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Synosia Begins Proof-of-Mechanism Trial For Nitisinone Print E-mail
10 Jun 2008

Compound to be Tested as Treatment for Parkinson’s Disease

Basel, Switzerland | June 10, 2008 | Synosia Therapeutics today announced the start of a proof-of-mechanism, clinical trial to evaluate the efficacy and safety of nitisinone (SYN-118), a potent and selective inhibitor of hydroxyphenylpyruvate dioxygenase (HPPD), as a treatment for Parkinson’s disease.

HPPD is an enzyme in the primary pathway responsible for the catabolism of tyrosine, the precursor of the neurotransmitter dopamine. This novel intervention point in the dopamine pathway offers the potential to induce a sustained increase in dopamine synthesis in the brain of Parkinson’s patients to improve the control of motor symptoms with an improved side effect profile compared to current therapy.

The dose-escalation trial in Parkinson’s patients is being conducted at the Karolinska Institutet in Stockholm using positron emission tomography (PET) and clinical ratings such as the Unified Parkinson’s Disease Rating Scale to evaluate efficacy.

“We know from extensive clinical studies and patient experience with nitisinone that the drug produces pronounced and reliable elevations in the levels of tyrosine, a precursor of dopamine,” said Stephen Bandak, Synosia’s chief medical officer. “We are now evaluating whether this results in increased synthesis of dopamine in the brain of patients with Parkinson’s.”

Synosia’s Chief Executive Officer and President Ian Massey said: “This data-rich, efficient trial will test our hypothesis on the pharmacology of the drug and help to zero in on the right dose for a Phase II efficacy study.” Parkinson’s disease is the second most common neurodegenerative disorder, after Alzheimer’s disease. It affects about one per cent of people aged 65-69 years, rising to up to three per cent of people aged 80 years and older.1

About Nitisinone

Nitisinone was discovered and developed by Syngenta, which has granted licensing rights to Swedish Orphan International (SOI) for orphan indications. SOI markets nitisinone in Europe and the United States for the treatment of hereditary tyrosinemia type 1, under the brand name Orfadin®. The development programme for nitisinone to date has generated over 5,000 years of clinical and market experience.

In 2007, Synosia obtained rights from Syngenta to develop and commercialise nitisinone in all non-orphan indications.
About Synosia Therapeutics Synosia Therapeutics develops and intends to commercialise innovative and clinically differentiated products for unmet medical needs in psychiatry and neurology. The privatelyowned company has six clinical-stage compounds in its pipeline acquired through key partnerships with Novartis, Roche and Syngenta. Two of the compounds are marketed drugs
that will be tested in new indications, extending their reach into neurological and psychiatric diseases with high unmet medical need, including anxiety and Parkinson’s disease. Synosia’s headquarters is in Basel, Switzerland. For more information visit www.synosia.com

SOURCE: Synosia





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