Progressive analogue: trials of a drug for a dangerous blood disease have been completed in Russia

Russia has completed the third phase of trials of a new drug for patients with immune thrombocytopenia. This is a more affordable biosimilar of a foreign drug, which is expensive in the Russian Federation. In people with this disease, the body destroys its own platelets, bruises appear and sudden bleeding occurs. Experts note that the Russian equivalent will be in demand. For more information about the results of drug testing, see the Izvestia article.

What is immune thrombocytopenia?

Researchers from the ITMO University Master's Degree program in Public Health Sciences, together with the biotechnology company Geropharm and other colleagues from Russia, have completed the third phase of trials of a new drug for patients with immune thrombocytopenia.

Immune thrombocytopenia (ITP) is a rare disease in which the immune system attacks and destroys platelets, that is, blood cells that help stop bleeding. A person with ITP can often bleed from the nose, bruises take longer to heal, and hemorrhages in the skin and mucous membranes are possible. In addition to creating physical discomfort, the disease also affects daily life: patients face anxiety, activity restrictions, and difficulties with treatment.

Despite the fact that there are few cases of ITP, the disease has been well studied, and modern drugs allow patients to live a full life, the scientists said. One of these drugs, enplate (also known as romiplostim, or synthesized protein AMG531), is included in the treatment recommendations of the Ministry of Health of the Russian Federation and the list of essential medicines in Russia.

However, the original enplate drug is considered expensive. Therefore, pharmaceutical companies are developing biosimilars, analogues of original biological products that have the same effectiveness and safety, but are much cheaper.

For example, a Russian pharmaceutical company has synthesized a new biosimilar of romiplostim, the GP40141 protein. A group of scientists, including researchers from ITMO, presented the results of the third and final phase of drug trials.

The study involved 136 patients from 16 medical centers in Russia. The participants were divided into two groups: one of them received the GP40141 biosimilar, and the other received the original enplate drug. The main purpose of the study was to evaluate how effective the new drug is and whether it helps to achieve a normal concentration of platelets in the blood.

After the tenth week of therapy, a positive response (i.e., the required platelet count) was observed in 78% of patients in the biosimilar group and in 85% in the original drug group. In addition, during the experiment, no significant differences were found in other key performance indicators, including stability and duration of action of the drug. The new drug also turned out to be safe, the frequency of side effects was the same as that of the original drug, while all adverse reactions were mild.

The scientists noted that the study also confirmed the low immunogenicity of the drug: protein-neutralizing antibodies were produced in only a few patients and even in these cases did not affect the overall effectiveness of therapy.

— This study is interesting because it involved patients who were already on enplate therapy, and within the study they were transferred to biosimilar therapy. The use of an unusual methodology allowed us to obtain not only data on the effectiveness of the biosimilar, but also data on the safety of switching from the original drug to an analog. The transition did not change the effectiveness and safety of the therapy," said Ekaterina Protsenko, a graduate student of Public Health Sciences at ITMO, a medical scientific adviser to R&D at Geropharm.

What is the difference between the Russian biosimilar

The researchers also managed to introduce two important innovations. First, the scientists used in silico simulation (a study based on computer models) to justify the endpoint and calculate the sample. This allowed optimizing the design of the study, reducing its duration and making it more economical.

— Secondly, for the first time, we included in the study patients who had previously received another TPO agonist, eltrombopag, and showed that even with a weak response to previous therapy, a good response to a drug change is possible. This opens up new possibilities for the treatment of complex patients," Vladislav Kovalik, a graduate of the Master's degree in Public Health Sciences, told Izvestia.

The new biosimilar has already received a registration certificate, and patients will be able to receive the medicine free of charge under the state program.

The development is promising and in demand. This is a biosimilar of a drug that "pushes" the bone marrow to make more platelets, reducing the risk of bleeding in immune thrombocytopenia, Albert Rizvanov, head of the Center for Excellence "Personalized Medicine" at Kazan (Volga Region) Federal University, corresponding member of the Academy of Sciences of the Republic of Tatarstan, told Izvestia.

— In a multicenter phase III study, it showed comparable efficacy and safety to the original, and in Russia it has already been registered and included in the list of VED. This means that the drug will be available under government programs for chronic ITP and long-term courses. This guarantees steady demand," the specialist said.

The successful testing of a Russian drug in such a complex and high-tech field as the treatment of autoimmune hematological diseases underscores the maturity of the Russian pharmaceutical industry and the sustainability of the healthcare system, Andrey Varivoda, an expert at NTI Helsnet and Chairman of the Board of Directors of the Deacon Group of companies, told Izvestia.

— For a healthcare organization, this means a double win. Firstly, we see the high level and transparency of the conducted clinical trials. Secondly, the availability of an effective analogue makes it possible to guarantee uninterrupted therapy and, quite likely, optimize budget expenditures. The released funds can be used to develop other areas, including early diagnosis and treatment of other rare pathologies," the expert noted.

The work was published in the eJHaem journal.