PIPELINE

SOM3355 is a β blocker with a novel Mechanism of Action (MoA) representing a different class of molecule for the treatment of Chorea in Huntington's Disease and is the truth alternative to the current available therapies.

Used safely as a mild anti-hypertensive for 40 years, has been characterized by SOM as having a rich pharmacology including also the modulation of dopamine through VMAT1 and VMAT2 inhibition, making the product well suited to treat movement disorders and the complexity of psychiatric disorders. SOM3355 successfully completed two Phase2 study on Huntington’s chorea patients showing efficacy with no sign of worsening depression, somnolence, anxiety and akathisia. The Company is now planning to approach FDA and run a pivotal study. SOM3355 has been granted with Orphan Drug Designation by FDA. SOM3355 development plan will than include also tardive dyskinesia indication.

Huntington's Disease (HD) is a genetic rare progressive neurodegenerative disease characterized by motor, cognitive and psychiatric abnormalities. Chorea is a key symptom of HD and occurs in approximately 90% of HD patients characterized by abnormal, jerky, muscular movements of extremities, facial and axial muscles compromising the daily living.

SOM3366 is R-enantiomer of SOM3355 with less effect on the β1-adrenoceptor, and it is a New Molecular Entity. SOM3366 is under evaluation for the treatment of Tourette Syndrome, a movement and neuropsychiatric disorder, as first indication and it may be expanded to other movement disorders. FDA guidance (pre-IND meeting) and EMA Scientific Advice were obtained.

SOM1311 is a small molecule chaperone of Phenylalanine Hydroxylase under development for the treatment of Phenylketonuria. The molecule showed promising preclinical data that supports moving directly to a Phase 2a PoC.

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