1Q19 Galafold® (migalastat) Strong Launch Trends Continue with Largest Quarterly Number of Net New Patients Added Globally (150+ Net New Galafold Patients in 1Q19)
1Q19 Galafold Revenue of
Maintaining FY19 Revenue Guidance of
AT-GAA in Pompe Disease Continues to Advance in PROPEL Pivotal Study with Full Enrollment on Track by Year-End
Gene Therapy Focus Advances with Positive Preclinical Pompe Gene Therapy Data and Significant Progress Across All Gene Therapy Programs - On Track to Report Additional Phase 1/2 Clinical Data in CLN6 Batten Disease in Mid-2019 (Q3)
Strong Balance Sheet with $438.3M+ Cash
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Corporate Highlights for 1Q19 and Early 2Q19
- Global revenue for Galafold (migalastat) in the first quarter of 2019 was
$34.0M, in line with management expectations, and continues to track toward full-year 2019 revenue guidance of $160M-$180Mand 1,000+ patients by year-end. The first quarter revenue represented a year-over-year increase of 104% from total revenue of $16.7 millionin the first quarter of 2018.
Patient adoption and wide prescriber experience continues globally for Galafold. Highest number since launch of net new patients added in 1Q19 (150+) with higher than anticipated prescriptions in EU,
Japanand US, and continued global compliance and adherence rates exceeding 90%. Over 200 patient referral forms (PRFs) written now by over 90 physicians in the United States, with continued strong reimbursement from payors, since the launch in August 2018.
- New intellectual property issued for Galafold. The new patent covers a method of treatment and provides additional protection through 2038.
- Positive Initial Proof-of-Concept for Preclinical Pompe Gene Therapy Data. Positive preclinical results showed robust uptake and glycogen reduction in multiple tissues, including brain and spinal cord. Additional preclinical studies underway to select a clinical candidate in 2019. This program is significantly ahead of schedule reflecting a strong collaboration with the
University of Pennsylvania.
- Enrollment momentum in ongoing pivotal PROPEL study in Pompe disease. The PROPEL study is now underway at 24 participating sites and is on track to achieve full enrollment of ~100 patients by year-end 2019. Significant enthusiasm from Pompe patient and physician community continues.
- Additional ERT-Switch Patients (Cohort 4) Fully Enrolled in Pompe Phase 1/2 Clinical Study: Initial 6-month data in Cohort 4, and complete 24-month data in Cohorts 1-3, anticipated in 2H19.
- Additional data from Phase 1/2 CLN6 Batten disease study anticipated 3Q19. A total of 12 patients have received a single administration of AAV9-CLN6 gene therapy (exposure ranging from ~5 to 39 months). The top-line data are expected to include two-year results in approximately half the patients in 3Q19 with more detail at a scientific congress later in 2019.
- Low dose cohort fully enrolled in Phase 1/2 study in CLN3 Batten disease. A total of three patients were dosed in the low dose cohort in the CLN3 Batten disease study with no serious adverse events to date following a single administration of AAV9-CLN3 gene therapy. The high dose cohort is expected to commence in the coming months.
- Robust gene therapy pipeline continues to advance toward important data milestones. Additional preclinical proof-of-concept data in Pompe, initial preclinical data in Fabry, and further advancement of neurologic lysosomal disorder preclinical programs, expected throughout 2019.
- 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 approximately
$438.3 millionat March 31, 2019is expected to fund ongoing operations into at least mid-2021.
First Quarter 2019 Financial Results
- Total revenue in the first quarter 2019 was
$34.0 million, a year-over-year increase of 104% from total revenue of $16.7 millionin the first quarter of 2018. This includes year-over-year operational revenue growth measured at constant currency exchange rates of 114.9%, offset by negative currency impact of $1.8M, or 11.0%.
- Cash, cash equivalents, and marketable securities totaled
$438.3 millionat March 31, 2019, compared to $504.6 millionat December 31, 2018.
- Total operating expenses of
$111.3 millionfor the first quarter of 2019 increased as compared to $70.3 millionin the first quarter of 2018, reflecting continued investments in the Galafold launch, Pompe program, and gene therapy pipeline.
- Net loss was
$120.3 million, or $0.56per share, compared to a net loss of $49.9 million, or $0.28per share, for the first quarter 2018.
2019 Key Strategic Priorities
- Nearly double 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
For the full-year 2019 the Company anticipates total Galafold revenue of
Cash, cash equivalents, and marketable securities totaled
Anticipated 2019 Milestones by Program
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.
- Additional preclinical data including next-generation gene therapies for Fabry and Pompe
- Selection of Pompe AAV gene therapy clinical candidate to move into IND-enabling studies
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.
Non-GAAP Financial Measures
Operational 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.
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)
|Three Months Ended March 31,|
|Net product sales||$||34,046||$||16,696|
|Cost of goods sold||4,055||2,615|
|Research and development||64,593||40,798|
|Selling, general and administrative||44,303||27,396|
|Changes in fair value of contingent consideration payable||1,383||1,100|
|Depreciation and amortization||991||969|
|Total operating expenses||111,270||70,263|
|Loss from operations||(81,279||)||(56,182||)|
|Other income (expense):|
|Loss on exchange of convertible notes||(36,123||)||—|
|Change in fair value of derivatives||—||4,861|
|Loss before income tax||(120,131||)||(51,308||)|
|Income tax (expense) benefit||(168||)||1,392|
|Net loss attributable to common stockholders||$||(120,299||)||$||(49,916||)|
|Net loss attributable to common stockholders per common share — basic and diluted||$||(0.56||)||$||(0.28||)|
|Weighted-average common shares outstanding — basic and diluted||213,519,287||175,977,700|
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
|Cash and cash equivalents||$||96,349||$||79,749|
|Investments in marketable securities||341,978||424,403|
|Prepaid expenses and other current assets||13,592||16,592|
|Total current assets||483,046||551,096|
|Operating and finance lease right-of-use assets, less accumulated amortization of $2,111 and $0 at March 31, 2019 and December 31, 2018, respectively||36,308||—|
|Property and equipment, less accumulated depreciation of $15,794 and $15,671 at March 31, 2019 and December 31, 2018, respectively||13,286||11,375|
|In-process research & development||23,000||23,000|
|Other non-current assets||11,265||6,683|
|Liabilities and Stockholders’ Equity|
|Accounts payable, accrued expenses, and other current liabilities||$||60,596||$||80,625|
|Operating and finance lease liabilities||2,523||—|
|Total current liabilities||65,869||86,125|
|Senior secured term loan||146,766||146,734|
|Contingent consideration payable||20,767||19,700|
|Deferred income taxes||6,465||6,465|
|Operating and finance lease liabilities||36,100||—|
|Other non-current liabilities||3,609||2,853|
|Commitments and contingencies|
|Common stock, $0.01 par value, 500,000,000 shares authorized, 230,180,714 and 189,383,924 shares issued and outstanding at March 31, 2019 and December 31, 2018, respectively||2,347||1,942|
|Additional paid-in capital||1,970,607||1,740,061|
|Accumulated other comprehensive loss:|
|Foreign currency translation adjustment||(1,309||)||495|
|Unrealized gain (loss) on available-for-sale securities||157||(427||)|
|Total stockholders’ equity||451,668||342,912|
|Total Liabilities and Stockholders’ Equity||$||764,702||$||789,951|
Source: Amicus Therapeutics, Inc.