British pharmaceutical company Phytopharm today announced that their Parkinson’s disease (PD) drug candidate Cogane “had no beneficial effects on patients symptoms measured" in a phase 2 clinical study.
Cogane was designed to promote the release of two trophic factors in the brain called GDNF and BDNF, proteins that researchers believe could restore life to some of the brain cells that die in PD. Trophic factors have shown good potential in Parkinson’s, but most approaches to getting them into the brain have involved invasive surgery. Cogane was a rare, orally-administered treatment designed to produce the same effect. Unfortunately, today’s results are negative.
According to Reuters, Phytopharm has now halted all of its research and development investment. An analyst interviewed by the news agency called the results a conclusive failure of Cogane.
MJFF previously funded Phytopharm for a pre-clinical study to evaluate the potentially effective dosing of the drug, work that readied Cogane for the clinical testing that followed. Although the MJFF study and other prior studies had suggested some neuroprotective potential for PD, results were not completely definitive. This new trial suggests that the potential wasn’t realized in humans. Often, results from pre-clinical testing don’t translate into the clinic; it’s one of the major challenges of drug development, and a reason that the Foundation continues to push hard to find biomarkers of PD that could better measure the efficacy of potential drugs in humans. And to do so faster, and for less money.
MJFF continues to support other trophic factors work, and the Foundation continues to believe in their potential as a next-generation therapy for people with PD. MJFF has funded and advised San Diego-based biotech Ceregene on three trophic factor clinical trials since 2005. Ceregene is currently in phase 2 testing for the trophic factor neurturin. Critical results from this trial are expected sometime this spring.