Arrowhead Pharmaceuticals(ARWR)2024-11-27 04:08Financial Data and Key Metrics Changes - The net loss for fiscal 2024 was 5 per share, compared to a net loss of 1.92 per share for 2023 [71] - Revenue in 2024 was 240.7 million in 2023, primarily due to no new partnership agreements or major milestones triggered during the year [72] - Total operating expenses for fiscal 2024 were 445.7 million in 2023, with R&D costs being the key driver of this increase [73][74] - Cash and investments totaled 403.6 million at the same date in 2023, primarily due to a 400 million debt facility [76] Business Line Data and Key Metrics Changes - The company is focusing on its cardiometabolic pipeline, with key programs including plozasiran, zodasiran, and ARO-INHBE, which are progressing towards commercialization [22][30] - The partnership with Sarepta includes select clinical candidates and non-clinical programs, which will allow Arrowhead to focus on its core cardiometabolic assets while generating substantial upfront and milestone payments [10][17] Market Data and Key Metrics Changes - The company expects to launch plozasiran for patients with familial chylomicronemia syndrome (FCS) in mid-2025, with potential expansion into the severe hypertriglyceridemia (SHTG) market thereafter [82] - The market for plozasiran is estimated to be significant, with potential annual revenues of 3 billion, particularly in the underserved SHTG population [33] Company Strategy and Development Direction - The company aims to build a focused pipeline around cardiometabolic programs while retaining select early-stage programs for future value creation [84] - The partnership with Sarepta is seen as transformational, providing immediate capital and a clearer path to profitability while allowing Arrowhead to concentrate on its core therapeutic areas [9][19] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's position, stating that it is funded into 2028 and has a robust pipeline that spans early discovery to Phase-3 ready programs [34][81] - The company is optimistic about the potential for plozasiran and other cardiometabolic programs to generate substantial revenue and address significant unmet medical needs [82][84] Other Important Information - The company has decided not to pursue further development of ARO-MUC5AC due to challenges in assessing target engagement, reallocating resources to more promising programs [28] - The collaboration with Sarepta includes a manufacturing component that will utilize Arrowhead's new Verona manufacturing plant, enhancing operational efficiency [25] Q&A Session Summary Question: Future of Arrowhead CNS franchise and plans for zodasiran - Management is excited about the subcutaneous CNS platform and is considering potential partnerships for non-core CNS assets while retaining key programs [86][88] - Regarding zodasiran, there is a protocol ready for a Phase-3 study to support HoFH, with ongoing exploration of other opportunities for the asset [89] Question: Reaction to Eli Lilly discontinuing their siRNA for APOC3 and competitiveness of the Sarepta deal - Management noted that while the reasons for Lilly's decision are unclear, plozasiran is a strong drug that may have set a high bar [94] - The Sarepta deal was competitive, with discussions ongoing with other companies, but this partnership was deemed the most beneficial [95] Question: Plans for paying down debt and R&D savings - Debt will be paid down through cash inflows from the Sarepta deal and other potential partnerships, with ongoing R&D costs expected to remain stable over the next two years [100] Question: Importance of obesity programs and Phase-1 study designs - The obesity programs ARO-INHBE and ARO-ALK7 are strategically important, with plans for placebo-controlled Phase-1 studies to assess safety and early efficacy [102][103]