PharmaCyte Biotech, Inc. (OTCQB:PMCB), a clinical stage
biotechnology company focused on developing targeted therapies for
cancer and diabetes using its live-cell encapsulation technology,
Cell-in-a-Box®, presents today another in a series of articles that
will serve to educate the public on its live-cell encapsulation
technology and its use in developing treatments for pancreatic
cancer and diabetes. This educational piece addresses PharmaCyte’s
intellectual property (IP) and the strategy that PharmaCyte will
employ to protect that property.
PharmaCyte’s IP consists of exclusive license
agreements that PharmaCyte has with other parties and IP that
PharmaCyte intends to create during the clinical development of its
product candidates. In the questions and answers below,
PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner,
discusses PharmaCyte’s IP protection strategy, which consists of
patents, patent term extensions, regulatory data exclusivity,
market exclusivity for orphan drugs, know-how/trade secrets and
trademarks.
PharmaCyte’s Intellectual Property
Protection Strategy
Which family of patents protects
PharmaCyte’s pancreatic cancer therapy and what is your plan to
extend those patents?
“Before I answer that question, let me say that
our pancreatic cancer therapy has two iron-clad protections that
will far surpass any protection that our patents could ever bring
if we can obtain FDA approval. I will discuss this a little later
in this piece, but keep in mind while reading these responses that
our pancreatic cancer therapy is going to remain protected long
after the patents expire.
“Everyone interested in PharmaCyte’s pancreatic
cancer therapy should be focused on the family of patents that deal
with the live-cell encapsulation of genetically altered human cells
that overexpress a form of the Cytochrome p450 enzyme system
(normally found in the liver), and specifically on only 2 patents
in that family. Those patents are set to expire in the United
States a little over a year from now on March 27, 2017. These are
the only patents that pertain to PharmaCyte’s pancreatic cancer
therapy. Now, while these patents are set to expire next year, we
certainly aren’t sitting idly by and allowing that to
happen.
“We do have a protection strategy in place,
which includes filing an application with the U.S. Patent and
Trademark Office for interim extensions extending the life of those
patents 1 year at a time for up to 5 years, which we believe will
be long enough to get us through clinical trials and the regulatory
approval process. The earliest the application can be filed is 6
months before the expiration of the patents, and the application
can be filed up to 15 days before the expiration date. PharmaCyte
can and plans to file its patent extension application between
September 27, 2016, and March 12, 2017.”
Can PharmaCyte apply for follow-on
patents? If so, can you explain what these patents
are?
“Yes. New patentable inventions related to a
pharmaceutical product, also called ‘follow-on patents,’ generally
encompass improvements to, or new uses for, the pharmaceutical not
disclosed or suggested in the original patent. PharmaCyte
anticipates extending its patent protection for its product
candidates through improvements to its core technology,
including:
1. New Formulations: New formulations of a known drug compound
that are clinically superior to the previous drug formulation may
be patentable. Developing new formulations that promote a patient’s
successful therapy through such things as reduced dosing or ease of
use, or that exhibit improved therapeutic outcomes or more
favorable side-effect profiles, are patentable. Examples include
sustained-release formulations, extended-release formulations and
dosing regimens.
2. New Routes of Administration: Additional patent protection
may be obtained for new formulations that permit new routes of
administration.
3. New Uses: Patents directed to new uses and treatments may be
obtained.
4. Combinations: Combining two or more drugs into one
treatment also may be patentable.
New discoveries that may be eligible for patent
protection as follow-on patents cannot be predicted at the current
time; however, PharmaCyte anticipates improvements to its
technology and product candidates to be generated as these product
candidates move through clinical testing.”
Being that PharmaCyte’s cancer product
candidates are biologics, does PharmaCyte qualify for regulatory
data protection and the 12 years of data exclusivity that comes
with it as outlined by the Biologics Price Competition and
Innovation Act (BPCIA), which was enacted as part of the Affordable
Care Act in 2010?
“Yes. This is one of the two iron-clad
protections that I was speaking of earlier in this article. We will
be seeking this protection and the 12 years of data exclusivity it
provides. ‘Reference product exclusivity’ or ‘regulatory data
protection’ is an IP right available for a limited duration, which
protects an innovator's proprietary safety and efficacy data for
its innovative product. This protection prevents any other party,
during an exclusivity term of 12 years, from relying on the
innovator's proprietary data in order to obtain marketing approval
or authorizations for a follow-on ‘biosimilar’ or generic drug
product. A biosimilar product is a follow-on version of an
innovator’s biological product.
“PharmaCyte’s cancer product candidates are
considered biological products because the capsules that are part
of those products contain living, albeit genetically altered, human
cells. Biological products include a wide range of products such as
vaccines, blood and blood components, allergenics, somatic cells,
gene therapies, tissues and recombinant therapeutic proteins. The
BPCIA created an abbreviated licensure pathway for biological
products shown to be biosimilar to, or interchangeable with, an
FDA-licensed biological reference product.
“The BPCIA establishes a period of 12 years of
data exclusivity for reference products in order to preserve
incentives for future innovation. Under this framework, data
exclusivity protects the data in the innovator’s regulatory
application by prohibiting others, for a period of 12 years, from
gaining FDA approval based in part on reliance on or reference to
the innovator’s data in their biosimilar application. PharmaCyte
anticipates its 12-year exclusivity will begin as soon as the FDA
approves its pancreatic cancer product candidate.
“Countries in the European Union (EU) also
provide for such data protection. Further, in October 2015 it was
agreed as part of the Trans-Pacific Partnership trade deal between
the United States, Australia, Brunei, Darussalam, Canada, Chile,
Japan, Malaysia, Mexico, New Zealand, Peru, Singapore and Vietnam
that biologic drugs will be given a minimum of 5 years data
exclusivity.”
Can you explain how the Orphan Drug
designation that PharmaCyte has received in both the United States
and in the European Union is a major source of protection for
PharmaCyte’s pancreatic cancer therapy?
“This is the second of the two protections I
spoke of earlier. PharmaCyte’s pancreatic cancer product candidate
was designated an orphan drug and listed in the official registry
of medicinal products for rare diseases by the FDA on December 17,
2014. This orphan drug status assures market exclusivity for
PharmaCyte in the United States for 7 years after market approval.
Similarly, PharmaCyte has orphan drug status in the EU for its
pancreatic cancer product candidate. This designation provides 10
years of market exclusivity in all of the countries in the EU and
assistance from the EMA in the product development.
“So it should be understood that once we gain
market approval, our pancreatic cancer therapy will have exclusive
protections with both the regulatory data protection and the orphan
drug designations. In addition, there are a number of
know-how/trade secrets and trademark protections built in that we
believe will make it exceedingly difficult for any company or
entity to ever duplicate our pancreatic cancer therapy and complete
clinical trials before we reach market approval. Conservative
estimates have indicated that, given the complex nature of the
trade secrets/know-how associated with our IP portfolio, we may be
able to extend the protection of our IP portfolio by at least
several years on this basis alone.”
In addition to PharmaCyte’s pancreatic
cancer therapy, PharmaCyte also has a diabetes therapy that it is
developing that will need protecting. Can you discuss how you’ll
protect the therapy that consists of
Cell-in-a-Box® and Melligen
cells?
“PharmaCyte has a License Agreement with the
University of Technology Sydney (UTS) in Australia that provides
PharmaCyte with an exclusive worldwide right to use genetically
modified human liver cells called ‘Melligen cells.’ Those cells
have been modified to contain pancreatic islet cell glucokinase for
use in developing a treatment for Type 1 diabetes and
insulin-dependent Type 2 diabetes. The Melligen cells are protected
by a patent issued in the EU that is in the process of being
validated in each of the major countries. In addition, there is a
patent pending in the United States. The License Agreement also
provides PharmaCyte with the non-exclusive worldwide rights to
‘know-how’ associated with the Melligen cells.
“PharmaCyte also licensed from Austrianova the
exclusive, worldwide rights to use the Cell-in-a-Box®
cellulose-based live-cell encapsulation technology for the
development of a treatment for diabetes and the use of
Austrianova’s Cell-in-a-Box® trademark for this technology. The
diabetes Licensing Agreement grants PharmaCyte exclusive worldwide
rights to use the Cell-in-a-Box® technology with genetically
modified or non-modified non-stem cell lines, designed to produce
insulin and/or other critical components for the treatment of
diabetes.”
In simple terms, how would you summarize
PharmaCyte’s strategy for protecting its IP portfolio?
“PharmaCyte has several diverse avenues
available to it for protecting its IP portfolio. We plan to pursue
all of these in order to extend our IP portfolio to the greatest
extent possible.”
About PharmaCyte BiotechPharmaCyte Biotech is a
clinical stage biotechnology company focused on developing and
preparing to commercialize therapies for cancer and diabetes based
upon a proprietary cellulose-based live cell encapsulation
technology known as “Cell-in-a-Box®.” This unique and patented
technology will be used as a platform upon which treatments for
several types of cancer and diabetes are being developed.
PharmaCyte’s treatment for cancer involves encapsulating
genetically modified live cells that convert an inactive
chemotherapy drug into its active or “cancer-killing” form. These
encapsulated live cells are placed as close to a cancerous tumor as
possible. Once implanted in a patient, a chemotherapy drug, which
needs to be activated in the body (ifosfamide), is then given
intravenously at one-third the normal dose. The ifosfamide is
carried by the circulatory system to where the encapsulated cells
have been placed. When the ifosfamide, which is normally activated
in the liver, comes in contact with the encapsulated live cells,
activation of the chemotherapy drug takes place at the source of
the cancer without any side effects from the chemotherapy. This
“targeted chemotherapy” has proven remarkably effective and safe to
use in past clinical trials.
In addition to developing a novel treatment for
cancer, PharmaCyte is developing a treatment for Type 1 diabetes
and Type 2 insulin-dependent diabetes. PharmaCyte plans to
encapsulate a human cell line that has been genetically engineered
to produce, store and release insulin in response to the levels of
blood sugar in the human body. The encapsulation will be done using
the Cell-in-a-Box® technology.
Safe Harbor This press release may contain
forward-looking statements regarding PharmaCyte Biotech and its
future events and results that involve inherent risks and
uncertainties. The words "anticipate," "believe," "estimate,"
"expect," "intend," "plan" and similar expressions, as they relate
to PharmaCyte or its management, are intended to identify
forward-looking statements. Important factors, many of which are
beyond the control of PharmaCyte, could cause actual results to
differ materially from those set forth in the forward-looking
statements. They include PharmaCyte's ability to continue as a
going concern, delays or unsuccessful results in preclinical and
clinical trials, flaws or defects regarding its product candidates,
changes in relevant legislation or regulatory requirements,
uncertainty of protection of PharmaCyte’s intellectual property and
PharmaCyte’s continued ability to raise capital. PharmaCyte does
not assume any obligation to update any of these forward-looking
statements.
More information about PharmaCyte Biotech can be
found at www.PharmaCyte.com. It can also be obtained by contacting
Investor Relations.
Investor Relations:PharmaCyte Biotech,
Inc.Investor Relations DepartmentTelephone: 917.595.2856Email:
info@PharmaCyte.com