Financial Performance & Guidance - Increased 2025 financial guidance by >20%[52] - 2025 revenue guidance is $725-$750 million, up from prior guidance of >$600 million[56] - Anticipated operating loss for 2025 is <$375 million, improved from prior guidance of <$495 million[56] - Cash and short-term investments stand at ~$1.9 billion[56] - Q1 2025 revenue was $132 million, including $76 million in commercial revenue and $56 million in R&D revenue[52] Product Launches & Pipeline - TRYNGOLZA generated over $6 million in net product sales in its first full quarter of launch[20,54] - TRYNGOLZA prescriptions are split ~60% commercial and ~40% government coverage[23] - Donidalorsen U.S approval PDUFA date is August 21, 2025[15] - Olezarsen sHTG Phase 3 (CORE and CORE2) data is expected in Q3 2025[15] - Median fasting baseline TG levels in CORE and CORE2 studies were 836 mg/dL and 749 mg/dL, respectively[40] - 84% of switch patients surveyed preferred Donidalorsen[46]

Core Insights - Ionis Pharmaceuticals reported a first-quarter EPS loss of 93 cents, an improvement from a loss of 98 cents, and better than the consensus estimate of a $1.12 loss [1] - Revenue for the first quarter of 2025 increased by 10% to $132 million, surpassing the consensus of $125.32 million, driven by higher commercial revenue [1] Financial Guidance - The company raised its 2025 revenue guidance by over 20% to a range of $725-$750 million, compared to previous guidance of over $600 million and the consensus of $659.37 million [4] - Ionis expects a 2025 adjusted operating loss of less than $375 million, improved from prior guidance of less than $495 million, with cash and equivalents projected at approximately $1.9 billion [4] Product Performance - The drug Tryngolza (olezarsen) generated over $6 million in net product sales in its first full quarter post-approval in the U.S. [6] - Wainua (eplontersen) for polyneuropathy generated $39 million in sales, resulting in $9 million in royalty revenue for the first quarter of 2025 [6] - Spinraza (nusinersen) for spinal muscular atrophy achieved global sales of $424 million, leading to $48 million in royalty revenue in the first quarter of 2025 [6]

Financial Data and Key Metrics Changes - Ionis Pharmaceuticals increased its 2025 financial guidance across all metrics, raising revenue expectations by more than 20% due to strong Q1 results and successful licensing transactions [31][36] - The company reported $132 million in revenue for Q1 2025, a 10% year-over-year increase, with over half of the revenue coming from commercial products, which grew 28% compared to the same period last year [31][34] - Non-GAAP operating expenses increased by less than 5%, reflecting disciplined investment and driving operating leverage [34][39] Business Line Data and Key Metrics Changes - The launch of TrINGOLZA generated over $6 million in product sales in its first full quarter, exceeding expectations [32][33] - SPINRAZA royalties contributed $48 million, a 25% year-over-year increase, while Waynua royalties amounted to $9 million, with expectations for growth driven by strong US demand [33][34] - The company is on track to add initial product revenue from the upcoming launch of Donadolorsen, with an FDA action date set for August 21 [37][38] Market Data and Key Metrics Changes - The initial prescriber mix for TrINGOLZA included approximately 50% cardiologists and 25% endocrinologists, with positive feedback from physicians regarding patient outcomes [15][14] - The company is focusing on patient finding efforts and healthcare provider education to increase awareness of familial chylomicronemia syndrome (FCS) and hereditary angioedema (HAE) [18][27] Company Strategy and Development Direction - Ionis is focused on executing strategic priorities, including multiple independent launches and expanding its pipeline of transformational medicines [7][11] - The company aims to leverage its first independent launch to build momentum for future launches, including Donadolorsen and olazarcen, which are expected to address significant unmet medical needs [10][12] - Ionis is committed to driving long-term value for shareholders through sustained revenue growth and positive cash flow [41][44] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the rapidly evolving environment, including changes at the FDA and new tariff policies, which introduce uncertainty but do not currently impact the business [6][62] - The company expressed confidence in its ability to deliver transformational medicines and achieve substantial revenue growth despite external challenges [9][41] - Ionis expects to generate over $3 billion in peak annual product sales from its owned medicines and over $2 billion from partnered medicines, positioning the company for significant top-line growth [40][41] Other Important Information - Ionis completed licensing transactions for sapaglutzin and ex-US commercialization rights for olazarcen, enhancing its financial outlook and enabling a focus on independent launches [36][38] - The company is actively engaging with payers to ensure broad access for patients and is monitoring the impact of tariffs on its operations [16][62] Q&A Session Summary Question: Overlap between physician prescribers for FCS and SHTG - Management indicated significant overlap among prescribers, with cardiologists and endocrinologists treating both FCS and SHTG patients, which presents an opportunity for growth [46][49] Question: Confidence in FCS patient estimates and conversion to commercial therapy - Management expressed confidence in the estimate of over 3,000 FCS patients and noted that awareness efforts are expected to drive patient identification and conversion to commercial therapy [67][70] Question: Impact of Medicare Part D redesign on uptake - Management highlighted that the redesign is expected to increase patient starts and compliance due to reduced out-of-pocket costs, positively impacting revenue growth [95] Question: Channel stocking for TrINGOLZA - Management confirmed that TrINGOLZA sales are demand-driven with no significant channel stocking, ensuring inventory levels are managed effectively [75][78] Question: Manufacturing footprint and exposure to tariffs - Management stated that the company has a robust manufacturing supply chain in place and has not experienced any meaningful impacts from tariffs to date [80][81]

Financial Data and Key Metrics Changes - The company increased its 2025 financial guidance across all metrics, raising revenue expectations by more than 20% due to strong Q1 results and successful licensing transactions [29][34] - Revenue for Q1 2025 was $132 million, a 10% year-over-year increase, with over half coming from commercial products, which grew 28% compared to the same period last year [29][30] - Non-GAAP operating loss guidance improved by nearly 25% to less than $375 million, with an expected cash balance of $1.9 billion by year-end [37][38] Business Line Data and Key Metrics Changes - The first independent commercial launch, TrINGOLZA, generated over $6 million in product sales in its first full quarter, exceeding expectations [6][30] - SPINRAZA royalties contributed $48 million, reflecting a 25% year-over-year increase, while Waynua royalties were $9 million, with expectations for growth driven by strong U.S. demand [30][31] - The company anticipates additional product revenue from the upcoming launch of Donadolorsen for Hereditary Angioedema (HAE) [36][59] Market Data and Key Metrics Changes - The company is focused on expanding its market presence, particularly in the rare disease space, with significant unmet needs in conditions like familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG) [6][18] - The estimated patient population for FCS in the U.S. is around 3,000, while SHTG has a much larger addressable market of over 1 million patients [70][72] Company Strategy and Development Direction - The company is executing on strategic priorities, including multiple independent launches and partnerships to enhance its product pipeline and revenue potential [5][8] - The focus remains on disease education and patient identification to maximize the potential of TrINGOLZA and prepare for the launch of Donadolorsen [35][96] - The company is well-positioned to leverage its first-mover advantage in both FCS and SHTG markets, aiming for substantial revenue growth and positive cash flow [22][39] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the evolving regulatory environment and potential disruptions due to new tariff policies but expressed confidence in the company's strategic execution [4][5] - The company is optimistic about achieving sustained revenue growth, with late-stage programs expected to deliver significant peak sales potential in the multibillion-dollar range [38][41] - Management emphasized the importance of ongoing patient identification efforts and payer engagement to ensure access to treatments [15][16] Other Important Information - The company completed licensing transactions for sapaglutzin and ex-U.S. commercialization rights for olazarcen, enhancing its financial outlook [33][34] - The company is actively engaging with payers to establish broad access for TrINGOLZA and Donadolorsen, with favorable coverage dynamics reported [12][13] Q&A Session Summary Question: Overlap between physician prescribers for FCS and SHTG - Management indicated significant overlap among cardiologists and endocrinologists treating both FCS and SHTG patients, which could facilitate the transition to SHTG treatment [48][49] Question: Preparation for Donadolorsen launch - The company is actively preparing for the launch of Donadolorsen, focusing on patient advocacy and physician education to facilitate transitions from existing therapies [59][60] Question: Impact of tariffs and FDA interactions - Management reported no significant impact from tariffs or changes in FDA interactions, with all programs on track [61][63] Question: Confidence in FCS patient estimates - The company expressed confidence in the estimated 3,000 FCS patients and is focused on increasing awareness and identification of these patients [68][70] Question: Channel stocking for TrINGOLZA - Management confirmed that TrINGOLZA sales are demand-driven, with no significant channel stocking observed [75][76] Question: Manufacturing footprint and tariff exposure - The company has a robust manufacturing supply chain in place and has not experienced any meaningful impacts from tariffs to date [78][79]

Revenue Performance - Total revenue for Q1 2025 was $132 million, a 10% increase from $119 million in Q1 2024[3] - Full year 2025 revenue guidance increased from over $600 million to $725-750 million, reflecting a more than 20% increase[5] - Total revenue for the three months ended March 31, 2025, was $132 million, a 10.9% increase from $119 million in the same period of 2024[33] - Total commercial revenue increased to $76 million, up 28.8% from $59 million year-over-year[33] - TRYNGOLZA generated over $6 million in net product sales in its first full quarter following U.S. approval[9] - SPINRAZA generated global sales of $424 million, resulting in royalty revenue of $48 million in Q1 2025[9] - WAINUA achieved sales of $39 million, leading to royalty revenue of $9 million in Q1 2025[9] - Ionis' commercial revenue increased by 28% year-over-year, driven by the launch of TRYNGOLZA and higher royalty revenues[14] Operating Performance - Operating loss on a non-GAAP basis is now expected to be less than $375 million, improved from less than $495 million[5] - Total operating expenses rose to $278 million, an increase of 3.4% compared to $269 million in the same quarter of 2024[33] - The net loss for the quarter was $147 million, compared to a net loss of $143 million in the same quarter of 2024[33] - Non-GAAP net loss was $118 million, compared to $112 million in the same period of the previous year[34] Cash and Investments - Cash, cash equivalents, and short-term investments are projected to be approximately $1.9 billion by year-end 2025[5] - Cash, cash equivalents, and short-term investments decreased to $2,145 million from $2,298 million at the end of December 2024[38] - Total stockholders' equity decreased to $476 million from $588 million at the end of December 2024[38] Research and Development - Donidalorsen is on track for a PDUFA date of August 21, 2025, with positive Phase 2 data showing a 96% reduction in HAE attack rates[9] - ION582 for Angelman syndrome is expected to initiate Phase 3 development in Q2 2025[13] - Key Phase 3 clinical events are expected for Olezarsen and Zilganersen in 2025, with data anticipated to be released[39] Regulatory Approvals - The company achieved regulatory approval for TRYNGOLZA in the EU for FCS and anticipates U.S. approval for Donidalorsen for HAE in 2025[39]

Core Insights - Ionis Pharmaceuticals reported revenue of $132 million for the quarter ended March 2025, reflecting a year-over-year increase of 10.9% and surpassing the Zacks Consensus Estimate of $120.16 million by 9.86% [1] - The company's EPS was -$0.75, an improvement from -$0.98 in the same quarter last year, with an EPS surprise of 32.43% against a consensus estimate of -$1.11 [1] Revenue Breakdown - Commercial revenue from SPINRAZA royalties was $48 million, exceeding the average estimate of $43.02 million by nine analysts, marking a 26.3% increase year over year [4] - Total commercial revenue reached $76 million, surpassing the average estimate of $66.61 million based on eight analysts, representing a 28.8% year-over-year change [4] - Total research and development revenue was $56 million, compared to the average estimate of $50.44 million [4] - WAINUA royalties generated $9 million, slightly below the average estimate of $9.89 million [4] - Other commercial revenue from TEGSEDI and WAYLIVRA was $6 million, falling short of the average estimate of $6.93 million, indicating a 33.3% decline year over year [4] - Collaborative agreement revenue within research and development was $46 million, exceeding the average estimate of $37.10 million, but reflecting a 6.1% decrease year over year [4] Stock Performance - Ionis Pharmaceuticals' shares have returned +9.4% over the past month, contrasting with a -0.2% change in the Zacks S&P 500 composite [3] - The stock currently holds a Zacks Rank 3 (Hold), suggesting it may perform in line with the broader market in the near term [3]

分组1 - Ionis Pharmaceuticals reported a quarterly loss of $0.75 per share, better than the Zacks Consensus Estimate of a loss of $1.11, and improved from a loss of $0.98 per share a year ago, representing an earnings surprise of 32.43% [1] - The company posted revenues of $132 million for the quarter ended March 2025, surpassing the Zacks Consensus Estimate by 9.86%, and compared to year-ago revenues of $119 million [2] - Ionis Pharmaceuticals has surpassed consensus EPS estimates for four consecutive quarters [2] 分组2 - The stock has underperformed the market, losing about 14.7% since the beginning of the year, while the S&P 500 declined by 5.5% [3] - The current consensus EPS estimate for the upcoming quarter is -$0.85 on revenues of $146.33 million, and for the current fiscal year, it is -$3.31 on revenues of $637.57 million [7] - The Zacks Industry Rank for Medical - Drugs is in the top 20% of over 250 Zacks industries, indicating a favorable outlook for the industry [8]

Core Insights - Ionis Pharmaceuticals is a developer of antisense oligonucleotide therapeutic drugs based in Carlsbad, California, and has compelling catalysts expected in 2025/26 [2] Company Overview - The company focuses on both novice and experienced biotech investors, providing insights on catalysts, buy and sell ratings, product sales forecasts, and integrated financial statements [2] Analyst Background - The analysis is led by a biotech consultant with over 5 years of experience covering biotech, healthcare, and pharma, having prepared detailed reports on over 1,000 companies [2]

Ionis Pharmaceuticals (IONS) shares rallied 8.9% in the last trading session to close at $27.77. This move can be attributable to notable volume with a higher number of shares being traded than in a typical session. This compares to the stock's 22.8% loss over the past four weeks.The rise in share price came after President Trump announced a 90-day pause on his sweeping tariffs against non-retaliating countries.This drug discovery and development company is expected to post quarterly loss of $1.07 per share ...

Ionis Pharmaceuticals (IONS) signed a license agreement with Sweden-based Sobi. Per the terms, Sobi will acquire exclusive rights to market its wholly owned drug Tryngolza outside the United States (except Canada and China).The FDA recently approved Tryngolzafor treating familial chylomicronemia syndrome (FCS), a rare genetic disease marked by extremely elevated triglyceride levels. This nod makes the drug the first approved treatment for FCS in the country. Italso marks Ionis’ first independent commercial ...