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 PLCCONSOLIDATED
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 PLCCONSOLIDATED
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 |
|
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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