$PMCB Bullet Points from the Call with respect to the pre-IND meeting PMCB:
• We had a great meeting with the FDA.
• Those individuals from CBER who spoke were very enthusiastic about our product.
• They told us we are on the right track.
• We have agreement on the cell line that will be used in the clinical trial.
• We have agreement on the patient population to be studied in the clinical trial.
• We have agreement on the comparator arms that would be acceptable to the FDA in a pivotal clinical trial.
• We have agreement on the key design aspects of a pivotal clinical study.
• We discussed and have agreement on the primary efficacy endpoints for a pivotal clinical trial.
• We discussed and have agreement on the number of patients needed to comprise an adequate safety database for a Biologics Licensing Application for CypCaps™.
• We discussed exploratory clinical study designs.
• We have agreement that the next step for PharmaCyte is to submit an IND.
• We have confirmed that the FDA believes this is a drug device combination product.
• Currently, there is still a “hard stop” at the six-month mark after a certain number of patients have been enrolled in the trial. This may change to when 50% of the patients have been treated. This will be part of the new trial design and won’t be finalized until the entire study synopsis has been revised to reflect what occurred at the pre-IND meeting.
• Most importantly, the primary endpoints of the trial may change as a result of our dialogue with the FDA. Going into the pre-IND meeting, our primary endpoints were Progression Free Survival (PFS) and safety.
They may change to Overall Survival (OS) and safety. This was discussed with the FDA in the context of making PharmaCyte’s clinical trial a “pivotal trial” rather than an “exploratory trial.”
• So our shareholders understand what a pivotal trial is, I will share its definition. A pivotal trial is a clinical trial or study intended to provide evidence for a drug marketing approval by the United States Food and Drug Administration. Phase III trials are “assumed” to be pivotal. The phrase “pivotal trial” is often used for the rare pivotal phase II trials.
• If this change takes place, the number of patients enrolled in the trial will increase. To be a pivotal trial, the FDA wants at least 100 patients treated with our therapy for purposes of safety. We are working with TD2’s statistician to see how many more patients will have to be enrolled in the trial to reach a statistically significant difference between PharmaCyte’s therapy and the comparator group’s therapy.
• A variety of secondary endpoints will be measured, several of which measure tumor progression. None of the secondary endpoints changed as a result of the pre-IND meeting, except that PFS will be added to the list if the trial becomes a pivotal trial.
• Both CT and PET scans will be heavily used to accurately measure changes in the patients’ tumors, although their frequency will change depending upon the final determination of our primary endpoints.
• A major determination will be whether PharmaCyte’s treatment is better than the comparator treatment in reducing the size of patients’ tumors to the point that they become operable.
• Several study sites have now been identified and major documents, such as the Clinical Protocol, Patient Consent Forms, Investigator’s Brochure, Case Report Forms and the like, are in various stages of preparation.
• During the pre-IND meeting, the FDA noted that our pancreatic therapy is considered a combination product and not a single entity like a single drug, such as gemcitabine or other form of cancer drug.
• In addition, CypCaps™ are considered a biologic product. In other words, it is a living, breathing organism that will require the strictest of regulatory scrutiny. So, while we all would love to complete one step and then move immediately to the next, unfortunately a great deal of patience has been and continues to be necessary as our entire team works toward submitting our IND to the FDA.
Let's go PMCB