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Good afternoon, and
thank you all for coming.

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I know there's a lot
of different panels

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to choose from, so thank
you for coming to ours.

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My name is Katie Poulin I'm a
senior year at Mount Holyoke

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College.

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I'm a bio major
and a stats minor.

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So today, I'm going to talk a
little bit about basics of drug

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discovery and development.

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So first, this past summer I
worked at Tokai Pharmaceutical,

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which is a pharmaceutical
company located

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in Boston, Massachusetts.

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They're working on a
drug for prostate cancer.

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So a little bit on how I got
this internship is last spring,

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I was looking on the
Mount Holyoke website,

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and there was an
article about an Alum.

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And she is actually the
CEO of this company.

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I was really interested,
how did she get there,

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what was her career about?

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Because I was a
bio major, I knew

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I wasn't really
interested in research.

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But I was like, what am I
going to do with my bio degree?

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So this is what I found.

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And I contacted
her, and just want

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to hear how she got
to where she is today.

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And it was a great
conversation, learned a lot.

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And from that conversation,
turned into an internship.

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So this past summer,
I got to see how the--

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I worked, actually, in the
clinical department, which

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I'll talk more about later on.

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But I got to see
from the beginning

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to the end of drug
discovery and development.

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So in this presentation,
we're going

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to start with drug
discovery, and we're

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going to get to one
FDA-approved drug.

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So this is the end goal for
all the pharmaceutical company.

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They want to see their drug
on the market actually helping

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the patients that
it's intended for.

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But how do we get there?

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So drug discovery on average
takes 10 to 15 years,

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and usually costs between
$800 million to $1 billion.

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It takes multiple contributing
internal departments

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and additional
external organizations.

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One fact that I
really was surprised

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about this summer that
really stuck with me

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was that 5,000 to 10,000
compounds enter the research

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development pipeline,
but only one

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will see FDA approval, which
I was very shocked about.

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So drug discovery.

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First, the scientists need
to understand the disease

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that the drug will be
treating in order for it

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to best help the patient.

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They need [INAUDIBLE]
target identification,

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so where the target area--

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where the drug is affected,
where the drug can

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affect the disease the most.

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And they need to validate
this target site.

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So how does it
impact the disease,

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visit the correct site
that they are targeting?

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And we don't want to
target the wrong site.

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[INAUDIBLE] an unnecessary
adverse effects

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to patients without a
benefit will not do any good

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for any of the patients.

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So before the drug is
allowed in any patients,

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it goes through many
frequent testing.

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And this is safety testing which
is usually done in animals.

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And you may share that
and be safe for humans.

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Toxicology, a look
at how a drug could

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be toxic to different
organs, to species.

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It looks at broad range.

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And ADME, which looks
at the absorption

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of the drug in the body.

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[INAUDIBLE] how the
drug is distributed

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through the body, how it's
metabolized in the body,

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and finally, how it's
excreted from the body.

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So the clinical trials, which
is the part near and dear

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to my heart, because that's
where I worked this summer,

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has three phases.

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And we're going to go
into it a little bit

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about each one of these.

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So phase one is the
initial human testing.

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And it's usually a healthy
group of volunteers.

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The number of volunteers
range from 20 to 100,

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but this is different
for all clinical trials,

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because it just depends.

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It discovers if
this safe in humans,

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and it figures out the safe
dosing range for the drug.

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But once these
questions are answered,

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depending their answers,
the question is asked,

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should this drug continue,
or does it need to stop?

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All of these are answers
that we would want to see,

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[INAUDIBLE] safe and everything,
it will continue to phase two.

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So phase two is a
larger study, usually

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with 100 to 500 patients
with the disease.

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It looks at the drug safety and
the effectiveness of the drug.

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It also looks at the dose
strength and schedule

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for the drug.

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So phase three is the
largest, most costliest phase.

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It usually consists between
1,000 to 5,000 patients

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with the disease.

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It's there to produce.

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It looks at the
safety, the efficacy,

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and the overall
benefit the drug has.

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And to find statistically
significant data

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to use for the FDA.

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There are a large
number of sites

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all around the world, which is
necessary because we don't want

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to create any statistical bias.

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So in order [INAUDIBLE], yeah.

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[CHUCKLES]

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It's-- yeah.

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[INAUDIBLE]

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OK.

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So the new drug application.

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So once all phases
are passed, they

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file a new drug application,
which is, on average, greater

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than only 1,000 pages.

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But luckily, we can now
file it electronically,

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so we don't have
to kill many trees.

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But the final decision
lies with the FDA.

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And in every review is
the risk-benefit analysis,

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the advisory
committee evaluation.

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And they also do a
facility inspection.

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So where's galeterone?

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Galeterone is the drug that
Tokai Pharmaceuticals is

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working in for prostate cancer.

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It's in its phase three,
so it's almost there.

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They have multiple global sites
in the United States, Canada,

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and the United Kingdom.

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There is galeterone, and
it's being looked at.

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And the active comparator
is enzalutamide.

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And their phase three
has 148 patients.

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So my experience.

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I really got to learn how
the industry operates,

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because I never--

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go to school, you
don't get to see that.

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And it was hands-on knowledge,
and I just see how it operates.

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I got to discover my
future career path,

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because, again, I
was unsure of where

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I was going to go after
college, and this really

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opened up a few doors.

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I got to network with
people in the industry

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from US and abroad.

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I went on a business
trip, which was amazing,

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and got to meet many
different people.

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And I got to take action for
my next move after college,

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and to see this was something
I want to do, and understand

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what is that I like.

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I'd like to thank
Tokai Pharmaceuticals

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for giving me this wonderful
opportunity for our mentor

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and for mentoring us.

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And for you guys all
for coming out today.

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Thank you.

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