Kannalife Sciences Discusses Orphan Drug Opportunity For New ‘Super CBD’ Molecule
Kannalife Sciences, Inc. has announced its intentions to pursue an orphan drug opportunity for a new ‘Super CBD’ molecule listed as KLS-13019. This new molecule is a Novel Functionalized 1,3-Benzene Diol for the treatment of Hepatic Encephalopathy (HE). HE is a brain disorder associated with individuals who have developed liver disease. KLS-13019 aims to block negative toxins from reaching the brain, a process that would represent the biggest innovation in the history of HE treatment. It is estimated that 1.5Mil people in the Unites States alone suffer from Hepatic Encephalopathy, with approximately 200,000 of them suffering from a more serious, grade three classification. Such a classification qualifies HE as an orphan (rare) disease. By developing KLS-13019, an orphan drug to treat an orphan disease, Kannalife’s new molecule can be fast tracked through the FDA’s approval process and, if approved, will be granted seven years of exclusivity, in accordance to FDA orphan drug regulations.
Who is KannaLife Sciences, Inc.?
Founded in 2010, Kannalife Sciences, Inc. is a phyto-medical company involved in the research and development of novel therapeutic agents designed to be neuroprotectants and immuno-modulators. Kannalife began their research looking to work with cannabinoids to create better and more efficient medical treatments. Kannalife chose to work primarily with CBD and, after considering the wide range of effective applications, they chose to primarily focus on the development of brain disease treatments. Kannalife is currently conducting research and development at the Pennsylvania Biotechnology Center in Doylestown, PA, for target drug candidates to treat Hepatic Encephalopathy (“HE”) and Chronic Traumatic Encephalopathy (“CTE”). HE and CTE are oxidative stress related diseases that affect cognitive and behavioral functions.
What is Hepatic Encephalopathy?
Hepatic encephalopathy, or HE, is a brain disorder that develops in some individuals with liver disease. HE is characterized by personality changes, intellectual impairment, and a depressed level of consciousness. The development of hepatic encephalopathy is explained, to some extent, by the effect of neurotoxic substances, which occurs in the setting of cirrhosis and portal hypertension. Kannalife’s newly developed, CBD-like molecule aims to stop toxic substances like these from ever reaching the brain. Successful application could mean a decrease in overall drug consumption, in addition to a healthier intestinal system, as patients suffering from HE often take ten or more prescription pills per day.
“Our pre-clinical studies using cannabidiol (CBD) as a positive control, side-by-side with KLS-13019 and other cannabidiol-like molecules of this class, reveals dramatic potency and safety improvements for this novel chemotype. Kannalife’s patent application further describes the novel chemotype useful as neuroprotective agents in the treatment of diseases associated with hepatic encephalopathy. KLS-13019 demonstrated improved oral bioavailability and CNS penetration relative to CBD and bodes well for its ability to penetrate the blood brain barrier, ameliorate brain toxicities associated with HE, and address unmet medical needs”. – Dean Petkanas, CEO of Kannalife.
The Super CBD Goal
KLS-13019 is the official designation that has been given to this newly developed molecule. The molecule will be renamed once it has moved further down the process to becoming an FDA approved drug. This ‘Super CBD’ is ~295 times less toxic than its naturally occurring CBD counterpart. In effect, KLS-13019 blocks unwanted toxins, that directly lead to HE, from reaching the brain. Other medications designed to help treat HE encourage the patient to filter out toxins through their stomach. The overall goal of this new ‘CBD-like’ molecule is to provide a much safer and reliable method to combat HE. Currently, a patient who suffers from HE could take as many as 10 pills in a ‘cocktail’ like treatment, with each pill containing its own side effects and impact on the intestinal system. If successful, this new molecule could replace the majority of pills used to properly treat HE, creating a healthier patient.
“In 2010 we believed there was an opportunity to build upon the promising preclinical findings with CBD in HE and design a novel cannabidiol-like therapeutic agent that would have benefits in terms of therapeutic index and delivery by the oral route. Developing our New Chemical Entity via the orphan status pathway will provide advantages in terms of reduced development cost and greater exclusivity in the marketplace.” – Dean Petkanas, CEO of Kannalife.
What is an Orphan Drug?
The FDA’s new drug application process allows for priority review as it pertains to orphan drugs designed to treat orphan diseases. Priority review is designed to expedite programs on new drug applications for serious conditions. Orphan status grants seven years exclusivity. It is estimated that there are 1.5 million sufferers of hepatic encephalopathy (HE) in the US, of which, approximately 200,000 patients suffer from overt hepatic encephalopathy (Grade 3), representing an orphan drug opportunity for a novel drug in use.
Orphan status allows the FDA to fast track the development and application of new treatments due to the smaller patient population associated with orphan diseases (also known as rare diseases). Once the new drug is approved, a seven year exclusivity period is granted to the discovering entity. Without orphan drug status, the FDA approval process is much longer and, unless a patent is filed, exclusivity is not a factor. Furthermore, because CBD is a natural substance, it is not patentable. By creating a molecule that is ‘CBD-like’, Kannalife has created a patentable product that is ~295 times less toxic than its natural CBD counterpart.
What happens next?
Kannalife Sciences, Inc. is continuing to develop and test KLS-13019 as they work towards FDA approval and the eventual market entrance of this new drug. In addition to the treatment of HE, Kannalife is using CBD to develop a treatment for CTE, a disease that is synonymous with concussions, especially pertaining to contact sports like football, and has been brought to the public’s attention by Dr. Bennett Omalu. Dr. Omalu is on the scientific advisory board at Kannalife and was the first to identify Chronic Traumatic Encephalopathy (CTE) in pro-football players.
The Hepatic Encephalopathy Market
Xifaxan®, produced by Salix Pharmaceuticals, is one of the chemical treatments that Kannalife is aiming to enhance or replace with their new drug. According to a report by Credit Suisse, Xifaxan® sales to treat Hepatic Encephalopathy accounted for roughly 70% of total Salix sales. Those sales are estimated to deliver approximately $900Mil in 2015 with estimates driving towards $1.4Bil by 2018.