PTC Therapeutics(US:PTCT)2025-02-27 21:32Financial Performance - Translarna achieved sales of $339.9 million during the year ended December 31, 2024[745]. - Emflaza net sales reached $207.2 million for the year ended December 31, 2024[745]. - The company had an accumulated deficit of $3,646.9 million as of December 31, 2024[772]. - The net loss for the fiscal year ended December 31, 2024, was $363.3 million[772]. - Net product revenue for the year ended December 31, 2024, was $601.0 million, a decrease of $60.3 million, or 9%, from $661.2 million in 2023[814]. - Total revenues for PTC Therapeutics, Inc. in 2024 were $806.78 million, a decrease of 13.9% from $937.82 million in 2023[911]. - The company reported a net loss of $363.30 million in 2024, compared to a net loss of $626.60 million in 2023, indicating an improvement of 42.0%[914]. - Total current assets increased to $1.37 billion in 2024 from $1.22 billion in 2023, representing a growth of 12.1%[909]. - Cash and cash equivalents rose to $779.71 million in 2024, up from $594.00 million in 2023, a significant increase of 31.2%[909]. - Total liabilities increased to $2.80 billion in 2024 from $2.71 billion in 2023, marking a rise of 3.3%[909]. - The company’s accumulated deficit grew to $3.65 billion in 2024 from $3.28 billion in 2023, an increase of 11.1%[909]. Research and Development - For the year ended December 31, 2024, total research and development expenses were $534.5 million, a decrease from $666.6 million in 2023[791]. - The increase in milestone expenses for 2024 was primarily due to achieving three regulatory milestones for sepiapterin totaling $65.0 million[794]. - The company incurred $219.6 million in development expenses for the year ended December 31, 2024, reflecting a strategic focus on high-potential research programs[791]. - Research and development expenses were $534.48 million in 2024, down from $666.56 million in 2023, a decrease of 19.8%[911]. Regulatory Approvals and Milestones - The FDA accepted the NDA resubmission for Translarna for review in October 2024, with no specified action date[750]. - Upstaza received accelerated approval from the FDA for AADC deficiency in November 2024[752]. - The CHMP is expected to provide an opinion on sepiapterin in the second quarter of 2025[757]. - The EMA validated and accepted the marketing authorization application for sepiapterin in May 2024[757]. - The marketing authorization for Translarna in the EEA is pending the European Commission's decision on a negative opinion from the CHMP[746]. - The FDA accepted the NDA for vatiquinone in February 2025, with a target regulatory action date of August 19, 2025[759]. - The Company achieved the primary endpoint in its Phase 3 trial for sepiapterin for PKU, with an MAA submitted to the EMA in March 2024 and an NDA submitted to the FDA in July 2024[934]. - Vatiquinone's NDA was submitted to the FDA in December 2024, with priority review granted and a target regulatory action date of August 19, 2025[938]. Collaborations and Agreements - The Novartis Agreement includes an upfront payment of $1.0 billion and potential additional payments of up to $1.9 billion in milestones[770]. - The company received an upfront payment of $1.0 billion from Novartis under the Novartis Agreement, with potential total payments of up to $1.9 billion in development, regulatory, and sales milestones[785]. - The company is exploring opportunities for in-licensing or acquiring rights to products and technologies to expand its pipeline[775]. Expenses and Financial Obligations - The company anticipates significant expenses related to commercialization efforts and ongoing clinical trials[774]. - The company expects to continue incurring significant selling, general, and administrative expenses in future periods to support commercialization efforts[801]. - The company expects to make additional payments of $57.5 million in cash upon achieving certain regulatory milestones for sepiapterin in 2025[778]. - The company has a total of $127.7 million in obligations stemming from operating leases, with leases expiring in 2039[881]. - The company has obligations totaling $24.0 million from a commercial manufacturing services agreement with MassBio for a term of 12.5 years[882]. Market Performance and Revenue Trends - Translarna net product revenues were $339.9 million for 2024, a decrease of $15.9 million, or 4%, compared to $355.8 million in 2023, attributed to timing of bulk patient orders and a negative opinion from CHMP[814]. - Emflaza net product revenues were $207.2 million for 2024, a decrease of $47.9 million, or 19%, compared to $255.1 million in 2023, driven by the expiration of orphan drug exclusivity[814]. - Collaboration revenue dropped to $0.3 million in 2024, a decrease of $99.7 million, or 100%, from $100.0 million in 2023 due to the absence of a sales milestone[815]. - Royalty revenue increased to $203.9 million in 2024, an increase of $35.0 million, or 21%, from $168.9 million in 2023, driven by higher Evrysdi sales[816]. Cash Flow and Liquidity - As of December 31, 2024, the company had cash and cash equivalents and marketable securities totaling $1,139.7 million[866]. - Net cash used in operating activities was $107.7 million for the year ended December 31, 2024, compared to $158.4 million in 2023 and $356.7 million in 2022[867]. - The company has $1,139.7 million in cash and cash equivalents and marketable securities as of December 31, 2024, which is believed to be sufficient to fund operating expenses for at least the next twelve months[890]. - The company recorded a gain of $99.90 million on the sale of a priority review voucher in 2024[911]. - The company made significant investments in marketable securities, totaling $607,984 thousand in purchases for 2024, compared to $174,086 thousand in 2023[920]. Stockholder Equity and Shares - The total stockholders' equity (deficit) as of December 31, 2024, was $(1,098,071) thousand, compared to $(818,555) thousand in 2023[917]. - PTC Therapeutics, Inc. had 76.85 million weighted-average shares outstanding in 2024, compared to 74.84 million in 2023, reflecting an increase of 2.8%[911]. - The company issued common stock in connection with an employee stock purchase plan, raising $5,199 thousand in 2024[920]. - The company reported share-based compensation expense of $74,615 thousand in 2024, down from $101,636 thousand in 2023[920].