Not A Competitor
I may be wrong on this (experts please advise), but from my reading of this report, the new, proposed Alzheimer’s drug will suppress cytotoxicity induced by beta-amyloid wastes. The molecule binds to a protein, FLN (filamin A), essential to beta-amyloid toxicity. It is also projected that the accumulations of this waste protein will be reduced or absent.
Additionally, it is proposed that the new drug also inhibits new deposits of both beta-amyloid and tau tangles, the two protein waste products universally understood to be the direct cause of all the consequent Alzheimer’s symptoms.
Lastly, PTI-125, the new drug, is claimed to be highly anti-inflammatory. Inflammation is a major factor in a host of neurological (and other) diseases.
Is PTI-125 promising? Looks to be, at least conceptually. I’ve not read (haven’t searched for) any of the background murine lab studies, so I can’t comment in any detail regarding molecular or physiologic mechanisms of actions, other than above, the binding to the FLN protein.
The drug is just going into an early, first-with-humans clinical trial, to test both safety and dosage tolerance, not efficacy. No human data are posted or, to me, known.
As with Anavex 2-73, I can surmise PTI-125 might have its greatest use as an Alzheimer’s prophylactic, a prevention.
But, here’s the difference between the two new drugs, as I see it. PTI-125 may effectively reduce or adequately inhibit the toxicities emanating from waste protein accumulations in neurons and in nerve tissues. And, it may also cause removal of those wastes. All well and good. So far, nothing (except Anavex 2-73, putatively) does that.
Anavex 2-73 gets at its job much earlier in the disease process, at the very start of it, when reaction-controlling enzymes (properly folded proteins) are no longer produced, a result of mitchondrial/endoplasmic reticular disconnection and chemical transfer difficulties (ATP, Ca++, perhaps others). That’s a distinct advantage: attack and conquer the problem at its start (early disease stage), not at the end, targeting solely the accumulated waste proteins.
With Anavex 2-73's restoration of proper, normal mitochondrial/ER connections, the effective and normal synthesis of health-maintaining enzymes is restored. Waste proteins are once again effectively removed before they accumulate and poison things in the neuron and nerves. Anavex 2-73 attacks the problem at the start of the disease process.
Could PTI-125 be a competitor to Anavex 2-73? If so, it will first have to demonstrate safety and tolerability. Then, in a large human trial, it will have to demonstrate efficacy.
But should all of that occur, I don’t see this as a suppressing Anavex competitor. Anavex 2-73 has a multitude of benefits beyond mitochondrial function. It crosses the blood/brain barrier with ease (unknown, yet, if this happens with PTI-125 — it may have to be injected). The combination of oral administration and penetration into nerves and nerve cells is a distinct advantage for Anavex 2-73. The soporific traits are profound, and highly desired in an Alzheimer’s treatment.
And, the safety profile, lack of disqualifying adverse events (side effects) is just superb for Anavex 2-73.
I’ve seen nothing that would cause me to sell a few of my AVXL shares to buy a few Pain Therapeutics shares. The Anavex story is stronger, better, and in a much later chapter.
