PR84946

PLYMOUTH MEETING, Pennsylvania, July 29, 2020 /PRNewswire=KYODO JBN/ --

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(R). 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, +1 267-440-4211, jrichardson@inovio.com

Investors: Ben Matone, +1 484-362-0076, 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.

SOURCE: INOVIO Pharmaceuticals, Inc.