EGEN, Inc.

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October 22, 2012 Issue

The Most Powerful Name In Corporate News and Information


Developing Nucleic Acid-Based Therapeutics for Cancer and other Difficult to Treat Diseases using proprietary Nanoparticle Delivery Systems and Expression Vectors, EGEN, Inc. has been Successful in Coming Up with Delivery Systems that are Safe and Efficient

Khursheed Anwer, Ph.D., M.B.A.
President and Chief Scientific Officer

Dr. Anwer joined EGEN, Inc. in July, 2002 as Vice-President of Research and Development. During his tenure at the company he has directed the clinical and preclinical research and development of the company and has been at his current position in the company since 2009. Dr. Anwer has a Ph.D. in Physiology/Pharmacology from Ohio University, an M.B.A. from University of Alabama and post-doctoral training from the University of Texas Health Science Center at Houston. Before joining EGEN, Dr. Anwer was Director of Pre-Clinical Development at Valentis, Inc. from 2000 to 2002. From 1993 to 1999, he served in several positions at Valentis/GeneMedicine, Inc. where he led several research projects in the area of non-viral gene therapy. He has co-authored more than 40 publications in the area of non-viral gene therapy resulting from his active career in research and development. Dr. Anwer is also an adjunct professor in the Biological Sciences Department at the University of Alabama in Huntsville where he has been involved in graduate level teaching and supervising doctoral students.

About EGEN, Inc.
EGEN, Inc., is a clinical stage biopharmaceutical company focused on developing nucleic acid-based therapeutics for cancer and other difficult to treat diseases using proprietary nanoparticle delivery systems and expression vectors. The Company's synthetic biocompatible delivery vehicles can be used to deliver therapeutic genes, inhibitory RNA (RNAi), and small molecules by protecting the therapeutic cargo from degradation, promoting uptake by target cells, and facilitating intracellular trafficking.


EGEN, Inc.
601 Genome Way

Huntsville, AL 35806



Interview conducted by: Lynn Fosse, Senior Editor, CEOCFO Magazine, Published – October 22, 2012

CEOCFO: Dr. Anwer, you have been with EGEN for a long time and have been president there for over three years. How has the company changed since you have taken over the helm?

Dr. Anwer: I took the company leadership at a critical time. We were embarking upon our phase II clinical trial and starting additional clinical development of our lead product, considering our business strategy of handing over our product when we finish our phase I or phase II trials. In that sense, over the last year and a half our focus has been more towards gearing up for monetizing some of our assets. We have increased the activity in that area. We have also made collaboration with government agencies such as NCI and as a result brought a significant non-diluted funding to the company. We have expanded our clinical development program to additional indications in collaboration with a private hospital, again bringing significant non-diluted funding to the company. Hence, since I took over the office, we have expanded our clinical program through private and government collaborations bringing significant non-diluted funding and consequently adding to the shareholder value. One of our most significant achievements in these tough economic times is the expansion of our clinical program to conducting multiple clinical trials with the help of government and private collaborations bringing non-diluted funding to raising the shareholder value.


CEOCFO: What is the basis? What are the technology and premise behind EGEN?

Dr. Anwer: EGEN is primarily a delivery company. We discover and design new methods to deliver nucleic acid drugs. Nucleic acid drugs are mainly DNA and RNA. These drugs are novel, cutting edge therapies that have not been approved yet, but have promise for future unmet clinical needs. The delivery of these biologics, the nucleic acids, has been very challenging over the years. Our company designs and develops delivery systems that are safe and efficient. We have technical expertise for the design, synthesis and development of the synthetic delivery systems. We have an integrated approach to development where we use in-house expertise in chemistry, formulation, and biology pharmacology to achieve proof of concept. We do discovery and development from the start to the end. The delivery expertise company has is the key to our strength and our core technology. We also have novel expression vectors that are also the basis of our core technology.


CEOCFO: What have you figured out at EGEN that other people do not know, or do not know how to do?

Dr. Anwer: We have been very successful in coming up with delivery systems that are safe and efficient. We have been able to show success of a gene therapy product through a delivery route (intraperitoneal) that has not been used in the past by other gene therapy companies. Our strength is essentially a lot of experience in DNA and RNA delivery. I have over twenty years of experience in nucleic acid delivery, so based on our knowledge and understanding of the delivery issues and biology we come up with new ideas and develop new products that are different from other people.


CEOCFO: How is your product reacting or acting in the body that is not the typical manner?

Dr. Anwer: The typical treatment approach for cancer is surgery, irradiation or chemotherapy. That is the standard way of treating cancer. Our approach involves immuno therapy, which means that we boost the immune system of a patient to fight cancer. Our approach is to give gene for one of the most potent immune stimulating agents locally at the cancer site to get the production of the anti cancer molecules that boosts the immune system for several days after a single injection without causing systemic toxicity. Compared to the available cancer therapies, this is a local therapy which boosts the immune system, a distinct difference from chemotherapy or cytotoxic therapies. Most importantly, it does not have the classical toxicity that you see with chemotherapy or some other biologics such as antibodies. Typical cancer therapeutics have side affects in bone marrow toxicity where patients have low red blood cell count, low white blood cell count, and also liver toxicity, neuro toxicity and gastrointestinal perforation. A clear advantage of our investigative cancer product is that it is given weekly for several months without the toxicity profile typically observed with current cancer therapies.


CEOCFO: That is quite a big difference.

Dr. Anwer: Yes.


CEOCFO: Has the medical community been paying attention?

Dr. Anwer: Yes. We had done two phase I clinical trials of this product on our own and the results have been found intriguing. The Gynecologic Oncology Group (GOG) of the National Cancer Institute has interest in new cancer therapies is conducting a phase II study of our cancer product alone and a Phase I study of it in combination with chemotherapy. GOG is a large group of gynecologic oncologists, which has found this drug interesting to do two clinical trials. The product information is reviewed by the oncologists in the field before giving approval for a clinical study. Our immunotherapy product also caught attention of a private hospital in the country. Their clinical development people with expertise in immunology are co-developing this product for colorectal cancer. The GOG and NCI trials are for ovarian cancer and the trial with private hospital is for colorectal cancer. Also, the Nanotechnology Characterization Laboratory (NCL) is developing this product for Glioblastoma, since we have intriguing data in animal models of brain cancer by direct injection of our product into experimental brain tumors. Therefore, the NCI, the private hospital and the NCL have enough interest to invest their time and money into this product for development. We have published a number of papers and made presentations on this novel cancer product. Our product was considered among the top hundred oncology drugs by R&D magazine.


CEOCFO: Why the choice of specific cancers?

Dr. Anwer: Most of our work with this product is for local therapy of cancers that spread into the abdominal cavity. The drug is given into the abdominal cavity and that is where it works the best. Our specific strategy has been to go after cancers that spread into the abdominal cavity. Ninety percent of ovarian cancer patients have a recurrence into the abdominal cavity. Therefore, when we talked to the private hospital for extending into colorectal cancer our strategic intent was to extend to a similar disease which is not ovarian but also metastasized to the abdominal cavity. Therefore, the colorectal cancer patients and appendix cancer patients who have metastasis in the abdominal cavity became part of our extended development plan. The same immuno therapy works in small cavities also as evident from its activity in glioblastoma model. The NCL funding is for pre-clinical development of the drug for glioblastoma. The GOG’s focus is women’s cancers, and since we are developing a novel therapy for ovarian cancer, our goals are overlapping. GOG’s interest in novel therapies and intraperitoneal delivery has been the reason for collaboration and our focus for the syndication.


CEOCFO: What is ahead for the next year or two?

Dr. Anwer: Right now we have three clinical trials, either actively running or they will be active soon, possibly within a month. We would like to finish our phase II clinical trial of our lead product EGEN-001 in ovarian cancer. Our target is to finish that by early spring 2014. This month or next month, depending on when the approval finally takes place, we will be starting a combination trial where our immunotherapy product is combined with chemotherapy for ovarian cancer. We would like to finish that study by the end of next year. The colorectal study that I told you about at the private hospital, will also be reaching some conclusive results by the end of next year or early 2014. Our objective is to complete these clinical trials by the end of next year, or early 2014. During this time we will try to out-license the clinical product to a pharmaceutical company or put the asset for merger acquisition by an interested party. That is the outlook over the next year and a half to two years for this clinical product. We also have a ground breaking technology by which we can engineer a cell to produce RNA and secrete it outside the cell. We call this RNA Amplification and Secretion Technology or RAST. This technology is very unique. Normally cells do not secrete RNA. The actively expressing genes make mRNA and mRNA make protein inside the cell. When that protein is secreted RNA is not secreted, and yet many new potential therapies are RNA. Therefore, people deliver RNA into cells to change the cell function and this delivery is specific to a specific cell. It goes inside the cell and that is where the RNA is consumed. The delivery of RNA is not very efficient; you cannot transfect or engineer a lot of cells. You can only do some cells. That is the limitation of the technology. What we have done is we have the technology where, even though we introduce RNA initially into few cells, we modify these cells to secrete the RNA that cells normally do not. With the current methods, once the RNA is delivered inside the cell it is consumed in that cell and a relevant cell function is modified only in that cell. With our RAS technology we engineer a cell to not only produce RNA but also to secrete it to cell exterior. The secreted RNA then influences the function of neighboring cells by either binding to the cell membrane or penetrating the cell interior. Therefore, amplifying the initial signals. Our RNA amplification and secretion technology is potentially a game changer, because the cells are not known to secrete RNA and if you can make them secrete RNA and make the RNA penetrate the neighboring cells you can create a totally new types of product lines in therapeutics and life science research. This project is in the early stage for us now. We have demonstrated proof of concept of this technology in cell culture. Over the next year and a half our goal for RAST platform is to demonstrate proof of concept in an animal studies. If we successfully demonstrate proof of concept in in vivo, then RAST can really be a game changing technology. It is at the early stage. We have a lot of work to do to be able to demonstrate proof of concept in vivo. As I said to you earlier, our strength or our specialty is the delivery of nucleic acid therapeutics. You can divide nucleic acid into two classes; DNA and RNA. The product that is in the clinic for ovarian and colorectal cancer is a DNA product. This oncology product comprises gene for IL-12, a potent immune stimulating molecule, and a proprietary delivery system for IL-12 DNA. We also have a gene silencing technology based on our proprietary RNAi delivery systems. Here we have developed a unique RNAi delivery system that causes gene silencing in lungs. You had asked me earlier how our technology differs from other companies. If you look at the landscape, the publications and other companies in the field, they have developed RNAi delivery technology or gene silencing technology that works on the liver after IV injections. Our systems work in lungs and so it creates new opportunities in lung therapeutics. Using these RNAi delivery systems we have demonstrated therapeutic effects in animal models of lung cancer and pulmonary hypertension. Therefore, we have three technology platforms in the company. One of these platforms deals with DNA delivery where our lead product is an immunotherapy for the treatment of ovarian and colorectal cancer and in preclinical development for brain cancer. The idea there is to give a DNA of a potent anticancer molecule to stimulate the immune system. You give it once every week and it is so safe that you can give it for several months and you do not see the typical toxicity that you see with chemotherapy. That is our lead program that is in the clinic. The second program, the RNA secretion and amplification, is in the early stage, but a game changer. The third program deals with gene silencing where we have demonstrated gene silencing in lungs, which is a distinguishing feature over liver specific delivery technologies from other companies. The physic-chemical properties of our delivery system are different, from other delivery systems that is why these systems are distributed to lung and not to the liver. In addition to these technologies we have modest revenue from DNA and RNA products that are commercialized in the research market. These research products are sold in the US, China and soon to be in India. We also provide pre-clinical contract work because we have a state of the art animal facility. Both preclinical contract work and regent sale business has limited revenue due to limited resource commitment to these projects. In the next year and two, we would like to complete the clinical trials of the lead product and try out-license it and use the revenue to grown the RAST platform where we can have very novel ground breaking products.


CEOCFO: You are very busy at EGEN!

Dr. Anwer: It has been very busy. Being a small company with multiple ongoing clinical trials and active discovery platforms and reagent business, we stay very busy.


CEOCFO: How do you stay focused and make sure the company is focused with so much happening?

Dr. Anwer: It is important that we keep the end in our mind. In clinical product our goal is not to get into too many diseases just for the sake of expanding. The goal is to develop the technology where it works the best. Therefore we focus only on those studies where there is great potential for success. It is easy in science to be able to do a lot of different things, but our goal has been keeping our business model in mind; that we will develop clinical product to phase I or phase II in conditions which are compatible with our technology. In our discovery program, our goal has been very focused that we will be developing only those products that are where our technology fits the best. That helps us stay away from straying into different areas and getting too diluted.


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We have been very successful in coming up with delivery systems that are safe and efficient. We have been able to show success of a gene therapy product through a delivery route (intraperitoneal) that has not been used in the past by other gene therapy companies. - Khursheed Anwer, Ph.D., M.B.A.


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