OREANDA-NEWS. Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of therapies for rare and orphan diseases, today announced financial results for the third quarter ended September 30, 2017. The Company also summarized recent program updates and reiterated full-year 2017 financial guidance.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, stated, “During the third quarter we continued to successfully execute across our strategic priorities for our core programs in Fabry and Pompe. We are pleased with the significant growth and expansion of our international Galafold launch, and the number of patients treated with this precision medicine for Fabry disease. In Pompe disease, the data cascade for our novel treatment paradigm ATB200/AT2221 has continued to exceed our expectations in terms of the consistency, durability and magnitude of effect across patients and across functional outcomes, key disease biomarkers and safety. There has been extraordinary patient and physician demand for this important potential treatment option. We are committed to increasing access to this investigational medicine for as many people as possible living with Pompe as soon as we can.”

Amicus is on track to achieve four key strategic priorities between now and the end of 2017:

1)  Target of 300 patients on reimbursed Galafold;
2)  New Drug Application (NDA) submission for migalastat to the U.S. FDA;
3)  Continued advancements with the Pompe program, including now ongoing collaborative discussions with regulators to determine the best and fastest pathway; and
4)  A strong balance sheet with more than 18 months of cash at year-end.

Third Quarter 2017 Financial Results

  • Total revenue in the third quarter 2017 was approximately $10.9 million, a sequential increase of 51.4% from total revenue of $7.2 million in the second quarter 2017, and a year-over-year increase of 419% from total revenue of $2.1 million in the third quarter 2016. Total revenue represents commercial sales of Galafold (migalastat) which commenced in May 2016, as well as reimbursed Expanded Access Programs (EAPs).
  • Cash, cash equivalents, and marketable securities totaled $426.6 million at September 30, 2017 compared to $330.4 million at December 31, 2016.
  • Total operating expenses increased to $284.3 million compared to $46.7 million for the third quarter 2016 primarily from non-cash charges related to the Phase 3 ESSENCE study in epidermolysis bullosa (EB).
  • Operating expenses, as adjusted, excludes the impact of the non-cash charges related to the ESSENCE study, were $73.5 million, representing a $26.8 million increase over the third quarter of 2016 primarily due to increased investments in the Pompe and EB programs as well as increased investment in the Galafold commercial launch.
  • Net cash spend was $147.3 million for the nine months ending September 30, 2017.
  • Net loss was $111.7 million, or $0.69 per share, compared to a net loss of $46.7 million, or $0.33 per share, for the third quarter 2016. Net loss, as adjusted excludes the impact of the non-cash charges related to the ESSENCE study was $65.6 million or $0.41 per share.

2017 Financial Guidance

Cash, cash equivalents, and marketable securities totaled $426.6 million at September 30, 2017 compared to $330.4 million at December 31, 2016. The current cash position includes $243.0 million in net proceeds from a follow on public offering in July 2017.

Amicus continues to expect full-year 2017 net operating cash spend of between $175 million to $200 million and full-year 2017 total net cash spend (including third-party milestone payments and capital expenditures) of between $200 million and $225 million. The current cash position is anticipated to fund ongoing operations into at least the second half of 2019.

Program Highlights

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 European Union, Switzerland, Israel, Canada and Australia have granted full approval for migalastat under the trade name Galafold. The EU approval may serve as the basis for regulatory approvals in more than two-thirds of the global Fabry market that is outside the U.S. In the U.S., as previously announced, the FDA has confirmed that Amicus may submit a new drug application (NDA) for migalastat.

International Launch and Expanded Access Programs (EAP) Updates:

  • More than 260 patients (na?ve and ERT-switch) on reimbursed Galafold as of October 31, 2017
  • 13 countries with reimbursement (commercial or EAP) including the top four largest EU countries
  • Reimbursement dossiers submitted and pricing discussions are now underway in 12 countries
  • Target of 300 patients treated with reimbursed Galafold on track for year-end 2017

Global Regulatory Updates:

  • Four additional approvals secured outside the EU (Switzerland, Israel, Canada and Australia)
  • Regulatory submissions completed in seven additional countries outside the EU, including Japan
  • NDA submission to U.S. FDA to be based on existing data on track for 4Q17

Anticipated Upcoming Fabry Disease Program Milestones:

  • Commercial launch and EAPs in additional international countries
  • Additional regulatory submissions including a U.S. NDA (4Q17)
  • Regulatory decision in Japan (1H18)
  • Final preclinical data and announcement of path forward for novel Amicus Fabry ERT cell line for Fabry patients with non-amenable mutations (1Q18)

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. Additional positive data were reported in October 2017 from an ongoing global Phase 1/2 clinical study (ATB200-02) to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ATB200/AT2221 across three cohorts, including ambulatory ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2), and ERT-na?ve patients (Cohort 3). The Company has commenced 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.           

In order to continue to build a robust data set and to meet the needs of the Pompe community, Amicus announced the following four key status updates for this important program:

  • A retrospective natural history study of Pompe patients treated with approved standard of care ERT has been initiated at leading global Pompe disease treatment centers (POM-002 Study)
  • A prospective observational study has also been initiated in Pompe patients currently receiving approved standard of care ERT at leading global Pompe disease treatment centers (POM-003 Study)   
  • All engineering runs successfully completed and GMP production of ATB200 has commenced at the large commercial scale (1,000 Liters)
  • Analytical and in vivo (preclinical) comparability studies completed between the 250 Liter and 1,000 Liter scale 

Anticipated Upcoming Pompe Disease Program Milestones:

  • Ongoing discussions with U.S. and EU regulators
  • Additional data from ATB200-02 clinical study at 14th Annual WORLDSymposium™ (February 5-9, 2018)
  • Pompe regulatory pathway update (1H18)

SD-101 for Epidermolysis Bullosa (EB)

During the third quarter Amicus reported that top-line data from the randomized, double-blind, placebo-controlled Phase 3 clinical study (ESSENCE, SD-005) to assess the efficacy and safety of the novel topical wound-healing agent SD-101 did not meet the primary endpoints or secondary endpoints in participants with EB. Based on these top-line data Amicus has no current plans to invest in any additional clinical studies or commercial preparation activities for SD-101. The Company continues to make SD-101 available to all patients currently enrolled in the ongoing extension study (SD-006).

Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, November 8, 2017 at 8:30 a.m. ET to discuss third quarter 2017 financial results and corporate updates. Interested participants and investors may access the conference call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international); conference ID: 5995789.

An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/ and will be archived for 30 days. Web participants are encouraged to go to the web site 15 minutes prior to the start of the call to register, download and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 11:30 a.m. ET today. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); conference ID: 5995789.

Non-GAAP Financial Measures

In addition to the United States generally accepted accounting principles (GAAP) results, this earnings release contains non-GAAP financial measures that we believe, when considered together with the GAAP information, provides useful information to investors that promotes a more complete understanding of our operating results and financial position for the current period. Management uses these non-GAAP financial measures internally for planning, forecasting, evaluating and allocating resources to the Company's programs.  The non-GAAP results exclude the impact of the following GAAP items specifically related to the Phase 3 ESSENCE study in EB: changes in fair value of contingent consideration, impairment of assets and adjustments to income tax expense. These non-GAAP financial measures should be considered in addition to, and not as replacements for, or superior to, measures of financial performance prepared in accordance with GAAP. Please refer to the attached Reconciliation of Non-GAAP Financial Measures for explanations of the amounts adjusted to arrive at non-GAAP total operating expense, net loss attributable to common stockholders and net loss attributable to common stockholders per common share – basic and diluted for the three month period ended September 30, 2017.

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.

About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.

 

 
TABLE 1
Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(Unaudited)
(in thousands, except share and per share amounts)
 
  Three Months Ended
September 30,		   Nine Months Ended
September 30,
    2017       2016       2017       2016  
Revenue:              
Net product sales $   10,874     $   2,127     $   22,201     $   2,127  
Cost of goods sold   1,790       344       3,626       344  
Gross Profit   9,084       1,783       18,575       1,783  
Operating Expenses:              
Research and development   40,641         32,457       103,502       74,163  
Selling, general and administrative   21,647       17,469       60,090       52,470  
Changes in fair value of  contingent consideration payable   (244,250 )       (4,110 )     (238,622 )     9,228  
Loss on impairment of assets   465,427       -       465,427       -  
Restructuring charges   -       11       -       69  
Depreciation   851       896       2,486       2,336  
Total operating expenses   284,316       46,723       392,883       138,266  
Loss from operations   (275,232 )     (44,940 )     (374,308 )     (136,483 )
Other income (expenses):              
Interest income   1,190       460       2,702       1,098  
Interest expense   (4,351 )     (1,517 )     (12,820 )     (3,517 )
Other income (expense)   2,044       (910 )     5,054       (3,199 )
Loss before income tax benefit   (276,349 )     (46,907 )     (379,372 )     (142,101 )
Income tax benefit     164,683       253         164,578       706  
Net loss attributable to common stockholders $ (111,666 )   $ (46,654 )   $ ( 214,794 )   $ (141,395 )
Net loss attributable to common stockholders per common share — basic and diluted $ (0.69 )   $ (0.33 )   $ (1.44 )   $ (1.07 )
Weighted?average common shares outstanding — basic and diluted   160,796,841       140,656,109       148,963,864       131,675,690  
                               
TABLE 2
 Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(Unaudited)
(in thousands, except share and per share amounts)
 
    September 30,
   December 31,
    2017 
   2016
Assets        
Current assets:        
Cash and cash equivalents   $ 64,133     $ 187,026  
Investments in marketable securities, current portion     347,388       143,325  
Accounts receivable     5,974       1,304  
Inventories     7,272       3,416  
Prepaid expenses and other current assets     6,246       4,993  
Total current assets     431,013       340,064  
Investments in marketable securities     15,109       -  
Property and equipment, less accumulated depreciation of $13,273 and $12,495 at September 30, 2017 and December 31, 2016, respectively     9,641       9,816  
In-process research & development     23,000       486,700  
Goodwill     197,797       197,797  
Other non-current assets     4,219       2,468  
Total Assets   $ 680,779     $ 1,036,845  
         
Liabilities and Stockholders’ Equity        
Current liabilities:        
Accounts payable, accrued expenses, and other current liabilities   $ 53,709     $ 41,008  
Deferred reimbursements, current portion     6,250       13,850  
Contingent consideration payable, current portion     8,200       56,101  
Total current liabilities     68,159       110,959  
Deferred reimbursements     16,906       21,906  
Convertible notes     161,635       154,464  
Contingent consideration payable     12,900       213,621  
Deferred income taxes     9,186       173,771  
Other non-current liability     2,313       1,973  
Commitments and contingencies        
Stockholders’ equity:        
Common stock, $0.01 par value, 250,000,000 shares authorized 165,491,141 and 142,691,986  shares issued and outstanding at September 30, 2017 and December 31, 2016, respectively     1,707       1,480  
Additional paid-in capital     1,387,767       1,120,156  
Accumulated other comprehensive loss:        
Foreign currency translation adjustment     (1,367 )     1,945  
Unrealized gain on available-for-sale securities     (101 )     102  
Warrants     16,076       16,076  
Accumulated deficit     (994,402 )     (779,608 )
Total stockholders’ equity     409,680       360,151  
Total Liabilities and Stockholders’ Equity   $ 680,779     $ 1,036,845  
TABLE 3
 
Amicus Therapeutics, Inc.
Reconciliation of Non-GAAP Financial Measures
Dollars in Thousands Except Per Share Data
 
  Three Months
  Ended
  September 30, 2017
   
Total operating expenses – as reported $ 284,316  
Loss on impairment of assets related to the  
Phase 3 ESSENCE study in EB   465,427  
Changes in fair value of contingent  
consideration payable related to the  
Phase 3 ESSENCE study in EB   (254,650 )
Total operating expenses – as adjusted $ 73,539  
   
Net loss attributable to common stockholders – as reported $ (111,666 )
Loss on impairment of assets related to the  
Phase 3 ESSENCE study in EB   465,427  
Changes in fair value of contingent  
consideration payable related to the  
Phase 3 ESSENCE study in EB   (254,650 )
Income tax benefit (1)   (164,683 )
Net loss attributable to common stockholders – as adjusted $ (65,572 )
   
Net loss attributable to common stockholders per common  
share – basic and diluted – as reported $ (0.69 )
   
Net loss attributable to common stockholders per common  
share – basic and diluted – as adjusted $ (0.41 )
   
Weighted-average common shares outstanding – basic and  
diluted – as reported and adjusted   160,796,841  
   

(1) Related to the reversal of the deferred tax liability associated with the Scioderm in process research and development asset.