CARLSBAD -
Presentations from Ionis leaders and external physicians will highlight Ionis' comprehensive integrated research, development, and commercialization capabilities, along with key pipeline and technology highlights.
'Our leadership in RNA-targeted medicine includes multiple modalities empowering us to advance the right medicines for the right targets,' said
Event location, webcast and replay information
The event will take place at the
Highlights include: Near-Term Commercial Medicines
Ionis has made important progress in building our commercial and medical affairs organizations led by individuals with deep, relevant expertise across rare and broad diseases.
With the eplontersen PDUFA date of
Eplontersen has the potential to be the treatment of choice, backed by strong efficacy and safety data, a self-administration profile and a strong global alliance with
Last week, positive Phase 3 NEURO-TTRansform data were published in The Journal of the
The Phase 3 CARDIO-TTRansform trial, the largest trial ever conducted in ATTR cardiomyopathy patients, is fully enrolled and expected to read out as early as 1H 2025.
Olezarsen is poised to be Ionis' first wholly owned independent commercial launch.
Last week, Ionis announced positive Phase 3 olezarsen Balance results in
Phase 3 studies continue for severe hypertriglyceridemia (SHTG), a more prevalent disease affecting more than three million people in the
Donidalorsen was recently granted
Ionis plans to report Phase 3 results with donidalorsen for prophylactic treatment of hereditary angioedema (
Two-year results from the Phase 2 open-label-extension trial showed that donidalorsen treatment resulted in a 96% overall sustained mean reduction from baseline in HAE attack rates. These data will be presented at a medical congress later this year.
Next Wave of Wholly Owned Medicines
Building on Ionis' proven track record in neurology with two approved medicines, 12 in clinical trials and more than 10 in lead optimization/preclinical development, Ionis is advancing a wholly owned neurology pipeline.
The lead independent program, zilganersen for the treatment of
In addition, Ionis will announce today that four new wholly owned medicines in rare pediatric neurological diseases and dementias are planned to enter first-in-patient studies by the end of 2024: ION356 (PLP1), for the treatment of children with Pelizaeus-Merzbacher Disease, a severe leukodystrophy, which recently received Rare Pediatric Disease Designation and Orphan Drug Designation from the
Advancing Technologies to Reach More Patients in Need
Ionis' leading medicinal chemistry platform is enabling technological advances to enhance existing medicine profiles and expand into new tissues and/or therapeutic areas.
Ionis is applying new chemistries such as the Mesyl Phosphoramidate (MsPA) backbone to improve the duration of effect and therapeutic index of investigational medicines.
The company is using multiple drug modalities, including antisense (ASO) and small interfering RNA (siRNA), to identify the best potential medicine for a particular target and/or tissue.
By leveraging targeted delivery approaches, including Bicycle technology via our collaboration with
Clear Path to Unlocking Next-Level Value
Ionis has a solid financial foundation with a strong balance sheet with
Ionis is investing efficiently to bring our near-term medicines to patients.
Ionis anticipates that revenue growth from multiple medicines as well as ongoing partner and royalty revenue will drive future positive cash flow.
About
For more than 30 years, Ionis has been a leader in RNA-targeted therapy, pioneering new markets and changing standards of care. Ionis currently has four marketed medicines and a promising late-stage pipeline highlighted by cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision to become the leader in genetic medicine, utilizing a multi-platform approach to discover, develop and deliver life-transforming therapies.
This press release includes forward-looking statements regarding our business, financial guidance and the therapeutic and commercial potential of QALSODY (tofersen), SPINRAZA (nusinersen), TEGSEDI (inotersen), WAYLIVRA (volanesorsen), eplontersen, olezarsen, donidalorsen, ulefnersen, zilganersen, pelacarsen, bepirovirsen, IONIS-FB-LRx, Ionis' technologies, and Ionis' other products in development. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended
Contact:
Email: info@ionisph.com
Tel: 760-603-2331
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