From the CEO’s Desk
The Best Is Yet To Come
Thank you for visiting the Cellceutix Corporation website. My name is Leo Ehrlich, Chief Executive Officer of Cellceutix. Cellceutix is a Beverly, Massachusetts-based clinical stage biotechnology company focused on discovering small molecule drugs for hard to treat diseases, including drug-resistant cancers, psoriasis, autism and inflammatory disease. There are many great things happening at Cellceutix.
Our flagship compound, Kevetrin, is a novel drug that has shown extremely promising laboratory data as a new cancer treatment. Clinical trials have already started at Harvard University’s Dana-Farber Cancer Institute and partner Beth Israel Deaconess Medical Center, arguably the pinnacle of cancer research hospitals in the world, to test Kevetrin against advanced solid tumors. Additional studies are being conducted at Beth Israel Deaconess to research Kevetrin in conjunction with two Pfizer multikinase inhibitors as potential new therapies for renal cancer and melanoma. In the first half of 2014, we expect to start a clinical trial against Acute Myelogenous Leukemia (AML), sponsored by a European university.
In preparation for human trials, we dedicated years to pre-clinical research of Kevetrin to test our proprietary compound against many different cancer lines and delineate its Mechanism of Action. What we discovered was potent anticancer activity against every type of cancer that we tested, regardless of origin. The data collected showed Kevetrin to be effective against many different types of cancer, including lung, breast, colon, prostate, squamous cell carcinoma, a leukemia tumor model and malignant glioma (brain tumor). In all of our research, tumor growth delay and tumor size reduction was shown.
Kevetrin has demonstrated the potential for a major breakthrough in cancer research by exhibiting an activation of p53. p53, often referred to as the “Guardian Angel Gene” or the “Guardian Angel of the Human Genome” due its crucial role in controlling cell mutations, is a tumor suppressor protein that is encoded by the TP53 gene in humans and has been widely regarded as possibly holding a key to the future of cancer therapies. As a potent anti-proliferative and pro-apoptotic protein, p53 has been shown to play critical roles in the homeostatic health of the human body by activating proteins required to repair DNA and plays a major role in the life cycle of cells by inducing cell cycle arrest and apoptosis (or programmed cell death) to maintain cellular and genetic stability.
The p53 gene is the most commonly disrupted gene in cancer. In more than 50 percent of all human carcinomas, p53 is limited in its anti-tumor activities by mutations in the protein itself. Currently, there are greater than 10 million people with tumors that contain inactivated p53, while a similar number have tumors in which the p53 pathway is partially abrogated by inactivation of other signaling components. This has left cancer researchers with the grand challenge of searching for therapies that could restore the protein’s protective function, which Kevetrin appears to be doing the majority of the time.
Because of the abundance of cancers with p53 disruption, a multi-billion dollar opportunity is present for a new drug that addresses p53 damage to restore the key protein to its normal role as a master regulator of the cell cycle. Nearly a decade ago, Roche began developing a series of molecules called cis-imidazolines analogs, which they called “Nutlins,” as selective antagonists of p53-MDM2 binding that were heralded as a major breakthrough in oncology. While contributing invaluable knowledge to the industry, Nutlins were shown to be genotoxic, meaning that they damage healthy DNA surrounding cancerous tumors.
Importantly, our research has shown that Kevetrin, which is structurally different from Nutlins and other anti-cancer agents in development or currently on the market, is non-genotoxic.
We are unaware of any other company that is actively in clinical trials with a non-genotoxic drug utilizing the p53 pathway as its Mechanism of Action; positioning us as a clear leader in the space.
Additionally, the Cellceutix research team has discovered that Kevetrin’s Mechanism of Action may also be connected with the retinoblastoma protein (RB), a second major component in controlling tumor progression. Primarily, RB is responsible for stopping S-phase (synthesis phase – the point where DNA is replicated) entry of a cell cycle at the G1 checkpoint. In nearly 100 percent of cancers, regardless of origin, RB or p53 is dysfunctional.
If clinical trials show that Kevetrin restores damaged RB and p53 in humans to their normal tumor suppressing status , it could be one the greatest breakthroughs in cancer history.
Kevetrin is thoroughly protected through pending patents which covers tens of thousands of possible future chemical combinations for new drugs; a potentially very valuable proposition for multiple drug development in the future.
Our second drug in development is an anti-psoriasis compound called Prurisol. Prurisol is a small molecule acting on the principles of immune modulation and PRINS reduction that has been found to be effective against psoriasis in animal models, both in induced psoriasis as well as a xenograft model with human psoriatic tissue. It is easily synthesized and has the potential for oral dosing, the preferred drug delivery method of patients worldwide. With more than 150 million people globally suffering from psoriasis and 150,000 new cases of psoriasis per year in the U.S. alone, Prurisol represents a very unique opportunity to potentially generate substantial revenue for Cellceutix.
Prurisol clearly outperformed methotrexate, a standard of care today for psoriasis, in human xenografts models of mouse studies.
In 2012 we announced that we had signed an agreement with Dr. Reddy’s Labs to manufacture Prurisol. We are working on advancing Prurisol immediately into mid-to-late stage clinical trials based on the fact that the active moiety of Prurisol has already received U.S. Food and Drug Administration approval. After meeting with the FDA in June regarding the regulatory pathway for Prurisol, the agency informed us that a 505(b)(2) application would be an acceptable approach for the new drug candidate. With that guidance from the FDA, we are now moving forward with planning the first Phase II clinical trial for Prurisol.
Our final compound in development is KM-391, a novel compound being developed for the treatment of autism. While still early in development, our initial research is very encouraging. Neonatal serotonin depletion and reduced plasticity of the brain are salient features well documented in the autistic brain. We focused on these benchmarks in our research and showed that the brains of KM-391 treated animals exhibited increased brain plasticity and serotonin levels as compared to paired twins serving as controls. Additionally, specific behavior characteristics – such as repetitive behavior and self-induced injury – were greatly reduced.
There are currently no drugs available on the market and only a very limited number of drugs in development that address the core issues of autism in the manner that KM-391 does. Again, we feel that this drug puts us in a strong position for growth and partnering opportunities.
At Cellceutix, we feel that optimism and hard work is the only option. Our optimism is based on our team which consists of some of the most passionate and brightest minds in drug development. They have a proven track record of success being involved in the development of drugs which have generated billions of dollars in revenues while providing a better standard of life for patients in need. This is the reason we have come together as a team.
I’d like to personally welcome everyone to Cellceutix Corporation and urge you to sign up on our home page for news alerts. If you have any questions, do not hesitate to contact us.
Chief Executive Officer
Cellceutix CEO Discusses Corporate Developments in 2012 And Plans For 2013
My fellow Cellceutix shareholders,
Looking back at the past 12 months, I could not be more pleased with the progression of our Company or more excited about 2013. It has always been our contention that Kevetrin™, our flagship anti-cancer drug, holds a great deal of promise as a potential therapeutic for a wide array of cancers and this past year has provided evidence that many leading institutions also see the potential. I’d like to take a moment and look at the progression of Kevetrin in 2012 and where it stands moving into 2013.
Company-sponsored clinical trials at Harvard University’s Dana-Farber Cancer Center and partner Beth Israel Deaconess Medical Center began in November testing Kevetrin for safety against solid tumors. At this time, first cohort patients are about to begin their third cycle of dosing and the second cohort – in which dosing levels were doubled – are in their first cycle of dosing. The information that we have received at this early stage of the trial so far has been very encouraging with no data being delivered outside of the parameters of the protocol. We expect some information on pharmacokinetics from the early cohorts in the near term.
European trials that are being sponsored by and hosted at a leading European University (the “University”) testing Kevetrin against Acute Myelogenous Leukemia (AML) are scheduled to begin in the first half of 2013. We have been advised that the study, a phase 1b trial, will be titled “A Multi-Center, Open-Labe, Phase 1B Study of Escalating Doses of Kevetrin (Thioureidobutyronitrile) Administered Intravenously, with Cytarabine Adminstered A) Subcutaneously, or B) Intravenously, in Patients with Acute Myelogenous Leukemia (AML).”
The protocol for the clinical trials is completed and undergoing a review process as well as other regulatory preparations progressing smoothly. The University has conducted pre-clinical research that they shared with us but asked that we not disclose the data as they “plan to publish it at a later day.” A very brief summary of the data showed Kevetrin causing apoptosis (programmed cell death) in cancerous cells in addition to sensitizing and optimizing the cells to maximize the effectiveness of drugs used in combination with Kevetrin. We will be releasing details about the University very shortly.
The University has indicated they are interested in a second study of Kevetrin in combination with a compound of a major European pharmaceutical company. We will update shareholders as this moves forward in 2013.
Preclinical tests are being performed at Beth Israel Deaconess Medical Center testing Kevetrin in combination with Pfizer, Inc.’s multikinase inhibitor drugs as potential new therapies for renal cancer and melanoma. We have seen the data and it is very exciting to us, but, again, confidentiality agreements prevent us from providing any details at this time. Moreover, data of this nature is generally not discussed so that the institutions may present their findings at industry conferences when they deem appropriate.
We disclosed in an 8-K filing with the Security and Exchange Commission on December 27 that the Company has recently been approached by one of the world’s most prominent cancer centers (the “Cancer Center”), located in the Southwest United States. The organization is interested in conducting and sponsoring studies on Kevetrin at their facilities. As stated in the regulatory filing, no assurances are made or implied that an agreement will be executed, but we are optimistic that the relationship will move forward early in 2013. We have now signed a Non-Disclosure Agreement with the Cancer Center and are quickly moving forward with a Material Transfer Agreement. Further details will be provided as permitted, but we are free to say that the Cancer Center is interested in the possibility of Kevetrin as a potential therapy for myeloma. Just like the other institutions, they will perform their own preclinical testing of Kevetrin.
Regarding Prurisol™, our leading anti-psoriasis drug candidate, a Company-sponsored Phase II/III Proof of Concept trial is on target to begin late first quarter/early second quarter 2013. This will be a relatively short trial with only 30 days of treatment and 30 days of follow-up to evaluate the efficacy and safety of Prurisol. Dr. Reddy’s Laboratories Ltd. has condensed the manufacturing process, saving us time and money. Manufacturing of cGMP Prurisol is expected to begin in about a months time. Given the outstanding data from our laboratory studies and the guidance from the Food and Drug Administration that a 505(b)(2) pathway is acceptable, we have high expectations for these trials to support a larger scale, late stage clinical trial. I believe updates on these trials will be quite frequent.
Although many of the expected studies on Kevetrin will be sponsored by outside organizations, financing is still critical to advancing those drugs further into human trials. To achieve this, we are extremely pleased to have received institutional support through a $10 million common stock purchase agreement with Aspire Capital Fund LLC in December. Aspire has committed to purchase up to $10 million of our Company’s common stock over the next three years at prices based on the market price at the time of each sale.
2012 has put us at the forefront of some of the leading cancer research centers in the world and I could not be more proud. Kevetrin is an extremely unique drug, actually classified as a new class of chemistry in medicine, and its ability to re-activate p53 could simply be unprecedented if it continues to show low toxicity. There is no comparison of Kevetrin to any other compound. I think that the interest that has been shown by organizations approaching us wishing to conduct and sponsor research speaks for itself as to what some of the brightest minds in oncology see in the data. It has been well publicized what a major impact a p53 drug can have on cancer research and we are excited to be at the forefront of pharmaceutical companies in that space.
We are also very eager to evaluate Prurisol in clinical trials in a matter of months. While not as high-profile as a new cancer drug, psoriasis represents a multi-billion dollar opportunity and an area of great unmet medical need. Soon clinical research on Prurisol will begin which we anticipate will validate our laboratories studies and allow us to have a very promising late-stage clinical trial in the mid-term.
Of course, a big key to this is the new association with Aspire Capital Fund LLC. Their commitment to provide us funding at such favorable terms will prove invaluable to developing our pipeline.
I have stated in the past, “the best is yet to come” and I believe that 2012 was full of our Company meeting milestones that has us firmly moving forward and gaining recognition as an innovative biotechnology company. We have distinguished ourselves through Kevetrin as one of only a handful of companies that is developing a p53 drug, but that is not all that we have to offer. Prurisol is jumping straight into mid-stage trials and we have six other compounds in our pipeline that hold great potential as well. 2012 has seen us transition into a clinical stage company and provided us with the financing to continue to move ahead, but I believe that 2013 will be a bigger year for us with clinical data coming from multiple trials and that the best is still yet to come.
We appreciate the support that we have received from our loyal shareholders throughout the year and pledge to continue to immediately provide as much information as possible through public disclosures as they happen in the coming year.
Chief Executive Officer, Cellceutix Corporation
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To the extent that statements in this press release are not strictly historical, including statements as to revenue projections, business strategy, outlook, objectives, future milestones, plans, intentions, goals, future financial conditions, future collaboration agreements, the success of the Company’s development, events conditioned on stockholder or other approval, or otherwise as to future events, such statements are forward-looking, and are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The forward-looking statements contained in this release are subject to certain risks and uncertainties that could cause actual results to differ materially from the statements made. Factors that may impact Cellceutix’s success are more fully disclosed in Cellceutix’s most recent public filings with the U.S. Securities and Exchange Commission.