Pharmsinthesis: Vamotinib

Main article: Leukemia (leukemia, blood cancer)

Vamotinib, a Russian drug developed for the treatment of chronic myeloid leukemia in patients with resistance, including T315I mutation, and intolerance to previous therapy, is an oral tyrosine kinase inhibitor.

History

2025: Presentation of "Vamotinib"

Pharmasintez is preparing to register and put into civilian circulation the drug product Vamotinib. Vamotinib, the first original fully developed and manufactured 3rd generation tyrosine kinase (TKI) inhibitor in Russia, is a highly selective TKI for the treatment of patients with chronic myeloid leukemia (CML) who are resistant to previous therapy. The company announced this on April 25, 2025.

Chronic myeloid leukemia is a rare and serious tumor disease blood that occurs due to genetic breakdown, when parts of the 9th and 22nd chromosomes change places, which leads to the appearance of the Philadelphia chromosome in the cell as a precursor to hematopoiesis, which leads to uncontrolled proliferation of tumor cells and development. Every leukosis Russia year, about a thousand new patients with CML are identified. This disease accounts for about 15% of all leukemias in adults. The peak incidence falls on a workable, socially active age of 50-60 years.

Before the advent of tyrosine kinase inhibitors, the diagnosis of chronic myeloid leukemia sounded like a sentence - most patients lived only 3-5 years. However, in 2001, the drug imatinib (the first 1st generation tyrosine kinase inhibitor (TKI)) appeared, revolutionizing the treatment of CML. With the introduction of 1st generation TKIs into clinical practice, and later 2nd generation TKIs (dasatinib, nilotinib, bosutinib), CML has passed from a fatal to a potentially treatable disease, and about 90% of patients with CML live for decades.

Tyrosine kinase inhibitors have made a breakthrough in the treatment of CML, but a quarter of patients with TKI develop resistance associated with the absence or achievement of an insufficient response or with a loss of effect on therapy over time.

Patients with CML receive TKI on a continuous basis, which allows disease control, therefore, ensuring the safety of patient therapy and preventing severe life-threatening complications is extremely important. An important unresolved medical problem in addition to the development of resistance in the treatment of CML is TKI intolerance and safety.

Differences in safety between TKIs seem to be significant. Various TKIs, along with the general toxicity profile, for example, hematological and cutaneous toxicity, demonstrate specific types of complications characteristic of a particular drug. A specific type of toxicity can be pleural effusion, pulmonary hypertension, cardio-vascular complications, hepatotoxicity, occlusive lesions of the arteries, increased blood glucose, pancreatitis, dyspeptic events (diarrhea, nausea, vomiting). The development of adverse events significantly affects the quality and life expectancy of CML patients. Ensuring safe therapy becomes a priority in the current stage of CML therapy.

The Phase 1 clinical trial and the data obtained in the Phase 3 clinical trial have shown that vamotinib has high efficacy and demonstrates high specific safety, including cardio-vascular, making it a promising solution not only for patients with resistance, but also with intolerance to previous therapy.

As of April 2025, the drug is at the stage of the third phase of clinical trials to confirm its effectiveness and safety in a wider group of patients. Interim analysis data support high efficacy and a favorable safety profile.