SOM Innovation Biotech, a biopharmaceutical company developing treatments for neurological orphan diseases with a proprietary AI-based drug discovery technology, has passed the milestone of 80% enrollment in its pivotal Phase IIb study, SOMCT03, for the treatment of chorea in Huntington’s disease (HD) with its lead drug SOM3355.
This international multicentric study, conducted in 22 centres across seven European countries (Spain, France, Italy, Germany, Poland, the UK and Switzerland) in collaboration with the European Huntington’s Disease Network (EHDN) and the Cure Huntington’s Disease Initiative (CHDI) Foundation, started in Q4 2022. It is a 12-week double-blind randomised study assessing in parallel the efficacy and safety of two dose regimens of SOM3355 (200mg or 300mg given twice a day) compared to placebo in HD patients with choreic movements. A total of 130 patients diagnosed with manifest HD, mildly to moderately impaired, suffering from choreic movements will be included.
The primary outcome measure of efficacy is the change in the Total Maximal Chorea score (TMC) from baseline to the end of treatment. The TMC is a sub-score of the Unified Huntington’s Disease Rating Scale (UHDRS). There have been no safety concerns to date and a sub-set of 24 patients have been enrolled in a PK sub-study to perform PK-PD assessments. Top-line results are expected by the end of this year thanks to the great performance of Ergomed, the selected CRO for the trial.
EudraCT Number: 2021-003453-28; ClinicalTrials.gov ID: NCT05475483
Catherine Scart, Chief Medical Officer of SOM Biotech, highlights: “SOM3355 has already shown ,with statistical significance, to reduce chorea in patients with Huntingdon’s Disease in a previous Phase 2a study with a good safety profile. Given the significant unmet need, related to the safety profile of current treatments, SOM3355 has the potential to treat chorea in HD with a much better side-effect profile than the current standard-of-care.” Results were published in the British Journal of Pharmacology: [Gamez et Al. A proof of-concept study with SOM3355 (bevantolol hydrochloride) for reducing chorea in Huntington’s disease. British. J. Clin. Pharmacol. 2022 Dec 9. doi: 10.1111/bcp.15635].
The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SOM3355. The FDA Office of Orphan Products Development grants orphan status to support the development of drugs for the safe and effective treatment of rare disorders. Receiving ODD may qualify SOM Biotech for a seven-year period of US marketing exclusivity upon approval of SOM3355.
SOM Biotech is engaged in a series B funding round seeking for €32 million with the help of US Capital Global in USA and CaixaBank in EU.
About SOM3355
SOM3355 has the potential to be a treatment for chorea in Huntington’s disease with a significantly improved side-effect profile over current VMAT2 inhibitors.
Using its proprietary artificial intelligence SOMAIPRO technology, SOM Biotech identified SOM3355 (bevantolol hydrochloride, a drug commonly used for years to treat hypertension) as a potent vesicular monoamine transporter type 2 (VMAT2) inhibitor and is developing SOM3355 to treat chorea in HD with a much better side-effect profile than the current standard-of-care.
About Chorea in Huntington’s Disease
HD is an inherited disorder whereby progressive degeneration of brain centres involved in movement can result in chorea, an involuntary, jerky, movement disorder that randomly affects somatic muscles and flows from one part of the body to another, impacting daily living activities and quality of life.