PTC Therapeutics, Inc. (NASDAQ:PTCT) Q4 2023 Earnings Conference Call February 28, 2024 4:30 PM ET Company Participants Ron Aldridge - Senior Director, IR Matthew Klein - CEO Eric Pauwels - CBO Kylie O'Keefe - CCO Pierre Gravier - CFO Conference Call Participants Brooke Schuster - William Blair Rick Miller - Cantor Fitzgerald Eric Joseph - JPMorgan Jeff Hung - Morgan Stanley Brian Abrahams - RBC Capital Markets Peyton Bohnsack - TD Cowen David Lebowitz - Citi Gena Wang - Barclays Paul Choi - Goldman Sachs J ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☑ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended: December 31, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission file number: 001-35969 PTC THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 04-3416587 (State or other jurisdiction of incorporati ...

PTC Therapeutics, Inc. (NASDAQ:PTCT) Q3 2023 Earnings Conference Call February 8, 2023 9:00 AM ET Company Participants Jane Hanlon - Associate Director, Investor Relations Matthew Klein - Chief Executive Officer Eric Pauwels - Chief Business Officer Kylie O'Keefe - Chief Commercial Officer Pierre Gravier - Chief Financial Officer Conference Call Participants Eric Joseph - J.P. Morgan Kristen Kluska - Cantor Fitzgerald Samantha Corwin - William Blair David Lebowitz - Citi Brian Abrahams - RBC Capital Markets ...

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, 2023 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) (State or other juris ...

Financial Data and Key Metrics Changes - The company reported total revenue of $214 million for the second quarter of 2023, representing a 29% increase compared to the same quarter in 2022 [46] - The DMD franchise revenue totaled $162 million, reflecting a 21% year-over-year growth [55][75] - Non-GAAP R&D expenses were $170 million for the second quarter of 2023, up from $143 million in the same quarter of 2022 [90] Business Line Data and Key Metrics Changes - Translarna generated $96 million in revenue, a 25% increase over the same quarter in 2022 [84] - Emflaza achieved net product revenue of $66 million, representing a 16% growth compared to the second quarter of 2022 [85] - The APHENITY trial for sepiapterin in PKU patients met its primary endpoint with a 63% reduction in blood phenylalanine levels [77] Market Data and Key Metrics Changes - Evrysdi's global revenue for the second quarter was approximately $380 million, with PTC earning $37 million in royalties [63] - The company expects continued growth in the Latin American and Eastern European markets, despite unpredictability in large government orders [28][56] Company Strategy and Development Direction - The company is focused on launching sepiapterin in the US and Europe, with expectations of a $1 billion-plus global commercial opportunity [49][88] - A strategic portfolio review has led to a reduction in operational expenses, with anticipated savings of approximately $150 million in 2024 [76] - The company plans to submit an NDA for sepiapterin in the fourth quarter of 2023 following a pre-NDA meeting with the FDA [77] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving the revenue guidance of $940 million to $1 billion for 2023, which would represent a year-over-year growth of 34% to 43% [75][116] - The company remains optimistic about the potential for Translarna to convert from conditional to standard marketing authorization in Europe [11][52] - Management highlighted the importance of the upright stability subscale in the mFARS scale as a predictor of time to loss of ambulation in Friedreich ataxia [120] Other Important Information - The company has approximately $338 million in cash, cash equivalents, and marketable securities as of June 30, 2023 [117] - The company is well-positioned for multiple product launches, leveraging its existing infrastructure and expertise [123] Q&A Session Summary Question: Clarity on CHMP opinion for Translarna - Management explained that the initial timing for the CHMP opinion was pushed to the third quarter due to additional questions during the review process [10] Question: Capital deployment strategy with $330 million in cash - Management indicated that they are well-capitalized to support upcoming product launches and maintain a robust discovery and development infrastructure [13] Question: Main questions for the PKU pre-NDA meeting - The focus will be on the structure of the NDA and ensuring alignment with the FDA on clinical and safety data [18] Question: Potential decline in Duchenne revenue guidance - Management acknowledged the unpredictability of large orders but expressed confidence in achieving growth despite potential fluctuations [30] Question: Overlap in physician communities for Huntington's disease - Management noted the recognition of the splicing platform's power and the importance of leveraging learnings from previous product launches [20]

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, 2023 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 ...

Financial Data and Key Metrics Changes - Total revenue for Q1 2023 reached $220 million, marking a 40% increase compared to Q1 2022, which is the highest quarterly revenue ever for the company [5][27] - The DMD franchise generated $170 million in revenue, reflecting a 33% increase year-over-year [5][24] - Evrysdi contributed $31 million in royalty revenue, with growth driven by both therapy-naive patients and those previously treated with other therapies [5][6] - The company expects total revenue for 2023 to be between $940 million and $1 billion, indicating potential growth of up to 43% year-over-year [6][27] - Non-GAAP SG&A expenses were $73.4 million for Q1 2023, compared to $59.7 million in Q1 2022 [28] Business Line Data and Key Metrics Changes - Translarna net product revenues were $115.1 million, with strong growth across all geographies [27] - Emflaza had net product revenues of $54.6 million, representing a 12% growth compared to Q1 2022 [27] - The DMD franchise is expected to achieve revenue guidance of $545 million to $565 million for 2023, supported by new patient starts and improved payer access [43][53] Market Data and Key Metrics Changes - The company is expanding its geographic presence, particularly in Latin America and Asia, with successful launches of Waylivra in Brazil and ongoing growth in the DMD franchise [23][24] - The company anticipates a strong rollout of Upstaza in Europe, with positive guidance received from NICE in England and Wales for AADC patients aged 18 months and older [45] Company Strategy and Development Direction - The company aims to leverage its strong commercial portfolio to drive growth in neurological and metabolic disorders [22] - A strategic portfolio review and potential operational expense reduction are expected based on study results to be reported in Q2 2023 [6] - The company is focused on expanding its clinical pipeline, with results from four clinical studies anticipated in the second quarter [7][10] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving the 2023 revenue guidance, citing strong fundamentals and execution across all geographies [22][26] - The company is optimistic about the upcoming clinical trial results and their potential impact on future product launches and market expansion [21][48] Other Important Information - The company reported cash, cash equivalents, and marketable securities totaling approximately $286.3 million as of March 31, 2023, down from $410.7 million at the end of 2022 [28] - Non-GAAP R&D expenses for Q1 2023 were $179.8 million, reflecting increased investment in research programs and clinical pipeline advancement [48] Q&A Session Summary Question: What has driven new patient starts in the DMD portfolio? - Management highlighted geographic expansion, early diagnosis, and rapid treatment initiation as key drivers of growth in new patient starts [31][53] Question: What are the expectations for the APHENITY trial? - The primary analysis population includes subjects with a greater than 30% reduction in phenylalanine levels, with confidence in achieving significant results based on prior data [90][91] Question: How does the company view the competitive landscape for vatiquinone? - The company is exploring ways to strengthen and extend the patent life for vatiquinone, primarily relying on orphan exclusivity [62] Question: What is the confidence level regarding the MOVE-FA study's statistical significance? - Management believes there is sufficient power to detect differences similar to those observed in previous studies, with confidence in capturing treatment benefits [118] Question: What are the implications of the FDA's queries regarding the BLA submission? - The company anticipates potential delays in the BLA submission to Q3 2023 due to ongoing interactions with the FDA [97]

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, 2023 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 ...

Financial Data and Key Metrics Changes - The company achieved total revenue of $699 million in 2022, representing a 30% growth over 2021, despite significant foreign exchange headwinds [169] - For Q4 2022, total revenue was $167 million, consisting of $128 million from net product revenue and $40 million from Evrysdi royalty revenue [14][169] - Non-GAAP R&D expenses for Q4 2022 were $175 million, up from $136 million in Q4 2021, reflecting increased investment in research programs [41] Business Line Data and Key Metrics Changes - The DMD franchise generated $507 million in revenue for 2022, a 20% year-over-year growth, with Q4 revenue of $114 million [162] - Translarna achieved $56 million in revenue for Q4 2022, totaling $289 million for the year, marking a 22% growth [12][178] - Emflaza reported Q4 revenue of $58 million, bringing total annual revenue to $218 million, a 17% increase over 2021 [207] Market Data and Key Metrics Changes - The company is expanding its geographic presence in Latin America, the Middle East, and Asia Pacific, with ongoing efforts to launch Upstaza and grow the DMD franchise [12][38] - The company anticipates significant growth in 2023, with revenue guidance set between $940 million to $1 billion, reflecting over 30% year-over-year growth [155] Company Strategy and Development Direction - The company aims to deliver one new therapy every two to three years, focusing on rare diseases by modulating gene and protein expression [168] - The company is preparing for the launch of Upstaza in the US, with a dedicated team focusing on disease awareness and key publications [125] - The company plans to request a Type C meeting with the FDA to discuss the potential path for NDA resubmission for Translarna [160] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong performance and growth trajectory for 2023, highlighting the excitement around upcoming clinical trial readouts [4][118] - The company noted the importance of addressing the unmet medical needs of patients, particularly in the PKU and mitochondrial disease markets [6][50] Other Important Information - The company was awarded the 2023 EURORDIS Black Pearl Company Award for Innovation, recognizing its contributions to rare disease research [154] - The company has initiated additional dosing cohorts in the PTC518 Huntington's disease program, with results expected in the second quarter of 2023 [36] Q&A Session Summary Question: Can you break down revenue components in your full year 2023 revenue guidance? - The company expects revenue guidance of $940 million to $1 billion, reflecting growth in the DMD franchise, Upstaza, and Evrysdi, including a $100 million milestone for Evrysdi [43] Question: Can you clarify the nature of your discussions with the agency on Translarna? - The company had an informal meeting with the FDA and plans to request another Type C meeting to review the totality of data collected [55][160] Question: What is the expected reduction in seizure frequency considered clinically meaningful for the MIT-E study? - The study is powered for a 40% differential reduction between treatment and placebo groups, with a hypothesized reduction of 50% in the active population [65] Question: Can you provide an update on the APHENITY study? - The company reported that 156 subjects passed screening, with 102 achieving greater than 30% reduction in phenylalanine levels, indicating a high responder rate [203] Question: What is the rationale for dose escalation in the Part 2 study? - The company plans to utilize a two-week dose escalation approach to assess the safety and efficacy of higher doses in the study [134]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☑ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended: December 31, 2022 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission file number: 001-35969 PTC THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 04-3416587 (State or other jurisdiction of incorporati ...