Amicus Therapeutics Announces Full-Year 2013 Financial Results and Corporate Updates

Amicus Therapeutics Announces Full-Year 2013 Financial Results and Corporate Updates    Executing 3-in-3 Strategy to Advance 3 Next-Generation ERTs into Clinic in                                  Next 3 Years             Reiterating FY14 Cash Spend Guidance of $54-$59 Million  CRANBURY, N.J., March 3, 2014 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced financial results for the full-year ended December 31, 2013. The Company also provided program updates and reiterated full-year 2014 operating expense guidance.  John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, "During 2013 we focused on strengthening our biologics business strategy to develop next-generation ERTs for patients with lysosomal storage diseases. Through our purchase of Callidus Biopharma, we have acquired a proprietary Pompe ERT as well as a peptide tagging technology that is complementary to our CHART platform. We believe that these technologies together provide a unique tool set to enhance enzyme activity, increase enzyme uptake into tissues, and potentially address the tolerability and immunogenicity associated with current ERTs. During 2014 we are strongly positioned and well capitalized to execute our 3-in-3 strategy to advance three next-generation ERTs into the clinical in the next three years, with lead programs in Fabry, Pompe and MPS I."  Financial Highlights for Full Year Quarter Ended December 31, 2013    *Cash, cash equivalents, and marketable securities totaled $82.0 million at     December 31, 2013 compared to $99.1 million at December 31, 2012.   *Total operating expenses decreased to $64.5 million compared to $71.3     million for the full-year 2012 primarily due to decreases in clinical     development costs on the Fabry monotherapy program.   *Net cash spend was $47.1 million, within the full-year 2013 guidance range     of $47-53 million.   *Net loss was $59.6 million, or $1.16 per share, compared to a net loss of     $48.8 million, or $1.07 per share, for the full-year 2012.  2014 Financial Guidance  Cash, cash equivalents, and marketable securities totaled $82.0 million at December 31, 2013 compared to $99.1 million at December 31, 2012. The Company's balance sheet was strengthened in the fourth quarter of 2013 with a $15.0 million equity financing and a $25.0 million debt financing under which $15.0 million was drawn and $10.0 million remains available. Amicus continues to expect full-year 2014 net cash spend between $54 million and $59 million. The current cash position is projected to fund operations into the second half of 2015.  Program Updates  Amicus owns exclusive global rights to its next-generation ERTs, as well as all applications of its Chaperone-Advanced Replacement Therapy (CHART™) and enzyme targeting technology platforms. In each CHART program, a unique pharmacological chaperone is designed to bind to and stabilize a specific therapeutic enzyme in its properly folded and active form. Through its purchase of Callidus Biopharma Amicus has also acquired a differentiated peptide tagging technology that can be used to uniquely engineer bio-better ERTs. These platform technologies provide a complementary tool set to design next-generation therapies for enhanced tissue uptake of active enzyme, greater lysosomal activity, more reduction of substrate, and potentially address the tolerability and immunogenicity associated with currently marketed ERTs.  Next-Generation ERT for Pompe Disease  Amicus is advancing a recombinant human acid alpha-glucosidase (rhGAA) for Pompe disease into late preclinical development. The Company's acquisition of Callidus Biopharma, brings a differentiated Pompe ERT, designated AT-B200, with a unique carbohydrate structure. In preclinical studies AT-B200 has shown superior tissue uptake and activity when compared to current standard of care. This ERT may be further optimized through co-formulation with Amicus' pharmacological chaperone AT2220 to improve enzyme stability and tolerability, and by applying the Company's peptide tagging technology for better targeting.  Next-Generation ERT for Fabry Disease  In combination with ERT, Amicus' pharmacological chaperone migalastat HCl is designed to bind and stabilize the infused alpha-Gal A enzyme, independent of a patient's genetic mutation. Amicus believes this approach has the potential to benefit all patients with Fabry disease.  Amicus has completed a Phase 2 clinical study (Study 013) of migalastat HCl co-administered with currently approved ERTs for Fabry disease (Fabrazyme^® and Replagal^®) as well as preclinical studies of migalastat HCl co-formulated with a proprietary investigational ERT for Fabry disease (JCR Pharmaceutical Co Ltd's JR-051). JR-051 is a human recombinant alpha-Gal A enzyme that is designed to be biosimilar to Fabrazyme. Positive results from these clinical and preclinical studies demonstrated increased enzyme activity in plasma and greater enzyme uptake into tissues in the presence of the chaperone compared to any of these ERTs alone^1,2.  In the first half of 2014 Amicus plans to conduct a Phase 1 study to assess the pharmacokinetics of an intravenous formulation of migalastat HCl in healthy volunteers to identify the optimal dose for co-formulation with ERT. In the second half of 2014, Amicus expects to initiate a Phase 1/2 study to evaluate migalastat HCl co-formulated with JR-051. Amicus is currently evaluating its long-term strategy for supplying late-stage clinical and commercial ERT, which may include developing or in-licensing a recombinant alpha-Gal A enzyme comparable to JR-051.  Next-Generation ERT for MPS I  Amicus is leveraging its CHART platform to develop a proprietary human recombinant alpha-L-iduronidase (rhIDUA) enzyme for MPS I. In support of its development of this next-generation ERT, Amicus has received funding of up to $250,000 from a private U.S.-based donor that provides medical research grants to find better treatments and cures for rare genetic disorders, including lysosomal storage diseases.  Migalastat HCl Monotherapy for Fabry Disease  Migalastat HCl monotherapy is being investigated in two ongoing Phase 3 studies for Fabry patients with amenable mutations. Interim 6-month data from the first ongoing Phase 3 study (Study 011) have been reported, and 12- and 24-month data from this study are anticipated in the second quarter of 2014. Top-line, 18-month clinical data from the second ongoing Phase 3 study (Study 012) are expected in the second half of 2014.  Novel Small Molecules for Parkinson's Disease  In September 2013 Amicus and Biogen Idec entered a multi-year collaboration to discover of a new class of small molecules that target the glucocerobrosidase (GCase) enzyme for further development and commercialization by Biogen Idec. Biogen Idec is responsible for funding all discovery, development, and commercialization activities. Amicus will be reimbursed for all full-time employees working on the project. In addition Amicus is eligible to receive development and regulatory milestones, as well as modest royalties on global net sales.  Conference Call and Webcast  Amicus Therapeutics will host a conference call and audio webcast today, March 3, 2014 at 5:00 p.m. ET to discuss full-year 2013 financial results and program updates. Interested participants and investors may access the conference call at 5:00 p.m. ET by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international).  An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at http://www.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 8:00 p.m. ET today. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); participant code 5821419.  About Amicus Therapeutics  Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of therapies for rare and orphan diseases. The Company is developing novel, first-in-class treatments for a broad range of human genetic diseases, with a focus on delivering new benefits to individuals with lysosomal storage diseases. Amicus' lead programs include the small molecule pharmacological chaperones migalastat HCl as a monotherapy and in combination with enzyme replacement therapy (ERT) for Fabry disease; and AT2220 (duvoglustat HCl) in combination with ERT for Pompe disease.  About Chaperone-Advanced Replacement Therapy (CHART)  The Chaperone-Advanced Replacement Therapy (CHART™) platform combines unique pharmacological chaperones with enzyme replacement therapies (ERTs) for lysosomal storage diseases (LSDs). In a chaperone-advanced replacement therapy, a unique pharmacological chaperone is designed to bind to and stabilize a specific therapeutic enzyme in its properly folded and active form. This proposed CHART mechanism may allow for enhanced tissue uptake of active enzyme, greater lysosomal activity, more reduction of substrate, and lower immunogenicity compared to ERT alone. Improvements in enzyme stability may also enable more convenient delivery of next-generation therapies. Amicus is leveraging the CHART platform to develop proprietary next-generation therapies that consist of lysosomal enzymes co-formulated with pharmacological chaperones.  ^1Bichet, et al., American Society of Human Genetics, November 2012  ^2Benjamin, et al., Molecular Therapy, April 2012  Forward-Looking Statements  This press release contains, and the accompanying conference call will contain, "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of Amicus' candidate drug products, the timing and reporting of results from preclinical studies and clinical trials evaluating Amicus' candidate drug products, and the projected cash position for the Company. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "potential," "plan," "targets," "likely," "may," "will," "would," "should" and "could," and similar expressions or words identify forward-looking statements. Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. The inclusion of forward-looking statements should not be regarded as a representation by Amicus that any of its plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong. They can be affected by inaccurate assumptions Amicus 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 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 the business of Amicus, including, without limitation: the potential that results of clinical or pre-clinical 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 may not grant or may delay approval for our product candidates; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we will need additional funding to complete all of our studies and, our dependence on third parties in the conduct of our clinical studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. With respect to statements regarding projections of the Company's cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2012. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and Amicus undertakes no obligation to revise or update this news release to reflect events or circumstances after the date hereof. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.  Table 1 Amicus Therapeutics, Inc. (a development stage company) Consolidated Statements of Operations (Unaudited) (In thousands, except share and per share amounts)                                                                                                                            Period from                                                              February 4,                                                                   2002                                                              (inception)                  Three Months            Twelve Months           To                  Ended December 31,      Ended December 31,      Dec.31,                  2012        2013        2012        2013        2013 Revenue:                                                       Research revenue  $--        $324        $11,591     $363        $57,856 Collaborationand --         --         6,820      --         64,382 milestone revenue Total revenue     --         324        18,411     363        122,238                                                               Operating                                                      Expenses: Research and      11,047     9,120      50,273     41,944     357,837 development General and       4,455      4,605      19,364     18,893     151,506 administrative Restructuring     --         1,988      --         1,988      3,510 charges Impairment of leasehold         --         --         --         --         1,030 improvements Depreciation and  421        401        1,705      1,719      13,487 amortization In-process research and      --         --         --         --         418 development Total operating   15,923     16,114     71,342     64,544     527,788 expenses Loss from         (15,923)   (15,790)   (52,931)   (64,181)   (405,550) operations Other income                                                   (expenses): Interest income   81         27         316        174        14,563 Interest expense  (12)       (20)       (89)       (46)       (2,468) Change in fair value of warrant  2,594      34         653        908        2,461 liability Other income      --         --         21         --         252 Loss before tax   (13,260)   (15,749)   (52,030)   (63,145)   (390,742) benefit Benefit from      3,245      3,512      3,245      3,512      12,220 income taxes Net loss          (10,015)   (12,237)   (48,785)   (59,633)   (378,522) Deemed dividend   --         --         --         --         (19,424) Preferred stock   --         --         --         --         (802) accretion Net loss attributable to   $(10,015) $(12,237) $(48,785) $(59,633) $(398,748) common stockholders Net loss attributable to common            $(0.20)   $(0.22)   $(1.07)   $(1.16)    stockholders per common share – basic and diluted Weighted-average common shares     49,477,596  56,173,260  45,565,217  51,286,059   outstanding – basic and diluted                                                                Table 2  Amicus Therapeutics, Inc. (a development stage company) Consolidated Balance Sheets (Unaudited) (in thousands, except share and per share amounts)                                                                                                                      December 31, December 31,                                                     2012         2013 Assets:                                                           Current assets:                                                   Cash and cash equivalents                            $33,971      $43,640 Investments in marketable securities                 65,151       38,360 Receivable due from GSK                              3,225        759 Prepaid expenses and other current assets            2,270        5,519 Total current assets                                 104,617      88,278                                                                  Property and equipment, less accumulated depreciation and amortization of $8,501 and          5,029        4,120 $9,973at December 31, 2012 and 2013, respectively In-process research & development                    —            23,000 Goodwill                                             —            11,613 Other non-current assets                             442          552 Total Assets                                         $110,088     $127,563                                                                  Liabilities and Stockholders' Equity                              Current liabilities:                                              Accounts payable and accrued expenses                $8,845       $10,162 Current portion of secured loan                      398          299 Total current liabilities                            19,256       9,243                                                                  Deferredreimbursements, less current portion        30,418       36,677 Warrant liability                                    908          — Secured loan, less current portion                   299          14,174 Contingent consideration payable                     —            10,600 Deferred tax liability                               —            9,186 Other non-current liability                          —            714                                                                  Commitments and contingencies                                                                                                      Stockholders' equity:                                             Common stock, $.01 par value, 125,000,000 shares authorized, 49,631,672 shares issued and outstanding 556          679 at December 31, 2012,61,975,416 shares issued and outstanding at December 31, 2013 Additional paid-in capital                           387,539      423,593 Accumulated other comprehensive income               14           1 Deficit accumulated during the development stage     (318,889)   (378,522) Total stockholders' equity                           69,220       45,751 Total Liabilities and Stockholders' Equity           $110,088     $127,563                                                                   FOLD–G  CONTACT: Investors/Media:          Sara Pellegrino          spellegrino@amicusrx.com          (609) 662-5044                   Media:          Dan Budwick          dan@purecommunicationsinc.com          (973) 271-6085