Country for PR: United States
Contributor: PR Newswire New York
Thursday, January 07 2021 - 00:00
INOVIO's VGX-3100 Demonstrates Positive Phase 2 Efficacy In Treatment of Precancerous Vulvar Dysplasia Caused by HPV-16/18
PLYMOUTH MEETING, Pa., Jan. 6, 2021 /PRNewswire-AsiaNet/ --

  -- DNA immunotherapy candidate VGX-3100 demonstrated clinically 
     significant reduction of HPV-16/18-associated precancerous vulvar 
     lesions in 63% of treated participants six months after treatment
  -- Phase 2 results indicate that VGX-3100 can be a safe, tolerable and
     efficacious adjunct to current standard of care for women with 
     vulvar dysplasia
  -- Phase 3 trial planned in 2021

INOVIO (NASDAQ:INO), a biotechnology company focused on bringing to market 
precisely designed DNA medicines to treat and protect people from infectious 
diseases, cancer and HPV-associated diseases, today announced positive efficacy 
results for an open-label Phase 2 trial of VGX-3100 to treat HPV-16 and 
HPV-18-associated vulvar dysplasia. A 25% or more reduction in 
HPV-16/18-associated vulvar HSIL (high-grade squamous intraepithelial lesion) 
was observed for 63% of trial participants (12 of 19) treated with VGX-3100 at 
six months post-treatment. Three out of the 20 participants with histology data 
(15%) resolved their vulvar HSIL and had no HPV-16/18 virus detectable in the 
healed area. By comparison, the spontaneous resolution of vulvar HSIL caused by 
HPV-16/18 is estimated to be only 2%. The trial also showed VGX-3100 to be safe 
and well-tolerated. Based upon these results INOVIO is planning to pursue Phase 
3 development.

Dr. Robert Edwards, MD, Milton Lawrence McCall Professor and Chair, Department 
of Obstetrics, Gynecology & Reproductive Sciences, University of Pittsburgh and 
Principal Coordinating Investigator for the Phase 2 clinical trial said, "These 
Phase 2 efficacy results are a very promising non-surgical advance for a 
recalcitrant disease that normally requires repetitive ablation and excision 
procedures to achieve disease and risk reduction, and may offer a more safe, 
tolerable and efficacious treatment option for patients."

Prakash Bhuyan, M.D., Ph.D., Senior Vice President and Head of HPV Therapeutic 
Clinical Development at INOVIO, said, "The reduction of precancerous disease 
offers a meaningful improvement in the management of this devastating disease 
and is a step forward towards the enhancement of women's healthcare."

VGX-3100 Phase 2 Vulvar Dysplasia Trial Highlights
     - Trial participants were 24 women between 22 and 70 years of age 
       at entry and other than having HSIL were healthy. 
     - 80% (20/25) had VIN3 disease (the more severe form of VIN) and 
       88% (22/25) had a history of 1 or more recurrences 
     - There were no discontinuations due to adverse events; five 
       participants elected to withdraw for reasons unrelated to 
       adverse events. 
     - 19 participants had digital photography data, and 12 (63%) 
       had clinically significant lesion reduction as defined by a 
       reduction of lesion area of more than 25%. 
     - 20 participants had histology and virology data and 3 (15%) 
       resolved their vulvar HSIL and had no HPV-16/18 virus detectable 
       in the healed area. 
     - Histology was assessed by up to two independent pathologists and 
       non-detectability of HPV-16/18 from lesion tissue using PCR 
       (polymerase chain reaction)-based testing, at six months following 
       the administration of VGX-3100. 
     - Efficacy endpoints were measured six months post-treatment. 
     - Safety will continue to be assessed for 18 months following the 
       last dose. 
     - The most observed adverse event was injection site pain, the 
       majority of which were mild-to-moderate. 
     - No cases of vulvar cancer have been observed in the trial. 

For more information about the Phase 2 clinical trial, please visit (search identifier NCT03180684).

About Vulvar Dysplasia

Vulvar dysplasia is a rare disease that affects women in immunocompetent and 
immunocompromised populations. Fewer than 2% of women with HPV-16/18-associated 
precancerous vulvar dysplasia exhibit spontaneous resolution. 
HPV-16/18-associated precancerous vulvar lesions (vulvar HSIL) are known to be 
difficult to treat, and typically require repeat ablation and excision 
procedures to achieve disease and risk reduction. Without adequate treatment 
vulvar HSIL can progress to vulvar cancer. In 2017, vulvar cancer was diagnosed 
in 1354 women in the U.S. and 411 died from vaginal cancer.1

About VGX-3100

VGX-3100 is a DNA medicine in clinical trials for the treatment of three 
HPV-16/18 related disease states – anal dysplasia, vulvar dysplasia and 
cervical dysplasia. The cervical dysplasia program is in late Phase 3 clinical 
trials (REVEAL1 and REVEAL2). VGX-3100 is designed to utilize the patient's own 
immune system to clear HPV-16/18-associated high-grade precancerous lesions 
with the aim of reducing the risk of cancer.

About INOVIO's HPV-Associated DNA Medicines Clinical Programs

This Phase 2 clinical trial builds on significant clinical benefits 
demonstrated with INOVIO's HPV-associated DNA medicines across multiple 
clinical trials. Specifically, VGX-3100 in a Phase 2 proof-of-concept trial for 
cervical dysplasia demonstrated a response in 43 out of 107 patients in 
regression of high-grade cervical lesions and elimination of the underlying HPV 
infection2. A Phase 2 trial of VGX-3100 in the treatment of anal HSIL was also 
conducted with positive efficacy results3. Additionally, two out of four 
metastatic HPV-associated head and neck cancer patients treated with MEDI0457 
and a PD-1 check point inhibitors in a Phase 1 trial experienced a long-term 
complete response for more two years and counting; 18 out of 22 head and neck 
patients treated with MEDI0457 have not progressed for over four years 
post-treatment. Lastly, a pilot study of INOVIO's DNA medicine INO-3107 in 
recurrent respiratory papillomatosis (RRP) resulted in two out of two patients 
delaying surgery due to lack of tumor recurrence.

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 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.


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


Investors: Ben Matone, 484-362-0076, 
Media: Jeff Richardson, 267-440-4211,

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, 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.

1 accessed on 04JAN2021
2 Trimble et al., Lancet, 386, 10008, P2078-2088, 2015;
3 Presented at ASCCP 2020 Scientific Meeting

Source - INOVIO Pharmaceuticals, Inc.