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2016-02-09

Prothena Corporation plc: Data From Prothena's Ongoing Phase 1/2 Clinical Trial of NEOD001 in AL Amyloidosis Published in the Journal of Clinical Oncology

Interim Results Previously Presented at Meetings of the American Society for
Clinical Oncology and the European Hematology Association

DUBLIN, Feb. 09, 2016 (GLOBE NEWSWIRE) -- Prothena Corporation plc
(NASDAQ:PRTA), a late-stage clinical biotechnology company focused on the
discovery, development and commercialization of novel protein
immunotherapies, today announced that interim data from its Phase 1/2
clinical study of NEOD001 in patients with AL Amyloidosis and persistent
organ dysfunction have been published in the Journal of Clinical Oncology.
The data appeared online in an article titled "First-in-Human Phase 1/2 Study
of NEOD001 in Patients With Light Chain Amyloidosis and Persistent Organ
Dysfunction."

The published data, which were previously presented in oral presentations at
the American Society for Clinical Oncology Annual Meeting and the European
Hematology Association Congress, showed that monthly infusions of NEOD001
were safe and well tolerated and that patients showed improvements in
functional biomarkers predictive of improvement of disease and survival. Data
from the ongoing expansion cohort of this study are expected in the second
quarter of 2016.

"The published data provide strong support for the continued development of
NEOD001 in AL amyloidosis, and we hope that through Prothena's ongoing
clinical development efforts, we may one day soon have an approved
immunotherapy treatment that positively impacts organ function for this
difficult-to-treat and deadly disease," said Morie A. Gertz, M.D., of the
Mayo Clinic, principal investigator of the study and lead author of the
manuscript.

A total of 27 patients with AL amyloidosis and persistent organ dysfunction
were enrolled in seven dose cohorts in the dose escalation portion of the
study. Cardiac and renal responses were exploratory endpoints.

Of the 14 cardiac-evaluable patients, 57 percent met the criteria for cardiac
response, which compares favorably to the expected cardiac best response rate
of 26.5 percent from historical data in patients treated solely with
off-label standard of care (Comenzo, et al., Leukemia. 2012; 26:2317-2325).
Forty-three percent of patients had stable disease. In cardiac responders,
the median NT-proBNP, a clinical biomarker used to predict disease
progression and mortality, decreased from 1768.5 pg/mL at baseline to 1054
pg/mL at the best response assessment. The mean decrease from baseline was
890 pg/mL, which represented a 48 percent reduction and was statistically
significant (P<0.008). NT-proBNP decline for responders significantly
correlated with increased number of NEOD001 infusions.

Of the 15 renal-evaluable patients, 60 percent met the criteria for renal
response which compares favorably to the expected renal best response of
approximately 24 percent from historical data in patients treated solely with
off-label standard of care (Palladini, et al., Blood. 2014 124:2325-2332).
Forty percent of patients had stable disease. Increased levels of proteinuria
and decreased eGFR predict faster progression to dialysis whereas decreased
levels of proteinuria in the absence of eGFR worsening predict delayed time
to dialysis. In the responders, the median proteinuria value decreased from
4834 mg/24 hours at baseline to 1647 mg/24 hours at the best response
assessment. The mean decrease from baseline was -2647 mg/24 hours, which
represented a 62 percent decrease and was statistically significant
(P<0.004).

"The positive results from our Phase 1/2 study of NEOD001 in patients with AL
amyloidosis suggest that NEOD001 may effectively recruit the immune system to
clear the circulating soluble and deposited insoluble proteins that cause
organ dysfunction in this progressive and life-threatening condition," said
Dale Schenk, Ph.D., President and Chief Executive Officer of Prothena. "Based
on these data, Prothena has initiated The VITAL Amyloidosis Study, a global
registration study for NEOD001, and is planning to initiate the PRONTO study,
which will evaluate cardiac response as assessed by changes in NT-proBNP, a
cardiac functional biomarker that has been shown to be highly predictive of
survival in patients with AL amyloidosis."

Monthly infusions of NEOD001 were safe and well tolerated. There were no
drug-related serious adverse events (AEs), discontinuations due to
drug-related AEs, dose-limiting toxicities or antidrug antibodies reported in
the study. The most frequently reported AEs were fatigue, upper respiratory
tract infection, cough, and dyspnea. The recommended dose level was 24 mg/kg
and the pharmacokinetic profile of the drug support intravenous dosing once
every 28 days. As previously reported, there was one patient death that was
determined to be unrelated to NEOD001.

About NEOD001

NEOD001 is a humanized monoclonal antibody that specifically targets the
circulating soluble amyloid and deposited insoluble amyloid that accumulates
in both the AL and AA forms of amyloidosis. NEOD001 received Fast Track
designation from the FDA in December 2014. There are three clinical trials
for NEOD001 in patients with AL amyloidosis. The multi-center Phase 1/2
clinical trial is evaluating the safety, tolerability, pharmacokinetics and
immunogenicity of NEOD001 in patients with AL amyloidosis and persistent
organ dysfunction. The trial is also evaluating exploratory biomarkers for
cardiac and renal function as well as peripheral neuropathy. The VITAL
Amyloidosis Study, a double-blind, placebo-controlled, global Phase 3
registrational trial, will evaluate NEOD001 in newly-diagnosed,
treatment-naïve patients with AL amyloidosis, and will assess a composite of
all-cause mortality or cardiac hospitalizations in addition to biomarker,
functional and quality of life endpoints. The PRONTO trial, a double-blind,
placebo-controlled, global Phase 2b registration-directed trial, will
evaluate NEOD001 in previously-treated patients with AL amyloidosis and
persistent cardiac dysfunction, and will assess NT-proBNP best response over
12 months in addition to other biomarker, functional, and quality of life
endpoints. More information on the Phase 1/2 trial, The VITAL Amyloidosis
Study, and the PRONTO trial is available at www.clinicaltrials.gov.

About AL Amyloidosis

Systemic amyloidoses are a complex group of progressive diseases caused by
tissue deposition of misfolded proteins that result in progressive organ
damage. The most common type, AL amyloidosis or primary amyloidosis, involves
a hematological disorder caused by plasma cells that produce misfolded
immunoglobulin light chain resulting in deposits of abnormal AL protein
(amyloid) in the tissues and organs of individuals with this disease. There
are no approved treatments for AL amyloidosis, and none that directly target
potentially toxic forms of the AL protein. AL amyloidosis is a rare disorder
and it is estimated that about 30,000 to 45,000 patients in the U.S. and
Europe suffer from this disease. Both the causes and origins of AL
amyloidosis remain poorly understood. For more information on AL amyloidosis,
please visit the websites of the Amyloidosis Support Group and the
Amyloidosis Foundation.

About Prothena

Prothena Corporation plc is a late-stage clinical biotechnology company
focused on the discovery, development and commercialization of novel protein
immunotherapies 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 potential clinical benefits of NEOD001;
the initiation of the PRONTO clinical trial; the PRONTO trial design; and the
expected timing for announcing data from the expansion cohort of the Phase
1/2 clinical trial. 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 13, 2015 and our subsequent Quarterly Reports on
Form 10-Q filed with the SEC. 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.

Contacts

Investors: Tran Nguyen, CFO
650-837-8535, IR@prothena.com

Media: Ellen Rose, Head of Communications
650-922-2405, ellen.rose@prothena.com

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This announcement is distributed by NASDAQ OMX Corporate Solutions on behalf of NASDAQ OMX Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Prothena Corporation plc via Globenewswire

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