Ionis Pharmaceuticals(IONS) - 2022 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company reported revenues of $134 million for Q2 2022 and $276 million for the first half of the year, showing an increase compared to the same periods last year [16] - SPINRAZA's global sales were $431 million for Q2 and $904 million year-to-date, resulting in $60 million and $113 million in royalty revenue for the corresponding periods [17] - Cash and investments remained flat at $2 billion from Q1 to Q2 [17] - Non-GAAP operating expenses increased due to higher R&D expenses, while SG&A expenses decreased year-over-year [23][24] - The company anticipates Q3 revenues to be similar to Q2, with R&D expenses projected to increase by 25% to 30% compared to last year [25][26] Business Line Data and Key Metrics Changes - The company is advancing its late-stage pipeline, with positive results from the Phase III NEUROTTRansform study of eplontersen in patients with TTR polyneuropathy [8][30] - Enrollment in the BALANCE study of olezarsen for familial chylomicronemia syndrome has been completed, with data expected next year [11][33] - The company reported positive data from six mid-stage programs, with plans to advance two new medicines into Phase III studies next year [13] Market Data and Key Metrics Changes - SPINRAZA sales in the US stabilized year-to-date, with a decrease in discontinuations during the quarter [18] - The company expects SPINRAZA to return to growth as positive trends in the US market are observed [18] - The Lp(a) HORIZON cardiovascular outcome study of pelacarsen has enrolled over 8,000 patients, solidifying the company's first-mover advantage [12][40] Company Strategy and Development Direction - The company aims to deliver transformational medicines to the market, with a focus on expanding its Phase III pipeline to at least eight medicines across ten indications [14][46] - The partnership with AstraZeneca is crucial for the global go-to-market strategy for eplontersen and other products [9][76] - The company is also focused on advancing new chemistries and expanding its LICA platform beyond the liver [47] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the positive results from ongoing studies and the potential for eplontersen and tofersen to enter the market soon [10][48] - The company is well-positioned to maximize value for patients and shareholders through its strategic investments and pipeline advancements [14][27] - Management highlighted the importance of generating robust clinical data to support the competitive positioning of its products in the market [111][114] Other Important Information - The NDA for tofersen is under priority review with the FDA, with a PDUFA date set for January 25, 2023 [10][36] - The company is preparing for additional studies related to eplontersen as part of its life cycle management strategy [70] Q&A Session Summary Question: What do you hope to show at the upcoming presentation at the ISA meeting regarding Eplontersen? - Management expressed excitement about sharing the efficacy, safety, and tolerability data at the ISA meeting, emphasizing the importance of the primary and secondary endpoints [50][51] Question: Will you consider filing any of your other neuro or ALS programs with NFL as a surrogate endpoint? - Management indicated that while biomarkers are important, they do not plan to file based solely on biomarkers but will use them as part of a robust study design [62][64] Question: Can you provide an update on the HBV program with GSK? - Management highlighted encouraging data from GSK's Bepirovirsen and the intention to move into Phase 3 development [84][85] Question: What is the differentiation of your Angelman program from other therapies? - Management noted that their drug targets the antisense transcript and aims to be the best molecule in the space, with a focus on safety and target engagement [102][104] Question: Can you confirm if you have done an interim analysis on cardio transform before expanding to 1,000 patients? - Management clarified that the decision to expand the study was based on demographics rather than event rates, emphasizing the importance of a robust data set [131][132]