There is a significant unmet need for Alzheimer's Disease. That fact, in combination with the fact that millions of people suffering from Alzheimer's and the projected growth rate for patients with the disease, creates a significant monetary incentive for healthcare companies to come up with solution for Alzheimer's. According to estimates from the Alzheimer's Association, the disease affects more than 5.4 million people in the U.S. and is projected to be diagnosed in as many as 16 million by 2050. That translates into a global market worth as much as $20 billion, according to some analysts, for an effective treatment for Alzheimer's. Multinational drug manufacturers such as Pfizer (NYSE:PFE), Eli Lilly (NYSE:LLY), and Johnson & Johnson (NYSE:JNJ) see the potential of this market and are heavily invested in Alzheimer's-related research. Separately, there is an under the radar microcap that is also heavily betting on making inroads into the Alzheimer's Disease market. That company is Anavex (OTCQB:AVXL).
Anavex Life Sciences is a biopharmaceutical company engaged in the discovery and development of new drugs for the treatment of neurological diseases and cancer, utilizing its proprietary drug discovery SIGMACEPTOR platform. The Anavex portfolio comprises novel, wholly owned sigma receptor agonists and antagonists. The company's lead drug candidate for Alzheimer's Disease (AD), ANAVEX 2-73, has successfully completed a Phase I single ascending dose (SAD) clinical trial. The company has also started scale-up manufacturing for ANAVEX 1-41, its second lead compound, targeting depression and AD. With sufficient quantities of ANAVEX 1-41 in hand, the company will be in a position to advance the program and begin preclinical studies on large animals in the near term.
The results for the Phase I trial for ANAVEX 2-73 were released late last year. In the Phase I study, the maximum tolerated single dose was defined per protocol as 55-60 mg. This dose is above the equivalent dose shown to have positive effects in mouse models of AD. There were no significant changes in laboratory or electrocardiogram (ECG) parameters. ANAVEX 2-73 was well tolerated below the 55-60 mg dose with only mild adverse events in some volunteers. Observed adverse events at doses above the maximum tolerated single dose included headache and dizziness, which were moderate in severity and reversible. These side effects are often seen with drugs that target central nervous system (CNS) conditions, including AD.
The results in the Phase I trial helped the product candidate gain national recognition. ANAVEX 2-73 was recognized by the independent publication Alzheimer's Weekly as "The number one most promising drug in Alzheimer's Disease" after their analysis of more than 100 trials that treat dementias and following the review of more than 10,000 articles.
Eli Lilly has been making noise recently in Alzheimer's after it released top-line and full results on Solanezumab Phase III Clinical Trials in patients with Alzheimer's Disease. In late August, the announced that the primary endpoints, both cognitive and functional, were not met in either of the two Phase III, double-blind, placebo-controlled solanezumab EXPEDITION trials in patients with mild-to-moderate Alzheimer's disease. However, a pre-specified secondary analysis of pooled data across both trials showed statistically significant slowing of cognitive decline in the overall study population of patients with mild-to-moderate Alzheimer's disease. Although the primary endpoints were not met, the market was impressed with the secondary analysis and sent the shares up over 3% on a big jump in volume.
The complete detailed results of the trial were released a month ago and both doctors and investors were encouraged. "It's certainly not the home run we all wanted, but we're very encouraged by these results," said Maria C. Carrillo, senior director of medical and scientific relations for the Alzheimer's Association. Investors sent the shares up 5% in response to the news.
News on the Johnson & Johnson and Pfizer Alzheimer's drug candidate has been mixed. In August, the companies announced plans to scrap further studies of their product candidate, bapineuzumab, after the drug failed to help patients with the memory-robbing condition in a second high-profile late stage clinical trial. The companies said they would discontinue all other studies of the drug bapineuzumab in its intravenous (IV) form, including two more late stage trials and follow-up extension studies, in patients with mild to moderate Alzheimer's.
However, the news turned around for the two healthcare giants in September after new data on bapineuzumab showed the treatment reduced underlying markers of the disease in some patients, suggesting the failed medication might work at an earlier stage. The biomarker results showed that bapineuzumab significantly reduced levels of the protein beta amyloid on the brain scans of patients with a gene mutation that increases their risk of Alzheimer's, compared with subjects who were given a placebo. The drug also significantly reduced the amount of a toxic form of the protein tau in spinal fluid, a sign of brain cell death, compared with patients who were given a placebo.
On a side note, in September, Medtronic (NYSE:MDT) announced its intention for Alzheimer's. The company said that it is seeking pharmaceutical industry partners to couple the company's pump technology with Alzheimer's medicines that may be more effective if pushed directly into the brain. "We have proof of concept and we're looking for partners," said Lisa Shafer, director of CNS Drug Delivery at Medtronic. Less than 1% of the intravenous drugs are able to cross the blood-brain barrier, a natural layer of protection in the vessels within the organ, Shafer said in a telephone interview. A product that can deliver drugs straight into the brain can yield higher and broader concentrations using 10-fold to 100-fold less medication, she said.