There is very little doubt that developing drugs for neuroscience disorders in general and dementias in particular is a high-risk business. Axovant Sciences, unfortunately, seems to be finding that out the hard way.
There is very little doubt that developing drugs for neuroscience disorders in general and dementias in particular is a high-risk business. Axovant Sciences, unfortunately, seems to be finding that out the hard way.
The company announced today that its Phase II clinical trial of nelotanserin for the treatment of REM sleep behavior disorder (RBD) in patients with Lewy body dementia (LBD) failed to meet its primary efficacy endpoint. The endpoint was reduction in frequency of RBD events as measured by sleep laboratory video.
“While secondary measures of efficacy suggest biologic activity for nelotanserin, Axovant has been focused on developing innovative gene therapies and we will not undertake further clinical studies with our legacy small molecule portfolio, including nelotanserin,” stated Pavan Cheruvu, Axovant’s chief executive officer. “The completion of this study closes a chapter in the company’s history. We are grateful to the patients and clinical investigators who participated in this study, and we look forward to advancing Axovant’s gene therapy pipeline through multiple important milestones in 2019.”
Axovant is under Vivek Ramaswamy’s Roivant Sciences’ umbrella. Broadly speaking, Ramaswamy’s business model is to acquire cast-off drugs from larger pharma companies to develop them in smaller, more focused or “other” indications. At least in Axovant’s case, this isn’t working well.
Most notoriously—and one suspects Axovant and Ramaswamy wish the media would stop bringing it up—Axovant’s first drug, intepirdine, failed a Phase III MINDSET clinical trial in Alzheimer’s disease in September 2017. Ramaswamy and Axovant bought the drug for $5 million from GlaxoSmithKline. It showed a favorable safety and tolerability profile, and even demonstrated immediate and sustained efficacy over placebo in a Phase IIb trial.
But GSK had run four clinical trials, all failures, and abandoned the drug. Axovant studied the drug in 1,315 patients on a stable background therapy of donepezil (Aricept) and received either intepirdine or a placebo. But the drug failed to meet its co-primary efficacy endpoints. The company stated at the time, there was “essentially no difference between the intepirdine and placebo arms in change from baseline in activities of daily living.”
The company then spent the next six months reorganizing. In May 2018, it announced these changes. David Hung, the founder, president and chief executive officer of Medivation, had taken over Axovant in April 2017 after Pfizer acquired Medivation in September 2016. He stuck around until the intepirdine failure, then left.
Gavin Corcoran joined Axovant as executive vice president of Research & Development, and Michael Hayden was appointed senior scientific advisor and chairman of the Scientific Advisory Board. Cheruvu came on as chief executive in February.
The Axovant restructuring was also part of an overall Roivant restructuring, where more of the companies under the Roivant umbrella shared resources.
In June, 2018, Axovant licensed OXB-102, now called AXO-Lenti-PD, from Oxford BioMedica. The drug is an investigational gene therapy for Parkinson’s disease, designed to deliver three genes that encode a set of enzymes required for dopamine synthesis in the brain.
The company indicates that since it licensed OXB-102, it has been focused on gene therapies. It is currently in the SUNRISE-PD Phase II clinical trial with data expected in March 2019. Axovant is also developing AXO-AAV-OPMD, a gene therapy using Silence-and-Replace technology to restore normal muscle function in patients with oculopharyngeal muscular dystrophy (OPMD). It expects to launch a clinical trial for that therapy in the second half of 2019.
Meanwhile, today’s news was met with a 27 percent drop in stock.
