DISCLAIMER: The research supporting Apatone is ongoing. IC-MedTech makes no claims as to the safety or efficacy of Apatone. The drug has not been fully evaluated or approved by the Food and Drug Administration. Therefore, Apatone is not intended to diagnose, treat, cure, or prevent any disease.
The Apatone® Development Project
Apatone is a small molecule combination based on the synergistic action of two well-known pharmacological agents, menadione and ascorbic acid. (Vitamins K3 and C) Administered to animals for more than 60 years, safety is well established. These small molecules have long been approved by the FDA. Therapeutic effects from the vitamin combination were first observed by academic researchers examining bioenergetics, cancer and other diseases. There are now more than one hundred publications presenting results on this approach, termed Apatone.
Menadione is available as a human dietary supplement in many other countries. However, at doses 1,000 to 10,000 times higher than contained in Apatone, menadione can be toxic. For this reason, in the US, menadione is regulated by the FDA. It is unavailable to the public and unapproved in combination with ascorbic acid as a drug.
IC-MedTech has sponsored development of Apatone as a drug, and has submitted pre-clinical and clinical results to the FDA. The drug has recently been awarded four orphan drug designations and is considered safe for advanced clinical study.
Research demonstrates that diseased cells often exhibit intracellular reducing environments. This is counter to the long-held belief that cancer cells are more oxidizing relative to normal cells. While this is true extracellularly, cancer cells up-regulate intracellular anti-oxidant pathways that protect them from oxidative stress. Consequently, cancers and many human diseases have been found to respond to pro-oxidant treatments.
Although the Apatone Development Project is based on academic discoveries, the technology is considered by industry as a financial orphan. That is, it does not fit the pharmaceutical industry business development model, chiefly for lack of new chemical entity intellectual property. For this reason, IC-MedTech is funding the research and clinical drug development as funds and reasonable regulatory pathways become available. In published early phase clinical trials, orally administered Apatone produced no serious drug-related adverse events and is considered by several independent clinical investigating groups to be safe and potentially efficacious in humans.
Considering the risk/benefit relationship, we believe in the potential importance of Apatone to satisfy unmet medical needs. IC-MedTech has raised money from private investors and donors and recently spent over a million dollars developing a new tablet dose form, together with the supporting analytical methods that are required by the FDA for advanced clinical study. It is the first investigational new drug of its kind aimed at utilizing redox modulation as a treatment rationale. After years in development, the non-trivial tablet program is expected to yield tablets that meet CMC and cGMP standards. It’s finally expected to be available for advanced clinical study in early 2018.
In preclinical efficacy studies conducted in vitro it has been shown that Apatone slows, or even stops growth of transformed cells. Animal studies have confirmed the effect and clinical studies in cancer, non-cancerous neoplastic diseases and inflammatory disease suggest this pharmacologic approach offers promise. Assuming that its activity is confirmed, the eventual goal is to add Apatone to existing protocols in order to improve treatment outcomes over a wide range of diseases. However, the costs, expertise and time required for commercial drug development, including pivotal registration clinical trials required to comply with the regulatory authorities, are a challenge. IC-MedTech has accepted that challenge and an equally difficult challenge to accomplish this without significantly inflating the ultimate cost of the drug to the patient.
We have recently licensed Apatone outside the US and plan to use the licensee fees and royalties to further this important research and regulatory development effort. We invite proposals from those interested and capable of helping to advance Apatone through regulatory approval.