Friday, May 27, 2016 12:56:21 AM
We have discovered and are developing three potentially transformational drugs, nusinersen, IONIS-TTRRx and volanesorsen, which we believe are close to commercialization. We designed each of these three drugs to treat patients with orphan diseases who have limited or no therapeutic options. In total, we are developing these three drugs for six different patient populations. In 2015, we completed target enrollment in the first pivotal Phase 3 study for each of these three drugs, and we anticipate reporting data from these studies in the first half of 2017. We designed nusinersen to treat patients with spinal muscular atrophy, or SMA, a severe motor-neuron disease that is the leading genetic cause of infant mortality. We designed IONIS-TTR Rx to treat patients with transthyretin amyloidosis, or TTR amyloidosis, a fatal disease in which patients experience progressive buildup of amyloid plaque deposits in tissues throughout the body, including peripheral nerves, heart, intestinal tract, kidney and bladder. We designed volanesorsen to treat patients with diseases associated with extremely high levels of triglycerides, including patients with two severe and rare genetic conditions called familial chylomicronemia syndrome, or FCS, and patients with familial partial lipodystrophy, or FPL. We anticipate that the data from our pivotal Phase 3 studies of these drugs, if positive, will support global regulatory filings for each drug. We believe that the significant unmet medical need and the severity of these diseases could warrant a rapid path to market. Already our partners for these programs, Biogen for nusinersen and GSK for IONIS-TTR Rx , are preparing to commercialize these drugs. Our wholly owned subsidiary, Akcea Therapeutics Inc., or Akcea, is preparing to commercialize volanesorsen. All three companies are engaging in pre-commercialization activities to understand the patient journey, build disease awareness with physicians and patients and develop their launch plans. We believe all three of these drugs have the potential to reach the market in the next several years.
To maximize the value of volanesorsen and other earlier-stage drugs for serious cardiometabolic disorders, we formed Akcea Therapeutics to focus on developing and commercializing these drugs. Akcea’s pipeline includes volanesorsen, IONIS-APOCIII-L Rx , IONIS-APO(a)-L Rx and IONIS-ANGPTL3-L Rx . Moving these drugs into a company that we own and control allows us to retain substantial value from them and ensures Ionis’ core focus remains on innovation. Akcea is building development and commercialization expertise in lipid and cardiometabolic diseases, including highly trained, specialized medical, marketing and sales teams, to successfully commercialize volanesorsen and the other lipid drugs in its pipeline. To learn more about Akcea Therapeutics, visit Akceatx.com.
Recent IONS News
- Form 8-K - Current report • Edgar (US Regulatory) • 06/18/2024 01:29:02 PM
- Ionis announces expanded licensing agreement with Otsuka in Asia Pacific for investigational medicine donidalorsen in hereditary angioedema • PR Newswire (US) • 06/18/2024 11:00:00 AM
- Ionis to present at TD Cowen Genetic Medicines & RNA Summit • PR Newswire (US) • 06/17/2024 08:09:00 PM
- Form 144 - Report of proposed sale of securities • Edgar (US Regulatory) • 06/17/2024 02:59:16 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 06/10/2024 08:33:55 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 05/31/2024 11:15:31 AM
- Ionis presents positive results from OASIS-HAE and OASISplus studies of investigational medicine donidalorsen in patients with hereditary angioedema • PR Newswire (US) • 05/31/2024 11:00:00 AM
- Ionis to hold donidalorsen Phase 3 data webcast • PR Newswire (US) • 05/28/2024 11:00:00 AM
- New data from the Phase 3 NEURO-TTRansform study evaluating WAINUA™ (eplontersen) to be presented at the 2024 International Symposium on Amyloidosis (ISA) • PR Newswire (US) • 05/23/2024 11:00:00 AM
- Biogen and Ionis Announce Topline Phase 1/2 Study Results of Investigational Drug in Amyotrophic Lateral Sclerosis • GlobeNewswire Inc. • 05/16/2024 11:30:14 AM
- Ionis and Biogen Announce Topline Phase 1/2 Study Results of Investigational Drug in Amyotrophic Lateral Sclerosis • PR Newswire (US) • 05/16/2024 11:30:00 AM
- Ionis announces positive topline results from Phase 1/2a trial of ION582 for Angelman syndrome • PR Newswire (US) • 05/16/2024 11:00:00 AM
- Ionis to host 2024 virtual Annual Meeting of Stockholders • PR Newswire (US) • 05/15/2024 11:05:00 AM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 05/13/2024 09:25:49 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 05/07/2024 08:55:46 PM
- Form 10-Q - Quarterly report [Sections 13 or 15(d)] • Edgar (US Regulatory) • 05/07/2024 07:55:10 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 05/07/2024 03:15:41 PM
- Ionis reports first quarter 2024 financial results • PR Newswire (US) • 05/07/2024 11:00:00 AM
- Ionis Publishes 2023 Corporate Responsibility Report • PR Newswire (US) • 04/25/2024 11:00:00 AM
- Ionis to hold first quarter 2024 financial results webcast • PR Newswire (US) • 04/23/2024 11:05:00 AM
- Ionis presents positive results from Phase 3 Balance study of olezarsen for familial chylomicronemia syndrome • PR Newswire (US) • 04/07/2024 01:48:00 PM
- Ionis to present at upcoming investor conferences • PR Newswire (US) • 04/01/2024 11:05:00 AM
- Ionis to hold olezarsen Phase 3 data webcast • PR Newswire (US) • 03/28/2024 11:05:00 AM
- Positive olezarsen Phase 3 data in familial chylomicronemia syndrome to be presented at 2024 American College of Cardiology (ACC) annual meeting • PR Newswire (US) • 03/25/2024 12:18:00 PM
- Ionis announces positive results from Phase 2 study of ION224, an investigational medicine demonstrating clinical efficacy in the treatment of NASH/MASH • PR Newswire (US) • 03/13/2024 11:00:00 AM
Last Shot Hydration Drink Announced as Official Sponsor of Red River Athletic Conference • EQLB • Jun 20, 2024 2:38 PM
ATWEC Announces Major Acquisition and Lays Out Strategic Growth Plans • ATWT • Jun 20, 2024 7:09 AM
North Bay Resources Announces Composite Assays of 0.53 and 0.44 Troy Ounces per Ton Gold in Trenches B + C at Fran Gold, British Columbia • NBRI • Jun 18, 2024 9:18 AM
VAYK Assembling New Management Team for $64 Billion Domestic Market • VAYK • Jun 18, 2024 9:00 AM
Fifty 1 Labs, Inc Announces Acquisition of Drago Knives, LLC • CAFI • Jun 18, 2024 8:45 AM
Hydromer Announces Attainment of ISO 13485 Certification • HYDI • Jun 17, 2024 9:22 AM