The trial was scheduled to complete in January 2020, but in May 2020, recruitment was suspended and the end date adjusted to December 2020, due to the Covid-19 pandemic. Called VALOR, the study will enroll an additional 99 people with SOD1-ALS, who will receive an undisclosed, fixed dose of BIIB067 or placebo for 28 weeks. A route of administration in pharmacology and toxicology is the path by which a drug, fluid, poison, or other substance is taken into the body.. Name: Tofersen Synonyms: BIIB067, Ionis-SOD1Rx, ASO1 Therapy Type: DNA/RNA-based Target Type: Other Condition(s): Amyotrophic Lateral Sclerosis U.S. FDA Status: Amyotrophic Lateral Sclerosis (Phase 3) Company: Biogen, IONIS Pharmaceuticals Background. No one developed myelitis, an inflammation of the spinal cord accompanied by sensory and motor deficits, but the investigators noted this condition was seen in some people dosed with BIIB067 in an ongoing Phase 3 and long-term extension (see below). Tofersen (formerly IONIS-SOD1 Rx ), also known as BIIB067, is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS). Intrathecal administration of tofersen is being studied for the treatment of amyotrophic lateral sclerosis (ALS) due to SOD1 mutations. It is designed to bind to the SOD1 mRNA — a blueprint of the gene read by the cell machinery to make SOD1 protein — preventing production of the SOD1 protein. Secondary outcomes are other clinical measures such as time to needing a ventilator, muscle strength, survival, and CSF levels of phosphorylated neurofilament heavy chain. Approximately 10 percent of people receiving BIIB067 showed elevated CSF white cell counts, and a similar number had elevated protein in their CSF. NCI concept code for route of administration: C38114 Injection of ASO1 into the brain or spinal cord of mice or rats expressing mutant SOD1 reduced spinal-cord levels of SOD1 mRNA and protein. The latest report Superoxide Dismutase [Cu-Zn] - Pipeline Review, H2 2019, outlays comprehensive information on the Superoxide Dismutase [Cu-Zn] (Superoxide Dismutase 1 … Dublin, April 01, 2021 (GLOBE NEWSWIRE) -- The "Antisense Oligonucleotide Therapeutics Market by Target Indication, Type of Therapy, Type of Molecule Type of Generation and Key Geographies: Industry Trends and Global Forecasts, 2020-2030" report has been added to ResearchAndMarkets.com's offering. Currently, treatment options for patients with ALS are extremely limited with no drugs that significantly slow disease progression. Background: Tofersen is an antisense oligonucleotide that mediates the degradation of superoxide dismutase 1 (SOD1) messenger RNA to reduce SOD1 protein synthesis. Mutations in SOD1can cause the enzyme to misfold as it is being made, which may l… Routes of administration are generally classified by the location at which the substance is applied. The trial is to be conducted at 31 sites in North America, Europe, Australia, and Japan, and finish in July 2021. In particular, in the fast-progressing patient subgroup, there was a very high level prior to administration of tofersen, and that fast-group's level appeared to come down after treatment with tofersen. Synonyms: BIIB067, Ionis-SOD1Rx, ASO1 Route of Administration shall consist of an alphabetic term which has a maximum length … In March 2019, Biogen added a Phase 3 efficacy arm to the same trial. The study took place at 18 sites in the U.S., Canada, and Western Europe. Seven participants had serious adverse events. Tofersen (formerly IONIS-SOD1Rx), also known as BIIB067, is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS). The small trial was not designed to measure efficacy, but exploratory outcomes included change from baseline in the ALS functional Rating Scale-Revised (ALSFRS-R), lung vital capacity and muscle strength, and blood and CSF neurofilament concentrations. The Phase III study of Tofersen is also progressing very well. As a result, patients with SOD1-ALS experience muscle weakness, loss of movement, difficulty in breathing and swallowing and eventually succumb to their disease. Tofersen (BIIB067), previously called IONIS-SOD1Rx, is an ASO that specifically binds to SOD1 mRNA. General practitioners can expect… The Lou Gehrig’s disease pipeline report provides detailed information of the Lou Gehrig’s disease pipeline products from the initial phase of product development until its commercialization in the Lou Gehrig’s disease market. The highest-dose group appeared to decline more slowly on the ALSFRS-R, showing a loss of 1.19 points at week 12, compared with 5.63 in the placebo group. Tofersen (Biogen) 9.4.6.1. In December 2018, a research study began to evaluate the distribution of BIIB067 in the central nervous system by SPECT/CT imaging after co-administration of a microdose of radiolabeled BIIB067 and high- or low-dose BIIB067 in 20 healthy volunteers. Novara Therapeutics will focus on pre-clinical development of the proprietary imaging and therapeutic technology to advance diagnosis and treatment of bone disease in patients. There is substantial evidence that mutations in the SOD1 gene … Antisense technology is now beginning to deliver on its promise to treat diseases by targeting RNA. In January 2016, Biogen and Ionis began a Phase 1/2 safety trial in SOD1-ALS. Prevalence is about 5-7 per 100,000. One person in the treatment group and one on placebo died of respiratory failure; one person in the treatment group died from a lung embolism. It also includes the Amyotrophic Lateral Sclerosis therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive ALS pipeline products. For details on BIIB067 trials, see clinicaltrials.gov. Patients with a mutation in the SOD1 gene develop a form of ALS referred to as SOD1-ALS. The U.S. Federal Aviation Administration had insufficient personnel to oversee the certification of new Boeing airplanes and should make significant reforms in how it approves new aircraft, an international aviation panel said Friday. Routes can also be classified based on where the target of action is. Common examples include oral and intravenous administration. Concentrations of neurofilament heavy and light chains in CSF and blood decreased from baseline to 12 weeks in the highest-dose group. BIIB067 is a second-generation antisense oligonucleotide (ASO) targeting the mRNA for superoxide dismutase 1. Three died. Tofersen is an ASO designed to target mutated SOD1 mRNA to prevent protein production, thereby slowing ALS progression. Intraperitoneal injection is widely used in rodents as a route of drug administration. Intrathecal administration of tofersen is being studied for the treatment of amyotrophic lateral sclerosis (ALS) due to SOD1 mutations.. Methods: We conducted a phase 1-2 ascending-dose trial evaluating tofersen in adults … In 2013, Ionis tested a first-generation SOD1 ASO, which proved safe in people, but lacked potency (May 2013 news). Biogen licensed ASO1 from Ionis, and is leading its clinical development. Antisense Oligonucleotide Therapeutics Market by Target Indication (Duchenne Muscular Atrophy, Spinal Muscular Atrophy, Familial Chylomicronemia Syndrome and others), Type of Therapy (Monotherapy and Combination), Type of Molecule (DNA Molecule and RNA Molecule) Type of Generation (First, Second and Third) and Key Geographies (North America, Europe, Asia- Pacific and … RSS Approximately 90 percent of patients with ALS have no known family history, while approximately 10 percent of patients have a known genetic cause for their disease. ASO1 treatment preserved neuromuscular innervation and stemmed the rise of phosphorylated neurofilament heavy chain in blood, a prognostic biomarker for ALS. Although the exact pathological mechanism remains unknown, mutant SOD1 is believed to exert a toxic action on motor neurons, and reducing its levels may be beneficial. It mediates mRNA degradation to prevent SOD1 protein synthesis and reduce levels of SOD1 protein. Name: Tofersen NCI Thesaurus OID: 2.16.840.1.113883.3.26.1.1. Tofersen, previously known as IONIS-SOD1Rx and BIIB067, is an experimental antisense oligonucleotide (ASO) or antisense therapy for people with SOD1-associated familial ALS. All Rights Reserved. The disappearance of drugs from the peritoneal cavity upon injection is because of diffusion into the surrounding tissues. Tofersen is being developed for ALS patients who carry the SOD1 mutation, which accounts for just 2% of ALS cases, but nevertheless plays an important part in the drug’s mechanism. An ongoing, open-label safety extension will run through June 2023, with treatment offered for up to five years. It is estimated that there are ~55,000 patients diagnosed with ALS in major markets1. Sales Forecast ... Distribution by Route of Administration 9.10. The Investor Relations website contains information about Biogen's business for stockholders, potential investors, and financial analysts. BIIB067 is being developed for ALS caused by SOD1 mutations, which account for about 20 percent of all familial ALS and 2 percent of all ALS cases. U.S. FDA Status: Amyotrophic Lateral Sclerosis (Phase 3) ALS is a progressive neurodegenerative disorder where damage to and death of motor neurons (the nerve cells that control the movement of muscles) leads to a loss of muscle control and paralysis. Paper Alert: Antisense Oligonucleotide Therapy Safe for ALS? It also includes the Amyotrophic Lateral Sclerosis therapeutics assessment by product type, stage, route of administration, and molecule type … In the multiple-dosing phase, 48 participants in four cohorts were randomized 3:1 to receive 20, 40, 60, or 100 mg BIIB067 or placebo five times over 12 weeks. The exact cause of the disease is unknown, but some inherited forms of ALS are caused by a mutation in thesuperoxide dismutase 1 (SOD1) gene.SOD1 encodes for a protein, which neutralizes harmful radical oxygen molecules. Antisense Oligonucleotides: Can They Take on ALS, SMA, Prions? This was largely consistent with the route administration lumbar punctures. Route of Administration: Infusion ; Intradermal ; Intramuscular ; Intranasal ; Oral ; Parenteral ; Subcutaneous ; Topical ; Product Types: Monotherapy ; Combination SOD1 protein levels in cerebrospinal fluid dropped by 3 percent in the low-dose group, and 36 percent in the high-dose group, compared with placebo. Finally, one of the limits of gene therapy is to identify a safe route of administration to make sure that the treatment gets into the CNS and reaches as many affected cells as possible. Orthopaedic Research UK (ORUK) and Embryo Ventures (Embryo) announce the formation of Novara Therapeutics, a joint venture company, to commercialise a patented bone-targeting contrast agent. Tofersen (previously known as IONIS SOD1Rx, ISIS 333611 and BIIB 067), an antisense therapy, is being developed by Biogen for the treatment of amyotrophic Route of Administration Abbreviations | en Espańol Below is an alphabetical list of … The company then screened 2,000 SOD1 ASOs and identified two targeting the mRNA 3' untranslated region that were most effective at lowering SOD1 mRNA and protein levels in cells. According to results presented at the May 2019 American Academy of Neurology conference, and later published, ASO treatment was safe and reduced mutant SOD1 protein (May 2019 conference news; Miller et al., 2020). Tofersen is an investigational molecule for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), the second most common genetic form of ALS, a … The panel also faulted assumptions made by the airplane manufacturer in designing the 737 MAX and for not disclosing more information to the FAA. Condition(s): Amyotrophic Lateral Sclerosis A single injection delayed disease onset, improved motor function, and increased survival in the animals. Nine single-stranded antisense oligonucleotide (ASO) … Target Patient Population 9.4.6.2. The multiple-dosing phase of the trial was completed in January 2019. To circumvent IV administration challenges and to provide an alternative route of administration, ... Tofersen. Administered through multiple routes of delivery The elegant structure of single-stranded antisense therapies enables us to administer antisense therapies by nearly all routes of administration, reaching parts of the body that cause disease while minimizing drug exposure to other parts of the body, thereby providing improved safety and enabling us to expand the number of diseases we can treat. To make a comment you must login or register. ©1989 – 2021 Ionis PharmaceuticalsTM is a trademark of Ionis Pharmaceuticals, Inc. Akcea TherapeuticsTM is a wholly owned subsidiary of Ionis Pharmaceuticals, Inc. You are now leaving https://www.ionispharma.com to visit. 2088232-70-4 - Tofersen [USAN] - Searchable synonyms, formulas, resource links, and other chemical information. Target Type: Other (timeline) Overview. In nonhuman primates, ASO1 lowered SOD mRNA in the CNS after spinal-cord injection (Jul 2018 news on McCampbell et al., 2018). Those are 2 pieces of very important biochemical data. Facebook Most adverse events were mild to moderate; the most common were headache, procedural pain, and postlumbar syndrome related to the spinal taps. BIIB067 is a second-generation antisense oligonucleotide (ASO) targeting the mRNA for superoxide dismutase 1. Intraperitoneally applied drug forms are supposed to be located in the peritoneal cavity. Primary outcomes were adverse events and PK; the secondary outcome was levels of SOD1 protein in cerebrospinal fluid after the last dose. ALS is a rare, fatal neurodegenerative disorder characterized by loss and dysfunction of neurons in motor pathways. The patients also experienced falls and post-lumbar puncture syndrome or headache that occurred due to the persistent leakage of CSF. A phase I and II double-blind, randomized, placebo-controlled trial showed a reduction in dysfunctional cerebrospinal fluid SOD1 concentrations in the Tofersen treated group . The study enrolled 84 people who received single or multiple ascending doses by intrathecal injection into their lumbar spinal cord. Next-Gen Antisense and Small Protein-Protein Disruptors Benefit SOD1 Models, Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS, Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models. There is substantial evidence that mutations in the SOD1 gene are responsible for a toxic gain of function that can lead to rapid progressive loss of motor neurons in patients with SOD1-ALS. Company: Biogen, IONIS Pharmaceuticals. MND is relatively uncommon with an annual incidence of about 2 cases per 100,000 population. Falls were also common. The primary outcome is change from baseline in the ALSFRS-R at week 28. Copyright © 1996–2021 FBRI LLC. Patients with a mutation in the SOD1 gene develop a form of ALS referred to as SOD1-ALS. 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