History of creation

25.11.2013

Hepatitis B is a worldwide public health problem. This potentially life-threatening infection affects a liver. The complications may result in the development of cirrhosis and cancer. More than 325 million people live with the hepatitis B virus and most of them are at risk of developing chronic liver pathology (HBV) and death.

About 686,000 people die annually from hepatitis B infection. This virus has spread across the whole world. Australian scientists from the Panacea Infarm company (the largest drug developper and researcher in Australia) decided to launch a large-scale research project based on their study according with hepatitis B death statistics. The aim of this project is developping an effective infection cure. The clinical trials started last month (10/20/2013).

The ambitious plans of Panacea infarm for 2013 year drew an attention among the highly specialized investors and doctors of science around the world. The best scientists who worked previously at CSL Behring company were invited to take part in this research. With a sinking heart, the patients who suffer from chronic hepatitis B are following the research on the up-to-date drug, that potentially might be a cure for the previously incurable disease.

The purpose of preclinical studies

It will take more than one month or even more than one year from the main active ingredient synthesis to the drug’s release at the market. The drug is currently going through a series of trials. The results will show how effective the new drug can be as a treatment of a specific pathology, can it be used at all and what side effects are possible. The new drug must withstand all clinical trials stage by stage.

The formula or recipe is not yet a drug. At first, scientists should figure out the required concentration of active substances and the optimal dose of the certain drug for achieving the maximum effect. Therefore, the main biological link in the series of trials are mice. The development of an effective medicine for treating hepatitis B is not a simple task. The drug should ideally correspond with the course of various biochemical reactions and change them respectively.

The first results revealed how many rodents survived after the initial trials. The group of scientists continues to work on defining and adjusting the required dose and the concentration of the drug’s active substances. The living processes of animals and the work of their internal organs are monitored for 12 months. The preclinical studies are necessary to find out if the drug can be tested on humans at all.

According to Mark Pellegrini, the head of the research team working on the project at Walter and Elsa Hall Institute in Melbourne (Australia), the active drug’s ingredients must destroy the liver cells infected with the hepatitis B virus without affecting healthy tissues.

In a few months of the daily trials, the major body functions were significantly recovered.

Usually, at the very beginning of these studies, the chemical and molecular drug formula is developed. Later the preclinical studies begin, including biological, microbiological, pharmacological, chemical, physical, and toxicological tests with the participation of laboratory animals. Then the form of a drug release is determined (tablets, injection solution, suspension).

Key phases of the clinical trials

The first phase. The tolerability and safety of the pharmacokinetic and pharmacodynamic features of a new drug are determined. The first phase of the clinical trials can be going during several weeks and months. Only a small percentage of drugs during the 1st phase will be eventually approved by the FDA, WHO and other organizations and later will be released at the global pharmacological market.

The second phase. The purpose of clinical trials at this phase — to define the optimal dosage of the drug to achieve the required concentration of active substances in a human body. Tolerability and efficacy are also compared with other drugs that were already defined as standard treatment. The placebo may be used for the same purpose.

The third phase. The pharmaceutical company that is responsible for the research and development confirms the safety and effectiveness of a new medical product. During this phase the registration dossier (new drug application) is created. This dossier contains full information on the method of use and the detailed results of the previous clinical trials. Then the following tests will be performed with the participation of humans.

The fourth phase. The volunteer groups take part in the clinical trials. Patients from all over the world are invited to become volunteers and test a new drug. The purpose of this phase is to give an objective review of the body’s reaction to the new drug in people of different races and nationalities. If a new medication successfully goes through all phases and is approved by the healthcare organizations, it will be approved for sale as a treatment method.

Then the information about the treatment duration, interaction with other drugs, tolerability by different age groups is gathered. If any dangerous complications are detected during the trails, this drug is withdrawn from sale. After the completion of all studies and trials, the medication is approved by the U.S. Department of Health and Human Services (HHS) and the World Health Organization (WHO). The drug receives an official trade name. After the drug’s approval, doctors may use it in medical institutions, according to the manufacturer’s guidelines.

Unlike most direct antiviral drugs, FABI256 completely blocks the RNA of pathogenic enzymes and the virus, in general. Thanks to its active ingredients, the recovery is possible even for the patients with cirrhosis, fibrosis and after a few types of hepatitis. FABI256 was developed based on the mechanism of action of its major predecessor called Birinapant, its development and release at the global pharmaceutical market was not successful.