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 ...

Call Start: 16:30 January 1, 0000 5:52 PM ET PTC Therapeutics, Inc (NASDAQ:PTCT) Q4 2020 Earnings Conference Call February 25, 2021 16:30 PM ET Company Participants Kylie O'Keefe - Head of Investor Relations Stuart Peltz - Founder and Chief Executive Officer Emily Hill - Chief Financial Officer Eric Pauwels - Chief Business Officer Matthew Klein - Chief Development Officer Conference Call Participants Eric Joseph - JPmorgan Li Watsek - Cantor Fitzgerald & Co Minh Vong - Truist Securities Joel Beatty - Citi ...

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, 2020 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 2021 J.P. Morgan Healthcare Conference Stuart W. Peltz, Ph.D., CEO THERAPESTIES 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 release, other than statements of historic fact, are forward-looking statements, including statements with respect to 2021 net product revenue guidance, statements with respect to the 2021 operating expenditure guidance and statements r ...

Financial Data and Key Metrics Changes - Total revenues for Q3 2020 were $118.4 million, a 66% increase year-over-year, including $82.7 million in net product revenue and $35.7 million in collaboration and royalty revenue [38][42] - Net product revenue for Emflaza was $38.5 million, up from $22.9 million in Q3 2019, reflecting strong growth driven by new patient prescriptions [40] - Translarna net product revenues were $43.4 million, down from $48.3 million in Q3 2019, primarily due to delays in Brazil's group purchase orders [39][42] - Non-GAAP R&D expenses were $83.8 million, up from $58.1 million in Q3 2019, reflecting increased investment in research programs and clinical pipeline advancement [40][42] - Net loss for Q3 2020 was $69.7 million, compared to a net loss of $60 million in Q3 2019 [42] Business Line Data and Key Metrics Changes - Evrysdi showed strong launch performance in the U.S., with a positive response from the SMA community and significant uptake among previously treated patients [7][9] - Emflaza product revenues increased by 68% year-over-year, with high compliance and low patient discontinuations [32] - Translarna continued to exceed expectations outside of Brazil, with a purchase agreement secured with Brazil's Ministry of Health [32][39] Market Data and Key Metrics Changes - Evrysdi has been approved in multiple countries, including Brazil, Ukraine, and Chile, with an NDA filed in Japan triggering a $7.5 million milestone payment [9][10] - The company estimates approximately 12,000 commercially addressable mitochondrial epilepsy patients globally [15] - There are an estimated 50,000 PKU patients globally, with 20,000 in the U.S., indicating a significant unmet medical need [16][28] Company Strategy and Development Direction - The company aims to create value by developing novel therapeutics for patients with medical needs and expanding its global commercial engine [6] - PTC is focused on advancing its R&D pipeline, including the splicing platform and gene therapy programs, to bring more therapies to patients [11][18] - The company is actively pursuing patient finding activities in preparation for potential launches in Europe next year [34] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong launch of Evrysdi and the ongoing growth of the commercial portfolio despite COVID-19 challenges [6][10] - The company anticipates reporting top-line results for several clinical trials in the first half of 2021, including PTC 518 for Huntington's disease and PTC 299 for COVID-19 [19][27] - Management highlighted the importance of collaboration with partners and the SMA community to address high unmet medical needs [9][10] Other Important Information - The company has a strong cash position of approximately $1.14 billion as of September 30, 2020, allowing for continued investment in its pipeline [42] - The gene therapy manufacturing facility is ready for production, with ongoing progress in the ADC deficiency program [18][29] Q&A Session Summary Question: Can you confirm if the IND for PTC 518 has been filed? - Management confirmed that all regulatory and ethics documents have been approved, and the study is set to start soon [45][46] Question: What are the expectations for the mitochondrial epilepsy trial enrollment timeline? - Management expects the trial to be completed by the end of 2022, with strategies in place to minimize COVID-19 impact [55][56] Question: What are the expectations for the Translarna data and approval timelines? - Management indicated that positive dystrophin data could lead to an accelerated approval submission in the first half of 2021 [62][63] Question: How is the patient identification process for AADC gene therapy going? - Management reported active efforts in patient education and screening, with a goal of identifying 300 or more addressable patients [66][68]

Financial Data and Key Metrics Changes - Total revenues for Q2 2020 were $75.2 million, down from $85.5 million in Q2 2019, reflecting a decrease primarily due to delays in Brazil's group purchase order [28] - Translarna net product revenues were $38.6 million for Q2 2020, compared to $57.8 million in Q2 2019, impacted by the Brazil order delay [28] - Emflaza reported net product revenues of $36.2 million for Q2 2020, up from $27.6 million in Q2 2019, indicating over 30% year-over-year growth [28] Business Line Data and Key Metrics Changes - The Duchenne muscular dystrophy (DMD) franchise experienced nearly 20% growth year-over-year, excluding Brazil [20] - Emflaza had a strong quarter with over 30% year-over-year growth, driven by new patient prescriptions and operational efficiencies [23] - Translarna continued to grow in key markets outside Brazil, with new patient identification despite COVID-19 challenges [21][20] Market Data and Key Metrics Changes - The COVID-19 pandemic has caused administrative delays in Brazil, affecting the group purchase order for Translarna, but new patient identification has continued in Europe and Latin America [22][20] - The market for refractory mitochondrial epilepsy is estimated at approximately 11,000 to 13,000 patients in the U.S., EU, Latin America, and Japan [14] - The market size for Friedreich ataxia is estimated to be around 25,000 patients in aggregate across the U.S., EU, Australia, and Latin America [14] Company Strategy and Development Direction - The company aims to expand its splicing platform, anticipating 3 to 5 new development candidates over the next 3 to 5 years [7] - The acquisition of PTC923 for phenylketonuria (PKU) enhances the company's platform capabilities and addresses unmet medical needs in inborn errors of metabolism [7] - The company is focused on advancing key programs despite COVID-19, including trials for PTC518 for Huntington's disease and PTC299 for COVID-19 [9][8] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of risdiplam as a competitive product for spinal muscular atrophy (SMA), with a PDUFA date approaching [5] - The company has taken proactive steps to mitigate risks associated with COVID-19 and has continued to execute on key programs [9] - Management remains optimistic about the growth trajectory of Emflaza and the DMD franchise, expecting strong performance to continue into the second half of the year [23] Other Important Information - The company completed a transaction with Royalty Pharma, bringing in $650 million in non-dilutive capital, which will support ongoing operations and growth [26] - Non-GAAP R&D expenses for Q2 2020 were $168 million, significantly higher than $54.5 million in Q2 2019, due to one-time charges related to acquisitions and manufacturing agreements [29] - Cash, cash equivalents, and marketable securities totaled $498.9 million as of June 30, 2020, with pro forma cash exceeding $1.1 billion after the Royalty Pharma deal [30] Q&A Session Summary Question: Importance of business development versus splicing platform development - Management emphasized that both business development and splicing platform development are critical, with a focus on maintaining a broad pipeline to address unmet medical needs [34][36] Question: Types of programs sought for business development - The company is looking for selective and strategic opportunities within its existing therapeutic areas, including gene therapy and inborn errors of metabolism [40] Question: Confidence in FDA approval for risdiplam - Management expressed strong confidence in receiving FDA approval by the PDUFA date, highlighting the product's broad efficacy and competitive advantages [42] Question: Remaining steps for BLA filing for AADC - The company is awaiting completion of surgical procedures with the SmartFlow cannula, which is essential for the BLA submission [46] Question: Patient identification efforts for mitochondrial epilepsy trial - The company is leveraging existing relationships with patient foundations to enhance patient identification and enrollment for the trial [71] Question: Impact of COVID-19 on Translarna revenues - The decline in Translarna revenues is primarily attributed to delays in Brazil, while key markets outside Brazil have shown growth [76]