InDex Pharmaceuticals: Improving quality of life in inflammatory disease
Severe treatment-refractory ulcerative colitis, a form of inflammatory bowel disease, is an unpleasant and devastating disease that leads to weight loss, increases the risk of colon cancer, and severely impacts patients' quality of life. For severely-affected patients who have failed all available therapies, surgery to remove the colon (colectomy) is the only remaining treatment option. However, research at Stockholm-based InDex Pharmaceuticals could provide an alternative to this invasive form of treatment, which can results in reliance on a colostomy bag for many patients.
Kappaproct, InDex Pharmaceuticals' lead product, has Orphan Drug designation for active ulcerative colitis in Europe.
A DNA-based immunomodulatory sequence (DIMS) that binds to toll-like receptor 9 (TLR 9), Kappaproct has just moved into phase III trials and is being tested in 131 patients in seven countries across Europe. The study will follow patients with severe treatment-refractory ulcerative colitis for one year, looking at the rate of clinical remission at 12 weeks and rate of colectomy over a year. It's expected to close at the end of the first quarter 2014, with results available around May 2014.
"If the results are positive, we may be able to submit for fast-tracked conditional approval to the European Medicines Agency after the trial, and could launch as early as 2016. This would be important for the patients suffering from this disease and for InDex Pharmaceuticals. Kappaproct would be our first product to market, and it is a product we have discovered and developed in house. It would also validate our platform technology," says Jesper Wiklund, chief executive officer, InDex Pharmaceuticals.
Kappaproct is currently positioned as a fourth-line treatment for ulcerative colitis, following anti-inflammatories, steroids and TNF-alpha inhibitors, but has potential as a third-line treatment, after steroid treatment.
"We are working on developing a companion diagnostic for Kappaproct that would help doctors determine the right treatment choices," says Wiklund.
After Kappaproct, the next product in the company's pipeline, DIMS 9054, is a development candidate for treatment resistant pulmonary inflammation. According to Wiklund, as InDex Pharmaceuticals is a venture capital-backed company, getting positive phase III results for Kappaproct will help them to raise further financing to move DIMS 9054 and other products in our pipeline forward.
The company's pipeline also includes diagnostics in inflammatory bowel disease. The most advanced product is DiBiCol, which is designed to support diagnosis by differentiating between the two most common forms of inflammatory bowel disease: Crohn's disease, which affects the entire gastrointestinal tract, and ulcerative colitis, which affects only the colon. This product, which is currently available on the Swedish market, could be used to confirm or rule out diagnosis. It received a CE mark in Europe in June 2012.
"We launched DiBiCol in Sweden in 2009, and despite no active marketing we already have a good reach based only on word-of-mouth recommendations," says Wiklund.
Wiklund joined InDex Pharmaceuticals in 2011, and explains: "I was attracted by InDex Pharmaceuticals' approach and achievements – it had a late stage product that had shown efficacy from early days of development, that addressed an area of unmet medical need and that was ready to go into phase III. And as Kappaproct offers hope for people with a severe disease, it has potential to be a real game changer."
InDex Pharmaceuticals is seeking regional or global partners for Kappaproct, and has already contacted a range of large and small biopharma companies, including global and regional players, and a number of discussions are ongoing.
Around two million people in Europe and the United States suffer from ulcerative colitis, and each year 500-1000 colectomies are performed in Europe.
As Kappaproct targets severely ill patients who are referred to tertiary care centers, its sales would require a relatively small and focused sales team. As an example, Wiklund explains, doctors in five or six treatment centers in Sweden actually treat around 80% of the country's patients. This means that the company could retain the option to keep sales and marketing in house. Whichever route is taken, Wiklund sees InDex Pharmaceuticals as a company with an exciting future ahead.
"Over the next five years, I expect to see InDex Pharmaceuticals as a standalone company with products on the market and moving through the pipeline, or as part of a larger company. Both are very possible."