Ionis Pharmaceuticals, Inc. - IONS STOCK NEWS

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.

Ionis Pharmaceuticals (Nasdaq: IONS) has announced a live webcast scheduled for Thursday, August 1st at 11:30 a.m. Eastern Time to discuss its second quarter 2024 financial results. Interested parties can access the webcast through the company's investor relations website at https://ir.ionis.com/events-and-presentations/upcoming-events. A replay of the webcast will be available for a time at the same address. This event provides an opportunity for investors and analysts to gain insights into Ionis' financial performance and potentially its future outlook.

Ionis Pharmaceuticals (Nasdaq: IONS) has completed enrollment in the pivotal trial of zilganersen (ION373), an investigational RNA-targeted medicine for Alexander disease (AxD). This rare, progressive neurological disorder affects an estimated one in one million people in the U.S. The trial's primary endpoint is percent change from baseline in gait speed, assessed by the 10-Meter Walk Test. Topline data are expected in the second half of 2025.

Zilganersen aims to address the underlying cause of AxD by targeting excess GFAP accumulation resulting from genetic variants in the GFAP gene. This approach could potentially slow or stabilize disease progression in AxD patients, who currently lack treatments that address the root cause of their condition.

Ionis Pharmaceuticals (NASDAQ: IONS) has announced a live webcast scheduled for Monday, July 22, at 8:00 a.m. Eastern Time. This webcast will discuss the results from the Phase 1/2a HALOS study of ION582, a treatment for Angelman syndrome. The findings will also be presented at the 2024 Angelman Syndrome Foundation (ASF) Family Conference and Research Symposium on Wednesday, July 24, in Sandusky, Ohio. Interested parties can access the webcast via the provided link, with a replay available for a time.

Ionis Pharmaceuticals announced the FDA's acceptance of its New Drug Application (NDA) for olezarsen, an investigational RNA-targeted medicine for treating familial chylomicronemia syndrome (FCS). The FDA has granted Priority Review with a PDUFA date set for December 19, 2024. Olezarsen has also received Fast Track, Orphan Drug, and Breakthrough Therapy designations.

Additionally, Ionis completed Phase 3 enrollment for three studies (CORE, CORE2, and ESSENCE) evaluating olezarsen for severe hypertriglyceridemia (sHTG), with results anticipated in the second half of 2025. These studies involve over 2,400 patients and aim to address the life-threatening complications associated with sHTG.

The NDA is supported by positive Phase 3 study results presented at the 2024 ACC Annual Meeting and published in the New England Journal of Medicine.

Ionis Pharmaceuticals has entered an expanded licensing agreement with Otsuka Pharmaceutical for the investigational RNA-targeted medicine donidalorsen, aimed at treating hereditary angioedema (HAE).

Under the agreement, Otsuka gains exclusive rights to commercialize donidalorsen in the Asia-Pacific region while also handling regulatory filings and localized development. Ionis will continue to manage global development and plans to submit a New Drug Application to the FDA this year for U.S. approval, aiming for an independent U.S. launch.

Previously, Otsuka secured licensing for Europe and is preparing a Marketing Authorization Application to the EMA this year. Ionis will receive a $20 million upfront payment and milestone payments from Otsuka, along with tiered royalties.

Recent positive results from Phase 3 trials were presented at the EAACI Annual Congress and published in The New England Journal of Medicine.

Ionis Pharmaceuticals announced that its management will participate in a fireside chat at the TD Cowen Genetic Medicines & RNA Summit on June 20, 2024. The event will be virtual, and a live webcast will be available on the Ionis website. Interested parties can access the webcast on the Investors & Media section of the website, with a replay available within 48 hours for a time.