Prothena Reports Robust Reduction of Free Serum Alpha-Synuclein of up to 96% After Single Dose of PRX002, a Novel Protein Immunotherapy for Parkinson's Disease
(Thomson Reuters ONE) -
* All Doses of PRX002 Found to be Safe and Well Tolerated, Meeting Primary
Objective of Phase 1 Single Ascending Dose Study
* Results of Study Demonstrate Rapid and Dose-Dependent Reduction of Free
Serum Alpha-Synuclein, Potential Disease-Causing Protein in Parkinson's
Disease
DUBLIN, Ireland, March 19, 2015 (GLOBE NEWSWIRE) -- Prothena Corporation plc
(Nasdaq:PRTA), a late-stage clinical biotechnology company focused on the
discovery, development and commercialization of novel protein immunotherapy
programs, today 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 12(th) International Conference on Alzheimer's and
Parkinson's Diseases and Related Neurological Disorders (AD/PD(TM) 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.
Roche and Prothena to Co-Host Symposium During AD/PD(TM) 2015
Leading researchers in the field of Parkinson's disease will present during a
Roche and Prothena co-hosted symposium entitled 'Alpha-Synuclein as a Target in
Parkinson's Disease,' during the 12(th) International Conference on Alzheimer's
and Parkinson's Diseases and Related Neurological Disorders (AD/PD(TM) 2015) in
Nice, France. The symposium will be held on March 21, 2015 at 9:15 a.m. local
time, and will feature leading experts from Europe and the United States
including Wilma van de Berg, PhD, of the VU University Medical Center in the
Netherlands; Patrik Brundin, MD, PhD, of the Van Andel Research Institute in
Michigan; Eliezer Masliah, MD, of the University of California, San Diego; Mark
Frasier, PhD, of the Michael J. Fox Foundation in New York; and, Wagner Zago,
PhD, of Prothena Biosciences Inc in South San Francisco.
About Alpha-Synuclein
Alpha-synuclein is a protein found in neurons and is a major component of
pathology that characterizes several neurodegenerative disorders including
Parkinson's disease, dementia with Lewy bodies, and multiple system atrophy,
which collectively are termed synucleinopathies. While the normal function of
synuclein is not well understood, the protein generally occurs in a soluble
form. In synucleinopathies, the synuclein protein can misfold and aggregate to
form soluble aggregates and insoluble fibrils that contribute to the pathology
of the disease. There is also increasing evidence that this disease-causing
synuclein can be propagated and transmitted from neuron to neuron, resulting in
an infection-like spread of neuronal death. Recent studies in cellular and
animal models suggest that the spread of synuclein-associated neurodegeneration
can be disrupted by targeting aberrant forms of synuclein.
About PRX002
PRX002, a monoclonal antibody targeting alpha-synuclein, is the focus of a
license agreement between Prothena and Roche. The companies are evaluating
PRX002 in a multiple ascending dose study in patients with Parkinson's disease,
with results expected in the first half of 2016. PRX002 is designed to slow or
reduce the progressive neurodegeneration associated with synuclein misfolding
and/or the cell-to-cell transmission of the pathogenic forms of synuclein in
Parkinson's disease and other synucleinopathies. Prior to initiating clinical
trials, Prothena demonstrated the efficacy of PRX002 in various cellular and
animal models of synuclein-related disease. In transgenic mouse models of
Parkinson's disease, passive immunization with 9E4, the murine version of
PRX002, reduced the appearance of synuclein pathology, protected synapses and
improved performance by the mice in behavioral testing. For more information on
the ongoing multiple ascending dose study, please visit www.clinicaltrials.gov
and search identifier NCT02157714.
About Parkinson's Disease
Parkinson's disease is a degenerative disorder of the central nervous system
that affects one in 100 people over age 60, and after Alzheimer's disease is the
second most common neurodegenerative disorder. There are an estimated seven to
ten million patients living with Parkinson's disease worldwide. Current
treatments for Parkinson's disease are only effective at managing the early
motor symptoms of the disease, mainly through the use of levodopa and dopamine
agonists. As the disease progresses and dopaminergic neurons continue to be
lost, these drugs eventually become less effective at treating the symptoms. In
contrast, PRX002 targets disease-causing alpha-synuclein, and may slow or reduce
the neurodegeneration associated with aberrant forms of alpha-synuclein.
About Prothena
Prothena Corporation plc is a late-stage clinical biotechnology company focused
on the discovery, development and commercialization of novel protein
immunotherapy programs for the potential treatment of diseases that involve
amyloid or cell adhesion. The Company is developing antibody-based product
candidates that target a number of potential indications including AL
amyloidosis (NEOD001), Parkinson's disease and other related synucleinopathies
(PRX002) and psoriasis and other inflammatory diseases (PRX003).
For more information, please visit the Company's web site at www.prothena.com.
Forward-Looking Statements
This press release contains forward-looking statements. These statements relate
to, among other things, the timing of reporting data from our Phase 1 multiple
ascending dose study for PRX002; the possible clinical benefit of PRX002 in
patients with Parkinson's disease; the potential of Prothena's development
pipeline; and the timing of initiating clinical trials for PRX003. These
statements are based on estimates, projections and assumptions that may prove
not to be accurate, and actual results could differ materially from those
anticipated due to known and unknown risks, uncertainties and other factors,
including but not limited to the risks, uncertainties and other factors
described in the "Risk Factors" sections of our Annual Report on Form 10-K filed
with the Securities and Exchange Commission (SEC) on March 12, 2015. Prothena
undertakes no obligation to update publicly any forward-looking statements
contained in this press release as a result of new information, future events or
changes in Prothena's expectations.
CONTACT: Investors: Tran Nguyen, CFO
650-837-8535, IR(at)prothena.com
Media: Angela Bitting
925-202-6211, angela.bitting(at)prothena.com
This announcement is distributed by GlobeNewswire on behalf of
GlobeNewswire clients. The owner of this announcement warrants that:
(i) the releases contained herein are protected by copyright and
other applicable laws; and
(ii) they are solely responsible for the content, accuracy and
originality of the information contained therein.
Source: Prothena Corporation via GlobeNewswire
[HUG#1904969]
Unternehmensinformation / Kurzprofil:
Datum: 19.03.2015 - 21:05 Uhr
Sprache: Deutsch
News-ID 379951
Anzahl Zeichen: 12348
contact information:
Town:
DUBLIN
Kategorie:
Business News
Diese Pressemitteilung wurde bisher 176 mal aufgerufen.
Die Pressemitteilung mit dem Titel:
"Prothena Reports Robust Reduction of Free Serum Alpha-Synuclein of up to 96% After Single Dose of PRX002, a Novel Protein Immunotherapy for Parkinson's Disease"
steht unter der journalistisch-redaktionellen Verantwortung von
Prothena Corporation (Nachricht senden)
Beachten Sie bitte die weiteren Informationen zum Haftungsauschluß (gemäß TMG - TeleMedianGesetz) und dem Datenschutz (gemäß der DSGVO).
