Curtana Pharmaceuticals, a privately-held, preclinical stage biopharmaceutical company, announced that the FDA has granted orphan drug designation to CT-179, the company’s lead Olig2 inhibitor for the treatment of gliomas, including glioblastoma (GBM) in adults and pediatric high grade glioma in children. 

Glioblastoma is the most common and most aggressive of the malignant primary brain tumors in adults. According to the American Brain Tumor Association, an estimated 12,390 new cases of GBM are predicted in 2017. Conventional therapeutic approaches for GBM, including surgery, chemotherapy and radiation therapy, target the tumor bulk, but have limited effect on the glioma cancer stem cells (CSCs), which are responsible for the recurrence of disease that occurs in most of the GBM patients. Accordingly, GBM represents a significant unmet clinical need as the median survival is less than 15 months and five-year survival rate is less than 10%. 

Curtana is targeting Olig2, a transcription factor important to normal brain development that has been shown to be a critical oncogene controlling tumor initiation, growth, invasion, differentiation and radiation resistance. Typically, Olig2 is not active in normal brain tissue. However, it is expressed in all diffuse gliomas and nearly 100% of the glioma CSCs, which makes it a highly attractive drug target. 

“We are pleased that the FDA has recognized the potential for CT-179 to provide a significant therapeutic benefit to patients with GBM,” said Gregory Stein, M.D., M.B.A. and Chief Executive Officer, Curtana Pharmaceuticals. “GBM is a devastating disease with a generally very poor prognosis even with the best available care. CT-179, projected to enter the clinic in the first half of 2018, has been shown to significantly prolong survival in relevant animal models, especially when combined with the standard of care treatments, temozolomide and radiation therapy.” 

The FDA Orphan Drug Designation program provides special status to drugs and biologics intended to treat, diagnose or prevent so-called ‘orphan’ diseases that affect fewer than 200,000 people in the U.S. This designation provides for a seven-year marketing exclusivity period against competition, as well as other incentives, such as federal grants, tax credits and PDUFA filing fee waivers.