Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in ocular and rare
diseases, today reported financial results for the first quarter
ended March 31, 2019 and provided a corporate update.
“Our primary focus is on advancing ADVM-022, our novel gene
therapy designed as a single-administration treatment for wet AMD,”
said Leone Patterson, chief executive officer of Adverum
Biotechnologies. “For ADVM-022, we presented new 30-month
preclinical safety and expression data at ASGCT and second eye
preclinical safety data at ARVO. We look forward to presenting the
first clinical data from the OPTIC phase 1 trial for ADVM-022 in
the second half of this year. Beyond wet AMD, we continue to
evaluate other ocular VEGF-related indications for this novel gene
therapy to treat additional patients with ocular disease.
Ms. Patterson continued, “In addition, we recently welcomed Dr.
Aaron Osborne as our new chief medical officer and Thomas Leung as
our new chief financial officer as we assemble a leadership team
with deep industry experience and commitment to developing novel
gene therapies for patients. At the board level, we recently
appointed three new members who broaden the set of financial,
operational, and development experiences and can add valuable
insights as we execute our development plans for our pipeline of
gene therapies.”
Recent Program Updates
• At the ASGCT 22nd Annual Meeting in May 2019,
Adverum presented new 30-month safety data on ADVM-022
(AAV.7m8-aflibercept) in wet age-related macular degeneration (wet
AMD) from a preclinical study. Data presented in an oral
presentation showed a single intravitreal (IVT) injection of
ADVM-022 (2 × 10^12 vg/eye) was safely administered and
provided long-term, sustained delivery of aflibercept up to 30
months at therapeutic levels in non-human primate (NHP) eyes.
Long-term intraocular expression of aflibercept did not affect the
retina structure or function and long-term suppression of VEGF
activity did not cause retinal atrophy.
• At the ARVO 2019 Annual Meeting in April 2019,
Adverum presented for the first time preclinical data on dosing the
second eye with a single IVT injection of ADVM-022. Data showed
ADVM-022 was safely administered to the contralateral eye, two
months after administering to the first eye, in NHPs. Findings from
this study could prove valuable when designing AAV-mediated gene
therapy protocols to treat wet AMD patients with pre-existing
vector immunity, or those with bi-lateral disease.
• In April 2019, Adverum announced the completion of
enrollment and dosing of patients (n=6) in the first cohort in the
OPTIC phase 1 clinical trial of ADVM-022 in patients with wet AMD.
The independent data monitoring committee (DMC) determined that
enrollment and dosing of patients in the second cohort could
proceed. This was based on a review of the preliminary safety data
from the first cohort of patients, which has shown no serious
adverse events (SAEs) or dose-limiting toxicities (DLTs) following
a single intravitreal injection of ADVM-022 at the initial trial
dose of 6 x 10^11 vg/eye. No patient has experienced an SAE, with a
follow up period of up to five months.
• In early April 2019, Adverum received a
notification from the FDA requesting additional chemistry,
manufacturing, and controls (CMC) information and requirements on
the ADVM-022 manufacturing process and placing Adverum’s IND
application for ADVM-022 for the treatment of wet AMD on clinical
hold. Adverum subsequently submitted its response and should the
agency decide to lift the clinical hold, Adverum expects to resume
dosing patients in the OPTIC trial.
Recent Corporate Updates
• In May 2019, Adverum announced the appointment of
Rekha Hemrajani, M.B.A., James Scopa, J.D., M.B.A., and Mark
Lupher, Ph.D. to its board of directors and the appointment of
current board member Patrick Machado, J.D. as board chair.
• In April 2019, Aaron Osborne, MBBS MRCOphth joined
Adverum as chief medical officer and Thomas Leung joined as
chief financial officer.
Future Outlook - Planned Milestones
ADVM-022 for Wet AMD
• Report 24-week primary and secondary outcomes from
the first cohort of patients in the OPTIC phase 1 clinical trial at
a scientific meeting in the second half of 2019
Rare Disease Programs
• Provide an update on Adverum’s A1AT deficiency and
hereditary angioedema programs by midyear 2019
Manufacturing Capabilities
• Occupy Adverum’s new facility in Redwood City by
the end of this year. This facility will serve as Adverum’s
new corporate headquarters and includes the expansion of its
in-house process development capabilities to the 1000 liter
scale
Upcoming Events
• Adverum plans to participate in the following
upcoming conference:
- American Society of Virology (ASV) 2019 Annual Meeting in
Minneapolis, MN, July 20-24, 2019 oral presentation:-
Abstract Title: Analysis of the structural differences
between AAV2.7m8 and its parental capsid AAV2 by Cryo-EM-
Program Number: W5-2- Session Title: Oncolytic
Viruses- Date: July 20, 2019- Time: 7:00 pm
CT- Location: University of Minnesota, Moos 2-690
Financial Results for the Three Months Ended March 31,
2019
• Cash, cash equivalents and short-term
investments were $189.5 million as of March 31, 2019,
compared to $205.1 million as of December 31, 2018.
Adverum expects this quarter-end cash position to fund operations
into 2021.• Revenue, consisting of
revenue from collaborative research, was $0.0 million the three
months ended March 31, 2019, compared to $0.2 million for the same
period in 2018. Revenue decreased as no research activities were
performed under collaboration agreements for the three month period
in 2019.• Research and development
expenses were $10.1 million for the three months ended
March 31, 2019, compared to $12.8 million for the same period in
2018. Research and development expenses decreased due to the
discontinuation of Adverum’s ADVANCE phase 1 clinical trial for
ADVM-043, partially offset by an increase related to the ongoing
OPTIC phase 1 clinical trial for ADVM-022.•
General and administrative expenses were
$5.6 million for the three months ended March 31, 2019, compared to
$5.4 million for the same period in 2018. General and
administrative expenses increased primarily due to higher
professional expenses.• Net loss attributable
to common stockholders was $14.5 million, or $0.23 per
basic and diluted share, for the three months ended March 31, 2019,
compared to $17.2 million, or $0.30 per basic and diluted share,
for the same period in 2018.
About Adverum Biotechnologies,
Inc.Adverum is a clinical-stage gene therapy company
targeting unmet medical needs in ocular and rare diseases. Adverum
develops gene therapy product candidates designed to provide
durable efficacy by inducing sustained expression of a therapeutic
protein. As a leader in ophthalmic gene therapy, Adverum has
collaboration agreements with Regeneron Pharmaceuticals and Editas
Medicine. Adverum’s core capabilities include clinical development,
novel vector discovery and in-house manufacturing expertise,
specifically in scalable process development, assay development,
and current Good Manufacturing Practices quality control. 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
Adverum’s plans for advancing ADVM-022, statements regarding the
expected timing of reporting first clinical data for the OPTIC
trial, the statements under the heading “Future Outlook -
Planned Milestones,” and Adverum’s expectations that its current
cash position will fund its operations into 2021, 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 consummate any of
these 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, without limitation, the risk that
Adverum’s resources will not be sufficient for Adverum to conduct
or continue planned development programs and planned clinical
trials, the risk that the FDA’s clinical hold may not be lifted in
a timely manner or at all, and the risk of a delay in the
enrollment of patients in Adverum’s clinical studies or in the
manufacturing of products to be used in such clinical studies.
Risks and uncertainties facing Adverum are described more fully in
Adverum’s Form 10-K filed with the SEC on March 6, 2019,
particularly 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.
ADVERUM BIOTECHNOLOGIES, INC. |
|
CONDENSED CONSOLIDATED BALANCE SHEETS |
|
(In thousands) |
|
(Unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
March 31, |
|
|
December 31, |
|
|
|
2019 |
|
|
2018 |
|
ASSETS |
|
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
144,107 |
|
|
$ |
154,949 |
|
Short-term investments |
|
|
45,381 |
|
|
|
50,130 |
|
Prepaid expenses and other current assets |
|
|
3,649 |
|
|
|
3,675 |
|
|
|
|
|
|
|
|
|
|
Total current assets |
|
|
193,137 |
|
|
|
208,754 |
|
|
|
|
|
|
|
|
|
|
Operating lease right to use asset |
|
|
22,592 |
|
|
|
— |
|
Property and equipment, net |
|
|
4,145 |
|
|
|
3,586 |
|
Restricted cash |
|
|
999 |
|
|
|
999 |
|
Deposits and other long-term assets |
|
|
174 |
|
|
|
156 |
|
|
|
|
|
|
|
|
|
|
Total assets |
|
$ |
221,047 |
|
|
$ |
213,495 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
LIABILITIES AND STOCKHOLDERS'
EQUITY |
|
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
|
Accounts payable |
|
$ |
1,752 |
|
|
$ |
1,707 |
|
Accrued expenses and other current liabilities |
|
|
5,032 |
|
|
|
8,784 |
|
Lease liability, current portion |
|
|
3,826 |
|
|
|
– |
|
Deferred rent, current portion |
|
|
— |
|
|
|
228 |
|
|
|
|
|
|
|
|
Total current liabilities |
|
|
10,610 |
|
|
|
10,719 |
|
|
|
|
|
|
|
|
|
|
Deferred rent, less current portion |
|
|
— |
|
|
|
1,366 |
|
Lease liability, less current portion |
|
|
22,078 |
|
|
|
— |
|
Other non-current liabilities |
|
|
216 |
|
|
|
243 |
|
|
|
|
|
|
|
|
|
|
Total liabilities |
|
|
32,904 |
|
|
|
12,328 |
|
|
|
|
|
|
|
|
|
|
Stockholders’ equity |
|
|
188,143 |
|
|
|
201,167 |
|
|
|
|
|
|
|
|
|
|
Total liabilities and stockholders’ equity |
|
$ |
221,047 |
|
|
$ |
213,495 |
|
|
|
|
|
|
|
|
ADVERUM BIOTECHNOLOGIES, INC. |
CONDENSED CONSOLIDATED STATEMENTS OF
OPERATIONS |
(In thousands, except per share amounts) |
(Unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
|
2019 |
|
2018 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Collaboration and license revenue |
$ |
- |
|
|
$ |
216 |
|
|
|
|
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
10,131 |
|
|
|
12,794 |
|
|
General and administrative |
|
5,576 |
|
|
|
5,368 |
|
|
|
|
|
|
|
|
|
|
Total operating expenses |
|
15,707 |
|
|
|
18,162 |
|
|
|
|
|
|
|
|
|
|
Operating loss |
|
(15,707 |
) |
|
|
(17,946 |
) |
|
|
|
|
|
|
|
|
|
Other income (expense), net |
|
1,218 |
|
|
|
746 |
|
|
|
|
|
|
|
|
|
Net loss |
$ |
(14,489 |
) |
|
$ |
(17,200 |
) |
|
|
|
|
|
|
|
|
Net loss per share, basic and diluted |
$ |
(0.23 |
) |
|
$ |
(0.30 |
) |
|
|
|
|
|
|
|
|
|
Weighted-average common shares outstanding, |
|
|
|
|
|
|
basic and diluted |
|
63,125 |
|
|
|
57,420 |
|
|
|
|
|
|
|
|
|
|
Investor and Media Inquiries:
Amy Figueroa, CFA
Investor Relations Consultant
afigueroa@adverum.com
650-823-2704
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