New orphan drug by AstraZeneca

AstraZeneca Chief Strategic Officer Marc Dunoyer and CEO Alexion Pharmaceuticals have announced their first purchase since assuming their new role. Currently, a new drug is being developed for the disease, where several players have already failed. (Prothena and GlaxoSmithKline)

The key element of the portfolio is CAEL-101, an anti-lgG1k monoclonal antibody developed for the treatment of systemic light chain amyloidosis (a rare fatal disease). The median survival time after diagnosis is less than 18 months. CAEL-101 may become the very first drug that removes amyloid deposits from organs and tissues and improves the function of affected organs as well. The drug has also completed a Phase III clinical trial sponsored by Caelum. AstraZeneca is going to promote and accelerate the development of this project, and test it in patients with stage III light chain amyloidosis.

Source: endpts