MEDIBIOFARMA SL

First patient dosed in the Phase IIa clinical trial of MBF-015 in patients with Huntington’s disease   Navarra – February 2024 – Medibiofarma S.L., a clinical-stage biotechnology company developing a portfolio of new therapies for the treatment of neurodegenerative diseases and other pathologies announced the first patient dosed in the Phase IIa clinical trial of MBF-015 in patients with Huntington’s disease.   MBF-015 has previously completed pre-clinical development and a phase I trial in healthy volunteers. MBF-015 is a patented new chemical entity which acts as an inhibitor of both HDAC1 and HDAC2 and has demonstrated synaptogenesis efficacy in animal models. In human phase I studies, MBF-015 was safe and well tolerated at all doses tested and was confirmed to be fully brain-penetrant. The current single-centre phase IIa trial will be carried out at the Hospital de la Santa Creu i Sant Pau, Barcelona, Spain, under the supervision of Dr. Jaime Kulisevsky. Dr. Kulisevsky is head of the the Movement Disorders Unit at the Sant Pau hospital, Director of the Neurocognition Diagnostic & Intervention Centre (CDINC), and a member of the executive committee of the European Huntington’s disease Network (EHDN). Richard Roberts, CSO of Medibiofarma said, “Cognitive impairment is a common symptom across a wide range of neurological disorders, and an ever-increasing burden on healthcare services as the world’s population gets older. While available therapies for Huntington’s disease only address symptoms, MBF-015 represents Medibiofarma’s best shot at a disease-modifying therapy against cognitive decline, with demonstrated synaptogenesis in animal models and unhindered access to the CNS in humans. We are pleased to count on the extensive experience of Dr. Kulisevsky and his team as we look for the first clinical signs of efficacy in HD patients”.    About Huntington’s Disease Huntington’s disease (HD) is an autosomal dominant neurological disease that afflicts around one in 10,000 people. HD onset typically occurs in young adult life, and the disease is invariably fatal. Patients initially develop small choreic movements that later intensify in amplitude and frequency, causing an impairment of daily activities. Cognitive and psychiatric alterations are often the most disabling symptoms for patients with HD and are among the most difficult symptoms to treat. They are initially subtle and may go unnoticed for years but become significant later in the course of the disease, manifesting as dementia and difficulties in executive functioning. Currently available medications target selected symptoms, but do not provide a cure or slow its progression.