Date: Sep 12, 2013 Source: MedCity News (
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Having received the first installment of a $1.43 million SBIR grant from the National Institutes of Health, a Cleveland-based biotech is hoping for clinical trials next year for its new therapeutic for patients with a rare blood disease called paroxysmal nocturnal hemoglobinuria.
NovelMed is working with a humanized antibody that it thinks could block intra- and extra-vascular hemolysis in patients with PNH. The orphan disease causes part of the immune system called the complement system to attack and kill red blood cells, which can result in a slew of symptoms including fatigue, weakness, pale skin, shortness of breath, abnormal blood clotting and red urine. It is thought to affect about 10,000 people in the U.S.
The goal is for NovelMed to have its candidate in clinical trials in the first half of 2014, according to Jeff Green, the company's vice president of clinical affairs. Based on its pre-clinical work, the biotech says its platform may yield a self-injectable treatment that patients could administer at home.
A drug approved by the FDA back in 2007 has improved treatment of PNH dramatically, Green said. Alexion Pharmaceuticals' Soliris inhibits parts of the complement system and has demonstrated long-term efficacy in reducing patients' hemolysis and risk of thromboembolism.
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"In 2007, Soliris came along and it has been effective at treating the disease, but the problem is it affects the part of the pathway that is responsible for host defense," Green explained. "When you shut down the body's ability to fight infection, there are people who develop meningococcal infection. There's a black box warning on Soliris about the chance of getting severe infections."
What NovelMed must do, then, is demonstrate in clinical studies that its drug shows superior efficacy and safety to Soliris.
Green said the company is in production phase and is putting together plans to begin talks with the FDA. It's also looking for strategic partners.
NovelMed has a platform of antibodies it's developing for various complement-mediated diseases, including age-related macular degeneration and arthritis, Green said. Rekha Bansal formed the company back in 2003 after spending several years in R&D at another Ohio biotech, Gliatech.
