AI-Guided Drug Repurposing for Treatment of Huntington’s Successful at Phase II

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A Phase II clinical trial of an AI-selected drug to treat the abnormal movement symptoms of Huntington’s disease has met its primary endpoint of an improvement in total maximal chorea score of at least two points versus placebo.

The drug identified, bevantolol hydrochloride, is a β1‐adrenoceptor antagonist that is used to treat hypertension in some countries, but is not currently approved in the US. It is being developed by Spanish biotech company SOM Biotech.

“Huntington’s disease causes a progressive degeneration of nerve cells in the brain and as such, has a massive adverse effect on the quality of patient’s lives, both physically and mentally. There is a huge unmet medical need for new treatment options to treat symptoms of HD, including chorea, and we are very encouraged by the results we have seen thus far,” commented Raúl Insa, CEO of SOM Biotech.

New treatments for Huntington’s, an inherited disease, are desperately needed. The condition is currently incurable and only limited options exist to treat the abnormal movement (chorea) and other symptoms associated with the disease that come with a range of adverse effects including excessive sleepiness, parkinsonism and the risk of depression and suicidality.

SOM Biotech, based in Barcelona, have a proprietary AI technology that can quickly search for new drug targets and possible drugs to repurpose for a variety of different conditions in the neurological space such as Huntington’s, Parkinson’s disease, phenylketonuria, glioblastoma and others.

The company has a mixed team of tech developers and preclinical and clinical researchers so is able to develop and trial new drugs identified by its AI system, as well as work with partners who wish to trial their own drugs.

They are developing and testing bevantolol in-house as a potential treatment for the chorea symptoms seen in patients with Huntington’s. In a Phase IIa ‘proof of concept’ study, presented at the recent European College of Neuropsychopharmacology hybrid congress, the researchers assigned 32 patients with Huntington’s disease to the two arms of the study where over four periods of 6 weeks the participants received placebo and bevantolol (SOM3355) 100 and 200 mg twice daily in a crossover fashion.

The primary endpoint was defined as “improvement of at least 2 points in the total maximal chorea (TMC) sub-score of the Unified Huntington’s Disease Rating Scale (UHDRS) in any active drug period compared with the placebo period,” by the researchers. This was met by 57% of the participants, with 28.6%, 25.0%, 17.9%, and 10.7% of the cohort achieving even better improvements of 3, 4, 5, or 6 points, respectively.

Importantly, only mild-moderate side effects were seen in the patients given bevantolol such as headache, fatigue and nausea, but not the more severe side effects sometimes seen in patients treated with the currently approved drugs to treat these symptoms such as suicidality.

“We will continue to progress SOM3355 through the clinic and anticipate starting a Phase 2b trial later this year in order to bring this promising product candidate to patients as soon as possible,” commented Insa.

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