The University of California San Diego, Skaggs School of Pharmacy and Pharmaceutical Sciences Drug Commercialization course brings you lectures from both faculty and industry experts. With this course, recorded on campus at UCSD, we seek to share our access to top people in the field who bring an unprecedented range of expertise on drug commercialization. This course will cover pharmacoeconomic, marketing strategy, intellectual property strategy, portfolio management, managed markets and strategic alliances. It will also have a lecture case study from startup to success. In addition, the course will discuss post-marketing clinical trials or Phase 4 trials. These are conducted after a new drug has been approved by the regulatory agencies and launched. In these studies, the new drug is prescribed in an everyday healthcare environment using a much larger group of patients. This enables new treatment uses for the new drug to be developed, comparisons with other treatments for the same indication to be made, and determination of the clinical effectiveness of the new drug in a wider variety of patient types, and more rare side effects, if any, may be detected . Pre-marketing strategy should be instigated as early as Phase 1 clinical trials to ensure that the market's needs are incorporated into the new drug's overall development. Later phases when clinical results are presented at international medical conferences the marketing strategy is then refined in order to develop an awareness amongst the medical community who will be prescribing the new drug. In addition to the marketing strategy, pricing strategy and a tactical plan will be developed. Promotional material, and the sales force will be trained so that when the product is approved they can promote the drug to physician, pharmacist and nurses. This course is intended as part 3 of a series: Drug Discovery (https://www.coursera.org/learn/drug-discovery), Drug Development (https://www.coursera.org/learn/drug-development) and Drug Commercialization. We would highly recommend that you take the courses in order since it will give you a better understanding on how a drug is discovered in the lab before being tested in clinical trials and then launched in the market place.
From Start up to Success in Biotech II
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来自 University of California San Diego 的课程
Drug Commercialization
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从本节课中
From Start up to Success in Biotech, Barry Quart, Pharm.D.
与讲师见面
Williams S. Ettouati, Pharm.D.Director, Industrial Relations & Development; Health Sciences Associate Clinical Professor, N.S.
Skaggs School of Pharmacy and Pharmaceutical Sciences
Joseph D. MaAssociate Professor of Clinical Pharmacy
Skaggs School of Pharmacy and Pharmaceutical Sciences
And I'll show you in a minute that it was pretty much ideal opportunity for the
shareholders at the time. >> Yeah.
>> How do you find that new technology to bring in.
>> great question. I spent about nine months turning over
rocks and in, in everywhere I could find to look for technology, and in fact, what
we ended up finding. so most of the time what happens [SOUND]
is when you're out looking for technology there's.
[INAUDIBLE]. Lots of different sources you can go to
because there is lot of, lot of small bio techs that are out there shopping their
wears. you can go to universities so you make a
stop you at USSD and and, and everybody has got office its looking to have
licensed technology. So, there's different places you go to.
in this particular case, we found a small mid-size firm that was interested in
getting out of R and D. they just decided that's not a place to
where they wanted to invest money anymore.
And they wanted to get out, and they had some interesting pre-clinical compounds.
So, they had never been in any yet the pre-clinical work hadn't been quite
finished, in order to put them in a man. But they looked quite promising.
And we were able to acquire those pre-clinical compounds.
They, there were some for HIV. And NNRTIs and MEK inhibitors,
predominantly focused towards cancer. so these were two great areas to start a
company around. Because their very well defined end
points they're looking at. Particularly HIV at the time seem like an
ideal place to be and although at the same there was lot of other HIV products
by then in 2006. So, you really had to have something that
was more interesting. Know what was on the market.
and we also were able to lease their laboratories, get all their equipment.
So, as a way of jump starting the organization it was really quite amazing
opportunity. And because there was a few things that
they wanted tied up in their R and D program.
We, we got an agreement with them to continue doing some research.
So, we also got some funding and from them to keep some of the scientists, paid
for so we didn't have to burn our own dollars.
so that was an ideal situation and then because these compounds really didn't
have any value to that organization. we were able to get them with very low
milestones and royalties most of which were back loaded.
So, from the point of view of what it caused to requirities that was say was a
great deal for shareholders. and I think that was clearly demonstrated
by, by the reaction of the market to restarting the company.
when we restarted the company, at the arrow and put out the press release of
the, you know [LAUGH]. Well, guess what, you're not getting your
money back [LAUGH]. But we're, we brought in a bunch of
interesting technology. you know, the concern is always, should
it, the stock is going to drop even further.
As it turned out, it actually went up very quickly.
So, the investors that were in this company at the time, to make a small, a
small increase in their investment when they got their money back.
So, the company was selling for 85% of cash and so if they got all the cash back
they'd make 15% in let's say six months. here they made almost double their money
in that same period of time. So, I got very few complaints from
investors which is nice. We restarted the company and we were able
to with the 50 people that I hired out of that Mid-sized company.
Have really a fully integrated organization that had all the components
that are necessary to do discovery and development.
Obviously not a lot of resources, not like a Pfizer, but certainly we had
individuals in the company that had expertise in all of these areas.
And enough to actually make some progress.
And so we focused, as I mentioned, on HIV and cancer.
And, so, you know, why HIV? Well, at the time, and now it's probably
10 billion. I'm not sure what the worldwide sales
are. but you know, when we were looking this
in early 2007, this was an $8 billion market.
So, there is a commercial opportunity clearly there, it had the highest success
rate of any phase three programs. So, if you had a drug that worked in
phase two when you put it in the phase three.
All but one of the HIV drugs that it moved in the phase three it made it to
approval. a very high success rate, so again, if
you're going to invest money, that looks like a nice profile.
We took a look at the main, three main classes.
Lots of protease inhibitors, as I mentioned, that we develop one here at
Aragon. Lots of nucleosides, very few NNRTIs, one
of which was not even on the market anymore.
and so we decided to go after the lead in RTI, which was doing over a billion sales
but still had significant issues. A lot of toxicity, drug interactions,
reproductive tox and mutations were developing against that product.
That we felt gave us a very interesting opportunity to try to get rid of the
three problems I defined and get something that worked versus resistant
mutations. So we did significant high throughput
screening to get initial hits for something that worked against reverse
[UNKNOWN]. and then moved into the design phase.
And so when you're looking at, you know, the HIV, reverse transcript A's and you
put your normal NRTI fits very nicely into a pocket.
But with mut, mutations in that pocket you find that, that drug doesn't fit
anymore. it's still a functional packet, but
you're no longer getting activity. So what do you have to do?
Well, you have to design something that fits into both the, the wild type virus
tightly, but also fits into the mutant virus.
with very little loss of activity. You have to be able to work against both.
and when it comes to drug design process inter rest and interactive process I
won't go through all of the chemistry and bore you.
But, needless to say, from the original hit on the left, we eventually got to,
you know, the Ferrari on the right, that was very potent.
Showed all the properties that we were interested in, and worked very nicely
against resistant mutations. And, as you can see the Y181C.
And why Y188L had very good activity as well.
and obviously dramatic improved activity compared to the original hit.
so we took that compound into phase 1. Phase 1 being in healthy volunteers, make
sure it gets absorbed, it's not toxic, check for the CNS toxicity that's
[UNKNOWN] had. And make sure there's no laboratory
abnormalities and lo and behold we find something interesting, it lowers uric
acid levels. and it lowers them quite profoundly and
in both a dose related and base line related fashion.
And here you can see the baseline values of serum uric acid and anything above 7
is where uric acid starts to crystallize out in joints.
And eventually can lead to galp and you can see that the decline.
The change in uric acid in those patient was healthy voluntarious I should say was
the greatest across. what we saw, so here we had something
that worked particularly in people that had high levels of uric acid.
Look interesting so you know it's easy just to say that's an interesting
observation. And move on to the next page but we were
fortunate that we, we took a second and said okay, this is an interesting
observation. you know, now what do we do about it?
So, the first step was to determine why. Why did it do this?
Because this was clearly an unanticipated activity.
so we wanted to know the mechanism of action.
I wanted to find out, once we knew the mechanism of action, was it the drug or
was it a metabolite. Because we could tell that the drug was
metabolized in animals and so culture and in humans at that point.
And so you know, was a metabolite that was working versus the parent drug.
And then finally do we care you know hopefully this not something that's
going to harm patients and then you move on.
