On 16 July, the biopharmaceutical company Oryzon Genomics, which develops epigenetics-based therapeutics in oncology and neurodegenerative diseases, presented data from its ongoing Phase II clinical trial in Alzheimer’s disease (AD) at the Alzheimer's Association International Conference (AAIC) held in Los Angeles.
ETHERAL is a Phase IIa multicentre, multinational, randomised, double-blind and placebo-controlled study evaluating the safety, tolerability and preliminary efficacy of two doses of ORY-2001 in participants with mild-moderate AD. ORY-2001 is an oral and brain penetrant drug that may reduce cognitive impairment, memory loss and neuroinflammation.
The company presented data from the first 104 recruited from the 125 European participants (49% with mild AD and 59% with moderate AD). Safety data from these first 104 participants, being treated for more than 1 month, reported that ORY-2001 is safe and well tolerated so far. Furthermore, no significant safety issues have been reported from the 36 participants who have already passed the 6-month threshold of treatment.
An initial blind analysis on the first 33 participants who have completed the first 24 weeks of treatment was presented. Although some participants show disease progression in cognition and behaviour measures, others maintain baseline values or show improvement. Similarly, encouraging changes have been detected in cerebrospinal fluid levels of S100A9, a pro-inflammatory biomarker described as an important contributor to AD pathology. The initial blind analysis showed that only 6/33 participants showed a strong increase of S100A9 levels while all the others have maintained or significant decreased values.