Farmas USA

Ya se empieza a caldear, dadle su tiempo que le cuesta! Eso sí, se va dejando gaps por el camino...
Los 1,75$ sí serán una buena prueba de fuego de si esto va en serio, o volvemos a la AMRN de estos últimos meses.

500k cromos en dos velas de 5 minutos.

bueno, no esta mal ... ahi las dejo  a ver si de verdad se animan y vemos los 2$ en unos dias.

Esos 1,75, como dices, son el punto de corte

AMRN

Está la bicha como para ponerle un stoploss...te salta antes de dar la orden.Vaya gap!

NVAX

OCAT

Aquí tienes la conferencia; todo buenas noticias. No hay fecha para los datos del Q4, pero será algún días de estos.

Cowen and Company 35th Annual Health Care Conference, Boston, MA, March 3, 2015
Transcript By, Patti

Paul Wotton: Thanks Jeff. Thanks to Cowen for inviting us to speak at this conference. It's a real pleasure to be here. On to the presentation, the first slide we come to is just a cautionary statement, so I'll whizz through that.

What we're trying to do, as Jeff mentioned just now, we're a company that's focused on regenerative ophthalmology. Our lead programs are addressing macular degeneration with what we think is ground-breaking retinal pigmented epithelial cell therapy. We had a busy second half of the year last year. We published data in the Lancet in October, which showed for the first time in any disease the long-term safety of a cell therapy transplant in humans. We're just about to initiate our advanced studies in Stargardt's macular degeneration, which in actual fact will be a Pivotal Study as a result of meetings we've had with the European Medicine Agency recently, and secondly dry AMD where we're going into Phase II studies to assess whether or not we need any immune suppression in that patient population. We don't think we do.

We have an extensive proprietary position in major markets in protecting the entire ___ chain of the cell therapy we're working on. One of the benefits of this company is that it's been able to benefit from a lot of work that's been done in the cell therapy area over the last 10 or 15 years as a result of which we have numerous patents that cover our technology and programs.

The ophthalmology market itself is a large growing and actually underserved market, particularly in the AMD market. Age-related macular degeneration affects actually now almost 2 million new patients per year, and there is no treatment available for dry AMD. There is very effective treatment for wet AMD, but the segment we're going after is dry AMD.

The second program which is Stargardt's, is a form of inherited onset of macular degeneration that affects juveniles. It's an Orphan disease indication and right now there is no available treatment for this either. There is a third disease we're going after which is myopic macular degeneration, or MMD, which is particularly prevalent in Asian affecting up to 13 million people in China and Japan. The common link with all of these diseases is that every single one of them results from loss of the RPE layer at the back of the eye, and what we're doing to fix this is putting a small cell transplant in the back of the eye of RPE cells which we make.

So the Lancet article was important. It was the first time, as I mentioned just now, that long-term safety had been seen following transplant of a cell therapy, and we also saw an efficacy signal which was icing on the cake really for the company, and what we showed the first time, and in fact we have almost four years data on some patients, the patients could take this therapy and years later the cell transplant is still in place and you can find it.

As a company, we're a world leader in the terminal differentiation of pluripotent to target cells, so our technology is being able to take a pluripotent stem cell and turn it into a fully differentiated cell that can then be used for transplants. In the case of the Stargardt's and dry AMD work for example, we are manufacturing retinal pigment epithelium cells, which are really an off-the-shelf solution to being able to transplant these cells to the back of the eye. We've also made corneal, retinal ganglion, and photoreceptor cells, also terminally differentiated, and just recently, and this work will be published soon, we did show that we can manufacture photoreceptor cells, progenitor cells in fact, give those to rodents and we found that they actually recovered their vision when previously hadn't been able to see anything.

The stem cells are our starting material, but it's fully differentiated cells, the treatment that we give patients, and this is an important difference.

So we actually as a company are able to make the spectrum of cells that are required to treat ocular disorders and this technology platform we have is able to also do other interesting things like screening small molecules for activity in some of these cells, but our goal for the company is to own the eye with the technology platform we have, and I think as a company, our focus on regenerative ophthalmology is one that is quite unique, but we're able to do it. As a result of which we have a pipeline that is quite comprehensive. We have, as I mentioned, programs in Stargardt's, dry AMD and MMD. I'll talk about these individually in a minute, but we have multiple shots on goal here. The Stargardt's program is about to enter Pivotal studies. The dry AMD program is about to enter Phase II where we're assessing whether or not we need any immune suppression in those patients, and myopic macular degeneration is a program that's ready to go into Phase I. Behind that, we have an opportunity for additional programs, photoreceptor progenitor work, ganglion cells and corneal work which we're doing. Our potential European launch for Stargardt's, we believe, could be as early as 2019.

Why the eye? So the eye is well suited for cellular transplants. It's an immune privileged organ, which means that it's less likely you'll see any rejection, and in fact in the patients who have transplant for up to nearly four years now in some cases, we haven't seen any evidence of transplant rejection, and these patients have been off immune suppression four years as well, so obviously the cells are well tolerated at the back of the eye. It's a compact structure as well, which means you do not need to give millions of cells in order for the therapy to work. We're just giving a couple hundred thousand cells to the back of the eye. It requires a dose of about 150 microliters in volume, which is well tolerated as a subretinal injection. And we're using straightforward delivery using currently available technology. These subretinal injections are also used in some of the gene therapy programs, and above all, and this is important when you're pioneering with a new therapy, the clinical outcome assessment tools we are using are all validated with the agency. The other thing about the ophthalmology market, which I think is chief for an emerging biotechnology company, the market is readily accessible with a relatively small sales force. It's a well-defined prescriber base. There are about 1500 to 2000 vitreoretinal surgeons in this country who perform the type of work that we do, who can diagnose and manage the subsequent patient care. This means that you need a sales force in the sort of 20 to 30 range. It's easily manageable. The product itself is easy to administer, and as I mentioned just now, the cellular transplantation is already performed with current technology. There are about 300,000 vitrectomies performed every year in this country. The small dosage requirement and the fact that it's off-the-shelf means that it's scalable in terms of manufacturing and we're able to distribute it quite readily. And lastly, and this is the most important thing, there are patients out there who have nothing to treat either dry AMD or Stargardt's today. So there's real patient need for this type of therapy. We believe in the case of dry AMD it's an opportunity to treat the disease before they go to wet AMD where there are already some successful drugs on the market, but with Stargardt's there's nothing, and Stargardt's patients begin to go blind quite early on, and our mission in life is really to make life better for them in helping them to recover what they might otherwise lose.

If you look at the epidemiology of Stargardt's, it's an orphan disease indication. There's around 32,000 patients in the US today who have Stargardt's disease. The segment which we are targeting as our primary target population is in fact the 9,000 to 10,000 patients who have early vision loss with Stargardt's. This means that we're going to get these patients and be able to preserve their vision and possibly improve it at an early age. By the age of 45 years old, most patients with Stargardt's already are legally blind.

With dry AMD, this is a much larger market opportunity. There's about 2 million patients each year diagnosed in the US with dry AMD and about 10% have what's known as advanced dry AMD, or severe. This is where they lose their central vision, and of those, there's about 165,000 patients we believe that can benefit from the RPE therapy that we are actually developing.

Going back to clinical programs and moving on to this slide, which actually talks about the RPE layer. When you actually look at the biology of what happens at the back of the eye, it's logical that an RPE transplant should work. We're not asking these cells to do anything complicated other than to reside in the back of the eye and just function as a normal RPE cell, which is to nurse the photoreceptors above them, and then also phagocytose some of the debris that might be at the back of the eye as well. So these are responsible for basically maintaining the health of the back of the eye. You don't need to wire them up to any neuronal circuits. They're required for vision. Without an effective RPE layer, your photoreceptors will eventually go out and you will go blind. The RPE layer itself delivers and metabolizes Vitamin A, which is important to vision. It also transports waste away from the retina, and it secretes most importantly growth and survival factors that are needed for photoreceptor differentiation. So what we're doing is we're putting an active nursing cell into the back of the eye that maintains the health of the rest of the eye.

This is a picture of what it looks like if you actually do have central vision loss. In the case of Stargardt's disease, this begins to occur when you're typically a teenager. In the case of dry AMD, it occurs usually into the 50s and 60s, but it's actually being diagnosed sooner than it used to be, and there is an epidemic of dry AMD now in this country.

Let's talk about biology here for a second. So what we can see from this slide is the rods and the cones are intimately linked with the pigment epithelium layer at the back of the eye. Those pigment epithelium cells nurse the rods and the cones. When you have macular degeneration, where your RPE layer disappears, those photoreceptors literally shrivel up and die. So our approach is to give a subretinal injection of pigment epithelial cells and then these cells, once they're injected into the back of the eye as a bleb, can then implant. They do take up residence. They are polarized. They find their own seat at the back of the eye and begin to regenerate some of the dormant photoreceptors at the back of the eye, which restores some of the visual capability.

What we showed in the Lancet when we published this in October of last year, we did see evidence of engraftment of these cells at the back of the eye, and this is important. It's easier to see in the room here, but the micrographs of the eye you can see here that there is almost no RPE layer here, but when you actually do the transplant, you can see that it's taken up and you've got a clear transplantation engraftment there of RPE cells, and we continue to see this during the time the patients have been on these cell therapies. So we've seen successful engraftment now in 13 out of 18 subjects that were reported in the Lancet. I'd just like to point out we now have actually dosed nearly 40 patients across the world with this therapy and to date we haven't seen any evidence of transplant rejection. So we've actually demonstrated the safety of this approach. I think the other thing that's important here is that the feedback that we received from both the FDA, the MHRA and now the EMA has shown us that the work we're doing is quite pioneering and they are very supportive of this company being able to get this product developed and approved.

So let's just look at the data on the Stargardt's, where we had patients with follow up for up to 24 months. You can see here that after two years of transplantation of four subjects who had the transplant in place for 24 months, they've actually maintained a difference between the treated eye and the fellow eye, which was used as a control. So in these early Phase I/II studies, these were safety studies that were designed to test whether patients would be able to take a transplant. The types of patients we looked at here were patients with really, really poor vision. If you're talking on a scale of 0 to 100 in terms of the letters you can see, these were 5's or 6's, which is basically legally blind. All you're looking for here was successful uptake of the transplant and what happened in fact was in some of the patients they reported improved visual signal, and what we saw here was that after two years, the transplant still gives you better vision in the treated eye than the control eye.

Let's talk about what we've got in terms of the technology. As a company, we've invested very heavily in the manufacturing process that we have at Ocata. We have spent a lot of time and money on the GMP suite that is FDA reviewed. We also have a pilot facility at Ocata and we have been able to create cell lines. That means that we basically have an inexhaustible supply of cells in order to manufacture the RPE cells. We have been able to expand those cells. We shift those to terminal differentiation. We have 12 full-time employees. In fact, we just hired a head of manufacturing. He came to us via Genzyme. And then we purify these RPE cells and we ship them to the study sites where they're actually implanted into the patients.

So I just think that one of the important pieces here is that we have a good intellectual property base for the company. We actually cover all of the IP from the stem cell line itself to the patient's treatment. We have patents covering the derivation of the cells, the manufacturing of the cells. One thing that's important is the product release assays. We actually have IP around this, because it's important to know that the final cell you've made is going to stay at the final cell before it goes into the patient. And we have a number of patents around that, because that is a key piece of the technology. We also have technology around the pharmaceutical preparations for these cells too, because keeping these cells in an optimal state of health in order to be able to deliver them is obviously important. We have IP around that. And lastly, we have a number of patent families around the method of treatment for these products.

We've also got a management team that we've assembled which I think is key for us to move forward successfully. Myself, I've been with a number of drug companies. I started out my career at Merck. I have primarily been involved in NASDAQ listed companies, public companies. Ted Myles is the CEO sitting right here, has also been involved in a number of successful companies in the past. Bob Lanza is actually the Scientific Officer. He was named one of Time Magazine's 100 Most Influential People last year. He's a pioneer in the cell therapy space. He's extremely well known as an individual. He also has honorary positions at Wake Forest right now, School of Medicine. Eddy Anglade is our Chief Medical Officer. He is a well experienced, not just transplant guy, but also an ophthalmologist by training, so he's really the ideal guy to lead our clinical program, which involves both types of technology. And more recently we have LeRoux Jooste join us who, amongst other things, his career at Lilly and Wyeth launched both Prozac and Enbrel as products, who has joined us as the Chief Commercial Officer and has a lot of experience, particularly from his time when he worked with Frank Valdeno at Cephalon at Business Development. So LeRoux's job is to help us build this business.

In terms of the corporate and scientific board that we have assembled, we've got a number of well-known names here on the board, including Bob Langer, Greg Perry, Mike Heffernan. We also have world class Scientific Advisory Board that we've actually put in place more recently. In fact we had a meeting here in Cambridge just a couple of weeks ago. It's chaired by Bob Langer, with people like Connie Cepko, George Daley, John Gearhart and Mike Longaker and Joe Vacanti. We have leaders from institutes like Harvard, Stanford and Massachusetts General Hospital. We have leaders in this area to help us pioneer this therapy.

I myself was also at a meeting down in Miami recently where we had the Key Opinion Leaders on the clinical side working with us on this program, and again, it's the who's who of ophthalmology that are working with us in pioneering this work, people from Bascom Palmer, Jules Stein out at UCLA, Mass Eye and Ear here. So we're trying to affiliate ourselves with the best and the brightest in the world when it comes to getting these therapies approved.

Lastly, over the next 12 to 24 months, I think what you're going to see is a number of key milestones hit. We just uplisted the company to NASDAQ, which was an important event for us. It was a key deliverable for us when I came into the company. We also had an opportunity to work through the clinical trial protocols more rigorously as a result of meetings with various regulatory bodies in the past six months as well. We actually have completed the dosing of our Phase I/II studies. We now know what dose we can go to in the Pivotal Stargardt's trial and the Phase II trial on the dry AMD. So we'll be planning on working on our Stargardt's Pivotal trial with the first patient treated some time in the next month or two. One of the things that we've changed actually, which everyone should recognize, is we're actually taken the advice of the FDA and the European Medicine Agency and we're moving away from using the fellow eye as a control in these studies, to actually moving towards control patients where they're actually receiving no cell therapy, so this is going to be, if you will, a placebo controlled study for both Stargardt's and for dry AMD. The Stargardt's study will be a Pivotal study. It's going to involve 100 patients, 50 of whom will be dosed, 50 of whom will be control patients and it's powered to provide us with the outcomes we're looking for. And in fact, the end points we're looking at are either anatomy or visual signal improvement, they are either/or end points, and the agencies have been very, very helpful indeed in helping us to identify those.

The other thing we're working on is making sure we select the right patients for these trials as well, because that is vitally important when you're actually designing clinical trials in terms of getting the best possible outcome.

With the dry AMD study, we will be treating up to about 60 patients now with dry AMD with three dosing levels with immune suppression up to 12 weeks. Obviously what we're trying to do here is minimize the amount of immune suppression we need. It's something that would actually expand the available patient population, which I think is important and commercially would actually be really helpful, but most importantly, if you can minimize immune suppression you need in a patient, then that's obviously beneficial for everybody.

So our first study in this treatment of AMD patients, dry AMD patients, should give us a good readout this time next year and that will be a big inflection point for the company. It will be a controlled study with patient control group in dry AMD, and I think this is going to be key to this company moving forward to have a successful outcome there. If we just repeat the data we saw in the Lancet that we had in October with a control study group, then that's going to be a very important inflection point, not just for this company, but for the cell therapy space in general.

We have additional readouts in the other cohorts in the dry AMD study coming through in 2016 as well. Then in the middle of 2016, we'll start to get interim reads, really just for the statistical side of things in our Stargardt's Pivotal Program, as well as completion of our AMD program.

So to summarize, where we are, is we're initiating the pivotal trial for Stargardt's macular degeneration and we have a Phase II trial starting for dry AMD with what I believe to be a novel and potentially curative therapy in areas where there are no approved products today. These are markets where there is a significant unmet need for patients. The Stargardt's is an orphan indication. We're very committed to getting the Stargardt's product to market as quickly as possible, both in Europe as well as here in the United States. We have dry AMD as a potential blockbuster indication. It's a precursor to wet where there's at least some successful treatments out there. We've shown that we can achieve safety in patients with a cell therapy transplant and that is key. I don't think anyone can overlook the importance of this finding that we published in the Lancet in October. And lastly, we have an established network now of key opinion leaders in ophthalmology who really believe in what we're doing. There are a number of very sort of well known sites who are actively seeking to become part of these studies, and it's not surprising because it is a world leading therapy, but on the other hand, I'm pleased to see that we're being associated with the very best now in terms of moving these programs forward.

We also have a good IP position. This company was actually one of the pioneers in cell therapy and I've been lucky to move into the company as the CEO at a time when I think it's just about ready to break out. Ted is here in the audience. He's been responsible for re-engineering the company last year, and we're now in a position where we can grow from the platform we've now created. The pipeline itself is world leading. I believe that it offers us the opportunity to grow the company and get a much closer focus on the ophthalmology area.

For the programs which are non-core to the company, we've got a lot of work done in lupus, multiple sclerosis and Crohn's disease for example. We have a lot of preclinical data there and we'll be seeking to find partners for those programs in the future.

And lastly, I think that it's all about the people. We have a very experienced management team coming from manufacture-based companies. We have a scientific leadership who now can develop and deliver products in this therapy area. We have very strong corporate and scientific boards, and I think that we've built ourselves a network of individuals as well that can help us get these important therapies to market and help the patients who need them most.

Thank you.

Fuente: http://investorstemcell.com/forum/ocata-main-forum-general-topics-science-prs-media-etc/49901-7.htm

DVAX 20.41 +0.69 (3.50%) Real-time: 10:40AM EST NASDAQ real-time data - Disclaimer Currency in USD Range 19.53 - 20.39 52 week 12.50 - 20.90 Open 19.70 Vol / Avg. 121,499.00/344,247.00 Mkt cap 532.97M P/E - Div/yield EPS -3.5 Shares 26.29M Beta 2.44 Inst. own 79% mugiWara como que no la miras mucho, aunque llama la atención que tras la dilucion y al contrario de lo habitual no para de subir desde 16, cada día marca un nuevo máximo , lo mejor es que sin S.Alpha pump , con poco ruido y bajo volumen todo un clásico de acumulación y a pesar de los pesares de sin catalizadores relevantes cercanos, Sigo pensando que sera la estrella de la Hepatitis B en los próximos años por lo en cualquier momento nos podrá sorprender un fuerte rumor de B.O

3 SOLDADOS BLANCOS

DVAX

Como que no la miro mucho? Si es la niñita de mis ojos! La verdad es que va viento en popa, miedo me dan los resultados de Q4 para el día 9 y que nos frene en seco. Toda la razón en eso que comentas, ruido ninguno, 0 relevancia mediática, como si no existiera. Y como dices, no me extrañaría nada que en algún momento empezaran los rumores para posible BO...

Yo esa me la compre en 1,8 alla por 2013 pero no la tuve paciencia y tras el reverse split la perdi de vista ya ...

DVAX

 

Ya hace más de un año que la tengo ahí, durante muchos meses un poco muerta... Pero bueno, hasta el día de hoy como inversión en $ no está mal, que la compré con el USD/EUR a 1,38$. Ahora a esperar a Q4 para gloria o batacazo. Hasta que Zoka no diga de vender ahí seguiremos! Además le tiene un precio objetivo de 70$, ahí es ná!

DVAX