Hang in there Bayluv, remember the interview with DeMare (w/Facet) back in May...
DeMare explains what few items were left to complete in order for PharmaCyte to file the IND:
Sarah DeMare: “The single most important item PharmaCyte needed to learn from the FDA at the pre-IND meeting was the acceptability of PharmaCyte’s proposed cell line. Without that understanding, PharmaCyte was unable to move forward with the manufacture of its product candidate for the LAPC clinical trial.”
“An IND encompasses many things, dealing with the manufacture and characterization of the product candidate, the clinical study design and nonclinical studies. PharmaCyte’s therapy for pancreas cancer has undergone or been part of several studies, so incorporation of that information into the IND is not ‘rate limiting’ or ‘slowing down the progress towards submitting an IND.’ The clinical study design that PharmaCyte agreed to undertake after meeting with the FDA is currently being drafted. This is also not rate limiting. To conduct the clinical trial, PharmaCyte will need to manufacture, test and release a Master Cell Bank, a Working Cell Bank and the encapsulated live cells. This information is also required to be described and documented in detail in the IND. The manufacture of these items is the rate limiting factor involved in submitting the IND.”
“Before the IND is filed, the following items must be available for inclusion:
(a) Documentation of preclinical work done on the cells.
(b) Toxicology studies.
(c) Documentation of preclinical work done on the capsules themselves.
(d) A wide array of CMC (Chemistry, Manufacturing and Controls) documentation that verifies that the final biologic product (the encapsulated cells) has been produced under current Good Manufacturing Practices (cGMP)-compliant conditions.
(e) Labeling for the final investigational biologic product.
(f) Previous evidence of human experience with the pancreas cancer therapy (low-dose ifosfamide plus Cell-in-a-Box® encapsulated genetically modified human cells).
(h) The Investigator’s Brochure.
(i) The Informed Consent Form
(j) The Case Report Form”
Can you talk about the items on the list in relation to PharmaCyte’s progress and what further rate limiting steps are on the path to filing the IND?
Sarah DeMare: “Documentation of work done on items (a), (b) and (c) and a significant amount of material concerning item (d) have been accumulated to date. As for item (f), publications of Phase 1/2 and Phase 2 trials that were published in scientific journals are “in-house,” as are original clinical reports of those clinical trials. The clinical trial protocol is nearing the “final” stage. Items (h), (i) and (j) will be written by Translational Drug Development (TD2), the Contract Research Organization (CRO) PharmaCyte has retained to conduct the clinical trial in the U.S. and oversee its conduct in Europe by Clinical Network Services (CNS).”
“The rate limiting step to being able to file the IND and, ultimately, begin the clinical trial is the manufacture of the combination drug product. For live cell-based products, a Master Cell Bank or ‘MCB’ first needs to be manufactured, characterized, tested and released. The speed at which the MCB can be produced is dependent on several different things. First, is the availability of starting materials. Some starting materials can have a significant lead-time when ordering, and you cannot begin manufacture until you have all your starting materials. Second, is the growth rate of the live cells. Cells grow at different rates. There isn’t much that can be done to speed up that process. Unfortunately, it is not a 1 to 2-day event. “
