BIOCAD has initiated clinical studies for its original hemophilia targeted drugs

The biotechnology company BIOCAD has initiated clinical studies for the first Russian original gene therapies targeting hemophilia: ANB-010 for hemophilia A and ANB-002 for hemophilia B. Authorizations for the clinical studies were issued by the Ministry of Health of the Russian Federation.

The main aim of the clinical studies will be to evaluate efficacy and safety of the investigational drugs. It’s expected that ANB-010 and ANB-002 will be administered as a single dose, and their sustained positive effect will be lasting up to several years as minimum.

“Both products are next-in-class drugs and are rAAV vectors carrying the human clotting factor genes: FVIII gene in hemophilia A or FIX gene in hemophilia B. rAAV-based gene therapies are specific in a way that a single dose of such drugs usually allows to achieve a sustained therapeutic effect lasting for many years”, said Yulia Linkova, Vice President, Clinical Research and Development, BIOCAD.

Nonclinical studies of ANB-010 and ANB-002 have been already successfully completed and have demonstrated efficacy and safety in animals. Data on pharmacodynamics, toxicity and pharmacokinetic properties of the drugs were obtained in these studies.

Patients over 18 years of age with established hemophilia A (ANB-010-1/EDELWEISS study) or hemophilia B (ANB-002-1/SAFRAN study) who have not received prior gene therapies are eligible to participate in the clinical studies.

The clinical studies are conducted at specialized study centers in Moscow, St. Petersburg, Ufa, Novosibirsk, Nizhny Novgorod, Samara, Kirov, Syktyvkar and other Russian cities. It should be noted that until recently, no gene therapies for hemophilia have been approved in the world. The first such product was approved in Europe in summer 2022.

Currently most patients in Russia and worldwide must receive lifelong injections of recombinant coagulation factors up to 3 times a week, depending on the disease severity. Hemophilia is one of the most common severe hereditary disorders. In Russia, more than 8.000 people live with hemophilia A and B. Worldwide, this number is as high as hundreds of thousands of people. In 2022, the Russian hemophilia treatment market was valued at least 14 billion rubles per year (USD 206 mln).
With the launch of new therapies including gene therapy, the market may grow by 50% by 2030.

Detailed information on the clinical studies, inclusion criteria and the list of study centers will be available at the website ct.biocad.ru. If you have any questions about participating in a clinical study, please call the hotline: 8 (800) 511–00-37.