PharmaCyte Biotech Updates Status of Investigational New Drug Application to FDA
February 14 2022 - 9:00AM
Business Wire
PharmaCyte Biotech, Inc. (NASDAQ: PMCB), a biotechnology company
focused on developing cellular therapies for cancer and diabetes
using its signature live-cell encapsulation technology,
Cell-in-a-Box®, today provided an update on PharmaCyte’s activities
to lift the U.S. Food and Drug Administration’s (FDA) clinical hold
on PharmaCyte’s treatment for locally advanced, inoperable
pancreatic cancer (LAPC). After submission of an initial
Investigational New Drug Application (IND), the FDA requested
additional studies and information as a prerequisite for approval
of PharmaCyte’s IND. A number of additional studies and assays have
already been completed; several others are quite lengthy and are
underway or are slated to begin soon. As each study and assay is
completed, the results are being compiled and will make up
PharmaCyte’s complete IND submission package to the FDA.
PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner,
explained, “Given our treatment is a biologic and our technology is
a one of a kind live-cell encapsulation, we completely understand
the FDA’s need for more information. We are confident that with the
data we’re producing in our additional studies and assays that the
FDA will grant us an open IND just as the FDA granted us the Orphan
Drug Designation for our treatment for LAPC.
“Last June, we provided an update on efforts being made by our
team of regulatory and scientific experts that are addressing the
FDA’s requirements to have the clinical hold lifted. Today, we
would like to update that list and explain where we are in the
process of delivering our updated IND package to the FDA. We have
brought additional regulatory and scientific experts onboard the
team, and we continue to engage closely with leading Contract
Research Organizations (CROs) and our partner Austrianova to ensure
a successful IND submission.
“We also want to make it clear that PharmaCyte and its service
providers are under the same constraints as everyone else globally
with supply chain issues, Covid related delays, and late delivery
of materials that are needed to complete studies and assays
required by the FDA. This also applies to the manufacturing of
empty capsules and encapsulated live cells that are necessary to
complete many of these required studies and assays. Moreover, many
of the laboratories that are conducting our studies and assays are
being met with long delays in receiving the consumables needed to
conduct the specific studies for which they are responsible. So,
we’re at the mercy of what is a global problem, which obviously
makes it more difficult to offer any accurate timelines for
completion of the FDA required assays and studies.”
Below is the list of items on which PharmaCyte has been working,
including updates on those tests previously reported.
- Additional Regulatory Expertise Added to IND Team - In
addition to its established team of experts, PharmaCyte has
retained Biologics Consulting as a fresh set of regulatory eyes to
perform a regulatory “Gap Analysis” and to assist with PharmaCyte’s
IND submission. Biologics Consulting is a full-service regulatory
and product development consulting firm for biologics,
pharmaceuticals and medical devices and has personnel with
extensive FDA experience. Although it took a lengthy amount of time
to onboard Biologics Consulting, this should augment PharmaCyte’s
ability to submit an acceptable IND to the FDA.
- Stability Studies on PharmaCyte’s Clinical Trial Product
- PharmaCyte has now successfully completed a product stability
study after 3, 6, 9, 12 and 18-months of storage frozen at -80C on
PharmaCyte’s clinical trial product known as CypCaps™, including
container closure integrity testing for certain timepoints. The
next time point in this ongoing stability study will be at 24
months of product stability of the CypCaps. This 24-month time
point analysis is ready to commence, and data will be available in
the coming weeks.
- Additional Studies Requested by the FDA - PharmaCyte has
designed and commenced various additional studies requested by the
FDA, including a stability study on the cells from its Master Cell
Bank (MCB) used to make the CypCaps. PharmaCyte is already at the
3-year stability timepoint for the cells from its MCB.
- Determination of the Exact Sequence of the Cytochrome P450
2B1 Gene - PharmaCyte has completed the determination of the
exact sequence of the cytochrome P450 2B1 gene inserted at the site
previously identified on chromosome 9 using state-of-the-art
nanopore sequencing, a cutting edge, unique and scalable technology
that permits real-time analysis of long DNA fragments. The result
of this analysis of the sequence data confirmed that the genes are
intact.
- Biocompatibility Studies – PharmaCyte has designed and
commenced 8 biocompatibility studies, 6 of which have been
completed successfully. The remaining 2 studies are underway. Those
studies are the Acute Systemic Toxicity Study of Empty Cellulose
Sulphate Capsules in Mice and the Skin Sensitization Study of Empty
Cellulose Sulphate Capsules in Guinea Pigs. To enable these studies
to be performed, Austrianova manufactured and delivered an
additional 400 syringes of empty capsules. Some of the data being
generated will also be used to demonstrate comparability with the
CypCaps successfully used in two earlier clinical trials for
pancreatic cancer.
- Micro-Compression and Swelling Assays - This project is
underway. The project is developing and optimizing two reproducible
methods for testing and confirming the physical stability and
integrity of the CypCaps produced under GMP. These studies required
the acquisition of new equipment by Austrianova as well as
validation and integration into Austrianova’s Quality Control
laboratory.
- Break Force and Glide Testing - PharmaCyte is in the
process of developing a protocol to measure whether the syringe,
attached to the catheter when used to expel the capsules, will
still have a break and glide force that is within the specification
that PharmaCyte has established. PharmaCyte will set this
specification based on the syringe/plunger manufacturer’s measured
break and glide forces, or alternatively, accepted ranges for glide
forces routinely used in the clinic.
- CypCaps Capsules Compatibility with the Syringe and Other
Components of the Microcatheter Delivery System - PharmaCyte
has commenced studies designed to show that CypCaps are not in any
way adversely affected by the catheters used by interventional
radiologists to deliver them into a patient. Compatibility data is
being generated to demonstrate that the quality of the CypCaps is
maintained after passage through the planned microcatheter
systems.
- CypCaps Capsules and Cell Viability after Exposure to
Radiological Contrast Medium - PharmaCyte has designed and
commenced a project to test the effect of the exposure of CypCaps
to two routinely used types of contrast medium that interventional
radiologists use to implant the CypCaps in a patient. The contrast
medium is used to visualize the blood vessels during implantation
of the CypCaps.
- Master Drug File Information- Austrianova is providing
additional detailed confidential information to the FDA on the
manufacturing process, including information on the improvements
and advancements made to the product since the last clinical trials
were conducted with respect to reproducibility and safety. However,
Austrianova has not changed the overall physical characteristics of
the CypCaps. PharmaCyte is supporting Austrianova financially in
this work.
- Additional Documentation Requested by the FDA -
PharmaCyte is in the process of updating its documentation
including extending its discussion on immunological aspects of its
LAPC treatment.
- Pig Study - Finally, the Company has designed a study in
pigs to address biocompatibility and long-term implantation and
dispersion of the CypCaps. This animal study will complement the
positive data already available from the previous human clinical
trials showing the safety of CypCaps implantation in human
patients.
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 is being 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 we believe 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. The
encapsulation of the cell line will be done using the Cell-in-a-Box
technology. Once the encapsulated cells are implanted in a diabetic
patient, we anticipate that they will function as a “bio-artificial
pancreas” for purposes of insulin production.
Safe Harbor
This press release may contain forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of 1995
that express the current beliefs and expectations of the management
of PharmaCyte. Any statements contained herein that do not describe
historical facts are forward-looking statements that are subject to
risks and uncertainties that could cause actual results,
performance, and achievements to differ materially from those
discussed in such forward-looking statements. Factors that could
affect our actual results include our ability to raise the
necessary capital to fund our operations and to find partners to
supplement our capabilities and resources, our ability to
satisfactorily address the issues raised by the FDA in order to
have the clinical hold on our IND removed, as well as such other
factors that are included in the periodic reports on Form 10-K and
Form 10-Q that we file with the U.S. Securities and Exchange
Commission. These forward- looking statements are made only as of
the date hereof, and we undertake no obligation to update or revise
the forward-looking statements, except as otherwise required by
law, whether as a result of new information, future events or
otherwise.
More information about PharmaCyte Biotech can be found at
www.PharmaCyte.com. Information may
also be obtained by contacting PharmaCyte’s Investor Relations
Department.
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version on businesswire.com: https://www.businesswire.com/news/home/20220214005399/en/
Investor Relations: Dr. Gerald W. Crabtree PharmaCyte
Biotech, Inc. Telephone: 917.595.2856 Email: InvestorRelations@PharmaCyte.com
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