Phase 3 Archives

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.

Ulefnersen

Ulefnersen, formerly known as ION363, is an investigational antisense medicine designed to reduce the production of the fused in sarcoma (FUS) protein to treat people with amyotrophic lateral sclerosis (ALS) caused by mutations in the FUS gene. Ulefnersen is also known as Jacifusen (not an official USAN name) in honor of Jaci Hermstad, the first patient treated with the drug under an expanded access program. Because antisense-mediated reduction of mutant FUS protein in a FUS-ALS mouse model demonstrated the ability to prevent motor neuron loss, it is hypothesized that reduction of FUS protein will reverse or prevent disease progression in FUS-ALS patients.

About FUS-ALS

FUS-ALS is a rare, fatal, neurodegenerative disorder characterized by muscle weakness, loss of movement, and difficulty breathing and swallowing, resulting in a severely declining quality of life and eventually death. Current treatment options are extremely limited, with no medicines that significantly slow disease progression. It is estimated that there are approximately 350 patients with FUS-ALS in G7 countries.

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

Olezarsen

Olezarsen, formerly known as IONIS-APOCIII-L_Rx and AKCEA-APOCIII-L_Rx, is an investigational ligand-conjugated antisense (LICA) medicine designed to inhibit the production of apoC-III for patients who are at risk of disease due to elevated triglyceride levels.

About FCS

ApoC-III is a protein produced in the liver that regulates triglyceride metabolism in the blood. People with severely elevated triglycerides, such as people with FCS, are at high risk for acute pancreatitis and an increased risk of cardiovascular disease, or CVD. It is estimated that FCS affects one to two individuals per million worldwide and more than three million patients have severe hypertriglyceridemia in the U.S.

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

Eplontersen

Eplontersen, formerly known as IONIS-TTR-L_Rx and AKCEA-TTR-L_Rx, is an investigational ligand-conjugated antisense (LICA) medicine designed to inhibit the production of TTR protein. We are developing eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR.

About ATTR

ATTR amyloidosis is a systemic, progressive and fatal disease in which patients experience multiple overlapping clinical manifestations caused by the inappropriate formation and aggregation of TTR amyloid deposits in various tissues and organs, including peripheral nerves, heart, intestinal tract, eyes, kidneys, central nervous system, thyroid and bone marrow. The progressive accumulation of TTR amyloid deposits in these tissues and organs leads to organ failure and eventually death.

ATTR-CM is caused by the accumulation of misfolded TTR protein in the cardiac muscle. Patients experience ongoing debilitating heart damage resulting in progressive heart failure, which results in death within three to five years from disease onset. ATTR-CM includes both the genetic and wild-type form of the disease. There are an estimated 300,000 to 500,000 patients with ATTR-CM worldwide.

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

IONIS-FB-L

IONIS-FB-L_Rx, also known as RG6299, is an investigational ligand-conjugated antisense (LICA) medicine designed to inhibit the production of complement factor B (FB), and the alternative complement pathway.

About IgA Nephropathy
Genetic association studies have shown that overaction of the alternative complement pathway has been associated with the development of several complement-mediated diseases, including immunoglobulin A, or IgA, nephropathy, or IgAN.

IgAN is one of the most common causes of inflammation that impairs the filtering ability of kidneys and is an important cause of chronic kidney disease and kidney failure. Also known as Berger’s disease, IgAN is characterized by deposits of IgA in the kidneys, resulting in inflammation and tissue damage.

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