Applied Genetic Technologies Corporation (Nasdaq: AGTC), a clinical
stage biotechnology company focused on the development and
commercialization of adeno-associated virus (AAV)-based gene
therapies for the treatment of rare and debilitating diseases with
an initial focus on inherited retinal diseases (IRDs), today
announced financial results for the quarter ended December 31,
2021.
“We enter 2022 with significant momentum from
the recent buildout of our management team, completion and
reporting of several milestones, and updates on future milestones
across our development pipeline, including key events for our
achromatopsia and X-linked retinitis pigmentosa programs in the
first half of the year,” said Sue Washer, President and CEO of
AGTC. “In achromatopsia, we believe we have identified an
appropriate dose for a potential Phase 2/3 trial in achromatopsia
B3 program and intend to move that program forward subject to the
receipt of end of phase 2 feedback from the FDA, expected in the
first half of this year. In the second quarter, we expect to
provide an update on our XLRP program with interim 3-month data
from the SKYLINE trial. We believe both the XLRP and achromatopsia
B3 programs have the potential to be best in class therapies for
patients and are excited to move these important therapies one step
closer to potential approval and commercialization. We are also
seeing progress in our earlier stage programs in ophthalmology,
otology and CNS and look forward to providing additional updates on
each of those programs throughout the year in addition to the
completion of our new manufacturing facility.”
Recent Highlights
AGTC-501 for X-linked Retinitis
Pigmentosa (XLRP)
The Company is currently conducting multiple
clinical trials of AGTC-501, its lead gene therapy development
program for the treatment of XLRP, including the ongoing Phase 1/2
dose ranging trial, the Phase 2 SKYLINE trial, and the Phase 2/3
VISTA trial in support of a potential filing of a Biologics License
Application (BLA). Earlier this year, the Company announced it had
exceeded the enrollment target in the Company’s Phase 2 SKYLINE
trial with a total of 14 patients enrolled across the high and low
dose groups.
In May 2021, the Company reported positive
12-month data from the highest dose groups in the Phase 1/2
clinical trial, including 12-month data from Groups 5 and 6 that
reflected a 50% response rate among patients who meet the inclusion
criteria for the SKYLINE and VISTA trials. Based on the data
generated to date, AGTC-501 also has a favorable safety profile.
These data, together with the data presented at the Fourteenth
International Symposium on Retinal Degeneration in September 2021
that indicated a correlation between improvements in macular
structure and visual function, add to the growing body of evidence
suggesting that AGTC-501 has the potential to be a best-in-class
treatment for patients with XLRP.
Over the next 12 months, the Company expects to
achieve the following milestones for AGTC-501:
- Release of
3-month interim SKYLINE trial results in 2Q 2022;
- Release of
24-month results from the ongoing Phase 1/2 clinical trial in 3Q
2022;
- Release of
12-month SKYLINE trial results in 1Q 2023; and
- Release of
interim VISTA trial results in 1H 2023.
Achromatopsia (ACHM)
The Company recently announced data from the
highest dose pediatric cohorts in the AGTC-401 and AGTC-402
clinical trials, which were consistent with the previously reported
adult data for these programs. Enrollment in both clinical trials
is complete.
The recent data from the Phase 1/2 clinical
trial of AGTC-401 in achromatopsia B3 (ACHMB3) patients are
supportive of prior results. The trial met its primary objective,
which was the identification of a well-tolerated dose that provides
clinical benefit to patients, and the Company intends to advance
AGTC-401 into the next stage of clinical development pending end of
Phase 2 feedback from the U.S. Food and Drug Administration (FDA)
that is expected in the first half of 2022. The most recent data
from the AGTC-401 trial included interim 3-month pediatric results
and additional adult and pediatric safety results, with follow up
as long as 24 months. In the 1.1E+12 vg/mL dose group, the Company
defined two pediatric patients (17 and 7 years old) as responders
based on improvements in visual sensitivity. Therefore, of the
three adults and four children (total n=7) in the 1.1E+12 vg/mL
dose group when including the adult data, four (>50%) are visual
sensitivity responders. These patients also had improvements in
quality of life as measured by a patient reported outcome survey
developed specifically for patients with achromatopsia. The two
other pediatric patients in the 1.1E+12 vg/mL dose group and two
pediatric patients under 7 years in the 3E+12vg/mL dose group
(total n=4) could not sufficiently concentrate and consistently
complete the visual sensitivity testing. Similar to other trials
where endpoints are adapted for young children, AGTC plans to work
closely with clinicians and regulators to develop potential
adaptations for younger patients for visual sensitivity testing.
The final pediatric patient in the 3E+12 vg/mL dose group could
complete the test but was a non-responder.
Results for the AGTC-402 clinical trial in
achromatopsia A3 (ACHMA3) patients are also consistent with
previously reported results in adults, providing no indication of
clinical improvements, and do not support further clinical
development. Most patients with ACHMA3 express a mutant protein
which the Company believes may have impacted results seen in
patients that received AGTC-402. This mutant protein is not
typically found in patients with CNGB3 mutations.
As previously reported, in both the ACHMB3 and
ACHMA3 trials and only in pediatric patients, treatment with the
highest doses of AGTC-401 and AGTC-402 led to three cases of severe
ocular inflammation, which were reported as Suspected Unexpected
Serious Adverse Reactions (SUSARs). No new additional SUSARs or
SAEs in adults or children, have been reported and the inflammation
in all previously reported SUSARs improved with an adjusted steroid
regimen. Two SUSARs (one in ACHMA3 and one in ACHMB3) have since
fully resolved and one (ACHMA3) continues to resolve, with all
three patients’ best corrected visual acuity returning to
baseline.
Bionic Sight Collaboration
In March 2021, the Company’s collaborator,
Bionic Sight, LLC (Bionic Sight), announced promising initial Phase
1/2 data in a retinitis pigmentosa optogenetics study that showed
that treated patients, all of whom have complete or near-complete
blindness, can now see light and motion, and, in two cases, can
detect the direction of motion. Bionic Sight is now moving to
enroll patients at higher doses in this trial.
Manufacturing Facility
Construction of AGTC’s new leased manufacturing
and quality control facility is in process with initial operations
at the facility expected to begin in 4Q 2022. The facility is a key
part of the Company’s strategy to expedite the potential BLA filing
and commercial launch, subject to FDA approval, of its candidate
for the treatment of XLRP, support the manufacture and supply of
materials for ACHMB3 late stage clinical trials, and other earlier
stage programs.
Preclinical Programs
In addition to its advanced clinical stage
development programs in XLRP and ACHMB3, AGTC has a broad and
diversified preclinical pipeline addressing patient populations
with significant unmet needs in ophthalmology and central nervous
system (CNS) indications, including AGTC-701 for the treatment of
the dry form of age-related macular degeneration (dAMD), AGTC-601
for the treatment of Frontotemporal Dementia (FTD) and AGTC-801 for
the treatment of Amyotrophic Lateral Sclerosis (ALS). In addition,
AGTC has a program targeting non-syndromic hearing loss through its
collaboration with Otonomy, Inc. (Otonomy). AGTC-701 and AGTC-601
are in advanced preclinical development with toxicology and
biodistribution studies planned for 2022 which if successful will
support IND filings. The program with Otonomy is expected to move
forward with an Investigational New Drug (IND) application in the
first half of 2023.
Leadership Appointments
With the recent additions of Abraham Scaria,
Ph.D. as Chief Scientific Officer and Hope D’Oyley-Gay as General
Counsel, AGTC has now completed the buildout of a world class
management team capable of leading the Company through the next
stages of anticipated growth. Dr. Scaria has more than 25 years of
experience in the biotech and pharmaceutical industries, with deep
knowledge in discovery research, preclinical studies and Phase 1/2
clinical trials, with a focus on gene therapies to treat rare
diseases. Ms. D’Oyley-Gay has more than 25 years of legal
experience, with the majority of her career spent in the healthcare
industry, and deep knowledge of pharmaceutical, gene therapy and
life sciences companies.
Financial Results for the Three and Six
Months Ended December 31, 2021
R&D Expenses: Research and
development expenses for the three and six months ended December
31, 2021, were $14.5 million and $26.8 million, respectively,
compared to $11.8 million and $23.4 million, respectively, during
the comparable 2020 periods. The increase of $3.4 million during
the 2021 six-month period was primarily due to: (i) increased
external spending for AGTC’s research and discovery program
targeting FTD, which was primarily due to planned material
production costs; (ii) increased external spending for the SKYLINE
trial due to planned clinical activities; and (iii) higher
employee-related costs from increased headcount. These increases
were partially offset by decreased external spending on the XLRP
development program, primarily due to a reduction in clinical
manufacturing activities to support the VISTA clinical trial, and
overall reduced spending on the ACHM development program.
G&A Expenses: General
and administrative expenses for the three and six months ended
December 31, 2021 were $4.0 million and $8.1 million, respectively,
compared to $3.3 million and $6.7 million, respectively, during the
comparable 2020 periods. The increase of $1.4 million during the
2021 six-month period was primarily due to compensation for new
employees, incremental share-based compensation costs and higher
operating and business development costs pertaining to AGTC’s
recurring operations, partially offset by lower legal fees.
Interest
Expense: Interest expense for the
three and six months ended December 31, 2021 increased by
$0.3 million and $0.7 million, respectively, when compared to
the comparable 2020 periods due to supplemental borrowing by the
Company in May 2021 under its amended loan agreement with Hercules
Capital, Inc.
Net Loss: The Company’s
net loss for the three and six months ended December 31, 2021 was
$19.1 million and $36.3 million, respectively, compared to $15.5
million and $30.8 million, respectively, during the comparable 2020
periods.
Financial Guidance: As
of December 31, 2021, the Company's cash and cash equivalents
totaled $72.8 million. Management believes that these
funds will be sufficient to allow the Company to generate data from
its ongoing and planned clinical programs and fund currently
planned research and discovery programs into calendar year
2023.
Conference Call and Webcast
AGTC will host a conference call and webcast to
review business operations and discuss financial results for the
quarter ended December 31, 2021 today at 8:00 a.m.
ET. To access the call, dial 877-407-6184 (US) or 201-389-0877
(outside of the US). A live webcast will be available in the Events
and Presentations section of AGTC’s Investor Relations site
at http://ir.agtc.com/events-and-presentations.
Please log in approximately 10 minutes prior to
the scheduled start time. The archived webcast will be available in
the Events and Presentations section of the Company's website.
About AGTC
AGTC is a clinical-stage biotechnology company
developing genetic therapies for people with rare and debilitating
ophthalmic, otologic and central nervous system (CNS) diseases.
AGTC is a leader in designing and constructing all critical gene
therapy elements and bringing them together to develop customized
therapies with the potential to address unmet patient needs. AGTC’s
most advanced clinical programs in X-linked retinitis pigmentosa
(XLRP) and achromatopsia (ACHM CNGB3) leverage its best-in-class
technology platform to potentially improve vision for patients with
inherited retinal diseases. Its preclinical programs build on the
Company’s industry leading AAV manufacturing technology and
scientific expertise. AGTC is advancing multiple important pipeline
candidates to address substantial unmet clinical need in
optogenetics, otology and CNS disorders, and has entered into
strategic collaborations with companies including Bionic Sight, an
innovator in the emerging field of optogenetics and retinal coding
and Otonomy, Inc., a biopharmaceutical company dedicated to the
development of innovative therapeutics for neurotology. For more
information, please visit https://agtc.com/.
Forward-Looking Statements
This release contains forward-looking statements
that reflect AGTC's plans, estimates, assumptions and beliefs,
including statements about the potential and expected timeline for
the Company’s late-stage development programs in X-Linked Retinitis
Pigmentosa (XLRP) and Achromatopsia (ACHM), the timing and
potential of the Company’s preclinical programs, partnered programs
and collaborations, and the timing for an impact of its planned
manufacturing facilities. Forward-looking statements include
information concerning possible or assumed future results of
operations, financial guidance, business strategies and operations,
preclinical and clinical product development and regulatory
progress, potential growth opportunities and potential market
opportunities. Forward-looking statements include all statements
that are not historical facts and can be identified by terms such
as "anticipates," "believes," "could," "seeks," "estimates,"
"expects," "intends," "may," "plans," "potential," "predicts,"
"projects," "should," "will," "would" or similar expressions and
the negatives of those terms. Actual results could differ
materially from those discussed in the forward-looking statements,
due to a number of important factors. Risks and uncertainties that
may cause actual results to differ materially include, among
others: gene therapy is still novel with only a few approved
treatments so far; AGTC cannot predict when or if it will obtain
regulatory approval to commercialize a product candidate or receive
reasonable reimbursement; uncertainty inherent in clinical trials
and the regulatory review process; risks and uncertainties
associated with drug development and commercialization; risks and
uncertainties associated with constructing and potentially
commencing operations in the Company’s leased manufacturing and
quality control facility; the direct and indirect impacts of the
ongoing COVID-19 pandemic on the Company’s business, results of
operations, and financial condition; factors that could cause
actual results to differ materially from those described in the
forward-looking statements are set forth under the heading "Risk
Factors" in our most recent annual report on Form 10-K, as updated
by subsequent quarterly reports on Form 10-Q and in other reports
we file with the SEC. Given these uncertainties, you should
not place undue reliance on these forward-looking statements. Also,
forward-looking statements represent management's plans, estimates,
assumptions and beliefs only as of the date of this release. Except
as required by law, we assume no obligation to update these
forward-looking statements publicly or to update the reasons actual
results could differ materially from those anticipated in these
forward-looking statements, even if new information becomes
available in the future.
APPLIED GENETIC TECHNOLOGIES
CORPORATIONCONDENSED BALANCE
SHEETS(Unaudited)
In thousands |
|
December 31, 2021 |
|
June 30, 2021 |
ASSETS |
|
|
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
72,771 |
|
|
$ |
105,052 |
|
Investments |
|
|
— |
|
|
|
2,000 |
|
Prepaid and other current assets |
|
|
2,597 |
|
|
|
2,655 |
|
Total current assets |
|
|
75,368 |
|
|
|
109,707 |
|
Property and equipment, net |
|
|
4,416 |
|
|
|
4,658 |
|
Intangible assets, net |
|
|
1,345 |
|
|
|
1,287 |
|
Investment in Bionic Sight,
LLC |
|
|
7,937 |
|
|
|
8,000 |
|
Right-of-use assets - operating
leases |
|
|
3,167 |
|
|
|
3,167 |
|
Right-of-use asset - financing
lease |
|
|
11 |
|
|
|
34 |
|
Other assets |
|
|
126 |
|
|
|
113 |
|
Total assets |
|
$ |
92,370 |
|
|
$ |
126,966 |
|
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
|
Current liabilities: |
|
|
|
|
Accounts payable |
|
$ |
1,674 |
|
|
$ |
1,879 |
|
Accrued and other liabilities |
|
|
14,486 |
|
|
|
14,500 |
|
Lease liabilities - operating |
|
|
1,227 |
|
|
|
1,116 |
|
Lease liability - finance |
|
|
13 |
|
|
|
38 |
|
Current portion of long-term debt |
|
|
6,714 |
|
|
|
2,181 |
|
Total current liabilities |
|
|
24,114 |
|
|
|
19,714 |
|
Lease liabilities - operating,
net of current portion |
|
|
3,133 |
|
|
|
3,418 |
|
Long-term debt, net of debt
discounts and deferred financing fees |
|
|
13,538 |
|
|
|
17,727 |
|
Other liabilities |
|
|
95 |
|
|
|
299 |
|
Total liabilities |
|
|
40,880 |
|
|
|
41,158 |
|
Stockholders’ equity: |
|
|
|
|
Preferred stock |
|
|
— |
|
|
|
— |
|
Common stock |
|
|
43 |
|
|
|
43 |
|
Additional paid-in capital |
|
|
327,235 |
|
|
|
325,245 |
|
Treasury stock |
|
|
(256 |
) |
|
|
(211 |
) |
Accumulated deficit |
|
|
(275,532 |
) |
|
|
(239,269 |
) |
Total stockholders’ equity |
|
|
51,490 |
|
|
|
85,808 |
|
Total liabilities and
stockholders’ equity |
|
$ |
92,370 |
|
|
$ |
126,966 |
|
APPLIED GENETIC TECHNOLOGIES
CORPORATIONCONDENSED STATEMENTS OF
OPERATIONS(Unaudited)
|
|
Three MonthsEnded December
31, |
|
Six MonthsEnded December 31, |
In thousands, except per share data |
|
|
2021 |
|
|
|
2020 |
|
|
|
2021 |
|
|
|
2020 |
|
Revenue |
|
$ |
— |
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
— |
|
|
|
|
|
|
|
|
|
|
Operating
expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
|
14,454 |
|
|
|
11,811 |
|
|
|
26,779 |
|
|
|
23,437 |
|
General and administrative and other |
|
|
3,989 |
|
|
|
3,304 |
|
|
|
8,089 |
|
|
|
6,740 |
|
Total operating expenses |
|
|
18,443 |
|
|
|
15,115 |
|
|
|
34,868 |
|
|
|
30,177 |
|
Loss from operations |
|
|
(18,443 |
) |
|
|
(15,115 |
) |
|
|
(34,868 |
) |
|
|
(30,177 |
) |
Other income (expense),
net: |
|
|
|
|
|
|
|
|
Investment income, net |
|
|
6 |
|
|
|
29 |
|
|
|
12 |
|
|
|
93 |
|
Interest expense |
|
|
(675 |
) |
|
|
(335 |
) |
|
|
(1,344 |
) |
|
|
(667 |
) |
Total other income (expense), net |
|
|
(669 |
) |
|
|
(306 |
) |
|
|
(1,332 |
) |
|
|
(574 |
) |
Loss before provision for income
taxes |
|
|
(19,112 |
) |
|
|
(15,421 |
) |
|
|
(36,200 |
) |
|
|
(30,751 |
) |
Provision for income taxes |
|
|
— |
|
|
|
20 |
|
|
|
— |
|
|
|
41 |
|
Loss before equity in net losses
of an affiliate |
|
|
(19,112 |
) |
|
|
(15,441 |
) |
|
|
(36,200 |
) |
|
|
(30,792 |
) |
Equity in net losses of an
affiliate |
|
|
(32 |
) |
|
|
(21 |
) |
|
|
(63 |
) |
|
|
(50 |
) |
Net loss |
|
$ |
(19,144 |
) |
|
$ |
(15,462 |
) |
|
$ |
(36,263 |
) |
|
$ |
(30,842 |
) |
Weighted average shares
outstanding: |
|
|
|
|
|
|
|
|
Basic |
|
|
42,886 |
|
|
|
25,883 |
|
|
|
42,855 |
|
|
|
25,850 |
|
Diluted |
|
|
42,886 |
|
|
|
25,883 |
|
|
|
42,855 |
|
|
|
25,850 |
|
Net loss per common
share: |
|
|
|
|
|
|
|
|
Basic |
|
$ |
(0.45 |
) |
|
$ |
(0.60 |
) |
|
$ |
(0.85 |
) |
|
$ |
(1.19 |
) |
Diluted |
|
$ |
(0.45 |
) |
|
$ |
(0.60 |
) |
|
$ |
(0.85 |
) |
|
$ |
(1.19 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
IR Contact: David Carey Lazar FINN
PartnersT: (212) 867-1768 david.carey@finnpartners.com
Corporate Contact:Jonathan LieberChief
Financial OfficerApplied Genetic Technologies CorporationT: (617)
843-5778jlieber@agtc.com
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