The cannabis sector continues to evolve. “Medical marijuana” has become medicinal cannabis. As the body of empirical evidence has grown, it is now apparent that cannabis is real medicine – with bona fide applications involving thousands of medical conditions.
More recently, some cannabis biopharma companies are taking this evolution much further. They are producing official, licensed, cannabinoid-based drugs. One of the cannabis biopharma companies leading the way in such clinical R&D is Kalytera Therapeutics Inc. (TSX: V.KALY, OTCQB: KALTF, Forum). This Company was originally introduced to the Stockhouse audience in a full-length feature article on February 6, 2018.
Kalytera’s clinical research focuses on cannabinoids, the active ingredients in the cannabis plant. With more than 80 cannabinoids already identified, this provides the potential for an enormous spectrum of drug development.
On April 19, 2018; KALY announced it has been issued a U.S. patent covering the use of cannabidiol (CBD) for the treatment of “severe and refractory graft versus host disease” (GVHD). GVHD is a life-threatening condition. It is a severe complication that commonly occurs following a bone-marrow transplant. This disorder results in the transplanted donor cells attacking the patient’s organs, including the skin, gastrointestinal tract, liver, lungs and eyes.
Cannabidiol is one of the cannabinoids that occurs in the highest concentration in the cannabis plant. This makes CBD-based therapies particularly attractive as potential commercial drug candidates.
Stockhouse recently had the opportunity to learn more about this important development from Kalytera’s President and CEO, Robert Farrell. We asked him for more details about the significance of this news as well as an update on the Company’s GVHD treatment and general operations.
For investors who are new to Kalytera Therapeutics, please introduce the Company.
Kalytera is focused on CBD. We are developing CBD (cannabidiol), as well as proprietary CBD analogues, for the treatment of human disease, and we are an acknowledged leader in the field of CBD pharmaceuticals.
In our lead program we are evaluating CBD in the prevention of graft versus host disease (
"GVHD"). This program is in late-stage clinical testing, and we are working with the FDA’s Division of Hematology Projects now to advance CBD into a Phase 3 pivotal registration study in treatment of acute GVHD later this year.
Even the filing of a new patent is a significant development since it provides the applicant with immediate protection for their discovery/innovation. What additional benefits are conferred when a patent is actually awarded?
We recently announced that the U.S. Patent and Trademark Office ("USPTO") has issued our patent covering the use of CBD in the treatment of GVHD. We anticipate that our patent covering the use of CBD in prevention of GVHD will be issued next month. The significance of patent issuance is that, until the patent is issued, it is always possible that the USPTO may find some reason to not issue the patent. Once the patent issues it is final. Our patents covering the use of CBD in the treatment and prevention of GVHD will give us market exclusivity through early 2034.
In addition to our patents and patent applications covering the use of CBD in GVHD, we have also obtained four orphan drug designations for the treatment and prevention of GVHD in the U.S. and Europe, and orphan drug designations provide additional market exclusivity.
Now let’s get to specifics. Please explain how KALY’s clinical research into GVHD can potentially help the sufferers of this affliction.
GVHD is a multisystem disorder that is a life-threatening complication commonly occurring after bone marrow transplant procedures. GVHD occurs when the transplanted donor cells attack the patient’s organs, including the skin, gastrointestinal tract, liver, lungs and eyes. GVHD is associated with acute and chronic illness, infections, disability, reduced quality of life and death.
There are currently few options to treat persons with GVHD, a critically underserved market. The data from our previous clinical study evaluating CBD in the treatment of acute GVHD were exceptional and unprecedented. Ten patients with acute (Grade 3-4) GVHD who were refractory to standard treatment with high-dose steroids were administered daily doses of CBD for up to three months. Nine of the ten patients responded to treatment, with seven achieving complete remissions, and with two achieving near-complete responses. Based on these very positive data, we believe that our CBD product may provide a major advance in the treatment of this disease.
Similarly, if we can develop a safe and effective way to prevent GVHD, this would also be a major advancement. It is estimated that up to 50% of patients who undergo a bone marrow transplant from a fully matched sibling donor, and up to 70% of patients who undergo a bone marrow transplant from an unrelated donor, will develop some level of GVHD. Data from our previous clinical studies evaluating CBD in the prevention of acute GVHD were positive and highly encouraging. In a published study of 48 patients who received daily doses of CBD for 7 days prior to the bone marrow transplant procedure and for 30 days after, the results were as follows:
- No patients developed acute GVHD while being treated with CBD
- The rates of grades 2-4 acute GVHD by day 100 were 12.1%, compared with 46% in 101 historical control subjects given standard GVHD prophylaxis
- CBD was found to be safe and well tolerated
Currently, how far has Kalytera progressed in its clinical trials of its CBD-based treatment for GVHD?
We are currently working with the FDA Division of Hematology Projects to define the protocol design that will permit us to advance CBD into a pivotal registration study in the treatment of acute GVHD. The Phase 3 pivotal registration study will enroll approximately 135 patients and will assess the safety and efficacy of multiple doses of CBD for the treatment of acute GVHD. The study will be a multicenter, multinational, placebo controlled, randomized clinical trial, for the evaluation of the safety and efficacy of CBD for the treatment of Grade 3-4 acute GVHD. The study will be conducted in up to 20 sites in Australia, Israel, The United Kingdom and the U.S. The Principal Investigator of the study is Edmund Waller, M.D., PhD., Professor, Hematology and Medical Oncology, Medicine, and Pathology at Emory University School of Medicine, and Director, Division of Stem Cell Transplantation and Immunotherapy at Winship Cancer Institute of Emory University.
What is the economic potential of a breakthrough treatment for this medical condition?
The commercial opportunity for our CBD products in the treatment and prevention of GVHD is large. According to the January 2018 Market Forecast Report by DelveInsight Perspective, projected annual sales in the 7 major markets market (the U.S., Germany, France, Italy, Spain, the U.K. and Japan) is estimated to be more than USD $408 million in 2018, and could grow to approximately USD $1.3 billion by 2027.
Does the Company have a corporate partner in these clinical trials?
We do not have a corporate partner for our GVHD program. GVHD is an orphan disease, and the number of patients required for the clinical studies is not large. Therefore the clinical studies are not as expensive as they would be in a disease setting such as pain, where there are any patients. Because the costs of clinical studies are not large, we believe that we will be able to raise sufficient capital to fund these studies without a corporate partner. This will enable us to retain the entire value of the program. Later, after the data are submitted for approval, we will probably seek marketing partners for jurisdictions outside North America. Because GVHD patients are primarily found at a relatively small number of centers of excellence in the U.S. and Canada, we believe we may be able to commercialize the product in the North American market without the need for a marketing partner there.
What are the next steps in advancing these clinical trials?
The next step in our program in prevention of GVHD would be to complete the ongoing Phase 2 clinical study later this year, and then advance the program into Phase 3 testing in 1H next year.
The next step in our program in treatment of GVHD will be to work with the FDA to advance CBD into a Phase 3 pivotal registration study, which we would like to start later this year.
With drug development costs exploding, more and more often the large-cap drug pharmaceutical companies are seeking to buy newly-licensed drugs to replenish their depleted pipelines. What is the significance of this for junior biopharma companies like Kalytera?
The significance of this dynamic between biopharma companies and big-pharma companies is that the big pharma companies rely on the smaller biopharma companies to de-risk new drug products by advancing them through Phase 1 and Phase 2 testing. After successful completion of Phase 2, there will be data demonstrating that the drug is safe, and there will be data demonstrating what the appropriate dose will be, and, most importantly, there will also be preliminary data indicating that the drug works. The big pharma companies continually surveil the field, looking for promising drug candidates that have successfully completed Phase 1 and Phase 2 studies, that can be bought or licensed from the biopharma companies. We have a new program to develop a proprietary CBD analogue for the treatment of pain. Our strategy for this program is a build to sell strategy. We don’t want to invest the time and money that would be required to take this product all the way through Phase 3 testing. Rather, we will advance this product through Phase 1 and Phase 2, and, if our data are positive, we will seek to out-license or sell this product to a big pharma partner that will have the ability to fund the costly Phase 3 studies and commercialize the product on a global basis.
What other cannabis-based research is the Company currently focused on in its own R&D pipeline?
In addition to our programs in treatment and prevention of GVHD, we have also recently announced the initiation of our program to develop a novel, proprietary CBD analogue for the treatment of acute and chronic pain. This novel compound is designed to provide effective pain reduction, without the risks of addiction or respiratory suppression that exist with opioid analgesics. We have also found a way to make this compound water soluble, which will allow for treatment of acute pain in in-patient settings, such as childbirth, short surgical procedures, and post-operative pain care. As discussed above, our strategy for this program will be to evaluate this compound through Phase 1 and Phase 2 clinical testing, and then seek to out-license or sell this program to a multinational pharmaceutical company. If successful, the commercial opportunity for this program could be very significant.
Over the long term, please describe your vision for Kalytera Therapeutics five years from now.
We believe that CBD and CBD analogues have the potential to be among the most important and versatile pharmaceutical compounds for years to come, and Kalytera is well positioned to become a global leader in this exciting area of research. We have a highly accomplished team of managers and consultants with world-class scientific, medical and pharmaceutical industry experience. Our goal for the next five years is to utilize our skills, knowledge and experience to bring the extraordinary promise of CBD pharmaceuticals to the market for the benefit of patients, as well as the stockholders who are supporting our work.
kalytera.co
FULL DISCLOSURE: Kalytera Therapeutics Inc. is a paid client of Stockhouse Publishing.