179 Fabry Disease Patients on Reimbursed
Galafold (migalastat) as of July 31 – On Target to Reach 300
Patients by Year-End 2017
Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company
at the forefront of therapies for rare and orphan diseases, today
announced financial results for the second quarter ended June 30,
2017. The Company also provided near-term program updates and
reiterated full-year 2017 financial guidance.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, stated, “During the second quarter
and into the third quarter we have continued to successfully
execute across all five of our strategic priorities which include
the international launch for our oral precision medicine Galafold
(migalastat) for Fabry disease, the advancement of our lead
clinical programs in Epidermolysis Bullosa and Pompe disease, and
further strengthening of our balance sheet. Our progress during the
first half of this year has been driven by our focused execution
and groundbreaking science which is tremendous for patients as well
as our shareholders. We have created a solid foundation to continue
building on this momentum as we approach several important
milestones in the second half of this year. I believe that today,
more than ever before, Amicus is well-positioned to become a
leading global biotechnology company with the potential to deliver
significant benefits to people living with rare devastating
diseases.”
Second Quarter 2017 Financial
Results
- Total revenue in the second quarter 2017 was approximately $7.2
million, a sequential increase of 71.4% from total revenue of $4.2
million in the first quarter 2017. Total revenue represents
commercial sales of Galafold (migalastat) which commenced in May
2016, as well as reimbursed Expanded Access Programs (EAPs).
- Cash, cash equivalents, and marketable securities totaled
$227.2 million at June 30, 2017 compared to $330.4 million at
December 31, 2016.
- Total operating expenses increased to $53.2 million compared to
$48.5 million for the second quarter 2016 primarily due to
increases in manufacturing scale-up investments on the Pompe
program.
- Net cash spend was $103.5 million for the six months ending
June 30, 2017.
- Net loss was $48.1 million, or $0.34 per share, compared to a
net loss of $51.1 million, or $0.40 per share, for the second
quarter 2016.
2017
Financial Guidance
Cash, cash equivalents, and marketable securities totaled $227.2
million at June 30, 2017 compared to $330.4 million at December 31,
2016. The Company added to the quarter-ending cash position with
$243.2 million in net proceeds from a follow on public offering in
July 2017.
Amicus continues to expect full-year 2017 net operating cash
spend of between $175 million to $200 million and full-year 2017
total net cash spend (including third-party milestone payments and
capital expenditures) of between $200 million and $225 million. The
current cash position, in addition to the net proceeds of $243.2
million from the follow-on public offering, is anticipated to fund
ongoing operations into at least the second half of 2019.
Program Highlights
Migalastat for Fabry
Disease
Migalastat is an oral precision medicine
intended to treat Fabry disease in patients who have amenable
genetic mutations. The European Commission (EC), in addition to
regulatory authorities in Switzerland and Israel, have granted full
approval for migalastat under the trade name Galafold. The EC
approval may serve as the basis for regulatory approvals in more
than two-thirds of the global Fabry market that is outside the U.S.
In the U.S., as previously announced, the FDA has confirmed that
Amicus may submit a new drug application (NDA) for migalastat.
International Launch and Expanded Access
Programs (EAP) Updates:
- 179 patients (naïve and ERT-switch) on reimbursed Galafold as
of July 31, 2017
- 12 countries with reimbursement (commercial or EAP) including
the top four largest EU markets
- Reimbursement dossiers submitted and pricing discussions are
now underway in 13 countries
- Target of 300 patients treated with reimbursed Galafold by
year-end 2017
Global Regulatory Updates:
- Two additional approvals secured outside the EU (Switzerland
and Israel)
- Regulatory submissions completed in seven additional
territories outside the EU, including Japan, Canada and
Australia
- U.S. FDA confirmed NDA submission may be based on existing data
(no additional gastrointestinal symptoms study required)
Anticipated Upcoming Fabry Disease Program Milestones:
- International commercial launch and EAPs in additional
countries
- Additional regulatory submissions including a U.S. NDA
(4Q17)
- Regulatory decision in Japan (1H18)
- Fabry ERT cell line development and optimization
ATB200/AT2221 for Pompe
Disease
ATB200/AT2221 is a novel treatment paradigm that
consists of ATB200, a unique recombinant human acid
alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate
structures, particularly mannose-6 phosphate (M6P), to enhance
uptake, co-administered with AT2221, a pharmacological chaperone.
Positive functional data in initial patients were reported during
the second quarter 2017 from an ongoing global Phase 1/2 clinical
study (ATB200-02) to evaluate safety, tolerability,
pharmacokinetics (PK), and pharmacodynamics (PD) of ATB200/AT2221.
The study enrolled a total of 20 patients across three cohorts,
including ambulatory ERT-switch patients (Cohort 1), non-ambulatory
ERT-switch patients (Cohort 2), and ERT-naïve patients (Cohort
3).
Anticipated Upcoming Pompe Disease Program
Milestones:
- Full ATB200-02 study data, including 6-month functional data in
all patients (late 3Q17)
- Meetings with US and EU regulators
SD-101 for Epidermolysis Bullosa
(EB)
SD-101 is a novel, late-stage, proprietary
topical treatment with the potential to be the first approved
therapy for EB. SD-101 is currently being investigated in a
registration-directed Phase 3 study (ESSENCE, also known as SD-005)
to support global regulatory submissions. As previously announced,
Amicus has completed the analysis plan for the primary endpoints in
the blinded ongoing ESSENCE study, and top-line Phase 3 data are on
track for late in the third quarter of 2017. More than 95% of
patients completing the primary treatment period have elected to
continue in the open-label extension study
SD-101 was the first investigational treatment
to show improvements in wound closure across all major EB types in
completed Phase 2 clinical studies. SD-101 has been granted FDA
Breakthrough Therapy designation, rare pediatric disease
designation, and orphan drug designation.
Anticipated EB Program Milestones:
- Top-line Phase 3 data (late 3Q17)
Conference Call and
WebcastAmicus Therapeutics will host a conference call and
audio webcast today, August 7, 2017 at 8:30 a.m. ET to discuss
second quarter 2017 financial results and corporate updates.
Interested participants and investors may access the conference
call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784
(international); participant code 60127813.
An audio webcast can also be accessed via the
Investors section of the Amicus Therapeutics corporate web site at
http://ir.amicusrx.com/, and will be archived for 30 days. Web
participants are encouraged to go to the web site 15 minutes prior
to the start of the call to register, download and install any
necessary software. A telephonic replay of the call will be
available for seven days beginning at 11:30 a.m. ET today. Access
numbers for this replay are 855-859-2056 (U.S./Canada) and
404-537-3406 (international); participant code 60127813.
Important Safety
InformationTreatment with GALAFOLD should be initiated and
supervised by specialists experienced in the diagnosis and
treatment of Fabry disease. GALAFOLD is not recommended for use in
patients with a nonamenable mutation.
- GALAFOLD is not intended for concomitant use with enzyme
replacement therapy.
- GALAFOLD is not recommended for use in patients with Fabry
disease who have severe renal impairment (<30 mL/min/1.73 m2).
The safety and efficacy of GALAFOLD in children 0–15 years of age
have not yet been established.
- No dosage adjustments are required in patients with hepatic
impairment or in the elderly population.
- There is very limited experience with the use of this medicine
in pregnant women. If you are pregnant, think you may be pregnant,
or are planning to have a baby, do not take this medicine until you
have checked with your doctor, pharmacist, or nurse.
- While taking GALAFOLD, effective birth control should be used.
It is not known whether GALAFOLD is excreted in human milk.
- Contraindications to GALAFOLD include hypersensitivity to the
active substance or to any of the excipients listed in the
PRESCRIBING INFORMATION.
- It is advised to periodically monitor renal function,
echocardiographic parameters and biochemical markers (every 6
months) in patients initiated on GALAFOLD or switched to
GALAFOLD.
- OVERDOSE: General medical care is recommended in the case of
GALAFOLD overdose.
- The most common adverse reaction reported was headache, which
was experienced by approximately 10% of patients who received
GALAFOLD. For a complete list of adverse reactions, please review
the SUMMARY OF PRODUCT CHARACTERISTICS.
- Call your doctor for medical advice about side effects.
For further important safety information for Galafold, including
posology and method of administration, special warnings, drug
interactions and adverse drug reactions, please see the European
SmPC for Galafold available from the EMA website at
www.ema.europa.eu.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a biotechnology company at the
forefront of therapies for rare and orphan diseases. The Company
has a robust pipeline of advanced therapies for a broad range of
human genetic diseases. Amicus’ lead programs in development
include the small molecule pharmacological chaperone migalastat as
a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa
(EB), as well as novel enzyme replacement therapy (ERT) and
biologic products for Fabry disease, Pompe disease, and other rare
and devastating diseases.
Forward-Looking StatementsThis
press release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995
relating to preclinical and clinical development of our product
candidates, the timing and reporting of results from preclinical
studies and clinical trials, the prospects and timing of the
potential regulatory approval of our product candidates,
commercialization plans, financing plans, and the projected cash
position for the Company. The inclusion of forward-looking
statements should not be regarded as a representation by us that
any of our plans will be achieved. Any or all of the
forward-looking statements in this press release may turn out to be
wrong and can be affected by inaccurate assumptions we might make
or by known or unknown risks and uncertainties. For example, with
respect to statements regarding the goals, progress, timing, and
outcomes of discussions with regulatory authorities, and in
particular the potential goals, progress, timing, and results of
preclinical studies and clinical trials, actual results may differ
materially from those set forth in this release due to the risks
and uncertainties inherent in our business, including, without
limitation: the potential that results of clinical or preclinical
studies indicate that the product candidates are unsafe or
ineffective; the potential that it may be difficult to enroll
patients in our clinical trials; the potential that regulatory
authorities, including the FDA, EMA, and PMDA, may not grant or may
delay approval for our product candidates; the potential that we
may not be successful in commercializing Galafold in Europe or our
other product candidates if and when approved; the potential that
preclinical and clinical studies could be delayed because we
identify serious side effects or other safety issues; and the
potential that we will need additional funding to complete all of
our studies. Further, the results of earlier preclinical studies
and/or clinical trials may not be predictive of future results for
any of our product candidates. With respect to statements regarding
projections of the Company's cash position, actual results may
differ based on market factors and the Company's ability to execute
its operational and budget plans. In addition, all forward-looking
statements are subject to other risks detailed in our previous
filings with the SEC and in our Annual Report on Form 10-K for the
year ended December 31, 2016 and our Quarterly Report on Form 10-Q
for the quarter ended June 30, 2017. You are cautioned not to place
undue reliance on these forward-looking statements, which speak
only as of the date hereof. All forward-looking statements are
qualified in their entirety by this cautionary statement, and we
undertake no obligation to revise or update this news release to
reflect events or circumstances after the date hereof.
TABLE 1 |
Amicus Therapeutics, Inc. |
Consolidated Statements of
Operations |
(Unaudited) |
(in thousands, except share and per share
amounts) |
|
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
|
|
2017 |
|
|
|
2016 |
|
|
|
2017 |
|
|
|
2016 |
|
Revenue: |
|
|
|
|
|
|
|
Net product sales |
$ |
7,158 |
|
|
$ |
— |
|
|
$ |
11,327 |
|
|
$ |
— |
|
Cost of goods sold |
|
1,061 |
|
|
|
— |
|
|
|
1,836 |
|
|
|
— |
|
Gross
Profit |
|
6,097 |
|
|
|
— |
|
|
|
9,491 |
|
|
|
— |
|
Operating
Expenses: |
|
|
|
|
|
|
|
Research and
development |
|
31,985 |
|
|
|
18,281 |
|
|
|
62,861 |
|
|
|
41,706 |
|
Selling, general and
administrative |
|
19,311 |
|
|
|
19,300 |
|
|
|
38,443 |
|
|
|
35,001 |
|
Changes in fair value
of contingent consideration payable |
|
1,050 |
|
|
|
10,186 |
|
|
|
5,628 |
|
|
|
13,338 |
|
Restructuring
charges |
|
— |
|
|
|
8 |
|
|
|
— |
|
|
|
58 |
|
Depreciation |
|
812 |
|
|
|
767 |
|
|
|
1,636 |
|
|
|
1,440 |
|
Total operating
expenses |
|
53,158 |
|
|
|
48,542 |
|
|
|
108,568 |
|
|
|
91,543 |
|
Loss from
operations |
|
(47,061 |
) |
|
|
(48,542 |
) |
|
|
(99,077 |
) |
|
|
(91,543 |
) |
Other income
(expenses): |
|
|
|
|
|
|
|
Interest income |
|
753 |
|
|
|
331 |
|
|
|
1,512 |
|
|
|
638 |
|
Interest expense |
|
(4,179 |
) |
|
|
(1,055 |
) |
|
|
(8,469 |
) |
|
|
(2,000 |
) |
Other income
(expense) |
|
2,400 |
|
|
|
(2,237 |
) |
|
|
3,010 |
|
|
|
(2,289 |
) |
Loss before income tax
(expense)/benefit |
|
(48,087 |
) |
|
|
(51,503 |
) |
|
|
(103,024 |
) |
|
|
(95,194 |
) |
Income tax (expense)/
benefit |
|
(49 |
) |
|
|
453 |
|
|
|
(105 |
) |
|
|
453 |
|
Net loss
attributable to common stockholders |
$ |
(48,136 |
) |
|
$ |
(51,050 |
) |
|
$ |
(103,129 |
) |
|
|
(94,741 |
) |
Net loss attributable
to common stockholders per common share — basic and
diluted |
$ |
(0.34 |
) |
|
$ |
(0.40 |
) |
|
$ |
(0.72 |
) |
|
|
(0.75 |
) |
Weighted‑average common
shares outstanding — basic and diluted |
|
143,000,718 |
|
|
|
129,122,175 |
|
|
|
142,886,614 |
|
|
|
127,160,943 |
|
TABLE 2 |
Amicus Therapeutics, Inc. |
Consolidated Balance Sheets |
(Unaudited) |
(in thousands, except share and per share
amounts) |
|
|
|
|
|
|
June 30, |
December 31, |
|
|
|
2017 |
|
|
2016 |
|
Assets |
|
|
|
|
Current assets: |
|
|
|
|
Cash and cash
equivalents |
|
$ |
37,394 |
|
|
$ |
187,026 |
|
Investments in
marketable securities |
|
|
189,838 |
|
|
|
143,325 |
|
Accounts
receivable |
|
|
3,786 |
|
|
|
1,304 |
|
Inventories |
|
|
3,948 |
|
|
|
3,416 |
|
Prepaid expenses and
other current assets |
|
|
6,023 |
|
|
|
4,993 |
|
Total current
assets |
|
|
240,989 |
|
|
|
340,064 |
|
Property and equipment,
less accumulated depreciation of $ 13,951 and $12,495 at June 30,
2017 and December 31, 2016, respectively |
|
|
10,471 |
|
|
|
9,816 |
|
In-process research
& development |
|
|
486,700 |
|
|
|
486,700 |
|
Goodwill |
|
|
197,797 |
|
|
|
197,797 |
|
Other non-current
assets |
|
|
3,009 |
|
|
|
2,468 |
|
Total
Assets |
|
$ |
938,966 |
|
|
$ |
1,036,845 |
|
|
|
|
|
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts payable,
accrued expenses, and other current liabilities |
|
$ |
35,645 |
|
|
$ |
41,008 |
|
Deferred
reimbursements, current portion |
|
|
18,850 |
|
|
|
13,850 |
|
Contingent
consideration payable, current portion |
|
|
46,188 |
|
|
|
56,101 |
|
Total current
liabilities |
|
|
100,683 |
|
|
|
110,959 |
|
Deferred
reimbursements |
|
|
16,906 |
|
|
|
21,906 |
|
Convertible
notes |
|
|
159,171 |
|
|
|
154,464 |
|
Contingent
consideration payable |
|
|
219,162 |
|
|
|
213,621 |
|
Deferred income
taxes |
|
|
173,869 |
|
|
|
173,771 |
|
Other non-current
liability |
|
|
2,283 |
|
|
|
1,973 |
|
Commitments and
contingencies |
|
|
|
|
Stockholders’
equity: |
|
|
|
|
Common stock, $0.01 par
value, 250,000,000 shares authorized, 143,371,243 and
142,691,986 shares issued and outstanding at June 30, 2017
and December 31, 2016, respectively |
|
|
1,485 |
|
|
|
1,480 |
|
Additional paid-in
capital |
|
|
1,132,229 |
|
|
|
1,120,156 |
|
Accumulated other
comprehensive loss: |
|
|
|
|
Foreign currency
translation adjustment, less tax expense of $ 1,293 at June 30,
2017 and December 31, 2016 |
|
|
(192 |
) |
|
|
1,945 |
|
Unrealized gain on
available-for securities |
|
|
31 |
|
|
|
102 |
|
Warrants |
|
|
16,076 |
|
|
|
16,076 |
|
Accumulated
deficit |
|
|
(882,737 |
) |
|
|
(779,608 |
) |
Total stockholders’
equity |
|
|
266,892 |
|
|
|
360,151 |
|
Total
Liabilities and Stockholders’ Equity |
|
$ |
938,966 |
|
|
$ |
1,036,845 |
|
FOLD–G
CONTACTS:
Investors/Media:
Amicus Therapeutics
Sara Pellegrino, IRC
Senior Director, Investor Relations
spellegrino@amicusrx.com
(609) 662-5044
Media:
W2O Group
Brian Reid
breid@w2ogroup.com
(212) 257-6725
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