IONIS PHARMACEUTICALS INC (IONS)

Expected Completion of Olezarsen Clinical Trial (NCT05079919)

Ionis Pharmaceuticals Inc. is on track to complete its clinical trial (NCT05079919) for Olezarsen, a treatment aimed at reducing transthyretin mRNA and protein expression, which is significant for addressing transthyretin amyloidosis. The study's completion on July 1, 2025, comes as Ionis seeks to advance its portfolio in this therapeutic area. Additionally, the related patent for the product TEGSEDI, which is assigned to Ionis and AKCEA Therapeutics, is set to expire on April 29, 2031, further highlighting the strategic importance of their developments in mitigating the condition associated with this amyloidosis.

Expiration of Patent US8101743 for WAINUA (AUTOINJECTOR)

The patent US8101743 related to the autoinjector device WAINUA, which modulates the expression of transthyretin, expires on April 1, 2026. The patent was held by Ionis Pharmaceuticals Inc and AstraZeneca AB. This expiration could potentially impact the competitive landscape for therapies related to transthyretin-related diseases, allowing for increased market access for generics and competitors in this therapeutic area.

CARDIO-TTRansform MRI Sub-Study Clinical Trial Completion

Ionis Pharmaceuticals, Inc. has announced the completion of the CARDIO-TTRansform MRI Sub-Study, part of a multicenter, open-label, Phase 3 clinical trial assessing the long-term safety and efficacy of Eplontersen (formerly known as ION-682884). The trial, aimed at patients with hereditary transthyretin-mediated amyloid polyneuropathy, is currently active and has enrolled 151 participants. It includes a treatment period of up to 157 weeks, with a post-treatment evaluation period of 24 weeks. Eplontersen is being evaluated in conjunction with daily vitamin A supplementation, and the trial is expected to influence the future commercialization of the drug.

Expected Completion of Clinical Trial for Sapablursen in Patients with Polycythemia Vera

Ionis Pharmaceuticals, Inc. is expected to complete a clinical trial for sapablursen in patients with Polycythemia Vera on June 1, 2026. This Phase 2a study evaluates the drug, formerly known as ISIS 702843, in approximately 40 participants and includes four periods: screening, treatment, treatment extension, and post-treatment. The treatment involves subcutaneous injections, with a total of 18 doses administered over roughly 16 months. Currently, the trial is listed as active but not recruiting. The outcome of this study may influence the future market positioning of sapablursen and its potential financial impact on Ionis Pharmaceuticals.

• www.clinicaltrials.gov/study/NCT05143957

Completion of MECP2 Duplication Syndrome Study

Ionis Pharmaceuticals, Inc. is set to complete its observational study on MECP2 Duplication Syndrome by June 1, 2026. The study, which began recruitment in October 2023, aims to analyze biomarkers and disease progression in up to 40 participants. It utilizes various assessments, including fluid biomarker collection and electrophysiological evaluations, to support the development of potential therapies for this rare genetic condition. The completion of this study may influence future treatment options and market opportunities for Ionis Pharmaceuticals in the area of rare diseases.

• www.clinicaltrials.gov/study/NCT06014541

Expected Completion of Clinical Trial for ION269

Ionis Pharmaceuticals, Inc. is expected to complete its clinical trial evaluating ION269 for participants with Down Syndrome at risk for Alzheimer's Disease on March 1, 2027. This Phase 1b study will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of ION269, which is being tested in a population showing evidence of brain amyloid positivity. The trial currently involves approximately 30 adult participants and includes a single ascending dose methodology throughout a 36-week treatment period followed by a 4-week follow-up. The trial is currently in the recruiting phase, having started on December 20, 2024, and is registered under Clinical Trial ID NCT06673069.

Expected completion of clinical trial for Olezarsen in severe hypertriglyceridemia

Ionis Pharmaceuticals is set to complete a Phase 3 clinical trial for its drug Olezarsen, aimed at treating severe hypertriglyceridemia by March 1, 2027. The trial, involving approximately 144 participants, assesses both the long-term safety and efficacy of the treatment. This clinical trial is part of Ionis Pharmaceuticals' broader strategy to expand its offering in lipid management therapies, which may impact its positioning in the competitive landscape of treatments for dyslipidemia-related conditions.

• www.clinicaltrials.gov/study/NCT05392114

Expected completion of Phase 3 clinical trial for donidalorsen in HAE

Ionis Pharmaceuticals, Inc. expects to complete its Phase 3 clinical trial of donidalorsen, a drug aimed at assessing the long-term safety and efficacy for patients with Hereditary Angioedema (HAE), on March 1, 2027. This global study currently has an enrollment target of approximately 144 participants, who will be divided into two groups: those transitioning from a previous study and new participants previously treated with standard HAE prophylactic therapies. The trial is pivotal as it explores Extended Treatment Periods for participants, indicating potential long-term benefits of donidalorsen in managing HAE.

• www.clinicaltrials.gov/study/NCT05392114

Completion of Olezarsen Clinical Trial

Ionis Pharmaceuticals, Inc. is set to complete its Phase 3 clinical trial of Olezarsen on June 1, 2027. This trial focuses on adults with Familial Chylomicronemia Syndrome (FCS) who have previously been treated with Volanesorsen. The study has an active status and involves 24 participants receiving Olezarsen every four weeks over a treatment period extending up to 209 weeks. This prolonged treatment aims to assess the safety and efficacy of Olezarsen, likely influencing future commercialization plans if the trial results are positive.

• www.clinicaltrials.gov/study/NCT05185843

Expected Completion of Clinical Trial for ION717

Ionis Pharmaceuticals, Inc. is expected to complete its clinical trial for ION717 on July 1, 2027. This study, which is a first-in-human trial focused on individuals with prion disease, aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of ION717. The trial involves a total of 56 participants and includes a randomized, double-blind treatment phase followed by an open-label extension. ION717 is being evaluated for its potential therapeutic effects in this specific patient population.