Marketing authorization application submitted to
the European Medicines Agency for the approval of volanesorsen
Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis
Pharmaceuticals, Inc., focused on developing and commercializing
drugs to treat patients with serious cardiometabolic diseases
caused by lipid disorders, today reported financial results for the
second quarter ended June 30, 2017, and provided an update on
upcoming events and key achievements.
“We recently submitted a marketing authorization application
(MAA) to the European Medicines Agency (EMA) for the approval of
volanesorsen in Europe and we are on track to file for regulatory
approval in the U.S. and Canada in September. In anticipation of a
potential 2018 launch, we are actively preparing to commercialize
volanesorsen globally. We are building a small, highly specialized
salesforce and a comprehensive patient support program tailored to
meet the needs of patients with familial chylomicronemia syndrome
(FCS) and their treating physicians,” said Paula Soteropoulos,
president and chief executive officer of Akcea. “Last month, we
raised over $190 million with the completion of our initial public
offering (IPO) and concurrent $50 million strategic investment by
our partner Novartis Pharma AG and $25 million investment from
Ionis Pharmaceuticals, who discovered and is co-developing the
drugs in our pipeline. We now have approximately $300 million in
cash, which should allow us to advance our mature pipeline of four
novel cardiometabolic drugs, including building the infrastructure
necessary to commercialize our drugs globally. We believe we are
well-positioned to pursue our mission to bring important medicines
to patients with lipid-driven cardiometabolic diseases.”
Upcoming Events
- File for regulatory approval of volanesorsen in the U.S. and
Canada in September.
- Present results from the Phase 1/2 study evaluating
AKCEA-APOCIII-LRx in healthy volunteers and patients with elevated
triglycerides in the second half of 2017.
- Initiate Phase 2 studies with AKCEA-ANGPTL3-LRx in the second
half of 2017, including a study in patients with non-alcoholic
fatty liver disease (NAFLD) and metabolic complications and studies
in patients with rare hyperlipidemias.
- Initiate a Phase 2b dose-ranging study evaluating
AKCEA-APOCIII-LRx in patients with hypertriglyceridemia and
established cardiovascular disease in the second half of 2017.
Financial ResultsAll pro forma amounts referred
to in this press release exclude non-cash compensation expense
related to equity awards. Please refer to the reconciliation of pro
forma and GAAP measures, which is provided later in this
release.
Revenue Akcea’s revenue for the three and six
months ended June 30, 2017 was $14.1 million and $23.7 million,
respectively, all of which was related to the Company’s
collaboration with Novartis. In January 2017, Akcea and Novartis
entered into a strategic collaboration and Novartis paid Akcea a
$75 million upfront payment, which Akcea is amortizing into
revenue. As part of the strategic collaboration, Novartis also
purchased Ionis’ common stock at a premium. Since Akcea is
providing the services under the Novartis collaboration, Akcea is
also amortizing into revenue the $33.4 million premium. Under its
license agreement with Ionis, Akcea paid a $15 million sublicense
fee to Ionis related to the $75 million upfront payment and
recorded a $33.4 million non-cash charge related to the premium
Novartis paid to acquire Ionis’ common stock as described in
Operating Expenses below.
Operating Expenses Akcea’s operating expenses
for the three and six months ended June 30, 2017 on a GAAP basis
were $25.4 million and $94.9 million, respectively, and on a pro
forma basis were $21.5 million and $87.8 million, respectively.
These amounts compare to GAAP operating expenses of $13.7 million
and $29.7 million and pro forma operating expenses of $11.7 million
and $24.6 million for the same periods in 2016. Akcea’s operating
expenses increased for the six months ended June 30, 2017 compared
to the same period in 2016 primarily due to $48.4 million of
sublicensing expenses related to the Company’s collaboration with
Novartis. The $48.4 million is comprised of $15 million the Company
paid to Ionis related to the $75 million upfront payment and a
$33.4 million non-cash charge related to the premium Novartis paid
to acquire Ionis’ common stock. Akcea’s operating expenses for the
six months ended June 30, 2017 and 2016 included $12.0 million and
$4.1 million, respectively, of expenses for development and support
services Ionis provided to Akcea. Akcea expects its G&A
expenses to continue to increase as the Company continues to
prepare to launch volanesorsen.
Net Loss Akcea reported a net loss of $11.9
million and $72.3 million on a GAAP basis for the three and six
months ended June 30, 2017, respectively, compared to $13.6 million
and $29.6 million for the same periods in 2016. Akcea reported
a pro forma net loss of $8.0 million and $65.2 million for the
three and six months ended June 30, 2017 compared to $11.6 million
and $24.4 million for the same periods in 2016. For the three and
six months ended June 30, 2017, basic and diluted net loss per
share were $0.41 and $2.50, respectively. Basic and diluted net
loss per share for the same periods in 2016 were $0.47 and
$1.02.
Balance Sheet As of June 30, 2017, Akcea had
cash, cash equivalents and short-term investments of $118.7 million
compared to $7.9 million at December 31, 2016. Akcea’s cash balance
increased in the first half of 2017 due to the $75 million
upfront payment the Company received from Novartis and the $106
million from borrowings under Akcea’s line of credit with Ionis,
which converted into Akcea’s common stock in connection with the
completion of the IPO. Akcea’s second quarter cash balance did not
include the $182 million it received in net proceeds from its IPO
and Novartis’ strategic investment. Concurrent with the Company’s
IPO, the series A preferred stock that Ionis owned converted into
Akcea’s common stock. Akcea’s working capital was $53.6
million at June 30, 2017 compared to $(19.3)
million at December 31, 2016.
Key Achievements
- Submitted a marketing authorization application (MAA) to the
European Medicines Agency (EMA) for the approval of volanesorsen
for the treatment of patients with familial chylomicronemia
syndrome (FCS).
- Raised over $190 million in the Company’s initial public
offering, including the underwriters' full exercise of their
overallotment option, Novartis’ $50 million strategic investment
and $25 million strategic investment from Ionis, who discovered and
is co-developing the drugs in Akcea’s pipeline. The IPO generated
over $180 million in net proceeds.
- Established a strategic collaboration with Novartis, a leader
in cardiovascular medicines, worth up to more than $1.6 billion
plus royalties for the development and commercialization of
AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx for large populations of
patients who have high cardiovascular risk due to inadequately
treated lipid disorders.
- Successfully completed the Phase 3 program of volanesorsen for
the treatment of FCS.
- Expanded the Company’s independent board of directors with the
appointments of Elaine Hochberg, Sandford Smith, Edward Fitzgerald
and Christopher Gabrieli, as chairman of the board.
- Initiated a Phase 2b dose-ranging study with AKCEA-APO(a)-LRx
in patients with elevated Lp(a) and established cardiovascular
disease to support the design of the Phase 3 cardiovascular outcome
study.
- Published key preclinical findings with angiopoietin-like 3
(ANGPTL3)-targeting drugs and Phase 1/2 clinical study results with
AKCEA-ANGPTL3-LRx in the New England Journal of Medicine.
- Published results from the IN-FOCUS (Investigation of Findings
and Observations Captured in Burden of Illness Survey in FCS
Patients) survey, the largest survey in patients with FCS,
demonstrating the considerable daily and life-long burden of
disease for these patients.
ABOUT AKCEA THERAPEUTICSAkcea Therapeutics, an
affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical
company focused on developing and commercializing drugs to treat
patients with serious cardiometabolic diseases caused by lipid
disorders. Akcea is advancing a mature pipeline of four novel drugs
with the potential to treat multiple diseases, including
volanesorsen, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx and
AKCEA-APOCIII-LRx. All four drugs were discovered and are being
co-developed by Ionis, a leader in antisense therapeutics, based on
Ionis’ proprietary antisense technology. The most advanced drug in
its pipeline, volanesorsen, is under regulatory review in the EU
for the treatment of familial chylomicronemia syndrome, or FCS, and
is currently in Phase 3 clinical development for the treatment of
familial partial lipodystrophy, or FPL. Akcea is building the
infrastructure to commercialize its drugs globally with a focus on
lipid specialists as the primary call point. Akcea is located in
Cambridge, Massachusetts. Additional information about Akcea is
available at www.akceatx.com.
FORWARD-LOOKING STATEMENTThis press release
includes forward-looking statements regarding financial position,
outlook and business of Akcea Therapeutics, Inc. and the
therapeutic and commercial potential of volanesorsen and other
products in development. Any statement describing Akcea’s goals,
expectations, financial or other projections, intentions or beliefs
is a forward-looking statement and should be considered an at-risk
statement. Such statements are subject to certain risks and
uncertainties, particularly those inherent in the process of
discovering, developing and commercializing drugs that are safe and
effective for use as human therapeutics, and in the endeavor of
building a business around such drugs. Akcea’s forward-looking
statements also involve assumptions that, if they never materialize
or prove correct, could cause its results to differ materially from
those expressed or implied by such forward-looking statements.
Although Akcea’s forward-looking statements reflect the good faith
judgment of its management, these statements are based only on
facts and factors currently known by Akcea. As a result, you are
cautioned not to rely on these forward-looking statements. These
and other risks concerning Akcea’s programs are described in
additional detail in its final prospectus for its initial public
offering, which is on file with the SEC.
In this press release, unless the context requires otherwise,
“Akcea,” “Company,” “we,” “our,” and “us” refers to Akcea
Therapeutics.
Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc. Ionis Pharmaceuticals™ is a trademark of Ionis
Pharmaceuticals, Inc.
|
|
AKCEA THERAPEUTICS, INC.SELECTED
FINANCIAL INFORMATIONCondensed Consolidated Statements of
Operations(In Thousands, Except Per Share
Data) |
|
|
|
|
|
|
|
|
|
Three months ended, |
|
Six months ended, |
|
|
|
June 30, |
|
June 30, |
|
|
|
|
2017 |
|
|
|
2016 |
|
|
|
2017 |
|
|
|
2016 |
|
|
|
|
(unaudited) |
|
(unaudited) |
|
R&D Revenue |
|
$ |
14,128 |
|
|
$ |
- |
|
|
$ |
23,725 |
|
|
$ |
- |
|
|
Expenses: |
|
|
|
|
|
|
|
|
|
Research
and development |
|
|
18,487 |
|
|
|
10,944 |
|
|
|
83,282 |
|
|
|
22,734 |
|
|
General
and administrative |
|
|
6,915 |
|
|
|
2,762 |
|
|
|
11,590 |
|
|
|
7,014 |
|
|
Total
operating expenses |
|
|
25,402 |
|
|
|
13,706 |
|
|
|
94,872 |
|
|
|
29,748 |
|
|
Loss from
operations |
|
|
(11,274 |
) |
|
|
(13,706 |
) |
|
|
(71,147 |
) |
|
|
(29,748 |
) |
|
Other income
(expense): |
|
|
|
|
|
|
|
|
|
Investment income |
|
|
295 |
|
|
|
91 |
|
|
|
358 |
|
|
|
177 |
|
|
Interest
expense |
|
|
(965 |
) |
|
|
- |
|
|
|
(1,507 |
) |
|
|
- |
|
|
|
|
|
|
|
|
|
|
|
|
Net loss |
|
$ |
(11,944 |
) |
|
$ |
(13,615 |
) |
|
$ |
(72,296 |
) |
|
$ |
(29,571 |
) |
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share |
|
$ |
(0.41 |
) |
|
$ |
(0.47 |
) |
|
$ |
(2.50 |
) |
|
$ |
(1.02 |
) |
|
Shares used in
computing basic and diluted net loss per share |
|
|
28,885 |
|
|
|
28,885 |
|
|
|
28,885 |
|
|
|
28,885 |
|
|
AKCEA THERAPEUTICS,
INC.Reconciliation of GAAP to Pro Forma Basis:Condensed
Consolidated Operating Expenses, Loss From Operations, and Net
Loss(In Thousands) |
|
|
|
|
|
|
|
Three months ended,June 30, |
|
Six months ended,June 30, |
|
|
|
2017 |
|
|
|
2016 |
|
|
|
2017 |
|
|
|
2016 |
|
|
|
(unaudited) |
|
(unaudited) |
As reported
operating expenses according to GAAP |
|
$ |
25,402 |
|
|
$ |
13,706 |
|
|
$ |
94,872 |
|
|
$ |
29,748 |
|
Excluding
compensation expense related to equity awards |
|
|
(3,942 |
) |
|
|
(1,978 |
) |
|
|
(7,122 |
) |
|
|
(5,168 |
) |
|
|
|
|
|
|
|
|
|
Pro forma
operating expenses |
|
$ |
21,460 |
|
|
$ |
11,728 |
|
|
$ |
87,750 |
|
|
$ |
24,580 |
|
|
|
|
|
|
|
|
|
|
As reported
loss from operations according to GAAP |
|
$ |
(11,274 |
) |
|
$ |
(13,706 |
) |
|
$ |
(71,147 |
) |
|
$ |
(29,748 |
) |
Excluding
compensation expense related to equity awards |
|
|
(3,942 |
) |
|
|
(1,978 |
) |
|
|
(7,122 |
) |
|
|
(5,168 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Pro forma loss
from operations |
|
$ |
(7,332 |
) |
|
$ |
(11,728 |
) |
|
$ |
(64,025 |
) |
|
$ |
(24,580 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
As reported net
loss according to GAAP |
|
$ |
(11,944 |
) |
|
$ |
(13,615 |
) |
|
$ |
(72,296 |
) |
|
$ |
(29,571 |
) |
Excluding
compensation expense related to equity awards |
|
|
(3,942 |
) |
|
|
(1,978 |
) |
|
|
(7,122 |
) |
|
|
(5,168 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Pro forma net
loss |
|
$ |
(8,002 |
) |
|
$ |
(11,637 |
) |
|
$ |
(65,174 |
) |
|
$ |
(24,403 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Reconciliation of GAAP to Pro Forma BasisAs
illustrated in the Selected Financial Information in this press
release, pro forma operating expenses, pro forma loss from
operations, and pro forma net loss were adjusted from GAAP to
exclude compensation expense related to equity awards, which are
non-cash. Akcea has regularly reported non-GAAP measures for
operating results as pro forma results. These measures are provided
as supplementary information and are not a substitute for financial
measures calculated in accordance with GAAP. Akcea reports these
pro forma results to better enable financial statement users to
assess and compare its historical performance and project its
future operating results and cash flows. Further, the presentation
of Akcea's pro forma results is consistent with how Akcea's
management internally evaluates the performance of its
operations.
|
|
AKCEA THERAPEUTICS,
INC.Condensed Consolidated Balance Sheets
(In Thousands)
|
|
|
|
|
|
June 30, |
|
December 31, |
|
|
|
|
2017 |
|
|
|
2016 |
|
|
|
|
|
|
|
|
Assets: |
|
|
|
|
|
Cash,
cash equivalents and short-term investments |
|
$ |
118,668 |
|
$ |
7,857 |
|
|
Other
current assets |
|
|
3,939 |
|
|
|
1,209 |
|
|
Licenses,
net |
|
|
1,281 |
|
|
|
1,341 |
|
|
Other
assets |
|
|
216 |
|
|
|
277 |
|
|
Total
assets |
|
$ |
124,104 |
|
|
$ |
10,684 |
|
|
|
|
|
|
|
|
Liabilities and
stockholders’ deficit: |
|
|
|
|
|
Other
current liabilities |
|
$ |
3,648 |
|
|
$ |
4,055 |
|
|
Payable
to Ionis |
|
|
11,244 |
|
|
|
24,355 |
|
|
Current
portion of deferred contract revenue |
|
|
54,155 |
|
|
|
- |
|
|
Long-term
portion of deferred rent portion |
|
|
18 |
|
|
|
21 |
|
|
Line of
credit with Ionis |
|
|
107,507 |
|
|
|
- |
|
|
Long-term
deferred contract revenue |
|
|
30,515 |
|
|
|
- |
|
|
Stockholders’ deficit |
|
|
(82,983 |
) |
|
|
(17,747 |
) |
|
Total
liabilities and stockholders’ equity |
|
$ |
124,104 |
|
|
$ |
10,684 |
|
|
Media and Investor Contacts:
D. Wade Walke, Ph.D.
Vice President, Corporate Communications and Investor Relations
760-603-2741
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