It has the backing of a young, media-savvy entrepreneur and was earlier this year helmed by the maestro of biotech CEOs, David Hung, and with a $2.6 billion valuation, all seemed bright for a company hoping to reverse the fortunes of Alzheimer’s R&D.
But what matters in biotech is data, and Axovant, following the pattern of flops of the past decade, is now just another company that tried and failed in a space that has caused nothing but big disappointments in R&D.
In a statement posted this morning, near the end of the month it guided for its release, Vivek Ramaswamy’s biotech says its phase 3 Mindset test failed to hits its primary endpoints with its investigational drug intepirdine in patients with mild to moderate Alzheimer’s disease (AD).
The drug was being tested in patients who were taking an older AD med, donepezil. At 24 weeks, patients treated with 35 mg of intepirdine “did not experience improvement in cognition or in measures of activities of daily living as measured by the Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) and by the Alzheimer's Disease Cooperative Study-Activities of Daily Living scale (ADCS-ADL), respectively, compared to patients treated with placebo,” the biotech says.
And after 24 weeks of treatment, change from baseline in cognition was “non-significantly improved in the intepirdine arm versus the placebo arm,” and there was essentially no difference between the intepirdine and placebo arms in change from baseline in activities of daily living.
Of the endpoints analyzed to date, the only endpoint in which any significant improvement was seen in the intepirdine arm versus the placebo arm was in the first key secondary endpoint, the Clinician Interview-Based Impression of Change plus caregiver interview. This is not enough, however, to save the drug. Axovant says it will work with investigators to “conclude” the Mindset open-label extension study.
“While we are deeply disappointed by these trial results, we also are saddened for the millions of patients and families impacted by Alzheimer's disease. However, we believe that the fight against Alzheimer’s and other important areas of unmet need in neurology is too important to be derailed by this setback,” said Hung, who left Medivation after its $14 billion sale to Pfizer for Axovant, seeing a spike in the biotech’s shares and belief his arrival may signify strong data.
Hung added that Axovant remains “committed to advancing our pipeline.” This includes its phase 2b study of intepirdine, and ex-Arena med nelotanserin, the highly selective inverse agonist of the 5-HT2A receptor in phase 2, both of which are being evaluated in patients with dementia with Lewy bodies.
This will be a major hit not just for the company but also for the Alzheimer’s community in general. It could also leave some open questions about the rest of Ramaswamy’s “Vant” family of biotech companies. Axovant was down a painful 71% in early premarket trading.
To its credit, the company did not try to play this up as anything other than a failure. A statement from Roivant, the parent company of the biotech, said: "Alzheimer's disease is one of the most difficult areas of drug development. We are proud of the tireless efforts of the team at Axovant who conducted the Mindset trial in the face of those challenges. Today's results are a setback for our company, and more importantly for patients with Alzheimer's disease. But it is not the end of the road. Axovant remains committed to future research in this area, and we are rooting for all companies who share that commitment."
Intepirdine, a former GSK med, has always been considered a high-risk project after similar drugs developed by Pfizer and Lundbeck failed late-stage trials.