Continued Global Galafold® (Migalastat) Adoption and Expansion – Reaffirms FY18 Revenue Guidance of
Significant Momentum with Pompe Clinical, Regulatory and Manufacturing Activities - GMP Manufacturing Campaigns of ATB200 Drug Substance and Drug Product Completed at 1000L Scale
Conference Call and Webcast Today at
2018 Key Strategic Priorities
- Double global revenue for Galafold (
$75 million - $85 million)
- Secure approvals for migalastat in
Japanand the U.S.
- Achieve clinical, manufacturing and regulatory milestones to advance AT-GAA (also known as ATB200/AT2221) toward global regulatory submissions and approvals as soon as possible
- Develop and expand preclinical pipeline to ensure at least one new clinical program in 2019
- Maintain a strong balance sheet
First Quarter 2018 Financial Results and Full-Year 2018 Financial Guidance
- Total revenue in the first quarter 2018 was
$16.7 million, a year-over-year increase of 300% from total revenue of $4.2 millionin the first quarter of 2018.
- Cash, cash equivalents, and marketable securities totaled
$605.2 millionat March 31, 2018, compared to $358.6 millionat December 31, 2017.
- Total operating expenses increased to
$70.3 millionfor the first quarter 2018 compared to $55.4 millionin the first quarter 2017 reflecting increased investment in Pompe clinical and manufacturing activities as well as Galafold commercial launch and launch preparations.
- Net cash spend was
$48.0 millionfor the first quarter 2018.
- Net loss was
$49.9 million, or $0.28per share, compared to a net loss of $55.0 million, or $0.39per share, for the first quarter 2017.
“The first quarter of 2018 marked another period of continued growth for Galafold and our strongest balance sheet in our Company’s history,” said
2018 Financial Guidance
For the full-year 2018 the Company anticipates total Galafold revenue at the high end of the
The Company continues to expect full-year 2018 net cash spend between
Migalastat for Fabry Disease
Amicus is committed to advancing the highest quality therapies for all people living with Fabry disease. Migalastat is an oral precision medicine intended to treat Fabry disease in patients 16 years or older who have amenable genetic mutations. Regulatory authorities in the
For people with non-amenable mutations who are not eligible for migalastat as an oral precision medicine, the strategy is to advance next-generation therapies such as a novel Fabry ERT (ATB101) co-formulated with migalastat or other innovative technologies that continue to be evaluated.
Global Fabry Updates:
- Pricing and reimbursement secured in 18 countries with first commercial patients treated in multiple new countries in 2018
- Approvals secured in EU,
Australia, Canada, Japan, Israel, South Koreaand Switzerland
- Launch team hired and trained in
Japanto execute upcoming launch
- U.S. leadership, including
Mike Keavany, to serve as Senior Vice President of the U.S. Business, and majority of field team now in place to support planned U.S. launch
- Japanese launch (2Q18)
FDAregulatory decision (3Q18)
Advanced and Targeted GAA (AT-GAA, also known as ATB200/AT2221) for Pompe Disease
AT-GAA 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. During WORLDSymposium™ in
The Company is engaged in ongoing collaborative discussions with U.S. and EU regulators regarding a registration-directed study for full approval, manufacturing activities, and the best and fastest pathway forward for this novel treatment regimen. Amicus is pursuing formal scientific advice from the
Amicus also continues to make progress with clinical and manufacturing activities to support the needs of the Pompe community.
Pompe Clinical Activities
- All 19 patients for whom extension study data have been previously reported continue treatment with AT-GAA in the ongoing Phase 1/2 ATB200-02 clinical study
- Currently enrolling Cohort 4 (up to 10 additional ambulatory ERT-switch patients) in ATB200-02 study
- Supportive studies underway including a retrospective study (POM-002) on the natural history of Pompe disease in up to 100 ERT-treated Pompe patients to help provide context for the ATB200-02 clinical study results; and a prospective observational study (POM-003) to assess safety and functional outcomes in patients currently treated with standard of care ERT, and to serve as a potential run-in for a registration study
Pompe Manufacturing Activities:
- As previously announced,
FDAagreed on comparability between 250L scale and 1000L engineering batches, as well as the testing strategy for demonstrating comparability between 250L scale and 1000L GMP batches
- GMP production runs of ATB200 drug substance and drug product completed at 1,000L commercial scale
Anticipated Upcoming Pompe Program Milestones:
- Full enrollment of additional patients in ATB200-02 clinical study
- Final demonstration of comparability between 1,000L GMP material and 250L material
- Pompe regulatory updates (2Q18 and 3Q18)
- Release of 1,000L GMP material for initiation of registration-directed study (2H18)
- 18-month data from ATB200-02 clinical study (4Q18)
Conference Call and Webcast
An audio webcast can also be accessed via the Investors section of the
Non-GAAP Financial Measures
In addition to
EU Important Safety Information
Treatment 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.
This 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
Vice President, Investor Relations & Corporate Communications
|Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
|Three Months Ended March 31,|
|Net product sales||$||16,696||$||4,169|
|Cost of goods sold||2,615||775|
|Research and development||40,798||30,876|
|Selling, general and administrative||27,396||19,132|
|Changes in fair value of contingent consideration payable||1,100||4,578|
|Total operating expenses||70,263||55,409|
|Loss from operations||(56,182||)||(52,015||)|
|Other income (expense):|
|Change in fair value of derivatives||4,861||-|
|Loss before income tax||(51,308||)||(54,936||)|
|Income tax benefit (expense)||1,392||(56||)|
|Net loss attributable to common stockholders||$||(49,916||)||$||(54,992||)|
|Net loss attributable to common stockholders per common share — basic and diluted||$||(0.28||)||$||(0.39||)|
|Weighted‑average common shares outstanding — basic and diluted||175,977,700||142,770,629|
|Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
|Cash and cash equivalents||$||114,322||$||49,060|
|Investments in marketable securities||448,198||309,502|
|Prepaid expenses and other current assets||10,607||19,316|
|Total current assets||592,033||391,965|
|Investments in marketable securities||42,673||-|
|Property and equipment, less accumulated depreciation of $13,461 and $12,515 at March 31, 2018 and December 31, 2017, respectively||8,910||9,062|
|In-process research & development||23,000||23,000|
|Other non-current assets||5,592||5,200|
|Liabilities and Stockholders’ Equity|
|Accounts payable, accrued expenses, and other current liabilities||$||43,678||$||53,890|
|Contingent consideration payable||8,700||8,400|
|Total current liabilities||140,705||70,040|
|Contingent consideration payable||17,800||17,000|
|Deferred income taxes||6,465||6,465|
|Other non-current liability||2,494||2,346|
|Commitments and contingencies|
|Common stock, $0.01 par value, 250,000,000 shares authorized 187,972,218 and 166,989,790 shares issued and outstanding at March 31, 2018 and December 31, 2017, respectively||1,929||1,721|
|Additional paid-in capital||1,621,479||1,400,758|
|Accumulated other comprehensive loss:|
|Foreign currency translation adjustment||(3,847||)||(1,659||)|
|Unrealized loss on available-for-sale securities||(877||)||(436||)|
|Total stockholders’ equity||521,617||352,850|
|Total Liabilities and Stockholders’ Equity||$||870,005||$||627,024|
Source: Amicus Therapeutics, Inc.