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«New York Forum Part 1 September 20, 2014 Page 1 of 30 Speakers: Virginia Klimek, MD Boglarka Gyurkocza, MD Simon Yeung, PharmD, MBA, Lac Jayshree ...»

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Anybody that treats this disease knows the importance of encouraging people to at least consider enrolling in… receiving treatment in a clinical study or giving blood or bone marrow samples for research because we have so much to learn about this disease and we really want to develop and need to develop better treatments.

One of the last things I wanted to do was touch on this question of iron chelation. Do people know…? Does this term sound familiar to a lot of people in the room? Do you know what iron chelation is? So, when people are treated with supportive care with transfusions, you can develop something we call iron overload in your body. The iron can be deposited in your bone marrow, in your spleen, in your liver, in your heart and in some of your other organs like your thyroid, your glands and your pancreas. So, there’s a lot of concern about what that means to your overall health that there’s extra iron in your body and there’s been more interest recently in giving drugs to try to get rid of this iron because of concerns… because of these health concerns from the iron and in part that interest is stimulated by the development of some new drugs that can be used to actually get rid of that iron in your body. So, people read about it, there’s a lot of interest and a lot of… always a lot of questions about this.

This is a little bit of a busy slide, but I just want to draw your attention to what I put in red. These are two clinical trials for a drug called Deferasirox. It’s also known as Exjade which is an oral chelating agent that’s available for treating iron overload. What I wanted to draw your attention to is the fact that in these two clinical studies although the ferritin level, which is a measure of iron, although the ferritin level does go down, it’s fairly modest by only about a quarter here and maybe about a third here, but importantly a lot of people have to stop taking this drug because of side effects. It’s not an easy drug to take. Getting back to that slide I showed you with the scales where is there more risk than benefit. So, we’re using these drugs very judiciously, very carefully in people with MDS because know it can cause more side effects than benefit in many people and also because there has not yet been a study that’s done in MDS to show that using these iron chelation drugs changes your survival or your outcomes with your disease. It may bring down your ferritin level, but it has yet to be proven that it’s going to change things for you in the long run. So, we have to be careful and one of the most concerning side effects that we have with this disease is problems with kidney function as well as problems with your intestines – diarrhea and so. So, it is not insignificant.

This is an ongoing clinical study. I don’t know if anybody in this room is in this study or they heard about it, but this is an ongoing study comparing one of these iron chelation drugs to

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placebo because that’s the only way that we can tell if these drugs are affecting the disease and the survival of people who have MDS. So, this is a study that’s ongoing and hopefully we’ll be able to get enough people on this study so that five years from now we’ll be able to say whether or not this drug is worth it to give to people on a regular basis.

And then so the last thing I wanted to go over quickly is about what’s new in MDS. As I mentioned, clinical trials are always ongoing. We’re looking at drug chemotherapy combinations. We’re always looking for new drugs. Shortly, we’ll hear more about new transplant… stem cell transplant techniques that might make transplant available to more people and also we may hear also about things that you can do even after transplant to help prevent the MDS from coming back.

Some of the drug combinations that we’re using are the Vidaza and the Decitabine or the 5Azacitidine and Decitabine in combination with other new drugs or some of these newer drugs by themselves to see if some of these drugs will work by themselves and some of them have kind of funny names like Hedgehog inhibitors, MAP Kinase inhibitors. So, there’s strange names, but these are drugs that don’t really have a company name assigned to them yet. So, they have all these funny names and numbers when they’re first being tested, but suffice to say that clinical trials, I think, can and should be considered as part of the treatment option menu, if you will.

A very exciting and I think will be ultimately very fruitful line of research is to look at the genetics of people who have Myelodysplastic Syndromes to look at things like the chromosome changes and very specific changes called mutations, very tiny changes in your DNA that can have a big impact on how your bone marrow works and the last time I hosted this MDS forum, this paper was new. It had just come out and this was a paper that showed that most patients with MDS when we really drill down and we looked at these very, very subtle changes in the DNA, most people with MDS have these changes and we’re now starting to learn about what those changes mean and even more exciting we’re now in partnership with pharmaceutical companies are now starting to develop clinical studies to try to test new drugs that will target those abnormalities. So, it’s not just that we’re finding things. Now, we’re finding things that we can actually drugs to target, so I find that very exciting. We have a number of studies here and there may be studies at the center where you go to where we can use these new drugs to target these genetic changes. Importantly what we’re also learning is not just how to treat it, but we’re learning more about why people… maybe why people actually develop it and, again, I have a specific interest in therapy related MDS and leukemia and we’re taking a close look at the changes… in these genetic changes in therapy related leukemia and MDS and as you can see from the differences in the height of the red and the blue bars there are differences in people who develop MDS or leukemia after they’ve had breast cancer therapy, for example, or lymphoma therapy. There are differences between people who’ve had that type of MDS and have MDS without having another cancer beforehand and so they’re starting to sort of dissect out to kind of separate out these people based on their genetics with the hope that we can develop targeted therapies and more personalized therapy, if you will, for people who have MDS.

MDSF2014-NYC-1 New York Forum Part 1 September 20, 2014 Page 11 of 30 This is a very busy slide that I use for my regular talks that I give to my colleagues but I just want you to look at the upper middle circle where you see mostly the pink. That pink portion of the pie, if you will, it shows that the majority of people who have a certain type of MDS called RARS. I don’t know if anybody here has RARS, but if you have RARS you are very likely to have this SF3B1 mutation. I don’t expect you to memorize this and there’s not going to be a quiz, but just to get across a point that we’re finding these mutations not only in MDS in general, but we’re finding specific mutations in people with specific types of MDS and as we speak there are companies that are making drugs to target mutations like this. So, we’re really looking forward to what’s coming down the road in terms of new treatments for this disease based on this type of new research.

And with that, I’ll say thank you and just on the subject of genetics and research, I brought some posters from my group and some of the laboratory colleagues. I put them back there just to sort of give you an idea of the type of research that we’re doing here spanning from the laboratory to clinical studies, looking at treatments, looking at new ways to establish a diagnosis of MDS and I’ll be here through lunch and we can… if you have questions, I can go and kind of explain to you what some of this new studies mean and what we’re trying to do here at Sloan Kettering and with that I’ll take some questions.

Q1: What if anything is the benefit of a genome sequence (inaudible 45:33)?

Virginia Klimek, MD: Oh, not fair. Not a fair question. It’s a very good question. I’m not surprised that somebody asked that actually. We don’t know. We don’t know yet because the technology is getting a little bit ahead of what we can act on. So, we now know that we can look at the genetic sequence, the entire genetic sequence of a human being and we’re starting to find things that we don’t understand completely. We have… As an example, there are mutations, some of these genetic changes I was talking about, there are mutations that are commonly seen in people with MDS and leukemia. We think that based on laboratory studies, we think that those are important and are causing this disease, causing the MDS and leukemia. However when we start… We can actually look at normal people and find some of these abnormalities as well, people who are normal. They don’t have MDS. They have normal blood counts. So, we have to be very careful about taking a laboratory finding and saying, “Okay, if you have that laboratory finding that means you have a disease,” because we’re finding that it doesn’t necessarily mean that you have a disease and it’s very difficult to counsel somebody about that. If they have this mutation, for example, we don’t know if that means like five years from now or 10 years from now they’re going to develop MDS. We don’t know that. So, we have to be very cautious about how we interpret these findings. If you already have an established disease, that’s a more defined setting and we feel a little more confident about how to interpret that because we ca look at a lot of people, hundreds of people who have that abnormality and make clinical correlations to how they do what that disease, but when we start finding things that are new and really not validated, it’s kind of risky territory.

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Q1: Thank you.

Q2: You mentioned hypomethylating agents, but you really didn’t say much about what they were.

Virginia Klimek, MD: The hypomethylating agents, those are the Vidaza and the Decitabine chemotherapy drugs. They work… The name is that they’re called hypomethylating. That means they remove a chemical group called methyl and methyl chemical group that’s actually technically what it does. It removes it from your DNA. When people develop cancer, many cancers have this problem as well MDS and leukemia, there’s too much of that methyl group on the DNA and it interferes with the way the DNA works. It’s kind of like having like a locked file cabinet like your body needs to get that information to make your body work normally, but the methyl group and there’s a lot of methyl groups on the DNA, your body can’t access that information. So, the way we think these drugs work is that it moves those methyl groups and allows your body to use your genetic information, your genetic blueprint if you will, to help your bone marrow function more properly. When I’m in my office I use my telephone cord and I show how the DNA is all coiled up and you can’t access it, but I don’t have that, but that’s the basic idea.

Q2: Thank you.

Virginia Klimek, MD: You’re welcome.

Q3: Hi. Two questions. What sort of antibiotics do you use to fight or prevent infections and does that mean that there would be a larger percentage of MDS patients who actually have developed or develop C. difficile because of that connection with taking antibiotics and maybe getting C. difficile and the other question is could you mention if there’s been any literature that has tied AG221 to MDS and good effects of that as opposed to just AML?

Virginia Klimek, MD: So, the AG221 is targeting an abnormality that’s far more common in AML. The antibiotic question is… so we don’t use antibiotics in everybody. Even if you have a low white blood cell count, you may find that you’re not running into trouble with frequent infections. You’d be amazed at what your body can do. I mean, you can have a low white count, your neutrophil count can be low, but you won’t necessarily… it doesn’t necessarily guarantee that you’re going to have problems with infections. So, the majority of my patients who are neutropenic who have low white counts, they’re not on chronic antibiotics, but if somebody has a low white blood cell count or even if their white count isn’t so low, but they’re running into trouble with frequent infections. In that setting, the benefit… remember this is this whole benefit/risk balance. So, the benefit of preventing a bad pneumonia or the benefit of preventing skin infections, whatever you’re prone to, the benefit outweighs… in that setting it outweighs the risk of the C. difficile which is, obviously, (inaudible 51:44).

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Q3: When the MDS Foundation puts your verbal talk on their screen, will it also be accompanied by your slides that were great?

Virginia Klimek, MD: I don’t know.

Jayshree Shah: (inaudible 51:58) asked the MDS Foundation to do so. I know that her slides will be available. If you choose to just let Debra or Audrey know outside and just tell them to mark it.

Send it to your E-mail that (inaudible 52:14) provided and she will do so.

Q3: Thank you.

Virginia Klimek, MD: I think we’ll move on. I’ve invited one of my colleagues here to give a talk about transplant. Now remember, I mentioned earlier that transplant may not be an option for everybody, but we are working our hardest to try to make transplant available and safe for as many people as possible and so I really wanted her to come and talk to us about allogeneic stem cell transplants for MDS and she’s going to talk about for whom, when and how. This is Dr.

Boglarka Gyurkocza.

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