On February 13, 2017 PharmaCyte Biotech, Inc. (OTCQB:PMCB), a clinical stage biotechnology company focused on developing targeted treatments for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported the comparator arm for its upcoming clinical trial and provided additional clarification on its recent pre-IND meeting with the U.S. Food and Drug Administration (FDA) regarding its upcoming clinical trial in locally advanced, inoperable pancreatic cancer (LAPC) (Press release, PharmaCyte Biotech, FEB 13, 2017, View Source [SID1234517708]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In the company’s upcoming trial, the comparator arm that PharmaCyte’s pancreatic cancer therapy will be compared to is the combination of the cancer drug 5-fluorouracil (FU) and the compound leucovorin (LV). The necessary and quick decision was made by Dr. Manuel Hidalgo, the Principal Investigator for the upcoming clinical, Dr. Daniel Von Hoff with Translational Drug Development (TD2), the CRO for PharmaCyte’s clinical trial, and Dr. Matthias Löhr, the Chairman of PharmaCyte’s Medical and Scientific Advisory Board.

"After our pre-IND meeting I am more confident and enthusiastic than ever about PharmaCyte’s ability to validate its therapy for locally advanced, inoperable pancreatic cancer in a human clinical trial," stated PharmaCyte’s Chief Executive Officer, Kenneth L Waggoner.

He continued, "And I am quite gratified that the FDA sees enough potential in our product to consider our trial a pivotal one under the right circumstances. Now the mission for our entire team is to work diligently towards the submission of our IND to the FDA. We all feel that the suggested changes we received from the FDA should not take long to make and will certainly be well worth it in the long run. For example, we quickly gained agreement on the comparator arm for the trial and, in doing so, we’ve moved closer to filing our IND with the FDA."

PharmaCyte’s management, Dr. Manuel Hidalgo, TD2 and TD2’s consulting statistician are actively working to finalize the number of patients that will be included in the trial. This is the final element and will complete the adjustments necessary for a newly designed trial.

In PharmaCyte’s recent pre-IND meeting with the FDA, the FDA stated that it would be willing to change PharmaCyte’s clinical trial from an "exploratory" trial to a "pivotal" trial under certain conditions. A pivotal trial is a clinical trial intended to provide evidence for a drug marketing approval by the FDA.

This indeed is a landmark moment in PharmaCyte’s history. Generally, a pivotal trial must be a Phase 3 trial (which PharmaCyte’s upcoming trial could be labeled); in such a trial several hundred patients can be treated. However, the FDA indicated that: (i) if PharmaCyte’s therapy shows real promise; (ii) includes a sufficient number of patients; and (iii) includes primary endpoints of overall survival (OS) and safety rather than progression free survival (PFS) and safety, the trial may be considered a pivotal trial.

Mr. Waggoner said of this opportunity, "This is good news for PharmaCyte shareholders since the change from an "exploratory" trial to a "pivotal" trial can eliminate one or two lengthy and costly trials and potentially make PharmaCyte’s Cell-in-a-Box-based product, CypCaps, "market-ready" in a much shorter period of time than anticipated. It may also accelerate the overall development timeline if the results are very positive, thus making the therapy more attractive to potential investors or suitors."

To be a pivotal trial, the FDA wants at least 100 patients treated with CypCaps for purposes of determining the safety of PharmaCyte’s therapy. In the trial’s original design only 40 or so patients would have received CypCaps and been evaluated for safety.

Other highly positive news provided by Mr. Waggoner concerning the pre-IND meeting with the FDA included:

agreement with the FDA that PharmaCyte is on the "right track" in its development program;
agreement with the FDA on the cell line that will be used in the clinical trial;
agreement with the FDA on the patient population to be studied in the clinical trial;
agreement with the FDA on the secondary endpoints of the clinical trial, except that PFS will be added to the list of secondary endpoints if the trial becomes a pivotal trial;
agreement with the FDA on the number of patients needed to comprise an adequate safety database for a Biologics Licensing Application for CypCaps;
agreement that the FDA believes CypCaps is a drug/device combination product;
agreement with the FDA that it will assist PharmaCyte in its development program; and
agreement with the FDA that the next step for PharmaCyte is to submit an IND.
There is still a "hard stop" at the six-month mark after a certain number of patients have been enrolled in the trial to review the data generated to that point. The timing of this hard stop may change, however, to the point in time when 50% of the patients have been treated. Because the clinical trial is an "open-label" trial (the trial is not a "blinded" study), this interim analysis should give PharmaCyte an important indication of the successfulness of its CypCaps therapy for LAPC.

Mr. Waggoner commented on the significance of following the FDA’s guidance moving forward stating, "We have one shot at this, and we intend to get it right. We greatly appreciate the continued patience and support of our shareholders during this process."