Last updated: Tuesday 24 March 2015
at 2am PST
Prothena
Corporation plc, a late-stage clinical biotechnology company focused on the
discovery, development and commercialization of novel protein immunotherapy
programs, has announced positive results from a Phase 1 single ascending dose
study of PRX002, a monoclonal antibody for the potential treatment of Parkinson's disease and other related synucleinopathies.
PRX002 is the focus of a worldwide collaboration between Prothena and Roche.
PRX002 was safe
and well-tolerated, meeting the primary objective of the study. Further,
results from this study showed that administration of PRX002 leads to mean
reduction of free serum alpha-synuclein levels of up to 96%. These overall
results were highly statistically significant (p<0.00001). Reduction of free
serum alpha-synuclein, a protein potentially involved in the onset and
progression of Parkinson's disease and the target of PRX002, was shown to be
robust, rapid and dose-dependent after just a single dose.
"There is
genetic and pathological evidence that supports a causal role of
alpha-synuclein in Parkinson's disease," said Todd Sherer, PhD, CEO of the
Michael J. Fox Foundation for Parkinson's Research. "We applaud Prothena
and Roche for their pioneering work in developing a potentially disease-modifying
therapy for this progressive neurodegenerative disease that affects millions
worldwide."
The Phase 1
double-blind, placebo-controlled, single ascending dose study enrolled 40
healthy volunteers. All volunteers enrolled were randomized 3:1 into five
escalating dose cohorts (0.3 mg/kg, 1 mg/kg, 3 mg/kg, 10 mg/kg or 30 mg/kg) to
receive either PRX002 or placebo. No hypersensitivity reactions or drug-related
serious adverse events were reported. PRX002 demonstrated favorable
pharmacokinetic properties, supporting the current dosing frequency in the
on-going Phase 1 multiple ascending dose study in patients with Parkinson's
disease. There were no treatment emergent adverse events (TEAEs) in greater
than 10% of subjects. The only TEAEs in greater than 5% of subjects were vessel
puncture site pain, headache and viral infection. All PRX002-related adverse
events were mild and no dose limiting toxicities were observed.
"We are extremely
pleased with the results of the Phase 1 single ascending dose study as the mean
reduction of free serum alpha-synuclein of up to 96% demonstrates the
pharmacodynamic effects of PRX002," commented Gene Kinney, PhD, Chief
Scientific Officer and Head of Research and Development at Prothena.
"Importantly and for the first time in humans, we demonstrated that this
robust, rapid and dose-dependent reduction of free serum alpha-synuclein was
safe and well-tolerated. Thus, this approach may translate into a clinically
meaningful delay or reversal of disease progression in patients with
Parkinson's disease. We look forward to building upon these data with results
from the on-going, multiple ascending dose study in patients with Parkinson's
disease expected in the first half of 2016, where we will also be measuring
levels of PRX002 in the cerebrospinal fluid and assessing additional
biochemical, imaging and clinical biomarker endpoints. Separately, we are
excited to co-host a symposium with Roche on March 21 at the 12th International
Conference on Alzheimer's and Parkinson's Diseases and Related Neurological
Disorders (AD/PD™ 2015) to continue to raise awareness of the role of
alpha-synuclein as a target for Parkinson's disease."
"The results
of the PRX002 study exemplify Prothena's deep domain expertise to develop novel
disease-modifying protein immunotherapies with unique specificities to their
targets," stated Dale Schenk, PhD, President and Chief Executive Officer
of Prothena. "Prothena's consistent ability to develop targeted potential
therapeutics has resulted in a strong and promising pipeline to transform
patient's lives, with NEOD001 in Phase 3 clinical studies for the treatment of
AL amyloidosis, PRX002 continuing in a Phase 1 multiple ascending dose study in
patients with Parkinson's disease and PRX003 ready to begin clinical studies
for the treatment of psoriasis and potentially other inflammatory diseases."
In December 2013,
Prothena and Roche entered into a worldwide collaboration to develop and
commercialize antibodies that target alpha-synuclein, including PRX002. To
date, Prothena has received $45 million of the potential $600 million in total
milestones through its collaboration with Roche. Prothena has an option to
co-promote PRX002 in the U.S., where the companies share all profits, as well
as development and commercialization costs, on a 30/70 basis (30% Prothena and
70% Roche). Outside the U.S., Roche will have sole responsibility for
developing and commercializing PRX002 and will pay Prothena up to double-digit
royalties on net sales.
Adapted by MNT
from original media release
http://www.medicalnewstoday.com/releases/291315.php?tw
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