Outlook Therapeutics, Inc. (Nasdaq: OTLK), a biopharmaceutical
company working to develop and launch the first FDA-approved
ophthalmic formulation of bevacizumab for use in
retinal indications, today announced new 12-month safety data from
the pivotal Phase 3 NORSE TWO trial that further confirm the strong
safety profile in this study of ONS-5010 / LYTENAVA™
(bevacizumab-vikg) for treatment of neovascular age-related macular
degeneration (wet AMD). In August 2021, Outlook Therapeutics
announced the topline readout of pivotal data from its NORSE TWO
trial, which included 11 months of safety data. The topline data
previously reported from NORSE TWO demonstrated clinically relevant
and highly statistically significant results as well as a robust
safety profile indicating that in the trial ONS-5010 was well
tolerated and showed no unanticipated safety signals.
“The full 12-month safety data results move us
one step closer to providing patients with an FDA-approved,
cGMP-produced drug product that meets ophthalmic FDA standards and
avoids the potential risks of repackaged IV bevacizumab,” said C.
Russell Trenary III, President and CEO of Outlook Therapeutics.
“Our goal is to offer patients, in the United States and globally,
a safe and effective, approved ophthalmic bevacizumab. ONS-5010 is
specifically formulated to meet requirements to treat retinal
diseases like wet AMD. We continue to work towards submitting a
Biologics License Application for ONS-5010 to the U.S. Food and
Drug Administration for ONS-5010 in the first calendar quarter of
2022.”
The NORSE TWO pivotal Phase 3 clinical trial
enrolled a total of 228 subjects with wet AMD across 39 clinical
trial sites in the United States. Participants in the trial were
followed for 12 months. The primary endpoint for the study was the
proportion of patients who gained at least 15 letters in the best
corrected visual acuity (BCVA) at 11 months. The trial compared
ONS-5010 dosed monthly to LUCENTIS®, which was dosed using one of
the regimens listed in the LUCENTIS® label (i.e., patients were
treated monthly for the first three months followed by less
frequent dosing; the PIER regimen). The key secondary endpoint for
NORSE TWO was the mean change in the BCVA through 11 months.
“The success of NORSE TWO continues to solidify
the potential of ONS-5010 ophthalmic bevacizumab as a highly
effective and safe treatment in treating wet AMD,” added Terry
Dagnon, Chief Operating Officer of Outlook Therapeutics. “We are
excited that the highly statistically significant and clinically
relevant results from NORSE TWO demonstrate the potential clinical
value of ONS-5010 for patients and will work closely with the FDA
and other global authorities to bring this new option to patients,
clinicians and payors as quickly as possible.”
Topline data from NORSE TWO showed that ONS-5010
bevacizumab-vikg met the primary and key secondary endpoints for
efficacy with clinically impactful change observed for treated
patients. The NORSE TWO primary endpoint difference in proportion
of subjects gaining at least 15 letters BCVA was met and was highly
statistically significant and clinically relevant. In the
intent-to-treat (ITT) primary dataset, the percentage of patients
who gained at least 15 letters who were treated with ranibizumab
was 23%, and the percentage of patients who gained at least 15
letters who were treated with bevacizumab-vikg was 41% (p =
0.0052). The primary endpoint was also statistically significant
and clinically relevant in the secondary per-protocol (PP) dataset
(p = 0.04) where the percentages were almost identical, at 24% with
ranibizumab and 41% with bevacizumab-vikg. The key secondary
endpoint BCVA score change from baseline to month 11 in the primary
ITT dataset was also highly statistically significant and
clinically relevant (p = 0.0043). A mean change in BCVA was
observed with ranibizumab of 5.8 letters and the mean change with
bevacizumab-vikg was 11.2 letters. The results were also
statistically significant in the secondary PP dataset (p = 0.05)
with a mean change in letters with ranibizumab of 7.0 letters and
with bevacizumab-vikg 11.1 letters.
The safety results demonstrated in NORSE TWO are
consistent with previously reported safety results from Outlook
Therapeutics’ NORSE ONE and NORSE THREE clinical trials. Following
exposure to bevacizumab-vikg, there was only one subject who
reported an adverse event of ocular inflammation in all three
trials. In NORSE TWO, there was only a single related ocular
serious adverse event reported in the bevacizumab-vikg trial arm,
which resolved, and no unanticipated safety signals were detected.
The most common ocular adverse event was intravitreal
injection-related hemorrhage in the tissues on the surface of the
eye (conjunctival hemorrhage) that resolved without any sequela.
The ONS-5010 safety database, now with 12 months of NORSE TWO data,
continues to be consistent with previously published results for
bevacizumab, such as in the 2011 CATT clinical trial.
ONS-5010 (bevacizumab) registration clinical trial
programOutlook Therapeutics’ wet AMD ONS-5010 clinical
program for the planned BLA consists of three clinical trials:
NORSE ONE, a proof-of-concept clinical experience trial in wet AMD
patients; NORSE TWO, the pivotal Phase 3 wet AMD trial; and NORSE
THREE, a supplemental safety study in patients with wet AMD and
other retina diseases undertaken to ensure that a sufficient number
of patients have been dosed with ONS-5010 to support the BLA
submission. Results from NORSE ONE and NORSE THREE demonstrated
positive proof-of-concept and a safety profile consistent with that
of prior published research on bevacizumab for ophthalmic use.
NORSE TWO provided pivotal data that demonstrated positive and
highly statistically significant and clinically relevant efficacy
and safety data for treatment of patients with wet AMD. With the
registration clinical trials now completed, Outlook Therapeutics
plans to submit a BLA under the Public Health Service Act (PHSA)
351(a) regulatory pathway in the first quarter of calendar 2022. If
the BLA is approved, it is expected to result in 12 years of
marketing exclusivity for ONS-5010 as the first and only ophthalmic
formulation of bevacizumab approved by the FDA to treat wet AMD.
Full data from NORSE TWO will be presented at the American Academy
of Ophthalmology’s Retina Subspecialty Meeting, RET13 - Section X:
Late Breaking Developments, Part II on November 13, 2021, at 9:17
AM ET. The NORSE TWO data will also be submitted for publication in
a peer-reviewed journal.Pre-commercialization planning
underway
Per the National Eye Institute (NEI), use of
unapproved repackaged IV bevacizumab from compounding pharmacies is
estimated to account for approximately 50% of all wet AMD
prescriptions in the United States each year. Globally, the nine
major markets account for an estimated $13.1 billion market for
anti-VEGF drugs to treat retina diseases. In anticipation of
potential FDA marketing approval in 2022 for ONS-5010, Outlook
Therapeutics has begun commercial launch planning, including
manufacturing with drug substance manufacturer FUJIFILM Diosynth
Biotechnologies and best-in-class drug product manufacturer Aji
Biopharma Services, distribution, sales force planning, physician
and payor advisory board outreach, key opinion leader support and
payor community engagement. To bring ONS-5010 to market in a way
that benefits all stakeholders – patients, clinicians, and payors –
Outlook Therapeutics has already commenced collaborative
discussions with payors and the retina community. Outlook
Therapeutics expects ONS-5010, if approved, to be a safe and
cost-effective choice for patients, payors and clinicians worldwide
for retinal indications.Outlook Therapeutics is also developing
registration documents on a parallel path for approvals in Europe
and expects to submit them shortly after completing the submission
to the FDA. Outlook Therapeutics continues to explore potential
strategic commercialization partners, such as Syntone Biopharma JV
in China.In addition to the clinical development program evaluating
ONS-5010 for wet AMD, Outlook Therapeutics has received agreements
from the FDA on three Special Protocol Assessments (SPAs) for three
additional registration clinical trials. These SPAs cover the
protocols for a planned registration clinical trial evaluating
ONS-5010 to treat branch retinal vein occlusion (BRVO, NORSE FOUR),
and two planned registration clinical trials evaluating the drug
candidate for the treatment of diabetic macular edema (DME, NORSE
FIVE and NORSE SIX). Outlook Therapeutics expects to initiate
registration clinical trials for ONS-5010 for DME and BRVO later in
2021 or in early 2022.About ONS-5010 / LYTENAVA™
(bevacizumab-vikg)
ONS-5010 is an investigational ophthalmic
formulation of bevacizumab under development to be administered as
an intravitreal injection for the treatment of wet AMD and other
retinal diseases. Because no currently approved ophthalmic
formulations of bevacizumab are available, clinicians wishing to
treat retinal patients with bevacizumab have had to use unapproved
repackaged IV bevacizumab provided by compounding pharmacies,
products that have known risks of contamination and inconsistent
potency and availability. If approved, ONS-5010 will replace the
need to use unapproved repackaged IV bevacizumab from compounding
pharmacies for the treatment of wet AMD.ONS-5010 is a full-length,
humanized anti-VEGF (Vascular Endothelial Growth Factor)
recombinant monoclonal antibody (mAb) that inhibits VEGF and
associated angiogenic activity. VEGF is a protein that
promotes the growth of abnormal new blood vessels and promotes
leakage from these vessels leading to retinal edema and hemorrhage.
With wet AMD, abnormally high levels of VEGF are secreted in the
eye and lead to loss of vision. Anti-VEGF injection therapy treats
the vision-threatening leakage and hemorrhage as wells as blocks
the growth of the abnormal blood vessels. Since the advent of
anti-VEGF therapy, it has become the standard-of-care treatment
option within the retina community globally.
About Outlook Therapeutics, Inc.
Outlook Therapeutics is a biopharmaceutical
company working to develop and launch ONS-5010/ LYTENAVA™
(bevacizumab-vikg) as the first FDA-approved ophthalmic formulation
of bevacizumab for use in retinal indications, including wet AMD,
DME and BRVO. If ONS-5010 ophthalmic bevacizumab is approved,
Outlook Therapeutics expects to commercialize it as the first and
only FDA-approved ophthalmic formulation of bevacizumab for use in
treating retinal diseases in the United States, United Kingdom,
Europe, Japan and other markets. Outlook Therapeutics expects to
submit ONS-5010 ophthalmic bevacizumab to the U.S. FDA as a BLA
under the PHSA 351(a) regulatory pathway. For more information,
please visit www.outlooktherapeutics.com.Forward-Looking
Statements
This press release contains forward-looking
statements. All statements other than statements of historical
facts are “forward-looking statements,” including those relating to
future events. In some cases, you can identify forward-looking
statements by terminology such as “may,” “might,” “will,” “should,”
“expect,” “plan,” “anticipate,” “project,” “believe,” “estimate,”
“predict,” “potential,” “intend” or “continue,” the negative of
terms like these or other comparable terminology, and other words
or terms of similar meaning. These include, among others,
statements about ONS-5010’s potential as the first FDA-approved
ophthalmic formulation of bevacizumab-vikg, including benefits
therefrom to patients, payors and physicians, including
expectations of market exclusivity, the timing of BLA submission
and commercial launch of ONS-5010, plans for regulatory approvals
in other markets and plans for future clinical trials. Although
Outlook Therapeutics believes that it has a reasonable basis for
the forward-looking statements contained herein, they are based on
current expectations about future events affecting Outlook
Therapeutics and are subject to risks, uncertainties and factors
relating to its operations and business environment, all of which
are difficult to predict and many of which are beyond its control.
These risk factors include those risks associated with developing
pharmaceutical product candidates, risks of conducting clinical
trials and risks in obtaining necessary regulatory approvals, as
well as those risks detailed in Outlook Therapeutics’ filings with
the Securities and Exchange Commission, including the Annual Report
on Form 10-K for the fiscal year ended September 30, 2020, as
amended, and subsequent Quarterly Reports on Form 10-Q, which
include the uncertainty of future impacts related to the ongoing
COVID-19 pandemic. These risks may cause actual results to differ
materially from those expressed or implied by forward-looking
statements in this press release. All forward-looking statements
included in this press release are expressly qualified in their
entirety by the foregoing cautionary statements. You are cautioned
not to place undue reliance on these forward-looking statements,
which speak only as of the date hereof. Outlook Therapeutics does
not undertake any obligation to update, amend or clarify these
forward-looking statements whether as a result of new information,
future events or otherwise, except as may be required under
applicable securities law.
CONTACTS:Media
Inquiries:Harriet UllmanVice PresidentLaVoie Health
ScienceT: 617-669-3082hullman@lavoiehealthscience.com
Investor
Inquiries: Jenene
ThomasChief Executive OfficerJTC Team, LLCT:
833.475.8247 OTLK@jtcir.com
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