Exhibit 99.1
INVESTOR CONFERENCE CALL - APRIL 26, 2005
DON WEINBERGER:
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Good morning. In a moment I will introduce the speaker for this conference call,
but first let me share certain safe harbor points to keep in mind as this
conference call proceeds.
During the course of this conference call, certain forward-looking statements
may be made by Mr. Pursley. Such statements are valid only as of today, and the
Company disclaims any obligation to update this information. These statements
are subject to known and unknown risks and uncertainties that may cause actual
future experience and results to differ materially from the statements made.
These statements are based on management's current beliefs and expectations as
to such future outcomes. The Company is in the early stages of drug discovery
and development. Drug discovery and development involve a high degree of risk.
Factors that might cause such a material difference include, among others,
uncertainties related to the ability to attract and retain partners for the
Company's technologies, the identification of lead compounds, the successful
preclinical development thereof, the completion of clinical trials, the FDA
review process and other government regulation, the Company's pharmaceutical
collaborator's ability to successfully develop, manufacture and commercialize
drug candidates, competition from other pharmaceutical companies, product
pricing and third party reimbursement. As is always the case with early stage
companies, the Company will also be affected by factors involving continued
access to capital.
BILL PURSLEY:
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Good morning and thank you for joining us for the Ceptor Corporation conference
call which is open to the public. A copy of this call will be available for one
month via the call-in number provided in our press release. For those of you who
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may be less familiar with the Company, I'll first provide a very brief overview
of the company and our technology.
Secondly, we would like to share our interpretation of the outcome of our
pre-IND FDA meeting (an IND is an individual new drug application, which is
necessary to obtain in order to move any drug into human testing).
Thirdly, we will provide both scientific reasoning and aspects of the orphan
drug legislation, which management believes mitigates our risk at this stage of
development more than one may normally assume. As a reminder the Orphan Drug
Legislation was passed in 1983 with the purpose to provide incentives to
companies to develop products for diseases that affect less than 200,000 people
in the U.S, are life threatening and have no current definitive therapy. Similar
legislation is in place in the EU and Japan.
After those opening remarks, we will then open it up for a question and answer
session.
As a brief background, Ceptor is a biopharmaceutical company focusing on the
development of proprietary, cell-targeted therapeutic products for neuromuscular
and neurodegenerative diseases, primarily for those which are categorized as
orphan diseases, with a clear and relentless mission to increase the quality and
quantity of life of people suffering with these devastating diseases. Our
primary efforts, are being focused on moving our lead product, Myodur, into
phase I/II clinical trials for Duchenne's muscular dystrophy (DMD), which the
Company believes may represent a potential $2B+ world market. We are fortunate
to have a broad platform technology which also provides for the potential
internal development of several additional orphan drugs, such as ALS (Lou
Gherig's disease) and CIDP ("MS of the extremities") as well as technology for
non-orphan diseases that we intend to offer for partnering opportunities such as
multiple sclerosis, retinal degeneration and epilepsy.
In looking at small molecules, or orally delivered compounds, what normally
happens is that a very small percentage of that drug gets to where it is
supposed to go to do what it is supposed to do while the rest of it goes where
it is not supposed to go and does what it is not supposed to do, thus, setting
up the oldest conundrum in the business: the risk-benefit ratio of efficacy
versus safety.
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What we believe makes our technology unique is that we use naturally occurring
endogenous carrier molecules, that exist in all of us, connect our therapeutic
compounds to those carriers so they are directly targeted and delivered to the
effected cell or tissue, which potentially greatly increases, in a positive way,
the risk-benefit ratio. This would suggest our technology is safer, and in terms
of efficacy, more efficient. And in fact, we now have proof of concept of that
in animal models both in muscular dystrophy and multiple sclerosis.
The primary carrier molecules that we are working with today are carnitine,
which transports things into muscle cells and taurine, which transports things
into nerve cells. Our primary therapeutic compounds are calpain inhibitors.
Calpain is a protease (which is a fancy word for an enzyme that degrades tissue)
that exists in all of us. The normal job of calpain is to tear down muscle or
nerve tissue in balance with production of new muscle or nerve tissue in our
normal everyday metabolism, where we constantly lose and create new cells. In
many neuromuscular and neurodegenerative diseases, calpain is highly
up-regulated (or abnormally highly activated) and, therefore, degrades muscle or
nerve tissue at a much greater rate than it can be replaced, leading directly to
the symptoms of these diseases.
With our technology we can carry calpain inhibitors directly inside the cell to
down regulate calpain and preserve the muscle or nerve tissue in those diseases.
For example, if we look specifically at our lead product, Myodur, for Duchenne
muscular dystrophy, it is well published that calpain is the primary culprit in
this horrible muscle wasting disease. We have shown, in animal models that we
can deliver Myodur directly to the wasting muscle, down regulate calpain, and
preserve the muscle tissue, slowing or stopping the disease process. This is
very provocative news when you realize just how devastating this disease is. The
average age of diagnosis is 3-5 years old. Around the ages of 9-12 you become
wheelchair bound. Then, it is not often that you live past your 20th birthday.
Anything we can do for these boys (DMD only affects males) would be wonderful.
Let's now shift gears to the pre-IND FDA meeting and results. Again, keep in
mind you must receive an IND from the agency to initiate human clinical trials.
In preparation for a pre-IND meeting, you normally send the FDA all your current
scientific data a month before the meeting, which they thoroughly review. Last
December, we provided the FDA with an 81 page confidential report containing our
scientific data on Myodur and DMD.
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You then meet with the agency to present your proposal for the pre-clinical and
clinical development plan for your product and ask permission to move forward
with that plan. Then you contemplate whether the FDA will be at least somewhat
agreeable with your plan.
Management believes we had a very positive meeting with the agency as is
evidenced by the meeting minutes. We proposed and asked for permission to carry
out a development plan based on nine critical issues. The agency agreed with all
nine points. In our team's experience, this is a very rare and very welcomed
outcome. This, simply stated, means we just have to carry-out our own proposed
plan, which has been and is our exact intention. The FDA was very collegial and
we believe recognizes the gravity of this disease (DMD) in supporting our plan.
There were three very important goals the Company had going into that meeting
which we will share with you. First and foremost, we wanted to provide a strong
enough package and take appropriate advantage of the Orphan Drug Legislation to
ask for permission to initiate our clinical studies directly in diseased DMD
kids. The agency agreed that we could. This means we do not have to start our
safety testing in normal volunteers as is usually required. We believe that this
could save us between one to two years of clinical safety development time.
Second, we wanted to be able to go directly into a true phase I/II combined
safety and efficacy trial. Again, the agency agreed. The advantages of this are
very important to understand as many companies will simply use phase I/II as
semantics. This is a true phase I/II because it is under one study protocol, one
patient consent is signed, it will include safety, dose ranging and efficacy
endpoints, and will be double-blinded and placebo controlled from start to
finish. We believe that this could save an additional one to two years of
clinical efficacy development time.
Third, we received permission from the FDA to submit a request for Fast Track
designation. If granted, this provides for FDA review of a final NDA submission
(new drug application for marketing approval) in six months instead of the
normal year or two. Although it is not possible to say with great precision, all
these things combined could cut somewhere between two and a half and four and a
half years off of our development time.
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More importantly, when put in historical prospective, if we are successful in
carrying out our own proposed development plan, by the end of this year, we
could have an IND in hand, that, as far as we know, will provide for the first
potential definitive therapy ever for DMD to be tested in an efficacy trial in
diseased kids.
Finally, before opening up for questions, let's address a very important issue
as it relates to the perception of our level of risk and the risk normally
associated with a company at our current early stage of development. The
perception in general is fair, but management believes there are valid
scientific and regulatory reasons in the case of the development of Myodur that
significantly reduce our development risk.
Although the risks of drug development at the preclinical phase are
statistically high, we are optimistic that a number of factors substantially
mitigate the risk for Myodur in all three areas that generally pose major
stumbling blocks to standard development pathways--safety, efficacy, and
regulatory.
Firstly, although the safety profile of Myodur has yet to be determined in
standard toxicology studies, it has not demonstrated toxicity at efficacious
doses in the mdx mouse, a standard, well-known and accepted genetic animal model
of DMD. In addition, Myodur is composed of two well-tested component parts,
L-amino-carnitine and the active leu-arg moiety of leupeptin. Carnitine (our
muscle targeting carrier molecule) is an approved drug product by itself that is
a naturally occurring replacement therapy for dialysis patients and has an
excellent safety record. While leupeptin is not an approved drug product, it has
been tested extensively in multiple animal models and given in clinical trials,
including in patients with DMD, without deleterious effects. Although safety of
its component parts is not a guarantee of safety of Myodur, it strongly suggests
to us that a favorable safety profile will be demonstrated. Formal data from our
initial toxicology studies is anticipated in May 2005.
Secondly, the potential risk for lack of efficacy is reduced by an understanding
of the DMD disease process, proposed mechanism of drug effect, and existence of
a well-described animal model of the disease, the mdx mouse. Published data
establish a firm cause-effect relationship between calpain activation and
myonecrosis (muscle wasting) in dystrophin deficient muscle and clearly indicate
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that calpain inhibition, the primary activity of Myodur, may be of therapeutic
value in children with DMD. Because many drugs in development lack a clear
animal model of disease, the first demonstration of efficacy for these products
comes from human clinical studies. For Myodur, however, proof-of-concept data
demonstrate to us that Myodur reduces histologic (tissue) evidence of muscle
necrosis in a well-documented animal model (mdx mouse).
Further, it is known that both calpain (the enzyme inhibited by Myodur) and the
carnitine transport mechanism (the muscle-targeting mechanism of Myodur) are
well-preserved across animal species, suggesting to us that animal models are
likely to be highly predictive of human efficacy.
In the regulatory area, Myodur is an orphan product for a devastating, fatal
disease of childhood without an approved therapy. Given this clinical scenario,
the required benefit-to-risk ratio for approval is likely to be lower than for a
product treating a mild chronic condition for which there are already
therapeutic options. The FDA has already favorably reviewed development plans
for Myodur as I mentioned earlier.
Making sense of the most important part of truly understanding management's
belief in mitigating our development risk is the animal model. Only in rare
genetic diseases, in some cases, has a genetic animal model been successfully
produced specifically to imitate that genetic disease in man (the mdx mouse is
one of those). This provides expectation for a significantly higher predictive
value of how a drug will act in that animal model as compared to man.
In the vast majority of development programs, these types of animal models don't
exist and thus the predictive value is low and the failure rate moving into
humans is high. This is what has fairly led to the general perception of risk at
this stage of development.
This concludes my prepared remarks, but I would also like to direct our
shareholders to our recently filed Form 10-KSB for further disclosures of our
business plans, financial statements and risk factors. Also, these opening
remarks will be filed in an 8K today. Thank you.
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BILL PURSLEY:
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I would once again like to thank you for your interest in CepTor and we look
forward to updating you on our progress through press releases and quarterly
conference calls such as the one held today.
Thank you and have a pleasant day.
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