Sales 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].

PTC 2022 J.P. Morgan Healthcare Conference Stuart W. Peltz, Ph.D., CEO Forward Looking Statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this presentation, other than statements of historic fact, are forward-looking statements, including statements with respect to preliminary unaudited 2021 financial information with respect to 2021 total net revenue and 2021 DMD franchise net product reven ...

PTC 2021 Corporate Deck Updated Nov 30, 2021 PTC Forward Looking Statements 2 This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 199 5. All statements contained in this release, other than statements of historic fact, are forward -looking statements, including statements with respect to 2021 net p roduct revenue guidance, statements with respect to the 2021 operating expenditure guidance and statements regarding: the future expectation ...

PTC Therapeutics, Inc. (NASDAQ:PTCT) Q3 2021 Earnings Conference Call October 28, 2021 4:30 PM ET Company Participants Kylie O'Keefe – Head-Investor Relations Stuart Peltz – Chief Executive Officer Matthew Klein – Chief Development Officer Eric Pauwels – Chief Business Officer Emily Hill – Chief Financial Officer Conference Call Participants Danielle Brill – Raymond James Tazeen Ahmad – Bank of America Raju Prasad – William Blair Brian Abrahams – RBC Capital Markets Peyton Bohnsack – Cowen and Company Kripa ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2021 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-35969 PTC Therapeutics, Inc. (Exact name of registrant as specified in its charter) Delaware 04-3416587 ( ...

Financial Data and Key Metrics Changes - The company reported revenues of $117 million for Q2 2021, a 55% increase compared to Q2 2020, including $103 million in net product sales and $14 million in royalty revenue from the Evrysdi program [56][57] - The DMD franchise revenue guidance for 2021 was raised to $370 million to $390 million from the previous guidance of $355 million to $375 million [53][55] Business Line Data and Key Metrics Changes - Translarna achieved $53 million in revenue for Q2 2021, a 36% increase from $39 million in Q2 2020, driven by geographic expansion [57][41] - Emflaza generated $49 million in revenue for Q2 2021, also a 36% increase from $36 million in Q2 2020, supported by high adherence and fewer discontinuations [57][42] Market Data and Key Metrics Changes - Evrysdi saw strong uptake in the US with 1,800 SMA patients on treatment, representing almost 20% market share within less than a year post-launch [14] - Evrysdi was approved in 53 markets outside the US, with early adoption expected to continue as pricing and reimbursement discussions progress [14] Company Strategy and Development Direction - The company aims to maintain a sustained pipeline of innovative treatments for patients with debilitating diseases, focusing on balancing the challenges of drug discovery and development [8][9] - The strategic plan includes expanding geographic reach and enhancing access to therapies, particularly in emerging markets [44][45] Management's Comments on Operating Environment and Future Outlook - Management acknowledged ongoing challenges due to the COVID-19 pandemic but expressed confidence in the company's ability to navigate these issues [10] - The company is optimistic about continued growth in the DMD franchise and expects significant orders from Latin America in the second half of the year [66] Other Important Information - The company has a fully functional gene therapy manufacturing facility in New Jersey, which may create additional revenue streams through service agreements with other companies [26] - The company plans to initiate a Phase 3 registration-directed study for its PKU program in September 2021, addressing a significant unmet medical need [22][23] Q&A Session Summary Question: Concerns about DMD franchise performance and potential risks - Management highlighted strong Q2 performance with significant growth in both Translarna and Emflaza, emphasizing confidence in continued growth despite potential lumpiness in orders [63][66] Question: Market opportunity for Envisat in outpatient settings - Management indicated that while the current trial is focused on hospitalized patients, there is potential for outpatient use depending on regulatory discussions post-trial [69][72] Question: Update on Huntington's program and patient information - Management confirmed that the Phase 1 study demonstrated dose-dependent lowering of Huntington mRNA and protein, with further data expected from the CSF cohort [78][80] Question: Addressable patient population for AADC program - Management stated that they are on track to identify 300 addressable patients globally, with ongoing efforts in high-risk populations [86][88] Question: Selection of ALS for PTC857 trials - Management explained that ALS was chosen due to its rapid progression and validated endpoints, allowing for measurable outcomes within a short treatment window [110][112]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2021 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant t ...

PTC Therapeutics, Inc. (NASDAQ:PTCT) Q1 2021 Results Conference Call May 4, 2021 4:30 PM ET Company Participants Kylie O'Keefe - Senior Vice President Commercial and Corporate Strategy Stuart Peltz - Chief Executive Officer Matthew Klein - Chief Development Officer Eric Pauwels - Chief Business Officer Emily Hill - Chief Financial Officer Conference Call Participants Eric Joseph - JP Morgan Robyn Karnauskas - Truist Securities Brian Abrahams - RBC Capital Markets Joseph Thome - Cowen & Company Colin Bristow ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2021 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-35969 PTC Therapeutics, Inc. (Exact name of registrant as specified in its charter) Delaware 04-3416587 (Stat ...

PTC518 Huntington's Disease Deep Dive April 15th, 2021 PTC) Forward Looking Statements: This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements with respect to the future expectations, plans and prospects for PTC, PTC's strategy, including with respect to the expected timing of clinical trials and stud ...