Special Report: Small cap pharma Actinogen (ASX:ACW) is on a mission to lead a “changing of the guard” in Alzheimer’s treatment through development of its lead drug Xanamem.

The leading Alzheimer’s treatment in use today was developed 25 years ago – and the last new drug approved by the FDA was way back in 2003.

Stagnation in the Alzheimer’s treatment space has scientists and researchers questioning where else to turn, prompting one of the world’s biggest drug companies Pfizer to exit the race for a cure earlier this year.

Consider that outlook against the market for cancer drugs. What was once a death sentence has in the last 20 years seen significant developments and advances in effective treatments, resulting in improved survival rates.

The major pharma players in the Alzheimer’s space have until now — for at least the past 40 years — focused on the amyloid hypothesis: the theory that a build-up of small sticky plaques of abnormal amyloid protein in between nerve cells of the brain can block the neurons from talking to each other.

As the plaques develop, loss of memory and learning abilities often occur, and this may gradually progress to loss of other functions, including immobility and difficulties with feeding or swallowing.

Despite hundreds of clinical trials of drug candidates targeting amyloid, none has proved successful.

That leaves a significant opportunity for new drugs with alternative targets to widen the scope.

“The failure of the drugs developed using the amyloid hypothesis necessitates a widened search for drugs with a different mode of action, including Actinogen’s,” a recent report from Life Science researcher NDF wrote.

“These high-profile drug failures have increasingly led researchers to explore other mechanisms where amyloid beta is perhaps an effect of the disease rather than a cause. The Cortisol Hypothesis may be one such approach that attracts more adherents as thought leaders in the field start to widen their horizons.”

Dr Bill Ketelbey, chief at Actinogen Medical
Dr Bill Ketelbey, chief at Actinogen Medical

A new approach

That brings us back to Actinogen’s lead drug Xanamem and its unique mode of action – targeting cortisol in the brain.  

Cortisol is often referred to as the body’s stress hormone and high levels may lead to long-lasting and wide-ranging health problems including impaired immune function, low bone density, high blood pressure, and high cholesterol.

Most significantly, high cortisol levels are toxic to the brain and are known to be associated with learning and memory problems, which may lead to cognitive decline and ultimately the development and progression of Alzheimer’s disease.

The evidence has been building for the cortisol hypothesis over the past 25 years, and most recently a 2016 study funded by the CSIRO showed a significant association between elevated cortisol and Alzheimer’s. The study encouraged the development of drug candidates that targeted lowering cortisol levels.

Actinogen’s two Phase 1 trials completed successfully in 2015, and now, its Phase 2 XanADu trial is well underway with patient enrolment already passing the halfway mark last month.

The XanADu study is being conducted at 20 study sites in the US, UK and Australia and tracks multiple efficacy measures alongside the widely-used Alzheimer’s disease Assessment Scale-cognitive subscale (ADAS-Cog).

XanADu is also one of the first studies to use the Alzheimer’s disease Composite Score (ADCOMS), a tool sensitive enough to measure changes in patients in the early stages of Alzheimer’s.

Too big to ignore

While Pfizer dropping out of the market rattled the industry, the growing global incidence of the disease is just too big for big pharma to ignore.

Despite the failures and the setbacks in the Alzheimer’s treatment space, those few drugs that have emerged as plausible treatments have struck gold.

Aricept, the leading treatment by Pfizer developed 25 years ago – and still in use today – has peaked at $5.4 billion in sales and Exelon and the Exelon Patch at just over $1.29 billion.

“Alzheimer’s is, prospectively, a $51.45 billion drug market globally… and is just too big to ignore,” the same report read.

“It may have been a drug developer’s graveyard since 2003, but Big Pharma has persisted in licensing and trialling candidates, as evidenced as recently as January 2018 when Pfizer, while announcing the closure of their neurodegeneration discovery labs, indicated that it was reallocating the capital to external alliances.”


This special report is brought to you by Actinogen.

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