Clinical Archives

ION717*

ION717 is an investigational antisense medicine designed to inhibit the production of prion protein (PrP) for the potential treatment of prion disease.

About Prion Disease
Prion disease is a rare, fatal neurodegenerative disease caused by misfolding of PrP which accumulates in the brain. People with prion disease often experience progressive memory impairment, personality changes, difficulties with movement and loss of independence. There are currently no effective disease-modifying treatments for prion disease. In most cases, a person succumbs to prion disease within a year following symptom onset.

Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.

Tofersen

Tofersen is an investigational antisense medicine designed to inhibit the production of superoxide dismutase 1 (SOD1), which is a well understood genetic cause of amyotrophic lateral sclerosis (ALS).

Tofersen is currently being studied in the Phase 3, randomized, placebo-controlled ATLAS study to evaluate whether tofersen can delay clinical onset when initiated in presymptomatic individuals with ALS and a SOD1 genetic mutation. More details about ATLAS(NCT04856982) can be found at clinicaltrials.gov.

About SOD1-ALS

SOD1-ALS is a rare, fatal, neurodegenerative disorder caused by a mutation in the SOD1 gene leading to a progressive loss of motor neurons. As a result, people with SOD1-ALS experience increasing muscle weakness, loss of movement, difficulty breathing and swallowing and eventually succumb to the disease. SOD1-ALS is diagnosed in approximately 2% of all ALS cases, impacting about 330 people in the United States. Approximately 5-10% of people with ALS are thought to have a genetic form of the disease; however, they may not have a known family history of the disease.

Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.

ION306

ION306, also known as BIIB115, is an investigational antisense medicine in development for spinal muscular atrophy (SMA). This program has the potential to help address additional unmet needs of patients as well as be administered with long interval dosing. ION306 is designed to target the root cause of SMA by increasing the production of functional SMN protein.

About Spinal Muscular Atrophy

Spinal Muscular Atrophy (SMA) is a rare, genetic, neuromuscular disease that affects individuals of all ages. It is characterized by a loss of motor neurons in the spinal cord and lower brain stem, resulting in progressive muscle atrophy and weakness. SMA is caused by a deficiency in the production of survival motor neuron (SMN) protein due to a damaged or missing SMN1 gene, with a spectrum of disease severity. Some individuals with SMA may never sit; some sit but never walk; and some walk but may lose that ability over time. In the absence of treatment, children with the most severe form of SMA would usually not be expected to reach their second birthday.

Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.

ION455

ION455, also known as AZD7503, is an investigational ligand-conjugated antisense (LICA) medicine designed to inhibit the production of hydroxysteroid 17-beta dehydrogenase 13 (HSD17B13) protein as a potential treatment for non-alcoholic steatohepatitis.

About Metabolic Dysfunction-Associated Steatohepatitis (MASH)

MASH is the more severe form of metabolic dysfunction-associated fatty liver disease (MASLD). It is related to the epidemic of obesity, pre-diabetes and diabetes. Unlike liver disease caused by alcohol consumption, MASH is the result of an accumulation of fat in the liver, which can lead to inflammation, cirrhosis and liver-related death.

Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.

Sapablursen

Sapablursen, formerly known as IONIS-TMPRSS6-L_Rx, is an investigational ligand-conjugated antisense (LICA) medicine designed to target the TMPRSS6 gene to modulate the production of hepcidin, which is the key regulator of iron homeostasis. By modulating hepcidin expression, sapablursen has the potential to positively impact diseases characterized by iron deficiency, such as polycythemia vera (PV).

About Polycythemia Vera

Polycythemia Vera (PV) is a rare, non-genetic and potentially fatal disease caused by overproduction of red blood cells. This overproduction leads to a thickening of the blood, which increases patients’ risk of life-threatening blood clots, including in the lungs, heart and brain. Patients with PV also experience severe iron deficiency due to hepcidin overexpression. There are no approved disease-modifying treatments for PV.

Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.