IONIS PHARMACEUTICALS INC (IONS)·Q1 2025 Earnings Summary

Executive Summary

• Q1 2025 revenue was $132.0M, up 10% YoY; GAAP EPS was $(0.93). Ionis beat Wall Street consensus on both revenue ($131.6M actual vs $122.5M*) and EPS ($(0.93) actual vs $(1.03)), driven by first-quarter TRYNGOLZA product revenue and stronger partner royalties .
• Management raised FY2025 guidance by more than 20%: revenue to $725–$750M (from >$600M), narrowed non-GAAP operating loss to <$(375)M (from <$(495)M), and year-end cash to ~$1.9B (from ~$1.7B), enabled by licensing (sapablursen $280M upfront) and ex‑U.S. olezarsen rights to Sobi .
• Commercial execution: TRYNGOLZA net product sales >$6M in first full quarter; SPINRAZA and WAINUA royalties strengthened. CFO emphasized path to sustained positive cash flow with growing product and royalty streams .
• Near-term catalysts: donidalorsen PDUFA Aug 21, 2025; ESSENCE (sHTG) data in Q2 and CORE/CORE2 topline in Q3; EU decision for TRYNGOLZA in H2 2025. These events, plus raised guidance, are primary stock reaction drivers .

What Went Well and What Went Wrong

What Went Well

• First independent launch underway with TRYNGOLZA delivering >$6M net product sales; early uptake from converted trial/EAP patients and newly identified FCS patients; favorable coverage (≈60% commercial/≈40% government) and ~90% zero out-of-pocket via Ionis Every Step support .
• Royalty strength: SPINRAZA global sales of $424M (Q1) drove $48M royalties; WAINUA sales of $39M delivered $9M royalties; commercial revenue +28% YoY .
• Management raised FY2025 guidance substantially. CFO: “We are increasing our 2025 financial guidance including raising revenue guidance by more than 20 percent… and now expect to end the year with approximately $1.9 billion in cash” .

What Went Wrong

• Continued GAAP operating losses: Q1 loss from operations $(146)M and GAAP net loss $(147)M; EBIT margin remained deeply negative given heavy launch and pipeline investments .
• SG&A increased to $76M (from $53M YoY) driven by TRYNGOLZA launch and donidalorsen preparation; R&D decreased to $201M as late-stage studies ended, but overall operating expenses ticked up .
• Medicare Part D redesign impacted some WAINUA revenue timing in Q1; however management expects net-positive impact on uptake/compliance going forward .

Financial Results

• YoY growth (Revenue): +10.9% vs Q1 2024 ($119.0M) .
• QoQ change (Revenue): down from Q4 2024 due to seasonality/mix shift post Q4 licensing/milestone inflows .

Segment revenue breakdown (Q1 2025 vs Q1 2024):

Selected KPIs:

Guidance Changes

Drivers: sapablursen global license ($280M upfront) and ex‑U.S. olezarsen commercialization agreement with Sobi (upfront, milestones, tiered royalties up to mid‑20%) .

Earnings Call Themes & Trends

Management Commentary

• CEO: “In its first full quarter on the market, TRYNGOLZA… exceeded expectations… Right behind TRYNGOLZA is our second independent launch, donidalorsen… approaching” .
• CFO: “We are increasing our 2025 financial guidance… raising revenue guidance by more than 20 percent… and now expect to end the year with approximately $1.9 billion in cash” .
• CEO on sHTG: “AP is a secondary endpoint and not powered… we are combining CORE and CORE2 in our secondary analysis of AP events… focused on demonstrating substantial reductions of triglycerides” .

Q&A Highlights

• TRYNGOLZA uptake and inventory: Sales directly reflect demand; minimal channel stocking; conversion of EAP/OLE patients and pent-up diagnosed patients; focus shifting to new patient identification .
• Donidalorsen launch: Switch market dynamics (~20% annual prophylactic switches); efficacy/tolerability/convenience profile; targeted sales force; hiring/training underway .
• Olezarsen sHTG AP endpoint: AP is secondary, not powered; combined CORE/CORE2 analysis at 12 months; topline focus on triglyceride reduction and safety; timing narrowed to Q3 .
• WAINUA and Part D: Redesign timing impact noted; expected benefits include new starts and improved compliance due to affordability .
• Pricing dynamics in TTR cardiomyopathy: Competitor approach observed; WAINUA pricing strategy will be AstraZeneca’s call; market evolution to guide decisions .

Estimates Context

How results compared to Wall Street consensus (S&P Global):

• Q1 2025: Revenue beat by ~$9.1M; EPS beat by ~$0.10. Q4 2024 was a significant revenue beat on licensing/milestone inflows. Q3 2024 modest beats. Values retrieved from S&P Global.*
• Target price consensus mean: ~$83.82; # of estimates: 24.*
• Consensus recommendation text not provided in source data.*

Key Takeaways for Investors

• Ionis delivered a clean Q1 beat on revenue and EPS alongside a >20% guidance raise—license monetization (sapablursen) plus ex‑U.S. olezarsen partnership structurally de-risk FY2025 top line .*
• Commercial flywheel is turning: TRYNGOLZA demand-led sales, strong payer dynamics, and rising partner royalties (SPINRAZA, WAINUA) support sequential growth through 2025 .
• Near-term binary events: ESSENCE safety (Q2) and CORE/CORE2 pivotal (Q3) data for sHTG could unlock broader olezarsen opportunity; donidalorsen PDUFA in August is a second 2025 launch catalyst .
• Operating leverage improving: Non-GAAP operating loss guidance tightened; CFO articulated path to sustained positive cash flow with increasing product revenue and royalties .
• Risk watch: AP data are secondary and not powered; pricing strategy in broader sHTG will be crucial; Medicare Part D redesign introduces timing effects but appears net supportive for WAINUA .
• Strategic positioning: Ex‑U.S. olezarsen commercialization by Sobi leverages existing rare disease channels, preserving U.S. economics while broadening global reach .
• Portfolio optionality: Continued licensing/partner launches (e.g., ION582 Phase 3 start) augment revenue streams and capital flexibility into 2026+ .

Citations: Earnings press release and 8-K ; Earnings call transcript ; Prior quarters . Licensing/partner releases . Estimates from S&P Global (*).