During my mothers visit I had an opportunity to speak to the Orthopedic surgeon involved in the study. I asked him what precipitated his involvement in the drug study, he quickly corrected me and reiterated it is a biologic and not a drug. (Somebody please tell me the difference.) He explained that many of his patients prefer to try conservative treatment before surgery. He was honest in acknowledging Hyaluronic acid is ineffective but patients are willing to try it regardless of its inability to alleviate pain or provide sustain relief. He said people in general would like to avoid surgery if possible. He went on to state he has limited options and not many alternatives to surgery and is looking for an alternative.to Nsaids , Ibuprofen and Steroids. . He said steroids provide temporary re leaf however they have been shown to adversely effect the knee joint and cause destruction of the bone. He believes the application for the Ampion is far greater for tendons and ligaments where steroids really compromise the strength of soft tissue and possibly induce ruptures of the ligaments. I have never witnessed a doctor so exited about the possibilities of potential applications. It was somewhat confounding because I thought they earned there living operating . It gave me a different appreciation for surgeons.
There appear to be multiple mechanisms of action. Part of the negative feedback loop, down-regulating pro-inflammatory cytokines in non-naive T-cells. It also decreases expression of class 2 MHC receptors & decreases vascular permeability, thereby slowing the rate of infiltration of immune cells to the inflammation site.
A biologic is a drug, just not a xenobiotic. Therefore, less potential of unknown adverse effects. Please remember that the FDA considers even KCl, NaCl, MgSO4 and dextrose as "drugs". The surgeon probably was using a less technical definition of "drug".
Biologics have 2-fold greater chance of approval than synthetic (small molecules). Still, from Phase II to approval, only 37% make it. (vs 21% for synthetic drugs). Data from a 2010 article by Dimasi, et al Clinical Pharmacology and Therapeutics, 87, 272. This success rate applies to repurposed drugs as well, since efficacy for new indications is generally not established with human data. Viagra is a well classic example of using clinical data to derive a new use. Most repurposed drugs still rely on in vitro and animal model data for the 501(b)(2) status. So, one hopes that analysts make reasonable valuations by properly risk-adjusting projected top line revenue prior to discounting cash flow for NPV.
Here's what I understand is the difference between a drug and a biologic - and how I think Ampion works. I am a layman, so take this with a grain of salt.
A biologic is a compound that is developed in the body - or manufactured in the body in the normal course of events. This is distinguished from a compound that never appears naturally in body - a drug. Drugs typically can have some effects on the body that are not readily known or knowable. A biologic typically doesn't have as many unknown consequences. However, an amount of the biologic in excess of what is normally in the body can have some consequences.
In the case of Ampion, the body regularly manufactures a cyclic-dipeptide from the cleaving of the amino acids on the n-terminus of albumin. This di-peptide (Ampion) apparently has anti-inflammatory effect in the body. However, as I understand it, when there is "dis-ease" (an abnormal state in the body, such as osteoarthritis or many, many other ailments that cause inflammation) there is an insufficient amount of made by the body. Administration of Ampion enhances and multiplies what the body produces to fight inflammation.
Despite being a biologic, the cyclic-dipeptide will be manufactured matching that within the body.