Ionis Pharmaceuticals - IONS STOCK NEWS

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.

Ionis Pharmaceuticals (Nasdaq: IONS) has announced its participation in two upcoming investor conferences. The company's management will engage in fireside chats at the 2024 Wells Fargo Healthcare Conference on September 4, 2024, and the Morgan Stanley 22nd Annual Global Healthcare Conference on September 5, 2024.

Investors and interested parties can access live webcasts of these presentations through the Investors & Media section of the Ionis website at https://ir.ionis.com/events-and-presentations/upcoming-events. Replays of the presentations will be available on the website within 48 hours and will be archived for a time, providing an opportunity for those unable to attend the live events to stay informed about the company's updates and strategies.

Ionis Pharmaceuticals reported Q2 2024 financial results, highlighting progress across its pipeline. Key points include:

- WAINUA launch for ATTRv-PN progressing well, with $16M in Q2 sales

- Olezarsen NDA accepted for Priority Review for FCS, with PDUFA date of Dec 19, 2024

- Positive Phase 3 data for donidalorsen in HAE; preparing regulatory submissions

- Q2 revenue increased 20% YoY to $225M, driven by R&D revenue growth

- Operating expenses rose due to investments in late-stage development and commercialization

- Cash position of $2.1B as of June 30, 2024

Ionis reaffirmed 2024 financial guidance and remains focused on advancing its pipeline of RNA-targeted therapies.

Alcyone Therapeutics has received FDA approval to continue enrollment in the PIERRE pivotal IDE clinical study for the ThecaFlex DRx™ System. This implantable device is being investigated for routine subcutaneous administration of nusinersen to treat spinal muscular atrophy (SMA). The first stage, involving 10 SMA patients, has been completed with no device-related adverse events. The FDA has approved enrollment of an additional 80 SMA patients across 30 centers in the U.S. and Europe.

Key observations include:

• Implantation duration: 1-2 hours
• Hospital discharge: Within 24 hours
• Infusion procedure: Less than 30 minutes
• Actual nusinersen infusion: Less than 10 minutes

The ThecaFlex system aims to provide a less invasive, more accessible alternative to repeat lumbar puncture for SMA patients receiving SPINRAZA® (nusinersen) therapy.

Ionis Pharmaceuticals (Nasdaq: IONS) announced positive results from the Phase 1/2 HALOS study of ION582 in people with Angelman syndrome (AS). The study showed consistent clinical improvement in communication, cognition, and motor function. Key findings include:

- 97% of patients in medium and high dose groups showed improvement in overall AS symptoms

- Improvements exceeded those observed in natural history studies

- ION582 demonstrated favorable safety and tolerability at all dose levels

Ionis plans to initiate Phase 3 development in H1 2025. The company views ION582 as a potential cornerstone of its next wave of transformational medicines for neurological conditions.