- Net cash used
in operating and investing activities was $41.1 million in the
fourth quarter and $133.9 million for the full year 2016; year-end
cash and restricted cash position of $391 million supports
advancement of clinical pipeline
- Presented
results in an oral presentation at ASH from the Phase 1/2 study of
NEOD001 demonstrating improvement in three organ systems (cardiac,
renal and peripheral nerve) in previously treated patients with AL
amyloidosis
- Reported
top-line results from the Phase 1b study of PRX002 demonstrating
robust antibody central nervous system penetration and rapid, dose-
and time-dependent mean reduction in levels of free serum
alpha-synuclein of up to 97 percent in patients with Parkinson's
disease
- Initiated a
Phase 1b multiple ascending dose, proof-of-biology study of PRX003
in patients with psoriasis, following presentation of Phase 1a
single ascending dose study in healthy volunteers demonstrating
target engagement
- Appointed Gene
G. Kinney, PhD President, Chief Executive Officer and
Director
DUBLIN, Ireland, Feb. 14, 2017
(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 reported financial results for the fourth quarter and full
year 2016. In addition, the Company provided 2017 financial
guidance and an update on its R&D programs.
"In 2016 we were saddened by the
loss of Dale Schenk, PhD, our friend and former CEO, and a true
scientific pioneer," said Gene Kinney, PhD, President and Chief
Executive Officer of Prothena. "As a testament to our team's talent
and commitment, our business continued to gain momentum and 2016
was a year of significant progress where we reported positive data
for each of our three clinical programs. As our pipeline continues
to mature, several key milestones in 2017 and into 2018 keep us on
track towards our goal of delivering novel disease modifying
therapies to patients. For NEOD001 in patients with AL amyloidosis,
we expect to complete enrollment in the PRONTO study during the
next several weeks and in the VITAL study during the second
quarter. Also in 2017, for PRX002/RG7935, we expect to initiate,
with our partners at Roche, a Phase 2 clinical study in patients
with Parkinson's disease. For PRX003 we expect to report full
topline results from a Phase 1b multiple ascending dose,
proof-of-biology study in patients with psoriasis, and for PRX004
we continue to advance our preclinical work toward the start of a
Phase 1 clinical study in patients with ATTR amyloidosis."
Full Year
2016 and Recent Highlights:
NEOD001 is a monoclonal antibody for the
potential treatment of AL amyloidosis:
- Presented positive results from
the Phase 1/2 study of NEOD001 in patients with AL amyloidosis and
persistent organ dysfunction in an oral presentation by Morie A.
Gertz, MD, of Mayo Clinic at the 58th Annual American Society
of Hematology (ASH) meeting. The results demonstrated best response
rates of 53 percent and 64 percent for cardiac- (n=36) and renal-
(n=36) evaluable patients, respectively. Improvement in peripheral
neuropathy was demonstrated by a mean 35 percent (median 23
percent, n=11) decrease in the Neuropathy Impairment Score-Lower
Limb (NIS-LL) as a change from baseline to month 10, leading to an
82 percent response rate. NEOD001 continued to be safe and well
tolerated. A total of 69 patients received 994 doses of NEOD001
over a mean duration of therapy of 12.8 months.
- Published preclinical data in
the peer-reviewed journal Amyloid that further supports the proposed
mechanism of action of NEOD001, demonstrating the binding and
immunotherapy-mediated clearance properties of NEOD001 and the
related murine form of the antibody in tissue samples from multiple
organs of patients with AL amyloidosis.
PRX002/RG7935 is a monoclonal antibody for
the potential treatment of Parkinson's disease and related
synucleinopathies, and is the primary focus of Prothena's worldwide
collaboration with Roche:
- Reported positive results from
an 80-patient Phase 1b double-blind, placebo-controlled, multiple
ascending dose study that supported advancing PRX002 into a Phase 2
clinical study. All dose levels had an acceptable safety and
tolerability profile, meeting the primary objective of the study.
Robust central nervous system penetration was demonstrated by a
dose-dependent increase of PRX002 levels in cerebrospinal fluid
(CSF), and a mean concentration of PRX002 in CSF of 0.3 percent
relative to serum across all dose levels, which exceeded our
expectations based on our preclinical experience. Target engagement
was further demonstrated in this study by a rapid, dose- and
time-dependent mean reduction of free serum alpha-synuclein levels
of up to 97 percent after a single dose, which was statistically
significant (p<0.0001), and maintained following two additional
monthly doses.
- Published clinical results from
the first-in-human assessment of PRX002 in the peer-reviewed
journal Movement Disorders.
PRX003 is a monoclonal antibody for the
potential treatment of inflammatory diseases, including psoriasis
and psoriatic arthritis:
- In an oral session at the European
League Against Rheumatism (EULAR) 17th Annual European
Congress of Rheumatology, presented positive
results from a Phase 1 clinical study of PRX003 in healthy
volunteers that demonstrated PRX003 was safe and well-tolerated
following a single infusion, up to and including the highest dose
level tested. Results from this study showed that administration of
PRX003 led to greater than 95 percent neutralization of CD146 at
saturating drug exposures. CD146 is a cell adhesion molecule which
is expressed on the surface of Th17 cells. The data from this study
also demonstrated a statistically significant (p<0.0001) dose-
and time-dependent duration of downregulation of CD146 on Th17
cells.
- Presented preclinical data for
PRX003 at the American Academy of Allergy, Asthma & Immunology
(AAAAI) 2016 Annual Meeting demonstrating the ability of PRX003 to
inhibit migration of disease-causing T cells.
PRX004 is a monoclonal antibody for the
potential treatment of ATTR amyloidosis:
- In an oral session at the 6th
International Charcot-Marie-Tooth and Related Neuropathy Consortium
(CMTR) meeting, presented preclinical data from a series of novel,
conformation-specific protein immunotherapy antibodies that
selectively bind to amyloidogenic (diseased) forms of the
transthyretin (ATTR) protein in tissues from ATTR amyloidosis
patients.
- Published preclinical data from
a series of novel, conformation-specific protein immunotherapy
antibodies that selectively bind to amyloidogenic (diseased) forms
of the transthyretin (ATTR) protein in the peer-reviewed
journal Amyloid.
Corporate:
- Appointed Gene G. Kinney, Ph.D. as
President, Chief Executive Officer and as a member of the Board.
Dr. Kinney was a founding member of Prothena's leadership team, and
has served as Prothena's Chief Scientific Officer and Head of
Research and Development, and also as Chief Operating
Officer.
- Appointed Carol D. Karp as
Chief Regulatory Officer to lead Prothena's Regulatory, Quality and
Safety functions. Ms. Karp brings an extensive and successful track
record of leading global registration activities for innovative new
products in the biotechnology and pharmaceutical sectors.
Upcoming
Research and Development Milestones
Prothena's pipeline includes four
protein immunotherapy programs.
NEOD001
- Complete planned
enrollment (N=100) in the Phase 2b PRONTO study expected
during the last week of February 2017. At that time, patients
already in screening will have an opportunity to complete this
process and will be randomized into the study provided they
meet eligibility requirements. The study is therefore likely to be
overenrolled, with the last patient randomized in March
2017
- Topline results in the Phase 2b
PRONTO study expected following the 12-month study period in the
second quarter of 2018
- Complete enrollment in the Phase 3
VITAL Amyloidosis Study expected in the second quarter of 2017
PRX002
- Phase 2 clinical study expected to
begin in 2017
PRX003
- Topline results from the completed
Phase 1b multiple ascending dose, proof-of-biology study in
patients with psoriasis expected in the third quarter of 2017
PRX004
- Clinical development expected to
begin in early 2018
Fourth
Quarter and Full Year of 2016 Financial Results and 2017 Financial
Guidance
Prothena reported a net loss of
$48.9 million and $160.1 million for the fourth quarter and full
year of 2016, respectively, as compared to a net loss of $24.2
million and $80.6 million for the fourth quarter and full year of
2015, respectively. Net loss per share for the fourth quarter and
full year of 2016 was $1.41 and $4.66, respectively, as compared to
a net loss per share for the fourth quarter and full year of 2015
of $0.76 and $2.66, respectively.
Prothena reported total revenue of
$0.2 million and $1.1 million for the fourth quarter and full year
of 2016, respectively, as compared to total revenue of $0.3 million
and $1.6 million for the fourth quarter and full year of 2015,
respectively. The decrease in revenue for the fourth quarter and
full year of 2016 was primarily due to lower revenue from
Prothena's collaboration agreement with Roche.
Research and development (R&D)
expenses totaled $39.8 million and $119.5 million for the fourth
quarter and full year of 2016, respectively, as compared to $17.9
million and $58.4 million for the fourth quarter and full year of
2015, respectively. The increase in R&D expenses for the fourth
quarter and full year of 2016 was primarily due to increased
expenses for product manufacturing, clinical trials and personnel
cost. R&D expenses included non-cash share-based compensation
expense of $1.9 million and $7.1 million for the fourth quarter and
full year of 2016, respectively, as compared to $1.3 million and
$4.3 million for the fourth quarter and full year of 2015,
respectively.
General and administrative
(G&A) expenses totaled $9.6 million and $41.1 million for the
fourth quarter and full year of 2016, respectively, as compared to
$6.6 million and $23.1 million for the fourth quarter and full year
of 2015, respectively. The increase in G&A expenses for the
fourth quarter and full year of 2016 was primarily due to increases
in personnel costs. The full year costs included $7.7 million of
non-cash share-based compensation expense related to the
accelerated vesting of stock options and payments due to the estate
of our former Chief Executive Officer, Dr. Dale B. Schenk, upon his
passing. G&A expenses included non-cash share-based
compensation expense of $3.3 million and $17.8 million in the
fourth quarter and full year of 2016, respectively (including $6.5
million, of non-cash share-based compensation expense in 2016
related to the accelerated vesting of Dr. Schenk's stock options),
as compared to $1.9 million and $6.1 million in the fourth quarter
and full year of 2015, respectively.
Total non-cash share-based
compensation expense was $5.2 million and $24.9 million for the
fourth quarter and full year of 2016, respectively, as compared to
$3.3 million and $10.4 million for the fourth quarter and full year
of 2015, respectively.
As of December 31, 2016,
Prothena had $391.0 million in cash, cash equivalents and
restricted cash and no debt.
As of February 10, 2017,
Prothena had approximately 35.0 million ordinary shares
outstanding.
The Company expects the full year
2017 net cash burn from operating and investing activities to be
$160 to $170 million, including an expected milestone payment from
Roche upon initiation of the Phase 2 study of PRX002, and
ending the year with approximately $224 million in cash
(mid-point). The estimated full year 2017 net cash burn from
operating and investing activities is primarily driven by an
estimated net loss of $177 to $191 million, which includes an
estimated $26 million of non-cash share-based compensation
expense.
Upcoming
Investor Conferences
Members of the senior management
team will present and participate in investor meetings at the
following upcoming investor conferences:
- RBC Capital
Markets 2017 Global Healthcare Conference on February
22, 2017 at 1:35 PM ET in New York, NY.
- Barclays
Global Healthcare Conference on March 16, 2017 at
10:15 AM ET in Miami, FL.
- Oppenheimer
27th Annual Healthcare Conference on March 21,
2017 at 8:35 AM ET in New York, NY.
A live webcast of the
presentations can be accessed through the investor relations
section of the Company's website at www.prothena.com.
Following the live presentations, a replay of the webcast will be
available on the Company's website for at least 90 days following
the presentation date.
Conference
Call Details
Prothena management will discuss
these results and its 2017 outlook in a live audio webcast and
conference call today, Tuesday, February 14, 2017, at 4:30 PM ET.
The webcast will be made available on the Company's website
at www.prothena.com under the Investors tab in the Events
and Presentations section. Following the live audio webcast, a
replay will be available on the Company's website for 90 days.
To access the call via dial-in,
please dial (877) 887-5215 (U.S. toll free) or (315) 625-3069
(international) five minutes prior to the start time and refer to
conference ID number 56336849. A replay of the call will be
available until February 28, 2017 via dial-in at (855) 859-2056
(U.S. toll free) or (404) 537-3406 (international), Conference ID
Number 56336849.
About
Prothena
Prothena Corporation plc is a
global, late-stage clinical biotechnology company establishing
fully-integrated research, development and commercial capabilities.
Fueled by its deep scientific understanding built over decades of
research in protein misfolding and cell adhesion - the root causes
of many serious or currently untreatable amyloid and inflammatory
diseases - Prothena seeks to fundamentally change the course of
progressive diseases associated with this biology. The Company's
pipeline of antibody therapeutic candidates targets a number of
indications including AL amyloidosis (NEOD001), Parkinson's disease
and other related synucleinopathies (PRX002), inflammatory
diseases, including psoriasis and psoriatic arthritis (PRX003), and
ATTR amyloidosis (PRX004). The company continues discovery of
additional novel therapeutic candidates where its deep scientific
understanding of disease pathology can be leveraged. For more
information, please visit the company's website
at www.prothena.com.
Forward-looking Statements
This press
release contains forward-looking statements. These statements
relate to, among other things, our goal of delivering therapies to
patients; the sufficiency of our cash position; the timing of
completing enrollment in the Phase 2b and Phase 3 studies and
announcing topline results from the Phase 2b study of NEOD001; the
timing of initiating a Phase 2 study of PRX002; the timing of
announcing full topline results from the Phase 1b study of PRX003;
the timing of advancing PRX004 into a Phase 1 clinical study; our
anticipated net cash burn from operating and investing activities
for 2017 and expected cash balance at the end of 2017; and our
estimated net loss and non-cash share-based compensation expense
for 2017. 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 February
25, 2016, our subsequent Quarterly Reports on Form 10-Q filed with
the SEC and our Annual Report on Form 10-K to be filed with the SEC
for our fiscal year 2016. 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.
PROTHENA CORPORATION
PLC
CONSOLIDATED STATEMENTS OF
OPERATIONS
(unaudited - amounts in thousands except per
share data)
|
Three Months Ended
December 31, |
|
Twelve Months Ended
December 31, |
|
2016 |
|
2015 |
|
2016 |
|
2015 |
Collaboration revenue |
$ |
171 |
|
|
$ |
307 |
|
|
$ |
1,055 |
|
|
$ |
1,607 |
|
Revenue-related party |
- |
|
|
- |
|
|
- |
|
|
- |
|
Total revenue |
171 |
|
|
307 |
|
|
1,055 |
|
|
1,607 |
|
Operating expenses: |
|
|
|
|
|
|
|
Research and development |
39,844 |
|
|
17,890 |
|
|
119,534 |
|
|
58,439 |
|
General and
administrative |
9,604 |
|
|
6,629 |
|
|
41,056 |
|
|
23,105 |
|
Total operating expenses |
49,448 |
|
|
24,519 |
|
|
160,590 |
|
|
81,544 |
|
Loss from operations |
(49,277 |
) |
|
(24,212 |
) |
|
(159,535 |
) |
|
(79,937 |
) |
Other income, net: |
727 |
|
|
57 |
|
|
571 |
|
|
26 |
|
Loss before income taxes |
(48,550 |
) |
|
(24,155 |
) |
|
(158,964 |
) |
|
(79,911 |
) |
Provision for income
taxes |
353 |
|
|
2 |
|
|
1,144 |
|
|
701 |
|
Net loss |
$ |
(48,903 |
) |
|
$ |
(24,157 |
) |
|
$ |
(160,108 |
) |
|
$ |
(80,612 |
) |
Basic and diluted net loss per
share |
$ |
(1.41 |
) |
|
$ |
(0.76 |
) |
|
$ |
(4.66 |
) |
|
$ |
(2.66 |
) |
Shares used to compute basic
and diluted net loss per share |
34,603 |
|
|
31,611 |
|
|
34,351 |
|
|
30,326 |
|
PROTHENA CORPORATION
PLC
CONSOLIDATED BALANCE
SHEETS
(unaudited - amounts in
thousands)
|
December 31, |
|
2016 |
|
2015 |
Assets |
|
|
|
Cash and cash equivalents |
$ |
386,923 |
|
|
$ |
370,586 |
|
Other current assets |
4,439 |
|
|
6,817 |
|
Total current assets |
391,362 |
|
|
377,403 |
|
Property and equipment,
net |
56,452 |
|
|
3,862 |
|
Restricted cash |
4,056 |
|
|
- |
|
Other assets |
8,106 |
|
|
3,971 |
|
Total non-current assets |
68,614 |
|
|
7,833 |
|
Total assets |
$ |
459,976 |
|
|
$ |
385,236 |
|
Liabilities and Shareholders' Equity |
|
|
|
Accrued research and
development |
$ |
19,073 |
|
|
$ |
12,794 |
|
Other current liabilities |
22,002 |
|
|
9,422 |
|
Total current liabilities |
41,075 |
|
|
22,216 |
|
Non-current liabilities: |
53,498 |
|
|
2,351 |
|
Total liabilities |
94,573 |
|
|
24,567 |
|
Total shareholders'
equity |
365,403 |
|
|
360,669 |
|
Total liabilities and
shareholders' equity |
$ |
459,976 |
|
|
$ |
385,236 |
|