US stock - ACTC.OB, now changed to OCAT (Ocata Therapeutics)

IpohMan69

jcslho 发表于 24-2-2015 10:27 AM
Ipohman, Happy Chinese New Year. May all ur  and Ur family wishes and dream comes true. And stay h ...

jsclho,

Same to you and your family! Thank you!

I am on vacation and have not really paying attention. Let the game begins and good luck to all who invested.

IpohMan69

My view about the potentials of the company have not changed and in facts over times it has gone up...LOL.

But to understand all these one has to do extensive DD on this company based on the understanding of regenerative medicine, unfortunately.

Below is the transcripts of the recent Quarterly report CC. Thanks to Patti who transcribed the whole CC as usual, credit goes to her. There is a play back on OCATA website for 90 days(I think) if one wants to listen to it or google it.

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Ocata Conference Call - March 12, 2015
Transcript By, Patti

Paul Wotton: Good afternoon everyone and thank you for joining today's call. I'd like to start the call by noting that this is our first public call as Ocata Therapeutics following our corporate name change in November of last year. We believe that Ocata has quickly become known as a leader in Regenerative Ophthalmology, as we have begun the process of marketing the company. Our goal is to own the eye with a novel therapeutic approach, as we are the leading company in the clinic with this type of technology. This is also the first call where we have been listed on the NASDAQ Global Market Exchange. I'm pleased that we have reached this milestone. NASDAQ is the appropriate exchange for a biotechnology company like Ocata to be traded on with its advanced clinical programs.

Since the beginning of the 4th quarter, we have continued to make significant progress on both our lead clinical development programs and on the corporate front. Our transition from a technology platform company into a product development company, with a pipeline of clinical and preclinical assets has progressed substantially. This was highlighted during the quarter by the October 14th publication in the Lancet as we reported for the first time long-term safety and size of efficacy for RPE transplants with fully differentiated RPE cells that have been manufactured from stem cells in patients with dry age-related macular degeneration, or dry AMD, and Stargardt's macular degeneration, or SMD.

We have discussed these results in other calls so I won't go into detail again today. However, I do want to point out that the exposure and interest generated from the publication with regard to physicians, patients and potential marketing partners continues to grow. The Lancet publication has clearly strengthened our leadership position in Regenerative Ophthalmology, and we believe a strong signal of our ability to generate positive data in future studies. It's worth noting, however, that as we continue to track these patients, it appears that visual acuity gains that were reported in the Lancet have persisted and in some cases for more than two years since the patients first received our cell therapy. We also are now approaching the four-year safety timeline for some patients, and we continue to see that the transplants are behaving as one would like to see, a very hopeful sign for the future.

Our focus on the ophthalmology space also continues to be supported by market dynamics as well. Our own market research has confirmed that our therapies, if approved, could treat substantial numbers of patients with dry AMD and Stargardt's disease where no meaningful therapy exists today. The ophthalmology market continues to grow rapidly with annual sales in the US alone approaching $12 billion dollars. One product, for example Eyelea, marketed by Regeneron for wet AMD, exceeded $1.7 billion in sales in 2014 in only its third year on the market. This highlights the extent of the AMD disorder, which is rapidly becoming an epidemic in this and other developed countries. It's critical for us to be able to offer our therapy to as many patients as possible in the shortest time possible, and we're getting ready to begin our Phase II program in dry AMD, and our pivotal trial program for Stargardt's following a recent productive meeting with the European Medicines Agency, or EMA. Our number one corporate objective for 2015 is the initiation of the next phase of our clinical trials.

We plan to initiate our Phase II trial for dry AMD in the second quarter of this year. The purpose of the dry AMD trial is to evaluate the safety and exploratory efficacy of our RPE cells in a study with a parallel control group of patients. This Phase II study will include up to three cohorts of approximately 20 patients each. Each of the three cohorts will be treated with a different immune suppression regimen to determine how much, if any, immune suppression is required. We intend to complete the first cohort dosing during 2015 and the second two cohorts by the end of 2016.

We also plan to initiate our pivotal trial for SMD in the near term. The purpose of this pivotal trial will be to evaluate the safety and efficacy of our hESC-derived RPE cells. This trial will include approximately 100 subjects. As usual, Dr. Eddy Anglade will provide further detail around our clinical programs, but I also want to highlight that during the 4th quarter, Ocata was granted Advanced Therapy Medical Product, or ATMP designation, for this therapy for macular degeneration by the European Medicines Agency. This designation is an essential step in allowing companies to commercialize products effectively across the European Union. We also interpret this designation as a favorable indication of how the European regulators view our therapy. As a reminder, Stargardt's is an inherited disease with no available treatment option. SMD has orphan indication and it has a devastating impact by robbing younger patients of their vision.

Dry AMD is a complex disease influenced by a number of factors. As with SMD, there is currently no approved drug treatment. There are about 30 million patients in the US and Europe suffering from AMD, and dry AMD accounts for 85 to 90% of this number. The majority is categorized in early stages of the disease with generally good vision. There are about 110,000 to 165,000 new patients a year diagnosed with advanced dry AMD in the US, with so-called geographic atrophy, the hallmark of which is RPE cell loss and is characterized by impaired vision and significant damage to the macula. This is expected to grow as the population ages and represents the primary target segment for our dry AMD development program.

With that overview, I'd like to turn the call over now to Eddy Anglade to provide some additional detail regarding our upcoming Phase II clinical trial for AMD and the pivotal trial for SMD.

Eddy Anglade: Thank you Paul. First, as an update on the SMD and AMD studies just discussed, we have now treated 38 patients safely, and have completed the dosing at the 200K cell level, the highest dosing cohort planned in the three ongoing Phase I/II trials. A planned safety review of patients treated at the highest dose level by the DSMB now provides us with additional knowledge and confidence regarding safety at this dose level. As Paul previously mentioned, our key corporate objective is the initiation of our Phase II study for dry AMD and pivotal study for SMD. I am pleased to let you know that we have finalized our trial design and we're strengthening our capabilities and expanding our network of trial sites to support this next phase of product development.

I'll now provide you with some of the highlights of our clinical trial design and execution plan.

The initiation of the pivotal SMD trial is the top priority for Ocata. So this pivotal trial will target an enrollment of approximately 100 patients. The goals of the study are to evaluate the safety and efficacy of RPE cellular therapy. Outcome parameters will include assessment of visual function, as well as structural changes. Importantly, we plan to incorporate an untreated control arm to our study design, which we believe will allow us to recruit patients more quickly, and may ultimately demonstrate a higher degree of clinical benefit in our study subjects. Importantly, we believe these revisions are aligned with the guidance received by the US FDA for a potential pivotal trial. We now have a greater degree of certainty that our SMD program can be initiated as a pivotal trial, potentially allowing us to apply for marketing authorization upon its completion. Additionally, we are in the process of exploring an SPA, or a Special Protocol Assessment, with the FDA as a way to further optimize our regulatory path toward approval. We will provide additional updates in due course.

We plan to initiate a Phase II trial of approximately 50 to 60 patients in age-related macular degeneration in the 2nd quarter of this year. The goals of this trial will be to optimize the duration of concomitant immunosuppressive therapy required in the peri and post-transplantation period, as well as to more fully explore the efficacy signal that we saw reported in the Lancet in October of last year, in comparison to a control group of untreated patients, rather than using the fellow eye as a control as we had planned in the past. We plan to provide Top Line Data on the first cohort of the Phase II AMD trial by this time next year, and this is expected to be a key inflection point for this program, as it will represent the first time a control group has been used in this type of study with cell therapy.

I will now turn the call back over to Paul.

Paul Wotton: Thanks Eddy. I'm going to ask Ted Myles now to provide a brief corporate update.

Ted Myles: Thanks Paul. A key objective on the corporate side is to properly capitalize the company. This includes attracting institutional capital from investors who can partner with us over the long term and who can invest the level of capital required to fully fund our clinical trials. A critical step in this process was our recent accomplishment of uplisting our stock to NASDAQ. As many of you know, we began trading on the NASDAQ Global Market on February 26th under the ticker symbol OCAT. Uplisting the company's stock to NASDAQ has been an objective of the company for a long time. Aside from all of the obvious benefits of being a NASDAQ-listed company, we also believe that this listing allows us to demonstrate that we have resolved the legacy issues inherited from prior management. This allows us to move forward with a fresh start for the company.

Moving to financial results for 2014, I want to highlight two line items.

First, R&D expenses, excluding non-cash stock compensation, were approximately $9.6 million dollars for the full year. While this was a decrease compared to 2013, we expect R&D to increase year over year in 2015 as we initiate the next phase of our clinical trials.

G&A expenses, excluding non-cash stock compensation, decreased to approximately $9.2 million dollars in 2014. We expect this trend to continue as we believe we have resolved our legacy legal matters and therefore don't expect to incur significant legal costs related to those matters.

As of December 31, 2014, we had approximately $4.4 million dollars of cash and cash equivalence on hand and approximately $18.6 million dollars available to us under our arrangement with Lincoln Park Capital. As previously disclosed, we initiated the process of marketing the company to institutional investors in the 4th quarter of last year. We were pleased with the reception we received from several high-quality investors, and now that we have achieved the NASDAQ listing and we're nearing the initiation of our Phase II clinical trials, we look forward to reengaging the market and ultimately attracting institutional capital into Ocata. We look forward to updating you further as we continue to make progress against our plan, and we appreciate your continued support. Now I'll turn the call back to Paul.

Paul Wotton: Thanks Ted. As you can see this has been another significant quarter for the growth of this company and we're continuing to add new and experienced personnel to Ocata. I'm pleased to welcome John Heffernan to Ocata to the newly created position of Vice President of Manufacturing. John has a breadth of experience in all facets of manufacturing in the cell therapy space, most notably with Genzyme for 18 years in the Cell Therapy Regenerative Medicine Business Unit. His last position there was Vice President of Global Operations with facilities in Europe, Australia and the US. He has also held positions at Gene-Trak, Behring Diagnostics, Boston Scientific and Baxter. We believe that his experience and expertise will prove to be highly valuable to us as we prepare to initiate the next phase of our clinical trials for our lead programs in dry AMD, SMD and beyond.

While we focused on Regenerative Ophthalmology, we also continued to make progress in other areas. On our last call I highlighted the platelet publication that we announced on October the 17th, which enables us to seek collaborations with external groups that have the resources necessary to bring the results of our ground breaking technologies to patients in need.

Another example of illustrating the versatilities of Ocata's core technology relates to our recent announcement that the United States Patent and Trademark Office, or USPTO, issued two patents directed to the company's hemangio-derived mesenchymal cells, or hMCs, which we anticipate publishing in a top tier journal with a view towards engaging a partner in the future. These cells have had a degree of antiinflammatory and immune regulatory activity. This new technology enables the generation of a potentially unlimited supply of potent hMCs from renewable pluripotent stem cell sources. Perhaps most importantly, in preclinical work in inflammatory disease models such as lupus nephritis and Crohn's disease, we have seen very compelling and highly potent antiinflammatory activity in relevant animal models. In addition to hMC formulations, the issued patents also broadly protected the manufacture of hMCs as well as the therapeutic use of these cells, and the treatment of ophthalmic, autoimmune and inflammatory diseases.

In addition to our ophthalmic uses, this platform also gives Ocata the ability to carve out the non-ophthalmic uses, and to potentially partner for the treatment of devastating autoimmune diseases.

2015 will be a year of consolidation and development execution for Ocata as we build upon the sound foundation for a biotechnology business.

I've now been with the company for almost nine months, and while I continue to be very impressed with the science, what I see here is a real opportunity to change the face of medicine for the better with the use of regenerative and targeted cell therapy.

Amongst the planned milestones that we are working to achieve in 2015 will be:

1. The initiation of the pivotal trial for Stargardt's, with a control group of patients.

2. The initiation of a controlled Phase II trial for dry AMD in the second quarter of 2015.

3. Publication of positive data on Asian patients treated for Stargardt's and dry AMD with our RPE therapy, and that's replicating the Lancet data independently.

4. Presentation at key ophthalmology conferences and investor conferences.

5. Continued strengthening of our intellectual property platform with additional patents.

We continue to believe strongly in our mission here at Ocata. There are patients who need us to succeed so that we might bring them a curative therapy for Stargardt's and dry AMD. This is now becoming an execution story and we are building an organization that is focused on execution.

Thank you for your continued support and we look forward to reporting further progress as the clinical trials role out.

Operator can you now open up the call to any analysts that are on the line?

Jason Kolbert: Hi guys. This is actually Jason McCarthy (Maxim Group Equity Research Associate - Biotechnology) for Jason Kolbert. It sounds like everything is moving in the right direction, and it sounds like the trials are starting up, it's going to be exciting. I wanted to go back to the financials a little bit, and I know it's disappointing to have not gotten a deal done at the end of last year, and if you're going to do it this year, what point in the trial, or how much data do you think you're going to need to kind of drive that forward, and just make it successful this time around?

Paul Wotton: Well, the question really is about the progress we're making on the clinical trial front, and the important thing that we learned over the last quarter. There's a number of things that happened, the first is we have positive data from the Lancet. The second thing is towards the end of December, early part of the year, we started realizing that we'd have to change the way we designed our clinical trials to include control patients, which actually just changed the way that people view our company. We did have a meeting with the European Medicines Agency February 4th of this year, and as a result of that meeting, as well as continued dialogue we've had with other agencies, we have changed the design of both the dry AMD study and the Stargardt's disease study to ensure that we include a control group of patients, which we believe will give us a better outcome. It also harmonizes, we think, the studies between what the FDA will require for approval and what the European Agencies will require for approval, and as we mentioned on the call as well, we are also looking into the possibility of getting a Special Protocol Assessment with the FDA, which will help to de-risk the programs that we are working on and make the approval process more expeditious for us. So what we've tried to do in the past quarter is focus quantitatively on improving the design of our clinical studies.

Jason McCarthy: Thank you. I caught it before, but the new control is going to be what? No treatment, I missed it.

Paul Wotton: No, we actually didn't specify what the control is going to be, so you didn't miss anything.

Jason McCarthy: Okay, so I didn't miss it then.

Paul Wotton: The way we're going to do this, patients who are getting the therapy will receive the therapy. Control patients will not be receiving the therapy, but they will be put into a procedure, including being taken into the surgical room, where they will appear to have received the therapy. It's fair to say that we won't be doing a subretinal injection of anything like saline in that study, but it's important, this is about as close a control as we can get for these patients without crossing over the boundary of being unethical.

Jason McCarthy: Right. Okay great, thank you so much.

Caroline Corner (For Reference: Cantor Fitzgerald & Co. Continues Expansion of Healthcare Equity Research Team | Cantor Fitzgerald): To see all of the progress you've been making lately, just a couple of more questions about your SMD trial. First of all, with regard to sites, I think in a previous conversation you said you probably needed somewhere around 12 sites for this trial. How many have you identified and started enrolling to date?

Paul Wotton: Carolyn, I'm going to hand this question over to Eddy Anglade, who is knee-deep in this process right now, but thank you for the question.

Eddy Anglade: Yes Carolyn, thanks very much for the question. So right now we've got in the US 4 trial sites that are participating in both the SMD and AMD trials, and we're gearing up obviously to start the AMD trial next quarter. With respect to SMD, as Paul mentioned and I did as well, we will submit the protocol under SPA to get better surety around the design and so forth. So in terms of the sites, we will ramp up quickly thereafter to hit the target, assuming that we've reached accord with FDA on the SPA.

Caroline Corner: Just a follow-up question on the question you just had on the control arm. So this isn't a blinded study because the control arm is not actually getting an injection, as I understand it, correct me if that's wrong. What exactly are you doing to the control patients when they come in, you bring them in for an eye exam, and then what happens?

Eddy Anglade: So let me just explain the control a bit more. So the idea is to minimize the likelihood that the patients can guess that they have received treatment or not. So the patients will be obviously consented and they will be randomized either to treatment or nontreatment, and those patients who are randomized not to treatment, they will be prepared as if they're going to have surgery, undergo just light sedation, and where permissible, they might actually have a small incision made to the very surface tissue of the eye, but never penetrating the eye for the very reasons that Paul alluded. It would be unethical to do that. So the trial will be masked to the extent possible, and obviously the surgeon will know, but the way that the trial is designed is that after the immediate postoperative treatment, all of the healing is done, the observations that are going to be made of the patient in terms of safety by the operating surgeon, will then be turned over to a masked physician, who won't know what the patient received, because a month out most of these patients, you won't be able to distinguish whether or not they've had surgery. So every effort will be made to make sure that patients are unaware of what treatment they have received.

Caroline Corner: Okay very good, and then the efficacy end points for this trial. Are you expecting them to be based on vision or is there some kind of imaging of the retina?

Eddy Anglade: So it will be both. The way that we would engage the FDA at the SPA level are co-primary end points, so we have the opportunity to win on either vision or change in the area of atrophy.

Caroline Corner: Okay, very good, then just quickly, 100 patients total, is it 50 control, 50 therapy?

Eddy Anglade: Exactly.

Caroline Corner: Okay, thank you. That's all the questions I have. Good luck to you guys.

Ted Tenthoff (Piper Jaffray - Analyst Information): Great, thank you very much for taking the time and for this thorough update. I guess question on the sequencing of this study and sort of when it comes to the discussions that you've had with overseas regulators, is that concordant with respect to the end points that you're looking at?

Paul Wotton: Ted, that's a good question. I'll ask Eddy to provide the answer for that. Suffice it to say, we have had very good and very recent dialogue with both FDA and EMA.

Eddy Anglade: Ted, the question is a great one, and that's precisely why we're implying a co-primary endpoint, so that we have the opportunity to win on either. Europe is pretty clear that they'd like to see a patient recognize clinical benefit, so obviously they're keenly interested in the vision. That said, when we met with MHRA they were also keenly interested in the supportive secondary endpoints. I think Europe is also willing to look at the totality of data, whether it's primary or secondary. The US I believe, based on the discussion that we've had so far and we'll pressure test it in the setting of an SPA, should be willing and will be willing to consider a win on either of the endpoints that we've described, be it vision or the area of atrophy.

Ted Tenthoff: Great, excellent. Thank you, good luck. Looking forward to some more results.

Paul Wotton: Thanks very much Ted. Appreciate that.

I think that was all the analysts on the call.

Just on behalf of everyone here at the company, we'd like to thank everyone for listening in today and I will say that I'd like to thank all of my colleagues here at Ocata for working real hard to try and expedite these clinical trials, which really are the key inflection points for our company as we move forward. We look forward to continuing to provide you with a good update on how we're doing here as we continue to move throughout 2015, which I think is going to be a key year for this company, more important than ever before. So thank you very much and we'll speak to you soon.

IpohMan69

Worth the time to watch in general. The sad thing is as she said, funding is always a big issues and cure will not be available soon enough to those needed if fund dried up and the research project have to be stopped way before any big pharma or venture capital invest in it. Similar case to OCAT in the past, thus huge dilution. Remember "valley of death" for biotech.

http://new.livestream.com/techon ... 671/videos/81739122

IpohMan69

This is what I am investing in, jaw dropping potentials.......LOL, that is, if it comes to fruition.

Oh well, bet of a life time for me! Do or die...LOL.

https://www.youtube.com/watch?v=4B8U7AfnbXk

IpohMan69

If you have been on the side line, hesitant to buy until now, you are one lucky son of the gun!!!! And I am very jealous of it!!


http://investorstemcell.com/foru ... media-etc/51470.htm

IpohMan69

The fundamental of the company has not changed, in fact progressing nicely; although PPS is eroding......

My view of the company is still the same.

Pay attention to this video embedded in International Business Times' article. Pay close attention of mentioning a safety trial for eye treatment published in North America.....


https://www.youtube.com/watch?v=hw_uOLuTiZI

http://www.ibtimes.com/are-stem- ... ye-possible-2021269

The ups and mostly downs of the PPS is part of growing pains of a young biotech company.......do your due diligence if you want to invest!!!!

IpohMan69

Moving along carefully and nicely....be patience. And DD please!

http://ir.ocata.com/press-releas ... from-nih-to-develop

http://ir.ocata.com/press-releas ... grant-from-the-nihs

http://ir.ocata.com/press-releas ... herapeutics-phase-2

Below link is the other potential pipeline that is to be partnered in the future...it is a presentation done by OCATA in this conference https://www.gtcbio.com/conferences/cell-gene-therapy-overview

https://www.dropbox.com/s/n9m0dm ... 209_9_2015.m4a?dl=0

IpohMan69

We have troops on the ground recorded and also transcribed, thanks to iCell members and other folks unselfish dedication. Below is the transcripts.

Second Cell and Gene Therapy Conference, Philadelphia, PA, September 9, 2015
Erin Kimbrel, Ph.D., Ocata Therapeutics

I'm very excited about being able to share some of our work at Ocata Therapeutics with you today.

Unlike adult stem cells, pluripotent stem cells have the ability to self-renew indefinitely, but also to differentiate into virtually any cell type of the body, many of which can be harnessed for therapeutic purposes. What you may or may not know is that the first wave of pluripotent stem cell-based therapies are actually already in clinical trials, the majority of which are ES cell-derived retinal pigment epithelium, and these are being used for ocular indications such as age-related macular degeneration and Stargardt's disease; however, other cell types are also being used in clinical trial for cardiac progenitors, pancreatic nuclear and also oligodendrocyte progenitors.

Now one of the biggest hurdles in developing pluripotent stem cell-based therapies is the risk of a pluripotent cell contaminant in your differentiated cell product so the risk of tumorigenicity is a great fear. However, the first report suggests that the clinical use of ES-derived RPE is safe, so you can mitigate the risks of tumorigenicity. So a couple of these publications show that among the first three trials, there are a total of 22 patients, they found no tumorous ectopic tissue formation or major adverse effects within one to two years after transplantation. So this is at least encouraging news that pluripotent stem cell-based therapies can potentially be safe for usage.

Now at Ocata Therapeutics, we not only have our ongoing RPE clinical trials, but we also have other ocular products in the pipeline such as photoreceptor progenitors for diseases such as retinitis pigmentosa and ganglion nerve cells. But what I'm going to talk to you about today is another cell type called HMCs, which we are developing for use in autoimmune diseases. So I will share with you a little bit of the characterization of what HMCs are, some in vitro data and then I'll go into some of our preclinical models.

So HMCs are hemangio-derived mesenchymal cells or mesenchymal stem cell-like population derived from ES cells or iPS cells. Starting with your self-renewing pluripotent stem cell population, you can undergo a brief MRA body differentiation followed by this intermediate cell type called the hemangioblast. We then use this intermediate cell type to go on and produce the HMCs, or ES-derived MSCs.

The therapeutic potential of this cell population is tied to their secretion of various bioactive factors, so in response to environmental stimuli, these cells will secrete a variety of different cytokines and growth factors. Ones that are involved in immunomodulation can affect both cells of the innate and adaptive immune system, and we really feel as though it's this property of the HMCs that give rise to their therapeutic potential. HMCs are very similar to tissue-derived MSCs and some of the properties they have. Among the factors that they secrete, they produce indolamine 2, 3-dioxygenase and prostaglandin-2 which are involved in the inhibition of T-cell proliferation and antiinflammatory properties. They also produce exosomes which are filled with a variety of different micro RNAs, RNAs and proteins that can affect cells with the immune system. Looking directly at their inhibition of different cells in the immune system in a dose-dependent manor, we see that HMCs can inhibit T-cell proliferation. They also can inhibit the maturation of dendritic cells, so in response to maturation stimuli, dendritic cells will up regulate expression to CD83. Co-culture with HMCs can reduce them. Likewise dendritic cells will secrete IL-12 p70 in response to maturation stimuli, but co-culture with the MSCs or HMCs can reduce this. These properties that I have just described are very similar to adult MSCs, as well as the expression of various cell surface markers. They are very similar to bone marrow and umbilical cord MSCs.

The MSC populations that are being used in clinical trials are all derived from adult tissue sources, the vast majority of which are bone marrow MSCs. However, as many of you may know, some of the MSC trials have lead to rather disappointing clinical trial results, and that is probably attributed to the fact that there are donor-dependent variability in the cells and also that the cells tend to lose their therapeutic potential if they have been expanded in culture for a very long time. So HMCs may be able to circumvent some of these issues that the adult MSCs have. We have some data that suggests that they may be more potent than the MSCs currently used in clinical trials and I will share some of that with you. The HMC manufacturing process is also readily scalable. By virtue of the fact that you are starting with a replenishable starting material such as embryonic stem cells, you can derive an unlimited number of early passage MSCs thereby avoiding the loss of function that you would normally see with extended in vitro culture.

So some of the molecular differences that we are finding for our HMCs is that they have faster migration than bone marrow or umbilical cord MSCs. This is an in vitro scratch assay showing that after a 6-hour time window that the HMCs will migrate into the red zone, or the empty space, faster than the umbilical cord or bone marrow MSCs. In relation, they also have higher expression of certain migration-associated proteins such as CD24 and MMP9 and this is an ongoing area of investigation. They also have a lower expression of the pro-inflammatory cytokine IL-6 as shown here by an antibody array. They have the lowest scores in comparison to umbilical cord or bone marrow. Not only do they express a lower amount of IL-6, but we also have in vivo data showing that the HMCs can actually reduce the level of disease-associated IL-6 and that's contributing to their therapeutic effects.

So now we'll go into the preclinical animal models using our HMCs, first in comparison to adult-derived MSCs and also proof of principle in other experiments. The first one I would like to share with you is the EAE model for multiple sclerosis. It's an induced model, whereby you can induce paralysis in the animals, and what you're looking at is the clinical severity scores. The higher the number here, the more severe the paralysis is. And what you can see is that in comparison to PBS controls, bone marrow MSCs had very little therapeutic activity and were not able to reduce the disease severity. Umbilical cord MSCs did have a therapeutic effect, but this effect was lost over time. However, our HMCs, either live or irradiated, were able to inhibit the disease process and lower the disease severity for the extent of the time period. And also in this EAE model, we looked at the migration of our HMCs into the inflamed CNS tissue. So you're looking at time focal images, either volume or iso surface rendered showing that GFP labeled HMCs were able to extravasate out of the vasculature and into the inflamed tissues. However, bone marrow MSCs also labeled with GFP, while they homed to the inflamed tissue, they were not able to extravasate out of the vessels and they remained trapped in the vasculature. So we think this migration phenomenon that we are observing could relate to improved therapeutic activity of HMCs.

Jumping to another autoimmune disease model, we have looked at the effects of our HMCs in lupus-prone mice. Here we are using the New Zealand black and New Zealand white F1 generation mice, which develop immune complex mediated glomerulonephropathy and they have a reduced lifespan because of this lupus nephritis effect. So as the control animals will die by around 35 weeks of age, if they are treated with either a single or two doses of HMCs, we can see they have a significant effect on the survival of these animals. They also can maintain kidney function as evidenced by blood urea nitrogen, serum creatinine and proteinuria levels. You can see that as the mice age the kidney function begins to fail; however, if the animals are treated with HMCs, we see a reduction in this disease-associated process.

If you look at the histopathology of these animals, you see that going from a pre-diseased to a diseased state, you undergo a process whereby there is hyperproliferation of mesangial cells and a loss of the Bowman space surrounding the glomeruli. When you treat the animals with the ES MSCs or HMCs, you see preservation of the kidney architecture. In addition, you do not see the protein casts that normally appear as the disease manifests itself if you treat with the HMCs. You also see a reduced number of infiltrating T-cells when you treat with the HMCs. A blinded veterinary histopathologist has scored the animals and what we can see is that in three different categories, the glomeruli, protein cast and interstitial scores, the HMCs had this therapeutic effect.

A third model that we have investigated is the EAU model for uveitis, which is an inflammatory eye disorder. You could see in the untreated animals that undergo the disease process, you see an infiltration of immune cells into the vitreous cavity and also a disruption of the retinal architecture. However, treating the animals with the HMCs you see much fewer infiltrating immune cells and preservation of the retinal architecture. Also, scored by a blinded histopathologist, there is an improvement in the histology and funduscope scores.

The last model I would like to share with you is actually a large animal model using canines. In collaboration with Tufts University Cummings School of Veterinary Medicine, we treated six dogs that had cyclosporin-resistant anal furunculosis with our HMCs, and we found that while the animals have between 1 and 6 fistulas upon presentation, after three months after the HMC injection there was an absence of fistulas in these dogs, which was a remarkable finding considering that they had basically undergone resistant cyclosporin-resistant fistula appearance for quite some time. Six months afterwards, two of the six animals did have a small recurrence of fistulas suggesting that perhaps more than one injection of the HMCs would be needed for a sustained effect, and yet the results were quite promising.

So the last part of the talk, I would like to touch upon the commercial scale of manufacturing that we have for our HMCs. The HMC name refers to this hemangioblast intermediate cell type that we have. This intermediate is highly expandable. It is also cryopreservable, so we can generate a large working cell bank with these hemangioblasts and then use them to generate several manufacturing lines, which provides greater consistency for our final HMC product.

Again, getting back to the issue of tumorigenicity from a pluripotent stem cell-derived product, QC lot release criteria are heavily focused on safety, so not only do we screen the HMCs for a variety of different viruses, mycoplasma, sterility, we also ensure they have a normal karyotype, and then we look at the expression of pluripotency-related genes in the final HMC product, and what you can see is there is a strong down regulation of pluripotency-associated genes as there is an up regulation of the MSC-associated genes. We can also detect the absence of pluripotency-associated cell surface markers trial 160 and trial 181 in the final HMC product.

Just the take-home message from the talk today, the HMCs are ES-derived mesenchymal stem cells that we are deriving through a prior observable hemangioblast intermediate. They are showing unique molecular properties, and they have a greater in vivo potency, at least in the EAE model, than tissue-derived MSCs. I have shared with you some of the evidence in four different preclinical models of autoimmune disease, that they have therapeutic potential. Our platform is scalable for commercial manufacturing in the capacity that we don't have to expand them very much in order to do this, so we can maintain the potency. The QC lot release criteria are heavily focused on safety and ensuring lack of tumorigenicity in the final product, and what I didn't touch upon, but which is important, obviously intellectual property surrounding the composition of matter and method of manufacturing for this product.

Lastly, I would just like to thank members of the Ocata research team, as well as our collaborators at Tufts University and UCLA. Thank you.

Q: What was the source of the human embryonic stem cells?

Erin: We used our FDA-approved MA09 embryonic stem cell line, which is derived from a single blastomere technology. It's a technology that unlike most technologies that derive the cells from the inner cell mass, single blastomere technology isolates cells from an 8-cell stage so you are not essentially destroying the embryo in the process.

Q: There's nothing to do with Geron?

Erin: No.

Q: I wonder if testing does compare it to MSCs, the effectiveness or efficacy or are you now just looking at your own product?

Erin: We made a lot of the comparisons in the EAE model, but currently we're looking at the lupus nephritis models and doing direct head to heads with umbilical cord MSCs.

IpohMan69

This company, as stated before, got bought out by a big company. But sadly, the pps is very low compared to what we were aiming for.

http://www.astellas.us/therapeutic/rnd/ocata.aspx

Astellas, is a big medical company headquartered in Japan with a market cap of 30 billions dollar, paid cheap to acquired Ocata, when the pps being ambushed by shorts. The company was offering a tender offer of $8.5 per share to shareholders, with about $380 millions total buy out cost for Astellas. And it took them 3 tries, with 2 extension of the offering deadline to be able to acquire more than 50% and declare to have power to force remaining percentage to tender unwillingly!!!!

At the time of the tender offer, it kept saying that the premium is 79% of the previous day close of about $4.46, Nov 9, 2015. On Nov 10, the news came out and the price jumped to around $8.45. From the surface of it, it seems good deal, but for many of us who knows the potential of the company, this is penny to a dollar sales.

I made out fine as I have kept buying all along to lower my average cost and lower it to below $7 per shares, thus I made some very small profit, compared to what I was expecting from it. I am not happy with it but this is investing, we retail shareholders really have no control, thus that is why doing due diligence and invest with what you can afford to lose is the key!!! I have moved on.

This will be the final post and good luck to all of you folks and thanks for reading all along.