Continued Strong Galafold Launch Trends in Early 2019 - On Track to Achieve FY19 Revenue Guidance of
FY18 Galafold Revenue of
Clinical Data Out to Month 24 and Breakthrough Therapy Designation in Late Onset Pompe Disease Continue to Support AT-GAA as Next Potential Standard of Care
Strong Balance Sheet with $500M+ Cash
Conference Call and Webcast Today at
Corporate Highlights for Full-Year 2018 and Year-to-Date 2019
- More than doubled global revenue for Galafold (migalastat) in 2018. As previously announced, revenue grew from
$36.9 millionin full-year 2017 to $91.2 millionin full-year 2018, exceeding the high end of the full-year 2018 guidance range of $80 million to $90 million.
- Strong Launch Trends Continue in Early 2019. On track to achieve full-year 2019 revenue guidance of
$160M-$180Mand 1,000+ patients on Galafold by year-end.
- Updated AT-GAA clinical data in Pompe disease presented at WORLDSymposium™ in
February 2019. As previously announced, consistent and durable responses continued across key measures of safety, functional outcomes and biomarkers for both ERT-naïve and ERT-switch patients treated with AT-GAA for up to 24 months in the ongoing Phase 1/2 clinical study.
- AT-GAA received Breakthrough Therapy Designation (BTD) in late onset Pompe disease. The BTD was based on clinical efficacy results from the ongoing ATB200-02 Phase 1/2 clinical study, and further strengthens the Company’s conviction in the potential for AT-GAA to become the next standard of care.
- Enrollment momentum in ongoing pivotal PROPEL study in Pompe disease and Phase 1/2 study in CLN3 Batten disease. The PROPEL study is on track to achieve full enrollment by year-end 2019. The first patient remains in the CLN3 Batten disease study with no serious adverse events after more than two months following a single administration of AAV9-CLN3 gene therapy. Further patient dosing is expected in the coming months.
- Robust gene therapy pipeline continues to advance toward important data milestones. Additional two-year data from Phase 1/2 study in CLN6 Batten disease expected mid-year. Preclinical proof-of-concept for Fabry and Pompe gene therapies anticipated throughout 2019.
Global Researchand Gene Therapy Centerof Excellence in Philadelphia. This new headquarters for the global Amicus science organization and the gene therapy leadership team advances the Company’s commitment to world-class science.
- Strong financial position to continue executing the Galafold launch and advance development programs. The current cash position of approximately
$504.2 millionat December 31, 2018is expected to fund ongoing operations into at least mid-2021.
Full-Year 2018 Financial Results
- Total revenue in the full-year 2018 was
$91.2 million, an increase from total revenue of $36.9 millionin the full-year 2017.
- Cash, cash equivalents, and marketable securities totaled
$504.2 millionat December 31, 2018compared to $358.6 millionat December 31, 2017.
- Total operating expenses decreased to
$405.6 millionfor the full-year 2018 compared to $472.7 millionin the full-year 2017. Operating expenses reflecting increased investments in the Galafold launch, Pompe program, and gene therapy pipeline.
- Net cash spend was
$189.3 millionfor the full-year 2018, which was below full-year 2018 net cash spend guidance of $200 million to $225 millionand reflects careful expense management.
- Net loss was
$349.0 million, or $1.88per share, compared to a net loss of $284.0 million, or $1.85per share, for the full-year 2017.
2019 Key Strategic Priorities
- Nearly double again, annual revenue for Galafold (FY19 guidance of
$160M-$180Min worldwide revenue) with 1,000+ Fabry patients on Galafold by year end.
- Complete enrollment in pivotal 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
Cash, cash equivalents, and marketable securities totaled
Anticipated 2019 Milestones by Program
Amicus previously announced full-year 2018 program updates as well as anticipated 2019 program milestones in early
Galafold (migalastat) Oral Precision Medicine for Fabry Disease
- On track to meet full-year 2019 revenue guidance range of
$160 million to $180 million.
AT-GAA for Pompe Disease
- Initial 6-month data in additional ERT-switch patients (Cohort 4) in Phase 1/2 ATB200-02 clinical study.
- 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 study.
- Advance agreed upon CMC requirements to support BLA.
Gene Therapy Pipeline:
- Additional two-year data from CLN6 Batten disease Phase 1/2 study.
- Full enrollment of ongoing CLN3 Batten disease Phase 1/2 study.
- Preclinical data for next-generation gene therapies for Fabry, Pompe and CDD.
- Preclinical work across additional neurologic LSDs.
Conference Call and Webcast
A live audio webcast can also be accessed via the Investors section of the
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,
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
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
For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf.
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 and Corporate Communications
Director, Corporate Communications
Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
|Years Ended December 31,|
|Net product sales||$||91,245||$||36,930||$||4,958|
|Cost of goods sold||14,404||6,236||833|
|Research and development||270,902||149,310||104,793|
|Selling, general and administrative||127,200||88,671||71,151|
|Changes in fair value of contingent consideration payable||3,300||(234,322||)||6,760|
|Loss on impairment of assets||—||465,427||—|
|Total operating expenses||405,618||472,679||186,015|
|Loss from operations||(328,777||)||(441,985||)||(181,890||)|
|Other income (expenses):|
|Change in fair value of derivatives||(2,739||)||—||—|
|Loss on extinguishment of debt||—||—||(13,302||)|
|Other income (expense)||(5,632||)||6,008||(4,793||)|
|Loss before income tax||(349,089||)||(449,121||)||(203,781||)|
|Income tax benefit||94||165,119||3,739|
|Net loss attributable to common stockholders||$||(348,995||)||$||(284,002||)||$||(200,042||)|
|Net loss attributable to common stockholders per common share — basic and diluted||$||(1.88||)||$||(1.85||)||$||(1.49||)|
|Weighted-average common shares outstanding — basic and diluted||185,790,021||153,355,144||134,401,588|
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
|Cash and cash equivalents||$||79,749||$||49,060|
|Investments in marketable securities||424,403||309,502|
|Prepaid expenses and other current assets||16,592||19,316|
|Total current assets||551,096||391,965|
|Property and equipment, less accumulated depreciation of $15,671 and $12,515 at December 31, 2018 and 2017, respectively||11,375||9,062|
|In-process research & development||23,000||23,000|
|Other non-current assets||6,683||5,200|
|Liabilities and Stockholders' Equity|
|Accounts payable, accrued expenses, and other current liabilities||$||80,625||$||53,890|
|Contingent consideration payable||—||8,400|
|Total current liabilities||86,125||70,040|
|Senior secured term loan||146,734||—|
|Contingent consideration payable||19,700||17,000|
|Deferred income taxes||6,465||6,465|
|Other non-current liabilities||2,853||2,346|
|Commitments and contingencies|
|Common stock, $.01 par value, 500,000,000 shares authorized, 189,383,924 shares issued and outstanding at December 31, 2018 Common stock, $.01 par value, 250,000,000 shares authorized, 166,989,790 shares issued and outstanding at December 31, 2017||1,942||1,721|
|Additional paid-in capital||1,740,061||1,400,758|
|Accumulated other comprehensive loss:|
|Foreign currency translation adjustment||495||(1,659||)|
|Unrealized loss on available-for securities||(427||)||(436||)|
|Total stockholders' equity||342,912||352,850|
|Total Liabilities and Stockholders' Equity||$||789,951||$||627,024|
Source: Amicus Therapeutics, Inc.