PTC Therapeutics(PTCT) - 2020 Q2 - Earnings Call Transcript

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]