Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in ocular and rare
diseases, today announced the initiation of INFINITY, a Phase
2, multi-center, randomized, double-masked, active
comparator-controlled trial to assess a single intravitreal (IVT)
injection of ADVM-022 in patients with diabetic macular edema
(DME). The company also reported financial results for the first
quarter ended March 31, 2020 and provided a corporate update.
The INFINITY trial will enroll approximately 33 patients and is
designed to demonstrate superior control of disease activity
following a single IVT injection of ADVM-022 compared to a single
aflibercept injection, as measured by time to worsening of DME
disease activity. Additional objectives include assessments of
treatment burden, visual acuity, retinal anatomy and safety
outcomes.
Participants in this double-masked trial will be randomized to
one of three arms for their study eye treatment:
- Arm 1 will receive the higher dose of ADVM-022 at 6x1011
vg.
- Arm 2 will receive the lower dose of ADVM-022 at 2x1011
vg.
- Arm 3 will receive aflibercept at a dose of 2 mg.
“I’m thankful for the strong commitment and resilience our
employees have exhibited towards advancing our pipeline despite the
challenges of COVID 19,” said Leone Patterson, president and chief
executive officer, Adverum Biotechnologies. “We continue to execute
on our goal to develop and commercialize our novel gene therapy
candidate ADVM-022 as a potential one-time treatment for patients
with wet age-related macular degeneration (AMD) and DME, the two
largest indications for anti-VEGF treatment. With the recent
approval of our Investigational New Drug application for ADVM-022,
we have initiated INFINITY in DME, a high-need subgroup of patients
within the larger DR population. Additionally, we look forward to
presenting data from all four cohorts of the OPTIC Phase 1 trial
for ADVM-022 in wet AMD by the end of this year.”
Aaron Osborne, MBBS, chief medical officer of Adverum
Biotechnologies stated, “Over 30 million people are impacted by
diabetes in the United States. DME affects approximately 5% of
people with diabetes and is the most common cause of vision loss in
people with diabetic retinopathy (DR). We believe that the exciting
data seen to date for ADVM-022 in the ongoing OPTIC trial in wet
AMD highlight this therapy’s transformative potential to deliver
long-term control of serious retinal vascular diseases, including
DME. INFINITY has been designed to provide robust, controlled data
on ADVM-022 in DME and will be conducted at trial sites across the
United States.”
Recent Progress
- The U.S. Food and Drug Administration (FDA) approved Adverum’s
IND application for ADVM-022 (AAV.7m8-aflibercept) for the
treatment of DR. The company has initiated INFINITY, a Phase 2,
multi-center, randomized, double-masked, active
comparator-controlled trial, to assess a single IVT injection of
ADVM-022 in patients with DME, the most common cause of vision loss
in patients with DR. INFINITY will enroll approximately 33 patients
and is designed to demonstrate superior control of disease activity
following a single IVT injection of ADVM-022 compared to a single
aflibercept injection, as measured by time to worsening of DME
disease activity. Additional objectives include assessments of
treatment burden, visual acuity, retinal anatomy and safety
outcomes.
- In early May, positive interim clinical data were presented
from Cohorts 1-3 of the OPTIC Phase 1 dose-ranging clinical trial
of ADVM-022 in patients requiring frequent anti-VEGF injections for
wet age-related macular degeneration (wet AMD). Following a single
IVT injection, ADVM-022 showed long-term durability beyond 1 year
with zero rescue injections in Cohort 1. Additionally, early
evidence from Cohort 3 suggested 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.° Began
dosing patients in April in Cohort 4 (n=9, dose 6 x 1011 vg and a
6-week prophylactic regimen of steroid eye drops).°
Data from all four cohorts expected to be presented by
year-end.
- Scott Whitcup, M.D. was appointed to Adverum’s Board of
Directors. Dr. Whitcup has over 20 years of biopharmaceutical
industry experience, with extensive expertise in drug development
and regulatory approvals, including products for the treatment of
patients with ocular disease.
- The company raised approximately $140.9 million in net proceeds
from an underwritten public offering in February 2020.
COVID-19In March 2020, the San Francisco Bay
Area of California, where Adverum has its corporate headquarters,
mandated a Shelter-in-Place Executive Order in response to the
World Health Organization declaring a pandemic related to
coronavirus (COVID-19). The company’s primary focus is on the
health and safety of its employees, patients, and healthcare
providers. In mid-March, the company implemented a number of
actions, including a work-from-home policy for employees whose jobs
have not required them to be on-site. The company has maintained
certain essential in-person laboratory functions in order to
advance key research and development initiatives supported by the
implementation of updated onsite procedures. The company believes
these measures and others have allowed it to mitigate, but not
eliminate, the effects on and risks of on-site operations posed by
the COVID-19 pandemic.
In the OPTIC Phase 1 trial, patients with wet AMD are in
high-risk categories for COVID-19 complications based on age,
comorbidities, or both. The company is working closely with
clinical trial sites to monitor and attempt to minimize the
potential negative impacts of the evolving COVID-19 outbreak on
patient safety, patient enrollment, continued participation of
patients already enrolled in the company’s clinical studies,
protocol compliance, data quality, and overall study integrity.
Despite these efforts, the company continues to assess whether the
COVID-19 pandemic will significantly impact trial enrollment or
completion of the current or planned clinical studies.
Additionally, although the company has sufficient drug supply for
its current clinical trials, it is working with its product supply
partners to implement measures where possible to attempt to
mitigate the COVID-19 pandemic’s effects on and risks to its future
clinical supply needs and long-term timelines, which may result in
additional expenses.
Financial Results for the Three Months Ended March 31,
2020
- Cash, cash equivalents and short-term
investments were $297.1 million as of March 31, 2020,
compared to $166.0 million as of December 31, 2019. In February
2020, Adverum raised approximately $140.9 million in net proceeds
from an underwritten public offering. Adverum expects this
quarter-end cash position to fund operations into 2022.
- Research and development expenses were $14.8
million for the three months ended March 31, 2020, compared to
$10.1 million for the same period in 2019. Research and development
expenses increased primarily due to higher material production
costs, personnel-associated costs, and increased facilities costs
related to the company’s new facility.
- General and administrative expenses were $9.0
million for the three months ended March 31, 2020, compared to $5.6
million for the same period in 2019. General and administrative
expenses increased primarily due to higher personnel-associated
costs, including stock-based compensation expenses, and
professional service and consultant expenses.
- Net loss was $22.9 million, or $0.31 per basic
and diluted share, for the three months ended March 31, 2020,
compared to $14.5 million, or $0.23 per basic and diluted share,
for the same period in 2019.
Conference Call
InformationAdverum will host a conference call and audio
webcast today at 1:30 pm PT / 4:30 pm ET to report its first
quarter 2020 financial results, discuss the INFINITY Phase 2 trial,
and provide an update on recent business progress. The live audio
webcast and accompanying slide presentation will be accessible
under Events and Presentations in the Investors section of the
company's website. To participate in the conference call dial
1-866-420-8347 (domestic) or 1-409-217-8241 (international) and
refer to the “Adverum Biotechnologies’ First Quarter 2020 Earnings
Call.” It is recommended call participants dial in15 minutes in
advance. The archived audio webcast will be available on the
Adverum website following the call and will be available for 30
days.
About the INFINITY Phase 2 Trial of ADVM-022 in
DMEINFINITY is a Phase 2, multi-center, randomized,
double-masked, active comparator-controlled trial designed to
assess a single intravitreal (IVT) injection of ADVM-022 in
patients with diabetic macular edema (DME), the most common cause
of vision loss in patients with DR.
The INFINITY trial will enroll approximately 33 patients and is
designed to demonstrate superior control of disease activity
following a single IVT injection of ADVM-022 compared to a single
aflibercept injection, as measured by time to worsening of DME
disease activity. Additional objectives include assessments of
treatment burden, visual acuity, retinal anatomy and safety
outcomes.
Across the United States, leading retinal clinical trial sites
will participate in the INFINITY trial. For additional information,
please visit www.INFINITYclinicaltrial.com.
About Diabetic Retinopathy (DR) and Diabetic Macular
Edema (DME)Over 30 million people are impacted by diabetes
in the United States. Diabetic retinopathy (DR) affects
approximately one in three adults with diabetes and can put
patients at risk of vision loss. DR can be diagnosed at different
severity levels, and is the most common cause of blindness in
working-age adults in the U.S.
Diabetic macular edema (DME) is a vision-threatening
complication of DR that can occur at any severity stage of DR. DME
is characterized by retinal thickening in the area of the macula,
and the risk of DME increases with the worsening of the DR severity
score (DRSS). DME affects approximately 5% of people with diabetes
and is the leading cause of vision loss in patients with DR.
The current standard-of-care therapy for DME is anti-VEGF
intravitreal injections. These are effective but typically require
frequent and long-term injections for patients to maintain good
vision. Compliance with these regimens can be difficult for
patients, leading to undertreatment and vision loss. Real-world
outcomes in DME with anti-VEGF therapy are meaningfully worse than
in clinical trials.1
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
(IVT), designed to deliver long-term efficacy and reduce the burden
of frequent anti-VEGF injections, optimize patient compliance and
improve vision outcomes for patients with wet age-related macular
degeneration (wet AMD) and diabetic macular edema (DME).
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 wet AMD.
Adverum is currently evaluating ADVM-022 in the OPTIC Phase 1
clinical trial in patients with wet AMD and the INFINITY Phase 2
trial in patients with DME.
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 advancing the clinical development of its
novel gene therapy candidate, ADVM-022, as a one-time, intravitreal
injection for the treatment of patients with wet age-related
macular degeneration and diabetic macular edema. For more
information, please visit www.adverum.com.
Forward-looking Statements
Statements 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 and DME; Adverum’s expectations as to its
plans to advance ADVM-022 in DME by beginning to enroll patients in
the INFINITY trial and the expected enrollment numbers; Adverum’s
expectations that its current cash position will fund its
operations into 2022; and Adverum’s expectations that it will
present data from all four cohorts of the OPTIC Phase 1 trial for
ADVM-022 in wet AMD by the end of this year. All of these
statements 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 and INFINITY
trials and vector production; the effects of the COVID-19 pandemic
on the company’s operations and on the company’s ongoing clinical
trials; and ability to fund operations through completion of the
OPTIC and INFINITY trials and thereafter. Risks and uncertainties
facing Adverum are described more fully in Adverum’s Form 10-Q
filed with the SEC on May 28, 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
_________
1 TA Ciulla, et al. Diabetes Care 2003 Sep; 26(9):
2653-2664.
|
Adverum
Biotechnologies, Inc. |
Consolidated Balance
Sheets |
(In thousands) |
|
|
|
|
|
|
|
March
31, |
|
December
31, |
|
|
|
2020 |
|
|
|
2019 |
|
|
|
(Unaudited) |
|
|
(1) |
|
Assets |
|
|
|
|
|
|
|
|
Current
assets: |
|
|
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
130,162 |
|
|
$ |
65,897 |
|
Short-term investments |
|
|
166,904 |
|
|
|
100,138 |
|
Prepaid expenses and other current assets |
|
|
3,382 |
|
|
|
9,835 |
|
Total current assets |
|
|
300,448 |
|
|
|
175,870 |
|
Operating
lease right-of-use asset |
|
|
20,406 |
|
|
|
20,963 |
|
Property and
equipment, net |
|
|
26,727 |
|
|
|
24,884 |
|
Restricted
cash |
|
|
999 |
|
|
|
999 |
|
Deposit and
other long-term assets |
|
|
19 |
|
|
|
11 |
|
Total assets |
|
$ |
348,599 |
|
|
$ |
222,727 |
|
Liabilities and stockholders' equity |
|
|
|
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
|
|
|
Accounts payable |
|
$ |
4,663 |
|
|
$ |
4,103 |
|
Accrued expenses and other current liabilities |
|
|
8,079 |
|
|
|
11,271 |
|
Lease liability, current portion |
|
|
4,025 |
|
|
|
4,034 |
|
Total current liabilities |
|
|
16,767 |
|
|
|
19,408 |
|
Lease
liability, net of current portion |
|
|
27,753 |
|
|
|
28,214 |
|
Other
noncurrent liabilities |
|
|
126 |
|
|
|
148 |
|
Total liabilities |
|
|
44,646 |
|
|
|
47,770 |
|
Stockholders' equity: |
|
|
|
|
|
|
|
|
Common stock |
|
|
8 |
|
|
|
7 |
|
Additional paid-in capital |
|
|
712,713 |
|
|
|
560,704 |
|
Accumulated other comprehensive loss |
|
|
(833 |
) |
|
|
(725 |
) |
Accumulated deficit |
|
|
(407,935 |
) |
|
|
(385,029 |
) |
Total
stockholders' equity |
|
|
303,953 |
|
|
|
174,957 |
|
Total liabilities and stockholders' equity |
|
$ |
348,599 |
|
|
$ |
222,727 |
|
|
|
|
|
|
|
|
|
|
(1) Derived from Adverum’s annual audited consolidated financial
statements. |
|
Adverum
Biotechnologies, Inc. |
Consolidated
Statements of Operations |
(In thousands except
per share data) |
(Unaudited) |
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
|
2020 |
|
|
|
2019 |
|
|
|
|
|
|
|
|
|
|
Operating
expenses: |
|
|
|
|
|
|
|
|
Research and
development |
|
|
14,751 |
|
|
|
10,131 |
|
General and
administrative |
|
|
9,040 |
|
|
|
5,576 |
|
Total
operating expenses |
|
|
23,791 |
|
|
|
15,707 |
|
Operating
loss |
|
|
(23,791 |
) |
|
|
(15,707 |
) |
Other
income, net |
|
|
885 |
|
|
|
1,218 |
|
Net
loss |
|
|
(22,906 |
) |
|
|
(14,489 |
) |
Net loss per
share — basic and diluted |
|
$ |
(0.31 |
) |
|
$ |
(0.23 |
) |
Weighted-average common shares outstanding - basic and diluted |
|
|
73,797 |
|
|
|
63,125 |
|
|
|
|
|
|
|
|
|
|
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