The following excerpt is from the company's
Migalastat for Fabry Disease Moving Forward to CHMP Opinion in European Union
Novel Pompe ERT Advancing to Enter Phase 1/2 Study in Pompe Patients
Rolling NDA Now Initiated for SD-101 for Epidermolysis Bullosa
CRANBURY, NJ, November 3, 2015
Amicus Therapeutics (Nasdaq: FOLD), a biotechnology company at the forefront of therapies for rare and orphan diseases, today announced financial results for the third quarter ended September 30, 2015. The Company also provided program updates and reiterated financial guidance for 2015 year ending cash balance of $200-$225 million.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, During the third quarter we made significant progress in executing our patient-centric vision to build one of the worlds leading biotechnology companies focused on devastating rare and orphan diseases. We are sharply focused on four key business priorities ahead that we believe have the potential to create substantial shareholder value and to deliver upon our mission for rare disease patients approval and commercialization of our personalized medicine migalastat for Fabry patients in Europe, development of an optimal U.S. approval pathway for migalastat, initiation of clinical studies in Pompe patients with our novel Pompe ERT ATB200, and completion of our Phase 3 study and rolling NDA submission for SD-101 for Epidermolysis Bullosa.
Financial Highlights for Third Quarter Ended September 30, 2015
Cash, cash equivalents, and marketable securities totaled $251.9 million at September 30, 2015, compared to $169.1 million at December 31, 2014.
Completed acquisition of Scioderm Inc. for approximately $224 million (approximately $141 million paid in cash and approximately $83 million paid through issuance of 5.9 million newly issued Amicus shares)
Total operating expenses increased to $38.0 million compared to $17.1 million for the third quarter 2014, primarily due to increases in preclinical and clinical development costs on the Fabry monotherapy and Pompe ERT programs as well as pre-commercial organizational costs.
Net loss was $37.8 million, or $0.32 per share, compared to a net loss of $17.1 million, or $0.22 per share, for the third quarter 2014.
2015 Financial Guidance
Cash, cash equivalents, and marketable securities totaled $251.9 million at September 30, 2015 compared to $169.1 million at December 31, 2014. During the third quarter, Amicus successfully completed its acquisition of 100% of the capital stock of Scioderm, Inc. With the closing of the Scioderm acquisition and the forecasted spending on the EB clinical development program, Amicus expects to end 2015 with between $200 million and $225 million of cash on hand. Current cash is expected to fund the Companys operating plan (including SD-101) into 2017.
Amicus has made significant progress in building the commercial organization and preparing for the launch of migalastat in Europe and other international geographies. The European Medicines Agencys (EMA) review of the marketing authorization application (MAA) for migalastat remains on track under accelerated assessment. The Day 120 questions have been received and Amicus continues to expect an opinion from the Committee for Medicinal Products for Human Use (CHMP) in late 2015 or early 2016. As previously reported, the timing of an NDA submission in the U.S. will be based on the determination of the optimal regulatory pathway. An update on the U.S. strategy for migalastat will be provided by Amicus in the first quarter of 2016.
Amicus is developing migalastat in combination with ERT for Fabry patients who do not have amenable mutations and cannot take monotherapy. Amicus is developing a novel Fabry ERT cell line, and preclinical proof-of-concept studies co-formulating Amicus Fabry ERT with migalastat will begin this quarter. As the Amicus internal novel ERT for Fabry has continued to advance, the Company plans to focus exclusively on this proprietary cell line for co-formulation with migalastat and no longer plans to conduct a co-administration study with commercially available ERT. Amicus believes that further development of its own proprietary Fabry ERT co-formulated with migalastat represents the fastest and best path for bringing a novel therapy and...