BIO Conference PresentationFebruary 20th BIO Conference Presentation by Dr. McPherson
Dr. McPherson: Thank you very much and thank you BIO for inviting us
to present at this particular meeting. The first comment is the usual
cautionary statement, which is necessary particularly in the United
States. The second is some information about Biomira. We're carried on
the Toronto Stock Exchange as well as NASDAQ and there you see our
shares outstanding. What I'm also going to talk about is the cash
position of the company which at the current time is US $43.3 million,
in relationship to a PIPE, which is a private investment in public
equity and we have another $57 million US to draw down in relationship
to a total of 8.8 million shares. What I'm not going to talk about
today, because of time limitations, is the product pipeline and I'm
not going to talk at any length about our superb management group.
I'm going to be concentrating on our two near term products, which
relate to our particular area of expertise in proprietary
immunotherapy and organic chemistry, with respect to the development
of cancer vaccines. I think its fair to say that Biomira, having
started this work in the early Seventies, and currently in phase III
trials in the largest immunotherapy trial in metastatic breast cancer
in the world, is probably amongst the leaders in the area of cancer
vaccines.
The molecular biology 101 of this particular program relates to, what
is known as, a cancer-associated mucin. And this particular compound
was found virtually on all cancer cells as well as all endothelial
cells. In the case of normal cells, its found crossing the plasma,
crossing the cell membrane into the plasma and it has these branched
carbohydrate chains. In the case of cancer, what happens is there's a
perturbed synthesis of the carbohydrates, so that you have these
aborted or low molecular weight carbohydrates as well as areas of the
polypeptide chain, which are exposed. So the two vaccines that I'm
going to talk about today relate to the STn, sialyated Tn, which is a
disaccharide carbohydrate, known as Theratope, and BLP, for Biomira
Lipo Peptide 25, which is a 25mer component of the core polypeptide.
We're in a phase III clinical trial program for metastatic breast
cancer with Theratope. We've been given FDA Fast Track designation.
The previous speaker has explained to you the importance of Fast Track
designation. It's also important to know that we have about a 400
patient database of patients who have been treated with different
doses in different indications in phase I and phase II, with
Theratope. So we have a very substantial safety background and a
substantial pre-phase III evidence of clinical efficacy with respect
to survival in metastatic breast cancer.
The current trial is to look at 900 evaluable patients. We currently
have 928 total patients enrolled in 120 sites in 11 different
countries and these patients are all patients with metastatic breast
cancer who have responded to the first-line chemotherapy of the
investigator's choice. So the patient has metastatic breast cancer,
they're treated with whatever chemotherapy program the investigator
thinks is appropriate for that patient, in that setting, and patients
who show a response and by that I mean either a complete response, a
partial response or substantial stable disease, are then randomized
into two groups. One group, the treatment group, gets the Theratope
vaccine, which in phase II trials showed a substantial survival
improvement in patients with metastatic breast cancer, and the other
group gets non-specific immunotherapy, which is everything in the
Theratope vaccine, except the STn. So you can see that this is a
designer trial with 450 evaluable patients, in each arm, looking at
study endpoints, two primary study endpoints, time to disease
progression and survival, which has been approved by the FDA to go
forward and as I say we will be completing enrollment by the end of
March of this year.
If we look at the clinical trial timelines, we expect to complete
enrollment by about the end of March. We will have the initial interim
analysis done, with the Data Safety Monitoring Board, in the third
quarter of 2001, which is event driven and it's important to
understand when I say it's event driven that it's an intent to treat
trial that is determined, with respect to timelines, on events, which
is code for progression or deaths. And so according to the number of
events, we will have the first analysis opened up in the third quarter
of 2001 and that will look primarily at time to disease progression.
The second interim analysis will open about the middle of 2002 and
will be a survival analysis. And the third analysis, if needed, will
open in the middle of 2003 and again will be a survival analysis. So
what we have here is an active specific immunotherapy, controlled
non-specific immunotherapy, blinded, prospective, randomized clinical
trial in a community based setting, which is particularly acceptable
and attractive to regulatory authorities.
The numbers of breast cancer annual deaths, that is the mortality, is
approximately 135,000 and that will represent the basis of the market
that one will be looking at. And if one looks at a 30% penetration of
available patients and looks at pricing which is comparable to say
HER2/Neu or something of that nature, then the market should be of the
order, in peak year, which is either fourth or fifth year after launch
of about 400 million US dollars.
Our second program is BLP, Biomira Lipo Peptide 25, which consists of
that 25 amino acid mer, which forms part of the core polypeptide,
encapsulated in a liposome. It's important to recognize that we have
very strong patent position, proprietary protection in relationship to
this particular program. We're looking at this in stage IIIB and stage
IV non-small cell lung cancer.
The trials that are underway or have been completed are a phase I
trial, in which we looked at two different doses of the BLP25, 20ug
and 200ug given subcutaneously and we were satisfied with the safety
and the immunogenicity. But the immunogenicity we got, although it was
T-cell related, was not the right kind of T-cell response that we
wanted, which was T-cell proliferation. So we moved to another phase
I/II trial in which we looked at a very much stronger dosing and a
more frequent dosing using the Biomira Lipo Peptide, and in eight
patients we demonstrated very strong T-cell proliferation response in
aggressive non-small cell lung cancer. So we were very gratified by
that kind of tumor response, which in pre-clinical models showed
substantial evidence of regression, in most models. In August of 2000,
we started a phase IIb BLP25, again in patients that are going to
receive the 1000ug of the BLP25, at weekly intervals, for eight
consecutive weeks, and overall we expect to admit 166 patients.
Currently, that trial is under way in 10 centers in Canada. It's
recruiting slower than we had hoped and we're making a variety of
adjustments, probably going to add centers, to that trial in order to
try to accomplish full accrual sometime next year. And then, finally,
we have an additional trial, which has completed enrollment, in 18
patients where we've added BLP25, in the 1000ug dose, to get that good
T-cell proliferation response that I talked about, along with
liposomal IL-2. The IL-2 is the Roche molecule; we have signed a
formal agreement for use with Roche. And in our own patented and
proprietary protected liposomal program. We've found that that
product, given at doses of 500,000 units subcutaneously in the first
group of ten patients and 2,000,000 units subcutaneously in the next
group of eight patients, was tolerated satisfactorily and we're
looking at the immunology at the present time. The important step here
is whether or not we go forward and finalize the 166 patient trial or
whether we move into additional changes to that trial, so as to
perhaps improve the possibility of it being a pivotal trial.
If you look at the market size, with respect to this horrible disease,
you can see that the mortality is showing here 332,000 deaths in North
America, Europe and Japan. That constitutes the market. This product,
if it is successful in showing improvement in survival and
prolongation, with satisfactory therapeutic index, should have a
market revenue generation of, again in peak year, very close to
$1,000,000,000. There is no question this is just a horrible disease
and I'm very pleased to say that part of what we're doing with
therapeutic cancer vaccines now is participating in a new paradigm,
which sees cytokines, which sees therapeutic antibodies and which sees
therapeutic cancer vaccines incorporated into the conventional
approaches to the treatment of solid tumor malignancy, which includes
surgery, radiation, chemotherapy and hormones. So the armamentarium,
that's available now for the oncologist as we move forward, is going
to be very substantial. And as was talked about by Entremed, one can
see that these immune concepts are going to be additional to
chemotherapy and hormones, in particular, in mopping up residual
disease, particularly clones of multi-drug resistance cells, that are
residual after combination chemotherapy.
If you look at our patent portfolio, we have a very strong proprietary
position and if you look at the world patents, of course you will all
recognize that these are individual patents, issued or applied for, in
a variety of different jurisdictions. So that although there's
approximately 25 there, or there is 25 there, they represent probably
over 100 countries in which they've either been issued or applied. We
have very, very strong patent position with both respect to Theratope
and to the MUC1 peptide.
We are also seeking a corporate alliance and various speakers have
commented upon the importance of corporate alliances for a variety of
reasons. We believe that there are a variety of components that are
important with respect to a corporate alliance. We believe that they
need to have a commitment to oncology and a culture that's appropriate
for the relationship to work. We believe they need to have a worldwide
marketing history for solutions in healthcare and particularly
oncology. They need to have financial and capital resources and they
need to have a vision for the future for innovative oncology products.
These are oncology products that you've been hearing about today, that
are not chemotherapeutic and not hormonal, are the leading edge of the
development with respect to the therapy of solid tumor malignancies in
particular in combination with conventional products. We're moving
very aggressively with respect to this partnership. We expect to
conclude this, either by the end of this quarter or sneaking,
depending on lawyers, into the next quarter. But certainly this isn't
something in the far distance; we expect this to occur in the first
half of this year.
So that is the Biomira story, "The Cancer Vaccine People", and I take
great comfort in thanking you for paying attention to me and look
forward to your questions in the outreach room.
Thank you very much.