PIPELINE

SOM3355 is an oral VMAT2 inhibitor (vesicular monoamine transporter 2) for the symptomatic treatment of chorea movements associated with Huntington’s disease (HD). SOM3355 has successfully completed Phase 2a trials in HD and shown a favourable safety profile with no depression effects. The program received positive results of Pre-IND with US Food and Drug Administration (FDA) and Scientific Advice with European Medicines Agency. SOM3355 has also been granted Orphan Drug Designation by the US FDA. A Phase 2b dose range trial for the drug will be initiated in the EU in 2022. 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.

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 was outlicensed to Corino Therapeutics based in the US and is currently in development for different forms of TTR amyloidosis

TTR amyloidosis occurs when the protein Transthyretin (a protein that is mainly made in the liver), “misfolds” or binds together to form 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, including heart failure.

SOM1311 is a small molecule pharmacological chaperone of Phenylalanine Hydroxylase for the treatment of Phenylketonuria. The response rate of the drug is higher compared with the current standard of care. Clinical Phase 2a trial initiation expected at the beginning of 2023.

Phenylketonuria (PKU) is characterized by a birth defect that causes the amino acid, phenylamine to build up in the body. Untreated, PKU can lead to intellectual disability, seizures, behavioral problems, and mental disorders.

SOM3366 is an oral VMAT2 inhibitor (vesicular monoamine transporter 2) for the symptomatic treatment of Tardive Dyskinesia (TD). SOM3366 is currently finishing preclinical development in TD, with Phase 1 clinical trial initiation expected in 2022.

Tardive Dyskinesia is an involuntary neurological movement disorder caused by the use of neuroleptic drugs that are prescribed to treat certain psychiatric or gastrointestinal conditions.

SOM0777 is a small molecule for the treatment, Glioblastoma. SOM0777 was outlicensed to U-Cell Therapeutics, based in Singapore, which is developing safe ways of administration and delivery in the tumour. When these new formulations are developed and ready for clinical testing, U-Cell Therapeutics will perform the clinical proof-of-concept study and the rest of clinical development.

Glioblastoma is an aggressive cancer that occurs in the brain and spinal cord. Initially, signs and symptoms of glioblastoma are nonspecific. They may include headaches, personality changes, nausea, and symptoms similar to those of a stroke. Worsening of symptoms is often rapid, and may progress to unconsciousness. The cause of most cases is unclear. Uncommon risk factors include genetic disorders, such as neurofibromatosis and Li–Fraumeni syndrome, and previous radiation therapy. Glioblastomas represent 15% of brain tumors.