Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in ocular and rare
diseases, today announced new interim clinical data from Cohorts
1-3 of the OPTIC Phase 1 dose-ranging clinical trial of ADVM-022
intravitreal (IVT) injection gene therapy in patients requiring
frequent anti-VEGF injections for their wet age-related macular
degeneration (AMD) and provided a business update.
For the first time, interim data are being presented from
patients in Cohort 31, 2 and updated data are being presented from
Cohorts 1 and 2 following treatment with a single IVT injection of
ADVM-022. April 1, 2020 is the cutoff date for all data being
presented. New data as detailed in the table below further
demonstrate the transformative potential of ADVM-022 to greatly
reduce anti-VEGF injection burden in wet AMD:
- ADVM-022 continues to show robust efficacy
- Long-term durability beyond 1 year from a single IVT injection
with zero rescue injections in Cohort 1
- Further evidence of a dose response:-- 6x1011 vg/eye: 6/6
patients rescue injection free-- 2x1011 vg/eye: 8/113
patients rescue injection free
- ADVM-022 continues to be well tolerated with a favorable safety
profile in all 3 cohorts:-- ADVM-022 related ocular AEs mild
(69%) to moderate (31%)-- No ADVM-022 related SAEs or
non-ocular adverse events-- No evidence of vasculitis,
retinitis, or choroiditis-- Ocular inflammation, when
observed, has been responsive to steroid eye drops
- Early evidence from Cohort 3 suggests that a 6-week
prophylactic regimen of steroid eye drops results in fewer adverse
events and less inflammation, compared to a 13-day prophylactic
regimen of oral steroids as used in Cohorts 1 and 2. In all 9
patients in Cohort 3:-- No cellular inflammation graded at a
score above 1+ in any patient-- No patients have required
more than steroid eye drops-- Only 8 ADVM-022-related AEs in
4 patients have been observed, all mild or moderate
- Early anatomic and vision improvements observed in Cohort 3,
with first 5 patients with 20 weeks follow-up showing:-- Mean
CRT4 reduction (-137.8 mm)-- Mean BCVA5 gain (+6.8
letters)
OPTIC Phase 1 Clinical Trial Data:
Results Following a Single ADVM-022 Dose: |
Cohort 1 |
Cohort 2 |
Cohort 31 |
Patients |
n=6 |
n=6 |
n= 9 |
Dose ADVM-022 |
Higher Dose6 x 10^11 vg/eye |
Lower Dose2 x 10^11 vg/eye |
Lower Dose2 x 10^11 vg/eye |
Follow-up (median) |
60 weeks |
36 weeks |
20 weeks |
Prophylactic steroid regimen |
13-day oral |
13-day oral |
6-week eye drops2 |
Rescue Injections: |
|
Number of patients requiring anti-VEGF rescue injections |
0/6 patients |
2/6 patients |
1/5 for first 5 patients with 20 weeks follow-up |
Total anti-VEGF rescue injections |
0 injections |
8 injections |
2 injections |
|
|
Follow-up BCVA5 and CRT4: |
52-64 weeks(median 60) |
32-40 weeks(median 36) |
First 5 patients with 20 weeks follow-up |
|
All Patients 100% (6/6) Rescue Free |
All Patients |
Rescue Free Patients 67% (4/6) |
All Patients |
Rescue Free Patients 80% (4/5) |
BCVA mean change from baseline (letters) |
-2.7 |
-2.8 |
+2.3 |
+6.8 |
+8.8 |
CRT mean change from baseline (mm) |
-26.2 |
-40.8 |
-30.0 |
-137.8 |
-149.8 |
1 The first 5 patients had 20 weeks of follow-up as of April 1,
2020. The remaining 4 patients had 4-12 weeks of follow-up,
insufficient for assessment of efficacy.2 In Cohort 3, patients
received 6-week prophylactic topical steroid regimen in place of
the 13-day prophylactic oral steroid regimen used in Cohorts 1 and
2.3 4/6 patients from Cohort 2 and 4/5 patients from Cohort 3 with
20 weeks follow up4 Central retinal thickness (CRT)5 Best
corrected visual acuity (BCVA)
Arshad M. Khanani, M.D., M.A., director of clinical research,
Sierra Eye Associates and principal investigator in the OPTIC trial
said, “It’s impressive to see the long-term durability demonstrated
at the higher dose of ADVM-022 in a patient population that
previously required frequent injections to maintain their vision
and are now beyond one year of follow-up with no rescue injections.
Additionally, preliminary evidence in Cohort 3 shows vision and
anatomical improvements and that the 6-week prophylactic steroid
eye drop regimen is effective at minimizing early ocular
inflammation. These are very positive data, and it is exciting to
see that this intravitreal gene therapy has the potential to
completely change the treatment paradigm for patients with wet
AMD.”
Aaron Osborne, MBBS, chief medical officer of Adverum, added,
“We are encouraged by the robust efficacy signal and evidence of a
dose response in the OPTIC trial with interim data from 3 cohorts.
Also, momentum in OPTIC is strong as we are currently enrolling
patients in Cohort 4 at the higher dose of 6 x 10^11 vg/eye using
the same steroid regimen as Cohort 3. We look forward to reporting
additional data in the second half of this year from OPTIC. Beyond
wet AMD, we are on track with our plans to advance ADVM-022 in
diabetic retinopathy, our second indication, and we continue to
expect to begin enrolling patients in our planned clinical trial in
the second half of this year.”
Business Update
- Adverum reports $297 million in cash, cash equivalents and
short-term investments as of March 31, 2020, compared to $166
million as of December 31, 2019. In February 2020, Adverum raised
approximately $140.8 million in net proceeds from an underwritten
public offering. The Company expects this quarter-end cash position
to fund operations into 2022.
- Due to COVID-19, and the shelter-in-place mandated in the State
of California, Adverum implemented a mandatory work-from-home
policy for all non-essential activities.-- To minimize the
chance of community infection, the company has limited on-site
activities to only the most time-critical or necessary operational
activities.-- Pursuant to the SEC’s recent order under
section 36 of the Securities Exchange Act of 1934, Adverum now
expects to file its Form 10-Q for the first quarter of 2020 with
the SEC on May 28, 2020.
2020 MilestonesFirst half
- Submit an investigational new drug application for ADVM-022 in
diabetic retinopathy, a key VEGF-driven cause of vision loss among
working-age adults
Second half
- Present data from all four cohorts of the OPTIC trial
- Begin enrolling patients in a planned Phase 1/2 clinical trial
for ADVM-022 in diabetic retinopathy to expand Adverum’s clinical
development pipeline
Conference Call and Webcast Today:In addition,
Adverum will host a conference call and webcast with expert retinal
specialists to discuss the new OPTIC data and the potential
opportunity for ADVM-022. The discussion will be held on Monday,
May 4, 2020 beginning at 2:15 pm PDT (5:15 pm EDT). Individuals can
participate in the conference call by dialing 1-866-420-8347
(domestic) or 1-409-217-8241 (international) and refer to
“Adverum Biotechnologies’ KOL Discussion Call.” It is recommended
call participants dial in 15 minutes in advance. The webcast and
slides for the call will be accessible under Events and
Presentations in the Investors section of the company's website.
The archived audio webcast will be available on the Adverum website
following the call and will be available for 30 days.
About the OPTIC Phase 1 Trial of ADVM-022 in Wet
AMDThe multi-center, open-label, Phase 1, dose-ranging
trial is designed to assess the safety and tolerability of a single
intravitreal (IVT) administration of ADVM-022 in patients with wet
AMD who are responsive to anti-vascular endothelial growth factor
(VEGF) treatment. In Cohort 1, patients (n=6) received ADVM-022 at
a higher dose of 6 x 10^11 vg/eye and in Cohort 2, patients (n=6)
received ADVM-022 at a lower dose of 2 x 10^11 vg/eye. In Cohort 3,
patients (n=9) also received a dose of 2 x 10^11 vg/eye and in
Cohort 4, patients (n=9) are receiving a dose of 6 x 10^11 vg/eye.
Patients in Cohorts 3 and 4 receive prophylactic steroid eye drops
instead of oral steroids which were used in Cohorts 1 and 2. The
primary endpoint of the trial is the safety and tolerability of
ADVM-022 after a single IVT administration. Secondary endpoints
include changes in best-corrected visual acuity (BCVA), measurement
of central retinal thickness (CRT), as well as the need for
anti-VEGF rescue injections. Each patient enrolled will be followed
for a total of two years.
Ten leading retinal centers across the United
States (U.S.) are participating in the OPTIC Phase 1 trial for
ADVM-022. For more information on the OPTIC Phase 1 clinical trial
of ADVM-022 in wet AMD, please
visit https://clinicaltrials.gov/ct2/show/NCT03748784.
About ADVM-022 Gene TherapyADVM-022 utilizes a
propriety vector capsid, AAV.7m8, carrying an aflibercept coding
sequence under the control of a proprietary expression cassette.
ADVM-022 is administered as a one-time intravitreal injection,
designed to deliver long-term efficacy and reduce the burden of
frequent anti-VEGF injections, optimize patient compliance and
improve vision outcomes for wet AMD and diabetic retinopathy
patients.
In recognition of the need for new treatment options for wet
AMD, the U.S. Food and Drug Administration granted Fast Track
designation for ADVM-022 for the treatment of this disease.
Adverum is currently evaluating ADVM-022 in the OPTIC study, a
Phase 1 clinical trial in patients 50 years and older with wet AMD.
Additionally, Adverum plans to initiate a planned Phase 1/2
clinical trial of ADVM-022 for the treatment of diabetic
retinopathy in the second half of 2020.
About Wet Age-related Macular Degeneration
(AMD)Age-related macular degeneration (AMD) is a
progressive disease affecting the macula, the region of the retina
at the back of the eye responsible for central vision. In patients
with wet AMD, an aggressive form of AMD, abnormal blood vessels
grow underneath and into the retina. These abnormal blood vessels
leak fluid and blood into and beneath the retina, causing vision
loss.
Wet AMD is a leading cause of vision loss in patients over 60
years of age, with a prevalence of approximately 1.2 million
individuals in the U.S. and 3 million worldwide. The incidence of
new cases of wet AMD in the U.S. is approximately 150,000 to
200,000 annually, and this number is expected to grow significantly
as the country’s population ages.
The current standard-of-care therapy for wet AMD is anti-VEGF
intravitreal injections. These are effective but typically require
eye injections every 4-12 weeks in order to maintain vision.
Compliance with this regimen can be difficult for patients,
caregivers, and healthcare systems, leading to undertreatment and
resulting in loss of vision.
About Adverum BiotechnologiesAdverum
Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy
company targeting unmet medical needs in serious ocular and rare
diseases. Adverum is evaluating its novel gene therapy candidate,
ADVM-022, as a one-time, intravitreal injection for the treatment
of its lead indication, wet age-related macular degeneration. For
more information, please visit www.adverum.com.
Forward-looking StatementsStatements contained
in this press release regarding events or results that may occur in
the future are “forward-looking statements” within the meaning of
the Private Securities Litigation Reform Act of 1995. Such
statements include, but are not limited to statements regarding:
the potential for ADVM-022 in treating patients with wet AMD
expressed in Dr. Khanani’s and Dr. Osborne’s quotes; Adverum’s
expectations as to the timing of reporting additional data in the
second half of 2020, and as to its plans to advance ADVM-022 in
diabetic retinopathy by filing an investigational drug application
in the first half of 2020 and begin enrolling patients in a planned
Phase 1/2 clinical trial in the second half of 2020; all of
which are based on certain assumptions made by Adverum on current
conditions, expected future developments and other factors Adverum
believes are appropriate in the circumstances. Adverum may not
achieve any of these in a timely manner, or at all, or otherwise
carry out the intentions or meet the expectations disclosed in its
forward-looking statements, and you should not place undue reliance
on these forward-looking statements. Actual results and the timing
of events could differ materially from those anticipated in such
forward-looking statements as a result of various risks and
uncertainties, which include risks inherent to, without limitation:
Adverum’s novel technology, which makes it difficult to predict the
time and cost of product candidate development and obtaining
regulatory approval; the results of early clinical trials not
always being predictive of future results; the potential for future
complications or side effects in connection with use of ADVM-022;
obtaining regulatory approval for gene therapy product candidates;
enrolling patients in clinical trials; reliance on third parties
for conducting the OPTIC trial and vector production; the effects
of the COVID-19 pandemic on the company’s operations; and ability
to fund operations through completion of the OPTIC trial and
thereafter. Risks and uncertainties facing Adverum are described
more fully in Adverum’s Form 10-K filed with the SEC on March 12,
2020 under the heading “Risk Factors.” All forward-looking
statements contained in this press release speak only as of the
date on which they were made. Adverum undertakes no obligation to
update such statements to reflect events that occur or
circumstances that exist after the date on which they were
made.
Investor and Media Inquiries:
Investors:Myesha LacyAdverum Biotechnologies,
Inc.mlacy@adverum.com1-650-304-3892
Media:Cherilyn Cecchini, M.D.LifeSci
Communicationsccecchini@lifescicomms.com1-646-876-5196
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