Verto’s Lupusorb™ Column: A Novel Device for the Treatment of Systemic Lupus Erythematosus.
- Prof. Yaakov Naparstek, MD., CSO and Founder
- Stuart Bernstein, COO
- Keren Saadon, M.Sc., Clinical Trial and R&D Manager
Sector and Field of Activity
Verto’s scientists discovered that the target self-antigen is a short peptide located in the C terminal of laminin, a major component of the extracellular matrix that lines all body organs. The peptide is termed VRT101. Verto has demonstrated that specific inhibition of the binding of these antibodies on the target organs ameliorates the disease in animal models. This provided the rationale for two potential treatments of Lupus – by injection of the peptide as a drug and by extracorporeal immunoadsorption via plasmapheresis.
Market
SLE is a chronic, often life-long autoimmune disease affecting millions of people worldwide. There is no drug treatment specific to lupus. The current therapy of lupus is based on corticosteroids and cytotoxic agents, which suppress the over-reactive immune system, and can lead to severe systemic infections that are a major cause of morbidity and mortality in lupus patients. These drugs have other severe side-effects, such as bone fractures, early arteriosclerosis, malignancies and infertility.
The systemic lupus erythematosus (SLE) market is about to enter an era of targeted biological therapies. A greater understanding of the pathogenesis of the disease has led to considerable development of biologic therapies that will provide therapeutic intervention at a number of targets - B-cells, T-cells and cytokines. The current pipeline consists of 25 products in total, about a third of which are in Phase III development. Genentech’s Rituxan (Rituximab) was the most advanced Phase III candidate for SLE, but failed to demonstrate clinical efficacy in SLE patients. The product is already used off-label in SLE patients’ refractory to standard therapy—with potential use in around 1% to 10% of lupus patients—leading to sales in the range of $125m–$650m.
Datamonitor estimates that the prevalent population in the seven major markets (7MM) is around 430,000 in 2008 (by some estimates even higher) and is set to experience minimal growth in these markets. By 2018, the SLE population will reach 450,000; representing a CAGR of 0.5% based on population growth. There is much greater patient potential in the emerging markets because of racial differences in disease prevalence. The combined SLE patient population in Mexico, China and India is estimated to be between 0.9 and 2.2 billion in 2008, suggesting companies should seek product approval in emerging markets, as well as in the EU and the US. Assuming that about 30% of SLE patients are candidates for the Lupusorb™ treatment and assuming a market penetration of 25% at 10 treatments per year at a price of $1,000 per treatment, the Luposorb™ column has a market potential of approximately $320M in the 7MM alone, without including the emerging markets.
Intellectual Property Status
Verto holds two worldwide patents protecting its unique peptide for therapeutic and diagnostic applications. A third patent for the use of VRT101 peptide in a plasmapheresis column is in process.
Development Status
A Single-Arm Open-Label Clinical Trial with Lupusorb™ Immunoadsorption Columns in Systemic Lupus Erythematosus (SLE or lupus) patients.
The company has completed a single-arm open-label clinical study to determine safety and the ability of the Lupusorb™ column to reduce the circulating levels of antibodies associated with SLE disease. Ten SLE patients were recruited for treatment with a single Lupusorb™ immunoadsorption session during routine plasmapheresis procedure. The Lupusorb™ immunoadsorption column is an affinity adsorption column comprising R38 (VRT101) peptide. Patient screening prior to enrollment into the study was between 3 months up to 7 days prior to the day planned for plasmapheresis. Patients were enrolled into the study on the plasmapheresis day and underwent an average time of 2 hours of treatment with the Lupusorb™ column. The patients were then followed up for 8 weeks after the Lupusorb™ column procedure.
The results indicated a statistically significant decrease in the level of disease-related antibodies in the patients’ blood (anti-VRT101). The antibody level remained low for three weeks and only than increased to lower levels than pre-treatment, therefore, no rebound effect was observed (in which the antibody level exceeds that before treatment), as is usually found in routine plasmapheresis therapies. There was also an improvement in other components of the immune system that bolster the body’s resistance to the disease, accompanied by a reduction of inflammatory non-specific markers and a reduction of urine protein levels. With regard to safety, no significant harmful side-effects associated with use of the device were observed.
Scientific Team
Verto's scientific team is headed by Prof. Yaakov Naparstek, MD, Chairman of Internal Medicine at Hadassah University Hospital, who is the company's founder and a pioneer in the fields of autoimmunity, SLE and autoimmune arthritis.
Collaboration / Investment Opportunity
Strategic industrial partners and investment funding in order to progress to a pivotal clinical trial with a larger sample size and longer treatment period.
Contact:
Stuart Bernstein
Tel: +972-2-6777906
stuart@hadasit.co.il
