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The recent approval of Biogen’s Qalsody in SOD1–ALS highlighted the potential of ASOs in CNS diseases, while recent failures make it clear there is still work to be done.
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Wave will discontinue development of WVE-004 in C9-associated ALS and frontotemporal dementia after the therapy failed to show any clinical benefit in a mid-stage trial.
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Ionis reports third quarter 2022 financial results
11/9/2022
Ionis Pharmaceuticals, Inc. reported financial results for the third quarter of 2022.
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Ionis reports second quarter financial results and recent business achievements
8/9/2022
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today reported financial results for the second quarter of 2022 and recent business achievements.
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Ionis announced that the FDA has accepted its NDA for tofersen for SOD1-ALS. The application has been given the priority review designation and an action date of January 25, 2023.
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Biogen has terminated its asset purchase agreement with Karyopharm Therapeutics, putting a halt to the development of KPT-350 (BIIB100), which was being evaluated as a treatment for ALS.
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Biogen and Ionis Pharmaceuticals have announced results from the Phase III VALOR trial and an open-label extension (OLE) trial of tofersen.
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Ionis reports first quarter financial results and recent business achievements
5/4/2022
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today reported financial results for the first quarter of 2022 and recent business achievements.
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It was a particularly busy week for clinical trial announcements. Let's take a look.
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Lately, there has been heightened attention to briefing documents in the CNS space, a notoriously tricky area to get drugs approved, which can affect company stock prices.
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Biogen and Ionis Pharmaceuticals announced that their Phase I study of BIIB078 (IONIS-C9Rx) intended for ALS did not meet clinical endpoints, so they will discontinue the program.
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Biogen and Ionis Announce Topline Phase 1 Study Results of Investigational Drug in C9orf72 Amyotrophic Lateral Sclerosis
3/28/2022
Biogen Inc. (Nasdaq: BIIB) and Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced topline results from the Phase 1 study of BIIB078 (IONIS-C9Rx), an investigational antisense oligonucleotide (ASO) for people with C9orf72-associated amyotrophic lateral sclerosis (ALS).
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Ionis reports fourth quarter and full year 2021 financial results and recent business achievements
2/24/2022
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today reported financial results for the fourth quarter and full year ended December 31, 2021, and recent business achievements.
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Biogen R&D Day to Provide Overview of Diversified Pipeline and Capabilities with Potential for Multiple Novel Therapies in Neuroscience
9/21/2021
Biogen Inc. (Nasdaq: BIIB) will hold a virtual Investor R&D Day today, providing a comprehensive overview of the company’s diversified pipeline in neuroscience. Biogen plans to advance innovative therapies for patients through the work of its world-class researchers and the strength of its global network of collaborators
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Ionis initiates Phase 3 trial of novel antisense medicine to treat leading cause of juvenile-onset ALS
4/5/2021
ION363, the first medicine to specifically target FUS-ALS, is among Ionis' wholly owned assets the company plans to commercialize
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Ionis' third novel antisense medicine for ALS, its first designed to treat a broad ALS population, begins clinical trial
10/22/2020
Tofersen and IONIS-C9Rx also currently in clinical trials targeting genetic forms of the disease
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Ionis' neurological franchise marks year of achievement
1/7/2020
Potential breakthrough therapies for Huntington's, Alzheimer's, ALS and Parkinson's among numerous programs advancing in the clinic
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After Recent Tau Failure, Biogen Licenses Tau Therapeutic from Ionis for Alzheimer's Disease
12/19/2019
As Biogen stays in the hunt for an Alzheimer’s treatment with the recent presentation of data about aducanumab made at the 12th Clinical Trials on Alzheimer’s Disease (CTAD) conference on December 5, it’s expanding its pipeline. -
Ionis licenses investigational Alzheimer's therapy
12/19/2019
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that Biogen, a collaboration partner for neurological diseases, has licensed IONIS-MAPTRx, an antisense therapy designed to selectively reduce production of microtubule-associated protein tau (MAPT), or tau, in the central nervous system.