Emmaus Medical Inc

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March 4, 2013 Issue

The Most Powerful Name In Corporate News and Information


With a focus on using Oral L-glutamine, Emmaus Medical, Inc. is the Only Company Currently Conducting a Phase III Clinical Trail for the Treatment of Sickle Cell Disease

About Emmaus Medical, Inc.:


Emmaus Medical, Inc. is a Torrance, California-based pharmaceutical company dedicated to developing and bringing to market new treatments for rare diseases and conditions. Emmaus Medical launched its first product, NutreStore® [L-glutamine powder for oral solution] for the treatment of Short Bowel Syndrome (SBS) in June 2008. Emmaus Medical is currently conducting a Phase 3 clinical trial to investigate the safety and efficacy of oral L-glutamine for the treatment of sickle cell disease. Emmaus Medical has received FDA orphan drug designation and fast track designation for its sickle cell treatment. Emmaus Medical is also developing regenerative medicine products.

Yutaka Niihara, MD, MPH


Dr. Niihara is a hematologist whose background treating patients with sickle cell disease led him to seek alternate treatments for sufferers of this debilitating disease. He became the principal inventor of the patented L-glutamine therapy for the treatment of sickle cell disease, and co-founded Emmaus Medical, Inc. Dr. Niihara is board-certified by the American Board of Internal Medicine/Hematology.

Dr. Niihara is licensed to practice medicine in both the U.S. and Japan. He is a Clinical Professor of Medicine, Division of Medical Oncology/Hematology at UCLA School of Medicine. Dr. Niihara is the recipient of numerous grants, including a completed NIH grant study for "L-glutamine Therapy for Sickle Cell Disease", and an FDA Orphan Drug Grant that supported an ongoing Phase 2/3 clinical trial.

Healthcare – Pharmaceutical

Sickle Cell Anemia


Emmaus Medical Inc

20725 S. Western Avenue, Suite 136
Torrance, CA 90501-1884



Interview conducted by: Lynn Fosse, Senior Editor, CEOCFO Magazine, Published – March 4, 2013


CEOCFO: Dr. Niihara, what is the vision at Emmaus Medical?

Dr. Niihara: Our vision is to bring treatment to those people who are suffering from diseases where the treatments are scarce. It is our hope that we will be able to reach out to those people who, for different reasons, have difficulty accessing medical care.


CEOCFO: What is your focus today?  

Dr. Niihara: Today our focus is to finish developing the treatment for sickle cell disease, which is one of the rare diseases. It affects mostly African American people here in this country, but throughout the world mostly people of African origin. There are many patients in Europe, Africa, the Middle East, India and South America. We want to bring this treatment to the population who are suffering from enormous pain and a shortened life span. They also have many social and economic difficulties because of the disease.


CEOCFO: What is the therapy or alternative treatment that you have helped develop?

Dr. Niihara: This is a treatment using one of the most common amino acids -- glutamine. We have discovered that giving glutamine to these patients increases a substance within their body that counteracts the effect of sickle cell disease. What we have seen is that the patients are having decreased pain levels and fewer complications due to sickle cell disease. The nice thing about this treatment is that it is very easy to administer and has minimal side effects.  


CEOCFO: What made you decide to try glutamine?

Dr. Niihara: We were looking for a molecule that acts as an antioxidant. We were looking for substances that counteract the oxidation process. Oxidation is sort of like sunburn. It can actually hurt our body and our cells, but is necessary in our body in a controlled manner, because having oxidation gets rid of the unwanted organisms, like bacteria or viruses, from our system.


However, when you have too much, it destroys red blood cells. What we saw in sickle cells is that each sickle cell had too much oxidation damage and was not able to counteract this oxidation process. We were looking at NAD, a molecule that counteracts this oxidation process. We noticed that the sickle cell patients’ blood cells were trying to produce more of this NAD, and we assumed that this was to counteract the oxidation process. However, the cells were not able to keep up with it. Therefore, we looked into different things to figure out what we could do to help the cell produce more NAD. We realized that providing the cells with building blocks for NAD would help, and glutamine happens to be one of the building blocks. Once we started to give the patients glutamine, the cells all of a sudden started to make much more NAD. As a result, the oxidation level decreased. Previously, because of the oxidation, the cells were damaged and getting deformed. However, after we started to treat the patients with glutamine, the deformities started to decrease to the point that the blood cells looked more like normal blood cells instead of sickle-shaped cells.


CEOCFO: Where are you in the process of development?

Dr. Niihara: We started this in 1992 and twenty years later we are in the very last phase of clinical trials. We conducted preclinical trials, clinical trials, Phase I, II, and now we are finishing up with Phase III. We anticipate finishing the Phase III clinical trial under the supervision of the FDA before the end of 2013. After this, based on the data, we are hoping to bring the data to the FDA for evaluation for FDA approval to start treatment.


CEOCFO: Has the medical community been paying attention?

Dr. Niihara: In a way yes and in a way no. That is because we were able to do this without making a lot of noise up until the Phase II clinical trials. Of course the FDA knew about this, and the National Institutes of Health knew about this, and leading thought leaders in the field of sickle cell disease knew about this. However, once we got into our Phase III clinical trials we really had to get help from all the people who are involved in the sickle cell community. We were able to get most of the academic institutions that deal with sickle cell disease to be a part of our Phase III clinical trials. Now, many people know about this study.


What was interesting was that just this last week I went to a health care conference in San Francisco and many people in the health care industry knew about sickle cell disease, but did not know about our research. Many of them came up to me and said, “We did not realize that there is a company like yours that is, by far, leading the pack in the development of a treatment for sickle cell disease.” That is because we happen to be the only company that has a product in Phase III clinical trial. Thousands of potential treatments or medications have been proposed to be studied, but most of them stopped before they were brought to a clinical trial, because they realized that it was either ineffective or unsafe. Out of the thousands, only about eighty or so were brought to clinical trials. Out of those some eighty potential sickle cell treatments, only three have actually reached Phase III. The first one is called hydroxyurea. This happened in 1995, and it was actually approved. However, the majority of the patients cannot use it because of the side effects. The next one is not really a treatment for sickle cell disease, but a treatment for the complications of sickle cell disease. Therefore, it does not really treat the disease at its root. Then ours came out, and ours is the third medication to reach Phase III clinical trials. Therefore, we are in a fairly important position. At this very moment, we are the only company that is actually conducting a Phase III clinical trial for the treatment of sickle cell disease. The other companies claim that they have gotten the FDA’s permission to move on to Phase III clinical trials, but so far there is no one else that is actually running Phase III clinical trials at this moment, except for us.  


CEOCFO: Is Emmaus Medical funded to get through the next steps?     

Dr. Niihara: So far, we have been funded by various supportive individuals. We have raised over thirty million dollars up till now for this project. However to complete the trial and prepare for potential commercialization, we are seeking an additional fifteen million dollars.


CEOCFO: What else is going on at Emmaus?

Dr. Niihara: We have important technology in regenerative medicine from Japan. This is called Cell Sheet Technology. For the first time Dr. Okano has accomplished production of cell sheets. We can grow and multiply cells as long as we have a good living cell. In research of cells, one of the major problems is that once we grow them, we need to harvest them but we have a hard time getting the cells off of the Petri dish where the cells were growing. In order to get the cell off the dish, we damaged cells significantly. It is like in the olden days when we made fried eggs on the frying pan and it did not have Teflon coating like today. Then the egg would get stuck on the pan and in the process of getting the egg off of the pan you would mess up the fried eggs. We mess up the cells in a much worse way trying to separate them from the Petri dish. However, Dr. Okano came up with this technology where now we can cleanly get the cells off of the Petri dish in a nice circular sheet without any problem. This makes the transplant of your own cells to various parts of your own body possible. For example, there are people who have a problem with the outer layer of the eye. Once the outer layer of the eye is damaged, you are officially blind in that eye. Before, we did not have any technology to replace the outer layer of the eye if it was completely gone. However, using this technology we can take some cells from the inside of your mouth and grow it into a cell sheet. Now, we can cleanly harvest this cell sheet and can simply place it onto the damaged eye. We do not even need to suture, because the cells have its own type of glue that sticks. Patients are recovering their eyesight with this technology. This is revolutionary. It is quite exciting! There are many dozens of cases in Japan, and in Europe they also have about the same number of cases that have been successful. This can be applied in many other tissues, too. Because the origin of the tissue is the patients themselves, there is no issue with rejection. That is one of the problems with a transplant from a donor. What is really exciting is the heart. There is a disease of the heart where it is so weak and the heart becomes ballooned up. Once the heart is ballooned up the patient goes into severe heart failure and he or she essentially waits to die unless he or she gets a heart transplant. This is actually one of the major indications for heart transplants. Many of these people become this way after suffering multiple heart attacks and the heart just gets so weak. People also can get this condition after a viral infection that can weaken the heart so that it can no longer pump effectively. Those people have to get heart transplants in order to survive in today’s standard of care. However, now we could take the cells from their own legs and then select out the younger cells from them. We call these younger cells myoblast. When these myoblasts are grown with the cell sheet technology, they can become a tool that can reconstruct the weakened heart. Because these are younger cells, they can become a leg muscle or transform into a heart muscle. We can paste this cell sheet without cutting the heart. This does require cutting the chest wall to access the heart, but avoids cutting into the heart at all. You just paste the cell sheet over the heart and in a period of three months, the sheet of cells will repair the heart and avoid a heart transplant. In Japan they have done over ten cases and they have been successful. This is something that not too many people here in the U.S. have heard of. Fortunately, our company has the license in the U.S. to co-develop this treatment. We are doing trials using animals and we have been successful with the eye and will probably move onto the heart pretty soon. For the eye, we hope that we can start working on treating human patient volunteers within a year or two on a clinical trial basis.     


CEOCFO: Why does Emmaus Medical stand out to investors and people in the business community?

Dr. Niihara: We have our sickle cell medication in Phase III and I believe that this is going to be approved very soon. Every year, there are only twenty-four to thirty medications that are approved. We believe we are at the point where we will provide our investors with a great opportunity. Aside from financial opportunity, this medication is going to treat the disease that has been for the most part untreatable up to this point. Now, you can provide this simple treatment and potentially change the lives of some hundred thousand patients in this country, and possibly four million patients in Africa. Along with moral satisfaction, there is a financial incentive in doing this. It is not every day that you will have this type of opportunity. Sickle cell disease is called a prolonged death sentence among the African people. There are social, medical and many other aspects that the patients suffer from. I started working on this as a physician because I had to deal with so many of these cases and I actually had to see many of these patients die. One of the most difficult things for me was that when I first started this I was in my late twenties and I had many patients who had just become my patients and they were all about my age. There were about ten of them and I can name each one. Over time we became pretty close as I realized that this was a devastating disease and I had to pay special attention to them. I did not think that I was doing anything special, but because I felt really bad for them, I paid a little extra attention to them. It is really just my responsibility as a physician, but they really appreciated that. I became very attached to them and was very happy that I was able to do some research for them. However, when I reached my forties and they reached their forties they all died like clockwork. When I saw that I said, “My goodness, this is real.” Based on the effects I observed so far, I have a feeling that this treatment is going to extend the life expectancy but, more importantly I have observed that there are so many people whose quality of life have improved dramatically on this treatment. I really hope that people will understand the devastation that the sickle cell patients are going through and there will be someone who will help us and help these patients get to a permanent cure. We hope that there will be many other effective treatments to be developed, like gene therapy and so forth, so that they will have a little bit better lives to live.


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“At this very moment, we are the only company that is actually conducting a Phase III clinical trial for the treatment of sickle cell disease.”- Yutaka Niihara, MD, MPH


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