The pipeline of SOM Biotech mainly contains programs focused on Orphan indications.
All programs are obtained through the proprietary AI-based technology.
|Product||Indication||Discovery||In vitro||In vivo||Phase I||Phase II||Patients WW||Estimated Peak Sales||Commercial Rights|
|Chorea in Huntington’s disease||
€0.8 – €1.1 B
€0.6 – €0.8 B
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 was out-licensed to the 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.
Chorea movements associated with Huntington’s disease
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 finished Phase 2a trials in HD and has shown a favorable safety profile with no depression effects. The program received positive results of Pre-IND with FDA and SA with EMA. The Phase 2b will be initiated in 2021. Application for ODD was performed. 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.
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.
Tardive Dyskinesia is an involuntary neurological movement disorder caused by the use of dopamine receptor blocking drugs that are prescribed to treat certain psychiatric or gastrointestinal conditions.
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.
Niemann–Pick type C
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.
SOM0044 is a small molecule for the treatment of Parkinson’s disease.
Parkinson’s disease is a slowly progressive neurodegenerative disorder that affects predominately dopamine-producing neurons in a specific area of the brain called substantia nigra. People with the disease may experience bradykinesia, rigidity, tremor at rest, shuffling gait and postural instability. The disease is caused by aging, genetic and environmental factors. Symptoms usually appear around 60 years of age. Available treatments are symptomatic, mainly involving dopaminergic agents for the control of motor function.
SOM0061 program includes three drug candidates for the treatment of COVID-19.
SOM Biotech has applied its artificial intelligence based technology (SOMAIPRO) to identify inhibitors of the 3CL proteases of SARS-CoV-2, SARS-CoV and MERS-CoV viruses as potential candidates to treat COVID-19. Identified drug candidates were validated in vitro in collaboration with the Department of Pharmacy and Pharmaceutical Sciences led by Professor Dong-Hae Shin from Ewha Womans University in South Korea.Clinical studies with the three repurposed drug candidates can be initiated immediately.
Coronavirus Disease 2019 (COVID-19) is a new infectious disease caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2; previously known as 2019-nCoV). The clinical spectrum of SARS-CoV-2 infection appears to be wide, ranging from asymptomatic infection, and mild upper respiratory tract illness to severe viral pneumonia with respiratory failure and even death.
SOM0777 is a small molecule for the treatment of different types of Cancer, specifically Glioblastoma. SOM0777 was out-licensed to the company based in Singapore.
Glioblastoma is the most aggressive cancer that begins within the brain. 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 often is 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.