– Accelerating value creation with numerous commercial, regulatory and pipeline milestones –– Ionis outlines clear path to sustained positive cash flow –CARLSBAD, Calif., Jan. 13, 2025 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced highlights from the Company's 2024 achievements and previewed important milestones expected in 2025. Ionis will provide a business update at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15 at 10:30am PT; the presentation is a ...

Portfolio Updates and Anticipated Milestones - Praxis has three blockbuster programs in late-stage development, with potential for four product launches between 2026 and 2028 [1] - The Essential3 program for ulixacaltamide in essential tremor (ET) includes two Phase 3 studies, with over 100,000 patients showing interest in participating since recruitment began in November 2023 [2] - Relutrigine is being evaluated in the EMBOLD study for developmental and epileptic encephalopathies (DEEs), with plans for the EMERALD study in a broader patient population [3] - Praxis shared an update at the 2024 AES Annual Meeting for an emergency use patient who achieved extended benefit after receiving relutrigine [4] - Ulixacaltamide is a highly selective small molecule inhibitor of T-type calcium channels, currently in late-stage development for essential tremor [5] - Praxis has completed discussions with global regulatory agencies to harmonize a registrational study design for elsunersen, with trial design and timelines expected in Q1 2025 [6] - UCB exercised its option to in-license global development and commercialization rights for a KCNT1 small molecule candidate, with Praxis eligible for up to $100 million in milestone payments and tiered royalties [6] - Praxis is on track to nominate development candidates for its early-stage ASO therapeutic initiatives in 2025, including PRAX-080, PRAX-090, and PRAX-100 [6] Clinical Trial Progress and Results - Vormatrigine demonstrated strong competitive differentiation in Phase 1 studies, with full results to be shared at an upcoming medical conference [10] - The EMPOWER observational study has enrolled over 2,000 patients, with early results shared at the 2024 AES Annual Meeting [10] - The RADIANT Phase 2 study for focal onset seizures (FOS) and generalized epilepsy is enrolling patients, with topline results expected in H1 2025 [10] - The POWER1 Phase 2/3 registrational study for treatment-resistant FOS is progressing, with topline results anticipated in H2 2025 [10] - Relutrigine showed a 46% placebo-adjusted reduction in monthly motor seizures in the EMBOLD study, with 77% reduction in motor seizures for patients continuing into the open-label extension [17] - Over 30% of patients achieved seizure freedom status while on relutrigine, with a median of 46 days of seizure freedom compared to 3 days at baseline [17] Regulatory and Commercial Developments - Praxis anticipates filing the NDA for ulixacaltamide in 2025, following positive results from the Essential3 program [9] - The company plans to re-initiate a study of ulixacaltamide in Parkinson's disease, addressing unmet needs for non-dopaminergic treatment options [9] - Praxis received a third rare pediatric drug designation (RPDD) for relutrigine in Dravet Syndrome, in addition to previous designations for SCN2A and SCN8A DEEs [17] - The company expects to initiate the pivotal trial for elsunersen in H1 2025, following regulatory feedback [8] Financial and Strategic Outlook - Praxis has a cash and investment position of approximately $470 million at the end of 2024, supporting a runway into 2028 [15] - The company is well-positioned for a readout-rich 2025, with results anticipated for ulixacaltamide, vormatrigine, and relutrigine, as well as regulatory submissions [8] - Praxis plans to provide a thorough update across its portfolio at the Investor R&D Day in Q2 2025 [8]

CARLSBAD, Calif., Jan. 8, 2025 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that Brett P. Monia, Ph.D., chief executive officer, will present a company overview at the 43rd Annual J.P. Morgan Healthcare Conference at 10:30 a.m. PT on Wednesday, January 15, 2025. A live webcast of the presentation can be accessed on the Investors & Media section of the Ionis website at https://ir.ionis.com/events. A replay will be available on the Ionis website within 48 hours and will be archiv ...

Shares of Ionis Pharmaceuticals (IONS) rose nearly 5% in after-market trading on Thursday after the FDA approved its RNA-targeted therapy, olezarsen, in adults with familial chylomicronemia syndrome (FCS). The drug will be marketed under the trade name Tryngolza.The FDA’s nod to Tryngolza makes it the first approved treatment for FCS in the country. The drug’s commercial launch is part of management's broad strategy to deliver a steady flow of wholly-owned medicines to patients. Besides being Ionis’ first w ...

TRYNGOLZA shown to significantly reduce triglycerides and substantially reduce acute pancreatitis events in adults with FCS; a rare, highly debilitating and life-threatening disease Indicated for adults with FCS regardless of genetically or clinically confirmed diagnosis  TRYNGOLZA is the first of four independent launches planned over the next three years, pending approvals Ionis to host webcast today at 6:45pm ET CARLSBAD, Calif., Dec. 19, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) ...

Ionis Pharmaceuticals (IONS) closed the last trading session at $38.26, gaining 1.3% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $59.65 indicates a 55.9% upside potential.The average comprises 20 short-term price targets ranging from a low of $37 to a high of $77, with a standard deviation of $10.76. While the lowest estimate indicates a decline of 3.3% from the current price l ...

Ionis Pharmaceuticals (IONS) could be a solid addition to your portfolio given its recent upgrade to a Zacks Rank #2 (Buy). This rating change essentially reflects an upward trend in earnings estimates -- one of the most powerful forces impacting stock prices.The Zacks rating relies solely on a company's changing earnings picture. It tracks EPS estimates for the current and following years from the sell-side analysts covering the stock through a consensus measure -- the Zacks Consensus Estimate.Since a chan ...

Ionis Pharmaceuticals (IONS) has been beaten down lately with too much selling pressure. While the stock has lost 13.5% over the past four weeks, there is light at the end of the tunnel as it is now in oversold territory and Wall Street analysts expect the company to report better earnings than they predicted earlier.How to Determine if a Stock is OversoldWe use Relative Strength Index (RSI), one of the most commonly used technical indicators, for spotting whether a stock is oversold. This is a momentum osc ...

Ionis Pharmaceuticals (IONS) incurred a loss of 95 cents per share for third-quarter 2024, which was narrower than the Zacks Consensus Estimate of a loss of $1.16 per share.Earnings include compensation expenses related to equity awards. Excluding these special items, adjusted loss per share was 72 cents compared with a loss of 85 cents per share in the year-ago quarter.Total revenues were $134 million in the third quarter, beating the Zacks Consensus Estimate of $129.0 million. Revenues declined 7% year ov ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Title of each class Trading symbol Name of each exchange on which registered Common Stock, $.001 Par Value "IONS" The Nasdaq Stock Market LLC Form 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Quarterly Period Ended September 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____ to _____ Com ...