Drug candidate PF-114 is a third-generation target drug developed by the Russian company Fusion Pharma and intended for the treatment of chronic myelogenous leukemia (CML) resistant to first- and second-generation drugs therapy.
Based on the results of the Phase I Multicenter Clinical Study, a dose of PF-114 with an optimal efficacy and safety ratio was selected in the target group of patients, which will be studied further in the Phase III Pivotal Clinical Study.
Pharmasyntez will conduct the Phase III Pivotal Clinical Study, register and commercialize the drug in the EAEU countries.
Ghermes Chilov, PhD in Chemistry, Chief executive Officer of Fusion Pharma: “The company has been the Skolkovo resident since 2011, and much of the work on the drug has been done in this status. We have been working on PF-114 for ten years, and we have gone through all stages – from drug design with molecular modeling to in vitro and animal studies, and finally, to human studies, after launching industrial production of the drug product. PF-114 has demonstrated the safety and efficacy in CML patients with resistance to previous therapy in a clinical study setting. But before launching the drug into the market, the Pivotal Clinical Study should be conducted in accordance with standards of pharmaceutical development. We hope that cooperation with Pharmasyntez will allow us to upgrade the project development."
Ksenia Tyumeneva, Head of Partnership Projects Department of the Pharmasyntez Group of Companies: “The development and launch of innovative drugs into the market is a strategically important area of development for the Pharmasyntez Group of Companies, in particular, there is a high interest in orphan and anticancer drugs. According to the agreement, Fusion Pharma grants us exclusive rights to manufacture and sell of the drug candidate PF-114. The available research results gave us hope that PF-114 would play an important role in the treatment of CML."
Vikram Punia, President of the Pharmasyntez Group of Companies: “The implementation of such an important project together with the Skolkovo Foundation improves the innovative capacity of the entire country. We carefully look for and select such “treasures” as PF-114 and we are ready to make large financial investments to launch the drug into the market, because we have no doubt about its mission. "
Kirill Kaem, Senior Vice President for Innovations at the Skolkovo Foundation: “Innovative pharmaceutics is one of the most complex, expensive and time-consuming areas of research activity. At the same time, the success rate for early stage projects is only 2-4% of the total. The very important thing is that the development of the PF-114 drug for the treatment of CML by Fusion Pharma has received recognition from our industrial partner, Pharmasynthez, and the signed license agreement, as a result of Pivotal Clinical Studies, will ensure patients' access to new therapy. We hope that Fusion Pharma team will move forward and will initiate the development of new drugs using the experience of working with PF-114."
Prof. Anna Turkina, Doctor of Medical Sciences, Head of Department of Chemotherapy of Myeloproliferative Disorders of the National Medical Research Centre of Hematology: “The Review Board that developed the design of the first Clinical Study of PF-114 included leading foreign experts in the field of CML, that is extremely important for the international recognition of the study results. To date, the drug has passed the Phase I Multicenter Clinical Study to assess the efficacy and safety in patients with chronic myelogenous leukemia with resistance and/or intolerance to two or more drugs, i.e. in patients with limited therapeutic options. We hope that PF-114 will give another chance to patients with CML when targeted therapies fail."
Prof. Olga Vinogradova, Doctor of Medical Sciences, Head of the Moscow City Hematology Center: “Our center joined the clinical study of PF-114, and it became clear pretty soon that the drug could be a real option for CML patients with resistance or intolerance to previous therapy. It is especially pleasant that this is a domestic development. Our center is interested in active participation in the Phase III Clinical Study for PF-114”.
Reference Information
Fusion Pharma was founded in 2010 by a group of scientists, with expertise in Medical Chemistry and Computer-Aided Drug Design, and private investors. The company is focused on the development of an innovative drug for the treatment of resistant forms of chronic myelogenous leukemia, including the T315I mutation. Since 2011, the company has been acting as a resident of the Skolkovo Foundation.
The Skolkovo Foundation is a noncommercial organization established in 2010 and acting as the managing company of the Skolkovo Innovation Center. The purpose of the Foundation is to support technological entrepreneurship in Russia and commercialize the results of research activities. The number of Skolkovo residents has already exceeded 2,700. The Foundation provides them a range of services necessary to introduce developments to the Russian and international markets. The resident status gives the company tax relief and customs privileges. In 2019, the residents' revenue exceeded 100 billion rubles and the amount of investments they attracted exceeded 13 billion rubles. Since 2019, the law allows any organization engaged in research activities on the territory of Russia to gain access to all services and benefits of the Innovation Center. The territory of the Skolkovo Innovation Center includes the Skolkovo Institute of Science and Technology (Skoltech), which is one of the world’s top 100 young universities in the world according to Nature Index. The infrastructure of the Innovation Center includes, in particular, the largest Technopark in Europe and research centers of industrial partners. In 2019, three new partner R&D centers of Sibur, TMK and Tatneft were opened in the Innovation Center. A total area of residential areas, research and educational centers that have been commissioned on the territory of Skolkovo is 750 thousand square meters. Extra-budget investments in infrastructure development exceeded 100 billion rubles. Website: sk.ru.