SOM0226 is a small molecule transthyretin (TTR) stabilizer for the treatment of TTR amyloidosis. It is active on different TTR mutations and has shown clinical efficacy in stabilizing plasma TTR in healthy volunteers and patients with Familial Amyloid Polyneuropathy (FAP) after oral administration. SOM0226 is currently in Phase 2a of the Clinical trials in different forms of TTR amyloidosis, including FAP and leptomeningeal amyloidosis. SOM0226 is out-licensed to a company based in the US.
TTR amyloidosis occurs when the protein Transthyretin (a protein that is mainly made in the liver), “misfolds” or changes its shape in an abnormal way, and forms into fibrous clumps. Depending on the specific type of TTR amyloidosis, the amyloid is deposited into various organs and/or nerves, which can lead to permanent damage and organ malfunction.
SOM3355 is an oral VMAT2 inhibitor (vesicular monoamine transporter 2) for the symptomatic treatment of chorea movements associated with Huntington’s disease (HD), and other hyperkinetic movement disorders. SOM3355 is currently in Phase 2a trials in HD and has shown a favorable safety profile with no depression effects. You can find more information about Clinical trials for SOM3355 here NCT03575676.
Huntington’s disease is an inherited disease that causes the progressive breakdown (degeneration) of nerve cells in the brain. Huntington’s disease has a broad impact on a person’s functional abilities and usually results in movement, thinking (cognitive) and psychiatric disorders.
SOM1201 and SOM1202
SOM1201 and SOM1202 are small molecules in development for adrenoleukodystrophy and adrenomyeloneuropathy, respectively, the two main forms of X-linked adrenoleukodystrophy (x-ALD). Currently, SOM1201 and SOM1202 are ready to enter to the Phase 2a.
X-linked adrenoleukodystrophy is a genetic neurodegenerative disease caused by the accumulation of very long chain fatty acids due to mutations on the peroxisomal transporter ABCD1. Some of the symptoms of the disease include behavior problems, seizures, learning disabilities, deafness, fatigue, trouble coordinating movements (ataxia), weakness and pain in the hands and feet (peripheral neuropathy), muscle spasms and weakness.
SOM1311 is a small molecule pharmacological chaperone of Phenylalanine Hydroxylase for the treatment of Phenylketonuria.
Phenylketonuria (PKU) is is characterized by an inborn error of metabolism that results in decreased metabolism of the amino acid phenylalanine. Untreated, PKU can lead to intellectual disability, seizures, behavioral problems, and mental disorders.
SOM0208 is a small molecule for the treatment of Niemann–Pick C disease.
Niemann–Pick type C is a lysosomal storage disorder caused by defects in the intracellular transport and metabolism of cholesterol and glycolipids. The disease has a broad clinical spectrum. Affected individuals may have enlargement of the spleen (splenomegaly) and liver (hepatomegaly), or enlarged spleen or liver combined (hepatosplenomegaly), but this finding may be absent in later onset cases. Prolonged jaundice or elevated bilirubin can present at birth.