BERLIN & GERMANTOWN, Md.—July 1 saw Epigenomics AG, the German-American cancer molecular diagnostics company, report on the status of its Pre-Market Approval (PMA) application with the U.S. Food and Drug Administration (FDA) and provide information regarding the design and objectives of an additional clinical study requested by the FDA for regulatory approval of Epi proColon, the company’s blood-based colorectal cancer (CRC) screening test in the United States.
In a recent meeting between the company and the FDA, detailed discussions were held to specify the details regarding the design of this clinical study. Epigenomics proposed to conduct a study to demonstrate that Epi proColon will increase participation in CRC screening in patients being offered the blood-based test as compared to those being offered a FIT stool-based test. The trial is intended to be conducted in a population that is non-compliant to CRC screening according to current screening guidelines and may include patients actively managed by CRC screening programs within healthcare systems.
Discussions with several healthcare organizations to participate in such a study are already underway. While a decision on the final study plan has not yet been taken, an adaptive study design could allow early completion of the study if statistical significance is clearly met. Based on these initial assumptions, Epigenomics foresees that conducting the study can likely be done with a reasonable number of patients that could be enrolled in a matter of a few months. However, additional time will need to be allocated for study initiation and logistics. Once the study has been completed, a PMA amendment with study results will be filed within a reasonable period of time.
The study’s secondary objectives will include a measurement of compliance to colonoscopy in those patients with positive test results with Epi proColon or FIT. For this endpoint, the results will be descriptive and will not be reported with statistical significance in the premarket setting. The company remains committed to studying the performance of its blood-based test in a programmatic setting after potential approval in the context of a post-marketing study as previously proposed in its PMA application. Based on initial estimates, Epigenomics expects additional costs for the study to be in the range of less than €1 million.
“After a productive dialogue with the FDA, we now have a better understanding of the issues and background of the agency's assessment,” said Dr. Thomas Taapken, CEO and chief financial officer of Epigenomics. “We are confident that we can complete the requested clinical study within a reasonable period of time. Although Epigenomics is currently able to fund these additional activities, we are constantly evaluating various options to maintain the company’s financial flexibility and to allow for the preparation of commercialization in the U.S. in order to rapidly enter the market post-approval.”
The discussed clinical study was requested as part of the FDA response letter in relation to Epigenomics’ PMA application for the company’s blood-based colorectal cancer screening test Epi proColon the company received at the beginning of June 2014.