PR87406

PLYMOUTH MEETING, Pa., Dec. 24, 2020 /PRNewswire=KYODO JBN/ --

-- Peer-reviewed Phase 1 data shows INO-4800 to be immunogenic in 100% of

subjects, inducing neutralizing antibody and/or T cell responses

-- INO-4800 demonstrates favorable safety and tolerability, with no serious

adverse events reported

-- Offers best-in-class thermostability, including a five-year projected shelf life at

normal refrigeration temperature and no frozen transport or storage requirements

INOVIO (NASDAQ:INO), a biotechnology company focused on bringing to market

precisely designed DNA medicines to treat and protect people from infectious

diseases and cancer, today announced the publication of peer-reviewed Phase 1

clinical data from the first cohort of 40 participants for its COVID-19 DNA

vaccine candidate, INO-4800, in EClinicalMedicine, an open access clinical

journal published by The Lancet.

The paper, titled "Safety and immunogenicity of INO-4800 DNA vaccine against

SARS-CoV-2: a preliminary report of an open-label, Phase 1 clinical trial,"

found that INO-4800 was immunogenic in all vaccinated subjects, effectively

generating an immune response of humoral (including neutralizing antibodies)

and/or cellular responses (both CD4 and CD8 T cells).

Additionally, Phase 1 clinical data found INO-4800 to have a favorable safety

and tolerability profile with no serious adverse events reported; only six

Grade 1 adverse events (AEs) were observed, primarily minor injection site

reactions. Notably, these only occurred on the day of the first or second

dosing, and the AEs did not increase in frequency with the second administration.

INO-4800, beyond being safe and tolerable, is stable at room temperature for

more than a year, at 37o C (98.6o F) for more than a month, has a five-year

projected shelf life at normal refrigeration temperature [i.e., at 2-8o C /

35.6 – 46.4o F] and does not need to be frozen during transport or storage –

all critical factors for timely global distribution in the fight against COVID-19.

Dr. J. Joseph Kim, President and CEO of INOVIO, said, "We are very pleased to

share peer-reviewed Phase 1 clinical data for INO-4800 published in The

Lancet's EClinicalMedicine, and are grateful for the support of all

participants and investigator staff involved in the clinical trial."

Dr. Stanley Plotkin, Professor Emeritus at The Wistar Institute, said,

"INOVIO's DNA vaccine appeared to be quite safe with few significant reactions

but yet induced both antibody and T cell responses to SARS-CoV-2."

Findings from the Phase 1 Clinical Trial

   -- The Phase 1 clinical trial of INO-4800 initially enrolled 40 healthy

      adult volunteers, ages 18 to 50, at two U.S. sites with funding from the

      Coalition for Epidemic Preparedness Innovations (CEPI).

   -- The participants were enrolled into 1.0 mg and 2.0 mg dose cohorts; each

      participant received two doses of INO-4800 four weeks apart. Each dose

      was administered by intradermal injection using INOVIO's proprietary

      smart device CELLECTRA(R).

   -- Thirty-nine subjects completed both doses. One subject in the 2.0 mg

      group discontinued trial participation prior to receiving the second dose

      due to lack of transportation to the clinical site; discontinuation was

      unrelated to the study or the dosing. One subject was deemed to be

      seropositive at trial entry.

   -- The 1.0 mg and 2.0 mg dose group both demonstrated seroconversion in 95%

      of the subjects, respectively, with 78% demonstrating neutralizing

      antibodies in the 1.0 mg dose group and 84% demonstrating neutralizing

      antibodies in the 2.0 mg dose group.

   -- Cellular (T cell) response were observed to multiple regions of the spike

      protein including the RBD region. 74% had measurable cellular responses

      at the 1.0 mg dose group and 100% of the subjects in the 2.0 mg dose

      group demonstrated cellular responses.

   -- Through week 8, no serious adverse events were reported. Only 6 related

      Grade 1 adverse events in 5 subjects were observed, primarily mild

      injection site reactions (e.g., redness); none of these increased in

      frequency with the second administration.

   -- All 38 subjects who were evaluable for immunogenicity had balanced

      cellular and humoral immune responses following the second dose of INO 4800.

   -- ClinicalTrials.gov identifier: NCT04336410.

INOVIO is currently conducting the Phase 2 segment of its planned Phase 2/3

clinical trial for INO-4800, called INNOVATE (INOVIO INO-4800 Vaccine Trial for

Efficacy). INNOVATE is a randomized, blinded, placebo-controlled safety and

efficacy trial of INO-4800 to be conducted in adults in the U.S. It will be

funded by the U.S. Department of Defense (DoD) Joint Program Executive Office

for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND) in

coordination with the Office of the Assistant Secretary of Defense for Health

Affairs (OASD(HA)) and the Defense Health Agency (DHA).

The DoD has agreed to provide funding for both the Phase 2 and Phase 3 segments

of the INNOVATE clinical trial, in addition to the $71 million of funding

previously announced in June 2020 for the large-scale manufacture of the

company's proprietary smart device CELLECTRA(R) 3PSP and the procurement of

CELLECTRA(R) 2000 devices.

INOVIO also recently announced (https://c212.net/c/link/?t=0&l=en&o=3022074-1&h=2277805837&u=http%3A%2F%2Fir.inovio.com%2Fnews-releases%2Fnews-releases-details%2F2020%2FINOVIO-and-Advaccine-Announce-First-Dosing-of-Subject-in-Phase-2-Clinical-Trial-for-COVID-19-DNA-Vaccine-Candidate-INO-4800-in-China%2Fdefault.aspx&a=announced )

the first dosing of its first subject in its Phase 2 clinical trial for INO-4800 in China,

in collaboration with Advaccine. The company is currently in Phase 1/2a trials for

INO-4800 in South Korea in partnership with The International Vaccine Institute and

the Korea National Institute for Health.

About the INO-4800 "INNOVATE" Phase 2/3 Clinical Trial

The lead Principal Investigator for the INNOVATE trial is Dr. Pablo Tebas,

Professor of Medicine at the Hospital of the University of Pennsylvania. The

Phase 2 segment of the clinical trial is designed to evaluate safety,

tolerability and immunogenicity of INO-4800 in a 2-dose regimen (1.0 mg or 2.0

mg), in a three-to-one randomization to receive either INO-4800 or placebo for

each dose, to confirm the more appropriate dose(s) for each of three age groups

(18-50 years, 51-64 years and 65 years and older) for the subsequent Phase 3

efficacy evaluation. The company intends to work diligently to ensure diversity

in enrollment, targeting specific populations that are working or residing in

environments with high infection rates and/or areas where there is greater risk

of exposure to SARS-CoV-2, for whom exposure may be relatively prolonged or for

whom personal protective equipment (PPE) may be inconsistently used, especially

in confined settings.

In the Phase 3 segment of the clinical trial, INOVIO intends to enroll healthy

men and non-pregnant women 18 years and older, to evaluate the efficacy of the

proposed dose(s) based on the data from the Phase 2 evaluation. Participants

will be enrolled in a one-to-one randomization to receive either INO-4800 or a

placebo. The Phase 3 segment will be case-driven with the final number of

enrollees to be determined by the incidence of COVID-19 during the Phase 3

segment. The primary endpoint of the Phase 3 segment will be virologically

confirmed COVID-19 disease.

About INOVIO's Global Coalition Advancing INO-4800

INOVIO has assembled a global coalition of collaborators, partners and funders

to rapidly advance the development of INO-4800. R&D collaborators to date

include The Wistar Institute, the University of Pennsylvania, the University of

Texas, Fudan University and Laval University. INOVIO has partnered with

Advaccine and the International Vaccine Institute to conduct clinical trials of

INO-4800 in China and South Korea, respectively. INOVIO is also assessing

nonclinical efficacy of INO-4800 in several animal challenge models with Public

Health England (PHE) and Commonwealth Scientific and Industrial Research

Organization (CSIRO) in Australia. INOVIO is working with a team of contract

manufacturers including Kaneka Eurogentec, Thermo Fisher Scientific,

Richter-Helm BioLogics, and Ology Bioservices to manufacture INO-4800 on a

commercial scale and is seeking additional external funding and partnerships to

further scale up manufacturing capacities to satisfy the urgent global demand

for safe and effective vaccines. To date, the Coalition for Epidemic

Preparedness Innovations (CEPI), the Bill & Melinda Gates Foundation, and the

U.S. Department of Defense have contributed significant funding to the

advancement and manufacturing of INO-4800.

About INO-4800

INO-4800 is INOVIO's DNA vaccine candidate against SARS-CoV-2, the novel

coronavirus that causes COVID-19. INOVIO has extensive experience working with

coronaviruses and was the first company to initiate a Phase 2a trial for

INO-4700, a DNA vaccine candidate for a related coronavirus that causes Middle

East Respiratory Syndrome (MERS).

Composed of an optimized DNA plasmid, INO-4800 is delivered directly into cells

in the body via a proprietary smart device to produce a robust, safe and

tolerable immune response. INO-4800 is the only nucleic-acid based vaccine that

is stable at room temperature for more than a year, at 37o C for more than a

month, has a five-year projected shelf life and does not need to be frozen in

transport of storage, which are important factors when implementing mass immunizations.

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, 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(R) 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(R) 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 observed 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), HIV Vaccines Trial Network, International Vaccine Institute

(IVI), Kaneka Eurogentec, 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,

Thermo Fisher Scientific, 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 and manufacture DNA medicines, our

expectations regarding our research and development programs, including the

conduct of the Phase 2/3 clinical trial of INO-4800, and our ability to

successfully manufacture and produce large quantities of our product candidates

if they receive regulatory approval. Actual events or results may differ from

the expectations set forth herein as a result of a number of factors, including

uncertainties inherent in preclinical studies, clinical trials, product

development programs and commercialization activities and outcomes, our ability

to secure sufficient manufacturing capacity to mass produce our product

candidates, 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 September 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.