PharmaCyte Presents Options for Phase II Pancreatic Cancer Trial
Laguna Hills, California-based PharmaCyte Biotech (Formerly Known As Nuvilex) recently discussed its options and plans for a Phase IIb clinical trial of its Cell-in-a-Box technology in pancreatic cancer.
The key issue was whether the company’s Phase IIb trial could be considered “pivotal,” which would funnel into registration for marketing. In a discussion in January 2017 with the U.S. Food and Drug Administration (FDA), PharmaCyte was told that it was possible, but the trial would need to be bigger than what the company was planning and that the data would have to be “markedly superior” to the data they had presented thus far.
After about a year’s consideration, PharmaCyte chose to conduct a Phase IIb clinical trial instead of a pivotal trial, largely on the basis of its consulting oncologists, chief medical officer and its advisory board. The company states, “PharmaCyte must rely on data from two European trials from 27 patients that were conducted about 20 years ago. The data from these trials are incomplete when compared to what is now required by the FDA to support a pivotal trial.”
As a result, the Phase IIb trial design will attempt to determine the efficacy and safety of multiple courses of itosfamide in patients with locally advanced, non-metastatic, inoperable pancreatic cancer (LAPC), as well as how the company’s therapeutic compares to a commonly used treatment for LAPC after patients’ cancer stops responding after four to six months of the combination therapy of gemcitabine and Abraxane. The company hopes the data will provide a more solid foundation for working with the FDA on getting its therapy to market.
The company states, “The planned Phase IIb trial will be significantly larger than the original Phase IIb trial PharmaCyte previously discussed with the FDA and will include multiple courses of low dose ifosfamide (the earlier trials used only two courses). This trial will also provide better statistical analyses of PharmaCyte’s therapy for LAPC and the comparator arms in terms of survival and safety. Also, the possibility exists that if the data from PharmaCyte’s therapy are significantly better than the data from the comparator arm, this may allow PharmaCyte to apply to the FDA for accelerated approval.”
The company’s therapeutic approach involves encapsulating genetically engineered human cells that convert an inactive chemotherapeutic into its active or “cancer-killing” form. In the case of pancreatic cancer, the encapsulated cells are placed in the blood stream close to the tumor. Ifosfamide is typically activated in the liver. PharmaCyte’s approach is to dose the patients with one-third the normal dose. The circulatory system carries the drug to where the encapsulated cells have been implanted. The drug seeps through the capsules’ pores and the live cells inside the capsules act as a “bio-artificial liver” that activates the chemotherapeutic at the cancer site.
This is the company’s Cell-in-a-Box technology. The company is also developing it for Type 1 diabetes and insulin-dependent Type 2 diabetes. In those diseases, a human cell line is used that is genetically engineered to produce, store and release insulin in response to the blood glucose levels in the body.
On February 26, PharmaCyte announced that a six-month study on storage of the frozen encapsulated cells had been successfully wrapped up by Austrianova. This is required to determine an initial shelf life for a medicinal product prior to an advanced-phase clinical trial.
“We are pleased with this significant result which will enable us to apply to the FDA for an initial shelf life of six months,” said Kenneth Waggoner, PharmaCyte’s chief executive officer, in a statement. “This study, one of many being performed for us by Austrianova, is an essential part of the Investigational New Drug Application (IND) that we will be submitting to the FDA.”