Ionis Pharmaceuticals(IONS) - 2022 Q3 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company reported revenues of $160 million for Q3 2022 and $435 million year-to-date, representing a 20% increase over the previous year [35] - Operating expenses increased due to investments in advancing the pipeline and commercial readiness, with cash and investments remaining steady at approximately $2 billion [36][40] - Non-GAAP operating expenses are projected to be at the lower end of guidance, between $825 million and $850 million, with an increase in cash guidance from $1.7 billion to $2 billion due to a recent sale-leaseback transaction [47][44] Business Line Data and Key Metrics Changes - SPINRAZA's global sales were $431 million for Q3 and $1.3 billion year-to-date, with royalty revenue of $62 million for Q3 [37] - R&D revenue for Q3 was $87 million, with significant contributions from collaborations with Biogen and Roche [39] - The company is advancing multiple Phase III studies, with eplontersen and tofersen expected to add to the commercial portfolio next year [7][50] Market Data and Key Metrics Changes - The company anticipates a potential approval for tofersen in the first half of 2023, with eplontersen's NDA filing expected by the end of 2022 [15][50] - The CARDIO-TTRansform study has increased its target enrollment to 1,400 patients to better reflect the patient population and improve study outcomes [19][20] Company Strategy and Development Direction - The company is focused on expanding its commercial pipeline and diversifying its technology, with plans for a new state-of-the-art manufacturing facility to support growth [10][42] - The recent sale-leaseback transaction is aimed at strengthening the balance sheet and funding growth initiatives [11][45] - The company is also exploring new technologies, including LICA technology and MSPA backbone chemistry, to enhance drug delivery and efficacy [9][92] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's position for substantial growth, with a steady cadence of Phase III data readouts expected in the coming years [8][52] - The management highlighted the importance of the evolving landscape in ATTR cardiomyopathy and the need to adapt study designs accordingly [58][60] - The company remains optimistic about the potential of its pipeline and the ability to deliver transformative medicines to the market [50][48] Other Important Information - The company is preparing for multiple product launches and has made significant advancements in its pipeline, including positive data from several mid-stage programs [25][29] - The company is also focused on addressing unmet medical needs in the market, particularly in the HAE and HBV treatment areas [99][106] Q&A Session Summary Question: Rationale for expanding the cardiomyopathy study - Management explained that the population with ATTR cardiomyopathy has changed, necessitating an increase in study size to ensure proper powering without extending the timeline [56][60] Question: NDA extension for tofersen - The NDA for tofersen was extended due to the FDA needing more time to review data, not because of a request for additional data [56][57] Question: Update on the Angelman program - The company is currently enrolling in the open-label extension study and plans to complete it as rapidly as possible [86][88] Question: Differentiation of HBV program - The company highlighted that pelacarsen as a single agent has shown potential for functional cures in HBV, differentiating it from competitor approaches [101][106]