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is developing chaperone proteins that target
receptor molecules that reside on the cells of certain tissue types to provide specific
tissue targeting of therapeutic molecules and reduce side-effects in other tissues
Raptor Pharmaceutical Inc.
9 Commercial Boulevard, Suite 200
Novato, CA 94949-6118
Christopher M. Starr, Ph.D.
Chief Executive Officer and Director
Kim R. Tsuchimoto, C.P.A.
CFO, Secretary and Treasurer
Interview conducted by:
Lynn Fosse, Senior Editor
Published - March 15, 2007
Christopher M. Starr, Ph.D.
Dr. Starr is a co-founder, Chief Executive Officer and a director of Raptor since its
inception in 2006. Dr. Starr co-founded BioMarin Pharmaceutical Inc. in 1997 where he last
served as Senior Vice President and Chief Scientific Officer. As Senior Vice President at
BioMarin, Dr. Starr was responsible for managing a Scientific Operations team of 181
research, process development, manufacturing and quality personnel through the successful
development of commercial manufacturing processes for its enzyme replacement products, and
supervised the cGMP design, construction and licensing of a manufacturing facility.
From 1991 to 1998, Dr. Starr supervised research and commercial programs at BioMarin's
predecessor company, Glyko, Inc., where he served as Vice President of Research and
Development. At Glyko, Inc., Dr. Starr directed the research and development and
commercial operations of the organization. Prior to his tenure at Glyko, Inc., Dr. Starr
was a National Research Council Associate at the National Institute of Health studying
nuclear membrane transport, cell surface receptor function and protein intracellular
trafficking. Dr. Starr earned a B.S. from Syracuse University and a Ph.D. in Biochemistry
and Molecular Biology from the State University of New York Health Science Center, in Syracuse,
Raptor develops novel biopharmaceuticals and drug-targeting platforms derived from the
human receptor-associated protein (RAP) and Mesd chaperone proteins. The RAP technology is
envisioned to selectively target the delivery of engineered biopharmaceuticals to organs,
tissues and cell types through the use of specific receptor systems. Raptor is developing
RAP as a blood-brain barrier delivery platform that can be used to transport a wide
variety of intravenously administered protein therapeutics into the brain for the
treatment of neurodegenerative diseases. Raptor is also exploring applications for RAP for
the treatment of cancer and will initially focus on a potential treatment hepatocellular
Raptor Pharmaceuticals Corp. is a publicly traded
company (OTCBB:RPTP), whose wholly owned subsidiary, Raptor Pharmaceutical Inc. (Raptor)
was founded in September 2006 based on technology and intellectual property acquired from
BioMarin Pharmaceutical Inc. (Nasdaq/SWX: BMRN.) This technology was developed by Raptors
founders while working at BioMarin. In May 2006, Raptor completed a $5M first-round
financing through a reverse merger with a non-operating public company.
CEOCFO: Dr. Starr, what was your
vision when you founded Raptor and how is that developing?
Dr. Starr: The technology we used to
found Raptor was actually developed through work done at a company called BioMarin
Pharmaceutical Inc. and we are a staff originally from BioMarin. I was a cofounder of
BioMarin and my chief scientist there and I were cofounders of Raptor. The basic
therapeutic technology that we founded Raptor on was developed through about three or four
years of work at BioMarin in the research laboratory and represents therapeutic
applications in areas, that BioMarin is not going to pursue. Through a series of events,
we acquired the technology and funded the new company. We are now pursuing the
applications around this new technology at Raptor.
CEOCFO: What are the applications for this
Dr. Starr: The therapy is based on the
use of a protein that is called receptor-associated protein or RAP, which is where the
name of the company comes from. We are looking at three different applications; one is in
the area of transporting protein therapeutics to the brain. In that regard, the RAP
protein, which is a normal human protein, is actually used to transport other potentially
efficacious therapeutics to the brain. It has the potential to delivering therapeutics
across the blood-brain barrier, which is a huge medical problem right now in the treatment
of neurodegenerative and CNS (Central Nervous System) diseases. We are working with some
in-house technology and also collaborating and developing technology with other folks to
develop potential therapeutic molecules. We are also working with a couple of indications
in the area of cancer, with our initial focus on primary liver cancer which affects
about 20,000 patients in the US every year, with over a million patients outside the US
and there is really no known therapy that works very well in this area. We are testing
RAP-drug conjugates to improve the liver delivery of already approved therapeutics to
treat that disease. Another cancer target that we are developing is in the area of lung
cancer. There is a type of lung cancer called mesothelioma, which is a severe type of lung
cancer that is associated with the exposure of patients to asbestos and takes 20 to 30
years to develop after the asbestos exposure. It affects about 3,000 patients in the US
with the incidence rate actually increasing in both the US and the developed world because
of the presence of asbestos in the environment. We also just recently in the last week or
so, licensed in some technology from Washington University called Mesd, which represents a
second type of therapeutic protein that works very similar to RAP in the human body. It is
going to be a transporter of therapeutics and also a therapeutic in its own right. We are
working with the folks at Washington University to develop that for the treatment of
cancer and aggressive end-stage breast cancer and also as a potential therapy for
Will you tell us about the trafficking, and how have you chosen the likely applications?
Dr. Starr: The trafficking or
targeting that we are trying to do with these molecules is a fundamental issue in drug
development. Many people are addressing this more creatively. Part of the problems with
the drugs that people take is if they are not targeting the tissues that are affected,
then you end up with the potential for a variety of side effects. They go to tissues that
really should not see the drug and you have these complications or safety issues. That is
why a lot of the drugs on the market have been withdrawn in the last few years. It is not
because they dont work; they do work, but the problem is that they go to places they
should not be delivered to. The technology we are using is referred to as first-in-class
in the sense that the proteins we are talking about are chaperone proteins and they target
individual receptor molecules that we know reside on the cells of certain tissue types. We
are effectively trying to increase the targeting of specific molecules to treat diseases.
We are targeting specific diseases because of the biology; we are looking at the receptor
molecules that these proteins target. We are looking at diseases where these molecules are
at the highest levels and those are the diseases that we go after initially because the
chances of having a successful targeting event are much improved. The list I just went
through is a partial list of all the potential applications that we think we can pursue.
They represent what we refer to here as the low hanging fruit, the ones that
are the easiest to develop and would have the shortest timeline to getting into the
What did you like specifically about the Mesd?
Dr. Starr: Our second protein, Mesd
binds to the same type of class of receptors that the RAP protein binds to. We know the
receptors very well, we know the distribution, and they are very well characterized. They
are the receptors that do the heavy lifting when it comes to getting lipids out of your
blood and regulating levels of HDL and LDL and fats in your blood. The difference is that
Mesd binds to a different subset of those receptors. It represents a protein that
complements what we are doing. It does not replace RAP in any of the areas we are working
in, but increases the potential clinical applications of the technology. It gives us the
second platform to work on. Each of these proteins, RAP and Mesd represent what we refer
to here as a platform for therapeutic applications. Mesd is a second type of chaperone
protein that expands the applications beyond what we think we can achieve with RAP alone.
Will you tell us about the financial picture at Raptor today?
Ms. Tsuchimoto: We raised $5 million at the end
of May. That will get us at least through the first calendar quarter of 2008 based on our
current plan. Our plan is to raise money before 2008.
You mentioned some partnerships; is there a specific plan on how you are going to develop
what you have?
Dr. Starr: We have a couple of
programs we believe can enter the clinic in the next 12 to 18 months. One of them is in
the area of the treatment of liver cancer; we have evaluated and we believe because of the
needs and the technology we have, we can move that quickly. The plan is to have a couple
of tracks in the company. One of the things we can do because of the nature of the
platform technology is we can develop a number of therapeutic areas simultaneously. What
we will do in the next 12 to 18 months is develop the liver cancer application through the
pre-clinical and hopefully into the clinical phase by the mid 2008. That project is going
to be on a track toward the clinical and will involve bringing in people who can prepare
regulatory documents and collaborate with people who can do preclinical work and
toxicology that we need to get done in order to file with the FDA to get into the clinic.
Therefore, that is in the clinical track.
What we have done with the brain delivery program is that is August of this year (2006),
we entered into a collaboration with Stanford University. We are working with the director
of the neurosciences institute at Stanford; he is a professor of neurology; Dr. William C.
Mobley, M.D., member of our medical and scientific Advisory Board. He and his team are
evaluating these RAP molecules as transporters. We support the work in his lab in addition
to the fact that he is putting some money into the project. We are incubating and moving
that project along virtually in the sense that there is not a lot of internal work that is
being done. What we are doing is providing reagents to test. They do all the animal
testing and antibody work that is generic work that can be done by people that are not
necessarily experts in RAP, but are looking at these molecules. We will continue to
develop collaborations like that with other institutes to develop the NeutroTransTM
technology. It is a complex technology. It is a more challenging therapeutic area;
certainly one with huge pay-offs with methods that will work well. It is incubating at a
research level over the next twelve months, and at the end of that period, we will
evaluate it to determine where we are in the project. At that point, if things are looking
particularly good we will be looking at partners in that project.
We have a delivery technology for the brain disease area. We are what I euphemistically
refer to as the FEDEX truck. We are the guys that deliver the cargo. The cargo
comes from other companies who have been working on a variety of therapeutics for brain
delivery but have run into challenges because the therapeutic they have, does not get into
the brain when injected intravenously and that is where we can help. We are not planning
on developing a therapeutic for Alzheimers disease, we are not going to be running
massive clinical trials on neurological diseases; we are going to be looking for partners
to help us with that. We will bring to them technology they can use, and license, we could
form partnerships around individual therapeutic proteins. That is the plan for that and
also for some of the cancer indications I mentioned, to find partners to help us move
those along as we further develop the technology in-house. We have a hybrid business
model. We have a small lab here at the company; it is a about 800 square feet. We have a
few people working in the lab along with my cofounder who directs the laboratory work. We
do a lot of esoteric testing; we develop the therapeutics that could be tested and then
send them out to be tested by our collaborators and at the same time, we can then run a
lot of our programs virtually. Despite the fact that we only have five people here, we
have a number of collaborations with Washington University and also at Stanford University
where there are people looking at these projects in addition to our internal work.
In closing, why should investors be interested in Raptor?
Dr. Starr: I think one of the reasons
is the management. There are a lot of startup companies out there in biotech. There are
many people with exciting new ideas and many of them are spin-offs from universities and
institutes because of the technology they are developing. One of the risks you run with
that kind of setup, with people that are not experienced in developing clinical molecules,
is that it takes a lot of experience to know how to take a molecule from the bench and
move it into the clinic. We talk a lot about our experience back in the BioMarin days, as
Kim Tsuchimoto and I worked together there for 8 years and we saw 2 molecules go from
discovery all the way to approval. I lead regulatory affairs during the time of our first
product approval. What you have here is a management team that is pretty well seasoned in
this area and that is an important factor, even for the layperson that is not familiar
with the science and has a difficult time evaluating the quality of the science. The fact
that you have people that are experienced; not only us internally but also our advisors;
you can see that on our website and we plan to continue to bring experienced professionals
into the company. We knew they were experienced people, these are people that when they
see a molecule at the discovery level, they are already trying to figure out what that
package is going to say and they are really seeing the molecules as a clinical and
ultimately a commercial molecule and that is really important.
Beyond that we are working in a number of areas where there is a huge unmet medical need
and this is one of the big successes behind BioMarin, which focused on niche areas. These
are where there are small number of potential patients, but areas where there was a huge
unmet medical need. I am talking in areas like our liver cancer application, and
mesothelioma. There are huge advantages to developing these types of drugs because the FDA
has created many vehicles for small companies to develop drugs quickly in the clinic for
areas where people are not being treated adequately. Like the BioMarin model, which I was
instrumental in creating. Raptor is very much a model in looking at small niche markets
and using things like fast track product designations to be able to accelerate into those
areas. The third point in terms of what Raptor is doing is in the neurological area. I
think that if you speak to any neuroscientist or anybody in the neuro field they will tell
you that delivering protein therapeutics across the blood-brain barrier is really the Holy
Grail of the industry. There are literally dozens of drugs that are stacked up against
this barrier, that if there was a way of delivering them, it could really open up a number
of potentially very exciting treatments for diseases such as Alzheimers, Parkinsons,
ALS and all the things that you hear about. This is a challenging area, there are many
companies that are trying to work on this that have not been successful. We think we have
as good a chance as anybody at cracking the problem.
The way we present ourselves to investors is a seasoned management who has been there
before, done it before, and BioMarin is now a $1.5 billion dollar biotech company and so I
think the track record of our success is clear. We are looking at utilizing orphan product
protection and accelerated regulatory guidance to help move our products into the clinic
as quickly as possible and areas of unmet medical needs. These applications could be
blockbusters in the sense that if it works in the neurological area, it could open up the
floodgates to development of a lot of new generation therapeutics for neurological
diseases. The FDA has recently issued public guidance on Alzheimers disease; it
references a study that says that a lot of the drugs being used in Alzheimers are
not very effective. The LA Times had an article a few weeks ago, that reports on a study
that points out that Alzheimers is not being treated adequately with the current
therapeutics. They are looking for a new generation of drugs and if we can provide a
transport technology that will allow protein therapeutics to be delivered; I think it is
going to change the practice of medicine and neurology. There is a lot of excitement
around these areas and Raptor offers the industry an experienced research and commercial
development team that has been involved in drug approvals in the past. We are in this
because we want to be successful.
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