PIPELINE

SOM Biotech’s pipeline mainly contains programs focused on orphan diseases with high unmet medical needs

All programs are obtained through the proprietary AI-based technology.

SOM3355

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 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 is ongoing whose final report is expected in 2024. 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

TTR amyloidosis
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

Phenylketonuria
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 during 2024.

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.

SOM0034

Duchenne Musculary Dystrophy
SOM0034 is related with stabilization of dystrophin. SOM0034 is ongoing in animal model testing and Phase 2 clinical trial initiation expected in 2025.

Duchenne Muscular Dystrophy
is a rare X-linked progressive and fatal muscle-wasting disease in males caused by mutations in the DMD gene (dystrophin protein).

SOM3366

Tardive Dyskinesia
SOM3366 is an oral VMAT2 inhibitor (vesicular monoamine transporter 2) for the symptomatic treatment of Tardive Dyskinesia (TD). SOM3366 is currently completing preclinical development. Phase 1 clinical trial planned in 2024.

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.

SOM3366

Methamphetamine Addiction
SOM3366 is an oral VMAT2 inhibitor (vesicular monoamine transporter 2) that have been shown to potentially decrease the neurochemical and behavioral effects of Methamphetamine (mAMP). Preclinical work planned in 2024.
 
Drug abuse, including Methamphetamine Addiction, is a major unmet health concern particularly in the US.
To learn more about the pipeline of SOM Biotech and 15 more Orphan indications that we are working on, you can contact us.