Parkinson's UK & MedGenesis Therapeutix Inc. announce top-line results of Phase 2 GDNF safet

Parkinson's UK and MedGenesis Therapeutix Inc. announced today that the Phase 2 clinical trial evaluating the safety and efficacy of glial cell line-derived neurotrophic factor (GDNF) in Parkinson's disease, delivered via intermittent bilateral intraputamenal infusions using convection enhanced delivery (CED) and conducted at Southmead Hospital by the North Bristol NHS Trust, did not meet the primary efficacy endpoint.

The primary endpoint of the study is the percentage change from baseline in the practically defined OFF-state Unified Parkinson's Disease Rating Scale (UPDRS) motor score (part III) after nine months of double-blind treatment.

The study was funded by Parkinson's UK, with support from The Cure Parkinson's Trust and in association with the North Bristol NHS Trust. Study drug, additional project resources and supplementary funding was provided by MedGenesis Therapeutix, who in turn received some program funding support from the Michael J. Fox Foundation for Parkinson's Research. Renishaw plc manufactured the CED device on behalf of the North Bristol NHS trust and provided additional technical and analytical support. PET scans were acquired at the Wales Research and Diagnostic PET Imaging Centre, Cardiff University and analysed at the PET Imaging Centre at the University of British Columbia. Most importantly, the researchers would like to gratefully acknowledge the contribution of the study participants.


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About GDNF

GDNF is the major neurotrophic factor for dopamine neurons and has an impact on several other relevant neurotransmitter systems. It has established disease-modifying efficacy in all key animal models of Parkinson's disease, having displayed neuroprotection, axonal sprouting, and improved motor function in preclinical research. Previous clinical trials include two open-label Phase I studies (n=15) with positive results that were sustained well beyond the end of treatment, and a placebo-controlled Phase II study (n=34) that did not confirm efficacy. The results of this Phase II study are likely to have been the result of poor delivery of the drug to the target tissue in the putamen, due to the immature state of CED expertise and limited technology available at that time to provide for accurate, precise and verified distribution of the therapeutic agent across a therapeutically relevant fraction of the target structure within the brain. On the basis of the available preclinical and clinical evidence, GDNF offers the potential for the first ever disease-modifying treatment of Parkinson's disease.

About Parkinson's UK

Every hour, someone in the UK is told they have Parkinson's.

It affects 127,000 people in the UK - which is around one in 500 of the population.

Parkinson's is a degenerative neurological condition, for which there currently is no cure. The main symptoms of the condition are tremor, slowness of movement and rigidity.

Parkinson's UK is the UK's leading charity supporting those with the condition. Its mission is to find a cure and improve life for everyone affected by Parkinson's through cutting edge research, information, support and campaigning.

For advice, information and support, visit or call our free, confidential helpline on 0808 800 0303.

About MedGenesis Therapeutix Inc.

MedGenesis is a privately held biopharmaceutical company committed to developing and commercializing innovative therapeutics to provide life-enhancing treatments to patients with serious neurologic diseases, employing convection-enhanced delivery (CED), a minimally invasive technique that provides for targeted, local treatment of serious conditions such as Parkinson's, epilepsy, brain cancer and other severely debilitating diseases of the central nervous system.

Learn more about MedGenesis at: