On 3 April, the biopharmaceutical company Oryzon Genomics, which develops epigenetics-based therapeutics in oncology and neurodegenerative diseases, presented data from its two Phase IIa clinical trial of vafidemstat in Alzheimer’s disease (AD) at the virtual AAT-AD/PD 2020 conference. Vafidemstat (ORY-2001) is an oral and brain penetrant drug that may reduce cognitive impairment, memory loss and neuroinflammation.
REIMAGINE-AD is a single center and open-label Phase IIa study assessing the efficacy, safety and tolerability of vafidemstat in agitation and aggression in moderate and severe AD. 12 participants were recruited and treated for 2, 4 and 6 months with vafidemstat. The drug was safe and well tolerated. The treatment showed a significant clinical improvement in agitation and aggression after six months of treatment. A statistical improvement in memory and cognition (measured by the MMSE test) was detected in severe and moderate AD participants by month 2, but the improvement was not maintained at 4 and 6 months. The company noted that these data are preliminary and support further clinical investigations.
ETHERAL is a Phase IIa multicentre, randomised, double-blind and placebo-controlled study evaluating the safety, tolerability and preliminary efficacy of two doses of vafidemstat in participants with mild-moderate AD. The company made an oral presentation of the 6 month data. 117 participants were enrolled in Europe, of which 96 completed the first 6 months of treatment. The trial met its primary endpoint at 6 months as vafidemstat was found to be safe and well tolerated. Some inflammatory and neuronal-damage biomarkers were reported to be reduced in the participants who received the drug. However, preliminary efficacy analyses showed no significant differences in cognition between groups after six months of treatment. The ETHERAL study is still ongoing and participants are to be treated up to 12 months.