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Dr. Raúl Insa, SOM Biotech founder and CEO, with the principal investigator of the study, Dr. Jaume Kulisevsky, scientific director at the Hospital de la Santa Creu i Sant Pau (Foto: IIB Sant Pau).

SOM Biotech initiates of Phase 2a clinical trial of SOM3355 in Huntington’s disease patients

SOM Biotech, a clinical-stage biopharmaceutical company –with headquarters in Barcelona Science Park– that discovers and develops drugs for orphan indications in the central nervous system,  has initiated of Phase 2a clinical trial of its compound SOM3355 for the treatment of the chorea movements associated to Huntington’s disease. The investigational product is a repositioned drug, currently commercialized for the treatment of hypertension. 


SOM3355 is an oral and brain penetrant drug that was discovered by SOM Biotech to be a potent selective inhibitor of Vesicular Monoamine Transporter-2 (VMAT2), using its artificial intelligence genetic algorithm platform (Phoenix®). After the promising results of the preclinical trials, the biopharmaceutical company starts the study is a Phase 2a proof-of-concept to evaluate the safety and efficacy of SOM3355 in Huntington’s disease (HD) in 30 patients with chorea movements. 

The principal investigator of the study is Dr. Jaume Kulisevsky, director of the Sant Pau Biomedical Research Institute (IIB Sant Pau) and the Movement Disorder Unit at Hospital de la Santa Creu i Sant Pau de Barcelona. It will also take place simultaneously in three other centers: the Hospital Clínic de Barcelona, the Bellvitge University Hospital and the Vall d’Hebron University Hospital, under the leadership of Dr Esteban Muñoz, Dr Matilde Calopa and Dr Josep Gàmez, respectively.

A rare and disabling disease 

According to SOM Biotech’s internal assessment, this pathology has a prevalence of 6-8/105  in western countries, and most of those affected develop the disease between 35 and 55 years of age. Life expectancy is long, between 15 and 20 years, during which the disease progresses gradually and slowly, causing significant functional, social and family disability.

“Huntington’s disease affects some 2,500 people in Spain and about 78,000 around the world. There is no treatment that modifies the progression and the available medications are symptomatic with side effects that often make them unusable. SOM3355 is a safe drug that has been administered for many years to patients with hypertension and which has been highly valued by a market study conducted by Insights in Life Science (ilS)”, says Dr Raúl Insa, SOM Biotech founder and CEO.

And he adds: “We hope to be able to demonstrate the efficacy expected in this clinical trial since it would mean a considerable reduction of the healthcare cost since it is a well-known product and is cheaper than other alternatives. If all goes well, it could reach the market within a period of five years and achieve global sales of around €600 million”.

SOM3355 is the second product from SOM Biotech to reach the clinical phase. It is preceded by the successful case of compound SOM0226 for the treatment of amyloidosis that the biopharmaceutical company licensed to the American company Corino Therapeutics after obtaining very positive results in the Phase II trial.

Efficacy and Safety to combat choreic movements

Chorea (from the Greek khoreia, dance), an involuntary jerky movement that is purposeless and abrupt, is the most characteristic symptom of HD. HD predominantly affects the distal extremities and facial muscles. Abnormal movements usually become gradually more severe and many patients finish their years with very severe chorea that compromises motor function.

SOM, after using a proprietary ligand analysis computational algorithm and completing the preclinical assays, determined that SOM3355 is a highly effective Vesicular Monoamine Transporter Type 2 (VMAT2) inhibitor, a class of compounds that has demonstrated to have success in treating Central Nervous System movement disorders such as Huntington’s Chorea. The validating preclinical studies also indicated that SOM3355 has the potential to have promising clinical efficacy as well as properties that are likely to avoid the side effects of other members of the VMAT2 inhibitor class that have led to restricted prescribing. Acording  the biopharmaceutical company, SOM3355 could also be extended to other related diseases such as Tourette syndrome, tardive dyskinesia and ballism.

“We are very proud to start this clinical trial of SOM3355 in Huntington patients. It will validate for the second time our business model and artificial Phoenix technology while providing patients with safer and more affordable drugs. The four hospitals taking part in the trial are of excellent profile and we are confident that the recruitment will go smoothly”, says Dr. Raúl Insa.

Dr. Jaime Kulisevsky adds: “Currently, the treatment of movement disorders in Huntington’s disease is limited by the side effects of the drugs used. SOM3355 offers a promising alternative to improve the tools available to treat the disease. The clinical trial that we begin has a double-blind and randomized design that will allow to verify objectively the ability of the drug to improve abnormal movements. The participation of four centers in our environment will facilitate the recruitment of the sufficient number of patients and guarantee the objectivity of the results.”

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