PTC Therapeutics(PTCT)2022-02-22 21:36Sales Performance - Translarna achieved sales of $236.0 million during the year ended December 31, 2021[896]. - Emflaza net sales reached $187.3 million for the year ended December 31, 2021[896]. - Net product sales in the United States for 2021 were $187.3 million, up 34.8% from $139.0 million in 2020[955]. - Net product sales outside the United States reached $241.6 million in 2021, a 24.2% increase from $194.4 million in 2020[955]. - Net product revenue for the year ended December 31, 2021, was $428.9 million, an increase of $95.5 million, or 29%, compared to $333.4 million for the year ended December 31, 2020[964]. - Total revenues for the year ended December 31, 2021, were $538.6 million, an increase of 41.5% compared to $380.8 million in 2020[1071]. - Collaboration revenue was $55.0 million for the year ended December 31, 2021, an increase of $12.5 million, or 29%, from $42.6 million for the year ended December 31, 2020, driven by milestone payments from Roche[965]. - Royalty revenue increased to $54.6 million for the year ended December 31, 2021, up $49.9 million, or over 100%, from $4.8 million for the year ended December 31, 2020, due to FDA approval of Evrysdi[966]. Regulatory Approvals and Clinical Trials - The European Commission renewed the marketing authorization for Translarna, effective through August 5, 2022, subject to annual review[897]. - Tegsedi has received marketing authorization in the United States, EU, and Brazil for treating hATTR amyloidosis, with commercial launch initiated in Brazil[901]. - Waylivra received marketing authorization in Brazil for treating familial chylomicronemia syndrome, with commercial launch also initiated[902]. - The company initiated a registration-directed Phase 3 trial for PTC923 for phenylketonuria in Q3 2021, expecting results by the end of 2022[907]. - The company is preparing a biologics license application for PTC-AADC for AADC deficiency in the United States, expecting to submit in Q2 2022[905]. - The company plans to submit a Biologics License Application (BLA) for PTC-AADC for the treatment of AADC deficiency in the United States in the second quarter of 2022[929]. - The company is preparing for a regulatory decision from ANVISA regarding Waylivra in the second half of 2022[23]. - The SMA program includes Evrysdi®, approved by the FDA in August 2020 and by the EU in March 2021 for treating SMA in patients aged two months and older[1087]. Financial Performance and Losses - The company reported a net loss of $523.9 million for the fiscal year ended December 31, 2021, compared to $438.2 million in 2020 and $251.6 million in 2019, indicating a continued increase in losses[928]. - As of December 31, 2021, the company had an accumulated deficit of $2,098.0 million, reflecting ongoing financial challenges[928]. - The company reported a net loss attributable to common stockholders of $523.9 million for 2021, compared to a net loss of $438.2 million in 2020, reflecting an increase in losses of 19.6%[1071]. - The company expects to continue incurring significant expenses and operating losses for at least the next fiscal year[998]. - The company has never been profitable and will need to generate significant revenues to achieve and sustain profitability[1040]. Expenses and Cash Flow - Total research and development expenses increased to $540.7 million in 2021 from $477.6 million in 2020, reflecting a 13.2% rise[941]. - The company anticipates ongoing increases in expenses related to commercialization efforts, including sales and marketing, legal and regulatory, and manufacturing, which will impact future financial performance[929]. - Selling, general and administrative expense was $285.8 million for the year ended December 31, 2021, an increase of $40.6 million, or 17%, from $245.2 million for the year ended December 31, 2020[972]. - Interest expense, net increased to $86.0 million for the year ended December 31, 2021, up $29.7 million, or 53%, from $56.4 million for the year ended December 31, 2020[975]. - Net cash used in operating activities was $251.3 million for the year ended December 31, 2021, compared to $194.1 million in 2020 and $98.6 million in 2019, primarily due to clinical development and international infrastructure expansion[18]. - The company has a total annual funding requirement of $4.5 million for cash interest payments on its outstanding 2022 Convertible Notes[28]. - The company expects to incur a $50.0 million sales-based milestone payment in connection with Emflaza sales in 2022[932]. Investments and Capital Structure - The company completed the acquisition of Censa for total upfront consideration of $15.0 million in cash and 845,364 shares valued at $42.9 million[924]. - The company received net proceeds of $650.0 million from the Royalty Purchase Agreement, selling 42.933% of its right to receive sales-based royalty payments[925]. - The Royalty Purchase Agreement generated $650.0 million in cash consideration, classified as debt on the balance sheet[959]. - The company has total obligations of $130.8 million stemming from operating leases, with significant leases expiring in 2024 and 2035[1031]. - The company has made significant investments in marketable securities, with purchases totaling $333.1 million in 2021[1080]. Pipeline and Future Outlook - The company has a pipeline of gene therapy product candidates for rare monogenic diseases affecting the central nervous system[905]. - The company has a robust pipeline of transformative medicines aimed at rare disorders, leveraging its scientific and clinical expertise[1084]. - The company aims to maximize value for stakeholders by providing access to best-in-class treatments for patients with limited options[1084]. - The company plans to initiate a Phase 2 study of PTC518 for Huntington's disease in Q1 2022, following positive Phase 1 results[1090]. - The company has a gene therapy pipeline for rare CNS diseases, including PTC-AADC for AADC deficiency, with a marketing authorization application submitted to the EMA in January 2020[1091].