The US Food and Drug Administration (FDA) has granted orphan drug status for Kinex Pharmaceuticals' dual src/pre-tubulin inhibitor 'KX02' for the treatment of gliomas, which are the most common and aggressive form of brain cancer.
KX02 is a small molecule drug that has shown potent inhibitory activity against a broad panel of brain tumor cell lines, such as those that are resistant to temozolomide (T98G), the most widely used chemotherapy for the treatment of deadly glioma.
The orphan drug status will help the company in developing KX02 as a potentially new and effective treatment for patients with gliomas.
The company said that the standard treatment for glioblastoma consists of surgical removal of the tumor bulk, followed by radiation and chemotherapy with temozolomide.
During the trials KX02 has showed the ability to clear gliomas after four weeks of therapy in a well-established brain tumor animal model and also prevent tumor recurrence in these animals.
The trials showed that KX02 induced more necrosis compared to Temodar in addition to creating an immune response to the glioblastoma tumor cells.
According to the company, pharmacokinetic trials demonstrated that KX02 is absorbed orally and has 76% penetration into brain tissue from plasma.
Kinex chief scientific officer David Hangauer said, "KX02 is a novel compound with good potential for these patients in terms of generating a durable immune response to tumor cells in mice that allow the animals to live to their full life expectancy without tumor recurrence or further drug treatment," Hangauer said.
XiangXue Pharmaceuticals is Kinex's joint development partner for KX02 in China and has an exclusive license from Kinex for the region.
XiangXue is developing its IND application for the Chinese State FDA and are planning to start KX02 trials for Chinese patients with brain tumor patients in 2014.
Image: Two MRI images of human brain (rear view on left, top view on right) showing a low grade, encapsulated cyst-like glioma (or multiple gliomas). Photo: courtesy of Magnus Manske.
