Outlook Therapeutics, Inc. (Nasdaq: OTLK), a late
clinical-stage biopharmaceutical company working to develop the
first FDA-approved ophthalmic formulation of bevacizumab-vikg for
use in retinal indications, today announced topline results
demonstrating anticipated safety and efficacy and positive
proof-of-concept of ONS-5010 / LYTENAVA™ (bevacizumab-vikg) for the
treatment of wet age-related macular degeneration (wet AMD) from
its NORSE 1 clinical study, the first of two registration clinical
trials. ONS-5010 is the first injectable ophthalmic
formulation of bevacizumab-vikg seeking U.S. Food and Drug
Administration (FDA) approval for the treatment of wet AMD under a
new Biologics License Application (BLA).
“Although a small study, we are excited to see
that both the efficacy signals that we anticipated in NORSE 1 for
an ophthalmic bevacizumab as well as the clinical safety data are
consistent with previously published results for ophthalmic
bevacizumab,” stated Mark Humayun, MD, PhD, Medical Advisor to
Outlook Therapeutics. “We are looking forward to seeing the results
of the NORSE 2 pivotal trial in the third quarter of 2021. If
ONS-5010 is approved to treat wet AMD and other retinal diseases,
it will be a significant development in the practice of
ophthalmology. Bevacizumab is a well understood anti-VEGF therapy
that is already widely used, and ONS-5010, if approved, will be a
valuable, FDA-approved treatment option across the spectrum of
retinal care.”
In NORSE 1, there were no statistical
differences between LUCENTIS® (ranibizumab) and ONS-5010 in the
study. Overall, 2 of 25 (8%) patients on the ONS-5010 arm achieved
> 15 letters best corrected visual acuity (BCVA) at Month 11
compared to 5 of 23 (22%) patients on the ranibizumab arm. In the
subgroup analysis of treatment-naïve subjects, 2 of 6 (33%)
patients on the ONS-5010 arm achieved > 15 letters at Month 11
compared to 4 of 13 (31%) patients in the ranibizumab arm.
Additionally, the subgroup analysis of patients who had a baseline
visual acuity of < 67 letters (20/50 or worse) at study entry
included 2 of 4 (50%) patients in the ONS-5010 arm and 4 of 9 (44%)
patients in the ranibizumab arm achieving > 15 letters at Month
11. These key subgroups represent the enrollment criteria for
patients in the fully enrolled, pivotal NORSE 2 clinical trial.
The results from NORSE 1 indicate that the
observed safety profile of ONS-5010 in this study is consistent
with that of previously reported bevacizumab ophthalmology studies.
There were no statistical differences in safety between LUCENTIS®
(ranibizumab) and ONS-5010 in the study and zero cases of ocular
inflammation.
“The results from NORSE 1 met our
proof-of-concept expectations and, importantly, validate our
confidence in the design of our ongoing NORSE 2 trial and the
potential data we may see from that study. As anticipated in NORSE
1, ONS-5010 provided us with positive trends in efficacy in
three-line visual acuity gains and was shown to be safe and well
tolerated. In fact, ONS-5010 had no adverse events associated with
inflammation, which has emerged as a concern for other anti-VEGFs
in treating retinal diseases,” said Lawrence Kenyon, President, CEO
and CFO of Outlook Therapeutics. “On behalf of Outlook
Therapeutics, I want to thank all of the patients and clinicians
who have persevered through the COVID-19 pandemic to bring this
clinical trial to completion.”
The results from NORSE 1 provide support for the
established design and protocol for the ongoing pivotal NORSE 2
clinical trial, the second of two registration clinical trials
evaluating ONS-5010 for treatment of wet AMD; NORSE 2 excludes
patients with vision better than 20/50 at baseline, as well as
patients who have received prior treatment for wet AMD. NORSE 2 is
powered for statistical significance, and by excluding such
patients, Outlook Therapeutics believes that NORSE 2 has enrolled
the optimal patients for meeting the endpoint of the
study.
Mr. Kenyon concluded, “With these results now in
hand, we turn our attention to our NORSE 2 Phase 3 pivotal trial,
which is similar in design to NORSE 1 in length of treatment and
dosing, but is designed with a larger, and treatment-naïve, patient
population and powered to show statistical significance. Based on
our end-of-Phase 2 discussions with the FDA, we believe that the
results from NORSE 1 and NORSE 2, combined with our upcoming NORSE
3 safety study that is designed to ensure an adequate number of
patient exposures to ONS-5010 are available, will be sufficient to
support a new BLA submission in the second half of next year.”
Outlook Therapeutics intends to complete
development of ONS-5010 for submission to the FDA as a new BLA
under the 351(a) PHSA regulatory pathway for the treatment of wet
AMD and also has plans to submit for regulatory approvals in
Europe, the United Kingdom and Japan, as well as other countries.
While bevacizumab is already widely used, if approved, ONS-5010
will be the first and only on-label ophthalmic formulation of
bevacizumab-vikg approved for treating retinal diseases and has the
potential to address a $9.1 billion anti-VEGF market.
About NORSE 1
The NORSE 1 clinical trial is a small
30-subject-per-arm clinical experience trial designed to provide an
initial safety and efficacy readout for ONS-5010 v. LUCENTIS®
(ranibizumab) in treating patients with wet AMD, and to provide the
initial safety data necessary to open an Investigational New Drug
Application (IND) with the FDA in March 2019. The first of two
registration clinical trials evaluating ONS-5010 in treating wet
AMD, NORSE 1 enrolled a total of 61 treatment-naïve and previously
treated patients diagnosed with wet AMD at nine sites in Australia.
Patients on ONS-5010 were dosed monthly compared to those on
ranibizumab, who were dosed using the PIER alternative dosing
regimen of three monthly doses followed by quarterly dosing. Study
design and randomization are consistent with an agreement reached
with the FDA at an end-of-Phase 2 meeting in April 2018.
About NORSE 2
Outlook Therapeutics recently completed patient
enrollment in NORSE 2, its 227-patient Phase 3 registration
clinical trial evaluating ONS-5010 for the treatment of patients
with wet AMD. Patients in the trial are being treated for 12
months. The primary endpoint for NORSE 2 is the difference in
proportion of patients who gain at least 15 letters in BCVA at 11
months for ONS-5010 dosed on a monthly basis, compared to
LUCENTIS®, which is being dosed quarterly per the PIER regimen.
Outlook Therapeutics expects to report pivotal safety and efficacy
data from NORSE 2 in the third calendar quarter of 2021.
About ONS-5010 / LYTENAVA™
(bevacizumab-vikg)
ONS-5010 / LYTENAVA™ (bevacizumab-vikg) is an
investigational ophthalmic formulation of bevacizumab-vikg under
development to be administered as an intravitreal injection for the
treatment of wet AMD and other retinal diseases. ONS-5010 is
currently being evaluated in two registration clinical trials for
wet AMD (NORSE 1 and NORSE 2) and, if successful, is expected to be
submitted to the FDA as a new BLA for this ophthalmic indication
under the PHSA 351(a) regulatory pathway. Because no currently
approved ophthalmic formulations of bevacizumab are available,
clinicians wishing to treat retinal patients with bevacizumab use
unapproved repackaged IV bevacizumab provided by compounding
pharmacists, products that have known risks of contamination and
inconsistent potency and availability.
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 new
abnormal blood vessels. With wet AMD, abnormally high levels of
VEGF are secreted in the eye and can lead to vision loss. Anti-VEGF
injection therapy blocks this growth. Since the advent of anti-VEGF
therapy, it has become the standard-of-care treatment option within
the retina community globally.
If approved, ONS-5010 will be the first and only
FDA-approved ophthalmic formulation of bevacizumab-vikg to treat
retinal diseases. Outlook Therapeutics currently intends to
commercialize ONS-5010 in both vials and single-use pre-filled
syringes.
About Outlook Therapeutics,
Inc.
Outlook Therapeutics is a late clinical-stage
biopharmaceutical company working to develop ONS-5010 / LYTENAVA™
(bevacizumab-vikg) as the first FDA-approved ophthalmic formulation
of bevacizumab-vikg for use in retinal indications, including wet
AMD, DME and BRVO. If ONS-5010 is approved, Outlook Therapeutics
expects to commercialize it as the first and only FDA-approved
ophthalmic formulation of bevacizumab-vikg for use in treating
retinal diseases in the United States, United Kingdom, Europe,
Japan and other markets. Outlook Therapeutics expects to file
ONS-5010 with the U.S. FDA as a new 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 statements about the
timing of completion of, and pivotal safety and efficacy data from,
NORSE 2, ONS-5010’s potential as the first FDA-approved ophthalmic
formulation of bevacizumab-vikg, including benefits therefrom to
patients, payors and physicians, the timing of BLA submission and
sufficiency of exposures to support such submission, statements
about commercial launch of ONS-5010, and plans for regulatory
approvals in other markets. 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, 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:Jessica
MorrisAccount ExecutiveLaVoie Health ScienceT: 857.701.9731
jmorris@lavoiehealthscience.com
Investor Inquiries: Jenene
Thomas Chief Executive Officer JTC Team, LLC T: 833.475.8247
OTLK@jtcir.com
Outlook Therapeutics (NASDAQ:OTLKW)
Historical Stock Chart
From Jun 2024 to Jul 2024
Outlook Therapeutics (NASDAQ:OTLKW)
Historical Stock Chart
From Jul 2023 to Jul 2024