Date: 2015-02-10
Type of information: Treatment of the first patient
phase: 2a
Announcement: treatment of the first patient
Company: Forum Pharmaceuticals (USA - MA)
Product: FRM-0334
Action
mechanism: enzyme inhibitor/histone deacetylase inhibitor. FRM-0334 is an orally bioavailable, small molecule inhibitor of histone deacetylase (HDAC) enzymes that enhances gene transcription of the healthy granulin (GRN ) gene copy and may have the promise to positively affect neuronal survival for patients with a genetic variation of frontotemporal dementia (FTD) associated with a granulin deficiency (FTD-GRN). FTD-GRN is a rare, early-onset and rapidly progressive neurodegenerative brain disorder that can affect behavior, cognition, language and motor skills, due to a reduction in the progranulin protein. In FTD-GRN, mutation renders one of the granulin alleles incapable of producing normal levels of the precursor protein progranulin. Low production of progranulin is associated with the neurodegeneration that arises in FTD-GRN. By activating the remaining healthy gene allele, FRM-0334 may have therapeutic potential to partially reverse the loss of protein function in granulin-associated FTD.
Disease: frontotemporal dementia with granulin mutation (FTD-GRN)
Therapeutic area: Rare diseases - Neurodegenerative diseases
Country:
Trial
details: The randomized, double-blind, placebo-controlled, dose-escalating Phase 2a clinical trial will evaluate the safety, tolerability and pharmacodynamic effects of two doses of FRM-0334 in subjects with prodromal to moderate FTD-GRN. Primary endpoints will assess the safety and tolerability of FRM-0334 in subjects with the FTD-GRN genotype, as well as effects of FRM-0334 on plasma and cerebrospinal fluid progranulin concentrations.
Latest
news: * On February 10, 2015, Forum Pharmaceuticals, a biopharmaceutical company singularly focused on the development and delivery of innovative medicines to treat serious brain diseases, announced dosing of the first patient in a Phase 2a proof of mechanism clinical trial to evaluate its investigational therapy FRM-0334 in patients with the ultra-orphan rare disease frontotemporal dementia with granulin mutation (FTD-GRN). Forum anticipates enrolling subjects at multiple sites in the United States and Western Europe for the trial. The company has previously completed a Phase 1 study of FRM-0334 in Europe, where it was found to be well tolerated at all doses tested.
