Several key announcements have pushed Advanced Cell Technology's
(OTCBB: ACTC) stock from just under a nickel in late November to an
intraday high of $.20 during Wednesday's session and volume has
made it apparent that a growing crowd of day traders momentum
players have now joined biotech investors in cheering on the small
biotech.
The excitement around ACTC comes after a recent series of key
positive announcements including the fact that the FDA granted
orphan drug status to the micro-cap's patented embryonic stem cell
derived treatment for specific forms of Macular Degeneration and
blindness (Stargardt's Macular Dystrophy and Dry Age-related
Macular Degeneration). The eye conditions destroy the pigmented
layer of retina (retinal pigment epithelium) which is the pigmented
cell layer just outside the neurosensory retina that nourishes
retinal visual cells.
The condition destroys the pigmented layer of retina (retinal
pigment epithelium) which is the pigmented cell layer just outside
the neurosensory retina that nourishes retinal visual cells.
Advanced Cell Technology has shown success introducing the
embryonic stem cell derived RPE cells into the eye in animal models
and this resulted in 100% improvement with no side effects for more
than 220 days. Human clinical trials are expected to start in the
first quarter of 2011, and can potentially take only a few weeks to
show positive results.
Advanced Cell Technology has shown success introducing the
embryonic stem cell derived RPE cells into the eye in animal models
and this resulted in 100% improvement with no side effects for more
than 220 days. Human clinical trials are expected to start in the
first quarter of 2011, and could potentially take only a few weeks
to show positive results according to some analysts.
In addition, for only the second time in history (following
Geron's therapy for spinal cord injury), the FDA granted approval
for clinical trials for a therapy derived from human embryonic stem
cells.
William M. Caldwell, Chairman and CEO of ACTC tells
BioMedReports that ACTC plans on building upon their orphan drug
status and accelerating clinical testing. In addition, they hope to
continue showing promising advancements in other forms of
regenerative medicine which the company is developing -- most
notably its Myoblast program for the treatment of heart failure.
The Myoblast program (part of the company's 2007 acquisition of
Mytogen, Inc.) has successfully completed Phase I human clinical
trials and the FDA has finished reviewing the data, thus allowing
Advanced Cell Technology to proceed with a Phase II human clinical
trial (in approximately 160 patients) early next year.
BioMedReports: It appears that suddenly your
company has a lot of attention given your news developments. What
do you make of all the activity in your stock during recent
days?
William M. Caldwell, Chairman and CEO of
ACTC: The market is starting to appreciate the significance of
the FDA approval of our particular therapy. I think they are now
beginning to understand the strategy of having filed for an orphan
indication designation for Stargardt's Macular Dystrophy (SMD is
one of the most common forms of juvenile macular degeneration in
the world) and they now realize that that represents the first
wave, with potentially huge commercial opportunities in Dry AMD
(Dry Age-Related Macular Degeneration reportedly afflicts more than
30 million people worldwide, including an estimated 13-15 million
Americans). Both indications do not have viable therapies and so to
the extent that our program can make an impact, it's going to not
only help a very large patient population, it -- as well as any
other therapies that are approved -- will help validate a very
large technology.
BioMedReports: Can you help us digest or
simplify what some of those implications are?
William M. Caldwell, Chairman and CEO of
ACTC: I can certainly try. Right now, Genentech has a drug on
the market for Wet AMD [Note: the FDA approved Lucentis in 2006
after a 6-month priority review] and that patient population is
substantially less than the Dry AMD component. Their procedure is
to apply a needle into the eye every two to three months with their
therapy and for that they get some $2500, plus or minus, for each
injection. In our particular situation, we are inserting a needle
into the eye- which is something that is done all the time, by the
way, this isn't something that's foreign to the practitioner that
does the application -- but our application takes place only once
or possibly twice over the life of the patient. It is our
expectation that the therapy which we'll apply will have an impact
on either slowing down or arresting the progression of the disease.
We've seen that in our animal models. There have been some very
dramatic results when we've applied it into animals and we are
extremely hopeful that we will see the same types of results when
we apply it into humans.
The problem has been that this technology is totally new to the
world of medicine. It is an embryonic stem cell derived therapy. It
turns out that our cells have been derived utilizing what we call a
blastomere technology which means that we have been able to develop
those stem cells without any embryo destruction, which somewhat
mitigates the issues that have been in the media.
So, we take our particular stem cell therapy -- and remember
that the stem cells are converted into a single cell type so
there's really no actual stem cells into the therapy that we are
applying only a certain cell-type and in this particular case, it's
what they call RPE (retinal pigment epithelial) cells. That RPE
layer is in the eye between the photoreceptor and the Bruch's
membrane. It protects that photoreceptor -- which gives us the
ability to see -- and it also nourishes it. With deterioration, all
sorts of different diseases occur. One of which is Stargardt's
Macular Dystrophy and another of which is Dry AMD. Now, there are
certainly different characteristics to those, but to the extent
that you can replenish that RPE layer with new, healthy, viable
cells you have the opportunity to dramatically impact the
deterioration that is occurring within the photoreceptor.
That's a layman's description of what we're doing with respect
to that therapy, but more importantly the market implication is
such that if you have in excess of ten million patients currently
suffering from that disease; which is age related and as we know
the baby boomers are getting older. Unfortunately for those of us
that are getting into that post fifty-five or sixty year-old age
range, those individuals are prime candidates for this disease.
That market is fairly significant. There is an opportunity of
tremendous magnitude for a company like ours.
BioMedReports: Let's talk about the structure
of the company for a bit. There have been some concerns that there
are a lot of shares out there and that a company that is set up in
this way could suddenly announce something like a reverse-split
during a run-up in price like this one. What are your comments in
regard to that as far as ACTC goes?
William M. Caldwell, Chairman and CEO of
ACTC: I'm an investment banker and I can tell you that it has
been my experience that reverse splits for the sake of reverse
splits are very problematic. There has to be a rationale behind why
someone would do such a thing and it has to be done around some
sort of event that is accretive to shareholders and makes logical
sense for all the stakeholders. I'm not inclined at all to
recommend a reverse split unless that opportunity presented itself.
If it does, based upon our charter, we would then have to go to the
shareholders for their approval. In that way they would have an
opportunity to understand our rationale and determine whether that
makes sense for the majority of them. I think that's about all I
can say about that subject at this stage.
BioMedReports: Can you talk about any of the
upcoming milestones for the company? Some think that is part of the
reason for this run-up, that there are some events worth looking
forward to on the horizon.
William M. Caldwell, Chairman and CEO of
ACTC: I think we've alluded to some things a couple of times
either on blogs or in conferences, and I can start with the
approval of our IND for Stargardt's Disease which we will be seeing
some time in the first half of the coming year. (That will mark) us
going into the clinic. And we have already alluded to the fact that
we will go into multiple sites, not just one particular site, for
the reason that we have filed for Phase I/Phase II. For those who
are not familiar with that, Phase I really focuses on safety.
That's going to be a very, very important piece, not just because
of the safety, but because it will ensure for the FDA that this
cell type can be safe in humans.
You know the first one is always the toughest one, so we've
designed the trial to be very, very slow in its evolution. We have
a dosage escalation schedule whereby we're only inserting a minimal
amount of cells at the outset per patient. Then we will increase
that with ensuing patients and we'll pause to allow the FDA to
review the results of that so that they can feel comfortable with
the safety issues related to the fact that the cells go where they
are supposed to go and do what they're supposed to do and that they
don't cause any side effects, or tumors or anything else that has
been ballyhooed around. By the way, we have never, in any of our
studies, ever seen that.
So, I can't speak for others, but for us; our patented
differentiation processes are such that our cells are terminally
and totally differentiated into the cell type that we're dealing
with. Once we do that, then we'll move quickly into efficacy and
that tells us, of course, "does it work?" And that's why we're
starting out with multiple sites. Right now, I'm in the process of
finalizing those sites and developing a relationship with the
primary investigator -- the surgeon at each of the sites. I'm
working with the internal review boards to gain approval on the
protocols on any specific issues that they may have relative to
their particular situation and then we will initiate those trials
when all of that is completed.
The second major milestone is that we've filed a second IND.
That one is for the Dry AMD and we anticipate that it will take
much less time for the FDA to evaluate that, versus the time they
took to evaluate the first one. We anticipate that sometime in the
first quarter (of 2011) there is a very reasonable chance that we
will see approval for that IND -- at which time we will then
initiate trials for that program as well. Just so you know, it's
the same cells. So we're really just taking the same cell-type and
treating a different disease-type. So that's why we're relatively
bullish on that particular program.
A third area that we've announced is that as big as the market
is, and the opportunity is here in the United States, the European
community offers a similar opportunity. And with the E.U.
controlling the regulatory perspective for the various countries on
the continent, we will be looking very hard at the opportunity to
take both our Stargardt's and Dry AMD programs over there. I've
been spending some time over there trying to ascertain what the
best way is to do it and where the best places are to initiate the
trials as well as learning a little bit about the process of how to
work through the regulatory situations over there. We should be in
position to make an announcement about that in the first half of
next year.
We've also mentioned another disease condition called our
Myoblast or heart program. It's an adult stem cell -- meaning it's
the patient's own cells -- in this particular instance. We extract
out of the thigh in a biopsy and then we re-place it into the heart
with a catheter system. Basically, it goes over the dead heart
tissue from a heart attack that a patient has had. And what those
cells do, those myoblast cells, is they integrate with the good
cardio myocyte cells -- the heart cells -- and help those cells
pump the blood in and out of the heart. That's important because
when you have a heart attack, part of your heart muscle is killed
or dead, and unlike other parts of the body the muscle doesn't
regenerate itself and so the remaining muscle has to work harder
and the heart becomes a little weaker. Because of the strain on the
muscle it gets, sometimes, enlarged. The walls get thinner and
that's when you start seeing heart failure. What this does is that
it helps mitigate that and the patient can start feeling better.
That's really where the FDA is focused on, is the quality of life
of the patient. Most of the patients that we're dealing with are in
advanced age heart failure. So that is another disease condition.
We have gotten approval from the FDA to move out of the Phase I,
where we did four trials, and we're moving into Phase II. I've made
an announcement that we intend to do that in the first half of next
year. So again, that is another program that you should be hearing
some things from us about during the first half of next year.
So just in the first half of the coming year we have some fairly
significant milestones that we have before us and then there are a
couple that we're working on now that we haven't announced yet.
Investors interested in reading the full interview can do so at:
http://biomedreports.com/2010120961168/advanced-cell-technologys-momentum-train-remains-on-track.html
Biotech investors interested in accessing the news portal's
complete database of clinical trials and upcoming FDA decisions can
also access that information at:
http://biomedreports.com/fda-calendar/fda-calendar.html
News developments and live healthcare sector updates are
available constantly via twitter at:
http://twitter.com/BioMedReports
About BioMedReports.Com
BioMedReports.com is a news portal covering the biomedical news
and financial sector. BioMedReports is not paid or compensated to
report the news and developments of publicly traded companies in
the healthcare sector of the markets.
Add to Digg Bookmark with del.icio.us Add to Newsvine
Media Contacts Only: Mary Davila Assistant Editor
BioMedReports.Com e-mail: Email Contact Tel: +1 323 472 4480 Fax:
+1 888 210 3556