- Axovant Sciences said Tuesday its experimental Alzheimer's disease drug failed to beat out placebo in a closely watched study, marking the latest setback in a long string of clinical failures for drug development in the field.
- Treatment with intepirdine, as Axovant's drug is known, didn't improve either cognition or daily living activities in patients with mild to moderate Alzheimer's disease when added to background treatment with donepezil.
- The study's failure — while not entirely surprising given the history of Alzheimer's research — sent shares in the company down more than 70% Tuesday. Axovant said it doesn't plan to invest further in developing intepirdine as a treatment for Alzheimer's, although studies in other indications will continue.
Investors in Axovant had pinned their hopes on the company's ability to find success where big pharma failed. The biotech, founded by Vivek Ramaswamy and now run by former Medivation CEO David Hung, acquired intepirdine from GlaxoSmithKline plc in 2014 for only $5 million after the drug's failure in mid-stage studies dimmed the British drugmakers's interest in further development.
But Ramaswamy, who left Wall Street to mine pharma pipelines for underappreciated assets, saw promise in the drug, taking Axovant public in 2015 with a $315 million initial public offering.
Over the past year, high-profile failures from Eli Lilly & Co., Merck & Co. and Danish drugmaker Lundbeck A/S helped to shift attention toward Axovant's efforts. Unlike most drugs developed for Alzheimer's that target the disease's hallmark amyloid plaques, Axovant's intepirdine is a 5-HT6 antagonist. Ramaswamy, and later Hung, believed combining intepirdine with the generic Alzheimer's drug donepezil could help improve cognition by spurring the release of a neurotransmitter associated with memory and alertness.
Success from Axovant's Phase 3 study would have meant potentially billions in sales opportunity, as the industry's continued failure to find an answer to the devastating disease has made it a dramatically underserved market.
Yet, intepirdine proved to be no better than placebo, failing to show any significant benefit on two scales used to assess Alzheimer's disease progression. On one, a measure of patient's ability to do daily activities, intepirdine's effect was essentially the same as placebo.
Speaking with analysts Tuesday morning, Hung said the data showed insufficient evidence of efficacy to justify further investment with intiperdine in Alzheimer's.
The CEO did sound a hopeful note on a Phase 2 study testing the drug in patients who have dementia with Lewy bodies. That trial will read out at the end of 2017, and Hung believes intepirdine could elicit a greater response than seen in Alzheimer's.
But the failure of MINDSET to show any signs of efficacy will erase much of the optimism in Axovant. In some ways, the setback echoes an ealier disappointment in Hung's career. While head of Medivation, Hung led the company's efforts to develop an Alzheimer's drug called Dimebon (latrepirdine), only to see the treatment miss its primary and secondary endpoints in a 2010 Phase 3 study.
Hung subsequently pivoted Medivation into oncology, where it found success with the prostate cancer drug Xtandi (enzalutamide) — later spurring Pfizer, Inc to buy the biotech for a princely $14 billion.
He'll have to work similar magic at Axovant. Hung said Tuesday the company began looking at potential assets to pick up several months ago and will continue to build its pipeline.
Axovant listed nearly $300 million in cash on its balance sheet, giving it the resources it will need to pivot from MINDSET's failure.
Editors' note: This article has been updated to reflect Axovant Sciences' share price as of close of market Tuesday.