U.S. Galafold® (Migalastat) Fabry Launch Tracking Significantly Ahead of Expectations- 100+ Patients Prescribed Galafold Since August Launch
3Q18 Global Galafold Net Product Sales of
Balance Sheet Strength Sufficient to Fund Operations into at least 2021
Gene Therapy Pipeline Provides 14 New Programs and Future Growth Platform
Conference Call and Webcast Today at
Third Quarter 2018 Financial Results and Full-Year 2018 Financial Guidance
- Total revenue in the third quarter 2018 was
$20.6 million, a year-over-year increase of 89% from total revenue of $10.9 millionin the third quarter of 2017. Third quarter revenue was impacted by uneven ordering patterns over the summer months in Europe.
- Cash, cash equivalents, and marketable securities totaled
$564.4 millionat September 30, 2018, compared to $358.6 millionat December 31, 2017.
- Total operating expenses for the third quarter 2018 were
$172.5 millioncompared to $284.3 millionin the third quarter 2017. The decrease is due primarily to a non-cash impairment charge incurred in 3Q17, partially offset by an upfront payment of $100 millionfor the Celenex asset acquisition which was reflected in the third quarter 2018 as a research and development expense.
- Net cash spend was
$35.2 millionfor the third quarter 2018. Net loss was $159.2 million, or $0.84per share, for the third quarter 2018 compared to a net loss of $111.7 million, or $0.69per share, for the third quarter 2017. Total net operating expenses were $172.5 million, which includes the $100 millionCelenex asset acquisition cost.
“We are very pleased with the momentum of the global Galafold launch,” said
2018 Financial Guidance
For the full-year 2018 the Company reiterated its total Galafold revenue guidance to
Galafold (Migalastat) Oral Precision Medicine for Fabry Disease
Galafold is an oral precision medicine for Fabry disease approved in the EU and other geographies to treat Fabry disease in patients 16 years or older who have amenable genetic mutations. The U.S.
Global Galafold Updates:
FDAapproval on August 10, 2018
- U.S. launch tracking ahead of internal expectations with 103 new patient prescriptions, also known as patient referral forms (PRFs), as of
October 31, 2018. Time to shipment is approximately 60 days, limiting 2018 revenue impact but providing a strong foundation for 2019.
- Launched in
Australiaon November 1following formal listing on life saving drugs program
- Pricing and reimbursement secured in 22 countries
- Approvals secured in eight total geographies including
Australia, Canada, EU, Israel, Japan, South Korea, Switzerland, and United Statesand pending in Taiwan
AT-GAA 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 to stabilize ATB200 while in the circulation to deliver active therapeutic enzyme.
Positive results from a global Phase 1/2 clinical study (ATB200-02) have shown consistent and durable responses across key measures of safety, functional outcomes and biomarkers in both ERT-switch and ERT-naïve Pompe patients following up to 18 months of treatment with AT-GAA. The Company’s strategy is to enhance the body of clinical data for AT-GAA in ongoing studies and the upcoming pivotal study (PROPEL, also referred to as ATB200-03) to deliver this potential new therapy to as many people living with Pompe disease as soon as possible.
Recent and Anticipated AT-GAA Program Milestones:
- Positive 18-month data from ATB200-02 Phase 1/2 clinical study at
World Muscle Society
- 1,000L GMP material released for pivotal study.
- Initiation of PROPEL pivotal study to support full approval in U.S. and EU, and other geographies (4Q18)
- Completion of retrospective natural history study in approximately 100 ERT-treated Pompe patients (4Q18)
- Additional ATB200-02 study data from up to 10 additional ERT-switch patients in Cohort 4 (2019)
- Initiation of studies in additional patient populations, including pediatric patients (2019)
- Update on long-term manufacturing strategy
As part of the Company’s long-term commitment to provide multiple solutions to address the significant unmet needs of the Pompe community, Amicus is also advancing a next-generation gene therapy as a potential cure for Pompe disease.
Gene Therapy Portfolio: 14 New Programs for Rare Metabolic Diseases
During the third quarter and early fourth quarter, Amicus expanded its pipeline [link here] to include 14 new gene therapy programs and future growth platform for rare metabolic diseases. The Company acquired 10 preclinical and clinical stage adeno associated virus (AAV) programs (intrathecal delivery) for neurologic lysosomal storage disorders (LSDs) currently in development at Nationwide Children’s Hospital. In collaboration with the
Gene Therapy Pipeline Highlights:
- Batten Disease: Compelling proof-of-concept demonstrated in preclinical studies in CLN6, CLN3, and CLN8, as well as initial clinical safety and efficacy in a Phase 1/2 study in patients with CLN6.
- Pompe Disease: Early proof-of-principle for Amicus DNA constructs for an optimized gene therapy to address all aspects of Pompe disease including the central nervous sytem, heart, and muscles.
- Fabry Disease: Early proof-of-principle for Amicus DNA constructs for an optimized gene therapy to deliver stable, active enzyme to lysosomes.
Upcoming Gene Therapy Pipeline Milestones in 2018 and 2019:
- First Patient in CLN3 Batten disease Phase 1/2 Study (4Q18)
- Completion of enrollment in CLN6 Batten disease Phase 1/2 study
- Preliminary data from CLN6 Batten disease Phase 1/2 study
- Enrollment of full initial cohort in 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
An audio webcast and slide presentation can also be accessed via the Investors section of the
GalafoldTM (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 & Corporate Communications
Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Net product sales||$||20,596||$||10,874||$||58,601||$||22,201|
|Cost of goods sold||4,310||1,790||10,060||3,626|
|Research and development||138,227||40,641||213,685||103,502|
|Selling, general and administrative||31,867||21,647||88,435||60,090|
|Changes in fair value of contingent consideration payable||1,300||(244,250||)||2,700||(238,622||)|
|Loss on impairment of assets||—||465,427||—||465,427|
|Total operating expenses||172,467||284,316||307,835||392,883|
|Loss from operations||(156,181||)||(275,232||)||(259,294||)||(374,308||)|
|Other income (expense):|
|Change in fair value of derivatives||—||—||(2,739||)||163|
|Other (expense) income||(1,039||)||2,044||(3,593||)||4,891|
|Loss before income tax||(159,214||)||(276,349||)||(272,018||)||(379,372||)|
|Income tax benefit||51||164,683||1,104||164,578|
|Net loss attributable to common stockholders||$||(159,163||)||$||(111,666||)||$||(270,914||)||$||(214,794||)|
|Net loss attributable to common stockholders per common share — basic and diluted||$||(0.84||)||$||(0.69||)||$||(1.47||)||$||(1.44||)|
|Weighted-average common shares outstanding — basic and diluted||189,162,841||160,796,841||184,606,790||148,963,864|
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
|September 30,||December 31,|
|Cash and cash equivalents||$||201,827||$||49,060|
|Investments in marketable securities||362,556||309,502|
|Prepaid expenses and other current assets||16,151||19,316|
|Total current assets||601,034||391,965|
|Property and equipment, less accumulated depreciation of $15,483 and $12,515 at September 30, 2018 and December 31, 2017, respectively||10,659||9,062|
|In-process research & development||23,000||23,000|
|Other non-current assets||6,099||5,200|
|Liabilities and Stockholders’ Equity|
|Accounts payable, accrued expenses, and other current liabilities||$||54,330||$||53,890|
|Contingent consideration payable||8,800||8,400|
|Total current liabilities||65,880||70,040|
|Senior secured term loan||146,622||—|
|Contingent consideration payable||19,300||17,000|
|Deferred income taxes||6,465||6,465|
|Other non-current liabilities||3,029||2,346|
|Commitments and contingencies|
|Common stock, $0.01 par value, 500,000,000 and 250,000,000 shares authorized, 189,254,341 and 166,989,790 shares issued and outstanding at September 30, 2018 and December 31, 2017, respectively||1,941||1,721|
|Additional paid-in capital||1,731,174||1,400,758|
|Accumulated other comprehensive loss:|
|Foreign currency translation adjustment||(875||)||(1,659||)|
|Unrealized gain on available-for-sale securities||(211||)||(436||)|
|Total stockholders’ equity||410,951||352,850|
|Total Liabilities and Stockholders’ Equity||$||838,589||$||627,024|
Source: Amicus Therapeutics, Inc.