PTBI is gene therapy..and gene therapy does not work. Check out CLDN.
We Are Initiating Coverage of Anavex with a Buy Rating and $7.00 Price Target. Anavex has a novel drug technology with several applications in central nervous system (CNS) conditions and cancers. The first indication is Alzheimer’s disease, in which the company plans to start a phase I/II trial in late 2014/early 2015. Additional research has shown potential in Parkinson’s disease, depression, epilepsy, stroke, pain, and solid tumors.
The Lead Alzheimer’s Drug Has Unique and Synergistic Mechanisms of Action. Anavex Plus is a drug that combines the company’s proprietary Anavex 2-73 molecule with generic Aricept (donepezil). Anavex 2-73 appears to reduce the accumulation of toxic proteins within cells, a key event early in the process of cellular dysfunction and disease pathways. This is a differentiated mechanism of action from the typical approaches of prior failed molecules, which have not shown benefits in Alzheimer’s disease.
Synergistic Effect with an Established Drug. Preclinical trials of Anavex 2-73 demonstrated that it prevents cell death, protects against cell damage, and results in improved memory. Donepezil, the most widely prescribed drug for Alzheimer’s, works by extending the action of neurotransmitters in surviving brain neurons. Study data have shown that the combination of both drugs may provide an 80% improvement over donepezil alone.
An Alzheimer’s Therapy Would Serve a Large Market. There are an estimated 5.2 million people with Alzheimer’s disease in the US and 11 million worldwide. Disabilities accumulate for the remainder of patients’ lives, requiring increasing care and higher medical expenses, so any therapy that has a benign AE profile and benefits is welcome in the space.
Anavex’s Pipeline Is Based on Sigma Receptor Technology. The company’s drug discovery platform is based on interactions of the sigma receptor, an intracellular receptor that activates several key pathways in the cell. Turning these pathways on or off has potential
There was a seven-point improvement in cognitive response in a three-month period, and a 5.5 point improvement after 26 weeks (as can be seen on this chart from the company's corporate presentation in early February). Why is this so encouraging? This test is strictly focused on symptomatic, not actual treatment to inhibit the disease. This cocktail has the ability to provide symptomatic relief, but also may be used for disease modification. There is no drug like that today. There is more potential to this drug than just symptomatic relief, though. The "cocktail" had a much higher response rate than Aricept (generic-donepezil) as a standalone.
The company is at the stage now where it is getting ready to run human trials in a combined 1b/2a clinical study. This study will get underway the first half of 2014, and should be put in the place fairly quickly. The trial will run three months (this is the company's highest Delta so far), six months and on to 12 months. After this trial is over, the company will need to run one more trial before it would qualify for FDA approval.
If trial results from this first three months look promising, I would not be surprised if Big Pharma took notice of this little company.
PRAN drugs work to prevent a build up of beta-amyloid deposits (well, it didn't work)....AVXL is approaching this differently ?
is it the sigma-1 receptors ?
presentation is in the afternoon 3:30 PST I think..San Diego.
are you new to biotechs ? They will have to raise more $ to start phase 2/3 obviously. ALL biotechs raise money by debt offering, stock sale. Nothing new.
If phase 2A is good, stock will be 3-4x this price. They will then raise for next phase. But I bet they get a partnership (Pfizer?) before that.
that is all that matters.
its a 1 time 1 person issue.
SeeThruEquity Initiates Research Coverage on Anavex Life Sciences Corporation with a Price Target of $1.33. This was back in Nov 2014. Read the research in yahoo news for AVXL from Nov.
there is a reason why out of 100 alz drugs, this was most promising. With all bios, there is no guarantees...but this one is showing the most promise. With all bios, we buy based on the promise.
Is JUNO going to work ? Is BLUE going to work ? Is KITE going to work...multi billion dollar stocks, they have no guarantee they will work. JUNO hasn't even STARTED phase I yet !!
I see plans for a reverse split to get this into the 2-3 dollar range very soon. Momentum building.
read the posts from earlier...they were looking for CEO, and funding. They got both now. The drug is still the same.
Anavex Life Sciences Corp. ("Anavex", AVXL.OB) announced today that ANAVEX 2-73 has been named 2010's "most promising trial drug" by the editorial staff at Alzheimer's Weekly. The publication selected the ANAVEX 2-73 trial from a field of over 100 trials that treat dementias such as Alzheimer's and following the review of more than 10,000 articles. Staffers and clinical experts of Alzheimer's Weekly said that Anavex stands out when scrutinized against their key criteria for potential treatments, including small molecule drugs, oral bioavailability, possibility for disease modification, targeting multiple pathways, near-term availability and entering human trials.
ANAVEX 2-73 is the first of a new class of compounds that act through sigma-1 receptor agonism as well as muscarinic cholinergic effects and modulation of endoplasmic reticulum stress thought to trigger a series of intracellular effects which modify ion channel signaling at the mitochondrial level.