IONIS PHARMACEUTICALS


Associated tags: Knowledge, RNA, Ionis, IONS, Ionis Pharmaceuticals, Pharmaceutical industry, Patient

Locations: LATIN AMERICA, US, NORFOLK, EU, CHINA, UNITED STATES, TG, ET, GEORGIA, CALIFORNIA, CR

Ionis Publishes 2023 Corporate Responsibility Report

Retrieved on: 
Thursday, April 25, 2024

Received approval for two Ionis-discovered medicines in 2023, and continued to advance a broad pipeline poised to bring a steady cadence of important new medicines to people with serious diseases

Key Points: 
  • Received approval for two Ionis-discovered medicines in 2023, and continued to advance a broad pipeline poised to bring a steady cadence of important new medicines to people with serious diseases
    CARLSBAD, Calif., April 25, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), today published its 2023 Corporate Responsibility Report: Action for a Healthier Future .
  • The report details Ionis' relentless passion to create better futures for people touched by serious disease as well as other stakeholders.
  • In 2023, Ionis extended its commitment to corporate responsibility (CR) through the establishment of three strategic pillars with actionable goals.
  • For more information on the 2023 Corporate Responsibility program, please visit: https://www.ionispharma.com/about/corporate-responsibility/
    For more information about QALSODY, visit https://www.qalsody.com/ .

Ionis to hold first quarter 2024 financial results webcast

Retrieved on: 
Tuesday, April 23, 2024

CARLSBAD, Calif., April 23, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Tuesday, May 7th at 11:30 a.m. Eastern Time to discuss its first quarter 2024 financial results.

Key Points: 
  • Webcast scheduled for Tuesday, May 7 at 11:30 a.m. Eastern Time
    CARLSBAD, Calif., April 23, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Tuesday, May 7th at 11:30 a.m. Eastern Time to discuss its first quarter 2024 financial results.
  • The webcast may be accessed at https://ir.ionispharma.com/events-and-presentations/upcoming-events .
  • A replay will be available for a limited time at the same address.

Ionis presents positive results from Phase 3 Balance study of olezarsen for familial chylomicronemia syndrome

Retrieved on: 
Sunday, April 7, 2024

CARLSBAD, Calif., April 7, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced full results from the Phase 3 Balance study of Ionis' lead independent investigational medicine, olezarsen, for the treatment of adults with familial chylomicronemia syndrome (FCS). The olezarsen 80 mg monthly dose met the primary endpoint of significantly reducing triglycerides (TGs) in patients with genetically validated FCS at six months. In addition, olezarsen demonstrated robust and sustained reductions in TGs and serum apolipoprotein C-III (apoC-III) levels. Importantly, olezarsen reduced the incidence of acute pancreatitis (AP) events over the 12-month treatment period compared to placebo. Olezarsen also demonstrated a favorable safety and tolerability profile. These results were presented in an oral presentation at the 2024 American College of Cardiology (ACC) Annual Meeting in Atlanta, Georgia and published simultaneously in The New England Journal of Medicine (NEJM). Based on these data, Ionis is pursuing regulatory approval of olezarsen as a potential breakthrough treatment for adults with FCS.

Key Points: 
  • Based on these data, Ionis is pursuing regulatory approval of olezarsen as a potential breakthrough treatment for adults with FCS.
  • In the study, patients were treated with olezarsen 80 mg (n=22), 50 mg (n=21) or placebo (n=23) once every four weeks.
  • Serious AEs occurred in 14% of patients treated with olezarsen 80 mg, 19% treated with olezarsen 50 mg, and 39% treated with placebo.
  • Ionis will host a webcast to discuss the detailed results from the Balance study on Monday, April 8 at 10:00 am ET.

Ionis to present at upcoming investor conferences

Retrieved on: 
Monday, April 1, 2024

CARLSBAD, Calif., April 1, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences:

Key Points: 
  • CARLSBAD, Calif., April 1, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences:
    23rd Annual Needham Virtual Healthcare Conference on Monday, April 8, 2024
    2024 RBC Capital Markets Global Healthcare Conference on Tuesday, May 14, 2024
    BofA Securities Health Care Conference 2024 on Tuesday, May 14, 2024
    45th Annual Goldman Sachs Global Healthcare Conference on Wednesday, June 12, 2024
    A live webcast of these presentations can be accessed on the Investors & Media section of the Ionis website at https://ir.ionispharma.com/events-and-presentations/upcoming-events .
  • Replays will be available on the Ionis website within 48 hours and will be archived for a limited time.

Ionis to hold olezarsen Phase 3 data webcast

Retrieved on: 
Thursday, March 28, 2024

CARLSBAD, Calif., March 28, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it will host a live webcast on Monday, April 8th at 10:00 a.m. Eastern Time to discuss the olezarsen Phase 3 Balance study results in patients with familial chylomicronemia syndrome (FCS) presented at the 2024 American College of Cardiology Annual Meeting.

Key Points: 
  • Webcast scheduled for Monday, April 8 at 10:00 a.m. Eastern Time
    CARLSBAD, Calif., March 28, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it will host a live webcast on Monday, April 8th at 10:00 a.m. Eastern Time to discuss the olezarsen Phase 3 Balance study results in patients with familial chylomicronemia syndrome (FCS) presented at the 2024 American College of Cardiology Annual Meeting.
  • The webcast may be accessed at https://ir.ionispharma.com/events-and-presentations/upcoming-events .
  • A replay will be available for a limited time at the same address.

New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs after Gene Therapy

Retrieved on: 
Wednesday, March 6, 2024

* The Phase 4 study evaluates clinical outcomes and safety following treatment with SPINRAZA over a 2-year period in infants and toddlers with spinal muscular atrophy (SMA) who have unmet clinical needs after treatment with Zolgensma® (onasemnogene abeparvovec).

Key Points: 
  • * The Phase 4 study evaluates clinical outcomes and safety following treatment with SPINRAZA over a 2-year period in infants and toddlers with spinal muscular atrophy (SMA) who have unmet clinical needs after treatment with Zolgensma® (onasemnogene abeparvovec).
  • The new data show that plasma neurofilament light chain (NfL) levels, an objective biomarker of axonal injury and neurodegeneration, were reduced in nearly all study participants treated with SPINRAZA.
  • These data will be presented at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (March 3-6, 2024).
  • “Our evolving understanding of gene therapy indicates there may be an opportunity for better outcomes,” said Crystal Proud, M.D., Pediatric Neurologist at Children’s Hospital of the King’s Daughters.

Ionis announces new chief global product strategy officer to lead next phase of commercial growth

Retrieved on: 
Thursday, February 29, 2024

CARLSBAD, Calif., Feb. 29, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that Kyle Jenne has rejoined Ionis as executive vice president, chief global product strategy officer, to lead all aspects of commercialization at the Company. Mr. Jenne has more than 25 years of biopharmaceutical commercial and executive leadership experience and has launched innovative medicines across a broad range of therapeutic areas including cardiovascular, neurology and allergy.

Key Points: 
  • Onaiza Cadoret-Manier, chief global product strategy and operations officer, is leaving the company to pursue another opportunity.
  • She has built a stellar team of professionals with deep experience across all aspects of commercialization," said Brett P. Monia, Ionis' chief executive officer.
  • Mr. Jenne previously spent five years in commercial leadership roles at Ionis and Akcea, which was fully acquired by Ionis in 2020.
  • His most recent role at Ionis was as EVP & chief commercial officer.

Eplontersen granted U.S. FDA Fast Track designation for patients with transthyretin-mediated amyloid cardiomyopathy

Retrieved on: 
Thursday, February 8, 2024

CARLSBAD, Calif., Feb. 8, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Ionis and AstraZeneca's eplontersen, an investigational therapy for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults.

Key Points: 
  • CARLSBAD, Calif., Feb. 8, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Ionis and AstraZeneca's eplontersen, an investigational therapy for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults.
  • The FDA grants development programs Fast Track designation to facilitate the development and expedite the review of drugs that demonstrate the potential to treat serious conditions and fill an unmet medical need.
  • "CARDIO-TTRansform is the largest, most comprehensive study ever conducted in ATTR-CM patients, with results expected as early as next year."
  • WAINUA was granted Orphan Drug Designation in the U.S. and in the EU for the treatment of transthyretin-mediated amyloidosis (ATTR).

Ionis to hold fourth quarter and full year 2023 financial results webcast

Retrieved on: 
Wednesday, February 7, 2024

CARLSBAD, Calif., Feb. 7, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, February 21st at 11:30 a.m. Eastern Time to discuss its fourth quarter and full year 2023 financial results.

Key Points: 
  • Webcast scheduled for Wednesday, February 21 at 11:30 a.m. Eastern Time
    CARLSBAD, Calif., Feb. 7, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, February 21st at 11:30 a.m. Eastern Time to discuss its fourth quarter and full year 2023 financial results.
  • The live webcast may be accessed at https://ir.ionispharma.com/events-and-presentations/upcoming-events .
  • A replay will be available for a limited time at the same address.

Praxis Precision Medicines Provides Update on Advancing Clinical Stage Portfolio

Retrieved on: 
Monday, January 8, 2024

BOSTON, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today provided an update on its portfolio and planned key milestones in 2024:

Key Points: 
  • Praxis made strong progress in developing PRAX-628 for focal epilepsy, which affects over 1 million Americans.
  • Initial data from PRAX-628 show it has a good tolerability profile, a potentially wide therapeutic index and reaching efficacious concentrations within 24 hours.
  • A preliminary analysis of the 15 mg cohort showed that this cohort exceeds the expectations in terms of drug activity.
  • In 2023, Praxis disclosed results from a Phase 1 dose escalation study of PRAX-628 in healthy volunteers:
    PRAX-628 was generally well-tolerated at all tested doses.