Financial Performance - The company recognized $67.2 million in net sales from Translarna™ (ataluren) during the quarter ended September 30, 2021[219]. - Emflaza® (deflazacort) generated $47.1 million in net sales in the same quarter[219]. - Net product revenues for the three months ended September 30, 2021, were $68.5 million outside the United States and $47.1 million in the United States, representing increases from $44.2 million and $38.5 million, respectively, in the same period of 2020[252]. - For the nine months ended September 30, 2021, net product sales outside the United States were $170.2 million, up from $124.0 million in 2020, while U.S. sales were $139.8 million, compared to $102.1 million in 2020[253]. - Net product revenue for the three months ended September 30, 2021, was $115.6 million, an increase of $32.9 million, or 40%, compared to $82.7 million for the same period in 2020[274]. - Net product revenues for the nine months ended September 30, 2021, were $310.0 million, an increase of $83.9 million, or 37%, compared to $226.1 million for the same period in 2020[288]. Research and Development - The company initiated a registration-directed Phase 3 trial for PTC923 for phenylketonuria in the third quarter of 2021, with results expected by the end of 2022[233]. - The company completed a Phase 1 trial for PTC857, which was well-tolerated with no serious adverse events reported[232]. - The company anticipates results from a Phase 2/3 trial of emvododstat for COVID-19 by the end of 2021[234]. - The company expects ongoing research and development expenses to increase due to commercialization efforts and regulatory submissions, including a BLA submission for PTC-AADC in the first quarter of 2022[244][246]. - Research and development expense was $130.8 million for the three months ended September 30, 2021, an increase of $37.8 million, or 41%, from $93.0 million for the same period in 2020[280]. - Total research and development expenses for the nine months ended September 30, 2021, were $390.8 million, compared to $359.6 million for the same period in 2020[274]. Regulatory Approvals and Milestones - The Brazilian health regulatory authority approved Waylivra™ (volanesorsen) in August 2021, marking it as the first treatment for familial chylomicronemia syndrome in Brazil, with a milestone payment of $4.0 million made to Akcea Therapeutics[218]. - The company expects an opinion from the CHMP regarding PTC-AADC for AADC deficiency in the fourth quarter of 2021[230]. - The marketing authorization for Translarna in the EEA is subject to annual review and renewal, with the next renewal effective through August 5, 2022[220]. - The company is preparing a biologics license application for PTC-AADC for the treatment of AADC deficiency in the United States, expected to be submitted in the first quarter of 2022[230]. Financial Position and Funding - The company had an accumulated deficit of $1,954.7 million as of September 30, 2021, with a net loss of $380.6 million for the nine months ended September 30, 2021[243]. - The 2022 Convertible Notes had an aggregate principal amount of $150.0 million, with net proceeds of approximately $145.4 million after expenses[239]. - The 2026 Convertible Notes consist of $287.5 million in aggregate principal amount, with net proceeds of $279.3 million after expenses[242]. - The company expects to continue incurring significant expenses and operating losses for at least the next fiscal year, with net losses fluctuating significantly from quarter to quarter[306]. - The company may need to obtain substantial additional funding to continue operations and achieve profitability, relying on equity offerings, debt financings, and collaborations[325]. - As of September 30, 2021, the company had cash, cash equivalents, and marketable securities totaling $867.9 million[311]. Expenses and Costs - Selling, general and administrative expense was $69.3 million for the three months ended September 30, 2021, an increase of $11.4 million, or 20%, from $57.8 million for the same period in 2020[281]. - Selling, general and administrative expenses increased by $29.5 million, or 17%, to $199.2 million for the nine months ended September 30, 2021, due to investments in commercial activities[296]. - Interest expense, net was $21.8 million for the three months ended September 30, 2021, an increase of $0.8 million, or 4%, from $21.0 million for the same period in 2020[283]. - Interest expense, net, was $63.5 million for the nine months ended September 30, 2021, an increase of $31.5 million, or 98%, from $32.1 million for the same period in 2020[300]. - The company anticipates increased expenses related to commercialization efforts in various regions, including the United States and EEA, and ongoing research and development[317]. Revenue Sources - Collaboration revenue decreased to $10.0 million for the three months ended September 30, 2021, a decline of $25.0 million, or 71%, from $35.0 million in the same period of 2020[275]. - Royalty revenue increased to $13.1 million for the three months ended September 30, 2021, up $12.4 million, or over 100%, from $0.7 million for the same period in 2020[277]. - Collaboration revenues decreased by $5.0 million, or 14%, to $30.0 million for the nine months ended September 30, 2021, due to milestone payments triggered in the current period[289]. - Royalty revenue increased by $32.7 million, or 100%, to $33.3 million for the nine months ended September 30, 2021, driven by the FDA approval of Evrysdi[292]. Market and Operational Risks - The company has never been profitable and may need to obtain substantial additional funding to sustain operations[249]. - The company has historically financed operations through various means, including public offerings and collaborations, and plans to maintain this approach moving forward[305]. - The company has no material changes in market risk management compared to previous disclosures[329]. - Disclosure controls and procedures were evaluated as effective at the reasonable assurance level as of September 30, 2021[330].

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]