Anavex’s Alzheimer’s Drug a Possible Game Changer After Patients Reported Remarkable Positive Response
April 27, 2016
By Mark Terry, BioSpace.com Breaking News Staff
New York-based Anavex Life Sciences Corp. ’s popped about 23 percent after two promising anecdotes about patients receiving its Alzheimer’s drug, Anavex 2-73, were reported.
Anavex’s drug, Anavex 2-73, is being administered to Alzheimer’s patients in Sydney, Australia as part of a Phase II clinical trial. Two patients showed what appears to be a remarkably positive response.
One patient is Pauline Stevens, who has been a pianist for 55 years. After losing her ability to play the piano three years ago, she was diagnosed with Alzheimer’s. After eight months on Anavex 2-73, she regained her ability to play the piano. Now 78 years old, Stevens told Economic Calendar, “I feel as though I can go and do things now that I couldn’t do. I am happy.”
Another patient, Valerie Lynch, an award-winning artist, had lost her ability to paint as part of her Alzheimer’s, which was diagnosed two years ago. Now 76 years old, Lynch has recovered her ability to paint and is preparing for an exhibition of her work. Her caregiver, Keith Everitt, told Economic Calendar, “It has improved her out of all sight. She is laughing and bubbly, she has absolutely blossomed since she has been on these tablets. Nobody can believe the difference in Valerie before the trials and now. It is a different person.”
So is Anavex 2-73 the long-awaited cure for Alzheimer’s? The answer is: it’s too early to say. And it’s important to point out that most of the 123+ Alzheimer’s drugs that have failed in clinical trials, failed in late-stage trials. Anavex 2-73 is only in a Phase II trial.
And there are other concerns with the trials. First, it’s a small trial, with only 32 patients. Secondly, all of the patients in this trial received the drug, so there is no control group receiving a placebo to compare it to.
“It’s looking good at this stage,” Stephen Macfarlane, the principal investigator of the trial told The Motley Fool. “What’s required is a much larger trial with a placebo control group to see if the changes we’ve noticed in this early study are actually real and pan out in a proper and well conducted study.”
Cory Renauer, writing in The Motley Fools, contacted an outside expert to discuss the trial. “What I learned was, they’re basically meaningless. James Hendrix, Ph.D., director, global science initiatives, at the Alzheimer’s Association, was quick to point out patients over the age of 65 often have several different health problems. When something else bothering them—think arthritis, asthma, even depression—is alleviated, some patients appear to have better cognitive abilities.”
Which isn’t to say that Anavex 2-73 isn’t going to be the drug the world is waiting for, it’s to say that it’s too early to say and the evidence to date is a little anecdotal and has not been rigorously tested. Which brings up another issue, not an uncommon one for late-stage clinical trials: money.
What the U.S. Food and Drug Administration (FDA) and other regulatory agencies worldwide are going to want to see, most likely, is a single clinical trial with at least two groups that have over 1,000 patients in both groups. It will be randomized, with half receiving the drug and the other half receiving the placebo. It will be blinded, meaning that the only people knowing who’s receiving the placebo or the drug is an independent data monitoring group. Which costs money, as does manufacturing the drug and organizing the trial.
Theoretically, the trial could start today, but Anavex probably doesn’t have the financing, although the recent stock jump probably gave them some. What would be expected in most situations like this is for a larger company with deep pockets to license the drug and continue trials.
Edward Vranic, writing for Seeking Alpha, is particularly concerned about this. He notes that realistically speaking the company may need $100 million to work with, and that even at its fastest route to approval, the company is several years away. Having taken a harder look at the company’s finances, he notes, “The company requires a lot of cash and is not going about it the best way for long-term shareholders,” and questions some of the companies that have invested in it.
Vranic writes, “The news about the elderly Alzheimer’s patients regaining abilities once thought to be lost forever as described in the first paragraph of this article has gotten the retail market engaged in the story, but as mentioned previously, institutional ownership of AVXL is only 2.8 percent. If the science had true basis beyond a very experimental stage at this point, the company should have been able to attract a real biotech venture capitalist to invest at least a part of the $10s of millions needed to keep the clinical trials and research going. The company’s inability to do so speaks to the lack of interest in its IP from big money.”
At least so far.
Although the anecdotes are compelling and a deeper understanding of what the company’s financial strategies might be of interest to investors, one other particularly interesting aspects of the Alvanex 2-73 is that it doesn’t function the way most Alzheimer’s drugs being tested do. That could be a positive thing—to date even the approved drugs aren’t all that impressive.
Most Alzheimer’s drugs are focused on eliminating or preventing the development of beta-amyloid plaques, or in some cases, tau proteins. Alvanex 2-73 acts through sigma-1 receptor activation and M1 muscarinic allosteric modulation. In Phase I trials, it seemed to show the ability to protect post-synaptic dendritic spines, as well as to reverse synaptic loss in hippocampal neurons.