Roche Trial of Ionis Huntington’s Disease Drug Halted After Side Effects

Roche Trial of Ionis Huntington’s Disease Drug Halted After Side Effects

Publication date: Mar 06, 2020

Xconomy San Diego – A 20-patient Phase 1 study of an RNA drug discovered by Ionis Pharmaceuticals that is being tested as a treatment for Huntington’s disease by Roche was suspended after two reports of infections related to a device used to take samples of cerebral spinal fluid during the trial.

The suspension doesn’t appear to be related to the drug, tominersen, and no other trials of the investigational treatment were impacted-including an ongoing global Phase 3 study due to read out in 2022.

Historically Ionis has partnered many of its programs, such as the potential Huntington’s disease drug.

All of its clinical-stage neurological programs, for example, in addition to the Huntington’s program, are partnered: potential treatments for amyotrophic lateral sclerosis, Alzheimer’s disease, and frontotemporal degeneration are being advanced by Biogen (NASDAQ: BIIB), and an investigational centronuclear myopathy drug is under evalution by Dynacure.

Of the 40 drugs Ionis has under development, it controls include investigational treatments for a handful of rare diseases, including agromegaly, hereditary angioedema, beta thalassemia, and cystic fibrosis; and an experimental drug for treatment-resistant hypertension.

Concepts Keywords
Amyotrophic Lateral Sclerosis Spinal muscular atrophy
Antisense Drugs Antisense RNA
Beta Thalassemia Therapeutic gene modulation
Biogen Ionis Pharmaceuticals
Boston Biogen
Brain Antisense therapy
Catheter Pharmaceutical industry
Catheters Pharmacokinetics
CEO Huntington’s disease
Cerebral Spinal Fluid Articles
Chief Operating Officer Life sciences
China Health
Cystic Fibrosis Investigational treatments
Diabetes Catheters
Fluid Catheter
Huntingtin Protein Infections
Huntington Neurological diseases
Hypertension Sclerosis Alzheimers disease
Intrathecal Diabetes
Intrathecal Injection Trial device
IONS Infections related device
Mani Brain disorder
Neurological Diseases
San Diego


Type Source Name
disease MESH infections
drug DRUGBANK Coenzyme M
pathway KEGG Prostate cancer
disease MESH prostate cancer
disease MESH hypertension
disease MESH cystic fibrosis
disease MESH beta thalassemia
disease MESH hereditary angioedema
disease MESH rare diseases
disease MESH development
drug DRUGBANK Nonoxynol-9
disease MESH centronuclear myopathy
disease MESH amyotrophic lateral sclerosis
disease MESH brain disorder


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