A new BDNF inducer enters the pipeline.

UPDATE: Preclinical work with the HD mice has shown that Cogane is not effective.  We have removed this drug from the Pipeline. - Marsha, 3/27/2011

A new BDNF inducer has entered the drug pipeline for Huntington's Disease. Phytopharm, a pharmaceutical firm located in the UK, has entered into an agreement with CHDI Foundation to evaluate the effectiveness of its orally active, neurotrophic factor inducer PYM50028 (Cogane) in a preclinical model of Huntington's Disease.

Cogane boosts levels of two neurotrophic factors, brain derived neurotrophic factor (BDNF) and glial cell derived neurotrophic factor (GDNF). Neurotrophic factors can promote the survival of neurons. Phytopharm is conducting a Phase I (safety and tolerability) trial in Parkinson's patients as well as in healthy volunteers. The company expects to have results available by the end of the year.

Since levels of BDNF are known to be reduced in the brains of HD patients and its reduction has been linked to pathology, CHDI Foundation is interested in Cogane's potential as a treatment for Huntington's Disease.

Cogane will be tested in the R6/2 mouse model and possibly others. If successful, CHDI and Phytopharm will move into clinical trials, first testing for symptom relief and then for neuroprotection. CHDI is also interested in testing for elevated levels of BDNF and GDNF to make sure the targeted action has been accomplished.

Dr. Robert Pacifici, Chief Scientific Officer at CHDI, is excited about the potential of Cogane. "Thanks to a lot of work that has been done throughout the HD community, both BDNF and GDNF along with their cognate receptors, are clearly well validated targets for therapeutic intervention in Huntington's Disease," said Dr. Pacifici. "We are excited at the possibility that the compounds from Phytopharm will allow us to conduct proof of concept experiments in rodent models and determine whether or not pharmacological intervention is indeed beneficial and if so, whether or not modulating levels of these neurotrophic factors will translate into a benefit for HD patients."


Phytopharm press release

Interview with Drs. Robert Pacifici, Richard Morse, and Larry Park of CHDI Foundation.

Marsha L. Miller, Ph.D.
Phytopharm enters an agreement with CHDI to evaluate the potential efficacy of Cogane in the treatment of Huntington's Disease


press release:

Godmanchester, Cambridge, UK, (23 July, 2009) Phytopharm today announces that it has entered into an agreement with CHDI Foundation, Inc., in the U.S.A. to evaluate the efficacy of its orally active neurotrophic factor inducer PYM50028 (Cogane) in a preclinical model of Huntington's Disease (HD).

CHDI will fund the testing of Cogane in its network of industrial contract research organisations, employing its standardised criteria for the rigorous evaluation of novel therapeutic approaches for HD treatment. The testing will begin immediately and is expected to be completed in Q1 2010.

In preclinical models, Cogane increases the body's own production of a group of proteins called neurotrophic factors. One of these factors, BDNF, is known to be decreased in the brains of HD patients. Increasing the brain level of BDNF has been postulated as a potential treatment for HD. However, since BDNF is a protein it cannot be given orally (in pill or liquid form) because it is degraded in the stomach and intestine, and also does not readily cross the blood brain barrier. Cogane, which can be taken orally, readily distributes into the brain and stimulates the release of endogenous BDNF in the brain. It therefore has the potential to overcome the technical difficulties associated with exogenous BDNF administration.

Mr. Sandy Morrison, CEO of Phytopharm said, "We are delighted to have entered an agreement with CHDI to explore the potential of Cogane in a preclinical model of HD, a new indication for our lead compound. Huntington's Disease is a area of extremely high unmet medical need and there is currently no treatment to slow the progression or delay the onset of this devastating disease. We look forward to reporting the findings of this study in the first half of 2010. This partnership with CHDI is indicative of Phytopharm's ongoing strategy of partnering with leading charities in order to facilitate the progression of our pharmaceutical pipeline and demonstrates the potential benefit of Cogane in other neurodegenerative diseases."

In April 2009, following approval by the Medical and Healthcare Products Regulatory Agency, Phytopharm commenced a safety, tolerability, and pharmacokinetic (PK) study of Cogane in both healthy volunteers and patients with Parkinson's Disease (PD).

press release