Pancreatic cancer is one of the hardest cancers to treat. 50,000 people are diagnosed with it in the U.S. each year, with about 40,000 of them dying from the disease annually. The one year survival rate is an abysmal 29% and only 1% survive past 5 years. A new oral cancer drug RX-3117 from Rexahn Pharmaceuticals offers hope and efficacy where other drugs have failed.
Rexahn just announced that based on preliminary efficacy data in patients with relapsed or refractory pancreatic cancer, RX-3117 will proceed into Stage 2 of a Phase Ib/IIa clinical trial. Patients in the trial had previously been treated with three other FDA approved therapies and none of them were effective. These patients would normally be offered palliative or supportive care. RX-3117 may offer a new hope. Supporting its development, the U.S. FDA has granted Orphan Drug Designation to RX-3117 for pancreatic cancer.
Clinical data from Stage 1 of the Phase Ib/IIa clinical trial will be presented at the European Society for Medical Oncology (ESMO) conference in October 2016.
RX-3117 is a novel, investigational small molecule nucleoside compound. The drug has shown broad spectrum anti-tumor activity against over 100 different human cancer cell lines and efficacy in 17 different mouse xenograft models including pancreatic, bladder, lung, cervical and colon cancers, as well as gemcitabine resistant cancer cells. Importantly, RX-3117 still retains its full anti-tumor activity in human cancer cell lines made resistant to the anti-tumor effects of gemcitabine.
Rexahn’s ongoing Phase Ib/IIa clinical trial is a multicenter, open-label single-agent study of RX-3117 being conducted at 10 clinical centers in the United States. Patients receive a 700 mg daily oral dose of RX-3117, five times weekly on a three weeks on, one week off dosing schedule in a 28 day cycle for up to eight treatment cycles, or until their disease progresses. The study follows a two-stage design. In stage 1 of the trial, up to 10 patients with relapsed or refractory metastatic pancreatic cancer were enrolled. Based on predefined criteria, if 20% or more of the patients had progression free survival of > 4 months, or an objective clinical response rate and reduction in tumor size, then an additional 40 pancreatic cancer patients would be enrolled into stage 2.
Patients enrolled into stage 1 of the clinical trial had actively progressing disease, with 44% of them having failed > 3 prior cancer therapies (including 5-FU and gemcitabine-based therapies). RX-3117 was shown to be safe and well tolerated in this patient group. The clinical study is still on-going. However since the predefined efficacy criteria have been achieved, stage 2 of the study has been initiated.
Rexahn has a broad oncology pipeline that includes two other anti-cancer compounds currently in clinical development: Supinoxin™, and Archexin®, and a novel nanopolymer-based drug delivery platform technology that may increase the bio-availability of FDA-approved chemotherapies.
For more info: http://www.rexahn.com/cms/