Ionis Pharmaceuticals(IONS) - 2023 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company reported revenues of $188 million for Q2 2023, representing a 40% increase compared to the same period last year, and $319 million year-to-date, a 16% increase [54] - R&D revenue for Q2 was $110 million, with a year-to-date total of $173 million, both significantly higher than the previous year [31] - Operating expenses and operating loss increased as the company advanced its commercial readiness and pipeline, with full-year R&D expenses projected to rise by 20% to 25% year-over-year [29][58] Business Line Data and Key Metrics Changes - The company has 12 medicines in clinical development for neurological diseases, with SPINRAZA continuing to be the global market leader for SMA treatment, generating $437 million in sales for Q2 [21][56] - Eplontersen is under regulatory review in the U.S. and additional filings are planned for the EU and Latin America, with the goal of bringing it to patients globally [9][13] - Olezarsen is expected to be the first independent commercial launch for the company, with a Phase 3 BALANCE study readout anticipated in the second half of the year [10][24] Market Data and Key Metrics Changes - The market for severely elevated triglycerides includes over 3.5 million patients, with a significant unmet need for effective treatments [38] - The company is preparing for the launch of eplontersen, olezarsen, and donidalorsen, which have a combined multibillion-dollar peak sales potential [59] Company Strategy and Development Direction - The company aims to transform into a fully integrated biopharma, focusing on delivering new medicines to patients and advancing its commercial capabilities [19] - The collaboration with AstraZeneca has been expanded to include Latin America, emphasizing the commitment to bringing eplontersen to patients worldwide [42] - The company is also expanding its cardiovascular franchise with a new cardiac muscle-targeting drug entering preclinical development [12] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving key regulatory milestones and emphasized the importance of upcoming data readouts for their late-stage pipeline [27][35] - The company remains focused on advancing its key priorities and is optimistic about the potential for substantial revenue growth and long-term value for shareholders [59][60] Other Important Information - The company has completed enrollment in the Phase 3 CARDIO-TTRansform study, which is the largest study ever conducted in the ATTR cardiomyopathy indication [20] - The company is also preparing for the launch of donidalorsen, with a Phase 3 OASIS-HAE study completed and data expected in the first half of next year [47] Q&A Session All Questions and Answers Question: What is the company's strategy for addressing the unmet need in the market for severely elevated triglycerides? - The company is focused on launching olezarsen for FCS and SHTG, addressing the significant unmet need for patients with severely elevated triglycerides [37][38] Question: How does the company plan to compete against siRNA options in the ATTR-PN market? - The company aims to be first in many global markets, with plans to file in various regions outside the U.S. in the second half of the year [91][92] Question: Can you provide an update on the Angelman syndrome clinical study? - The Angelman program is progressing well, with plans to have data available around midyear next year [80]