Santhera Pharmaceuticals Holding AG Santhera’s FJORD Phase IIb Study Demonstrates Efficacy of JP-1730/Fipamezole for the Treatment of Dyskinesia in Parkinson’s Disease Liestal, Switzerland, June 30, 2009 – Santhera Pharmaceuticals (SIX: SANN), a Swiss specialty pharmaceutical company focused on neuromuscular diseases, announced today positive data from its Phase IIb study (FJORD). The key finding of the trial is that JP-1730/ fipamezole reduces levodopa-induced dyskinesia in Parkinson’s patients. This beneficial effect was not associated with a worsening of Parkinsonian features of the disease. The data additionally suggest that JP-1730/fipamezole reduces “off time” and improves cognitive function as well as clinical global impression in dyskinesia. The compound was found to be safe and well tolerated.
The FJORD study was a double-blind, randomized, placebo-controlled, multiple dose-escalating
study of the safety, tolerability and efficacy of JP-1730/fipamezole in the treatment of levodopa-
induced dyskinesia in Parkinson’s Disease patients. The study investigated the efficacy of three
doses (30, 60 and 90 mg) compared to placebo over a treatment period of 28 days in 179 patients
enrolled at a total of 33 sites in the US (26) and India (7). The primary study endpoint was the
suppression of levodopa-induced dyskinesia as measured by the Levodopa Induced Dyskinesia
Scale (LIDS) [1]. The LIDS is a modified version of the Abnormal Involuntary Movement Scale, an
evaluation tool widely used in clinical research.
Webcast / Teleconference
At 15.00 CET / 14.00 UKT / 09.00 EST on June 30, 2009, Santhera’s management will discuss the
results of the FJORD study. Anyone interested in participating may join either the webcast on www.santhera.com/webcast or the teleconference(ID: 17327356) using one of the following
United Kingdom / International +44 (0) 1452 555 566
The webcast will be available for playback one hour after the presentation ends.
Santhera’s FJORD Phase IIb Study Demonstrates Efficacy of JP-1730/Fipamezole for the Treatment of Dyskinesia in Parkinson’s Disease
Ongoing medical and scientific oversight of the FJORD study was provided by a Scientific
Committee comprising a subgroup of the participating US Parkinson’s specialists and chaired by
Professor Peter LeWitt, MD, Departments of Neurology, Henry Ford Hospital & Wayne State
University School of Medicine, Detroit, Michigan (USA), and Principal Investigator of the FJORD
A pre-specified secondary analysis of 116 patients enrolled at the 26 participating US centers
showed that at the highest dose of JP-1730/fipamezole tested (90mg), patients experienced a
significant reduction in levodopa-induced dyskinesia as compared to placebo (p=0.047, n=29),
assessed by the LIDS. In addition, a highly significant dose dependency (p=0.0066) was
demonstrated. This beneficial effect was not associated with a clinically relevant worsening of
Parkinsonian features of the disease (a secondary endpoint assessed with a standard rating
instrument, the Unified Parkinson’s Disease Rating Scale, UPDRS III). A responder analysis, which
focused on those patients experiencing (i) a reduction in dyskinesia and (ii) no worsening of
Parkinsonism in the investigator’s clinical global impression, showed a higher proportion of
responders in the 90mg group as compared to placebo. Another positive outcome of the US
subgroup analysis was a significant treatment-dose effect in the reduction of hours spent with
diminished mobility (“off time”), as measured by patients using all-day self-assessment ratings.
The Committee carried out the US subgroup analysis due to strong evidence of inhomogeneity
between the US and Indian study populations with respect to several baseline clinical
characteristics differentiating the two patient populations. Of particular concern were the usage
patterns and doses of levodopa and additional clinical features that raised doubts about the
The original primary outcome, based on a change in the LIDS rating in the combined US and Indian
study population (n=179), showed a positive trend for benefit but did not reach significance.
However, the strong evidence of efficacy in the US subgroup, which comprised 65% of the study
population, led to the Committee’s endorsement for further development of JP-1730/fipamezole in
Professor Peter LeWitt commented: “Levodopa-induced dyskinesia are a common and frequently
disabling problem of advanced Parkinson’s Disease for which treatment options are limited. The
FJORD Scientific Committee is encouraged by the US study site findings. Given these results and
the good safety and tolerability observed, the Committee recommends proceeding with the
development of the drug, possibly including higher doses than those previously tested.”
Klaus Schollmeier, Chief Executive Officer of Santhera, commented: “I am pleased about the
outcome of our FJORD study. We fully share the Committee’s view that the FJORD study data
provide a clear path forward for the further development of JP-1730/fipamezole in the treatment of
dyskinesia. We now expect to partner this program to bring this important compound to the market
Santhera’s FJORD Phase IIb Study Demonstrates Efficacy of JP-1730/Fipamezole for the Treatment of Dyskinesia in Parkinson’s Disease
Further detailed analysis of the data from the FJORD Phase IIb study will be presented at upcoming
medical meetings and in peer-reviewed publications.
About Dyskinesia in Parkinson’s Disease
Parkinson’s Disease is the second most common neurodegenerative disease. Doctors prescribe
levodopa and other dopaminergic compounds as standard therapy. Over time, as the disease
progresses, the beneficial effects of this medication often diminish and additional movement
disorders can appear (sometimes quite severe). These movement disorders include dyskinesia
which can be described as sudden uncontrollable, often chaotic movements of limbs, face, tongue
and body. These complications derive principally from long-term levodopa use, but there is currently
no alternative to using levodopa or dopamine agonists. It is estimated that approximately 400,000
patients in Europe and North America are affected by troublesome dyskinesia associated with their
About Fipamezole
Fipamezole is an antagonist of the adrenergic alpha-2 receptor and offers a novel and unique mode
of action to treat Dyskinesia in Parkinson’s Disease. The rationale behind the development of
fipamezole is to increase noradrenergic release in certain areas of the brain, resulting in
rebalancing of the distorted brain network and alleviating symptoms of advanced Parkinson’s
Disease such as dyskinesia, motor fluctuations, orthostatic hypotension and cognitive impairment.
In addition, fipamezole is believed to extend the beneficial effects of commonly used levodopa
(prolongs “on-time”) and other dopamine agonists without the negative side effects associated with
these treatments. JP-1730/fipamezole has been tested previously in a study conducted at the US
National Institutes of Health in Bethesda, Maryland, in which patients with advanced Parkinson’s
disease achieved a reduction in dyskinesia and fluctuations in mobility [2]. Such therapy is expected
to improve the quality of life of Parkinson’s patients.
References
[1] Peter A. LeWitt ea: Validating Rating Performance of a New Rating Scale for Levodopa-Induced
Dyskinesia (LIDS). Poster presented at MDS 13th International Congress of Parkinson’s Disease
[2] Tzvetelina Dimitrova ea: Alpha-2 Adrenergic Antagonist Effects in Parkinson’s Disease. Poster
presented at MDS 13th International Congress of Parkinson’s Disease and Movement Disorders,
Santhera’s FJORD Phase IIb Study Demonstrates Efficacy of JP-1730/Fipamezole for the Treatment of Dyskinesia in Parkinson’s Disease About Santhera
Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on
the discovery, development and commercialization of small-molecule pharmaceutical products for
the treatment of severe neuromuscular diseases, an area of high unmet medical need which
includes many orphan indications with no current therapy. Santhera’s first product, Catena® to treat
Friedreich’s Ataxia, is marketed in Canada. Data of a second pivotal Phase III trial are expected for
the first half of 2010. The drug has also shown efficacy in a clinical trial as a potential treatment for
Duchenne Muscular Dystrophy. For further information, please visit the Company’s Web site
Catena® is a trademark of Santhera Pharmaceuticals. For further information, contact
Klaus Schollmeier, Chief Executive Officer
Thomas Staffelbach, Head Public & Investor Relations
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