Amicus Therapeutics(US:FOLD)2021-05-09 16:00Revenue and Sales Performance - Galafold (migalastat) generated $66.4 million in revenue for the three months ended March 31, 2021, an increase of $5.9 million compared to the same period in the prior year[86]. - Net product sales increased by $5.9 million to $66.4 million for the three months ended March 31, 2021, compared to $60.5 million in the same period of 2020, driven by growth in the U.S., Europe, and Japan markets[104]. Clinical Trials and Research - The Phase 3 PROPEL study of AT-GAA for Pompe disease included 123 patients across 62 sites in 24 countries, with topline results reported in February 2021[89]. - Patients treated with AT-GAA walked an average of 21 meters farther at 52 weeks compared to 7 meters for those treated with alglucosidase alfa, although statistical significance was not achieved (p=0.072)[89]. - AT-GAA demonstrated a 0.9% absolute decline in percent-predicted Forced Vital Capacity (FVC) compared to a 4.0% decline in the alglucosidase alfa arm, achieving statistical significance (p=0.023)[90]. - The company is conducting clinical programs for CLN6 and CLN3 Batten disease, with positive interim results indicating treatment was well tolerated and showed meaningful effects in slowing disease progression[99][100]. - The engineered hGAA AAV gene therapy demonstrated better targeting and clearance of glycogen storage in Pompe mice compared to unmodified hGAA AAV, indicating potential for effective treatment[96]. Financial Position and Cash Flow - Total cash, cash equivalents, and marketable securities as of March 31, 2021, was $417.4 million, sufficient to fund operations and ongoing research programs[86]. - The company reported a net loss attributable to common stockholders of $65.7 million for the three months ended March 31, 2021, an improvement of $23.3 million compared to a net loss of $88.9 million in the same period of 2020[104]. - Net cash used in operating activities was $72.4 million for the three months ended March 31, 2021, compared to $107.9 million for the same period in 2020[110][111]. - Net cash provided by investing activities was $86.6 million for the three months ended March 31, 2021, reflecting $163.7 million from the sale and redemption of marketable securities[112]. - Net cash provided by financing activities was $8.8 million for the three months ended March 31, 2021, primarily from the exercise of outstanding warrants and stock options[114]. Regulatory Designations and Approvals - The FDA granted Breakthrough Therapy designation to AT-GAA for late-onset Pompe disease, and orphan drug designation was received in Japan[86]. - Galafold is approved in over 40 countries, including the U.S., E.U., U.K., and Japan, with plans for further launches in 2021[86]. Strategic Alliances and Collaborations - The company is exploring strategic alliances and business development opportunities to enhance stockholder value and access necessary resources for developing therapies for rare diseases[103]. - The company has agreed to pay up to an additional $10 million in milestone payments and $220 million for regulatory approval milestones related to the acquisition of Celenex[118]. - Under the exclusive license agreement with Nationwide Children's, the company is obligated to pay up to $7.8 million in development and sales-based milestones for each product[119]. - The company has a research collaboration agreement with Penn, which allows for milestone payments up to $88 million per indication and annual funding of $10 million for discovery research[120]. - The collaboration agreement with GlaxoSmithKline includes potential post-approval and sales-based milestones up to $40 million, along with tiered royalties in the mid-teens across eight major markets outside the U.S.[121]. Debt and Financial Risks - The company holds a $400 million Senior Secured Term Loan due 2026, with an annual average variable interest rate of 7.5%[126]. - A hypothetical 100 basis point change in the average interest rate on the company's variable rate debt would result in a $1 million change in interest expense[126]. - The company faces foreign exchange risk from transactions in currencies other than U.S. dollars, but does not currently engage in foreign currency hedging activities[128]. - There have been no material changes in the company's financial instrument portfolio or market risk exposures since the fiscal year ended December 31, 2020[129]. Operational Outlook - The company expects to incur operational losses due to ongoing research and development expenses, particularly for clinical trials of drug candidates such as AT-GAA, CLN6, and CLN3[116]. - The company anticipates an increase in cash used in operations over the next several quarters and years without additional funding, despite generating revenue from product sales[117]. - Research and development expenses decreased by $25.0 million to $64.1 million for the three months ended March 31, 2021, primarily due to the timing of clinical research and manufacturing costs associated with the Pompe program[105]. - The company has maintained operations and supply chains during the COVID-19 pandemic, ensuring uninterrupted access to medicines[85].