DISCLAIMER: IC-MedTech makes no claims as to the safety or efficacy of its investigational new drugs. These drugs have not been fully evaluated or approved by the Food and Drug Administration. Therefore, these compounds are not intended to diagnose, treat, cure, or prevent any disease.
The Apatone® Platform
ICM was founded in 2004 to help explore anti-tumoral effects observed when certain quinones are combined with ascorbic acid. Profound, selective anti-cancer effects were initially reported by academic researchers in the 1980’s. Today there are hundreds of publications exploring the effects of quinones and ascorbic acid in cancer and other diseases. ICM has supported much of this research and these data offer insights for new drugs. Consequently, our research interest is in redox biology and our focus is on mitochondrial metabolism and signaling.
The concept of targeting cellular redox is based on sound science. But the pharmacology that’s paved the way to a better understanding is considered a financial orphan. That is, while a large and growing amount of data suggest this approach to treatment is safe and effective, the active pharmaceutical ingredients (API’s) used as a platform to study and demonstrate the mechanism do not fit the prevailing business model, nor do they readily capture the imagination of researchers.
There are many reasons. One, the intellectual property is more difficult to defend and considered marginal to justify significant investments needed for clinical development, drug approval and commercialization. Two, there is a stigma surrounding ascorbic acid’s role as a drug versus a vitamin. This is due in large part to controversy regarding its storied history and questionable claims of efficacy.
While high doses of intravenous ascorbic acid are widely administered in alternative medicine, therapeutic effects are relatively modest and vary widely. “Pharmacologic” doses of ascorbic acid have captured tens of millions in NIH grants, yet well-controlled clinical studies have yet to produce sufficient evidence to support regulatory approvals. We believe our approach is better.
Third, the cellular consequences of targeted redox modulation are pleiotropic. This does not fit the prevailing regulatory and business model which favors targeted drugs produced from new chemical entities. And fourth, the key compounds used to demonstrate the effects of targeted redox are widely available and inexpensive. These are not chemical entities the pharmaceuticals industry favor to derive sufficient profits. For these and other reasons progress has been slow but steady.
In spite of the hurdles IC-MedTech (ICM) has supported and encouraged research and development of redox combination drugs capable of targeting transformed and infected cells. We’ve funded basic research, meetings engaging the top thought leaders in redox biology, formulation development, and pilot clinical studies as regulatory pathways are identified. Along the way ICM has sponsored thousands of experiments.
Preclinical in vitro studies have demonstrated it is possible to safely slow, stop, and even kill solid and blood borne tumors. Animal studies confirm the effects can be translated in vivo with significant anti-cancer effects against cancers including difficult tumors such as glioblastoma. Three pilot clinical studies support safety, suggest efficacy, and clinical development is moving ahead.
In and outside of clinical trials, nearly 1,000 people have received the ICM lead compounds in some form. In this population there are strong indications of efficacy and ICM has been awarded four orphan drug designations together with our first commercial IND.
It’s taken years of research to get to this point, largely due to the complexities of redox biology and the challenges of formulating redox coupling chemistries to regulatory standards. Redox active small molecules are promiscuous and deceivingly difficult to formulate in a stable, bioavailable dose form to CMC standards. After years of failures and disappointing setbacks, manufacturing of the first ever GMP targeted redox drug produced under CMC guidelines for advanced clinical studies has only recently been accomplished. In addition, identifying an indication that affords an achievable regulatory pathway for the resources available is a formidable challenge.
While the costs, expertise and time required for commercial drug development are barriers that have slowed progress, IC-MedTech has accepted the challenge. We have also accepted the difficult challenge to accomplish this without significantly inflating the ultimate cost of the drug to the patient.