260+ Fabry Patients on Reimbursed
Galafold™ (Migalastat) and 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 third quarter ended September
30, 2017. The Company also summarized recent program updates and
reiterated full-year 2017 financial guidance.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, stated, “During the third quarter
we continued to successfully execute across our strategic
priorities for our core programs in Fabry and Pompe. We are pleased
with the significant growth and expansion of our international
Galafold launch, and the number of patients treated with this
precision medicine for Fabry disease. In Pompe disease, the data
cascade for our novel treatment paradigm ATB200/AT2221 has
continued to exceed our expectations in terms of the consistency,
durability and magnitude of effect across patients and across
functional outcomes, key disease biomarkers and safety. There has
been extraordinary patient and physician demand for this important
potential treatment option. We are committed to increasing access
to this investigational medicine for as many people as possible
living with Pompe as soon as we can.”
Amicus is on track to achieve four key strategic
priorities between now and the end of 2017:
1) Target of 300 patients on reimbursed Galafold;2)
New Drug Application (NDA) submission for migalastat to the U.S.
FDA;3) Continued advancements with the Pompe program,
including now ongoing collaborative discussions with regulators to
determine the best and fastest pathway; and4) A strong
balance sheet with more than 18 months of cash at year-end.
Third Quarter 2017 Financial
Results
- Total revenue in the third quarter 2017 was approximately $10.9
million, a sequential increase of 51.4% from total revenue of $7.2
million in the second quarter 2017, and a year-over-year increase
of 419% from total revenue of $2.1 million in the third quarter
2016. 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
$426.6 million at September 30, 2017 compared to $330.4 million at
December 31, 2016.
- Total operating expenses increased to $284.3 million compared
to $46.7 million for the third quarter 2016 primarily from non-cash
charges related to the Phase 3 ESSENCE study in epidermolysis
bullosa (EB).
- Operating expenses, as adjusted, excludes the impact of the
non-cash charges related to the ESSENCE study, were $73.5 million,
representing a $26.8 million increase over the third quarter of
2016 primarily due to increased investments in the Pompe and EB
programs as well as increased investment in the Galafold commercial
launch.
- Net cash spend was $147.3 million for the nine months ending
September 30, 2017.
- Net loss was $111.7 million, or $0.69 per share, compared to a
net loss of $46.7 million, or $0.33 per share, for the third
quarter 2016. Net loss, as adjusted excludes the impact of the
non-cash charges related to the ESSENCE study was $65.6 million or
$0.41 per share.
2017 Financial Guidance
Cash, cash equivalents, and marketable
securities totaled $426.6 million at September 30, 2017 compared to
$330.4 million at December 31, 2016. The current cash position
includes $243.0 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 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. Regulatory authorities in the European Union,
Switzerland, Israel, Canada and Australia have granted full
approval for migalastat under the trade name Galafold. The EU
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:
- More than 260 patients (naïve and ERT-switch) on reimbursed
Galafold as of October 31, 2017
- 13 countries with reimbursement (commercial or EAP) including
the top four largest EU countries
- Reimbursement dossiers submitted and pricing discussions are
now underway in 12 countries
- Target of 300 patients treated with reimbursed Galafold on
track for year-end 2017
Global Regulatory Updates:
- Four additional approvals secured outside the EU (Switzerland,
Israel, Canada and Australia)
- Regulatory submissions completed in seven additional countries
outside the EU, including Japan
- NDA submission to U.S. FDA to be based on existing data on
track for 4Q17
Anticipated Upcoming Fabry Disease Program Milestones:
- Commercial launch and EAPs in additional international
countries
- Additional regulatory submissions including a U.S. NDA
(4Q17)
- Regulatory decision in Japan (1H18)
- Final preclinical data and announcement of path forward for
novel Amicus Fabry ERT cell line for Fabry patients with
non-amenable mutations (1Q18)
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.
Additional positive data were reported in October 2017 from an
ongoing global Phase 1/2 clinical study (ATB200-02) to evaluate
safety, tolerability, pharmacokinetics (PK), and pharmacodynamics
(PD) of ATB200/AT2221 across three cohorts, including ambulatory
ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients
(Cohort 2), and ERT-naïve patients (Cohort 3). The Company has
commenced collaborative discussions with U.S. and EU regulators
regarding the best and fastest pathway forward for this novel
treatment option, and continues to anticipate a Pompe regulatory
pathway update in the first half of
2018.
In order to continue to build a robust data set
and to meet the needs of the Pompe community, Amicus announced the
following four key status updates for this important program:
- A retrospective natural history study of Pompe patients treated
with approved standard of care ERT has been initiated at leading
global Pompe disease treatment centers (POM-002 Study)
- A prospective observational study has also been initiated in
Pompe patients currently receiving approved standard of care ERT at
leading global Pompe disease treatment centers (POM-003
Study)
- All engineering runs successfully completed and GMP production
of ATB200 has commenced at the large commercial scale (1,000
Liters)
- Analytical and in vivo (preclinical) comparability studies
completed between the 250 Liter and 1,000 Liter scale
Anticipated Upcoming Pompe Disease Program
Milestones:
- Ongoing discussions with U.S. and EU regulators
- Additional data from ATB200-02 clinical study at 14th Annual
WORLDSymposium™ (February 5-9, 2018)
- Pompe regulatory pathway update (1H18)
SD-101 for Epidermolysis Bullosa
(EB)
During the third quarter Amicus reported that
top-line data from the randomized, double-blind, placebo-controlled
Phase 3 clinical study (ESSENCE, SD-005) to assess the efficacy and
safety of the novel topical wound-healing agent SD-101 did not meet
the primary endpoints or secondary endpoints in participants with
EB. Based on these top-line data Amicus has no current plans to
invest in any additional clinical studies or commercial preparation
activities for SD-101. The Company continues to make SD-101
available to all patients currently enrolled in the ongoing
extension study (SD-006).
Conference Call and
WebcastAmicus Therapeutics will host a conference call and
audio webcast today, November 8, 2017 at 8:30 a.m. ET to discuss
third 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); conference ID: 5995789.
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); conference ID: 5995789.
Non-GAAP Financial Measures
In addition to the United States generally
accepted accounting principles (GAAP) results, this earnings
release contains non-GAAP financial measures that we believe, when
considered together with the GAAP information, provides useful
information to investors that promotes a more complete
understanding of our operating results and financial position for
the current period. Management uses these non-GAAP financial
measures internally for planning, forecasting, evaluating and
allocating resources to the Company's programs. The non-GAAP
results exclude the impact of the following GAAP items specifically
related to the Phase 3 ESSENCE study in EB: changes in fair value
of contingent consideration, impairment of assets and adjustments
to income tax expense. These non-GAAP financial measures should be
considered in addition to, and not as replacements for, or superior
to, measures of financial performance prepared in accordance with
GAAP. Please refer to the attached Reconciliation of Non-GAAP
Financial Measures for explanations of the amounts adjusted to
arrive at non-GAAP total operating expense, net loss attributable
to common stockholders and net loss attributable to common
stockholders per common share – basic and diluted for the three
month period ended September 30, 2017.
EU 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, 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
other geographies, 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 September 30, 2017, filed
today. 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.
CONTACTS:
Investors/Media:Amicus
TherapeuticsSara Pellegrino, IRCSenior Director, Investor
Relationsspellegrino@amicusrx.com (609) 662-5044
Media: W2O GroupBrian
Reidbreid@w2ogroup.com(212) 257-6725
|
TABLE 1 |
Amicus Therapeutics, Inc. |
Consolidated Statements of
Operations |
(Unaudited) |
(in thousands, except share and per share
amounts) |
|
|
Three Months Ended September
30, |
|
Nine Months Ended September
30, |
|
|
2017 |
|
|
|
2016 |
|
|
|
2017 |
|
|
|
2016 |
|
Revenue: |
|
|
|
|
|
|
|
Net product sales |
$ |
10,874 |
|
|
$ |
2,127 |
|
|
$ |
22,201 |
|
|
$ |
2,127 |
|
Cost of goods sold |
|
1,790 |
|
|
|
344 |
|
|
|
3,626 |
|
|
|
344 |
|
Gross
Profit |
|
9,084 |
|
|
|
1,783 |
|
|
|
18,575 |
|
|
|
1,783 |
|
Operating
Expenses: |
|
|
|
|
|
|
|
Research and
development |
|
40,641 |
|
|
|
32,457 |
|
|
|
103,502 |
|
|
|
74,163 |
|
Selling, general and
administrative |
|
21,647 |
|
|
|
17,469 |
|
|
|
60,090 |
|
|
|
52,470 |
|
Changes in fair value
of contingent consideration payable |
|
(244,250 |
) |
|
|
(4,110 |
) |
|
|
(238,622 |
) |
|
|
9,228 |
|
Loss on impairment of
assets |
|
465,427 |
|
|
|
- |
|
|
|
465,427 |
|
|
|
- |
|
Restructuring
charges |
|
- |
|
|
|
11 |
|
|
|
- |
|
|
|
69 |
|
Depreciation |
|
851 |
|
|
|
896 |
|
|
|
2,486 |
|
|
|
2,336 |
|
Total operating
expenses |
|
284,316 |
|
|
|
46,723 |
|
|
|
392,883 |
|
|
|
138,266 |
|
Loss from
operations |
|
(275,232 |
) |
|
|
(44,940 |
) |
|
|
(374,308 |
) |
|
|
(136,483 |
) |
Other income
(expenses): |
|
|
|
|
|
|
|
Interest income |
|
1,190 |
|
|
|
460 |
|
|
|
2,702 |
|
|
|
1,098 |
|
Interest expense |
|
(4,351 |
) |
|
|
(1,517 |
) |
|
|
(12,820 |
) |
|
|
(3,517 |
) |
Other income
(expense) |
|
2,044 |
|
|
|
(910 |
) |
|
|
5,054 |
|
|
|
(3,199 |
) |
Loss before income tax
benefit |
|
(276,349 |
) |
|
|
(46,907 |
) |
|
|
(379,372 |
) |
|
|
(142,101 |
) |
Income tax benefit |
|
164,683 |
|
|
|
253 |
|
|
|
164,578 |
|
|
|
706 |
|
Net loss
attributable to common stockholders |
$ |
(111,666 |
) |
|
$ |
(46,654 |
) |
|
$ |
( 214,794 |
) |
|
$ |
(141,395 |
) |
Net loss attributable
to common stockholders per common share — basic and
diluted |
$ |
(0.69 |
) |
|
$ |
(0.33 |
) |
|
$ |
(1.44 |
) |
|
$ |
(1.07 |
) |
Weighted‑average common
shares outstanding — basic and diluted |
|
160,796,841 |
|
|
|
140,656,109 |
|
|
|
148,963,864 |
|
|
|
131,675,690 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
TABLE 2 |
Amicus
Therapeutics, Inc. |
Consolidated Balance Sheets |
(Unaudited) |
(in thousands, except share and per share
amounts) |
|
|
|
September 30, |
|
December 31, |
|
|
2017 |
|
2016 |
Assets |
|
|
|
|
Current assets: |
|
|
|
|
Cash and cash
equivalents |
|
$ |
64,133 |
|
|
$ |
187,026 |
|
Investments in
marketable securities, current portion |
|
|
347,388 |
|
|
|
143,325 |
|
Accounts
receivable |
|
|
5,974 |
|
|
|
1,304 |
|
Inventories |
|
|
7,272 |
|
|
|
3,416 |
|
Prepaid expenses and
other current assets |
|
|
6,246 |
|
|
|
4,993 |
|
Total current
assets |
|
|
431,013 |
|
|
|
340,064 |
|
Investments in
marketable securities |
|
|
15,109 |
|
|
|
- |
|
Property and equipment,
less accumulated depreciation of $13,273 and $12,495 at September
30, 2017 and December 31, 2016, respectively |
|
|
9,641 |
|
|
|
9,816 |
|
In-process research
& development |
|
|
23,000 |
|
|
|
486,700 |
|
Goodwill |
|
|
197,797 |
|
|
|
197,797 |
|
Other non-current
assets |
|
|
4,219 |
|
|
|
2,468 |
|
Total
Assets |
|
$ |
680,779 |
|
|
$ |
1,036,845 |
|
|
|
|
|
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts payable,
accrued expenses, and other current liabilities |
|
$ |
53,709 |
|
|
$ |
41,008 |
|
Deferred
reimbursements, current portion |
|
|
6,250 |
|
|
|
13,850 |
|
Contingent
consideration payable, current portion |
|
|
8,200 |
|
|
|
56,101 |
|
Total current
liabilities |
|
|
68,159 |
|
|
|
110,959 |
|
Deferred
reimbursements |
|
|
16,906 |
|
|
|
21,906 |
|
Convertible notes |
|
|
161,635 |
|
|
|
154,464 |
|
Contingent
consideration payable |
|
|
12,900 |
|
|
|
213,621 |
|
Deferred income
taxes |
|
|
9,186 |
|
|
|
173,771 |
|
Other non-current
liability |
|
|
2,313 |
|
|
|
1,973 |
|
Commitments and
contingencies |
|
|
|
|
Stockholders’
equity: |
|
|
|
|
Common stock, $0.01 par
value, 250,000,000 shares authorized 165,491,141 and
142,691,986 shares issued and outstanding at September 30,
2017 and December 31, 2016, respectively |
|
|
1,707 |
|
|
|
1,480 |
|
Additional paid-in
capital |
|
|
1,387,767 |
|
|
|
1,120,156 |
|
Accumulated other
comprehensive loss: |
|
|
|
|
Foreign currency
translation adjustment |
|
|
(1,367 |
) |
|
|
1,945 |
|
Unrealized gain on
available-for-sale securities |
|
|
(101 |
) |
|
|
102 |
|
Warrants |
|
|
16,076 |
|
|
|
16,076 |
|
Accumulated
deficit |
|
|
(994,402 |
) |
|
|
(779,608 |
) |
Total stockholders’
equity |
|
|
409,680 |
|
|
|
360,151 |
|
Total
Liabilities and Stockholders’ Equity |
|
$ |
680,779 |
|
|
$ |
1,036,845 |
|
TABLE 3 |
|
Amicus Therapeutics, Inc. |
Reconciliation of Non-GAAP Financial Measures |
Dollars in Thousands Except Per Share Data |
|
|
Three Months |
|
Ended |
|
September 30, 2017 |
|
|
Total operating
expenses – as reported |
$ |
284,316 |
|
Loss on
impairment of assets related to the |
|
Phase 3
ESSENCE study in EB |
|
465,427 |
|
Changes
in fair value of contingent |
|
consideration payable related to the |
|
Phase 3
ESSENCE study in EB |
|
(254,650 |
) |
Total operating
expenses – as adjusted |
$ |
73,539 |
|
|
|
Net loss attributable
to common stockholders – as reported |
$ |
(111,666 |
) |
Loss on
impairment of assets related to the |
|
Phase 3
ESSENCE study in EB |
|
465,427 |
|
Changes
in fair value of contingent |
|
consideration payable related to the |
|
Phase 3
ESSENCE study in EB |
|
(254,650 |
) |
Income
tax benefit (1) |
|
(164,683 |
) |
Net loss attributable
to common stockholders – as adjusted |
$ |
(65,572 |
) |
|
|
Net loss attributable
to common stockholders per common |
|
share –
basic and diluted – as reported |
$ |
(0.69 |
) |
|
|
Net loss attributable
to common stockholders per common |
|
share –
basic and diluted – as adjusted |
$ |
(0.41 |
) |
|
|
Weighted-average common
shares outstanding – basic and |
|
diluted –
as reported and adjusted |
|
160,796,841 |
|
|
|
(1) Related to the reversal of the deferred tax
liability associated with the Scioderm in process research and
development asset.
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