Country for PR: United States
Contributor: PR Newswire New York
Friday, March 27 2020 - 00:00
INOVIO Reports Positive Interim Phase 2 VGX-3100 Results In Patients with HPV-associated Anal Dysplasia
PLYMOUTH MEETING, Pennsylvania, March 26, 2020 /PRNewswire-AsiaNet/ --

- VGX-3100 DNA medicine cleared high-risk HPV 16/18-associated precancerous 
lesions in 50% of patients, and reduced the number of lesions in 75% of patients

- Data to be presented at the 2020 American Society for Colposcopy and Cervical 
Pathology (ASCCP) meeting 

INOVIO Pharmaceuticals, Inc. (NASDAQ: INO) today announced positive interim 
results from an open-label, Phase 2 study showing its lead DNA medicine 
candidate VGX-3100 to be safe and effective in treating men and women with anal 
dysplasia, also known as high grade squamous intraepithelial lesion (HSIL), a 
precancerous condition caused by high-risk human papillomavirus (HPV) types 
16/18. Of the 20 subjects who had results at the time of data review, 50% (10 
of 20 subjects) showed clearance of HPV-16/18 associated precancerous lesions 
and 75% (15 of 20 subjects) demonstrated an overall decrease in the number of 
lesions 6 months after the start of treatment. No cases of anal cancer have 
been observed in the trial. The results support the ability of VGX-3100 to 
effectively treat multiple HPV associated diseases and will be presented in the 
virtual session of the annual American Society for Colposcopy and Cervical 
Pathology (ASCCP) meeting titled: Preliminary Results of an Open-label Phase 2 
Study of VGX-3100 for the Treatment of HPV16 and/or HPV-18 (HPV16/18) related 
Anal HSIL.

Anal dysplasia or anal HSIL, is an orphan disease that affects men and women in 
both immunocompetent and immunocompromised populations. Fewer than 1 in 5 
people with HPV 16/18-associated precancerous dysplasia exhibit spontaneous 
resolution at one year.  Without adequate treatment anal HSIL could progress to 
anal cancer. HPV 16/18 cause more than 90% of all anal cancer, which is now 
considered one the most rapidly rising causes of cancer incidence and 
mortality. According to the American Cancer Society, anal cancer will claim the 
lives of more than 1,300 people in the U.S. and 8,590 news cases (5,900 in 
women and 2,690 in men) will be diagnosed in 2020 and according to a study 
published November 2019 in the Journal of the National Cancer Institute, from 
2001 to 2015 the overall incidence ( 
) of anal cancer increased by 2.7% per year and mortality jumped by 3.1% each 
year ( 

Prakash Bhuyan, M.D., Ph.D., INOVIO's Vice President and Head of HPV 
Therapeutic Clinical Development said, "These initial proof-of-concept efficacy 
results show that VGX-3100 has the potential to enable the immune system to 
clear HPV 16/18 HSIL that cause precancerous anal dysplasia and are consistent 
with the results of our VGX-3100 Phase 2b efficacy study in high-risk 
HPV-associated precancerous cervical dysplasia."

Currently, the treatments for anal dysplasia are surgical excision, 
electro-cautery or laser therapy. Up to 50% of those treated with these 
invasive options experience disease recurrence within one year of treatment and 
nearly 70% experience recurrence within three years. Therefore, many patients 
with this condition need multiple invasive treatments.

VGX-3100 Safety and Efficacy Highlights for Anal Dysplasia

  -- The first 20 subjects on VGX-3100 demonstrated safety results consistent
     with the known safety profile of VGX-3100. There were no drug-related
     serious adverse events. 
  -- 75% showed an overall decrease in the number of lesions 6 months after
     treatment and 50% of subjects showed no HPV-16/18 associated precancerous
  -- Results further support proof of concept for DNA medicines as also
     demonstrated in prior VGX-3100 Phase 2b study in high-risk HPV-associated
     precancerous cervical dysplasia 

"These results are very encouraging and represent a potential important step 
forward in the treatment of precancerous anal dysplasia," said Dr. Céline 
Bouchard, Gynecologist/ Anoscopist at Centre Médical Santé Femme in Québec 
City, Canada, former Associate Clinical Professor in the Department of 
Obstetrics and Gynecology and Reproduction of Université Laval, and 
Coordinating Principal Investigator for the study. "Having a DNA medicine that 
can destroy and clear lesions without the burden of repetitive, multiple, and 
painful surgical or invasive treatments would change the standard of care and 
provide patients with a meaningful benefit. I look forward to continuing this 
research and learning more about the potential advantages of VGX-3100."

About the Study

This open-label, multi-center Phase 2 study is designed to evaluate the safety 
and efficacy of VGX-3100 in adults with precancerous anal dysplasia caused by 
HPV-16 and/or HPV-18. Twenty three subjects who are human immunodeficiency 
virus (HIV) negative with histologically confirmed anal or anal/peri-anal 
high-grade squamous intraepithelial lesion (HSIL) associated with human 
papilloma virus (HPV)-16 and/or HPV-18 enrolled. One subject discontinued due 
to an unrelated adverse event. Twenty two received three doses of VGX-3100 
administered by INOVIO's CELLECTRA(TM) 5PSP smart device at Day 0, Week 4 and 
Week 12. Participants are scheduled to be followed to Week 88.

About INOVIO's HPV-Associated DNA Medicines Clinical Programs

This study builds on significant clinical benefits demonstrated with INOVIO's 
HPV-associated DNA medicines in multiple clinical trials. Specifically, 
VGX-3100 in a Phase 2 proof-of-concept trial for cervical dysplasia 
demonstrated a complete response in 43 out of 107 patients in regression of 
high-grade cervical lesions and elimination of the underlying HPV infection. 
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 
study experienced a long-term complete response for more two years and 
counting. 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 VGX-3100  

VGX-3100 is a DNA medicine under Phase 3 investigation for the treatment of 
HPV-16 and HPV-18 infection and precancerous lesions of the cervix. INOVIO is 
in open-label Phase 2 clinical trials evaluating its efficacy for treating 
HPV-related vulvar and anal dysplasia (HSIL). VGX-3100 has the potential to be 
the first approved treatment for HPV infection of the cervix and the first 
non-surgical treatment for precancerous cervical lesions. It works by 
stimulating a specific immune response to HPV-16 and HPV-18, which targets the 
infection and causes destruction of precancerous cells. In a randomized, 
double-blind, placebo-controlled phase 2b study in 167 adult women with 
histologically documented HPV-16/18 cervical dysplasia (CIN2/3 or HSIL), 
treatment with VGX-3100 resulted in a statistically significantly greater 
regression of cervical dysplasia and clearance of HPV-16/18 infection vs. 
placebo. The most common side effect was injection site pain, and no serious 
adverse events were reported. VGX-3100 utilizes the patient's own immune system 
to clear HPV-16 and HPV-18 infection and precancerous lesions without the 
increased risks associated with surgery, such as loss of reproductive health 
and negative psychosocial impacts.

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 
coronaviruses MERS and COVID-19 under grants from the Coalition for Epidemic 
Preparedness Innovations (CEPI). 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). CELLECTRA(R) uses a brief electrical pulse to open 
small pores in the cell reversibly to allow the plasmids to enter, overcoming a 
key limitation of other DNA and mRNA approaches. Once inside the cell, the 
plasmids are used by the cell's own machinery to generate specific coded 
antigens, which then stimulate an immune response. 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 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 constructed 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 6,000 applications across a range of clinical trials, INOVIO has a 
strong track record of rapidly generating DNA medicine candidates to meet 
urgent global health needs.


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 
produce a robust and tolerable immune response. Specifically, INOVIO's lead 
candidate VGX-3100, currently in Phase 3 trials for precancerous cervical 
dysplasia, demonstrated it 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, 90% 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 MERS and COVID-19. Partners and collaborators 
include Advaccine, 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, Plumbline Life Sciences, Regeneron, 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

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 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 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.

Media:        Jeff Richardson, +1 267-440-4211, 
Investors:    Ben Matone, +1 484-362-0076,

SOURCE: INOVIO Pharmaceuticals, Inc.