Applied Genetic Technologies Corporation (Nasdaq: AGTC), a
biotechnology company conducting human clinical trials of
adeno-associated virus (AAV)-based gene therapies for the treatment
of rare diseases, today provided additional information about the
proposed design of the planned Phase 2/3 trial for its X-linked
retinitis pigmentosa (XLRP) clinical program, which is expected to
commence in the first quarter of 2021, and new preliminary data on
a higher dose Group 5 from the ongoing Phase 1/2 XLRP trial.
The Company also announced financial results for the fourth quarter
and fiscal year ended June 30, 2020.
“We are excited to close out our 2020 fiscal
year with a path toward initiating a Phase 2/3 trial for our XLRP
program, and to provide new preliminary data for the higher dose
Group 5 in our Phase 1/2 trial, which supports inclusion of that
dose level in the Phase 2/3. In addition, we remain on track to
report additional data readouts from all three of our clinical
programs in the fourth quarter of the 2020 calendar year,” said Sue
Washer, President and CEO of AGTC. “The advancements we are making
in our clinical programs, highlighted by the proposed design of our
planned Phase 2/3 XLRP trial, underscore our continued
progress.”
Recent Highlights
X-linked Retinitis Pigmentosa
(XLRP)
The proposed design of the XLRP Phase 2/3 trial
is expected to include approximately 60 patients randomized across
three arms: a low-dose group (1.2E+11 vg/mL the Group 2 dose from
the ongoing Phase 1/2 trial), a high-dose group (1.1E+12 vg/mL the
Group 5 dose from the ongoing Phase 1/2 trial), and an untreated
control group. The primary endpoint will be based on visual
sensitivity, with a responder defined as having at least 7 decibel
improvement in visual sensitivity in at least 5 loci at month 12,
which is intended to represent a clinically meaningful benefit.
Responder rates in each active arm will be compared to responder
rates in the control arm. The Company plans to submit a 6-month
interim analysis of the data from the Phase 2/3 to the FDA to
obtain feedback on the Company’s development plan to support
approval. Based on any FDA feedback, the Company may modify
the final trial design, enrollment numbers, and/or statistical
analysis plan. The Company also will discuss with FDA the
possibility of finalizing dose selection for the treatment of the
contralateral eye based on this 6-month interim data. The Company
expects to begin enrolling patients in 1Q 2021 and to provide
results from the 6-month interim analysis in 3Q 2022, dependent on
future effects of the COVID-19 pandemic on clinical trial
enrollment.
- Analysis of visual sensitivity data
from patients in the ongoing Phase 1/2 XLRP trial shows that 7 of
15 patients in Groups 2, 4 and 5 are responders at month 6 (month 3
in one case) based on the responder criteria defined above.
Focusing on the Phase 1/2 Group 5 dose group, a dose level that the
Company is planning to use as the high dose in the Phase 2/3 trial,
4 of 7 patients met the response criteria. Of note, using the
planned inclusion/exclusion criteria for the Phase 2/3 trial, one
Phase 1/2 Group 5 patient would be removed such that the responder
rate would be 4 of 6, or 67%.
- The Company also plans to expand
its ongoing Phase 1/2 trial to include approximately 12 additional
patients who will be masked and randomized to doses of 1.2E+11
vg/mL (Group 2 in original trial plan) and 1.1E+12 vg/mL (Group 5
in original trial plan). The Company expects to begin enrolling
these additional patients in 4Q 2020 and to provide results from a
3-month interim analysis in 4Q 2021, dependent on future effects of
the COVID-19 pandemic on clinical trial enrollment.
- AGTC remains on track to provide
12-month data from the ongoing Phase 1/2 trial by 4Q 2020 for
Groups 1-4 to evaluate durability of effect and continued safety,
as well as full 6-month data analysis for Groups 5 and 6 to
evaluate safety and efficacy at higher doses.
- The Company remains on-track to
have clinical trial material produced in time for the initiation of
the Phase 2/3 trial with its advanced manufacturing process that
provides improved yields, purity and potency.
Achromatopsia (ACHM)
- In January 2020, AGTC announced
encouraging interim data from the dose-escalation cohorts of its
ongoing Phase 1/2 clinical programs in patients with ACHM due to
mutations in the ACHM CNGB3 or ACHM CNGA3 genes. In March, AGTC
completed the planned enrollment in all dose groups for adult
patients (age 18 years or older), including the two higher dose
groups, of both ACHM trials, bringing the total number of adults
dosed to 15 in the ACHM A3 trial and 22 in the ACHM B3 trial.
Pediatric dosing is ongoing with four pediatric patients dosed in
each trial to date. The Company expects that pediatric enrollment
may continue to be challenging.
- The Company plans to release
additional data for the adult dose groups in 4Q 2020, which will be
used to inform decision-making regarding readiness to move the
product candidates to pivotal trials.
Preclinical Programs
AGTC’s preclinical pipeline includes two
ophthalmology programs, one of which targets the dry form of
age-related macular degeneration (AMD), and three programs
targeting central nervous system (CNS) disorders. The CNS programs
target adrenoleukodystrophy (ALD) and two additional rare genetic
CNS indications—frontotemporal dementia (FTD) and amyotrophic
lateral sclerosis (ALS)—that have substantial patient populations
and well-defined clinical phenotypes. AGTC also has collaborations
with Otonomy and Bionic Sight for genetic forms of hearing loss and
optogenetics, respectively.
Mobile Vision Testing
Program
In June 2020, AGTC announced that it launched a
mobile vision testing program to conduct follow-up assessments
during the COVID-19 pandemic for patients enrolled in the Company’s
ongoing clinical trials in XLRP and ACHM. The mobile vision
testing program is available to patients enrolled in AGTC’s
clinical trials across the United States so that they are able to
maintain their follow-up study assessments while COVID-19
restrictions remain in effect. To date, more than 25 patients have
been seen in the mobile vision testing program.
Financial Results for the Fourth Quarter
and Fiscal Year Ended June 30, 2020
Revenue: There was no revenue
for the fourth quarter of 2020 and $2.5 million for the year ended
June 30, 2020, compared to $0.4 million and $41.7 million in the
comparable periods in fiscal year 2019. Revenue for the year ended
June 30, 2020 was primarily $2.2 million of non-cash collaboration
revenue in connection with the in-kind contributions made to Bionic
Sight.
R&D Expenses:
Research and development expenses were $10.5 million for the fourth
quarter of 2020 and $35.8 million for the year ended June 30, 2020,
compared to $8.3 million and $33.2 million in the comparable
periods in fiscal year 2019. The increase of $2.6 million during
the full year period was primarily due to increased external XLRP
spending primarily related to Phase 2/3 activities, increased
employee-related costs and increased external spending related to
ACHM, primarily due to patient enrollment. These expenses were
partially offset by decreased external research and discovery
spending and share-based compensation expenses.
G&A Expenses: General
and administrative expenses were $4.1 million for the fourth
quarter of 2020 and $13.6 million for the fiscal year ended June
30, 2020, compared to $3.5 million and $12.9 million in the
comparable periods in fiscal year 2019. The increase in general and
administrative expenses for the full year was primarily driven by
increased employee-related expenses partially offset by a decrease
in share-based compensation expenses.
Net Income (Loss): Net loss was
$14.5 million for the fourth quarter of 2020 and $45.9 million for
the year ended June 30, 2020, compared to net loss of $10.5 million
and $2.0 million in the comparable periods in 2019.
Financial Guidance: As of
June 30, 2020, the Company's cash, cash equivalents and investments
totaled $80.5 million. The Company believes these funds will
be sufficient to allow AGTC to generate data from its ongoing
clinical programs, initiate a Phase 2/3 on XLRP trial and to fund
currently planned research and discovery programs into the fourth
quarter of calendar year 2021.
Conference Call and WebcastAGTC will host a
conference call and webcast with accompanying slides to review the
Phase 2/3 XLRP trial design and discuss financial results for the
fourth quarter and fiscal year ended June 30, 2020 today
at 8:00am 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 AGTCAGTC 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 that address real patient
needs. The Company’s most advanced clinical programs leverage its
best-in-class technology platform to potentially improve vision for
patients with an inherited retinal disease. AGTC has active
clinical trials in X-linked retinitis pigmentosa and achromatopsia
(ACHM CNGB3 & ACHM CNGA3). Its pre-clinical 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.
About X-linked Retinitis Pigmentosa
(XLRP)XLRP is an inherited condition that causes
progressive vision loss in boys and young men. Characteristics of
the disease include night blindness in early childhood and
progressive constriction of the visual field. In general, XLRP
patients experience a gradual decline in visual acuity over the
disease course, which results in legal blindness around the 4th
decade of life. AGTC was granted U.S. Food and Drug Administration
(FDA) orphan drug designation in 2017, as well as European
Commission orphan medicinal product designation in 2016, for its
gene therapy product candidate to treat XLRP caused by mutations in
the RPGR gene.
About Achromatopsia (ACHM)Achromatopsia is an
inherited retinal disease, which is present from birth and is
characterized by the lack of cone photoreceptor function. The
condition results in markedly reduced visual acuity, extreme light
sensitivity causing day blindness, and complete loss of color
discrimination. Best-corrected visual acuity in persons affected by
achromatopsia, even under subdued light conditions, is usually
about 20/200, a level at which people are considered legally
blind.
Forward-Looking StatementsThis release contains
forward-looking statements that reflect AGTC's plans, estimates,
assumptions and beliefs, including statements regarding the timing
for and expected expansion of its planned Phase 2/3 XLRP clinical
trial, the timing for reporting data in both its XLRP and ACHM
clinical programs and the funding needs for these programs.
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, potential market opportunities, the effects of
competition and the impact of the COVID-19 pandemic, including the
impact on its ability to enroll patients. 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; the direct and indirect impacts of the ongoing
COVID-19 pandemic on our 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 or quarterly report and in other reports we have
filed 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
CORPORATION BALANCE SHEETS
(Unaudited)
|
|
June 30, |
|
In thousands, except per share data |
|
2020 |
|
|
2019 |
|
ASSETS |
|
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
38,463 |
|
|
$ |
26,703 |
|
Investments |
|
|
41,995 |
|
|
|
55,292 |
|
Grants receivable |
|
|
— |
|
|
|
13 |
|
Prepaid and other current assets |
|
|
2,506 |
|
|
|
2,276 |
|
Total current assets |
|
|
82,964 |
|
|
|
84,284 |
|
Property and equipment, net |
|
|
4,311 |
|
|
|
4,430 |
|
Intangible assets, net |
|
|
1,098 |
|
|
|
1,013 |
|
Investment in Bionic Sight,
LLC |
|
|
8,096 |
|
|
|
1,945 |
|
Right-of-use assets - operating
leases |
|
|
3,422 |
|
|
|
— |
|
Right-of-use asset - financing
lease |
|
|
80 |
|
|
|
— |
|
Other assets |
|
|
348 |
|
|
|
544 |
|
Total assets |
|
$ |
100,319 |
|
|
$ |
92,216 |
|
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
|
Accounts payable |
|
$ |
1,355 |
|
|
$ |
1,331 |
|
Accrued and other liabilities |
|
|
10,502 |
|
|
|
8,024 |
|
Lease liabilities - operating |
|
|
1,058 |
|
|
|
— |
|
Lease liability - finance |
|
|
48 |
|
|
|
— |
|
Total current liabilities |
|
|
12,963 |
|
|
|
9,355 |
|
Lease liabilities - operating,
net of current portion |
|
|
4,070 |
|
|
|
— |
|
Lease liability - finance, net of
current portion |
|
|
38 |
|
|
|
— |
|
Long-term debt, net of debt
discounts and deferred financing fees |
|
|
9,677 |
|
|
|
— |
|
Other liabilities |
|
|
2,555 |
|
|
|
4,152 |
|
Total liabilities |
|
|
29,303 |
|
|
|
13,507 |
|
Stockholders’ equity: |
|
|
|
|
|
|
|
|
Preferred stock, par value $0.001 per share, 5,000 shares
authorized; no shares issued and outstanding |
|
|
— |
|
|
|
— |
|
Common stock, par value $0.001 per share, 150,000 shares
authorized; 25,813 and 18,226 shares issued; 25,793 and 18,207
shares outstanding atJune 30, 2020 and 2019, respectively |
|
|
25 |
|
|
|
18 |
|
Additional paid-in capital |
|
|
252,519 |
|
|
|
214,324 |
|
Shares held in treasury of 20 and 19 at June 30, 2020 and 2019,
respectively |
|
|
(88) |
|
|
|
(85) |
|
Accumulated deficit |
|
|
(181,440) |
|
|
|
(135,548) |
|
Total stockholders’ equity |
|
|
71,016 |
|
|
|
78,709 |
|
Total liabilities and
stockholders’ equity |
|
$ |
100,319 |
|
|
$ |
92,216 |
|
APPLIED GENETIC TECHNOLOGIES
CORPORATION STATEMENTS OF OPERATIONS
(Unaudited)
|
|
Three Months Ended June 30, |
|
|
Year Ended June 30, |
|
In
thousands, except per share data |
|
2020 |
|
|
|
2019 |
|
|
|
2020 |
|
|
|
2019 |
|
|
Revenue: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Collaboration revenue |
|
$ |
– |
|
|
|
$ |
– |
|
|
|
$ |
2,297 |
|
|
|
$ |
41,128 |
|
|
Grant and other revenue |
|
|
– |
|
|
|
|
406 |
|
|
|
|
156 |
|
|
|
|
564 |
|
|
Total revenue |
|
|
– |
|
|
|
|
406 |
|
|
|
|
2,453 |
|
|
|
|
41,692 |
|
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
10,453 |
|
|
|
|
8,332 |
|
|
|
|
35,778 |
|
|
|
|
33,183 |
|
|
General and administrative and other |
|
|
4,127 |
|
|
|
|
3,514 |
|
|
|
|
13,617 |
|
|
|
|
12,859 |
|
|
Total operating expenses |
|
|
14,580 |
|
|
|
|
11,846 |
|
|
|
|
49,395 |
|
|
|
|
46,042 |
|
|
Loss from operations |
|
|
(14,580 |
) |
|
|
|
(11,440 |
) |
|
|
|
(46,942 |
) |
|
|
|
(4,350 |
) |
|
Other income
(expense), net: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Investment income, net |
|
|
122 |
|
|
|
|
505 |
|
|
|
|
1,185 |
|
|
|
|
2,009 |
|
|
Other income (expense), net |
|
|
(5 |
) |
|
|
|
446 |
|
|
|
|
(5 |
) |
|
|
|
446 |
|
|
Total other income (expense), net |
|
|
117 |
|
|
|
|
951 |
|
|
|
|
1,180 |
|
|
|
|
2,455 |
|
|
Loss before provision for income taxes |
|
|
(14,463 |
) |
|
|
|
(10,489 |
) |
|
|
|
(45,762 |
) |
|
|
|
(1,895 |
) |
|
Provision for income taxes |
|
|
20 |
|
|
|
|
19 |
|
|
|
|
83 |
|
|
|
|
76 |
|
|
Loss before equity in net losses of an affiliate |
|
|
(14,483 |
) |
|
|
|
(10,508 |
) |
|
|
|
(45,845 |
) |
|
|
|
(1,971 |
) |
|
Equity in net losses of an affiliate |
|
|
(20 |
) |
|
|
|
(6 |
) |
|
|
|
(47 |
) |
|
|
|
(35 |
) |
|
Net loss |
|
$ |
(14,503 |
) |
|
|
$ |
(10,514 |
) |
|
|
$ |
(45,892 |
) |
|
|
$ |
(2,006 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted average
shares outstanding: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic |
|
|
25,769 |
|
|
|
|
18,184 |
|
|
|
|
21,102 |
|
|
|
|
18,157 |
|
|
Diluted |
|
|
25,769 |
|
|
|
|
18,184 |
|
|
|
|
21,102 |
|
|
|
|
18,157 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per common
share: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic |
|
$ |
(0.56 |
) |
|
|
$ |
(0.58 |
) |
|
|
$ |
(2.17 |
) |
|
|
$ |
(0.11 |
) |
|
Diluted |
|
$ |
(0.56 |
) |
|
|
$ |
(0.58 |
) |
|
|
$ |
(2.17 |
) |
|
|
$ |
(0.11 |
) |
|
IR/PR CONTACTS: David Carey (IR) or Glenn
Silver (PR)Lazar FINN PartnersT: (212) 867-1768 or (646)
871-8485david.carey@finnpartners.com or
glenn.silver@finnpartners.com
Corporate Contact:Bill SullivanChief Financial OfficerApplied
Genetic Technologies CorporationT: (617)
843-5728bsullivan@agtc.com
Stephen PotterChief Business OfficerApplied Genetic Technologies
CorporationT: (617) 413-2754spotter@agtc.com
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