 mg quizartinib, tablet form). Researchers measured the total levelsof quizartinib and AC886 over time from plasma samples of the participants. The measurement unit isnanograms hours per milliliter (ng*h/mL). This shows how much of quizartinib and AC886 in nanogram (onethousand-millionth of a gram) was found in each milliliter of plasma over time.

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What were the other results of this study?
For the tablet form, did the plasma levels of quizartinib and AC886 increase withan increase in dose?
To answer this question, researchers compared the levels of quizartinib and AC886 in the plasma ofparticipants who took 30 mg (Group 3), 60 mg (Group 2), or 90 mg (Group 4) as a tablet(s).
The plasma levels of quizartinib and AC886 increased in proportion to the increase in dose of quizartinib foreach group.

What medical problems did the study participants have?
Side effects are medical problems (such as feeling tired) that happened during the study, which the study
doctor (investigator) thought could be related to the treatments in the study. This section provides a summaryof 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, require hospitalization, or are otherwise considered medically important by thedoctor. Some participants stop study treatment because of side effects.
Side effects other than those related to quizartinib are not reported here.

How many participants had side effects?

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What side effects did the participants develop during the study?
The side effects experienced by 2 or more participants are reported below:

How many participants had serious side effects?
There were no deaths or serious side effects reported during the study that were related to quizartinib.

How many participants had to stop treatment because of side effects?
None of the participants in the study stopped treatment early because of side effects.

How was this study useful for patients and researchers?
This study helped researchers to understand the effects of different formulations of quizartinib, in liquid andtablet form. Researchers studied the plasma levels of quizartinib and its breakdown product AC886 in healthy
participants. This study also demonstrated that as the dose of quizartinib increases, the levels of quizartinib
and AC886 in the plasma also increased in a proportional way.
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.

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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,Randomized, Single Dose, Parallel Group, Relative
Bioavailability Study of Two AC220 Formulations
in Healthy Volunteers

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: 29 Apr 2022

This summary was prepared by Syneos Health®.

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