Ionis Pharmaceuticals

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

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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

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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

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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.

WAYLIVRA (volanesorsen): An Antisense Oligonucleotide Inhibitor of Apolipoprotein CIII (apoCIII) mRNA - Market Size, Forecasts, and Emerging Insights, 2019-2023 and 2024-2032 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, February 27, 2024

Further, it also consists of future market assessments inclusive of the WAYLIVRA market forecast analysis for lipodystrophy in the 7MM, SWOT, analysts' views, comprehensive overview of market competitors, and brief about other emerging therapies in lipodystrophy.

Key Points: 
  • Further, it also consists of future market assessments inclusive of the WAYLIVRA market forecast analysis for lipodystrophy in the 7MM, SWOT, analysts' views, comprehensive overview of market competitors, and brief about other emerging therapies in lipodystrophy.
  • WAYLIVRA (volanesorsen), an antisense oligonucleotide inhibitor of apolipoprotein CIII (apoCIII) mRNA, is being developed by Ionis Pharmaceuticals through its subsidiary company, Akcea Therapeutics to treat familial partial lipodystrophy (FPL).
  • Volanesorsen is an antisense oligonucleotide that binds to apoC-III mRNA, leading to its degradation and preventing the translation of apoC-III protein.
  • What are the other emerging products available and how are these giving competition to WAYLIVRA for lipodystrophy?

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

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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.

Dr. Lisa Johnson-Pratt Joins Aspen Neuroscience as Senior Vice President, Therapeutic Program Lead

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Wednesday, February 14, 2024

SAN DIEGO, Feb. 14, 2024 /PRNewswire/ -- Aspen Neuroscience, Inc., a private biotechnology company developing induced Pluripotent Stem Cell (iPSC)-derived cell therapies, announced that Lisa Johnson-Pratt, MD has joined the company's executive management team in a new role as Senior Vice President, Therapeutic Program Lead.

Key Points: 
  • SAN DIEGO, Feb. 14, 2024 /PRNewswire/ -- Aspen Neuroscience, Inc., a private biotechnology company developing induced Pluripotent Stem Cell (iPSC)-derived cell therapies, announced that Lisa Johnson-Pratt, MD has joined the company's executive management team in a new role as Senior Vice President, Therapeutic Program Lead.
  • "We are delighted to welcome Dr. Johnson-Pratt to Aspen Neuroscience," said Damien McDevitt, PhD, Aspen Neuroscience president and chief executive officer.
  • "As we prepare to enter the clinic with our first-in-human trial for Parkinson's disease, we are deepening our investment in the company's development.
  • Lisa is a board director at Assembly Bio and Tracon Pharma, and is the board chair of Young People in Recovery.

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

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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.

Vect-Horus Enters Exclusive License Agreement with Ionis Pharmaceuticals to Advance Systemic Delivery of RNA-Targeted Medicines for Neurological Diseases

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Tuesday, January 30, 2024

The agreement provides Ionis with a worldwide, exclusive license for a specified number of targets using Vect-Horus’ platform technology ”VECTrans” for systemic delivery of RNA-targeted therapeutics that can cross the blood-brain barrier and address targets in the central nervous system.

Key Points: 
  • The agreement provides Ionis with a worldwide, exclusive license for a specified number of targets using Vect-Horus’ platform technology ”VECTrans” for systemic delivery of RNA-targeted therapeutics that can cross the blood-brain barrier and address targets in the central nervous system.
  • Ionis has an option to add additional targets to the license.
  • Vect-Horus retains the rights to use its technology, for these specific targets, for all non-RNA-Targeted Medicines.
  • “This license is aligned with our strategy of using our technology to deliver drug payloads, such as RNA-targeted therapeutics, to the brain and other tissues.

Biotech Industry Veterans Jeff Jonas and Al Robichaud Join Cure Ventures as Partners

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Monday, January 8, 2024

Cure Ventures (“Cure”), a life sciences venture capital firm focusing on company formation around groundbreaking curative technologies, today announced the appointment of industry leaders Jeff Jonas, M.D.

Key Points: 
  • Cure Ventures (“Cure”), a life sciences venture capital firm focusing on company formation around groundbreaking curative technologies, today announced the appointment of industry leaders Jeff Jonas, M.D.
  • and Al Robichaud, Ph.D. as partners to expand the firm’s work with biotech entrepreneurs in developing therapeutics that cure life-altering diseases.
  • It’s clear to me that Cure has the chops to strategically choose its partners and elevate their work.
  • More importantly, he is about as well respected a leader as you can find in our industry,” said Dave Fallace, co-founder and managing partner at Cure Ventures.