PR82814

Inovio Receives Authorization from the U.S. FDA To Begin Phase 1/2 Clinical Trial for INO-3107, a DNA Medicine To Treat a Rare Disease -- Recurrent Respiratory Papillomatosis (RRP)

PLYMOUTH MEETING, Pennsylvania, Feb. 10, 2020 /PRNewswire=KYODO JBN/ --

-- RRP is a rare, potentially fatal orphan disease caused by Human

Papillomavirus (HPV) 6 and 11

-- Inovio's DNA medicine pipeline includes 15 clinical programs focused on

HPV-associated diseases, cancer, and infectious diseases, including the novel

coronavirus (2019-nCoV)

Inovio Pharmaceuticals, Inc. (NASDAQ: INO) today announced the U.S. Food and

Drug Administration (FDA) has accepted its Investigational New Drug (IND)

application to evaluate its DNA medicine INO-3107 in a Phase 1/2 trial for

treatment of Recurrent Respiratory Papillomatosis or RRP. RRP is a rare disease

caused by the human papillomavirus (HPV) types 6 and 11 infections, a condition

that causes noncancerous tumor growths leading to life-threatening airway

obstructions, and occasionally can progress to cancer. Currently, the disease

is incurable and is mostly treated by surgery, which temporarily restores 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.

The open-label, multicenter Phase 1/2 trial will enroll approximately 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. Upon obtaining sufficient safety and potential efficacy data in

adults, Inovio plans to expand the trial to include pediatric patients as well

as a potential booster regimen.

"Inovio's investigational DNA medicine INO-3107 is designed to destroy and

clear tumors caused by HPV 6 and 11 infections from the body exactly where they

are hiding," said Jeffrey Skolnik, M.D., Inovio's Vice President of Clinical

Development. "We believe this DNA medicine has the potential to provide people

living with RRP a long-term, if not life-long, improvement in their disease,

especially as an alternative to often successive and debilitating surgeries

that may temporarily remove HPV growths from the airways but do not address the

underlying recurring virus."

J. Joseph Kim, Ph.D., Inovio's President and Chief Executive Officer, said "Our

mission at Inovio is to rapidly provide patients with urgent health needs

access to our novel DNA medicines. We are pleased the FDA has authorized our

INO-3107 clinical trial, and look forward to working closely with the RRP

patient and medical community to drive recruitment as quickly as possible."

In addition to initiating this efficacy trial, Inovio also plans to attain

Orphan Disease designation with the FDA's Office of Orphan Products Development

(OOPD). The FDA grants orphan status to drugs and biologic products that are

intended for the safe and effective treatment, diagnosis, or prevention of rare

diseases or disorders that affect fewer than 200,000 people in the United

States. OOPD provides a drug developer with certain benefits and incentives,

including a period of marketing exclusivity if regulatory approval is

ultimately received for the designated indication.

Inovio recently published data from its pilot clinical study of INO-3106 (DNA

medicine candidate targeting HPV6 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; with one patient able to

delay surgery for over a year and a half (584 days surgery-free) and a second

that remained surgery-free for over two and a half years (over 915 days

surgery-free).

About Inovio's DNA Medicines

Inovio has 15 DNA medicine clinical programs currently in development focused

on HPV-associated diseases, cancer, and infectious diseases, including the

novel coronavirus (2019-nCoV) under a grant from the Coalition for Epidemic

Preparedness Innovations (CEPI). DNA medicines are medicines 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). CELLECTRA uses a brief electrical pulse to open

small pores in the cell reversibly to allow the plasmids to enter. Once inside

the cell, the plasmids begin replicating, thereby strengthening the body's own

natural response mechanisms. Administration with the CELLECTRA device ensures

that the DNA medicine is delivered directly into the body's cells, where it can

go to work immediately mounting an immune response. Inovio's DNA medicines are

not interfering with or changing in any way an individual's own DNA, which is

the case with gene therapy or gene editing.

With more than 2,000 patients receiving Inovio investigational DNA medicines in

more than 6,000 applications across a range of clinical trials, Inovio's DNA

medicines have consistently activated safe, robust, and fully functional T cell

and antibody responses against targeted pathogens and cancers.

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 extremely

rare cases (less than 1%), 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 Pharmaceuticals, Inc.

Inovio is a biotechnology company focused on rapidly bringing to market

precisely designed DNA medicines to treat, cure, and protect people from

diseases associated with HPV, cancer, and infectious diseases. 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

safely produce a robust immune response to destroy and clear high-risk HPV 16

and 18, which are responsible for 70% of cervical cancer, 90% of anal cancer

and 69% of vulvar cancer. In addition to HPV, Inovio's optimized plasmid design

and delivery technology have been demonstrated to consistently activate robust

and fully functional T cell and antibody responses against targeted cancers and

pathogens. Inovio's most advanced clinical program, VGX-3100, is in Phase 3

development for the treatment of HPV-related cervical pre-cancer. Also in

development are Phase 2 immuno-oncology programs targeting HPV-related cancers

and GBM, as well as externally funded vaccine development programs in Zika,

MERS, Lassa, HIV, and the novel coronavirus (2019-nCoV). Partners and

collaborators include ApolloBio Corporation, AstraZeneca, The Bill & Melinda

Gates Foundation, Coalition for Epidemic Preparedness Innovations (CEPI),

Defense Advanced Research Projects Agency, GeneOne Life Science, HIV Vaccines

Trial Network, Medical CBRN Defense Consortium (MCDC), National Cancer

Institute, National Institutes of Health, National Institute of Allergy and

Infectious Diseases, Regeneron, Roche/Genentech, University of Pennsylvania,

Walter Reed Army Institute of Research, and The Wistar Institute. For more

information, visit www.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, as well as commercialization

activities, including the planned initiation and conduct of clinical trials,

the availability and timing of data from those trials and our commercialization

strategy and tactics. 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 vaccines, our ability to support our pipeline of SynCon(R) active

immunotherapy and vaccine 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, 2018,

our Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, 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.

CONTACTS:   

Investors: Ben Matone, Inovio, +1 484-362-0076, ben.matone@inovio.com

Media: Jeff Richardson, Inovio, +1 267-440-4211, jrichardson@inovio.com

SOURCE: Inovio Pharmaceuticals, Inc.