It must take real guts, or maybe hubris, to try to do what Allon Therapeutics is attempting. This Vancouver, BC-based biotech company has designed a drug that is made to work unlike anything else on the market against some of the world’s major neurodegenerative diseases, like Alzheimer’s.
Scientists don’t really know what causes Alzheimer’s, and they don’t have a consensus on what is going wrong at the molecular level that leads to the heartbreaking loss of cognition and memory that an estimated 4.5 million people in the U.S. suffer from as they grow old. Quite a few biotech companies have failed with once-promising drugs for this disease—San Francisco-based Medivation (NASDAQ: [[ticker:MDVN]]), and Salt Lake City-based Myriad Pharmaceuticals (NASDAQ: [[ticker:MYRX]]) are a couple that leap to mind.
Yet Allon (UH-lahn) remains in the game, and CEO Gordon McCauley insists that things are still looking up.
“What I find fascinating about the standard of care today is that there is $6.5 billion of drugs sold to treat Alzheimer’s per year, and they all have one common characteristic. They don’t do much,” McCauley says. “They have nasty side effects, and provide some relief from symptoms for about six months. There’s a real opportunity for a therapeutic with disease-modifying potential.”
Allon (TSX: [[ticker:NPC]]) is placing its bet on what is known as the “tau tangle” hypothesis. For years, many researchers and drugmakers have been pursuing the notion that a buildup of amyloid beta plaques in the brain is the primary culprit causing Alzheimer’s. Break up the amyloid, or prevent it from building up, and you can treat the disease. That effort hasn’t borne fruit yet, McCauley says.
A dueling school of thought, which Allon has thrown its resources behind, says that key structural components of neurons, called tau, get broken up into fragments and form tangles as neurons die over time. That process happens when people don’t produce enough of a couple proteins called activity-dependent neuroprotective protein (ADNP) and activity-dependent neurotrophic factor (ADNF). Allon’s idea was to create a small peptide fragment, called davunetide, which is meant to restore that neuroprotective function. If it works as designed, the drug ought to prevent tau tangles from building up, and keep people’s minds sharp.
The Allon drug has been in some small trials to date, and McCauley characterizes them in a bullish way. The company ran a study that randomly assigned 144 patients with a mild form of cognitive impairment to get a low or high dose of the Allon drug or a placebo. This study didn’t look at the main patient population of people with mild-to-moderate Alzheimer’s, and it didn’t measure one of the main goals that’s usually required in an Alzheimer’s study by the FDA—what is known as ADAS-cog. Nonetheless, Allon says it is encouraged because the study found a statistically significant improvement
