- Axovant Sciences released a mixed bag of interim results from a Phase 2 trial of its dementia drug nelotanserin, showing that it is meeting some endpoints but not others.
- Nelotanserin aims to treat hallucinations in patients with dementia, particularly those with Lewy body dementia. Patients receiving the drug displayed significant improvement in motor function and performing daily activities as measured by the Unified Parkinson's Disease Rating Scale (UPDRS).
- The drug didn't have a significant impact on secondary outcomes, however, including visual hallucinations. Given the results of the study and its small patient pool of 11, Axovant has decided to expand recruitment and expects final results in mid-2017.
The company is planning to push the drug into Phase 3 testing, which it anticipates will begin in the second half of 2017.
"We are pleased with the preliminary results of this small pilot study which supports our belief that nelotanserin could be a promising investigational drug candidate for patients suffering from Lewy body dementia," Axovant's Chief Development Office Lawrence Friedhoff said in a Feb. 13 statement. "We plan to discuss with FDA and other regulators the parameters of a potential Phase 3 registration program while we await results from the full cohort of patients in this study."
Piper Jaffray analyst Charles Duncan sees the results as somewhat varied.
"First off, nelotanserin did appear safe/well tolerated, which was a non-zero risk in this population," he said. 'In addition, the study suggested some benefit on activities of daily living and motor function — not exactly what we were expecting, but if replicated could address a key unmet need in dementia with Lewy bodies especially. We didn't yet see improvement on visual hallucinations but longer dosing in more patients may be helpful here."
Axovant's nelotanserin isn't designed to be a disease-modifying drug. It is an oral, once-daily drug targeting the 5HT2A receptor, and aims to treat symptoms of Lewy body dementia including visual hallucinations, which can be extremely distressing for patients.
However, developing a drug for a complex disorder like dementia is still going to be high risk and challenging, and the slew of high profile failures may have made investors cagey — despite reporting at least some positive data, Axovant Sciences' value has dropped by over 6%.
Nelotanserin is also in development for REM sleep behavior disorder (RBD) in patients with dementia with Lewy bodies, with a readout slated for the second half of 2017, and will use the results from that analysis and the hallucinations study to set up a Phase 3 pivotal trial later in 2017.