Aegerion Pharmaceuticals Announces Fourth-Quarter and Year-End 2012 Financial
Promising Early Progress in Commercial Launch of JUXTAPID(TM) (lomitapide)
CAMBRIDGE, Mass., March 6, 2013 (GLOBE NEWSWIRE) -- Aegerion Pharmaceuticals,
Inc. (Nasdaq:AEGR), a biopharmaceutical company dedicated to the development
and commercialization of innovative, life-altering therapies for patients with
debilitating, often fatal rare diseases, announced its financial results and
business highlights for the fourth-quarter and year-ended December 31, 2012.
As the company previously announced, JUXTAPID was approved by the U.S. Food
and Drug Administration (FDA) in December 2012 as an adjunct to a low-fat diet
and other lipid-lowering treatments, including LDL apheresis where available,
to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC),
apolipoprotein B (apo B) and non-high-density-lipoprotein cholesterol
(non-HDL) in patients with HoFH.
"2012 was an important year for Aegerion, and more importantly, for HoFH
patients," said Marc D. Beer, Chief Executive Officer. "Following FDA
approval, we are focused on strong commercial execution. We are very pleased
with the early acceptance of JUXTAPID with over 400 REMS trained physicians
and85 prescriptions globally to date. The initial ramp of our commercial
launch is on track to achieve our previously stated guidance of 250-300
patients globally on therapy by the end of 2013."
"The work needed to achieve our commitment to bring this important therapy to
HoFH patients globally continues. We expect a mid-year decision on JUXTAPID in
the European Union, and are simultaneously driving our development activities
in the pediatric and Japanese HoFH patient populations with planned or ongoing
nonclinical and clinical studies. In addition, we are building infrastructure
in key markets that allow for sales of JUXTAPID on a named patient sales basis
following FDA approval. We are executing on our plan to deliver JUXTAPID to
HoFH patients in need."
For the fourth-quarter ended December 31, 2012, net loss was $21.8 million, or
$0.86 per share, compared with a net loss of $13.9 million, or $0.66 per
share, for the same period in 2011. For the year-ended December 31, 2012, net
loss was $62.3 million, or $2.64 per share, compared with a net loss of $39.5
million, or $2.03 per share, for the same period in 2011.
Research and development expenses were $8.6 million for the quarter ended
December 31, 2012, compared to $8.4 million for the same period in 2011.
Research and development expenses were $25.2 million for the year ended
December 31, 2012, compared to $24.4 million for the same period in 2011. The
increase in research and development expenses in the fourth quarter and full
year of 2012 over the comparable periods in 2011 was primarily related to
increased headcount required to support the Company's regulatory and medical
affairs activities, as well as costs for production validation runs, partially
offset by decreases in clinical trial expenses related to trials which had
been substantially completed in 2011.
Selling, general and administrative expenses were $13.1 million for the
quarter ended December 31, 2012, compared to $4.3 million for the same period
in 2011. Selling, general and administrative expenses were $34.1 million for
the year ended December 31, 2012, compared to $14.0 million for the same
period in 2011. The increases in selling, general and administrative expenses
in the fourth-quarter and full-year 2012 over the comparable periods in 2011
were primarily related to costs of increased headcount to support the planned
commercial launch of JUXTAPID and in administrative functions.
Cash, cash equivalents and marketable securities totaled $82.2 million as of
December 31, 2012, compared to $73.2 million as of December 31, 2011.
Aegerion expects total operating expenses, excluding stock-based compensation
expense, to be between $75 and $85 million in 2013. In addition, Aegerion
confirmed the following previously stated financial guidance:
*Aegerion expects global net revenues of $15 million to $25 million for FY
2013 with 250 to 300 patients on JUXTAPID therapy globally by year-end
*In the second half of 2014, the Company expects to:
*generate global net revenue at a $100 million annualized run rate; and
*achieve cash flow breakeven from operations.
Conference Call Details
Aegerion will hold a conference call to discuss its financial results,
business highlights and outlook today, Wednesday, March 6, 2013 at 8:30 a.m.
EST. In addition, the Company will answer questions concerning business and
financial developments and trends, and other matters affecting the Company,
some of the responses to which may contain information that has not been
To listen to the conference call, dial (866) 516-3002 (international callers
dial (760) 298-5082). In addition, the presentation will be webcast live, and
may be accessed for up to 14 days following the call, by visiting the
"Investors" section of Aegerion's website, www.aegerion.com. An accompanying
slide presentation also can be accessed via the "Investors" section of the
About JUXTAPID™ (lomitapide) capsules
JUXTAPID was approved by the U.S. FDA in December 2012 as an adjunct to a
low-fat diet and other lipid-lowering treatments, including LDL apheresis
where available, to reduce low-density lipoprotein cholesterol (LDL-C), total
cholesterol (TC), apolipoprotein B (apo B) and non- high-density-lipoprotein
cholesterol (non-HDL) in patients with HoFH.
Important Safety Information, including BOXED WARNING:
WARNING: RISK OF HEPATOTOXICITY
JUXTAPID can cause elevations in transaminases. In the JUXTAPID clinical
trial, 10 (34%) of the 29 patients treated with JUXTAPID had at least one
elevation in alanine aminotransferase (ALT) or aspartate aminotransferase
(AST) ≥ 3x upper limit of normal (ULN). There were no concomitant clinically
meaningful elevations of total bilirubin, international normalized ratio
(INR), or alkaline phosphatase.
JUXTAPID also increases hepatic fat, with or without concomitant increases in
transaminases. The median absolute increase in hepatic fat was 6% after both
26 and 78 weeks of treatment, from 1% at baseline, measured by magnetic
resonance spectroscopy. Hepatic steatosis associated with JUXTAPID treatment
may be a risk factor for progressive liver disease, including steatohepatitis
Measure ALT, AST, alkaline phosphatase, and total bilirubin before initiating
treatment and then ALT and AST regularly as recommended.During treatment,
adjust the dose of JUXTAPID if the ALT or AST are ≥3x ULN.Discontinue
JUXTAPID for clinically significant liver toxicity.
Because of the risk of hepatotoxicity, JUXTAPID is available only through a
restricted program under a Risk Evaluation and Mitigation Strategy (REMS)
called the JUXTAPID REMS Program.
*Concomitant administration of moderate or strong CYP3A4 inhibitors
*Moderate or severe hepatic impairment or active liver disease including
unexplained persistent elevations of serum transaminases
WARNINGS AND PRECAUTIONS
JUXTAPID can cause elevations in transaminases and hepatic steatosis.Although
cases of hepatic failure have not been reported, there is concern that
JUXTAPID could induce steatohepatitis, which can progress to cirrhosis over
several years.Modify the dose of JUXTAPID if elevations of transaminases are
observed and discontinue JUXTAPID for persistent or clinically significant
elevations.If transaminase elevations are accompanied by clinical symptoms of
liver injury, increases in bilirubin ≥2x ULN, or active liver disease,
discontinue treatment with JUXTAPID and identify the probable cause.Use
JUXTAPID with caution when co-administered with agents known to be
hepatotoxic.Alcohol may increase levels of hepatic fat and induce or
exacerbate liver injury.
Measure ALT, AST, alkaline phosphatase, and total bilirubin before initiating
treatment.During the first year, measure liver-related tests (ALT and AST at
a minimum) prior to each increase in dose or monthly, whichever occurs
first.After the first year, do these tests at least every 3 months and before
any increase in dose.
Females of reproductive potential should have a negative pregnancy test before
starting JUXTAPID and should use effective contraception during therapy with
Given its mechanism of action in the small intestine, JUXTAPID may reduce the
absorption of fat-soluble nutrients.Patients treated with JUXTAPID should
take daily supplements that contain 400 international units vitamin E and at
least 200 mg linoleic acid, 210 mg alpha-linolenic acid (ALA), 110 mg
eicosapentaenoic acid (EPA), and 80 mg docosahexaenoic acid (DHA).
Gastrointestinal adverse reactions are common and may lead to treatment
discontinuation.To reduce the risk of gastrointestinal adverse reactions,
patients should adhere to a low-fat diet supplying less than 20% of energy
from fat and the dosage of JUXTAPID should be increased gradually.
Combination with CYP3A4 inhibitors increases exposure to lomitapide.Strong
and moderate CYP3A4 inhibitors should not be used with JUXTAPID.JUXTAPID
dosage should not exceed 30 mg daily when used concomitantly with weak CYP3A4
Due to risk of myopathy associated with simvastatin or lovastatin, doses of
these agents should be limited when co-administered with JUXTAPID.
JUXTAPID increases the plasma concentrations of warfarin.Increases or
decreases in the dose of JUXTAPID may lead to supra- or subtherapeutic
anticoagulation, respectively. Patients taking warfarin should undergo regular
monitoring of the INR, especially after any changes in JUXTAPID dosage.
Avoid use of JUXTAPID in patients with rare hereditary disorders of galactose
The most common adverse reactions were gastrointestinal, reported by 27 (93%)
of 29 patients.Adverse reactions reported by ≥8 (28%) patients in the HoFH
clinical trial included diarrhea, nausea, vomiting, dyspepsia and abdominal
pain.Other common adverse reactions, reported by 5 to 7 (17-24%) patients,
included weight loss, abdominal discomfort, abdominal distension,
constipation, flatulence, increased ALT, chest pain, influenza,
nasopharyngitis, and fatigue.
About Aegerion Pharmaceuticals
Aegerion Pharmaceuticals is a biopharmaceutical company dedicated to the
development and commercialization of innovative, life-altering therapies for
patients with debilitating, often fatal, rare diseases.Our first approved
product, JUXTAPID, is an oral once-daily capsule that offers a new treatment
option to patients with homozygous familial hypercholesterolemia (HoFH) – a
severe lipid disorder. For more information about the company, please visit
The Aegerion Pharmaceuticals, Inc. logo is available at
This press release contains forward-looking statements, including statements
regarding the commercialization of JUXTAPID in the U.S.; the potential for
JUXTAPID as a treatment for HoFH; forecasts as to the number of patients
expected to be on therapy at the end of 2013; the possibility of named patient
sales and other expanded access use of lomitapide outside the US; the
potential for and possible timing of approval of JUXTAPID in the E.U.; planned
nonclinical and clinical development activities directed at pediatric and
Japanese HoFH patient populations, and the potential for future opportunities
for expansion in those areas, and expectations as to future financial results,
including operating expenses, net revenues, annual revenue run rate and cash
flow break-even. These forward-looking statements are neither promises nor
guarantees of future performance, and are subject to a variety of risks and
uncertainties, many of which are beyond our control, which could cause actual
results to differ materially from those contemplated in these forward-looking
statements. In particular, the risks and uncertainties include, among other
factors: the risk that JUXTAPID may not gain market acceptance or that market
acceptance may be lower than expected; the risk that the actual number of
patients with HoFH may be lower than expected; the risk that the restrictions
imposed by the regulatory authorities or the side effect profile or the impact
of competitive products may limit the potential of JUXTAPID; the risk that
payers may decide not to provide reimbursement for JUXTAPID, or may impose
restrictions that hinder reimbursement; the risk that regulatory authorities
in the E.U. or other countries outside the U.S. may not be satisfied with the
efficacy or safety profile of JUXTAPID or our proposed risk management plan;
the risk that we do not receive approval of JUXTAPID in the E.U. or other
countries outside the U.S. on a timely basis, or at all; the risk that we are
not able to generate the level of named patient sales in countries outside the
U.S. that we expect; the risk that technical hurdles may delay initiation of
future clinical trials; the risk of unexpected results in use of our
commercial product or during our additional nonclinical or clinical
development work with JUXTAPID; the risk of unexpected manufacturing issues
affecting future supply; the risk that we may incur unanticipated expenses in
connection with our activities; and the other risks inherent in
commercialization, drug development and the regulatory approval process.
For additional disclosure regarding these and other risks we face, see the
disclosure contained in the "Risk Factors" section of Aegerion's Current
Report on Form 8-K filed on January 10, 2013, and our other public filings
with the Securities and Exchange Commission, available on the SEC's website at
http://www.sec.gov. We undertake no obligation to update or revise the
information contained in this press release, whether as a result of new
information, future events or circumstances or otherwise.
Aegerion Pharmaceuticals, Inc.
Condensed Consolidated Statements of Operations
(in thousands, except per share amounts)
Three Months Ended December Year Ended December 31,
2012 2011 2012 2011
Researchand development $8,558 $8,363 $25,164 $24,433
Selling, generaland 13,054 4,345 34,077 13,966
Restructuring costs (11) 912 1,366 912
Total operating expenses 21,601 13,620 60,607 39,311
Loss from operations (21,601) (13,620) (60,607) (39,311)
Interest expense (205) (335) (937) (1,114)
Interest income 46 44 162 209
Other income/(expense), (39) -- (883) 748
Net loss $(21,799) $(13,911) $(62,265) $(39,468)
Net loss per common share $(0.86) $(0.66) $(2.64) $(2.03)
- basic and diluted
outstanding - basic and 25,418 21,179 23,563 19,409
The Company has reclassified certain prior period amounts to conform to the
current period presentation. In 2012, the Company began allocating certain
overhead costs across its functional areas and as a result reclassified
certain amounts in the prior year from selling, general and administrative
expenses to research and development expenses.
Aegerion Pharmaceuticals, Inc.
Condensed Consolidated Balance Sheets
December 31, December 31,
Cash, cash equivalents and marketable securities $82,177 $73,163
Prepaid expensesand other current assets 1,571 914
Restricted cash 105 105
Propertyand equipment, net 1,143 526
Other assets 93 860
Total assets $85,089 $75,568
Accounts payable and accrued expenses $13,904 $7,525
Current portion of long-term debt 3,022 1,875
Long-term debt 7,589 8,125
Other noncurrent liabilities 173 842
Total liabilities 24,688 18,367
Total stockholders' equity 60,401 57,201
Total liabilities and stockholders' equity $85,089 $75,568
CONTACT: Aegerion Pharmaceuticals, Inc.
Manager, Investor Relations
Aegerion Pharmaceuticals, Inc. Logo
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