Annexon Reports Significant Progress with its Priority Programs and Third Quarter 2023 Financial Results
13 Novembre 2023 - 12:30PM
Annexon, Inc. (Nasdaq: ANNX), a clinical-stage biopharmaceutical
company developing a new class of complement-based medicines for
people living with devastating inflammatory-related diseases, today
highlighted portfolio progress and reported third quarter 2023
financial results.
“As the leading company focused on stopping C1q from initiating
and driving harmful inflammation where it starts on diseased
tissue, we’re highly encouraged by the consistency of the clinical
data demonstrating rapid and meaningful functional benefit across
multiple disease areas,” said Douglas Love, president and CEO of
Annexon. “With increasing regulatory recognition of our data in GBS
and GA in particular, and the potential of these programs to offer
significant therapeutic advantages over existing treatments, we are
sharply focused on bringing these classical complement therapies to
patients as swiftly as possible while also intentionally advancing
our first-in-kind classical complement small molecule program in an
array of autoimmune conditions. As a result, we have aligned our
efforts and resources on our three priority programs to drive
maximum value for patients and stakeholders in the near-to-mid
term.”
Recent Clinical Program Updates
- Continued Clinical and Regulatory Progress for ANX005
Pivotal Program in Guillain-Barré Syndrome (GBS): In the
third quarter, Annexon announced that the European Medicines Agency
(EMA) granted orphan drug designation to ANX005 for the treatment
of GBS. EMA orphan drug designation requires that a novel rare
disease therapeutic demonstrates the potential for significant
benefit over available therapies. For ANX005, the EMA’s designation
was based on a meta-analysis of past studies with ANX005 and
intravenous immunoglobulin (IVIg), demonstrating notable, early
improvement in muscle strength with ANX005 that translated into
observable gains in health status, including a reduction in the
need of mechanical ventilation. Importantly, Annexon also announced
that it has achieved target enrollment of 225 patients in the
randomized, double-blind, placebo-controlled Phase 3 trial of
ANX005 in patients with GBS. Coupled with prior FDA alignment on
the study’s primary endpoint and statistical analysis plan, this
enrollment milestone enables the company to deliver topline Phase 3
pivotal results in the first half of 2024.
- EMA PRIME Designation Granted to ANX007 for the
Treatment of Geographic Atrophy (GA): Also in the third
quarter, Annexon announced that EMA granted Priority Medicine
(PRIME) designation to ANX007 for the treatment of GA secondary to
age-related macular degeneration (AMD). EMA granted this
designation, which provides enhanced support to accelerate
development for priority medicines that target an unmet need, based
on the Phase 2 ARCHER trial data that showed a consistent, durable,
time and dose-dependent preservation of visual function in patients
with GA, as well as preclinical data supporting the protective
mechanism of ANX007 against photoreceptor damage and loss. ANX007
is the first therapeutic candidate to receive EMA PRIME designation
for GA, and the first ophthalmology product awarded the designation
that is not targeting a rare disease or is not a cell or gene
therapy. PRIME designation is a competitive process, with EMA
awarding PRIME to only approximately a quarter of all
requests.
- Additional Analyses from ARCHER Presented at the
American Academy of Ophthalmology (AAO) Conference: At AAO
in November, additional analyses from the ARCHER Phase 2 trial in
patients with GA were presented supporting time and dose-dependent
protection from vision loss. In a new analysis of best corrected
visual acuity (BCVA) in patients with ≥15 letter vision loss (loss
of three lines of vision on a standardized eye chart), ANX007’s
treatment effect increased over the course of the on-treatment
portion of the study. In that regard, the second six months of
ANX007 treatment provided greater protection against the loss of
visual function than the first six months compared to sham,
suggesting that ANX007 may provide a growing and durable treatment
effect over time. In addition, topline data from the final ARCHER
study of the full six-month off-treatment period were presented,
showing that ANX007’s protection against BCVA ≥15 letter vision
loss demonstrated during the treatment period was reversed once
ANX007 treatment was discontinued. These results underscore
the on-treatment effect of ANX007 therapy and further support the
strong and consistent benefit demonstrated of preserving vision as
measured by BCVA ≥15 letter vision loss. More than 25 GA Phase 2
and Phase 3 trials have been conducted, and none have shown
statistically significant preservation of BCVA ≥15 letter vision
loss, nor a significant false positive outcome, utilizing this
objective and well-established endpoint.
- Results of Single-Arm, Phase 1b Study of ANX009 Show
that the Classical Pathway is a Key Driver of Complement Activation
and Consumption in Patients with Active Lupus Nephritis
(LN): At the American Society of Nephrology's Kidney Week
2023 conference in November, data were presented from the Phase 1b
signal-finding trial of ANX009, which used a precision medicine
approach to identify patients with LN who have high baseline
complement activity. LN is an autoimmune disease for which
pathogenic anti-C1q antibodies (PACAs) enhance C1q activity and
uniquely amplify classical complement-mediated kidney inflammation
and damage. Initial results showed subcutaneous ANX009 was well
tolerated and demonstrated plasma C1q target engagement and
complement inhibition. Remarkably, inhibition of C1q rapidly
increased free/circulating PACA levels (consistent with decreased
deposition in the kidney) and inhibited all downstream markers of
complement consumption and activation (C4, C3 and C5). These
results indicate that C1q and the classical pathway are key drivers
of complement activation in LN, independent of the alternative and
lectin pathways, and that PACAs are a unique component of the
classical complement activation pathway. Consistent with the short
duration of this signal-finding study (3 weeks), changes in urinary
protein excretion were not observed as anticipated.
Key Near-Term Anticipated Milestones
Priority Programs
- ANX005 in GBS: Initial topline results from
the Phase 3 pivotal trial of ANX005 in patients with GBS are
anticipated during the first half of 2024. To enable focus on
priority programs, Annexon will not conduct an R&D Day in 2023,
and instead plans to host an event focused on the burden of
disease, current treatment paradigm and market opportunity for GBS
in the first half of 2024.
- ANX007 in GA: Interactions with U.S. and EU
regulatory authorities on a global Phase 3 pivotal program for
ANX007 are ongoing, with plans to provide an update by year-end
2023.
- ANX1502 in autoimmune diseases: Results from
the single and multiple-ascending dose Phase 1 trial in healthy
volunteers are expected to be reported by year-end 2023.
Next Wave Programs
- ANX005 in Amyotrophic Lateral Sclerosis (ALS):
Following encouraging preliminary results which showed slowing of
disease progression, full on-treatment data from the Phase 2a trial
are expected by early 2024.
- ANX005 in Huntington’s Disease (HD): Annexon
is assessing the initiation of a planned late-stage trial in HD in
2024 as the company prioritizes near-term pivotal development
activities for GBS, GA and ANX1502.
Third Quarter 2023 Financial Results
- Cash and operating runway: Cash and cash
equivalents and short-term investments were $165.3 million as of
September 30, 2023. Annexon continues to expect its cash, cash
equivalents and marketable securities as of September 30, 2023, to
be sufficient to fund the company’s planned operating expenses into
the second quarter of 2025.
- Research and development (R&D) expenses:
R&D expenses were $27.9 million for the quarter ended September
30, 2023, reflecting the advancement of the company’s priority
programs, including GBS, GA and ANX1502, compared to $27.9 million
for the quarter ended September 30, 2022.
- General and administrative (G&A) expenses:
G&A expenses were $6.9 million for the quarter ended September
30, 2023, compared to $8.2 million for the quarter ended September
30, 2022.
- Net loss: Net loss was $32.5 million or $0.43
per share for the quarter ended September 30, 2023, compared to
$35.1 million or $1.42 per share for the quarter ended September
30, 2022.
About Annexon
Annexon Biosciences (Nasdaq: ANNX) is a clinical-stage
biopharmaceutical company utilizing a distinct scientific approach
to stop C1q and all inflammatory aspects of classical complement
pathway activation before it starts. As the only company solely
focused on shutting down C1q, Annexon is developing a
fit-for-purpose pipeline of therapeutics designed to provide
meaningful benefits across multiple diseases of the body, brain and
eye. With proof-of concept data in both Guillain-Barré syndrome and
geographic atrophy, Annexon is rigorously advancing its mid-to
late-stage clinical trials to bring their potential treatments to
patients as quickly as possible. To learn more visit
annexonbio.com.
Forward Looking Statements
This press release contains forward-looking statements within
the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as
amended. In some cases, you can identify forward-looking statements
by terminology such as “aim,” “anticipate,” “assume,” “believe,”
“contemplate,” “continue,” “could,” “design,” “due,” “estimate,”
“expect,” “goal,” “intend,” “may,” “objective,” “plan,”
“positioned,” “potential,” “predict,” “seek,” “should,” “suggest,”
“target,” “on track,” “will,” “would” and other similar expressions
that are predictions of or indicate future events and future
trends, or the negative of these terms or other comparable
terminology. All statements other than statements of historical
facts contained in this press release are forward-looking
statements. These forward-looking statements include, but are not
limited to, statements about: anticipated milestones; timing of
program updates and enrollment completion for ANX005, ANX007 and
ANX1502; plans to engage with U.S. and EU regulatory agencies to
determine the optimal path forward for ANX007; cash operating
runway; the potential benefits from treatment with anti-C1q
therapy; potential of ANX007 to support strong beneficial impact on
preserving vision; timing of data reports; and continuing
advancement of the company’s portfolio. Forward-looking statements
are not guarantees of future performance and are subject to risks
and uncertainties that could cause actual results and events to
differ materially from those anticipated, including, but not
limited to, risks and uncertainties related to: the company’s
history of net operating losses; the company’s ability to obtain
necessary capital to fund its clinical programs; the early stages
of clinical development of the company’s product candidates; the
effects of public health crises on the company’s clinical programs
and business operations; the company’s ability to obtain regulatory
approval of and successfully commercialize its product candidates;
any undesirable side effects or other properties of the company’s
product candidates; the company’s reliance on third-party suppliers
and manufacturers; the outcomes of any future collaboration
agreements; and the company’s ability to adequately maintain
intellectual property rights for its product candidates. These and
other risks are described in greater detail under the section
titled “Risk Factors” contained in the company’s Annual Report on
Form 10-K and Quarterly Reports on Form 10-Q and the company’s
other filings with the SEC. Any forward-looking statements that the
company makes in this press release are made pursuant to the
Private Securities Litigation Reform Act of 1995, as amended, and
speak only as of the date of this press release. Except as required
by law, the company undertakes no obligation to publicly update any
forward-looking statements, whether as a result of new information,
future events or otherwise
Investor Contact:
Chelcie ListerTHRUST Strategic
Communicationschelcie@thrustsc.com
Media Contact:
Sheryl SeapyReal
Chemistry949-903-4750sseapy@realchemistry.com
ANNEXON, INC.Condensed Consolidated
Statements of Operations(in thousands, except
share and per share
amounts)(unaudited) |
|
|
|
Three Months
EndedSeptember 30, |
|
|
Nine Months
EndedSeptember 30, |
|
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
|
|
|
|
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development (1) |
|
$ |
27,893 |
|
|
$ |
27,862 |
|
|
$ |
90,489 |
|
|
$ |
83,966 |
|
General and administrative (1) |
|
|
6,888 |
|
|
|
8,207 |
|
|
|
23,225 |
|
|
|
24,938 |
|
Total operating expenses |
|
|
34,781 |
|
|
|
36,069 |
|
|
|
113,714 |
|
|
|
108,904 |
|
Loss from operations |
|
|
(34,781 |
) |
|
|
(36,069 |
) |
|
|
(113,714 |
) |
|
|
(108,904 |
) |
Interest and other income,
net |
|
|
2,299 |
|
|
|
1,015 |
|
|
|
7,368 |
|
|
|
1,340 |
|
Net loss |
|
$ |
(32,482 |
) |
|
$ |
(35,054 |
) |
|
$ |
(106,346 |
) |
|
$ |
(107,564 |
) |
Net loss per share, basic and
diluted |
|
$ |
(0.43 |
) |
|
$ |
(0.51 |
) |
|
$ |
(1.42 |
) |
|
$ |
(2.21 |
) |
Weighted-average shares used in
computing net loss per share,basic and diluted |
|
|
75,342,495 |
|
|
|
68,652,859 |
|
|
|
74,815,274 |
|
|
|
48,710,433 |
|
_______________________
(1) Includes the following
stock-based compensation expense: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
2,243 |
|
|
$ |
2,433 |
|
|
$ |
6,801 |
|
|
$ |
6,509 |
|
General and administrative |
|
$ |
2,306 |
|
|
$ |
2,478 |
|
|
$ |
7,015 |
|
|
$ |
7,174 |
|
ANNEXON, INC.Condensed Consolidated
Balance Sheets(in thousands) |
|
|
|
September 30, |
|
|
December 31, |
|
|
|
2023 |
|
|
2022 |
|
|
|
(Unaudited) |
|
|
|
|
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
133,159 |
|
|
$ |
140,020 |
|
Short-term investments |
|
|
32,112 |
|
|
|
102,637 |
|
Prepaid expenses and other current assets |
|
|
3,898 |
|
|
|
5,441 |
|
Total current assets |
|
|
169,169 |
|
|
|
248,098 |
|
Restricted cash |
|
|
1,032 |
|
|
|
1,032 |
|
Property and equipment, net |
|
|
15,310 |
|
|
|
16,838 |
|
Operating lease right-of-use
assets |
|
|
18,305 |
|
|
|
19,128 |
|
Total assets |
|
$ |
203,816 |
|
|
$ |
285,096 |
|
Liabilities and
Stockholders' Equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable |
|
$ |
4,809 |
|
|
$ |
7,416 |
|
Accrued liabilities |
|
|
10,490 |
|
|
|
13,448 |
|
Operating lease liabilities, current |
|
|
1,701 |
|
|
|
1,316 |
|
Other current liabilities |
|
|
170 |
|
|
|
180 |
|
Total current liabilities |
|
|
17,170 |
|
|
|
22,360 |
|
Operating lease liabilities,
non-current |
|
|
29,807 |
|
|
|
31,542 |
|
Total liabilities |
|
|
46,977 |
|
|
|
53,902 |
|
Stockholders’ equity: |
|
|
|
|
|
|
Common stock |
|
|
53 |
|
|
|
48 |
|
Additional paid-in capital |
|
|
701,517 |
|
|
|
669,780 |
|
Accumulated other comprehensive loss |
|
|
(123 |
) |
|
|
(372 |
) |
Accumulated deficit |
|
|
(544,608 |
) |
|
|
(438,262 |
) |
Total stockholders' equity |
|
|
156,839 |
|
|
|
231,194 |
|
Total liabilities and stockholders’ equity |
|
$ |
203,816 |
|
|
$ |
285,096 |
|
Grafico Azioni Annexon (NASDAQ:ANNX)
Storico
Da Ago 2024 a Set 2024
Grafico Azioni Annexon (NASDAQ:ANNX)
Storico
Da Set 2023 a Set 2024