Kinex Pharmaceuticals has received Orphan Drug designation for KX02 for the treatment of gliomas, which are the most common and aggressive form of brain cancer. Orphan drug status qualifies Kinex for seven years of exclusivity after formal marketing approval, as well as further development incentives.

KX02, a dual src/pre-tubulin inhibitor, is a small molecule drug that has shown potent inhibitory activity against a broad panel of brain tumor cell lines, including those that are resistant to Temozolomide (T98G), the most widely used chemotherapy for the treatment of malignant glioma.

In a well-established brain tumour animal model, KX02 consistently clears brain tumours after four weeks of therapy, in 30-60 per cent of treated animals. These studies showed that KX02 induced more necrosis compared to Temodar, and also generated an immune response to the glioblastoma tumour cells. Pharmacokinetic studies showed that KX02 is absorbed orally and has 76 per cent penetration into brain tissue from plasma.

Dr David Hangauer, chief scientific officer of Kinex stated, “Receiving Orphan Drug Status for KX02 speaks to the need for new treatment options for patients with gliomas. KX02 is a novel compound with good potential for these patients in terms of generating a durable immune response to tumour cells in mice that allow the animals to live to their full life expectancy without tumour recurrence or further drug treatment. We are looking forward to moving this novel compound into the clinic.”

“This is an important regulatory milestone for Kinex,” stated Dr Lyn Dyster, senior VP of Operations at Kinex. “The orphan drug designation will facilitate our goal of developing KX02 as a potentially new and effective treatment for patients with gliomas. The standard treatment for glioblastoma consists of surgical removal of the tumour bulk, followed by radiation and chemotherapy with temozolomide. KX02 has demonstrated the ability to clear gliomas in a well-established animal model and also prevent tumour recurrence in these animals and we are excited to move this novel compound into the clinical setting. We have worked diligently with clinical sites to establish the trial protocols and expect to initiate a phase I trial imminently.”

XiangXue Pharmaceuticals is Kinex’s collaborative development partner for KX02 in China and has an exclusive license from Kinex for this territory. YongHui Wang, chairman and CEO of XiangXue Pharmaceuticals added, “Kinex has been an excellent collaborative partner.  Their ability to develop novel drugs and work closely with regulatory agencies continues to impress us and we are very pleased that the FDA has granted Orphan Drug Status for KX02. Glioblastoma is an aggressive cancer with few treatment alternatives. In fact, the average patient with malignant glioblastoma has a life expectancy of less than 12 months once they are diagnosed with the disease.  I am hopeful, based on promising animal studies, that KX02 can provide a new treatment option for this devastating disease. XiangXue is developing our IND application for the Chinese State FDA and we are planning to initiate KX02 trials for Chinese patients with brain tumor patients in 2014.”