310+ People with Fabry Disease Treated with Reimbursed Galafold® at YE17
FY18 Galafold Revenue Guidance of
Regulatory Agreement on Comparability of 1,000L Scale Engineering Material for Pompe
Additional Pompe Phase 1/2 Clinical Study Results to be Presented at WORLDSymposium™ 2018
Key 2017 Accomplishments
- Exceeded “Target 300” goal with more than 310 people treated with reimbursed Galafold™ (migalastat) oral precision medicine for Fabry disease at year-end 2017. Full-year 2017 Galafold revenue totaled approximately
- Completed global regulatory submissions for migalastat in
Japan(J-NDA), the U.S. (NDA), and other key geographies
- Established important clinical proof-of-concept for novel, highly differentiated Pompe treatment regimen ATB200/AT2221 on safety, functional outcomes and key disease biomarkers
- Successfully scaled manufacture of Pompe biologic engineering batches at commercial scale (1,000L) with capacity plans to ensure that entire Pompe population can be served as quickly as possible
- Strengthened balance sheet with total cash, cash equivalents and marketable securities of
$359 millionat December 31, 2017and cash runway into the second half of 2019
Amicus is focused on five key strategic priorities in 2018:
- 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 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
Mr. Crowley will discuss Amicus' corporate objectives and key milestones in a presentation at the 36th Annual
Full-Year 2017 Financial Summary and 2018 Guidance
Amicus recorded approximately
Cash, cash equivalents, and marketable securities totaled approximately
Migalastat for Fabry Disease
Migalastat is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic mutations. Regulatory authorities in the
Amicus is committed to advancing the highest quality therapies for all people living with Fabry disease. For people with non-amenable mutations who are not eligible for migalastat as an oral precision medicine, the Company has established initial preclinical proof-of-concept for a novel Fabry ERT (ATB101) co-formulated with migalastat as part of our CHART® platform.
Global Fabry Updates:
- 310+ patients (naïve and ERT-switch) on reimbursed Galafold as of
December 31, 2017
- Total full-year 2017 revenue of
$36 millionfrom global commercial sales and expanded access programs (EAPs)
- Pricing and reimbursement secured in 15 countries
- Approvals secured in EU,
Australia, Canada, Israel, South Koreaand Switzerland
- Approvals pending in
Japan, U.S. and other key geographies
FDAacceptance of U.S. NDA for filing (1Q18)
- Regulatory decision on Japanese J-NDA (1H18)
- Total full-year 2018 revenue guidance of
$75 million to $85 million
- ATB101 co-formulated with migalastat advancing toward the clinic in 2019
ATB200/AT2221 for Pompe Disease
ATB200/AT2221 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. Throughout 2017, Amicus presented a cascade of positive data from an ongoing global Phase 1/2 clinical study (ATB200-02) to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ATB200/AT2221 across ambulatory ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2), and ERT-naïve patients (Cohort 3).
Amicus continues to assemble the highest quality data package and to scale up manufacturing to meet the needs of the Pompe community. The Company is in the midst of a series of collaborative discussions with U.S. and EU regulators regarding the best and fastest pathway forward for this novel treatment option, and continues to anticipate a Pompe regulatory pathway update in the first half of 2018.
Pompe Program Updates:
- Data collection underway in a retrospective natural history study (POM-002)
- Prospective observational study (POM-003) initiated
- GMP production of ATB200 commenced at the large commercial scale (1,000 Liters)
FDAagreement reached on comparability between 250L scale and 1000L engineering batches FDAagreement reached on testing strategy for demonstrating comparability between 250L scale and 1000L GMP batches
Anticipated Upcoming Pompe Program Milestones:
- Expansion of ongoing ATB200-02 clinical study to include four to six additional ambulatory ERT-switch patients
- Additional data from ATB200-02 clinical study at 14th Annual WORLDSymposium™ (
February 5-9, 2018)
- Final regulatory agreement on manufacturing comparability between 1,000L and 250L GMP scale
- Completion and release for clinic of 1,000L GMP commercial scale material
- Pompe regulatory pathway update (1H18)
- Initiation of larger registration-directed study
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
Senior Director, Investor Relations
Source: Amicus Therapeutics, Inc.