- Previously planned Phase 3 trial no longer required to
support Biological License Application (BLA) submission
- If approved, INO-3107 could potentially revolutionize
treatment options for patients with Recurrent Respiratory
Papillomatosis (RRP), a debilitating rare disease caused by human
papillomavirus (HPV)
- INO-3107 could be the first DNA medicine available in
the United States and the first
commercial product for INOVIO
PLYMOUTH
MEETING, Pa., Oct. 10,
2023 /PRNewswire/ -- INOVIO (NASDAQ:INO), a
biotechnology company focused on developing and commercializing DNA
medicines to help treat and protect people from HPV-related
diseases, cancer, and infectious diseases, today announced that it
has received feedback from the U.S. Food and Drug Administration
(FDA) that data from its completed Phase 1/2 trial of INO-3107 for
the treatment of RRP could support INOVIO's submission of a
BLA for review under the FDA's accelerated approval program. The
FDA also advised that the company's previously planned Phase 3
randomized, placebo-controlled trial would not be required to
support this submission. INOVIO will be required to initiate a
confirmatory trial prior to BLA submission for accelerated approval
and satisfy all other FDA filing requirements. The design of the
confirmatory trial has not yet been finalized. If approved,
INO-3107 would be the first DNA medicine in the United States and the first INOVIO
candidate to receive regulatory approval.
"Following the recent grant of Breakthrough Therapy Designation
for INO-3107 for the treatment of RRP, we're grateful for the
additional feedback from the FDA providing a potentially
accelerated development pathway. We believe INO-3107 could become a
game-changing treatment option for those suffering from RRP, a
serious and often difficult-to-treat disease," said INOVIO's
President and Chief Executive Officer, Dr. Jacqueline Shea. "We're now focused on
streamlining our development plan to support submission of a BLA
for accelerated approval. We would like to thank the patients and
investigators who have participated in our trials to date."
"I commend the FDA for recognizing the immense burden RRP puts
on patients and the critical need for a better standard of care,"
said the President of the Recurrent Respiratory Papillomatosis
Foundation, Kim McClellan. "I'm
thrilled by the progress being made for RRP patients who are
desperate for an alternative to surgery."
INOVIO's completed Phase 1/2 open-label, multicenter trial
assessed INO-3107's safety, tolerability, immunogenicity, and
efficacy in patients with HPV-6 and/or HPV-11-related RRP
(NCT:04398433). The trial evaluated the reduction in the number of
surgical interventions in the year following initial administration
of INO-3107 compared to the year prior to treatment. Patients
received four doses of INO-3107 on Day 0, and Weeks 3, 6, and 9.
Overall, 81.3% (26/32) of patients in the trial had a decrease in
surgical interventions in the year after INO-3107 administration
compared to the prior year, including 28.1% (9/32) that required no
surgical intervention during or after the dosing window. Patients
in the trial had a median range of 4 surgeries (2-8) in the year
prior to dosing. After dosing, there was a median decrease of 3
surgical interventions (95% confidence interval -3, -2). At the
outset of the study (Day 0), patients could have RRP tissue
surgically removed, but any surgery performed after Day 0 during
the dosing window was counted against the efficacy endpoint.
Treatment with INO-3107 generated a strong immune response in the
trial, inducing activated CD4 T cells and activated CD8 T cells
with lytic potential. T-cell responses were also observed at Week
52, indicating a persistent cellular memory response. INO-3107 was
well tolerated by participants in the trial, resulting in mostly
low-grade (Grade 1) treatment-emergent adverse effects such as
injection site pain and fatigue.
Data from this Phase 1/2 trial were presented earlier this year
at scientific and medical conferences, including the 2023 Annual
Meeting of the American Broncho-Esophageal Association (ABEA) in
May and at the European Laryngological Association's Annual Meeting
in June. Data from the trial was also published in May in the
peer-reviewed journal, The Laryngoscope, under the title
"Interim Results of a Phase 1/2 Open-Label Study of INO-3107 for
HPV-6 and/or HPV-11–Associated RRP." The Laryngoscope is the
official journal of the Triological Society (TRIO), the American
Laryngological Association (ALA), and the ABEA.
INO-3107 is INOVIO's lead candidate and one of three
clinical-stage DNA medicine candidates targeting HPV-related
disease. INOVIO's DNA technology has been studied in twelve
HPV-related trials, ranging from Phase 1 to Phase 3, involving more
than 900 patients in 20 countries with a variety of HPV-related
diseases, including RRP, HSIL (cervical, anal and vulvar) and head
and neck cancers. Shared observations in these trials include the
ability of DNA medicines to generate HPV antigen specific T cells
and a persistent cellular memory response, viral clearance and
lesion regression, and no anti-vector immune responses. All three
HPV-related product candidates (INO-3107, INO-3112 and VGX-3100)
have been well tolerated in these trials.
Updated Cash Guidance
INOVIO previously announced it expected its cash runway to fund
the company's operations into the third quarter of 2025. The
changes to the development plan for INO-3107 as a result of the FDA
feedback announced today could affect that prior guidance. The
company plans to provide updated cash guidance on its third quarter
earnings call scheduled to take place in November.
About RRP
RRP is a debilitating and rare disease caused primarily by HPV-6
and/or HPV-11. RRP is characterized by the development of small,
wart-like growths, or papillomas, in the respiratory tract. While
papillomas are generally benign, they can cause severe,
life-threatening airway obstruction and respiratory complications.
RRP can also significantly affect quality of life for patients by
affecting the voice box, limiting the ability to speak effectively.
Surgery to remove papillomas is the standard of care for RRP;
however, the papillomas often grow back because the underlying HPV
infection has not been eradicated.
The most widely cited U.S. epidemiology data published in 1995
estimated that there were 14,000 active cases and about 1.8 per
100,000 new cases in adults each year. More recent pediatric
epidemiology data cites a range of 0.5 - 0.7 per 100,000 new cases
in children in the U.S. each year.
About INO-3107
INO-3107 is INOVIO's lead DNA medicine product candidate and is
being developed as a potential treatment for RRP. INO-3107 is
designed to elicit a targeted T cell response against HPV-6 and
HPV-11, the HPV types responsible for causing RRP among other
HPV-related diseases. These targeted T cells are designed to seek
out and kill infected cells, with the aim of potentially preventing
or slowing the growth of new papillomas. INO-3107 was
designated a Breakthrough Therapy by the FDA in September 2023. INO-3107 received Orphan Drug
designation from the European Commission in May 2023 and from the FDA in 2020.
About INOVIO's DNA Medicines Platform
INOVIO's DNA medicines platform has two innovative components:
precisely designed DNA plasmids, delivered by INOVIO's proprietary
investigational medical device, CELLECTRA®. INOVIO uses proprietary
technology to design its DNA plasmids, which are small circular DNA
molecules that work like software the body's cells can download to
produce specific proteins to target and fight disease. INOVIO's
CELLECTRA® delivery devices help ensure its DNA medicines enter the
body's cells for optimal effect, without chemical adjuvants or
nanoparticles and without the risk of the anti-vector response seen
in viral vector platforms.
About INOVIO
INOVIO is a biotechnology company focused on developing and
commercializing DNA medicines to help treat and protect people from
HPV-related diseases, cancer, and infectious diseases. INOVIO's
technology optimizes the design and delivery of innovative DNA
medicines that teach the body to manufacture its own
disease-fighting tools. For more information, visit
www.inovio.com.
Contacts
Media: Jennie Willson (267)
429-8567 jennie.willson@inovio.com
Investors: Thomas Hong (267)
440-4298 thomas.hong@inovio.com
Forward-Looking Statements
This press release contains certain forward-looking statements
relating to INOVIO's business, including its plans to develop and
commercialize DNA medicines and its expectations regarding its
research and development programs, including plans to initiate a
confirmatory trial for INO-3107 instead of the previously-announced
Phase 3 trial, expectations with respect to INO-3107, if approved,
expectations that the updated development plan for INO-3107 may
affect prior financial guidance, and plans for regulatory
submissions. 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, INOVIO's ability to support its
pipeline of DNA medicine products, the ability of INOVIO's
collaborators to attain development and commercial milestones for
products INOVIO licenses and product sales that will enable INOVIO
to receive future payments and royalties, the adequacy of INOVIO's
capital resources, the availability or potential availability of
alternative therapies or treatments for the conditions targeted by
INOVIO or its collaborators, including alternatives that may be
more efficacious or cost effective than any therapy or treatment
that INOVIO and its collaborators hope to develop, issues involving
product liability, issues involving patents and whether they or
licenses to them will provide INOVIO 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 INOVIO can finance or
devote other significant resources that may be necessary to
prosecute, protect or defend them, the level of corporate
expenditures, assessments of INOVIO's technology by potential
corporate or other partners or collaborators, capital market
conditions, the impact of government healthcare proposals and other
factors set forth in INOVIO's Annual Report on Form 10-K for the
year ended December 31, 2022, its
Quarterly Report on Form 10-Q for the quarter ended June 30, 2023, and other filings INOVIO makes
from time to time with the Securities and Exchange Commission.
There can be no assurance that any product candidate in INOVIO's
pipeline will be successfully developed, manufactured, or
commercialized, that the 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 INOVIO undertakes no obligation to update
or revise these statements, except as may be required by law.
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SOURCE INOVIO Pharmaceuticals, Inc.