PTC Therapeutics(PTCT)2022-05-03 20:36Financial Performance - Translarna generated $79.2 million in net sales during the quarter ended March 31, 2022[213]. - Emflaza achieved $48.6 million in net sales for the same quarter[213]. - For the three months ended March 31, 2022, net product sales outside of the United States were $81.2 million, compared to $47.8 million for the same period in 2021, representing a 70.0% increase[246]. - Net product sales in the United States for the same period were $48.6 million, up from $43.5 million in 2021, indicating an 11.8% growth[246]. - Net product revenue for the three months ended March 31, 2022, was $129.8 million, an increase of $38.6 million, or 42%, from $91.3 million for the same period in 2021[267]. - Translarna net product revenues were $79.2 million for the three months ended March 31, 2022, an increase of $32.7 million, or 70%, compared to $46.5 million for the same period in 2021[267]. - Emflaza net product revenues were $48.6 million for the three months ended March 31, 2022, an increase of $5.1 million, or 12%, compared to $43.5 million for the same period in 2021[267]. - Royalty revenue for the three months ended March 31, 2022, was $18.9 million, an increase of $12.2 million, or over 100%, from $6.7 million for the same period in 2021[269]. Research and Development - The company anticipates reporting results from the placebo-controlled trial of Study 041 by the end of Q2 2022[214]. - The company initiated a Phase 2 study of PTC518 for Huntington's disease in Q1 2022, expecting initial results by the end of 2022[222]. - A registration-directed Phase 3 trial for PTC923 for phenylketonuria was initiated in Q3 2021, with results expected by the end of 2022[227]. - The company completed a Phase 1 trial for PTC857, which was well-tolerated, and initiated a Phase 2 trial for amyotrophic lateral sclerosis in Q1 2022[226]. - The company expects to incur ongoing research and development expenses for its products and product candidates, including studies for emvododstat for COVID-19 and other therapeutic areas[238]. - Research and development expenses for the three months ended March 31, 2022, were $140.1 million, an increase of $5.6 million, or 4%, from $134.5 million for the same period in 2021[274]. Regulatory and Market Access - The marketing authorization for Translarna in the EEA is subject to annual review and renewal, with a renewal request submitted in February 2022[214]. - The company expects a regulatory decision on Waylivra for familial partial lipodystrophy in the second half of 2022[220]. - The company’s ability to generate revenue from Translarna is dependent on maintaining marketing authorizations and securing market access in various territories[283]. - The company plans to submit a BLA to the FDA for PTC-AADC in the third quarter of 2022[294]. Expenses and Financial Obligations - The company had an accumulated deficit of $2,224.7 million as of March 31, 2022, with a net loss of $126.7 million for the three months ended March 31, 2022[236]. - Selling, general and administrative expenses were $73.3 million for the three months ended March 31, 2022, an increase of $12.2 million, or 20%, from $61.1 million for the same period in 2021[275]. - Interest expense, net was $23.5 million for the three months ended March 31, 2022, an increase of $4.4 million, or 23%, from $19.2 million for the same period in 2021[277]. - The company expects selling, general and administrative expenses to increase in future periods due to ongoing commercialization efforts[261]. - The company anticipates significant expenses related to commercialization efforts in various regions, including the United States and EEA, and ongoing research and development costs[294]. - The company expects to pay $70.0 million to former equityholders of Agilis upon achieving certain milestones in 2022 related to PTC-AADC[306]. - The company may need to obtain substantial additional funding to sustain operations and achieve profitability[308]. Cash Flow and Financing - As of March 31, 2022, the company had cash, cash equivalents, and marketable securities totaling $587.8 million[290]. - Net cash used in operating activities was $97.4 million for the three months ended March 31, 2022, compared to $100.2 million for the same period in 2021[291]. - The company incurred net cash provided by investing activities of $49.0 million for the three months ended March 31, 2022, primarily due to net sales and redemption of marketable securities[292]. - The company closed a private offering of $150.0 million in aggregate principal amount of 3.00% convertible senior notes due 2022, receiving net proceeds of approximately $145.4 million after expenses[285]. - The company believes its cash flows from product sales and existing cash will be sufficient to fund operating expenses for at least the next twelve months[298]. Impact of COVID-19 - The COVID-19 pandemic has not significantly affected revenue generation, but may impact future revenue due to operational challenges[210]. Product Pipeline - The company has a pipeline of product candidates focused on multiple therapeutic areas, including rare diseases and oncology[230]. - The company may seek to expand its product pipeline through in-licensing or acquiring rights to new products, which could increase future capital requirements[239]. - The company expects to incur substantial costs in connection with regulatory submissions and commercialization efforts for its products[294].