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]

Splicing Platform Deep Dive July 24, 2020 Forward Looking Statement 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 future revenue and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical tri ...

PTC Therapeutics Jefferies Virtual Healthcare Conference Stuart W. Peltz, Ph.D., CEO Forward looking statement 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 future revenue and statements regarding: the future expectations, plans and prospects for PTC, including with res ...

PTC Therapeutics, Inc. (NASDAQ:PTCT) Q1 2020 Earnings Conference Call April 30, 2020 4:30 PM ET Company Participants Alex Kane - Head of Investor Relations Stuart Peltz - Chief Executive Officer Emily Hill - Chief Financial Officer Matthew Klein - Chief Development Officer Eric Pauwels - Chief Business Officer Conference Call Participants Eileen Maysek - Cantor Fitzgerald Tazeen Ahmad - BofA Merrill Lynch Joel Beatty - Citigroup Joseph Thome - Cowen and Company Vincent Chen - Bernstein Peter Kim - Barclays ...

Fourth Quarter and Full Year 2019 Financial Results & Corporate Update PTC Therapeutics, Inc. Forward looking statement 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 2020 net product revenue guidance, statements with respect to the 2020 non-GAAP R&D and SG&A expense g ...

PTC Therapeutics, Inc. (NASDAQ:PTCT) Q4 2019 Earnings Conference Call March 2, 2020 4:30 PM ET Company Participants Alex Kane - Head of Investor Relations Stuart Peltz - Chief Executive Officer Marcio Souza - Chief Operating Officer Emily Hill - Chief Financial Officer Conference Call Participants Alethia Young - Cantor Fitzgerald Joe Thome - Cowen and Company Martin Auster - Credit Suisse Vincent Chen - Bernstein Robyn Karnauskas - SunTrust Robinson Humphrey Sami Corwin - William Blair Gena Wang - Barclays ...

PTC 2020 JP Morgan Healthcare Conference Stuart W. Peltz, Ph.D., CEO Forward looking statement 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 related to preliminary unaudited 2019 financial information with respect to 2019 net product revenue of Translarna for the treatment of nmDMD and ...