September is Sickle Cell Awareness Month, a time when the medical community will gather with the patient population in the hopes of advancing research in this significant unmet medical need.
One company, ADVENTRX Pharmaceuticals [NYSE: ANX], is attempting to engage the investor community and the sector’s most promising drug developers through the First Annual Sickle Cell Disease Therapeutics Conference, taking place September 19th, 2012 in New York City.
The conference is meant to be a forum to discuss the most important advancements and the future trends for patient management and finding a cure. Presentations from leading companies in the space will look at some of the most promising technologies being developed. The one day event also features key opinion leaders discussing the patient experience, segmenting the market and the therapeutic value of hydroxyurea (and marketed as Droxia), the only drug approved to treat sickle cell disease.
Hydroxyurea reduces the severity of sickle cell disease by stimulating production of HbF, which can block the sickling action of red blood cells. Hydroxyurea is recommended as frontline therapy to with moderate-to-severe recurrent pain (occurring three or more times a year), however it is not a cure-all. It is not approved for children, and a large percentage of the patient population do not respond to it. Further, there have been concerns that long-term use of hydroxyurea may increase the risk of developing leukemia.
ADVENTRX, based in San Diego, is in clinical phase with its lead candidate, ANX-188, a rheologic, antithrombotic and cytoprotective agent that improves microvascular blood flow. The company is set to enter Phase III trials exploring ANX-188 as a treatment for sickle cell disease.
Sickle cell disease is an autosomal recessive genetic blood disorder with overdominance, characterized by red blood cells that assume an abnormal, rigid, sickle shape. Sickling decreases cells flexibility and results in a risk of various complications, in which life expectancy is drastically lowered. According to the CDC, sickle cell disease affects between 90,000 and 100,000 Americans, and millions around the world. The disease is most commonly affects populations with sub-Saharan African ancestry; Spanish-speaking regions in the Western Hemisphere; and the Middle East and Mediterranean. It is estimated sickle cell disease occurs in 1 out of every 500 Black or African-American births, and the sickle cell trait is present in 1 out of every 12 Blacks or African Americans.
OneMedRadio spoke with ADVENTRX CEO Brian Culley about this under-researched area, and the importance of the Sickle Cell Disease Therapeutics Conference on September 19th. Click below for audio.
To learn more about the conference, visit www.scdconference.com.
Matt Margolis: Greetings from OneMed Radio, I’m Matt Margolis. Today, I’m with Brian Culley, CEO of ADVENTRX, a San Diego-based biopharmaceutical company trading on the NYSE under the symbol ANX. The company’s lead candidate ANX-188 is a drug that improves blood flow and is being tested in sickle cell disease. ADVENTRX is preparing for further phase 3 for ANX-188. Now ADVENTRX has also organized and produced the 1st Annual Sickle Cell Disease Therapeutics Conference, taking place at the Four Seasons Hotel in New York City on September 19th. So thank you for joining us, Brian.
Brian Culley: It’s my pleasure, Matt. Happy to be here. Thanks.
MM: So firstly, let’s start with the 1st Annual Sickle Cell Disease Therapeutics Conference. How did you get involved with the event?
BC: Well it’s pretty simple. Whenever you’re working in a disease that doesn’t have a large number of patients, you really got to be a little creative with how you bring awareness to your particular area of interest. So at one time we were thinking of doing an R&D day, but we realized that an R&D day would be better if we brought together all of the companies that are working in sickle cell disease. We thought we’d attract a lot more investors and potential partners so we abandoned the idea of doing and R&D day and turned it into the 1st Annual Sickle Cell Disease Therapeutics Conference.
MM: Great. So maybe you can give us a history of how the event was conceived. Why is there a need to for this type of gathering right now in 2012?
BC: Well there are a number of answers to that question. This September is Sickle Cell Disease Awareness Month, and so this is a good time to be kicking off a new event like this. But it’s also amazing that a disease that we’ve known about for more than a hundred years has never had a conference dedicated to drug development. Just about every other disease out there has a tremendous amount of activity and interest in finding new therapies and treatment. Sickle cell disease hasn’t enjoyed the same level playing field and so we’ve pulled this together. I called a bunch of CEOs frankly from my competitors and I said, ‘why don’t we get together and show the world that there’s a pretty important investment opportunity here?’ And slowly but surely, I think we’re going to be able to attract additional investment, capital into not only ADVENTRX but also other companies that working on important new therapies for sickle cell patients.
MM: What is the clinical progress would you say of this particular area? Do you feel that it’s an under researched area?
BC: It’s been incredibly under researched. There has been only one approved drug to date that’s made it through the FDA process for sickle cell disease. Partly that’s a result of not having enough money invested in companies that work in this area and partly that it can be a difficult area. Sickle cell disease just requires some more love and attention from companies like ours, but I do think things are changing. There have been a lot of companies in the last 12 months that have raised altogether north of $100MM dollars in operating capital and if you consider contingent costs from partnerships with large pharmaceutical companies, it’s actually north of [$500MM]. So I think the landscape is changing. I’ve been predicting that in the next five to ten years we’re going to see some new developments, we’re going to see some new drugs approved in sickle cell disease because the money is starting to finally flow into this area.
MM: So let’s get a little background about sickle disease. Who is the patient population globally and what’s the prevalence?
BC: Sickle cell disease primarily affects African-Americans, however it also affects Hispanics, and many people of Mediterranean origin, Greek. The mutation, which actually gives rise to sickle cell disease independently arose on the earth in three distinct places. It arose in the Indian subcontinent, it arose in the Middle East, and it arose in Africa. Of course it made its way over to North and South America via the slave trade.
The different mutations, which exist, all give rise to the same basic disease state. You can be either a carrier of the disease or you can have called fully blown disease. If you’re a carrier, in many cases you only have a very low level of awareness and sometimes you’re completely asymptomatic, you don’t even know that you have this, that you’re carrying what’s called a faulty gene. But if both copies of your gene, if you’ve inherited from your mother and father, from each of them a faulty copy of this gene, it creates a very horrible disease.
The red blood cells in your body become curved and when the number of red blood cells which are curved increases too high, they actually block and occlude the flow of blood and oxygen fails to get to tissues and it’s incredibly painful. It’s often described as being hit with a baseball bat repeatedly in the same place over and over and over. It’s called a vasoocclusive crisis and these crises will often send patients to the hospital where they receive very high doses of pain medication. It’s quite horrible because these patients deal with very high levels of pain, sometimes these very acute levels or hospital requiring levels of pain, but generally they do suffer from pain on a very frequent basis so it makes day-to-day living very challenging. So there’s a huge need, the patient demand for something new to help treat this difficult disease is extremely high.
MM: How is the medical community and the patient population rallying around this conference?
BC: The response has been tremendous. I mean I was apprehensive about which size room to reserve for the event and I’m glad I went with the larger room because we’ve just sold out this week. There have been a lot of things happening, which are very positive and encouraging for sickle cell disease. We have seen some new initiatives at the federal level. There has been a change in the rules and regulations and laws surrounding how uncommon diseases or rare diseases are able to navigate the approval process with FDA. We have seen large pharmaceutical companies like Pfizer and GSK creating rare disease, divisions and departments.
When I’ve gone out and when I was stress testing or field testing the concept of having a sickle cell disease conference for investors and for large pharmaceutical companies, the response is very positive. People said, yeah, that’s great. So we’ve been really thrilled. I think that there has been a ground swell of interest in new disease because quite frankly, some of the lower hanging fruit has really been picked off already by the major multinational pharmaceutical companies and there’s been an evolution towards some of these diseases which fundamentally we have the tools to treat these diseases. We know that we’ve got the new drugs and all the different techniques. They’re out there, they just haven’t been really applied in a committed way the way that they’ve been applied in some other areas. Cancer comes to mind, tremendous progress in cancer. If you had a fraction of that investment going into sickle cell disease, we’d have tremendous progress in sickle cell disease too. So I think we’re seeing that. People are aware there’s nothing magical about sickle cell disease, it just hasn’t had its turn. I believe we’re at the beginning of that now.
MM: Of course it’s very intriguing. Let’s turn to your lead candidate ANX-188. So can you talk about the technology here? Why does this have the potential to be a treatment of choice?
BC: Well what 188 does in the simplest terms possible is it improves blood flow. So I described a minute ago that patients with sickle cell disease will have these crises where their sickled cells, which are sticky and not very deformable, will all bind together and like logs in the river will clog the flow of blood and prevent the delivery oxygen. What 188 does is it binds to those sickled cells and it binds to the lining of those tiny capillaries in your circulatory system and it has the ability to help restore the flow of the blood. If you’ve got blood flowing, you’re able to deliver oxygen and you should be able to reduce the duration of time that patients are in pain.
So whereas previously we’ve seen patients who can have one of these painful crises that last for they can be two days, they can be five days, they can be ten days or even longer, we’re looking to use our, sometimes we joke about it as like a WD-40 for your circulatory system. But we’ll use our agent to help blood flow so that people can get the normal circulatory activity restored and they can get out of this crisis and they can get out of the hospital and go back to their normal lives. I’m pretty excited about the data that we’ve seen for it.
MM: So what is your clinical timeline and what data have you been talking about this year?
BC: Well we’re gearing up now for a phase 3 trial and typically drug development involves phase 1, 2, and 3 so we’ll be going into the last phase of clinical development. We’ll be conducting a very large study. It will be between 350 and 400 patients will be enrolled in this study and it’s going to be 30 or 40 centers across the US and we may also go outside of the US. There is some interesting markets in Western Europe and Brazil. That study will be gearing up, we’re targeting to initiate it before the end of the year and we’re hopeful the data that comes out of that will be sufficient to support a new approval.
MM: What do you think are the biggest challenges facing sickle cell drug developers? How can you overcome them?
BC: Sickle cell disease is very heterogeneous. Whereas one patient might have an acute crisis that lasts for two days and that crisis might only happen once a year, you can have another patient who has a crisis every month and their crisis could last for seven days. So if you’re looking to have an impact on crisis, we’ve got such a breadth of experience with the disease, it can be very difficult sometimes to control the experiments so that you’re able to see the effect that your drug has. That’s just something that we as a drug development company deal with. You can address this with large numbers of patients in your study and you try to give the physicians some guidelines on best practices so that everyone is treating their patients essentially in the same way and hopefully at the end of the day, your drug’s effect is able to be observed among the background noise that’s inherent in a diseases like sickle cell disease.
MM: What programming is a must-see and also how can our readers and listeners register and learn more about the conference?
BC: Well the conference started out as just being a panel of clinical stage companies, a bunch of CEOs like myself talking for 20 or 25 minutes about our drug programs, but we very quickly expanded it. Before lunch, we’ve got several presentations. We’ve got a presentation from a representative of the CDC who’s going to be coming in and talking about sickle cell disease. I’ve got three key opinion leaders, these are medical experts who are very well known in their field talking about topics such as the patient experience, talking about that one approved drug, why is it now used as widely as it could be or should be, how can that be changed. I’ve got someone talking about the differences in the disease between adults and children. And then I’ve got three academic clinicians who are looking at drugs that have already been approved and testing those in sickle disease and then there are six companies like ours. So the day is pretty full but from start to finish, I think it’s going to be pretty interesting.
For those people who are interested, the morning sessions are largely about sickle cell disease, cutting edge topics and there will be question and answer sessions that will be moderated by healthcare analysts. Then in the afternoon, I sort of call it the afternoon of hope because we’re going to see a lot of presentations from companies that are working on new treatments and therapies and again this has never been done before to my knowledge so if you’re interested in drug development in sickle disease, this is a must attend event. If people are interested, they can attend. They can just go to SCDConference.com, that’s sierra, Charlie, delta for sickle cell disease, SCDConference.com and register and I know we’re at capacity but we’ll do our best to make sure people can attend.
MM: That was Brian Culley, CEO of ADVENTRX discussing the significant unmet medical need in sickle cell disease as well as the 1st Annual Sickle Cell Disease Therapeutics Conference, taking place at the Four Seasons Hotel in New York City on September 19th. This is OneMed Radio signing off.
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