PTC 2022 Corporate Presentation September 2022 Forward-Looking Statements 2 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 guidance relating to 2022 total revenue, 2022 DMD franchise net product revenue, 2022 operating expenditure guidance and future revenue guidance and s ...

PTC Therapeutics (NASDAQ:PTCT) Q2 2022 Earnings Conference Call August 4, 2022 4:30 PM ET Company Participants Kylie O'Keefe - Senior Vice President, Head of Global Commercial & Corporate Strategy Stuart Peltz - Chief Executive Officer Matthew Klein - Chief Operating Officer Eric Pauwels - Chief Business Officer Emily Hill - Chief Financial Officer Conference Call Participants Eric Joseph - JPMorgan Raju Prasad - William Blair Kristen Kluska - Cantor Fitzgerald Joe Thome - Cowen & Company Tazeen Ahmad - Ban ...

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, 2022 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 (State ...

Translarna™ (Ataluren) Study 041 Topline Results 21st June 2022 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 regarding: the future expectations, plans and prospects for PTC, including with respect to the commercialization of its products and product candidates ...

PTC 2022 Corporate Presentation May 2022 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 guidance relating to 2022 total net product revenue, 2022 DMD franchise net product revenue, 2022 operating expenditure guidance and future revenue guidance a ...

Start Time: 16:30 January 1, 0000 5:36 PM ET PTC Therapeutics, Inc. (NASDAQ:PTCT) Q1 2022 Earnings Conference Call May 03, 2022, 16:30 PM ET Company Participants Stuart Peltz - Founder and CEO Matthew Klein - COO Eric Pauwels - Chief Business Officer Emily Hill - CFO Kylie O’Keefe - SVP, Global Commercial and Corporate Strategy Conference Call Participants Eric Joseph - JPMorgan Rick Krause - Cantor Fitzgerald Brian Abrahams - RBC Capital Markets Joseph Thome - Cowen and Company Gena Wang - Barclays Robyn K ...

Financial 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].

PTC Therapeutics, Inc. (NASDAQ:PTCT) Q4 2021 Earnings Conference Call February 22, 2022 4:30 PM ET Company Participants Kylie O'Keefe - VP, Head of Global Strategic Marketing & Business Intelligence Stuart Peltz - Co-Founder & CEO Matthew Klein - COO Eric Pauwels - Chief Business Officer Emily Hill - CFO Conference Call Participants Alethia Young - Cantor Fitzgerald & Co. Joseph Thome - Cowen and Company Eric Joseph - JPMorgan Chase & Co. Raju Prasad - William Blair & Company Brian Abrahams - RBC Capital Ma ...

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 ...