Adverum Biotechnologies Reports Third Quarter 2023 Financial Results and Pipeline Highlights
09 Novembre 2023 - 10:05PM
Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage
company that aims to establish gene therapy as a new standard of
care for highly prevalent ocular diseases, today reported financial
results for the third quarter ended September 30, 2023.
“In our OPTIC presentation at AAO, we’ve built upon our industry
leading dataset in wet AMD gene therapy,” stated Laurent Fischer,
M.D., president and chief executive officer of Adverum
Biotechnologies. “Patients who have received a single intravitreal
injection of Ixo-vec continue to receive benefit through three
years of follow-up, including maintenance of vision, durability of
anatomical improvements and sustained reduction in anti-VEGF
treatment burden, with half of the patients remaining entirely free
of injections. Our first data presented from the LUNA trial,
reported during the third quarter, reinforced what we’ve learned
from both the ongoing OPTIC extension trial and our nonclinical NHP
data, and suggests that both the 2E11 and 6E10 vg/eye doses of
Ixo-vec produce aflibercept levels that are associated with
significant clinical activity. We look forward to presenting our
initial safety and efficacy data from our ongoing LUNA trial during
the fourth quarter, and ultimately to bringing the potential of
this transformative therapy to patients with wet AMD.”
Recent Pipeline Highlights:
- Aflibercept Protein
Level Data from Ongoing LUNA Phase 2 Trial:
- In September 2023,
the company announced aflibercept protein data suggesting that
Ixo-vec at the 2E11 and 6E10 doses deliver similar levels of
aflibercept that are both within the therapeutically active range
based on OPTIC and non-human primate data.
- Long-Term Ixo-vec
OPTIC Trial Data at AAO 2023:
- In November 2023,
the company announced 3-year data from the OPTIC extension study of
patients with wet AMD during the Retina Subspecialty Day at the
American Academy of Ophthalmology (AAO) 2023 Annual Meeting in San
Francisco, California.
- Patients in the
OPTIC extension trial continue to experience long-term benefit from
Ixo-vec through 3 years of follow-up, including maintenance of
vision, durability of anatomical improvements and sustained
reduction in anti-VEGF treatment burden.
- Patients at the 2E11
dose had an 84% reduction in annualized anti-VEGF injections, with
53% of the participants at the 2E11 dose receiving zero
supplemental injections through three years.
- Aflibercept protein
levels have been sustained through follow-up, which is up to 4.5
years post-treatment.
- BCVA was maintained
and CST was improved through 3 years.
- Ixo-vec was
generally well tolerated with the most common adverse event of
dose-dependent inflammation that was responsive to topical
corticosteroids.
- Scientific Meeting
Presentations: In October 2023, Adverum showcased non-clinical data
in two presentations at the European Society of Cell and Gene
Therapy 2023 Annual Meeting.
- Oral Presentation
Title: Dose-dependent inflammation signatures following Ixo-vec
administration in non-human primates
- Poster Presentation
Title: Functional and molecular evaluation of IVT AAV gene therapy
vectors for the treatment of GA
Corporate Updates:
- Appointment of
Andrew Ramelmeier, Ph.D. as Chief Technology Officer: In August
2023, the company announced the appointment of Dr. Andrew
Ramelmeier, as Chief Technology Officer, to lead Adverum’s
technology and operations organization, including process and assay
development for Ixo-vec. Dr. Ramelmeier has over three decades of
experience in drug development, clinical and commercial CMC, and
quality controls for gene and cell therapy products.
- Appointment of C.
David Nicholson, Ph.D. to Board of Directors: In November 2023, the
company announced the appointment of C. David Nicholson, Ph.D., as
an independent member of its Board of Directors, effective November
3, 2023. Dr. Nicholson brings over forty years of pharmaceutical
experience, a proven track record in drug development, and deep
experience in ophthalmology, most recently as chief research and
development officer at Allergan.
Anticipated Milestones
- Q4
2023: LUNA preliminary efficacy and safety data.
-
Mid-2024: Additional LUNA data, including the
26-week interim analysis.
Financial Results for the Three Months Ended September
30, 2023
- Cash, cash
equivalents and short-term investments were $117.1
million as of September 30, 2023, compared to $185.6 million as of
December 31, 2022. Adverum expects the September 30, 2023 cash
position to fund operations into 2025.
- Research and
development expenses were $20.7 million for the three
months ended September 30, 2023, compared to $23.8 million for the
same period in 2022. Research and development costs decreased due
to lower compensation, material production and bioanalytics
expenses, partially offset by higher facilities related expenses.
Stock-based compensation expense included in research and
development expenses was $1.2 million for the third quarter of
2023.
- General and
administrative expenses were $13.8 million for the
three months ended September 30, 2023, compared to $17.2 million
for the same period in 2022. General and administrative costs
decreased due to reversal of sublease income in prior year and
lower compensation expense, partially offset by higher professional
services expense. Stock-based compensation expense included in
general and administrative expenses was $3.2 million for the third
quarter of 2023.
- Net
Loss was $32.9 million, or $0.33 per basic and
diluted share, for the three months ended September 30, 2023,
compared to $40.1 million, or $0.40 per basic and diluted share,
for the same period in 2022.
About Wet Age-Related Macular DegenerationWet
AMD, also known as neovascular AMD or nAMD, is a VEGF driven
advanced form of AMD affecting approximately 10% of patients living
with AMD associated with the build-up of fluid in the macula and
the retina. Wet AMD is a leading cause of blindness in people over
65 years of age, with approximately 20 million individuals
worldwide living with this condition. New cases of wet AMD are
expected to grow significantly worldwide as populations age. AMD is
expected to impact 288 million people worldwide by 2040, with wet
AMD accounting for approximately 10% of those cases. Additionally,
wet AMD is a bilateral disease, and incidence of nAMD in the second
eye is up to 42% in the first two to three years. The current
standard of care requires frequent life-long repeated bolus
injections of anti-VEGF in the eye. IVT gene therapy has the
promise to preserve vision and reduce most or all injections for
the life of the patient by delivering stable therapeutic levels of
anti-VEGF to control macular fluid.
About Ixo-vec in Wet AMDAdverum is developing
ixoberogene soroparvovec (Ixo-vec, formerly referred to as
ADVM-022), its clinical-stage gene therapy product candidate, for
the treatment of wet AMD. Ixo-vec utilizes a proprietary vector
capsid, AAV.7m8, carrying an aflibercept coding sequence under the
control of a proprietary expression cassette. Unlike other
ophthalmic gene therapies that require surgery to administer the
gene therapy under the retina (sub-retinal approach), Ixo-vec is
designed to be administered as a one-time IVT injection in the
physician’s office, deliver long-term efficacy, reduce the burden
of frequent anti-vascular endothelial growth factor (VEGF)
injections, optimize patient compliance and improve vision outcomes
for patients with wet AMD. In recognition of the need for new
treatment options for wet AMD, the U.S. Food and Drug
Administration granted Fast Track designation for Ixo-vec for the
treatment of wet AMD. Ixo-vec has also received PRIME designation
from the European Medicines Agency and the Innovation Passport from
the United Kingdom’s Medicines and Healthcare Products Regulatory
Agency for the treatment of wet AMD.
About Adverum BiotechnologiesAdverum
Biotechnologies (NASDAQ: ADVM) is a clinical-stage company that
aims to establish gene therapy as a new standard of care for highly
prevalent ocular diseases with the aspiration of developing
functional cures to restore vision and prevent blindness.
Leveraging the capabilities of its proprietary intravitreal (IVT)
platform, Adverum is developing durable, single-administration
therapies, designed to be delivered in physicians’ offices, to
eliminate the need for frequent ocular injections to treat these
diseases. Adverum is evaluating its novel gene therapy candidate,
ixoberogene soroparvovec (Ixo-vec, formerly referred to as
ADVM-022), as a one-time, IVT injection for patients with
neovascular or wet age-related macular degeneration. Additionally,
by overcoming the challenges associated with current treatment
paradigms for debilitating ocular diseases, Adverum aspires to
transform the standard of care, preserve vision, and create a
profound societal impact around the globe. 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 benefits of Ixo-vec in the treatment of wet AMD, the
design of the LUNA trial, and anticipated preliminary and interim
efficacy and safety data from the LUNA trial. Actual results could
differ materially from those anticipated in such forward-looking
statements as a result of various risks and uncertainties,
including risks inherent to, without limitation: Adverum’s novel
technology, which makes it difficult to predict the timing of
commencement and completion of clinical trials; regulatory
uncertainties; enrollment uncertainties; the results of early
clinical trials not always being predictive of future clinical
trials and results; and the potential for future complications or
side effects in connection with use of Ixo-vec. Additional risks
and uncertainties facing Adverum are set forth under the caption
“Risk Factors” and elsewhere in Adverum’s Securities and Exchange
Commission (SEC) filings and reports, including Adverum’s Quarterly
Report on Form 10-Q for the quarter ended June 30, 2023 filed with
the SEC on August 10, 2023. 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, except as required by
law.
Corporate, Investor and Media InquiriesAdverum
Biotechnologies, Inc.E: ir@adverum.com
Adverum Biotechnologies, Inc. |
Consolidated Balance Sheets |
(In thousands) |
(Unaudited) |
|
|
|
September 30 |
|
December 31 |
|
|
|
2023 |
|
|
|
2022 |
|
|
|
(Unaudited) |
|
(1) |
Assets |
|
|
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
105,366 |
|
|
$ |
68,431 |
|
Short-term investments |
|
|
11,716 |
|
|
|
117,158 |
|
Prepaid expenses and other current assets |
|
|
7,514 |
|
|
|
5,006 |
|
Total current assets |
|
|
124,596 |
|
|
|
190,595 |
|
Property and equipment,
net |
|
|
15,497 |
|
|
|
34,927 |
|
Operating lease right-of-use
asset |
|
|
52,757 |
|
|
|
78,934 |
|
Restricted cash |
|
|
2,650 |
|
|
|
2,503 |
|
Deposit and other long-term
assets |
|
|
1,270 |
|
|
|
1,413 |
|
Total assets |
|
$ |
196,770 |
|
|
$ |
308,372 |
|
|
|
|
|
|
Liabilities and
stockholders' equity |
|
|
|
|
Current liabilities: |
|
|
|
|
Accounts payable |
|
$ |
2,170 |
|
|
$ |
2,238 |
|
Accrued expenses and other current liabilities |
|
16,362 |
|
|
|
16,767 |
|
Lease liability, current portion |
|
|
10,324 |
|
|
|
13,241 |
|
Total current liabilities |
|
|
28,856 |
|
|
|
32,246 |
|
Lease liability, net of
current portion |
|
|
65,200 |
|
|
|
93,561 |
|
Other noncurrent
liabilities |
|
|
- |
|
|
|
1,047 |
|
Total liabilities |
|
|
94,056 |
|
|
|
126,854 |
|
Stockholders' equity: |
|
|
|
|
Common stock |
|
|
10 |
|
|
|
10 |
|
Additional paid-in capital |
|
|
999,319 |
|
|
|
985,651 |
|
Accumulated other comprehensive loss |
|
|
(552 |
) |
|
|
(1,531 |
) |
Accumulated deficit |
|
|
(896,063 |
) |
|
|
(802,612 |
) |
Total stockholders'
equity |
|
|
102,714 |
|
|
|
181,518 |
|
Total liabilities and stockholders' equity |
|
$ |
196,770 |
|
|
$ |
308,372 |
|
(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 September 30, |
|
Nine months ended September 30, |
|
|
|
2023 |
|
|
|
2022 |
|
|
|
2023 |
|
|
|
2022 |
|
|
|
|
|
|
|
|
|
|
License revenue |
|
$ |
- |
|
|
$ |
- |
|
|
$ |
3,600 |
|
|
$ |
- |
|
|
|
|
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
|
20,740 |
|
|
|
23,849 |
|
|
|
62,398 |
|
|
|
77,078 |
|
General and
administrative |
|
|
13,789 |
|
|
|
17,188 |
|
|
|
39,035 |
|
|
|
46,117 |
|
Total operating expenses |
|
|
34,529 |
|
|
|
41,037 |
|
|
|
101,433 |
|
|
|
123,195 |
|
Operating loss |
|
|
(34,529 |
) |
|
|
(41,037 |
) |
|
|
(97,833 |
) |
|
|
(123,195 |
) |
Other income, net |
|
|
1,661 |
|
|
|
923 |
|
|
|
4,437 |
|
|
|
1,450 |
|
Net loss before income
taxes |
|
|
(32,868 |
) |
|
|
(40,114 |
) |
|
|
(93,396 |
) |
|
|
(121,745 |
) |
Income tax provision |
|
|
(17 |
) |
|
|
(17 |
) |
|
|
(55 |
) |
|
|
(55 |
) |
Net loss |
|
|
(32,885 |
) |
|
|
(40,131 |
) |
|
|
(93,451 |
) |
|
|
(121,800 |
) |
Net loss per share — basic and
diluted |
|
$ |
(0.33 |
) |
|
$ |
(0.40 |
) |
|
$ |
(0.93 |
) |
|
$ |
(1.23 |
) |
Weighted-average common shares
outstanding - basic and diluted |
|
|
100,999 |
|
|
|
99,475 |
|
|
|
100,693 |
|
|
|
99,027 |
|
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