PLYMOUTH MEETING, Pa.,
July 29, 2020 /PRNewswire/
-- INOVIO (NASDAQ: INO) today announced that the U.S. Food and
Drug Administration (FDA) granted orphan drug designation for
INO-3107, its DNA medicine being evaluated in a Phase 1/2 trial for
treatment of recurrent respiratory papillomatosis (RRP).
RRP is a rare disease caused by human papillomavirus (HPV) types
6 and 11 infections. RRP causes noncancerous tumor growths leading
to life-threatening airway obstructions and can progress to cancer
in rare cases. Currently, the disease is incurable and is mostly
treated by surgery to remove the tumor growths to temporarily
restore the airway. The tumor almost always recurs and the surgery
must be repeated, often multiple times a year. RRP can severely
impact the quality of life for those living with the disease.
Orphan drug designation is intended to advance drug development
for rare diseases. FDA grants orphan drug status to medicines
intended for the prevention, diagnosis, and treatment of rare
diseases or conditions. In the United
States, an orphan disease is defined as a disease or
condition with a prevalence of less than 200,000 patients in
the United States annually. This
orphan drug designation from the FDA qualifies INO-3107 for various
development incentives, including a tax credit on expenditures
incurred in clinical studies, a waiver of the New Drug Application
(NDA) fee, research grant awarded by the FDA, and most importantly,
7 years of U.S. market exclusivity upon approval for the treatment
of RRP.
Ami Shah Brown, Ph.D., MPH,
INOVIO's Senior Vice President, Regulatory Affairs, said,
"Receiving FDA's orphan drug designation for INO-3107 is an
important milestone in the development of INOVIO's DNA medicine for
this rare disease and clearly underscores the importance of
addressing the unmet medical need for this debilitating
condition."
The open-label, multicenter INO-3107 Phase 1/2 trial is
currently open to enrollment to recruit 63 subjects in the U.S. and
will evaluate the efficacy, safety, tolerability, and
immunogenicity of INO-3107 in subjects with HPV 6 and/or
11-associated RRP who have required at least two surgical
interventions per year for the past three years for the removal of
associated papilloma(s). For this study, adult subjects will first
undergo surgical removal of their papilloma(s) and then receive
four doses of INO-3107, one every three weeks. The primary efficacy
endpoint will be a doubling or more in the time between surgical
interventions following the first dose of INO-3107 relative to the
frequency prior to study therapy.
Last year, INOVIO published data from its pilot clinical study
of INO-3106 (DNA medicine candidate targeting HPV 6-caused RRP) in
the scientific journal Vaccines (MDPI). Study results
demonstrated that INO-3106 generated immunogenicity and engagement
and expansion of an HPV 6-specific cellular response, including
cytotoxic T cells. The paper also showed that INOVIO's
immunotherapy allowed two out of two patients who previously
required approximately two surgeries per year for several years to
manage this disease to delay the need for surgery to a robust
degree; one patient was able to delay surgery for over a year and a
half (584 days surgery-free) and a second remained surgery-free for
over two and a half years (over 915 days surgery-free).
About RRP
Recurrent respiratory papillomatosis (RRP) is a rare disease
(estimated at 15,000 active cases in the U.S.) that is
characterized by the growth of tumors in the respiratory tract
caused by the human papillomavirus. Although benign, papillomas can
cause severe, even life-threatening airway obstruction and
respiratory complications. A distinguishing aspect of this disease
is the tendency for the papilloma to recur after surgical
procedures to remove them. Left untreated, if RRP develops in the
lungs, affected individuals can potentially experience recurrent
pneumonia, chronic lung disease (bronchiectasis) and, ultimately,
progressive pulmonary failure. In rare cases papillomas can become
cancerous (malignant transformation) developing into squamous cell
carcinoma. Additional symptoms of RRP can include hoarse voice,
difficulty in sleeping and swallowing, and chronic coughing. RRP
symptoms are usually more severe in children than in adults. In
children, the disorder is most often diagnosed at or around the age
of four years. In adults, the disorder occurs most often in the
third or fourth decade.
About INOVIO's DNA Medicines Platform
INOVIO has 15 DNA medicine clinical programs currently in
development focused on HPV-associated diseases, cancer, and
infectious diseases, including coronaviruses associated with MERS
and COVID-19 diseases being developed under grants from the
Coalition for Epidemic Preparedness Innovations (CEPI) and the U.S.
Department of Defense. DNA medicines are composed of optimized DNA
plasmids, which are small circles of double-stranded DNA that are
synthesized or reorganized by a computer sequencing technology and
designed to produce a specific immune response in the body.
INOVIO's DNA medicines deliver optimized plasmids directly into
cells intramuscularly or intradermally using INOVIO's proprietary
hand-held smart device called CELLECTRA®. The CELLECTRA
device uses a brief electrical pulse to reversibly open small pores
in the cell to allow the plasmids to enter, overcoming a key
limitation of other DNA and other nucleic acid approaches, such as
mRNA. Once inside the cell, the DNA plasmids enable the cell to
produce the targeted antigen. The antigen is processed naturally in
the cell and triggers the desired T cell and antibody-mediated
immune responses. Administration with the CELLECTRA device ensures
that the DNA medicine is efficiently delivered directly into the
body's cells, where it can go to work to drive an immune response.
INOVIO's DNA medicines do not interfere with or change in any way
an individual's own DNA. The advantages of INOVIO's DNA medicine
platform are how fast DNA medicines can be designed and
manufactured; the stability of the products, which do not require
freezing in storage and transport; and the robust immune response,
safety profile, and tolerability that have been demonstrated in
clinical trials.
With more than 2,000 patients receiving INOVIO investigational
DNA medicines in more than 7,000 applications across a range of
clinical trials, INOVIO has a strong track record of rapidly
generating DNA medicine candidates with potential to meet urgent
global health needs.
About INOVIO
INOVIO is a biotechnology company focused on rapidly bringing to
market precisely designed DNA medicines to treat and protect people
from infectious diseases, cancer, and diseases associated with HPV.
INOVIO is the first and only company to have clinically
demonstrated that a DNA medicine can be delivered directly into
cells in the body via a proprietary smart device to produce a
robust and tolerable immune response. Specifically, INOVIO's lead
candidate VGX-3100, currently in Phase 3 trials for precancerous
cervical dysplasia, destroyed and cleared high-risk HPV 16 and 18
in a Phase 2b clinical trial.
High-risk HPV is responsible for 70% of cervical cancer, 91% of
anal cancer, and 69% of vulvar cancer. Also in development are
programs targeting HPV-related cancers and a rare HPV-related
disease, recurrent respiratory papillomatosis (RRP);
non-HPV-related cancers glioblastoma multiforme (GBM) and prostate
cancer; as well as externally funded infectious disease DNA vaccine
development programs in Zika, Lassa fever, Ebola, HIV, and
coronaviruses associated with MERS and COVID-19 diseases. Partners
and collaborators include Advaccine, ApolloBio Corporation,
AstraZeneca, The Bill & Melinda Gates Foundation, Coalition for
Epidemic Preparedness Innovations (CEPI), Defense Advanced Research
Projects Agency (DARPA)/Joint Program Executive Office for
Chemical, Biological, Radiological and Nuclear Defense
(JPEO-CBRND)/Department of Defense (DOD), GeneOne Life
Science/VGXI, HIV Vaccines Trial Network, International Vaccine
Institute (IVI), Medical CBRN Defense Consortium (MCDC), National
Cancer Institute, National Institutes of Health, National Institute
of Allergy and Infectious Diseases, Ology Bioservices, the Parker
Institute for Cancer Immunotherapy, Plumbline Life Sciences,
Regeneron, Richter-Helm BioLogics, Roche/Genentech, University of Pennsylvania, Walter Reed Army
Institute of Research, and The Wistar Institute. INOVIO also is a
proud recipient of 2020 Women on Boards "W" designation recognizing
companies with more than 20% women on their board of directors. For
more information, visit www.inovio.com.
CONTACTS:
Media: Jeff Richardson,
267-440-4211, jrichardson@inovio.com
Investors: Ben Matone, 484-362-0076,
ben.matone@inovio.com
This press release contains certain forward-looking
statements relating to our business, including our plans to develop
DNA medicines, our expectations regarding our research and
development programs, including the planned initiation and conduct
of preclinical studies and clinical trials, and the
availability and timing of data from those studies and trials.
Actual events or results may differ from the expectations set forth
herein as a result of a number of factors, including uncertainties
inherent in pre-clinical studies, clinical trials, product
development programs and commercialization activities and outcomes,
the availability of funding to support continuing research and
studies in an effort to prove safety and efficacy of
electroporation technology as a delivery mechanism or develop
viable DNA medicines, our ability to support our pipeline of DNA
medicine products, the ability of our collaborators to attain
development and commercial milestones for products we license and
product sales that will enable us to receive future payments and
royalties, the adequacy of our capital resources, the availability
or potential availability of alternative therapies or treatments
for the conditions targeted by us or our collaborators, including
alternatives that may be more efficacious or cost effective than
any therapy or treatment that we and our collaborators hope to
develop, issues involving product liability, issues involving
patents and whether they or licenses to them will provide us with
meaningful protection from others using the covered technologies,
whether such proprietary rights are enforceable or defensible or
infringe or allegedly infringe on rights of others or can withstand
claims of invalidity and whether we can finance or devote other
significant resources that may be necessary to prosecute, protect
or defend them, the level of corporate expenditures, assessments of
our technology by potential corporate or other partners or
collaborators, capital market conditions, the impact of government
healthcare proposals and other factors set forth in our Annual
Report on Form 10-K for the year ended December 31, 2019, our Quarterly Report on Form
10-Q for the quarter ended June 30,
2020 and other filings we make from time to time with the
Securities and Exchange Commission. There can be no assurance that
any product candidate in our pipeline will be successfully
developed, manufactured or commercialized, that final results of
clinical trials will be supportive of regulatory approvals required
to market products, or that any of the forward-looking information
provided herein will be proven accurate. Forward-looking statements
speak only as of the date of this release, and we undertake no
obligation to update or revise these statements, except as may be
required by law.
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SOURCE INOVIO Pharmaceuticals, Inc.