PTC Therapeutics(US:PTCT)2021-10-28 20:42Financial Performance - The company recognized $67.2 million in net sales from Translarna™ (ataluren) during the quarter ended September 30, 2021[219]. - Emflaza® (deflazacort) generated $47.1 million in net sales in the same quarter[219]. - Net product revenues for the three months ended September 30, 2021, were $68.5 million outside the United States and $47.1 million in the United States, representing increases from $44.2 million and $38.5 million, respectively, in the same period of 2020[252]. - For the nine months ended September 30, 2021, net product sales outside the United States were $170.2 million, up from $124.0 million in 2020, while U.S. sales were $139.8 million, compared to $102.1 million in 2020[253]. - Net product revenue for the three months ended September 30, 2021, was $115.6 million, an increase of $32.9 million, or 40%, compared to $82.7 million for the same period in 2020[274]. - Net product revenues for the nine months ended September 30, 2021, were $310.0 million, an increase of $83.9 million, or 37%, compared to $226.1 million for the same period in 2020[288]. Research and Development - The company initiated a registration-directed Phase 3 trial for PTC923 for phenylketonuria in the third quarter of 2021, with results expected by the end of 2022[233]. - The company completed a Phase 1 trial for PTC857, which was well-tolerated with no serious adverse events reported[232]. - The company anticipates results from a Phase 2/3 trial of emvododstat for COVID-19 by the end of 2021[234]. - The company expects ongoing research and development expenses to increase due to commercialization efforts and regulatory submissions, including a BLA submission for PTC-AADC in the first quarter of 2022[244][246]. - Research and development expense was $130.8 million for the three months ended September 30, 2021, an increase of $37.8 million, or 41%, from $93.0 million for the same period in 2020[280]. - Total research and development expenses for the nine months ended September 30, 2021, were $390.8 million, compared to $359.6 million for the same period in 2020[274]. Regulatory Approvals and Milestones - The Brazilian health regulatory authority approved Waylivra™ (volanesorsen) in August 2021, marking it as the first treatment for familial chylomicronemia syndrome in Brazil, with a milestone payment of $4.0 million made to Akcea Therapeutics[218]. - The company expects an opinion from the CHMP regarding PTC-AADC for AADC deficiency in the fourth quarter of 2021[230]. - The marketing authorization for Translarna in the EEA is subject to annual review and renewal, with the next renewal effective through August 5, 2022[220]. - The company is preparing a biologics license application for PTC-AADC for the treatment of AADC deficiency in the United States, expected to be submitted in the first quarter of 2022[230]. Financial Position and Funding - The company had an accumulated deficit of $1,954.7 million as of September 30, 2021, with a net loss of $380.6 million for the nine months ended September 30, 2021[243]. - The 2022 Convertible Notes had an aggregate principal amount of $150.0 million, with net proceeds of approximately $145.4 million after expenses[239]. - The 2026 Convertible Notes consist of $287.5 million in aggregate principal amount, with net proceeds of $279.3 million after expenses[242]. - The company expects to continue incurring significant expenses and operating losses for at least the next fiscal year, with net losses fluctuating significantly from quarter to quarter[306]. - The company may need to obtain substantial additional funding to continue operations and achieve profitability, relying on equity offerings, debt financings, and collaborations[325]. - As of September 30, 2021, the company had cash, cash equivalents, and marketable securities totaling $867.9 million[311]. Expenses and Costs - Selling, general and administrative expense was $69.3 million for the three months ended September 30, 2021, an increase of $11.4 million, or 20%, from $57.8 million for the same period in 2020[281]. - Selling, general and administrative expenses increased by $29.5 million, or 17%, to $199.2 million for the nine months ended September 30, 2021, due to investments in commercial activities[296]. - Interest expense, net was $21.8 million for the three months ended September 30, 2021, an increase of $0.8 million, or 4%, from $21.0 million for the same period in 2020[283]. - Interest expense, net, was $63.5 million for the nine months ended September 30, 2021, an increase of $31.5 million, or 98%, from $32.1 million for the same period in 2020[300]. - The company anticipates increased expenses related to commercialization efforts in various regions, including the United States and EEA, and ongoing research and development[317]. Revenue Sources - Collaboration revenue decreased to $10.0 million for the three months ended September 30, 2021, a decline of $25.0 million, or 71%, from $35.0 million in the same period of 2020[275]. - Royalty revenue increased to $13.1 million for the three months ended September 30, 2021, up $12.4 million, or over 100%, from $0.7 million for the same period in 2020[277]. - Collaboration revenues decreased by $5.0 million, or 14%, to $30.0 million for the nine months ended September 30, 2021, due to milestone payments triggered in the current period[289]. - Royalty revenue increased by $32.7 million, or 100%, to $33.3 million for the nine months ended September 30, 2021, driven by the FDA approval of Evrysdi[292]. Market and Operational Risks - The company has never been profitable and may need to obtain substantial additional funding to sustain operations[249]. - The company has historically financed operations through various means, including public offerings and collaborations, and plans to maintain this approach moving forward[305]. - The company has no material changes in market risk management compared to previous disclosures[329]. - Disclosure controls and procedures were evaluated as effective at the reasonable assurance level as of September 30, 2021[330].