Press Releases
Synosia Begins Proof-of-Mechanism Trial For Nitisinone
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
Disclaimer
This communication, and oral statements made with respect to information contained in this communication, expressly or implicitly contains certain forward-looking statements concerning Synosia Therapeutics and its business. Such forward-looking statements include those which express plan, anticipation, intent, contingency, goals, targets or future development and/or otherwise are not statements of historical fact including, but not limited to our plans for our regulatory filings, enrollment and future plans for our clinical trials, progress of and reports of results from clinical studies, clinical development plans and product development activities. The words “potential”, “could” and similar expressions also identify forward-looking statements. These statements are based upon management's current expectations and are subject to risks and uncertainties, known and unknown, which could cause actual results and developments to differ materially from those expressed or implied in such statements. Factors that could affect actual results include risks associated with the possibility that the respective regulatory agencies refuse approval of our applications, outcome of any discussions with such regulatory agencies and unexpected delays in preparation of materials for submission to such respective regulatory agencies as a part of our filings.
Synosia Therapeutics is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise. Actual events could differ materially from those anticipated in the forward-looking statements.
References
- Guttmacher et al. Alzheimer’s Disease and Parkinson’s Disease. New England Journal of Medicine (2003); 348; 1356-64
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