NEW YORK, NY—PharmaCyte Biotech (OTCQB: PMCB) continues to work with consultants to prepare its Investigational New Drug application (IND). At the same time, PharmaCyte is reportedly completing the remaining tests that will provide the last major pieces of non-medical information for the company to complete the IND package that it will submit to the U.S. Food and Drug Administration (FDA) for its planned Phase 2b clinical trial to treat locally advanced, inoperable pancreatic cancer (LAPC) using its signature technology, Cell-in-a-Box® together with the cancer prodrug ifosfamide, at trial sites within the United States.
In light of new guidelines coming down from the FDA on conducting clinical trials and several biotechnology companies already pausing their clinical trials during this global COVID-19 pandemic, in addition to needing some clarification on two additional tests that were addressed in PharmaCyte’s recent Quarterly Report on Form 10-Q (10-Q), we talked to the Company’s Chief Executive Officer (CEO), Kenneth L. Waggoner, to gain a better understanding of how COVID-19 may be affecting the company’s work to complete its IND.
Question 1: First and foremost, we’d like you to address the two tests that were discussed in PharmaCyte’s recently filed 10-Q – the Stability Study and the Container Closure Integrity (CCI) test. What are these?
PharmaCyte CEO: “The Stability Study is a study designed to determine whether the capsules generated from the manufacturing run we will be using in our planned Phase 2b clinical trial remain intact and functional after being frozen at approximately -80°C for specified periods during a 24-month time span and then thawed for a total period of 24 months. During this period, there are set measuring points, such as month 0, 3, 6, 9, 12, 18 and 24 months. The parameters being measured at each time point are cell identity, purity, viability, potency and sterility.
“We have elected to measure sterility by using a CCI test on a syringe containing 300 encapsulated cells. So, this too is a 24-month test in total. This test essentially measures whether, over time, the frozen syringes full of our encapsulated cells remain intact. We will be using High Voltage Leak Detection to determine this.”
Question 2: You mentioned the duration of both of these tests being 2 years, which naturally begs the question, will you have to complete the entire study/test before you can submit the IND? If not, how much data is needed from the Stability Study to complete the IND package that you’ll submit to the FDA? And how much data is needed from the CCI test?
PharmaCyte CEO: “We do not need to complete the entire 24-month Stability Study to complete and submit the IND to the FDA. Our regulatory consultant advises us that we will need three months of data from the Stability Study, which is already underway, to include in the IND and not the entire 24 months’ worth of data. In terms of the CCI test, we have been advised that it would be good to have 3 months of data by the time we submit the IND, but if we’re ready to submit the IND and the CCI test is simply underway, we may be given some leeway by the FDA with the understanding that that three months of data will be available during the 30-day review period of our IND submission by the FDA.”
Question 3: Now let’s discuss how the disruptive effects of this global pandemic hinders PharmaCyte’s ability to complete the Stability and CCI testing, if at all?
PharmaCyte CEO: “It is unclear whether the global pandemic will slow down or hinder completion of the Stability Study. Thus far, we are not aware of anything like that. The Stability Study is underway at Austrianova’s manufacturing facility in Thailand. However, as we have experienced in California, the Thai government may close down non-essential facilities causing difficulties in completing certain aspects of the Stability Study in a timely fashion. We’ll just have to wait and see.
“There has already been a slowdown in receiving required component parts for the CCI test which is being done in the U.S. and which requires shipment of materials from Thailand. FedEx failed to deliver the parts needed to start the work of designing the test. The problem got corrected, but we lost about 30 days of much needed time. The same potential for another slow down or work stoppage exists due to the COVID-19 pandemic.”
Question 4: Prevention Bio, Arrowhead Pharmaceuticals, Addex Therapeutics, and Iveric Bio have all already paused their clinical trials. Given that we’re battling a global pandemic, what are some of the challenges that PharmaCyte and others face when beginning a clinical trial?
PharmaCyte CEO: “Challenges may arise, for example, from quarantines, site closures, travel limitations, interruptions to supply chain for the investigational product, or other considerations if site personnel or trial subjects become infected with COVID-19. These challenges may lead to difficulties in meeting protocol-specified procedures, including administering or using the investigational product or adhering to protocol-mandated visits and laboratory/diagnostic testing.
“Also, patient enrollment, trial sites being solely focused on COVID-19 and not having space/availability for clinical trials, etc. In PharmaCyte’s case, the age of many of the patients that we’d enroll falls in the danger zone and will have a compromised immune system. In fact, all of our enrolled patients will have a compromised immune system from our treatment or that of the three comparators.”
Question 5: The FDA is obviously very busy with all things related to developing and testing treatments for COVID-19 along with the monitoring of current clinical trials in this new, but likely temporary dangerous environment, so having said that, do you feel somewhat thankful that you haven’t submitted your IND yet?
PharmaCyte CEO: “Our original timeline had us submitting the IND in the first quarter of this year. So yes, we are thankful that we’re not waiting on an answer from the FDA in this current environment where our submission package may have been put on the backburner and our patient population may have been seen as a negative in the U.S. while the country is dealing with COVID-19 as a medical priority. Every day, we get new directives from the CDC, NIH, etc. on how hospital facilities are going to be used.
“COVID-19 patients seem to take priority over any other type of medical condition. For example, elective surgeries have been deferred, as hospitals ready themselves for an anticipated onslaught of COVID-19 patients.”
To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, watch the company’s documentary video complete with medical animations at: https://www.PharmaCyte.com/Cancer
About PharmaCyte Biotech
PharmaCyte Biotech, Inc. is a biotechnology company developing cellular therapies for cancer and diabetes based upon a proprietary cellulose-based live-cell encapsulation technology known as “Cell-in-a-Box®.” This technology will be used as a platform upon which therapies for several types of cancer and diabetes are being developed.
PharmaCyte’s therapy for cancer involves encapsulating genetically engineered human cells that convert an inactive chemotherapy drug into its active or “cancer-killing” form. For pancreatic cancer, these encapsulated cells are implanted in the blood supply to the patient’s tumor as close as possible to the site of the tumor. Once implanted, a chemotherapy drug that is normally activated in the liver (ifosfamide) is given intravenously at one-third the normal dose. The ifosfamide is carried by the circulatory system to where the encapsulated cells have been implanted. When the ifosfamide flows through pores in the capsules, the live cells inside act as a “bio-artificial liver” and activate the chemotherapy drug at the site of the cancer. This “targeted chemotherapy” has proven effective and safe to use in past clinical trials and results in little to no treatment-related side effects.
PharmaCyte’s therapy for Type 1 diabetes and insulin-dependent Type 2 diabetes involves encapsulating a human cell line that has been genetically engineered to produce and release insulin in response to the levels of blood sugar in the human body. PharmaCyte is developing the use of genetically modified liver cells and stem cells, as well as beta islet cells, to treat diabetes. The encapsulation will be done using the Cell-in-a-Box® technology. Once the encapsulated cells are implanted in a diabetic patient, they will function as a “bio-artificial pancreas” for purposes of insulin production.