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 ...

Core Insights - Praxis Precision Medicines, Inc. is set to present data from four late-stage programs focused on epilepsy and movement disorders at the American Academy of Neurology (AAN) 2025 Annual Meeting from April 5 to 9, 2025 in San Diego, California [1] Company Overview - Praxis is a clinical-stage biopharmaceutical company that translates genetic insights into therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance [11] - The company has developed a diversified, multimodal CNS portfolio, including multiple programs across epilepsy and movement disorders, with four clinical-stage product candidates [11] Pipeline and Clinical Programs - The company is advancing its innovative pipeline with four late-stage programs that aim to transform patient care [2] - Key clinical updates will be presented from the vormatrigine ENERGY program, along with newly initiated clinical trials enrolling patients across the U.S., Europe, and Latin America [2] - Praxis will host an In-Booth Speaker Showcase featuring experts in the field, aimed at sharing their mission to revolutionize therapies for CNS disorders [2] Presentation Details - Praxis will present multiple poster presentations at the AAN, including: - P5-011: Focus on Essential Tremor Management and Impact, featuring results from an online survey capturing healthcare provider and patient perspectives [3] - P5-012: Characteristics of Adult Essential Tremor Patients in a Decentralized US Clinical Trial [3] - P9-012: An observational study on epilepsy monitoring to better understand the patient journey [4] - P4-006: Updates from the first-in-human Phase 1 clinical trial evaluating the safety and tolerability of vormatrigine [5] Product Highlights - Relutrigine (PRAX-562) is a first-in-class small molecule targeting developmental and epileptic encephalopathies (DEEs), showing promise in preclinical studies for seizure control [6][7] - Vormatrigine (PRAX-628) is a next-generation small molecule for adult focal onset seizures and generalized epilepsy, demonstrating superior selectivity and potency in preclinical models [8] - Elsunersen (PRAX-222) is an antisense oligonucleotide targeting SCN2A gene expression, showing significant seizure reduction in clinical studies [9] - Ulixacaltamide is a selective small molecule inhibitor of T-type calcium channels, currently in development for essential tremor [10]

Core Viewpoint - Ionis Pharmaceuticals reported a narrower loss per share for Q4 2024 compared to estimates, with total revenues exceeding expectations but declining year-over-year Financial Performance - Ionis incurred a loss of 66 cents per share in Q4 2024, better than the Zacks Consensus Estimate of a loss of $1.12 per share [1] - Total revenues for Q4 were $227 million, surpassing the Zacks Consensus Estimate of $137.5 million, but down 30.2% year-over-year [2] - For the full year 2024, Ionis reported a loss of $3.04 per share, which was narrower than the Zacks Consensus Estimate of a loss of $3.47 per share, with total revenues of $705 million, beating the estimate of $618.6 million [12] Revenue Breakdown - Commercial revenues in Q4 were $86 million, up 8.9% year-over-year, exceeding the Zacks Consensus Estimate of $81 million [7] - Spinraza royalties contributed $64 million to commercial revenues, up 1.6% year-over-year, with Spinraza sales recorded at $421.4 million, up 2% year-over-year [8] - Wainua generated $10 million in royalty revenues, with sales of $42 million in Q4, reflecting an 84% sequential increase [8] - R&D revenues declined 42.7% year-over-year to $141 million, but still beat the Zacks Consensus Estimate of $57 million [10] Cost Management - Adjusted operating costs decreased by 1.3% year-over-year to $301 million in Q4, while SG&A costs rose 18.5% to support new product launches [11] Future Guidance - Ionis expects total revenues to exceed $600 million in 2025, with a shift towards increasing commercial revenues [13] - The company anticipates an adjusted operating loss of less than $495 million and expects SG&A costs to rise due to investments in marketing activities [14] Product Development Updates - Ionis' first wholly-owned drug, Tryngolza, was approved by the FDA in December 2024 for a rare disease, with a marketing application under review in the EU [6] - Olezarsen is being evaluated in late-stage studies for severe hypertriglyceridemia, with data expected in the second half of 2025 [15] - Donidalorsen is under FDA review for hereditary angioedema, with a decision expected on August 21, 2025 [16] - AstraZeneca and Ionis are developing Wainua for another form of amyloidosis, with data from a phase III study expected in the second half of 2026 [18]

Financial Data and Key Metrics Changes - Ionis Pharmaceuticals reported a non-GAAP operating loss of $345 million, which is a significant improvement compared to the 2024 guidance [48] - The company exceeded its 2024 revenue guidance by more than $130 million, earning revenues of $705 million last year [48] - SPINRAZA remained the primary source of commercial revenue, generating $216 million in royalties for the year [48] Business Line Data and Key Metrics Changes - WAYLIVRA product revenue achieved an 84% increase in the fourth quarter compared to the third quarter, with total sales of $85 million for the year [49][50] - The new-to-brand share for WAYLIVRA is approximately 40%, indicating strong demand and growth in the ATTR polyneuropathy market [75] - Tringolza, the first independent launch, is expected to gradually build momentum, with several hundred patients currently identified and in the process of being diagnosed and treated [104][105] Market Data and Key Metrics Changes - The total addressable US FCS patient population is estimated to be up to approximately 3,000 people, with a significant portion still unidentified and undiagnosed [15] - The company is actively working to increase awareness and diagnosis of FCS through healthcare provider education [16] - The SHTG patient population is significantly larger, with the company planning to scale its commercial organization to address this market [21] Company Strategy and Development Direction - Ionis Pharmaceuticals aims to deliver three additional independent launches over the next three years, including Donidalorsen and Zilganersen, which represent multibillion-dollar revenue potential [9] - The company is focused on maximizing the value of its innovative medicines while maintaining strong operating leverage [51] - Ionis is positioned to achieve substantial and sustained revenue growth and positive cash flow through its expanding pipeline and commercial efforts [59][60] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the ongoing launch of Tringolza and the potential for future launches, emphasizing the importance of patient identification and physician engagement [15][104] - The company anticipates a gradual buildup of launch momentum for Tringolza, especially in the first few quarters [52] - Management highlighted the potential for olezarsen to become the standard of care for both FCS and SHTG, based on positive clinical data [38] Other Important Information - The company has a rich pipeline with multiple late-stage data readouts and regulatory actions expected this year and next [32] - Ionis Pharmaceuticals has retained global rights for its alpha-synuclein and LRRK2 programs, indicating a strong commitment to advancing these therapies [94] Q&A Session Summary Question: Insights on olezarsen and SHTG - Management clarified that the ESSENCE study is a safety study and not directly targeting the commercial patient population, but it will provide important safety read-throughs for SHTG [68][70] Question: ATTR polyneuropathy market performance - Management reported that WAYLIVRA has captured about 40% of new patient starts, with strong demand and growth observed [75][76] Question: Design for SHTG phase three study - Management explained that the CORE studies are well-powered for triglyceride reductions, and while acute pancreatitis events are not the primary focus, they expect to see meaningful signals based on previous data [81][82] Question: Growth drivers for WAYLIVRA - Management noted that the majority of growth is driven by new patient starts, with a strong emphasis on self-administration and affordability contributing to the uptake [91][92] Question: Tringolza launch metrics - Management indicated that several hundred patients have been identified and are in the process of being diagnosed, with a focus on expediting the prescription process [104][105]

Financial Performance - Total revenue for 2024 was $705.1 million, a decrease of 10.5% from $787.6 million in 2023[376] - The company recorded a net loss of $453.9 million in 2024, compared to a net loss of $366.3 million in 2023, indicating an increase of about 23.9%[412] - The income tax benefit for 2024 was $6.2 million, a significant improvement from an income tax expense of $32.3 million in 2023[409] - Investment income increased from $89.0 million in 2023 to $107.0 million in 2024, a growth of about 20.2% driven by higher interest rates[403] - Interest expense increased from $12.7 million in 2023 to $17.0 million in 2024, representing a rise of approximately 33.9% due to new convertible notes[404] Operating Expenses - Total operating expenses increased to $1,180.2 million in 2024 from $1,141.4 million in 2023, resulting in a loss from operations of $475.1 million[376] - Non-cash compensation expense related to equity awards increased to $130.2 million in 2024 from $105.7 million in 2023, driven by higher headcount and stock prices[384] - SG&A expenses rose from $232.6 million in 2023 to $267.5 million in 2024, marking an increase of approximately 15% due to new product launches[402] - Medical affairs expenses increased to $31.9 million in 2024 from $22.9 million in 2023, reflecting advancements in the late-stage pipeline[396] - Total R&D support expenses increased from $96.0 million in 2023 to $101.2 million in 2024, reflecting a rise of about 6.3%[400] Revenue Sources - SPINRAZA royalties decreased to $216.1 million in 2024 from $240.4 million in 2023, impacted by a non-recurring annual order from a single country[378] - WAINUA joint development revenue decreased to $79.4 million in 2024 from $126.4 million in 2023, primarily due to winding down development activities[382] - The company launched WAINUA in the U.S. in late January 2024 and TRYNGOLZA in late December 2024, contributing to new commercial revenue sources[378] - R&D revenue is recognized from various collaboration agreements, including upfront payments, milestone payments, and license fees, requiring significant estimates and judgments[432] - Revenue from R&D services is recognized based on an input method estimating total costs to complete performance obligations, which may require adjustments if estimates change[433] Cash and Investments - Cash, cash equivalents, and short-term investments were $2,297.7 million as of December 31, 2024, down from $2,331.2 million in 2023[376] - The company raised approximately $489 million from a public stock offering in September 2024, contributing to increased cash available for investments[403] Development Costs - Total drug development expenses were $568.5 million in 2024, compared to $564.8 million in 2023, indicating stable development costs[392] - Manufacturing and development chemistry expenses decreased from $74.1 million in 2023 to $67.1 million in 2024, a reduction of approximately 9.4%[398] - As of December 31, 2024, a hypothetical 10% increase in liability for preclinical and clinical development costs would increase loss before income tax benefit by approximately $7.7 million[436] - The company maintains an accrual for preclinical and clinical development costs, estimating liabilities based on contracted fees and expected expenses[435] Future Estimates and Risks - Factors affecting future royalty payments include regulatory approvals and competition, which could lead to significant changes in revenue estimates[439] - The allocation of transaction price to performance obligations involves estimating relative stand-alone selling prices, which may require significant judgments[431] - The company assesses the propriety of revenue recognition and deferred revenue, along with estimating future royalty payments under its royalty purchase agreement[430] - The company recognizes revenue related to milestone payments based on the probability of achievement and the estimated period of performance[437] - In 2023, the company entered into a royalty purchase agreement with Royalty Pharma to monetize future royalties from SPINRAZA and pelacarsen, with estimates subject to change based on sales performance[438] Contractual Obligations - Total contractual obligations as of December 31, 2024, amounted to $1,513.4 million, with $31.6 million due within one year[417] - The company does not utilize derivative financial instruments to manage exposure to interest rate changes, believing it is not subject to material risks from such changes as of December 31, 2024[440] Historical Performance - The company has generated approximately $7.9 billion in revenue since its inception through December 31, 2024[413]

Group 1 - Brendan completed a Ph.D. at Stanford University in organic synthesis in 2009 [1] - He worked for Merck from 2009 to 2013 and has experience in biotech startups such as Theravance and Aspira [1] - Brendan is a co-founder of 1200 Pharma, which spun out of Caltech and received significant investment in the 8 figures [1] Group 2 - Brendan remains an avid investor focused on market trends, particularly in biotechnology stocks [1]

Core Insights - Ionis Pharmaceuticals reported better-than-expected revenue and a smaller-than-expected loss for Q4 2024, indicating strong operational performance [2][6]. Financial Performance - Q4 2024 revenue reached $227 million, significantly exceeding analysts' expectations of $135 million, marking a 68.1% increase [3][6]. - The earnings loss was $0.66 per share, which was better than the anticipated loss of $1.11 per share, reflecting effective operational execution [3][7]. - Total revenue decreased by 30.2% compared to Q4 2023, where it was $325 million [3]. Operational Highlights - Ionis' non-GAAP operating expenses were $301 million, a slight decrease of 1.3% from the previous year [3][7]. - The successful launch of Wainua contributed to the positive revenue surprise, while royalty revenue from Spinraza remained a significant revenue source [6][7]. Strategic Focus - The company is expanding its drug offerings and enhancing the reach of its marketed medicines, with a focus on collaborations to mitigate development risks [5][8]. - Ionis has formed strategic alliances with Theratechnologies and Otsuka, which have provided financial support through milestones and royalties [8]. Future Outlook - Ionis anticipates revenue exceeding $600 million in 2025, with a planned operating loss of less than $495 million [9]. - The company has cash reserves of $2.3 billion, which will support continued investments in product launches and R&D [9]. - Three independent product launches are planned for the year, including donidalorsen for hereditary angioedema [10].

YE:2024 Business Update and Financial Results February 19, 2025 Nasdaq: IONS 1 On Today's Earnings Call Beth Hougen Chief Financial Officer 2 Brett Monia, Ph.D. Chief Executive Officer Kyle Jenne Chief Global Product Strategy Officer Richard Geary, Ph.D. Chief Development Officer Eric Swayze, Ph.D. Executive Vice President, Research Forward-Looking Statements This presentation includes forward-looking statements regarding our business, financial guidance and the therapeutic and commercial potential of our c ...

On Wednesday, Ionis Pharmaceuticals Inc IONS reported a fourth-quarter EPS loss of $0.66, up from a loss of $0.06, beating the consensus of a $1.10 loss.The company reported an adjusted net loss of $68 million versus a net income of $17 million.Ionis reported quarterly sales of $227 million, down from $325 million a year ago, beating the consensus of $137.6 million.Ionis says 2024 revenue of $705 million exceeded guidance as the company continued to generate revenue from diverse sources. The company added n ...