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

All programs are obtained through our proprietary AI-based technology, SOMAIPRO®.


Chorea movements associated with Huntington’s disease
SOM3355 is an atypical VMAT2 inhibitor (vesicular monoamine transporter 2) inhibitor for the symptomatic treatment of chorea movements associated with Huntington’s disease (HD). SOM3355 has successfully completed a Phase 2a trials in HD and shown a favorable safety profile with no neurological or neuropsychiatric side effects. The Phase 2b dose-range finding trial program received guidance from Pre-IND discussions with the US Food and Drug Administration (FDA) and Scientific Advice from the European Medicines Agency (EMA). The study is ongoing and the outcome expected in Q3 2024. SOM3355 has also been granted Orphan Drug Designation by the US FDA. 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.


TTR amyloidosis
SOM0226 is an oral and brain penetrating 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 improves 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.


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).


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 2025.

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.


Methamphetamine Addiction
SOM3366 is an oral VMAT2 inhibitor (vesicular monoamine transporter 2) that has 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.