SOM3355: a new drug for the treatment of the "chorea" symptom in Huntington's disease

Thanks to artificial intelligence, a drug has been discovered to be tested in the treatment of the "chorea" symptom in Huntington's disease: it is called SOM3355

The US Food and Drug Administration has just granted orphan drug designation to SOM3355, an investigational drug for the treatment of chorea (involuntary jerking movements) in patients with Huntington's disease.

SOM3355 (bevantolol hydrochloride) works as a beta blocker, inhibiting the vesicular monoamine transporter 2 (VMAT2).

Produced by the Spanish company SOM Biotech, it was originally developed to treat hypertension and it is marketed in Japan, South Korea and China with this indication.

Its potential in Huntington's disease has emerged thanks to an AI-powered drug screening platform - run by SOM Biotech - that is used to identify existing drugs that can be reused for use in new indications.

SOM3355 has already passed a phase 2a study, improving the involuntary and jerky movements known as chorea in the participants.

Phase 2a was conducted on 32 patients, compared two doses of SOM3355 versus placebo and showed that 57.1% of patients treated with the drug had a reduction in chorea compared to the control group.

SOM Biotech has announced that it will launch a Phase 2b study later this year.

In fact, the company believes that its drug has important advantages over xenazine - currently on the market for the treatment of 'chorea' in Huntington's - which is associated with side effects that can sometimes become serious, including sedation, parkinsonism and risk of depression and suicide.

In contrast, patients who took SOM3355 in the phase 2a study only experienced mild or moderate adverse events, including headache, fatigue, nausea and vomiting, which are known side effects of beta-blockers, according to the Spanish company. The drug has, in fact, shown a favorable safety profile, with no depressive effects.

It is important to remember that orphan drug status encourages the development of therapies for the treatment of rare and serious diseases, through benefits for companies, such as seven years of market exclusivity and tax exemption and it is therefore very important that SOM3355 has received this designation.

"Chorea is an involuntary movement that progressively disables and increases the sense of stigma of those who suffer from it", says Prof. Ferdinando Squitieri, Scientific Director of the LIRH Foundation, who adds: "Having the possibility to test a drug that limits its gravity without interfering with autonomy is a great opportunity for patients".

For more information on the phase 2a study, please visit: clinigalstrials.gov 

For more info on the drug, please visit: SOM Biotech