GlobeNewswire Inc | Nov 16, 2020 04:05PM EST

November 16, 2020

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Nov. 16, 2020 (GLOBE NEWSWIRE) -- 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 announced financial results for the quarter ended September 30, 2020. The Company is also providing an update on its ongoing clinical trials in patients with X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM).

The updated XLRP data that we reported last week provide evidence that our XLRP product candidate provides durable improvements in visual sensitivity and visual acuity over a wide dose range and has a favorable safety profile, said Sue Washer, President and CEO of AGTC. Data publicly reported by our competitors this weekend at the American Academy of Ophthalmology (AAO) Annual Meeting further increase our confidence that we have the industry-leading XLRP program. We are on track to initiate enrollment in the planned Phase 1/2 expansion trial (Skyline) in 4Q 2020 and the Phase 2/3 trial (Vista) in 1Q 2021. Additionally, new data from our ongoing ACHM trials have provided important insights for the potential development of our ACHM product candidates. We are pleased that we met our goal of reporting out data from all three ongoing clinical trials in 4Q 2020 and are now focused on advancing the Skyline and Vista trials for XLRP as rapidly as possible.

XLRPOn November 11, 2020, the Company reported additional positive data from its Phase 1/2 clinical trial in patients with XLRP that indicated durable improvements observed in visual sensitivity and visual acuity over a wide dose range with a favorable safety profile out to month 12 in two of the dose groups. Based on comparison to publicly released data reported by competitors this weekend at the AAO Annual Meeting, the Company believes it has a best-in-class product that may provide significant benefit to patients with XLRP.

ACHMToday the Company is reporting 12-month ACHM data in the original three dose groups (low, medium and high), as well as 6- to 9-month data at two higher dose groups (higher and highest groups in the January 2020 data release), and data for 6 pediatric subjects (three in each study; 12-17 years old) at the first of three planned dose levels. While some patients showed improvements in at least one measure of visual function, no consistent sustained improvements were observed based on current assessments within the dose groups tested. However, anecdotal statements and assessments from patient-reported outcome surveys continue to provide the Company with confidence that patients are subjectively experiencing improved vision. Based on the characteristics of ACHM, the Company continues to believe that longer treatment durations and/or focusing on younger patients may be necessary to fully realize the potential of this treatment. To this end, the Company intends to complete the planned enrollment of pediatric patients in the two highest dose groups of the ongoing ACHM clinical trials and has amended the study protocol to allow enrollment of patients as young as four years of age and to include additional assessments such as functional MRI (fMRI) and improved color brightness tests.

Financial Results for the Three Months Ended September 30, 2020

R&D Expenses: Research and development expenses were $11.6million for the quarter ended September 30, 2020 compared to $8.6million during the comparable period in the prior fiscal year. The increase of $3.0million was primarily due to increased external XLRP spending (primarily related to Skyline and Vista activities) and increased external spending related to ACHM (primarily due to patient enrollment and deployment of the Companys mobile vision center). These expenses were partially offset by a reduction in employee-related costs and decreased external research and discovery spending on other programs.

G&A Expenses: General and administrative expenses were $3.4million for the quarter ended September 30, 2020 compared to $3.3million during the comparable period in the prior fiscal year. The increase of $0.1 million was primarily due to higher fees from outside legal counsel in the 2020 period, partially offset by lower employee-related expenses and share-based compensation expense.

Interest Expense: Interest expense increased by $0.3 million during the quarter ended September 30, 2020 when compared to the comparable period in the prior fiscal year due to the loan agreement that the Company entered into on June 30, 2020.

Net Loss The Companys net loss was $15.4million and $11.6 million for the quarters ended September 30, 2020 and 2019, respectively.

Financial Guidance: As of September30, 2020, the Companys cash, cash equivalents and investments totaled $66.6million. We believe that these funds will be sufficient to allow the Company to generate data from its ongoing clinical programs, initiate the Skyline and Vista studies and fund currently planned research and discovery programs into the fourth quarter of calendar year 2021.

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 Companys 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 (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its preclinical programs build on the Companys 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 XLRPXLRP 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 or 5th 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 ACHMACHM 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 ACHM, 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 projected timing for its planned Vista (Phase 2/3 XLRP)and Skyline (Expanded Phase 1/2 XLRP) clinical trials, the timing for reporting data in both its Skyline and Vista trials and the potential of its ACHM clinical 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 the Companys 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 AGTCs most recent annual and subsequently filed quarterly reports 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 CORPORATIONBALANCE SHEETS(Unaudited) September June 30, 30,In thousands, except per share data 2020 2020 ASSETS Current assets: Cash and cash equivalents $ 31,570 $ 38,463 Investments 34,987 41,995 Prepaid and other current assets 1,984 2,506 Total current assets 68,541 82,964 Property and equipment, net 4,314 4,311 Intangible assets, net 1,136 1,098 Investment in Bionic Sight, LLC 8,067 8,096 Right-of-use assets ? operating leases 3,337 3,422 Right-of-use asset ? finance lease 69 80 Other assets 151 348 Total assets $ 85,615 $ 100,319 LIABILITIES AND STOCKHOLDERS? EQUITY Current liabilities: Accounts payable $ 1,654 $ 1,355 Accrued and other liabilities 10,246 10,502 Lease liabilities ? operating 1,063 1,058 Lease liability ? finance 49 48 Total current liabilities 13,012 12,963 Lease liabilities ? operating, net of current 3,898 4,070 portionLease liability ? finance, net of current portion 26 38 Long-term debt, net of debt discounts and 9,759 9,677 deferred financing feesOther liabilities 2,718 2,555 Total liabilities 29,413 29,303 Stockholders? equity: Preferred stock, par value $0.001 per share,5,000 shares authorized; no shares issued and ? ? outstandingCommon stock, par value $0.001 per share, 150,000shares authorized; 25,901 and 25,813 sharesissued; 25,860 and 25,793 shares outstanding at 25 25 September 30, 2020 and June 30, 2020,respectivelyAdditional paid-in capital 253,208 252,519 Treasury stock at cost; 41 and 20 shares atSeptember 30, 2020 and June 30, 2020, (211 ) (88 )respectivelyAccumulated deficit (196,820 ) (181,440 )Total stockholders? equity 56,202 71,016 Total liabilities and stockholders? equity $ 85,615 $ 100,319

APPLIED GENETIC TECHNOLOGIES CORPORATIONSTATEMENTS OF OPERATIONS(Unaudited)

Three Months Ended September 30, In thousands, except per share data 2020 2019 Revenue: Collaboration revenue $ ? $ ? Grant revenue ? ? Total revenue ? ? Operating expenses: Research and development 11,626 8,642 General and administrative and other 3,436 3,348 Total operating expenses 15,062 11,990 Loss from operations (15,062 ) (11,990 )Other income (expense), net: Investment income, net 64 446 Interest expense (332 ) (2 )Total other income (expense), net (268 ) 444 Loss before provision for income taxes (15,330 ) (11,546 )Provision for income taxes 21 21 Loss before equity in net losses of an affiliate (15,351 ) (11,567 )Equity in net losses of an affiliate (29 ) (10 )Net loss $ (15,380 ) $ (11,577 )Weighted average shares outstanding: Basic 25,818 18,212 Diluted 25,818 18,212 Net loss per common share: Basic $ (0.60 ) $ (0.64 )Diluted $ (0.60 ) $ (0.64 )

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 Contacts:Bill SullivanChief Financial OfficerApplied Genetic Technologies CorporationT: (617) 843-5728bsullivan@agtc.com

Stephen PotterChief Business OfficerApplied Genetic Technologies CorporationT: (617) 413-2754spotter@agtc.com