Generium to Conduct Clinical Trials of Generic Drug for Treatment of Hypophosphatasia

The clinical trial approval will be valid until the end of 2025. A total of 115 healthy volunteers will take part in the study. GNR-104 is presented as a solution for subcutaneous administration at a dosage of 100 mg/ml.

As indicated in the entry from the State Register of Medicines, Generium attracted only one company from St. Petersburg, X7 Research, as the organization that will conduct the research.

Alexion first registered Strenzik in Russia in July 2019 and updated its registration at the beginning of 2024. Regions began purchasing the drug in 2019, and the Federal Center for the Prevention and Treatment of Diseases for the needs of the state fund "Circle of Good" - in June 2021. In 2024, 22 tenders were announced for the fund's wards for a total of about 5.4 billion rubles.

Hypophosphatasia is a rare genetic disorder in which the activity of the enzyme tissue-specific alkaline phosphatase is reduced, leading to impaired mineralization of bones and teeth.

According to the SPARK-Interfax system, Generium JSC was established in 2009 in the Vladimir region. The drug Lantesens (nusinersen) from the operator's portfolio, according to the analytical company Headway, is among the top ten drugs with the largest purchase volumes for the Circle of Good in 2024. During this period, the federal center purchased Lantesens for a total of 7.6 billion rubles. The drug is used to treat spinal muscular atrophy.

In February 2025, the biotech company received approval for phase I-II clinical trials of another drug that may be included in the Circle of Good procurement plans: the gene therapy drug GNR-097 for the treatment of Duchenne muscular dystrophy (DMD). According to information in the State Register of Medicines, the clinical trials should be completed by the end of March 2030. They will involve 42 pediatric patients with DMD. Since 2024, the gene therapy drug Elevidis (delandistrogen moxeparvovec) from the American Sarepta Therapeutics has been used in Russia to treat this disease. According to the state fund Circle of Good, about $189 million was allocated for the purchase of the imported drug in the second half of 2024.