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  • tony_montello2003 tony_montello2003 Dec 8, 2013 2:14 PM Flag

    World Stem Cell Forum, San Diego, CA, Dec. 4-6, 2013 (Part 1) $$$$$$$$$$$$$$$$$$$$$$$$$$$

    World Stem Cell Forum, San Diego, CA, Dec. 4-6, 2013

    I'm Gary Rabin, the CEO of Advanced Cell Technology. We are a publicly traded company, over-the-counter. The ticker is ACTC. We have three clinical trials ongoing right now involving retinal degenerative disorders. We have one age-related macular degeneration trial, which is really the big kahuna in the sector. It's a massive unmet medical need in the dry form, and we have two Stargardt's trials going on. Stargardt's is a juvenile onset, typically juvenile onset macular dystrophy, and we've got a trial going in the United States and in the UK. We are close to closing out Phase I of all three of these trials.
    One of the company's principal cornerstones and more of the special sauce of the company, is we believe that we have some of the world's best developmental biologists, so what we take is a pluripotent stem source and from that we culture it into a variety of different kinds of tissue. In the case of our lead program here, we're talking about turning it into something called retinal pigment epithelium, and I'll talk more about that in a little bit. We've got a lot of intellectual property around this, not only in deriving the RPE cells from a source of pluripotent stem cells, but also the clinical applications, how they're injected, the degrees of phagocytic property that are exhibited by the cells, waste removal properties, and all other kinds of important filings related to the function and capabilities of these cells. Again, we're using a renewable pluripotent source of stem cells, so we back in 2005 we created about a dozen cell lines. Because embryonic stem cells, which is the progenitor source for these cells, replicates infinitely, there is a limitless supply of our ability to make these doses. We can make millions, billions, trillions of doses of anything from our master cell bank, of course given enough time for expansion.

    Sentiment: Strong Buy

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    • BUMP FOR TONY MONTELLO

      ++++++++++++++++++++++++++++++++++++++++++++++++++

      WORLD STEM CELL FORUM, SAN DIEGO, CA, DEC. 4-6, 2013 $$$$$$$$$

I'm Gary Rabin, the CEO of Advanced Cell Technology. We are a publicly traded company, over-the-counter. The ticker is ACTC. We have three clinical trials ongoing right now involving retinal degenerative disorders. We have one age-related macular degeneration trial, which is really the big kahuna in the sector. It's a massive unmet medical need in the dry form, and we have two Stargardt's trials going on. Stargardt's is a juvenile onset, typically juvenile onset macular dystrophy, and we've got a trial going in the United States and in the UK. We are close to closing out Phase I of all three of these trials. 
One of the company's principal cornerstones and more of the special sauce of the company, is we believe that we have some of the world's best developmental biologists, so what we take is a pluripotent stem source and from that we culture it into a variety of different kinds of tissue. In the case of our lead program here, we're talking about turning it into something called retinal pigment epithelium, and I'll talk more about that in a little bit. We've got a lot of intellectual property around this, not only in deriving the RPE cells from a source of pluripotent stem cells, but also the clinical applications, how they're injected, the degrees of phagocytic property that are exhibited by the cells, waste removal properties, and all other kinds of important filings related to the function and capabilities of these cells. Again, we're using a renewable pluripotent source of stem cells, so we back in 2005 we created about a dozen cell lines. Because embryonic stem cells, which is the progenitor source for these cells, replicates infinitely, there is a limitless supply of our ability to make these doses. We can make millions, billions, trillions of doses of anything from our master cell bank, of course given enough time for expansion.

      Within the eye, which is the focus of course, we created a whole bunch of different kinds of tissue. We have actually got some interesting things going on in the corneal epithelium. We've developed ganglion nerve cells, which could be used for glaucoma. We've developed photoreceptors and photoreceptor precursors, and we've even seen some very interesting secreted neuroprotective biologic agents, so there's a possibility that we could make something a drug would allow, that could be very exciting. We also have some preclinical things going on. We've got something pretty exciting in uveitis using mesenchymal stem cells.

The main program of course is the RPE cells. We've got these three ongoing trials and the first quarter of next year we'll start a trial for myopic macular dystrophy, which is a severe form of myopia that causes distention and fissures in the RPE layer. This is the stats of our program. We've got these two programs that are in the clinic right now, about to wrap up Phase I. That's the dry AMD and the two SMD trials here and in the UK. And behind that we've got a variety of other things we've developed. 

I assume most the people here are relatively familiar with the retina, but it's sort of the back story of what Sanjay just said. The light goes through the nerve cells, the nerve layer and basically gets refracted at the retinal pigment epithelial layer. The RPE layer has multiple critical roles in the health and function of the eye. It provides nutrients and growth factors. Photoreceptors don't see any blood. They play an important role in protecting the photoreceptors. They recycle Vitamin A. They detoxify the photoreceptor layers. They play a very important role. Essentially what happens, and importantly also, they maintain and groom the Bruch's membrane there, which is the angiogenic barrier between the front and the back of the eye. The important part about these cells, is that the failure of these cells, yet typically happening as a result of aging, it results in many degenerative diseases. What happens is toxicity builds up in the back of the eye there and it kills off the photoreceptors. Interestingly, the photoreceptors have a relatively long period of sort of quiet stability, where they're not shedding their outer layer, but they seem to be rescuable. And we've seen the results of that in our clinical trials. We'll be talking to you more about that in January. 

AMD is a massive unmet medical need. It will represent the most important parts of an epidemic in the coming years with expected increase of about 50% in the occurrence of this as the population ages, both from increased life expectancy and for the baby boomers themselves. So you see that roughly tripling and quadrupling in frequency of recurrence by age group, starting you know the low 2% in the 50s, then you're sweeping up into the 6-7% in the 60s, and by the time you're into your 80s, it's close to 40%, so very significant disease. Anybody who has seen those commercials on television that talks about who's the oldest person you know, but the reality is, this disease 40 to 50 years ago no one really cared about, because if somebody was going blind at the age of 70, it would make no difference, but today, an otherwise healthy 72-year-old might have another 10 to 25 years life expectancy. The statistics are simply remarkable the number of people that are greater than 55 years of age, you can just see the numbers here, and greater than 80 years of age it's going to actually increase by about 200 million people in the next 30 years or so.

      The good thing about the cell therapy in working with the eye is that unlike other parts of the body where you're doing, the potential for significant transplants and other things, you've got to create 10's of millions or maybe 100's of millions or even billions of cells. The advantage of the eye is that the dosage that we're using right now in our clinical environment is less than 200K cells. So one six-well plate could effectively treat 50 or 75 patients. So the scalability of that is remarkable. Just in our little manufacturing facility in Marlboro, Massachusetts, we can make enough cells to treat about 500,000 cases a year. So you've got big scale opportunities with this. 

Very simple ease of administration. It's an off-the-shelf cannula. We're actually working on making this a little bit more mechanized so that you can take an extra doctor out of the room, because right now there are two docs in the OR who do the surgery. The back of the eye is an immune privileged site. In abundance of caution in Phase I, we have been using a pretty rough regime of immunosuppression, both Tacrolimus and MMF, and Phase II now we're working with our immune specialists, and we're going to have certainly multiple arms and perhaps some arms with no immune suppression at all. The patients are weaned off of the immune suppression after a relatively short period of time, and we've seen survival and persistence of visual acuity improvements and the cells surviving, engrafting and continuing to replicate until confluence in patients that are now over two years out. Like I said, the procedure is very simple. I won't show you the procedure today to keep things short, but all off-the-shelf materials. It's under 2-minute surgery. Obviously there's some prep time, but it's a very straightforward activity. It involves a vitrectomy first.

Our clinical trial partners are four of the top five eye hospitals in the country. The lead investigator was Stephen Schwartz from Jules Stein Eye Institute. We also have Mass Eye and Ear and Wills Eye Institute, and Bascom Palmer here in the US, and then in the UK the trial is being lead by Dr. Jim Bainbridge at Moorfields Eye Hospital, which is the top eye hospital in Europe. We have been thrilled by the fact that we're a little company, when we have really these important key ophthalmology leaders that are kind of helping us drive our trial forward, and the month before last we had a meeting with all of the investigators in Miami, as we were planning and designing Phase II, so we've got all these great minds helping us along. It really makes a big difference to us. 

We're close to hiring a new Head of Clinical Development for the company. He's an ophthalmologist with significant experience in both the back and front of the eye, so we're really looking forward to that. That will be an important step for us as we move into Phase II of these trials. 

Designing the trials for us is fairly straightforward. The original structure of the trial was four cohorts with 3 patients each, in an escalating dose in the trial. We started on 50K cells. Next cohort 100K, next cohort 150K, fourth cohort 200K. Our plan is probably to abandon the 200K cell treatment. We've seen very little dose response in both the cohorts, so we think we're going to move to Phase II probably without that. We may keep the 200K cells alive in maybe one of the trials just to see what that does and see whether or not there is any dose response there, but we don't think that that's going to be really very important. 

The good thing about these cells is that both in vitro and now we're seeing in vivo also, the cells continue to replicate until they achieve confluence. We saw that in the dish too. In the dish we saw these cells continue to replicate until they were basically brushing up against each other, so we're seeing evidence of that also in the human eyes as well.

We got approval earlier this year to do a special Cohort 2a in the US for the AMD and SMD trials, where we're treating better visual acuity patient population, patients with as good as 20/100 vision. It's been a little tricky to recruit that patient population, especially in the SMD trial because these patients are a little bit younger and because it's a Phase I trial, with this heavy immune suppression, we've had some reluctance to get some patients enrolled, so that cohort has taken us a little bit longer in the SMD trial, but we'll be wrapping it up pretty soon.

We talked about how we're expanding the programs. We're going to do this myopic macular dystrophy trial. We'll start that early next year. 

We treated our first two patients and published the results in the Lancet back in January of 2012, and that was a very preliminary report. Obviously it just covered the first two patients three months in, but the paper showed that there was engraftment, that there were visual acuity changes and interestingly, those visual acuity changes have persisted now for the entirety of the two years for these patients, so we're very pleased about that. Overall we can say that as of this date we've treated about 31 patients. A lot of the patients have two years of follow up. We're coming up on 19 patients with a year of follow up. So what we're seeing is some very exciting and measurable visual acuity changes that we're going to be talking about giving some Top Line Data to be released early next year. The visual acuity is improved for the majority of the patients. Only one patient's visual acuity has really significantly worsened, other than one patient who got wet AMD in the treated eye and another patient got a cataract, but both patients we believe are on the road to maintaining the visual acuity improvements that they had seen initially.

      Question: Only one patient with wet AMD?

Gary Rabin: One patient got wet AMD and one patient got a cataract, which is by the way, you know vitrectomy, doing all these vitrectomies, it's surprising actually this long out that we've only had one cataract and one wet AMD.

So what we have seen regularly in these patients, we met with all the doctors last month. We went through all the fundus photos and all the OCT of all the patients, and what we were seeing is increasing pigmentation over time and kind of across the board. You can see that in these photos. 

Then finally, we've got a very dedicated management team. Dr. Robert Lanza is our Chief Scientific Officer. He and Dr. Klimanskya are essentially the creators of this RPE that we've been able to derive, and as we refine these cells and able to identify the important characteristics about their pigmentation, their phagocytic activity and so forth, as we evolve, that's an important part of our development bio.

      Sentiment: Strong Buy

    • Re: $$$$$$$$$$$$$$$$$$$$$$$$$$$

      tony_montello2003 • Dec 2, 2013 6:54 PM
      Ladies and gentlemen, Boys and girls please mark December 5, 2013 on your calendar, ACTC PPS will breakthrough new 52 week high Dec 5-6 2013!
      Let it be written,
      Dr. Tony Montello
      Sentiment: Strong Buy

      : ) ......... so let it be done.

    • Part 2
      Within the eye, which is the focus of course, we created a whole bunch of different kinds of tissue. We have actually got some interesting things going on in the corneal epithelium. We've developed ganglion nerve cells, which could be used for glaucoma. We've developed photoreceptors and photoreceptor precursors, and we've even seen some very interesting secreted neuroprotective biologic agents, so there's a possibility that we could make something a drug would allow, that could be very exciting. We also have some preclinical things going on. We've got something pretty exciting in uveitis using mesenchymal stem cells.

      The main program of course is the RPE cells. We've got these three ongoing trials and the first quarter of next year we'll start a trial for myopic macular dystrophy, which is a severe form of myopia that causes distention and fissures in the RPE layer. This is the stats of our program. We've got these two programs that are in the clinic right now, about to wrap up Phase I. That's the dry AMD and the two SMD trials here and in the UK. And behind that we've got a variety of other things we've developed.

      I assume most the people here are relatively familiar with the retina, but it's sort of the back story of what Sanjay just said. The light goes through the nerve cells, the nerve layer and basically gets refracted at the retinal pigment epithelial layer. The RPE layer has multiple critical roles in the health and function of the eye. It provides nutrients and growth factors. Photoreceptors don't see any blood. They play an important role in protecting the photoreceptors. They recycle Vitamin A. They detoxify the photoreceptor layers. They play a very important role. Essentially what happens, and importantly also, they maintain and groom the Bruch's membrane there, which is the angiogenic barrier between the front and the back of the eye. The important part about these cells, is that the failure of these cells, yet typically happening as a result of aging, it results in many degenerative diseases. What happens is toxicity builds up in the back of the eye there and it kills off the photoreceptors. Interestingly, the photoreceptors have a relatively long period of sort of quiet stability, where they're not shedding their outer layer, but they seem to be rescuable. And we've seen the results of that in our clinical trials. We'll be talking to you more about that in January.

      AMD is a massive unmet medical need. It will represent the most important parts of an epidemic in the coming years with expected increase of about 50% in the occurrence of this as the population ages, both from increased life expectancy and for the baby boomers themselves. So you see that roughly tripling and quadrupling in frequency of recurrence by age group, starting you know the low 2% in the 50s, then you're sweeping up into the 6-7% in the 60s, and by the time you're into your 80s, it's close to 40%, so very significant disease. Anybody who has seen those commercials on television that talks about who's the oldest person you know, but the reality is, this disease 40 to 50 years ago no one really cared about, because if somebody was going blind at the age of 70, it would make no difference, but today, an otherwise healthy 72-year-old might have another 10 to 25 years life expectancy. The statistics are simply remarkable the number of people that are greater than 55 years of age, you can just see the numbers here, and greater than 80 years of age it's going to actually increase by about 200 million people in the next 30 years or so.

      Sentiment: Strong Buy

      • 1 Reply to tony_montello2003
      • part3
        The good thing about the cell therapy in working with the eye is that unlike other parts of the body where you're doing, the potential for significant transplants and other things, you've got to create 10's of millions or maybe 100's of millions or even billions of cells. The advantage of the eye is that the dosage that we're using right now in our clinical environment is less than 200K cells. So one six-well plate could effectively treat 50 or 75 patients. So the scalability of that is remarkable. Just in our little manufacturing facility in Marlboro, Massachusetts, we can make enough cells to treat about 500,000 cases a year. So you've got big scale opportunities with this.

        Very simple ease of administration. It's an off-the-shelf cannula. We're actually working on making this a little bit more mechanized so that you can take an extra doctor out of the room, because right now there are two docs in the OR who do the surgery. The back of the eye is an immune privileged site. In abundance of caution in Phase I, we have been using a pretty rough regime of immunosuppression, both Tacrolimus and MMF, and Phase II now we're working with our immune specialists, and we're going to have certainly multiple arms and perhaps some arms with no immune suppression at all. The patients are weaned off of the immune suppression after a relatively short period of time, and we've seen survival and persistence of visual acuity improvements and the cells surviving, engrafting and continuing to replicate until confluence in patients that are now over two years out. Like I said, the procedure is very simple. I won't show you the procedure today to keep things short, but all off-the-shelf materials. It's under 2-minute surgery. Obviously there's some prep time, but it's a very straightforward activity. It involves a vitrectomy first.

        Our clinical trial partners are four of the top five eye hospitals in the country. The lead investigator was Stephen Schwartz from Jules Stein Eye Institute. We also have Mass Eye and Ear and Wills Eye Institute, and Bascom Palmer here in the US, and then in the UK the trial is being lead by Dr. Jim Bainbridge at Moorfields Eye Hospital, which is the top eye hospital in Europe. We have been thrilled by the fact that we're a little company, when we have really these important key ophthalmology leaders that are kind of helping us drive our trial forward, and the month before last we had a meeting with all of the investigators in Miami, as we were planning and designing Phase II, so we've got all these great minds helping us along. It really makes a big difference to us.

        We're close to hiring a new Head of Clinical Development for the company. He's an ophthalmologist with significant experience in both the back and front of the eye, so we're really looking forward to that. That will be an important step for us as we move into Phase II of these trials.

        Designing the trials for us is fairly straightforward. The original structure of the trial was four cohorts with 3 patients each, in an escalating dose in the trial. We started on 50K cells. Next cohort 100K, next cohort 150K, fourth cohort 200K. Our plan is probably to abandon the 200K cell treatment. We've seen very little dose response in both the cohorts, so we think we're going to move to Phase II probably without that. We may keep the 200K cells alive in maybe one of the trials just to see what that does and see whether or not there is any dose response there, but we don't think that that's going to be really very important.

        The good thing about these cells is that both in vitro and now we're seeing in vivo also, the cells continue to replicate until they achieve confluence. We saw that in the dish too. In the dish we saw these cells continue to replicate until they were basically brushing up against each other, so we're seeing evidence of that also in the human eyes as well.

        We got approval earlier this year to do a special Cohort 2a in the US for the AMD and SMD trials, where we're treating better visual acuity patient population, patients with as good as 20/100 vision. It's been a little tricky to recruit that patient population, especially in the SMD trial because these patients are a little bit younger and because it's a Phase I trial, with this heavy immune suppression, we've had some reluctance to get some patients enrolled, so that cohort has taken us a little bit longer in the SMD trial, but we'll be wrapping it up pretty soon.

        We talked about how we're expanding the programs. We're going to do this myopic macular dystrophy trial. We'll start that early next year.

        We treated our first two patients and published the results in the Lancet back in January of 2012, and that was a very preliminary report. Obviously it just covered the first two patients three months in, but the paper showed that there was engraftment, that there were visual acuity changes and interestingly, those visual acuity changes have persisted now for the entirety of the two years for these patients, so we're very pleased about that. Overall we can say that as of this date we've treated about 31 patients. A lot of the patients have two years of follow up. We're coming up on 19 patients with a year of follow up. So what we're seeing is some very exciting and measurable visual acuity changes that we're going to be talking about giving some Top Line Data to be released early next year. The visual acuity is improved for the majority of the patients. Only one patient's visual acuity has really significantly worsened, other than one patient who got wet AMD in the treated eye and another patient got a cataract, but both patients we believe are on the road to maintaining the visual acuity improvements that they had seen initially.

        Sentiment: Strong Buy

 
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