Pipeline
Phase 2a PoC
Phase 2a PoC
Phase 2a PoC
Phase 2a PoC
Phase 2a PoC
SOM3355
Advancing Care for Huntington’s Disease
Huntington’s disease is a hereditary neurodegenerative disorder that causes progressive motor, cognitive, and behavioral changes due to the gradual breakdown of nerve cells in the brain.
SOM3355 is an investigational therapy with a first-in-class mechanism of action (MoA) designed to address the complex challenges of Huntington’s disease (HD). As a selective β-adrenergic blocker combined with VMAT1 and VMAT2 inhibition, SOM3355 aims to treat Huntington’s disease symptoms beyond chorea, broadening to behavioral and psychiatric symptoms throughout the progression of HD.
How It Works
- Selective β1-adrenergic modulation addresses behavioral symptoms such as anxiety, akathisia, and irritability.
- VMAT1 and VMAT2 inhibition works across key regions of the brain, including the prefrontal cortex, nucleus accumbens and striatum, to reduce chorea without creating side effects caused by the pure dopamine depletors agents
- This multimodal MoA is unique to SOM3355 and not shared with other β -blockers.
Clinical Progress
- Positive outcomes from proof-of-concept and Phase 2b trials demonstrated efficacy with no increases in depression, somnolence, anxiety, or akathisia with a very safe profile.
- Received an Orphan Drug Designation from EMA (Sept 2025) supporting clinically meaningful benefit beyond chorea including behavioral and neuropsychiatric symptomatology
- Holds Orphan Drug Designation in the United States.
- Following a successful end-of-Phase 2 FDA meeting (Sept 2025), SOM3355 is preparing to advance into Phase 3.
- Future development will also explore additional indications, including Tardive Dyskinesia.
SOM3366
Tourette Syndrome
Tourette Syndrome is a neurological condition characterized by involuntary motor and vocal tics, often beginning in childhood and varying in frequency and intensity over time.
SOM3366 is a New Molecular Entity and the R-enantiomer of SOM3355, engineered to have reduced β1-adrenoceptor activity. It is under development as a potential new treatment for Tourette Syndrome, a neurological condition characterized by motor and vocal tics, often accompanied by behavioral or psychiatric symptoms.
- FDA pre-IND guidance and EMA Scientific Advice obtained already
The program also has potential to expand into other movement-related disorders.
SOM1311
Phenylketonuria (PKU)
Phenylketonuria (PKU) is a rare metabolic disorder in which the body cannot properly process the amino acid phenylalanine, requiring lifelong dietary management to prevent neurological complications.
SOM1311 is a small-molecule chaperone of Phenylalanine Hydroxylase (PAH), designed to help restore enzyme function in patients living with Phenylketonuria (PKU). Preclinical data have shown strong promise, supporting advancement directly into a Phase 2a Proof-of-Concept study.