Amicus Therapeutics Announces Full-Year 2016 Financial Results and Corporate Updates
Growing Momentum for EU Galafold (Migalastat) Launch for Fabry Disease Tracking Toward 300 Patients by Year-End 2017
Target Enrollment Achieved in Phase 1/2 Pompe Study - Additional Data Expected in 2Q17 and 3Q17
Phase 3 EB Program Remains on Track for Topline Data in Mid-2017
"Throughout 2016 we made significant progress with the international launch of our first commercial
product Galafold and continued to advance and expand our robust pipeline of first- and/or best-in-class medicines for people living with devastating rare diseases," stated
2016 Full-Year Financial Results
- Total product revenue in the full year 2016 was approximately
$5.0 million , which represents commercial sales of Galafold (migalastat) inGermany as well as reimbursed Expanded Access Programs (EAPs) in two countries during the third and fourth quarter of 2016. - Cash, cash
equivalents, and marketable securities totaled
$330.4 million atDecember 31, 2016 compared to$214.0 million atDecember 31, 2015 . - Total operating expenses increased to
$186.0 million compared to$130.4 million for the full year 2015 primarily due to increases in commercial costs of the Fabry monotherapy program and manufacturing scale-up on the Pompe program. - Net cash spend was
$154.3 million , within the full-year 2016 guidance of$135-155 million . - Net loss was
$200.0 million , or$1.49 per share, compared to a net loss of$132.1 million , or$1.20 per share, for the full year 2015.
2017 Financial Guidance
Cash, cash equivalents, and marketable securities totaled
Program Highlights
Migalastat for Fabry Disease
Migalastat is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic mutations. As previously announced, the
International Launch and Expanded Access Programs (EAP) Updates:
- 75 patients (naïve and ERT-switch) on reimbursed Galafold as of
February 28, 2017 - 10 countries with reimbursement (commercial or EAP)
- Reimbursement dossiers submitted and pricing discussions are now underway in 14 countries
- Launch commenced in
United Kingdom followingNational Institute for Health and Care Excellence (NICE) publication of final guidance recommending reimbursement of Galafold inEngland - Target of 300 patients treated with reimbursed Galafold by year-end 2017
Regulatory Updates:
- One additional approval secured outside EU (
Switzerland ) - Regulatory submissions completed in six additional territories outside EU
- Phase 3 gastrointestinal (GI) symptoms study protocol nearly complete and detailed feasibility study underway to support
U.S. full approval pathway
Anticipated Upcoming Fabry Disease Program Milestones:
- EU commercial launch in additional countries and EAP in additional territories
- Additional regulatory submissions including a Japanese regulatory submission (J-NDA) targeted for 1H17
U.S. intermediate EAP- Phase 3 gastrointestinal (GI) symptoms study
- Fabry ERT cell line development and program update
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. Positive preliminary data were reported in the fourth quarter of 2016 and during the 13th Annual WORLDSymposium™ in San Diego, CA in
Key Preliminary Data Highlights from ATB200-02 Study in Initial ERT-Switch and ERT-Naïve Patients:
- No infusion-associated reactions following 150+ infusions in initial patients treated for a maximum of 36 weeks (n=13)
- Available PK and PD (muscle and glycogen biomarkers) data through week 18 in eight initial ERT-switch patients and two ERT-naïve showed:
- The desired PK profile
- Improvements in key muscle damage biomarkers (creatine kinase (CK) enzyme, alanine aminotransferase (ALT), and aspartate aminotransferase (AST)) in a majority of ERT-switch patients and both ERT-naïve patients
- Reductions in a biomarker of glycogen substrate urine hexose tetrasaccharide (Hex4) in all patients
- Target enrollment achieved across all patient cohorts
Anticipated Upcoming Pompe Disease Program Milestones:
- ATB200-02 study data in additional naïve and non-ambulatory patients, as well as extension-phase data on all patient cohorts, in the second and third quarter of 2017
- Meetings with US and EU regulators
SD-101 for Epidermolysis Bullosa (EB)
SD-101 is a novel, late-stage, proprietary topical treatment and potential first-to-market therapy for EB. SD-101 is currently being investigated in a registration-directed Phase 3 study (ESSENCE, also known as SD-005) to support global regulatory submissions.
SD-101 was granted FDA Breakthrough Therapy designation in 2013 based on results from a Phase 2a study for the treatment of lesions in patients suffering with EB. SD-101 is the first-ever treatment in clinical studies to show improvements in wound closure across all major EB types.
EB Phase 3 ESSENCE Study Highlights:
- Significant momentum enrolling patients diagnosed with Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB
- More than 95% of patients completing the primary treatment period have elected to continue in the open-label extension study
Anticipated EB Program Milestones:
- Top-line Phase 3 data anticipated mid-2017
Conference Call and Webcast
An audio webcast can also be accessed via the Investors section of the
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.
About
Forward-Looking Statements
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, financing plans, and the projected cash position for the Company. In particular, this press release relates to the preliminary data from a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221. The inclusion of forward-looking statements arising
from this preliminary data and study 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
TABLE 1 | ||||||||||||
Amicus Therapeutics, Inc. Consolidated Statements of Operations (in thousands, except share and per share amounts) | ||||||||||||
Years Ended
| ||||||||||||
2016 | 2015 | 2014 | ||||||||||
Revenue: | ||||||||||||
Net Product Sales | $ | 4,958 | $ | — | $ | — | ||||||
Research revenue | — | — | 1,224 | |||||||||
Total revenue | 4,958 | — | 1,224 | |||||||||
Cost of goods sold | 833 | — | ||||||||||
Gross Profit | 4,125 | — | 1,224 | |||||||||
Operating Expenses: | ||||||||||||
Research and development | 104,793 | 76,943 | 47,624 | |||||||||
Selling, general and administrative | 71,151 | 47,269 | 20,717 | |||||||||
Changes in fair value of contingent consideration payable | 6,760 | 4,377 | 100 | |||||||||
Restructuring charges | 69 | 15 | (63 | ) | ||||||||
Depreciation | 3,242 | 1,833 | 1,547 | |||||||||
Total operating expenses | 186,015 | 130,437 | 69,925 | |||||||||
Loss from operations | (181,890 | ) | (130,437 | ) | (68,701 | ) | ||||||
Other income (expenses): | ||||||||||||
Interest income | 1,602 | 929 | 223 | |||||||||
Interest expense | (5,398 | ) | (1,578 | ) | (1,484 | ) | ||||||
Loss on extinguishment of debt | (13,302 | ) | (952 | ) | — | |||||||
Other expense | (4,793 | ) | (80 | ) | (77 | ) | ||||||
Loss before income tax benefit | (203,781 | ) | (132,118 | ) | (70,039 | ) | ||||||
Income tax benefit | 3,739 | — | 1,113 | |||||||||
Net loss attributable to common stockholders | $ | (200,042 | ) | $ | (132,118 | ) | $ | (68,926 | ) | |||
Net loss attributable to common stockholders per common share — basic and diluted | $ | (1.49 | ) | $ | (1.20 | ) | $ | (0.93 | ) | |||
Weighted‑average common shares outstanding — basic and diluted | 134,401,588 | 109,923,815 | 74,444,157 |
TABLE 2 | ||||||||
Amicus Therapeutics, Inc. Consolidated Balance Sheets (in thousands, except share and per share amounts) | ||||||||
2016 | 2015 | |||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 187,026 | $ | 69,485 | ||||
Investments in marketable securities | 143,325 | 144,548 | ||||||
Accounts receivable | 1,304 | — | ||||||
Inventories | 3,416 | — | ||||||
Prepaid expenses and other current assets | 4,993 | 2,568 | ||||||
Total current assets | 340,064 | 216,601 | ||||||
Property and equipment, less accumulated depreciation of | 9,816 | 6,178 | ||||||
In-process research & development | 486,700 | 486,700 | ||||||
Goodwill | 197,797 | 197,797 | ||||||
Other non-current assets | 2,468 | 1,108 | ||||||
Total Assets | $ | 1,036,845 | $ | 908,384 | ||||
Liabilities and Stockholders' Equity | ||||||||
Current liabilities: | ||||||||
Accounts payable, accrued expenses, and other current liabilities | $ | 41,008 | $ | 32,216 | ||||
Deferred reimbursements, current portion | 13,850 | — | ||||||
Contingent consideration payable, current portion | 56,101 | 41,400 | ||||||
Total current liabilities | 110,959 | 73,616 | ||||||
Deferred reimbursements | 21,906 | 35,756 | ||||||
Convertible notes | 154,464 | — | ||||||
Due to related party | — | 41,601 | ||||||
Contingent consideration payable | 213,621 | 232,677 | ||||||
Deferred income taxes | 173,771 | 176,219 | ||||||
Other non-current liability | 1,973 | 681 | ||||||
Commitments and contingencies | ||||||||
Stockholders' equity: | ||||||||
Common stock, 142,691,986 shares issued and outstanding at Common stock, 125,027,034 shares issued and outstanding at | 1,480 | 1,306 | ||||||
Additional paid-in capital | 1,120,156 | 917,454 | ||||||
Accumulated other comprehensive loss: | ||||||||
Foreign currency translation adjustment, less tax benefit of | 1,945 | — | ||||||
Unrealized gain/ (loss) on available-for securities | 102 | (115 | ) | |||||
Warrants | 16,076 | 8,755 | ||||||
Accumulated deficit | (779,608 | ) | (579,566 | ) | ||||
Total stockholders' equity | 360,151 | 347,834 | ||||||
Total Liabilities and Stockholders' Equity | $ | 1,036,845 | $ | 908,384 | ||||
FOLD-G
CONTACTS: Investors/Media:Source:Amicus Therapeutics Sara Pellegrino Senior Director, Investor Relations spellegrino@amicusrx.com (609) 662-5044 Media: MWW PRSid Dinsay sdinsay@mww.com (646) 381-9017
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