Clinical Archives

ION582*

ION582 is an investigational antisense medicine designed to inhibit the expression of the UBE3A transcript (UBE3A-ATS) for the potential treatment of Angelman syndrome (AS).

About Angelman Syndrome

AS is a rare, genetic neurological disease caused by the loss of function of the maternally inherited UBE3A gene. AS typically presents in infancy and is characterized by intellectual disability, balance issues, motor impairment, and debilitating seizures. Some patients are unable to walk or speak. Some symptoms can be managed with existing drugs; however, there are no approved disease modifying therapies.

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.

ION904

ION904 is an investigational next-generation ligand-conjugated antisense (LICA) medicine designed to inhibit the production of angiotensinogen to decrease blood pressure in people with uncontrolled hypertension.

About Treatment-Resistant Hypertension

Treatment-resistant hypertension (TRH) is defined as failure to achieve a blood pressure goal of 140/90 (systolic/diastolic) despite the use of three or more antihypertensive medications. People with TRH have been found to have a three-fold higher chance of having fatal and non-fatal cardiovascular events relative to those with controlled hypertension.

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