Country for PR: United States
Contributor: PR Newswire New York
Tuesday, June 30 2020 - 21:30
INOVIO Announces Positive Interim Phase 1 Data For INO-4800 Vaccine for COVID-19
PLYMOUTH MEETING, Pennsylvania, June 30, 2020 /PRNewswire-AsiaNet/--

-- INO-4800 Selected for the U.S. Government's Operation Warp Speed 

-- 94% of Phase 1 trial participants demonstrated overall immune responses at 
Week 6 after two doses of INO-4800 in trial with 40 healthy volunteers in 
preliminary analyses

-- Through Week 8 INO-4800 regimen was deemed safe and well-tolerated with no 
serious adverse events; all reported adverse events were grade 1 in severity

-- In preclinical animal challenge study, INO-4800 provided full protection 
against SARS-CoV-2 replication in the lungs in mice challenged with the virus

-- INOVIO to begin U.S. Phase 2/3 efficacy study this summer upon regulatory 

INOVIO (NASDAQ:INO), a biotechnology company focused on rapidly bringing to 
market precisely designed DNA medicines to protect and treat people from 
infectious diseases and cancer, today announced positive interim clinical data 
of INO-4800, its vaccine candidate against novel coronavirus (SARS-CoV-2), from 
the first two Phase 1 clinical trial cohorts. In addition, INO-4800 has been 
selected to participate in a non-human primate (NHP) challenge study as part of 
the U.S. government's Operation Warp Speed, a new national program aiming to 
provide substantial quantities of safe, effective vaccine for Americans by 
January 2021. Furthermore, INOVIO has expanded its Phase 1 trial to add older 
participants in additional cohorts and plans to initiate a Phase 2/3 efficacy 
trial this summer upon regulatory concurrence.

Dr. J. Joseph Kim, President and CEO of INOVIO, said, "INOVIO would like to 
thank all of the trial participants and the investigator staff who have made 
this trial possible. We are very encouraged by the positive interim safety and 
preliminary cellular and humoral immune response results to date as well as the 
inclusion of INO-4800 in Operation Warp Speed. We are also pleased that 
INO-4800 vaccination abrogated viral replication in the lungs of mice 
challenged with SARS-CoV-2. We look forward to urgently advancing INO-4800, as 
it is the only nucleic-acid based vaccine that is stable at room temperature 
for more than a year and does not require to be frozen in transport or for 
years of storage, which are important factors when implementing mass 
immunizations to battle the current pandemic."

The Phase 1 clinical trial of INO-4800 initially enrolled 40 healthy adult 
volunteers 18 to 50 years of age 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 CELLECTRA(R) 2000 device. An independent Data Safety 
Monitoring Board reviewed the safety data. INO-4800 was generally safe and 
well-tolerated in all participants in both cohorts through week 8; all ten 
reported adverse events (AEs) were grade 1 in severity, and most were local 
injection site redness. There were no reported serious adverse events (SAEs).

Multiple immunology assays including those for humoral and cellular immune 
responses are being conducted for both 1.0 mg and 2.0 mg dose cohorts after two 
doses at week 6. Analyses to date have shown that 94% (34 out of 36 total trial 
participants) demonstrated overall immunological response rates based on 
preliminary data assessing humoral (binding and neutralizing) and T cell immune 
responses. One participant in the 1.0 mg dose cohort and two participants in 
the 2.0 mg dose cohort were excluded in the immune analyses as they tested 
positive for COVID-19 immune responses at study entry, indicating prior 
infection. One participant in the 2.0 mg dose cohort discontinued the study for 
reasons unrelated to safety or tolerability. INOVIO plans to publish the full 
data set in a peer-reviewed medical journal.

One key feature of INOVIO's DNA vaccines is the ability to generate balanced 
antibody and T cell immune responses, which in the case of SARS-CoV-2 infection 
could be important in the development of potential COVID-19 vaccines. In this 
regard, recent scientific reports have highlighted that SARS-CoV-2-specific T 
cells found in convalescent patients have been positively implicated in 
controlling the severity of their COVID-19 disease (Grifoni et al, Cell 2020) 
while other studies have shown that a significant proportion (33% to 40%) of 
convalescent individuals in their reports had neutralizing antibody below 
detectable levels (Robbiani et al, Nature 2020 and Payne et al, MMWR 2020).

In addition to positive interim Phase 1 data, INO-4800 has been shown to 
protect mice in SARS-CoV-2 viral challenge studies, where vaccination with 
INO-4800 prevented viral replication in the lungs of animals challenged with 
SARS-CoV-2. Moreover, INO-4800 is currently being tested in a ferret challenge 
model as well as in NHP challenge studies as part of Operation Warp Speed.

"While the pathophysiologic profile of SARS-CoV-2 is not completely understood, 
research and clinical studies suggest that both T cell and antibody immune 
responses will be important for protection in both mild and serious infections. 
Leveraging our previous expertise in MERS with INO-4700, where we demonstrated 
significant antibody and cellular responses, the breadth and profile of the 
responses observed to date with INO-4800 targeting SARS-CoV-2 provide a 
promising read towards further development and addressing the existing public 
health threat," said Dr. Kate Broderick, Senior Vice President of R&D at INOVIO.

As previously announced, INOVIO received $71 million funding from the U.S. 
Department of Defense to support the large-scale manufacture of the company's 
proprietary CELLECTRA® 3PSP smart device and the procurement of CELLECTRA® 2000 
devices. INO-4800 development has also been supported by generous funding from 
CEPI and the Bill & Melinda Gates Foundation.

About INO-4800

INO-4800 is INOVIO's DNA vaccine candidate being developed to protect against 
the novel coronavirus SARS-CoV-2, which causes COVID-19. INO-4800 was designed 
rapidly using INOVIO's proprietary DNA medicine platform after the publication 
of the genetic sequence of the coronavirus that causes COVID-19. INOVIO has 
extensive experience working with coronaviruses and is the only company with a 
Phase 2 vaccine for a related coronavirus that causes Middle East Respiratory 
Syndrome (MERS). 

INO-4800 is the only nucleic-acid based vaccine that is stable at room 
temperature for more than a year and does not require to be frozen in transport 
or 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 diseases being developed under 
grants from the Coalition for Epidemic Preparedness Innovations (CEPI) and the 
DoD. 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 
intradermally or intramuscularly 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 is designed to ensure 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.


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

          Jeff Richardson

          Ben Matone

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 availability and 
timing of data from the company's ongoing Phase 1 clinical trial of INO-4800 
and the company's plans and ability to outsource manufacturing of its delivery 
devices to contract manufacturers. 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, our ability to 
secure adequate third-party manufacturing resources for the production of our 
product candidates, including the transfer of necessary processes, 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 March 31, 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.