Biopharmaceutical developer Endocyte (NASDAQ:ECYT) overcame a major hurdle in its path to drug approval and can now resume enrollment in its phase 3 clinical trial of an experimental drug for ovarian cancer. The company announced Monday that U.S. regulators approved the importation of cancer drug Doxil from Europe for the study.
Endocyte’s phase 3 trial of EC145, a targeted small molecule drug that’s intended to attach chemotherapy drugs to folate receptors often overexpressed in cancer cells, was halted just a few months after it started in May 2011 due to the global shortage of Doxil. The trial aims to show that the drug, when used with Doxil, improves progression-free survival of ovarian cancer patients compared with the use of Doxil alone.
Endocyte President and CEO Ron Ellis said in a statement that the supply from Europe approved by the FDA is expected to support enrollment in the trial through the end of the year, but the company will continue looking for alternatives so that it can resume enrollment outside of the U.S.
Doxil has been in short supply since last fall due to quality concerns at a Johnson & Johnson supplier, Ben Venue Laboratories Inc. Late last month, the FDA announced that it would allow temporary imports of a replacement drug called Lipodox for the treatment of patients.
The West Lafayette, Indiana company plans to file for early drug approval of EC145 in Europe this year, based on phase 2 results, according to Businessweek. European regulators gave Endocyte the OK to file early based on phase 2 trial results, but wanted the Indianapolis company to at least be enrolling patients in a phase 3 trial at the time of filing. The FDA’s approval of imported Doxil will make this a possibility for Endocyte.
U.S. approval of EC145, however, will have to wait until the phase 3 trial is completed.
The pre-revenue company is currently operating on the $142 million raised during public stock offerings last year, the Indianapolis Business Journal reports. Analysts at Wedbush have estimated that EC145 could generate $1.5 billion to $2 billion in annual sales in the U.S. and EU if it’s approved.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Shares of ECYT have yet to recover from when they plunged in December after results from a small phase 2 study showed the drug led to shorter survival times than Doxil alone.
