Xonovo Inc. and the Oklahoma Medical Research Foundation (OMRF) today announced that they entered into an Exclusive Worldwide Licensing Agreement for intellectual property rights related to small molecule synthetic derivatives of the natural brain metabolite Lanthionine Ketimine (LK), developed at OMRF. The lead molecule - XN-001 – has shown promising results in treating multiple neurodegenerative diseases in animal models.
XN-001 has demonstrated encouraging effects in tests on mouse models of amyotrophic lateral sclerosis (ALS), stroke, and Alzheimer's disease (AD) across several study centers in the USA. Specifically, XN-001 has been shown to treat all three hallmarks of AD in experimental triple transgenic mice by reducing amyloid beta peptide (Aβ) burden, reducing phospho-tau (a component of Alzheimer's disease-associated neurofibrillary tangles), and relieving cognitive decline. These results have been published in multiple peer-reviewed journals. XN-001 is being tested in other preclinical models of pediatric brain disorders including Batten disease.
XN-001 affects the root cause of a wide range of neurodegenerative diseases. Its novel Mechanism of Action (MOA) is based on targeting a protein called CRMP2 to improve both axonal transport and autophagy.
"This agreement underscores our commitment to pursue treatments for AD patients, and we hope to successfully complete the pre-clinical phase in 18 months and to commence human studies" said Dr. Rafi Gidron and Dr. Ofir Levi, co-founders of Xonovo.
"We are very encouraged by the quality of the Xonovo team and their industrial experience, and believe that they are best qualified to turn this technology into a drug" said Larry Kennedy, Vice President for Technology Transfer at OMRF.
According to Dr. Kenneth Hensley, the Chief Scientific Officer for Xonovo, "The innovative mechanism of action of XN-001 rests in the drug's unique ability to enhance CRMP2 function which produces the dual benefits of improving the way cells move protein cargo packages, and also improves the efficiency of autophagy, a natural cell recycling system." The combination of these actions could simultaneously diminish the production of toxic Aβ, and make neurons more resistant to Ab stress. Dr. Hensley notes that CRMP2 is trapped within AD neuron tangles, thus diminishing the cell's functional pool of this important protein. "XN-001 likely takes advantage of the brain's own natural defense mechanisms to increase the efficiency of the remaining CRMP2 supply", Hensley said.
AD is the most common form of dementia, and the fourth most common cause of death in the developed world. To date, the disease has no cure. More than 30 million people are currently affected by AD worldwide. In the US alone, the direct cost of care in 2012 was $203B, growing at an alarming rate toward an estimated direct cost of $1.2T in 2050.
At the current time there are 5 approved drugs for AD which are described as having "low benefit", merely treating the symptoms of the disease with limited effect. The existing drugs are effective for about 6-12 months for only half of the treated patients. None of the drugs are indicated to slow or halt the progression of the disease.
Xonovo is a Delaware startup company founded in 2013 by Dr. Kenneth Hensley, the inventor of the XoNovo's technology, Dr. Ofir Levi, - an entrepreneur with over 10 years' experience in the life science arena. and Dr. Rafi Gidron, a serial entrepreneur and chairman of Israel Brain Technologies.
OMRF (omrf.org) is an independent, nonprofit biomedical research institute dedicated to understanding and developing more effective treatments for human diseases. Its scientists focus on such critical research areas as cancer, lupus and cardiovascular disease.
OMRF Public Affairs Specialist