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Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) is a leading biotechnology company headquartered in Carlsbad, California. Founded in 1989, Ionis has been pioneering the discovery and development of antisense drugs, with an impressive portfolio of over 1300 patents. Ionis focuses on creating innovative RNA-targeted therapies for complex diseases, particularly in the areas of cardiovascular, metabolic, neurological, and rare genetic disorders.
Ionis has successfully brought several groundbreaking medicines to market. Notable achievements include the development of Spinraza, in collaboration with Biogen, which was launched in 2016 to treat spinal muscular atrophy, a rare neuromuscular disorder. In 2023, Biogen also launched Qalsody for ALS, further extending the impact of Ionis' antisense technology.
Through its cardiovascular-focused subsidiary, Akcea Therapeutics, Ionis has introduced drugs like Tegsedi for ATTR amyloidosis and Waylivra in Europe for cardiology indications. The company continues to advance its pipeline with promising candidates such as olezarsen, an investigational RNA-targeted therapy for familial chylomicronemia syndrome (FCS). Ionis' ongoing Phase 3 trials for olezarsen have shown promising results, with significant reductions in triglyceride levels and improvements in related health outcomes.
Ionis' commitment to innovation and patient care is also evident in its robust financial health and strategic partnerships. With a deep understanding of disease biology and cutting-edge technology, Ionis continues to drive advancements in RNA therapies and gene editing, aiming to deliver life-changing medicines for patients around the world.
Recently, Ionis announced positive clinical trial results for several of its investigational drugs and plans to pursue regulatory approvals for these therapies. The company's pipeline is well-positioned to address unmet medical needs in various therapeutic areas, promising a steady flow of new treatments in the coming years.
For more detailed information about Ionis Pharmaceuticals, its products, and career opportunities, visit their official website at www.ionispharma.com.
Ionis Pharmaceuticals (NASDAQ: IONS) announced new data presentations for donidalorsen, their investigational RNA-targeted medicine for hereditary angioedema (HAE), at the 2025 AAAAI/WAO Joint Congress. The drug's New Drug Application is under FDA review with a PDUFA date of August 21, 2025.
The presentations will showcase additional data from the Phase 3 OASIS and OASISplus studies, along with three-year data from the Phase 2 open-label extension study. The company will present 11 posters covering various aspects including efficacy in adolescent patients, disease control after switching from prior treatments, quality-of-life improvements, and patient-reported outcomes.
The data demonstrates sustained HAE attack rate reduction and improved quality of life in patients previously on other prophylactic treatments, with the convenience of monthly or bi-monthly self-administration via autoinjector. If approved, donidalorsen would be Ionis' second independent commercial launch.
Ionis Pharmaceuticals (NASDAQ: IONS) reported its Q4 and full year 2024 financial results, marking significant milestones with multiple product launches. The company's total revenue for 2024 was $705 million, down from $788 million in 2023, with operating expenses of $1,180 million.
Key highlights include the launch of TRYNGOLZA for familial chylomicronemia syndrome and WAINUA for hereditary transthyretin-mediated amyloidosis, which generated $85 million in sales with $20 million in royalty revenue. SPINRAZA generated global sales of $1.6 billion, resulting in $216 million in royalty revenue.
The company maintains a strong financial position with $2.3 billion in cash and investments as of December 31, 2024. For 2025, Ionis projects revenue exceeding $600 million and expects to maintain cash reserves of approximately $1.7 billion.
Ionis Pharmaceuticals (Nasdaq: IONS) has announced a webcast scheduled for Wednesday, February 19th at 11:30 a.m. Eastern Time. During this event, the company will discuss its fourth quarter and full year 2024 financial results. The webcast will be accessible through the company's investor relations website, and a replay will be available for a time afterward.
Ionis Pharmaceuticals (IONS) enters 2025 with significant momentum following the U.S. approval and launch of TRYNGOLZA™, the first-ever therapy for familial chylomicronemia syndrome (FCS). The company anticipates four independent product launches over the next three years, plus four additional launches from partnered programs. Key 2025 milestones include potential European approval for TRYNGOLZA, expected FDA decision on donidalorsen for hereditary angioedema by August 21, and continued progress with WAINUA™ in collaboration with AstraZeneca.
The company plans to advance seven clinical-stage therapies for neurological diseases, including Phase 3 trials for ION582 in Angelman syndrome and data readouts for zilganersen in Alexander disease. Ionis expects increasing product and royalty revenue to drive positive cash flow, marking its transition to a fully integrated commercial-stage biotechnology company.
Ionis Pharmaceuticals (Nasdaq: IONS) has announced that CEO Brett P. Monia, Ph.D., will deliver a company overview presentation at the 43rd Annual J.P. Morgan Healthcare Conference. The presentation is scheduled for Wednesday, January 15, 2025, at 10:30 a.m. PT.
Investors and interested parties can access a live webcast of the presentation through the Investors & Media section of the Ionis website. The presentation recording will be made available on the company's website within 48 hours and will remain accessible for a time.
Ionis Pharmaceuticals (NASDAQ: IONS) announced FDA approval of TRYNGOLZA™ (olezarsen), the first-ever treatment for adults with familial chylomicronemia syndrome (FCS). The drug, administered monthly via auto-injector, demonstrated a 42.5% placebo-adjusted mean reduction in triglyceride levels at six months and 57% at 12 months in Phase 3 Balance trial. TRYNGOLZA significantly reduced acute pancreatitis events, with only one episode in the treatment group versus 11 episodes in the placebo group.
The treatment showed a favorable safety profile and will be available in the U.S. before year end. This approval marks Ionis's evolution into a commercial-stage biotechnology company, with TRYNGOLZA being the first of four planned launches over the next three years.
Ionis Pharmaceuticals (NASDAQ: IONS) has announced its participation in six major healthcare investor conferences throughout November and December 2024. The company's management will engage in fireside chats at events including Guggenheim's Inaugural Healthcare Conference, UBS Global Healthcare Conference, Stifel Healthcare Conference, Jefferies London Healthcare Conference, Citi's Global Healthcare Conference, and Piper Sandler Annual Healthcare Conference. Live webcasts will be available on the Ionis website's Investors & Media section, with replays accessible within 48 hours and archived for a time.
Ionis Pharmaceuticals announced the design of its pivotal Phase 3 REVEAL trial for ION582, following successful alignment with the FDA. The trial, targeting Angelman syndrome (AS), will enroll approximately 200 children and adults with maternal UBE3A gene deletion or mutation. The study will use Bayley-4 expressive communication as the primary endpoint, with patients randomized 2:1 to active therapy or placebo. The Phase 2 HALOS study showed promising results, with 97% of participants in medium and high dose groups showing improvement in overall AS symptoms. The Phase 3 trial is planned to begin in H1 2025.
Ionis Pharmaceuticals reported Q3 2024 financial results, highlighting the successful launch of WAINUA in the U.S. and its approval in the UK. Total revenue was $134M for Q3, down 7% year-over-year. The company is preparing for two key product launches: olezarsen for FCS with FDA decision date December 19, 2024, and donidalorsen for HAE with PDUFA date August 21, 2025. SPINRAZA generated global sales of $381M in Q3. The company reaffirmed its 2024 P&L guidance and increased cash guidance to $2.2B following an equity offering that raised $500M.
Ionis Pharmaceuticals (IONS) announced FDA acceptance of its New Drug Application for donidalorsen, a first-in-class RNA-targeted medicine for hereditary angioedema (HAE) prevention. The FDA set a PDUFA date of August 21, 2025. The application is supported by positive results from Phase 3 OASIS-HAE and OASISplus studies, showing a 96% mean reduction in HAE attack rates maintained up to three years. The drug was well-tolerated with no serious treatment-related adverse events. If approved, donidalorsen will be Ionis' second independent commercial launch. Otsuka holds exclusive commercialization rights in Europe and Asia Pacific.
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