On 2 February, the biopharmaceutical company Cassava Sciences announced results of an interim analysis from its open-label study of simufilam (formerly PTI-125) in Alzheimer’s disease (AD). Simufilam is a small molecule drug targeting the altered form of filamin A found in the AD brain to restore its normal shape and function. This one-year, open-label and multi-center study is evaluating the long-term safety and tolerability of simufilam (100mg twice daily) in 100 people with mild-to-moderate AD. This study was initiated in March 2020 and is now approximately 80% enrolled.
The pre-planned interim analysis was conducted with data of the first 50 participants who have completed at least 6 months of treatment. Results showed that participants who received sumifilam had improved cognition and behaviour scores. Simufilam was found to be safe and well-tolerated. Adverse events were mild and transient. Based on these results, the enrolment target for this open-label study will be increased by up to 50 additional participants, to a total target of approximately 150 participants. In addition, the company plans to initiate a Phase III clinical program with sumifilam in AD in 2021.