FDA fast tracks PI-88 for the treatment of post resection liver cancer

Progen Pharmaceuticals Limited has announced that its investigational anti-cancer drug PI-88 has been awarded Fast Track status by the U.S. Food and Drug Administration (FDA).

The fast track designation has been granted to PI- 88 for the prevention of tumor recurrence following curative liver resection in patients with hepatocellular carcinoma (HCC, primary liver cancer).

Under the FDA Modernization Act of 1997, the fast track program is designed to facilitate the development and expedite the regulatory review of new drugs that demonstrate the potential to treat serious or life-threatening diseases where there is an unmet medical need. The Fast Track designation will enable Progen to file a New Drug Application (NDA) on a rolling basis as data becomes available. This permits the FDA to review the different components of the Drug Master File as they are completed in advance of receiving the final submission.

Mr. Justus Homburg, Progen's CEO commented, "This designation will speed the process of bringing this potentially clinically very important drug to patients with liver cancer. The FDA decision to award fast track status to PI- 88 was based on recent Phase 2 clinical data and the high unmet need for treatments for patients with resectable primary liver cancer. The key Phase 2 study showed that PI-88 has the potential to improve the time a patient remains disease free following surgery. As we enter the final stages of clinical development and commence a multinational Phase 3 trial, we look forward to collaborating closely with the FDA to expedite the development and approval of PI-88."

Conservatively, over half a million new cases of HCC are diagnosed worldwide each year, making it the fifth most common cancer and the third major cause of deaths due to cancer worldwide. The most common causes of HCC are related to chronic infection with hepatitis B and C (HBV, HCV). Due to late disease diagnosis, currently only about 25% of patients are eligible for surgery and half of those patients will have their disease recur within 12-15 months. No products have been approved by the FDA to prolong the time a patient remains disease free following surgery.

The fast track designation is fully aligned with Progen's Phase 3 trial design. The double blinded, placebo-controlled Phase 3 trial will be powered for the primary endpoint of disease-free survival determining if PI-88 can prevent early recurrence following curative hepatic surgery. The trial will begin later this year and targets 600 patients with liver cancer post resection, in more than a dozen countries.

Progen Pharmaceuticals Limited is an Australian-based globally focused biotechnology company committed to the discovery, development and commercialization of small molecule therapeutics primarily for the treatment of cancer.

PI-88 is one of a new class of multi-targeted cytostatic cancer therapeutics. It is a novel anti-cancer compound with a first-in-class mechanism as a heparan sulfate mimetic. Its anti-tumor activity is based on inhibition of two biological processes - angiogenesis (the growth of new blood vessels) and metastasis (the spread of cancer to other sites) - critical to the growth and progression of cancer. In a Phase 2 trial in post-resection liver cancer, PI-88 demonstrated a 25% improvement in the primary endpoint of disease-free rate at 48 weeks and 78% improvement in secondary endpoint of disease-free survival. These results, combined with positive feedback from the FDA, provide Progen with confidence in the potential of PI-88 for this indication and we are therefore aggressively pursuing its development towards registration and commercialization.