The first gene therapy drug was approved  on the Russian market on December 7. Neovasculogen (VEGF) will be used for treatment of peripheral arterial disease (PAD) and its complication critical limb ischemia (CLI). This drug is manufactured by Moscow’s company Human Stem Cell Institute  (HSCI). The company’s CEO, Dr. Isaev kindly agreed to answer my questions about this remarkable approval.
1. Dear Dr. Isaev, first of all let me congratulate you and your company with this notable approval. I think, this is very important for the whole gene therapy community worldwide. Tell us please about when Neovasculogen will be available on the market and where?
The new gene therapy drug will be available on Russian Federation pharmaceutical market in second quarter 2012 after the first batch certification process. HSCI has decided that Novasculgen will be available on ex-USSR pharmaceutical markets, and primarily it will be the market of Ukraine.
2. Can you tell us briefly about the results of clinical efficacy studies? Some other gene products for treatment of CLI have failed in the late phases of clinical trials (for example, Sanofi’s FGF-based gene product ). What are your thoughts on that?
We can announce 3rd phase clinical trial results. It was the 6 month comparative trial of unreconstructable peripheral arterial disease patients. Patients received Neovasculgen as a part of conservative therapy. So, the results of our trial showed good therapeutic effect that we observed in April 2011. Neovasculgen led to the development of new collateral blood vessels which was contrasted on angiography. The main endpoint, PFWD (pain free walking distance), detected to increase by 110% that showed statistically significant difference versus the control.
The main and second endpoints have been integrated in combined rates named «success/failure». The score “success” was granted in 94% of clinical observation. The investigators of HSCI used plasmid vector as the most safe. As a result, the adverse events, side effects and complications of Neovasculgen were not recorded.
Fibroblast growth factor type 1 (FGF1) takes part in enhancing of new blood-vessel formation and activates some steps of multilevel therapeutic angiogenesis. Sanofi-Aventis tested safety and efficacy of naked DNA plasmid that includes the gene encoding for human FGF1. The expectations of Sanofi-Aventis investigators did not meet that was announced last year. However, I have to point out that the Sanofi-Aventis drug had original construction with another active gene component and study protocol was primarily addressed to the prevention of major amputation. I believe that feasible and clear end point criteria is the most important issue for the first-in-class drug on its way to the market.
3. What was the regulatory path for this gene drug in Russia? Can you compare the requirements for such products from Russian regulatory bodies with FDA and EMA?
The regulatory path was full of different questions. Neovasculgen is a first-in-class drug. It stimulates a vascular growth (so-called “biological bypass” or “therapeutic angiogenesis”). There are no any drugs with the same action mechanism, but Russian regulators wanted to know, for example, pharmacodynamics of the VEGF expressed from the plasmid. Unfortunately these requirements are not applicable for this sort of a gene therapy drug. In general, the requirements of FDA and EMA for authorization of gene therapy drugs have much in common with Russian requirements – safety and efficacy.
4. What will be the market price for Neovasculogen? Will it be covered by medical insurance? Can foreign citizens get this treatment in Russia or purchase a product?
The price of Neovasculgen will be around 200 thousand of rubles (~ $6600USD) for treatment course. We hope that Ministry of Health and Social Development of the Russian Federation will include this socially significant drug in the medical reimbursement list for special groups of patients. Our company plans to submit an application form in special commission. The Foreign citizens suffering from PAD can get a treatment by Neovasculgen in Russian healthcare hospital or ambulatory.
5. What is the market penetrance for this product? How many patients could benefit from it?
Peripheral Arterial Disease affects an estimated 10% to 20% of all people over the age of 55, with prevalence increasing with age. Prevalence of PAD is also significantly higher, about 30% to 35%, in people with diabetes. Every year 30-35 thousands of amputations are performed in Russian Federation. The symptomatic PAD patient continues to have significant functional disability. HSCI believes that for many patients Neovasculgen may become a real alternative to amputation and can improve a quality of life.
6. What is your projection for Neovasculogen sales in Russia? Are you going to expand your sales outside of Russia and register the product, for example in Europe?
The Company expects the number of patients will be increasing in the course of 5 years up to 20 000 – to start with, in 2012 HSCI plans to initiate Neovasculgen market authorization in Ukraine.
7. What do you think about cell therapy of CLI? Could it compete with gene therapy market?
The experimental and clinical studies show that different cell therapy methods lead to different results of efficiency. The modern understanding of therapeutic angiogenesis mechanism assumes that gene therapy and cell therapy can improve to each other. Nevertheless we must remember that a cell therapy process is locally associated with specialized laboratory or equipment. Neovasculgen is a finished medical product which is convenient and easy for usage in practical public healthcare.
Thank you very much for the interview! I wish you good luck in this business!
Disclosure: I’m on editorial board of Russian scientific journal “Cell Transplantation and Tissue Engineering” published by HSCI. I don’t own HSCI stock and have no plans to initiate any positions within the next 72 hours.