Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology
company focused on discovering, developing and delivering novel
medicines for rare metabolic diseases, today announced financial
results for the second quarter ended June 30, 2019. The Company
also summarized recent program updates and reiterated its full-year
2019 guidance.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, Inc. stated, “2019 has been a great
period of growth and achievement at Amicus as we continue to build
a leading global rare disease biotechnology company. We are on
track to meet or exceed all of our key strategic priorities for the
year - continued success for Galafold, tremendous momentum in our
Phase 3 Pompe clinical study, positive interim Phase 1/2 clinical
data for our first gene therapy for CLN6 Batten disease, and an
industry leading rare disease gene therapy portfolio. We are in a
very strong position to deliver on our mission for patients and
shareholders.”
Corporate Highlights for 2Q19 and Early
3Q19
- Global revenue for Fabry precision medicine Galafold in
the second quarter of 2019 was $44.1 million and continues to track
toward full-year 2019 revenue guidance of $160 million to $180
million. Now expecting to exceed 1,000 patients on therapy well
before year-end. Second quarter revenue represented a
year-over-year increase of 107% from total revenue of $21.3 million
in the second quarter of 2018, and a quarter over-quarter increase
of 30% from total revenue of $34.0 million in the first quarter of
2019. As of June 30, 2019, Galafold represented an
estimated 24% of global market share of treated amenable patients.
Global compliance and adherence rates continue to exceed 90%.
- Enrollment momentum in ongoing pivotal PROPEL study in
Pompe disease at participating sites in 29 countries. This
global study at the leading Pompe centers in the world is on track
to achieve full enrollment of ~100 patients by year-end 2019. The
study is now more than a majority enrolled. The Pompe patient and
physician community around the world continues to show strong
interest in AT-GAA, which has U.S. Breakthrough Therapy
Designation, as the next potential standard of care.
- Positive interim Phase 1/2 clinical data for gene
therapy in CLN6 Batten disease. The results for our
AAV-CLN6 gene therapy showed a positive impact on motor and
language function in seven of eight patients with available
efficacy data (treated from 16 to 25 months), compared to a natural
history dataset, as well as in comparisons within sibling
pairs.
- Strategic partnerships with Catalent Biologics and
Thermo Fisher for clinical and commercial gene therapy
manufacturing. Brammer Bio, a part of Thermo Fisher, is
assisting with clinical and commercial-scale capabilities for
Amicus gene therapy programs in CLN6 and additional forms of Batten
disease. Paragon Gene Therapy, a unit of Catalent Biologics, will
support clinical manufacturing capabilities and capacity for
multiple active preclinical lysosomal disorder programs in
collaboration with Penn.
- Major expansion of gene therapy collaboration with the
Perelman School of Medicine at the University of Pennsylvania
(Penn). Amicus now has exclusive worldwide rights to
Penn’s next generation gene therapy technologies for a majority of
lysosomal disorders and 12 additional rare diseases, including Rett
Syndrome, Angelman Syndrome, Myotonic Dystrophy and select other
muscular dystrophies.
- Positive initial proof-of-concept for preclinical Pompe
gene therapy data at American Society of Cell and Gene Therapy
meeting. Positive preclinical results showed robust uptake
and glycogen reduction in multiple tissues, including brain and
spinal cord, and were a key driver for the expanded Amicus-Penn
collaboration. Additional Pompe preclinical studies and selection
of a clinical candidate are on track in 2019.
- Exclusive license for Japanese rights to Ultragenyx’s
(Nasdaq: RARE) Mepsevii™ (vestronidase alfa), an enzyme replacement
therapy (ERT) for the lysosomal disorder Mucopolysaccharidosis type
VII (MPS VII, Sly syndrome). Amicus will leverage its
existing infrastructure, relationships, and experience in clinical
development, regulatory approvals, and commercialization in Japan.
Mepsevii™ is currently approved for the treatment of children and
adults with MPS VII in the U.S., EU, and Brazil. Amicus plans to
use the existing Mepsevii data package, as well as data from an
ongoing investigator-sponsored trial in Japan, to support a J-NDA
submission.
- Strong financial position to continue executing the
Galafold launch, advance development programs and build leadership
position in gene therapy science, development and manufacturing
capabilities and capacity. The current cash position of
$575.7 million at June 30, 2019, which includes $189.0 million in
net proceeds raised from our June 2019 public offering of common
stock, is expected to fund ongoing operations into 2021.
2Q19 Financial Results
- Total revenue in the second quarter 2019 was $44.1 million, a
year-over-year increase of 107% from total revenue of $21.3 million
in the second quarter of 2018, and a quarter over-quarter increase
of 30% from total revenue of $34.0 million in the first quarter of
2019. This includes year-over-year operational
revenue growth measured at constant currency exchange rates of
115%, offset by a negative currency impact of $1.7M, or 8%.
- Cash, cash equivalents, and marketable securities totaled
$575.7 million at June 30, 2019, compared to $504.2 million at
December 31, 2018.
- Total operating expenses of $115.2 million for the second
quarter of 2019 increased as compared to $65.1 million in the
second quarter of 2018, reflecting continued investments in the
Galafold launch, Pompe clinical study program, and our gene therapy
pipeline.
- Net loss was $84.6 million, or $0.36 per share, compared to a
net loss of $61.8 million, or $0.33 per share, for the second
quarter 2018.
2019 Key Strategic
Priorities
- Nearly double annual worldwide revenue for Galafold (FY19
guidance of $160 million to $180 million) with over 1,000 Fabry
patients on Galafold by year end.
- Complete enrollment in pivotal Phase 3 PROPEL clinical study in
Pompe disease and report additional Phase 2 data.
- Report additional two-year results from Phase 1/2 clinical
study in CLN6 Batten disease and complete enrollment in ongoing
CLN3 Batten disease Phase 1/2 study.
- Establish preclinical proof of concept for Fabry and Pompe gene
therapies.
- Maintain a strong financial position.
2019 Financial Guidance
For the full-year 2019 the Company anticipates
total Galafold revenue of $160 million to $180 million. Growth in
2019 is expected to be driven by continued growth in EU markets,
further geographic expansion, and successful adoption in the first
full year of launch in the U.S. and Japan. For the remainder of the
year, consistent with Galafold adoption trends and ordering
patterns seen in previous years, Amicus expects flat to moderate
revenue growth in the third quarter and higher revenue growth in
the fourth quarter of 2019.
Cash, cash equivalents, and marketable
securities totaled $575.7 million at June 30, 2019. The Company
expects to end 2019 with approximately $400 million in cash on
hand. The current cash position is anticipated to fund ongoing
operations into 2021.
Anticipated 2019 Milestones by
Program
Galafold Oral Precision Medicine for
Fabry Disease
- Continued growth in global market share of treated amenable
patients.
- Geographic expansion following recent approval in
Argentina
- On track to meet full-year 2019 revenue guidance range of $160
million to $180 million.
- Target of 1,000+ patients on Galafold before year-end.
AT-GAA for Pompe Disease
- Initial 6-month data in additional ERT-switch patients (Cohort
4) and full 24-month data from the first three cohorts in Phase 1/2
ATB200-02 clinical study at World Muscle Society, October 1-5, 2019
(Copenhagen, Denmark)
- Retrospective natural history study data in approximately 100
ERT-treated Pompe patients.
- Additional supportive studies, including an open-label study in
pediatric patients.
- Full enrollment in Phase 3 PROPEL clinical study.
- Advance agreed upon CMC requirements to support BLA.
Gene Therapy Pipeline:
- Detailed data presentation of interim clinical results in CLN6
Batten disease at Amicus Analyst Day on October 10, 2019 (New York,
NY) and in a poster at Child Neurology Society Annual Meeting,
October 23-26, 2019 (Charlotte, NC).
- Advancement of clinical, regulatory and manufacturing
activities for CLN6 Batten disease
- High dose cohort on track to commence in Phase 1/2 clinical
study of CLN3 Batten disease with full enrollment by year end.
- Additional preclinical data including next-generation gene
therapies for Fabry and Pompe.
- Continued advancement of preclinical gene therapy programs in
CLN8 and CLN1 Batten disease.
- Selection of Pompe AAV gene therapy clinical candidate to move
into IND-enabling studies.
- Selection of Amicus site for late process development and
manufacturing facilities.
Conference Call and
WebcastAmicus Therapeutics will host a conference call and
audio webcast today, August 8, 2019 at 8:30 a.m. ET to discuss the
second quarter 2019 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: 8871329.
A live audio webcast can also be accessed via
the Investors section of the Amicus Therapeutics corporate website
at http://ir.amicusrx.com/, and will be archived for 30 days. Web
participants are encouraged to register on the website 15 minutes
prior to the start of the call. A replay of the call will be
available for seven days beginning at 11:30 a.m. ET on August 8,
2019. Access numbers for this replay are 855-859-2056 (U.S./Canada)
and 404-537-3406 (international); conference ID: 8871329.
About Galafold
Galafold® (migalastat) 123 mg capsules is an oral
pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A)
for the treatment of Fabry disease in adults who have
amenable GLA variants. In these patients, Galafold works
by stabilizing the body’s own dysfunctional enzyme so that it can
clear the accumulation of disease substrate. Globally, Amicus
Therapeutics estimates that approximately 35 to 50 percent of Fabry
patients may have amenable GLA variants, though
amenability rates within this range vary by geography. Galafold is
approved in Argentina, Australia, Canada, European Union,
Israel, Japan, South Korea, Switzerland and the U.S.
U. S. INDICATIONS AND USAGE Galafold is
indicated for the treatment of adults with a confirmed diagnosis of
Fabry disease and an amenable galactosidase alpha gene (GLA)
variant based on in vitro assay data.
This indication is approved under accelerated approval based on
reduction in kidney interstitial capillary cell
globotriaosylceramide (KIC GL-3) substrate. Continued approval for
this indication may be contingent upon verification and description
of clinical benefit in confirmatory trials.
U.S. IMPORTANT SAFETY INFORMATION
ADVERSE REACTIONS The most common adverse
reactions reported with Galafold (≥10%) were headache,
nasopharyngitis, urinary tract infection, nausea and pyrexia.
USE IN SPECIFIC POPULATIONS There is
insufficient clinical data on Galafold use in pregnant women to
inform a drug-associated risk for major birth defects and
miscarriage. Advise women of the potential risk to a fetus.
It is not known if Galafold is present in human milk. Therefore,
the developmental and health benefits of breastfeeding should be
considered along with the mother’s clinical need for Galafold and
any potential adverse effects on the breastfed child from Galafold
or from the underlying maternal condition.
Galafold is not recommended for use in patients with severe
renal impairment or end-stage renal disease requiring dialysis.
The safety and effectiveness of Galafold have not been
established in pediatric patients.
To report Suspected Adverse Reactions, contact Amicus
Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088
or www.fda.gov/medwatch.
For additional information about Galafold, including the full
U.S. Prescribing Information, please
visit https://www.amicusrx.com/pi/Galafold.pdf.
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 global, patient-dedicated
biotechnology company focused on discovering, developing and
delivering novel high-quality medicines for people living with rare
metabolic diseases. With extraordinary patient focus, Amicus
Therapeutics is committed to advancing and expanding a robust
pipeline of cutting-edge, first- or best-in-class medicines for
rare metabolic diseases. For more information please visit the
company’s website at www.amicusrx.com, and follow on Twitter and
LinkedIn.
Non-GAAP Financial
MeasuresOperational revenue growth excluding the impact of
foreign currency exchange rates is a non-GAAP financial measure and
should not be considered a replacement for, and should be read
together with, the most comparable GAAP financial
measure.
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, manufacturing and supply plans, financing
plans, and the projected revenues and 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, Japan, the US and 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; the potential that we may not be
able to manufacture or supply sufficient clinical or commercial
products; and the potential that we will need additional funding to
complete all of our studies and manufacturing. Further, the results
of earlier preclinical studies and/or clinical trials may not be
predictive of future results. With respect to statements regarding
projections of the Company's revenue and 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 Annual Report on Form 10-K for the year ended December 31,
2018. 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, IRCVice President, Investor Relations
and Corporate Communicationsspellegrino@amicusrx.com (609)
662-5044
Media:Amicus TherapeuticsMarco Winkler
Director, Corporate Communications mwinkler@amicusrx.com(609)
662-2798
FOLD–G
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, |
|
2019 |
|
2018 |
|
2019 |
|
2018 |
Revenue: |
|
|
|
|
|
|
|
Net product sales |
$ |
44,130 |
|
|
$ |
21,309 |
|
|
$ |
78,176 |
|
|
$ |
38,005 |
|
Cost of goods sold |
5,367 |
|
|
3,135 |
|
|
9,422 |
|
|
5,750 |
|
Gross profit |
38,763 |
|
|
18,174 |
|
|
68,754 |
|
|
32,255 |
|
Operating expenses: |
|
|
|
|
|
|
|
Research and development |
70,981 |
|
|
34,660 |
|
|
135,574 |
|
|
75,458 |
|
Selling, general, and administrative |
42,578 |
|
|
29,172 |
|
|
86,881 |
|
|
56,568 |
|
Changes in fair value of contingent consideration payable |
480 |
|
|
300 |
|
|
1,863 |
|
|
1,400 |
|
Depreciation and amortization |
1,154 |
|
|
973 |
|
|
2,145 |
|
|
1,942 |
|
Total operating expenses |
115,193 |
|
|
65,105 |
|
|
226,463 |
|
|
135,368 |
|
Loss from operations |
(76,430 |
) |
|
(46,931 |
) |
|
(157,709 |
) |
|
(103,113 |
) |
Other income (expense): |
|
|
|
|
|
|
|
Interest income |
2,599 |
|
|
2,913 |
|
|
5,238 |
|
|
4,650 |
|
Interest expense |
(4,625 |
) |
|
(4,560 |
) |
|
(11,079 |
) |
|
(9,048 |
) |
Loss on exchange of convertible notes |
(4,501 |
) |
|
— |
|
|
(40,624 |
) |
|
— |
|
Change in fair value of derivatives |
— |
|
|
(7,600 |
) |
|
— |
|
|
(2,739 |
) |
Other income (expense) |
(877 |
) |
|
(5,316 |
) |
|
209 |
|
|
(2,554 |
) |
Loss before income tax |
(83,834 |
) |
|
(61,494 |
) |
|
(203,965 |
) |
|
(112,804 |
) |
Income tax (expense)
benefit |
(717 |
) |
|
(339 |
) |
|
(885 |
) |
|
1,053 |
|
Net loss attributable
to common stockholders |
$ |
(84,551 |
) |
|
$ |
(61,833 |
) |
|
$ |
(204,850 |
) |
|
$ |
(111,751 |
) |
Net loss attributable to
common stockholders per common share — basic and diluted |
$ |
(0.36 |
) |
|
$ |
(0.33 |
) |
|
$ |
(0.91 |
) |
|
$ |
(0.61 |
) |
Weighted-average common shares
outstanding — basic and diluted |
238,089,824 |
|
188,621,423 |
|
225,848,013 |
|
182,303,128 |
TABLE 2
Amicus
Therapeutics, Inc.Consolidated Balance
Sheets(Unaudited)(in thousands,
except share and per share amounts)
|
June 30, 2019 |
|
December 31, 2018 |
Assets |
|
|
|
Current assets: |
|
|
|
Cash and cash equivalents |
$ |
220,578 |
|
|
$ |
79,749 |
|
Investments in marketable securities |
355,078 |
|
|
424,403 |
|
Accounts receivable |
28,709 |
|
|
21,962 |
|
Inventories |
10,395 |
|
|
8,390 |
|
Prepaid expenses and other current assets |
20,116 |
|
|
16,592 |
|
Total current assets |
634,876 |
|
|
551,096 |
|
Operating lease right-of-use assets, less accumulated amortization
of $2,641 and $0 at June 30, 2019 and December 31, 2018,
respectively |
35,052 |
|
|
— |
|
Property and equipment, less accumulated depreciation of $16,890
and $15,671 at June 30, 2019 and December 31, 2018,
respectively |
15,273 |
|
|
11,375 |
|
In-process research & development |
23,000 |
|
|
23,000 |
|
Goodwill |
197,797 |
|
|
197,797 |
|
Other non-current assets |
12,035 |
|
|
6,683 |
|
Total
Assets |
$ |
918,033 |
|
|
$ |
789,951 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
Current liabilities: |
|
|
|
Accounts payable, accrued expenses, and other current
liabilities |
$ |
84,119 |
|
|
$ |
80,625 |
|
Deferred reimbursements |
2,750 |
|
|
5,500 |
|
Operating lease liabilities |
2,678 |
|
|
— |
|
Total current liabilities |
89,547 |
|
|
86,125 |
|
Deferred reimbursements |
11,406 |
|
|
10,156 |
Convertible notes |
2,070 |
|
|
175,006 |
Senior secured term loan |
146,994 |
|
|
146,734 |
Contingent consideration payable |
21,247 |
|
|
19,700 |
Deferred income taxes |
6,465 |
|
|
6,465 |
Operating lease liabilities |
36,259 |
|
|
— |
|
Other non-current liabilities |
3,987 |
|
|
2,853 |
Total liabilities |
317,975 |
|
|
447,039 |
|
Commitments and
contingencies |
|
|
|
Stockholders’ equity: |
|
|
|
Common stock, $0.01 par value,
500,000,000 shares authorized, 254,513,522 and 189,383,924 shares
issued and outstanding at June 30, 2019 and December 31, 2018,
respectively |
2,589 |
|
|
1,942 |
|
Additional paid-in
capital |
2,201,447 |
|
|
1,740,061 |
|
Accumulated other
comprehensive loss: |
|
|
|
Foreign currency translation adjustment |
352 |
|
|
495 |
|
Unrealized gain (loss) on available-for-sale securities |
355 |
|
|
(427 |
) |
Warrants |
12,387 |
|
|
13,063 |
|
Accumulated deficit |
(1,617,072 |
) |
|
(1,412,222 |
) |
Total stockholders’
equity |
600,058 |
|
|
342,912 |
|
Total Liabilities and
Stockholders’ Equity |
$ |
918,033 |
|
|
$ |
789,951 |
|
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