Phase 1 Archives

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.

ION532

ION532 (formerly IONIS-AZ5-2.5_Rx ), also known as AZD2373, is an investigational medicine designed to reduce the production of apolipoprotein L1 (APOL1) for the treatment of APOL1-associated chronic kidney disease (CKD). Genetic studies have shown that two risk alleles (termed G1 and G2) are highly associated with nondiabetic CKD and increased rate of progression towards kidney failure, providing an exciting opportunity for personalized medicine in CKD. These two variants are more frequently found in individuals of West-African ancestry. Reduction of APOL1 with ION532 in a preclinical mouse model of APOL1-related kidney disease demonstrated significantly reduced proteinuria, a hallmark of CKD. ION532 is being developed for the treatment of APOL1-associated nephropathies.

About Chronic Kidney Disease

CKD is a worldwide public health problem, affecting about 10% of the population. Within United States, over 37 million adults have CKD and millions of others are at increased risk. CKD may be caused by diabetes, high blood pressure and other disorders. Without treatment, CKD may progress and eventually lead to kidney failure, which requires dialysis or a kidney transplant to maintain life.

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