Ionis Pharmaceuticals, Inc. - IONS STOCK NEWS

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.

Ionis Pharmaceuticals (Nasdaq: IONS) has announced a webcast to discuss its third quarter 2024 financial results. The event is scheduled for Wednesday, November 6th at 11:30 a.m. Eastern Time. Investors and interested parties can access the webcast through the company's investor relations website, with a replay available for a time after the event.

Ionis Pharmaceuticals announced that WAINZUA (eplontersen), developed with AstraZeneca, has been recommended for approval by the CHMP in the EU for treating hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or 2 polyneuropathy (ATTRv-PN). If approved, WAINZUA will be the only EU-approved medicine for ATTRv-PN that can be self-administered monthly via an auto-injector.

The recommendation is based on the positive NEURO-TTRansform Phase 3 trial results, showing consistent and sustained benefits in serum TTR concentration, neuropathy impairment, and quality of life through 66 weeks. WAINZUA demonstrated a favorable safety and tolerability profile throughout the trial.

WAINZUA was approved in the U.S. in December 2023 and is gaining approvals worldwide. Ionis and AstraZeneca are commercializing it in the U.S. and seeking approvals globally. The drug is also being evaluated for ATTR-CM in the CARDIO-TTRansform Phase 3 study.

Ionis Pharmaceuticals (Nasdaq: IONS) will present new data on donidalorsen, its investigational RNA-targeted prophylactic medicine for hereditary angioedema (HAE), at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting. The presentations include:

1. Three-year data from the Phase 2 open-label extension study
2. Additional results from the pivotal Phase 3 OASIS and OASISplus studies
3. Impact on patient-reported outcomes
4. Safety, efficacy, and patient preference after switching to donidalorsen
5. Exposure-response analysis
6. Drug patterns, clinical outcomes, and economic costs among HAE patients

The new data demonstrate that donidalorsen significantly reduced HAE attacks, providing high levels of disease control and improved quality of life for up to three years with monthly or every two-month dosing.

Ionis Pharmaceuticals (Nasdaq: IONS) announced that the U.S. FDA has granted Fast Track designation to zilganersen for treating Alexander disease (AxD), an ultra-rare, progressive, and fatal neurological disorder. Zilganersen is the first investigational medicine in clinical development for adults and children with AxD. This designation aims to expedite the development and review of medicines addressing serious conditions with unmet medical needs.

The pivotal Phase 1-3 study of zilganersen has completed enrollment across 13 sites in eight countries. Topline data from this study is expected in the second half of 2025. Ionis designed zilganersen to target the underlying cause of AxD and potentially improve patient functioning. The company looks forward to working closely with the FDA to potentially bring forward the first approved AxD treatment.

Ionis Pharmaceuticals (Nasdaq: IONS) has announced the pricing of a public offering of 11,500,000 shares of common stock at $43.50 per share, expecting to raise approximately $500.3 million in gross proceeds. The offering, set to close on September 11, 2024, includes a 30-day option for underwriters to purchase up to an additional 1,725,000 shares. Net proceeds will fund independent commercial launches, late-stage clinical programs, earlier pipeline programs, research and development activities, working capital, and general corporate purposes. Morgan Stanley and Goldman Sachs & Co. are acting as joint book-running managers for the offering.

Ionis Pharmaceuticals (Nasdaq: IONS) has announced a proposed underwritten public offering of $500.0 million of its common stock, with an option for underwriters to purchase an additional $75.0 million of shares. The company plans to use the net proceeds to fund independent commercial launches, late-stage clinical programs, earlier pipeline programs, and research and development activities, as well as for working capital and general corporate purposes. Morgan Stanley and Goldman Sachs & Co. are acting as joint book-running managers for the offering. The offering is subject to market conditions, and its completion is not guaranteed.