UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

 

FORM 8-K

 

 

CURRENT REPORT

PURSUANT TO SECTION 13 OR 15(D)

OF THE SECURITIES EXCHANGE ACT OF 1934

Date of Report (Date of earliest event reported): September 9, 2020

 

 

APPLIED GENETIC TECHNOLOGIES CORPORATION

(Exact Name of Registrant as Specified in Charter)

 

 

 

Delaware   001-36370   59-3553710

(State or other jurisdiction

of incorporation)

 

(Commission

File Number)

 

(IRS Employer

Identification Number)

14193 NW 119th Terrace

Suite 10

Alachua, Florida, 32165

(Address of principal executive offices) (Zip Code)

(386) 462-2204

(Registrant’s telephone number, including area code)

Not Applicable

(Former Name or Former Address, if Changed Since Last Report)

 

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below).

 

Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

 

Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

 

Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

 

Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

 

Title of each class

 

Trading Symbol(s)

 

Name of each exchange on which registered

Common Stock, $0.001 par value   AGTC   Nasdaq Global Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company  ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.  ☐

 

 

 


Item 2.02.

Results of Operations and Financial Condition

On September 9, 2020, Applied Genetic Technologies Corporation (the “Company”) issued a press release entitled “AGTC Provides Trial Design Update for its Phase 2/3 XLRP Clinical Trial, New Data on XLRP Higher Dose Group and Financial Results for the Fourth Quarter and Year Ended June 30, 2020.” The press release is furnished as Exhibit 99.1.

 

Item 7.01.

Regulation FD Disclosure.

The Company will present during its earnings call on September 9, 2020 a summary of recent corporate and clinical developments. A copy of the slide presentation that will be used by officers of the Company in connection with the earnings call (the “Corporate Presentation”) is attached to this Current Report on Form 8-K as Exhibit 99.2. The Corporate Presentation is current as of September 9, 2020, and the Company disclaims any obligation to correct or update this material in the future.

The information in the press release attached as Exhibit 99.1 and the corporate presentation attached as Exhibit 99.2 is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934 (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933 or the Exchange Act, except as expressly set forth by specific reference in such filing.

 

Item 9.01.

Financial Statements and Exhibits.

(d) Exhibits.

 

Exhibit
No.

  

Description

99.1    Press release dated September  9, 2020, entitled “AGTC Provides Trial Design Update for its Phase 2/3 XLRP Clinical Trial, New Data on XLRP Higher Dose Group and Financial Results for the Fourth Quarter and Year Ended June 30, 2020”
99.2    Corporate Presentation current as of September 9, 2020

 

2


SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.

 

APPLIED GENETIC TECHNOLOGIES CORPORATION

By:  

/s/ William A. Sullivan

  William A. Sullivan
  Chief Financial Officer

Date: September 9, 2020

 

3

EX-99.1

Exhibit 99.1

 

LOGO

AGTC Provides Trial Design Update for its Phase 2/3 XLRP Clinical Trial, New Data on XLRP Higher Dose Group and Financial Results for the Fourth Quarter and Year Ended June 30, 2020

- Planned Phase 2/3 trial expected to include approximately 60 patients and a responder analysis based on at least 7 decibel improvement in visual sensitivity in at least 5 loci across two active dose groups and one control group -

- Data from Group 5 supports use of higher dose level in Phase 2/3 trial -

- Company on track to provide multiple data readouts for XLRP and ACHM clinical programs in 4Q 2020 -

- Company to host management update and webcast with slides today at 8:00am ET -

GAINESVILLE, Fla., and CAMBRIDGE, Mass., September 9, 2020 – 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.”

 

1


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.

 

2


   

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.

 

3


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.

 

4


Conference Call and Webcast

AGTC 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 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 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.

 

5


Forward-Looking Statements

This 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.

# # #

 

6


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 at June 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  
  

 

 

   

 

 

 

 

7


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 Partners

T: (212) 867-1768 or (646) 871-8485

david.carey@finnpartners.com or glenn.silver@finnpartners.com

 

8


Corporate Contact:

Bill Sullivan

Chief Financial Officer

Applied Genetic Technologies Corporation

T: (617) 843-5728

bsullivan@agtc.com

Stephen Potter

Chief Business Officer

Applied Genetic Technologies Corporation

T: (617) 413-2754

spotter@agtc.com

 

9

EX-99.2

Slide 1

2020 10-K Conference Call September 9, 2020 Exhibit 99.2


Slide 2

Forward-Looking Statements This presentation contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs. Forward-looking statements include statements regarding the timing for and expected expansion of its XLRP clinical development program, the timing for reporting data in its XLRP and ACHM clinical programs, and its ability to enroll patients, effectively design and successfully complete its ongoing clinical trials, financial guidance, business strategies and operations, regulatory progress, potential growth and market opportunities, and the effects of competition. 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: 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 related to the COVID-19 outbreak that may delay clinical trials; gene therapy is still novel with only a few approved treatments so far; 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 the Company's most recent Annual Report on Form 10-K and subsequent periodic 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 presentation. 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. 


Slide 3

Call Agenda Recent accomplishments and latest developments – Sue Washer, CEO Clinical program update & preclinical pipeline review – Dr. Mark S. Shearman, CSO 4Q 2020 financial results review – Bill Sullivan, CFO Q&A Closing Remarks – Sue Washer, CEO


Slide 4

Recent Accomplishments Received detailed EOP2 feedback from the FDA on the design of our Phase 2/3 protocol and future regulatory filings for our XLRP trial On track to initiate a Phase 2/3 trial in 1Q 2021 Expanding ongoing Phase 1/2 trial in order to dose approximately 12 patients in two masked dosing arms to collect additional data, including a mobility course added as a supplemental endpoint New preliminary data for Group 5 patients in the Phase 1/2 XLRP trial available Launched nationwide mobile vision testing to support our ongoing Phase 1/2 XLRP and ACHM trials Formalized our Patient Advisory Council to integrate patient and caregiver voices into our clinical and preclinical development strategies Initial focus on XLRP Insights from patients and caregivers with real-world understanding of XLRP and other inherited retinal disorders


Slide 5

A Pipeline with Significant Clinical and Commercial Potential   DISCOVERY IND ENABLING PHASE 1/2 PHASE 3 NEXT MILESTONE* XLRP RPGR         High Dose Data 4Q 2020 Expand ongoing Phase 1/2 trial 4Q 2020 Initiate Phase 2/3 trial 1Q 2021 ACHM CNGB3         Interim Data New Adult Groups 4Q 2020 ACHM CNGA3         Interim Data New Adult Groups 4Q 2020 Optogenetics Unique ChR         Partnership with Bionic Sight; Complete Dosing Otology GJB2         Partnership with Otonomy; IND Filing FTD, ALS Progranulin, c9orf72 Final product construct testing ALD ABCD1 IND enabling Studies Dry AMD Complement Factor H IND enabling Studies Stargardts ABCA4 IND Enabling Studies * Milestones based on calendar year and not AGTC’s fiscal year


Slide 6

Industry Leading Gene Therapy Company Poised for Success XLRP: Lead Product Candidate Advancing Toward Phase 2/3 Trial in 1Q 2021 Sustained Improvements in visual function for centrally dosed patients Expanding ongoing Phase 1/2 trial; expect dosing to begin in 4Q 2020 Expect enrollment of Phase 2/3 trial to begin in 1Q 2021 ACHM: Early Evidence of Biological Activity in Both ACHM Trials Dose escalation completed in adult patients Best-in-Class Technology Platform Process and scale expected to support late-phase development and commercialization Expertise in product design allows for best capsid, promoter and gene cassettes for each indication Robust use of disease specific animal modeling to guide clinical dosing Deep expertise and capabilities to support robust pipeline in ophthalmology, CNS, and otology


Slide 7

Positioned for Success in a Changing Landscape XLRP: Direct Path to Phase 2/3 in 1Q 2021 Expanding Phase 1/2 trial to increase total data set Received EOP2 FDA feedback Manufacturing of Phase 2/3 material on track Redundant manufacturing options contracted; CDMOs are considered essential businesses in their locations ACHM Adult enrollment complete in both trials 8 of 18 pediatric patients across both trials enrolled DSMC approval to highest pediatric dose


Slide 8

Clinical Programs Update


Slide 9

Leading XLRP Program Moving Toward 1Q 2021 Phase 2/3 Sustained treatment effect, favorable safety profile Evidence of increase and expansion in central visual sensitivity Encouraging improvements in visual acuity Quality of life survey results support meaningful impact on patients’ lives Phase 1/2 trial initial targeted enrollment complete—28 patients in six dose groups All patients dosed continue to demonstrate a favorable safety profile


Slide 10

Centrally Dosed Responders Show Sustained Increase in Visual Sensitivity (4 of 8 Evaluable Patients) = standard deviation Treated Untreated Baseline Month 1 Month 2 Month 3 Month 6 Change from Baseline Mean Sensitivity (dB) Data from Groups 2 & 4


Slide 11

Treated Eyes Show Positive Trends in Visual Acuity 7/9 patients show improving visual acuity UNTREATED EYE TREATED EYE BCVA (ETDRS letter score) BCVA (ETDRS letter score) Change from Baseline Change from Baseline Baseline Month 1 Month 2 Month 3 Month 6 Baseline Month 1 Month 2 Month 3 Month 6 Data from Groups 2 & 4


Slide 12

Re-Analysis of Visual Sensitivity Data At Month 6 * Month 3 data 7 of 15 centrally-treated patients in combined Groups 2, 4 & 5 had at least 7 decibel increase at 5 or more loci


Slide 13

GROUP 5 GROUP 5 GROUP 4 Octopus Perimetry Example Data – Pointwise Analysis Month 6 TREATED EYE UNTREATED FELLOW EYE Loss ≥ 7.0dB Gain ≥ 7.0dB Change ≤ 7.0dB 16 loci within 30° 41 loci within 30° 16 loci within 30° UNTREATED FELLOW EYE TREATED EYE UNTREATED FELLOW EYE TREATED EYE Loss ≥ 7.0dB Gain ≥ 7.0dB Change ≤ 7.0dB Loss ≥ 7.0dB Gain ≥ 7.0dB Change ≤ 7.0dB Month 3 Month 3


Slide 14

Strong XLRP Competitive Position Six dose groups over a 100-fold range in concentration Well designed vector construct Meaningful improvements in visual function sustained over time Favorable safety profile No product related SAEs No recurrent inflammation Earlier groups now 12-24 months past enrollment date with continued safety AGTC BIIB MeiraGTx Patients Dosed 28 18 10 BCVA + - Unknown Construct ++Expression +Stability -Expression +Stability -Expression -Stability Sensitivity ++ + + Safety ++ Secondary Inflammation Secondary Inflammation Competitive Comparison* *Based on publicly available information


Slide 15

Proposed Phase 2/3 Trial Design = Sub-retinal Treatment with AGTC-501 Group 2 (N~20) Group 5 (N~20) Untreated (N~20) M3b M6b M1b M9b M12b All eligible untreated control eyes will receive treatment after their M12 visit Screening & Randomization Interim Analysis at M6 * * * * 2 3 1 M12 M3 M6 M1 M9 M18 Y2 Y3 Y4 Y5 6M I/A 12M Endpoint Revised protocol includes two masked active arms, untreated control


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Phase 1/2 Trial Expansion Group 2 (N=6) Group 5 (N=6) Contralateral eye dosing Amend Phase 1/2 = Sub-retinal Treatment with AGTC-501 * * * * 3M I/A Informal Interim Analysis at M3 M12 M3 M6 M1 M9 Screening & Randomization 2 1 12M Endpoint Additional patients will be masked & randomized to two doses


Slide 17

XLRP Moving Forward to Phase 2/3 Trial in 1Q 2021 On track to finalize Phase 2/3 protocol for initiation in 1Q 2021 Clinical trial material in-process Overlapping CDMOs and GLP testing labs—on track to support trial initiation in 1Q 2021 Next data report 4Q 2020 Twelve-month analysis for Groups 1-4—evaluate durability of effect and continued safety Interim analysis for Groups 5 and 6—evaluate safety and efficacy at higher doses 2 1 3


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ACHM Clinical Trials Update Early signs of biologic activity supported by patient-reported outcomes No dose-limiting toxicity observed DSMC-approved dosing of pediatric patients to highest dose Adult enrollment complete 22 adult patients enrolled in ACHM B3 15 adult patients enrolled in ACHM A3 Pediatric enrollment ongoing 4 of 9 patients enrolled in ACHM B3 4 of 9 patients enrolled in ACHM A3 Data on File


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DISCOVERY IND ENABLING PHASE 1/2 PHASE 3 Milestone/Product Differentiation Dry AMD Complement Factor H IND Enabling Tox Studies; engineered gene cassette Stargardts ABCA4 IND Enabling Tox Studies Otology GJB2         IND Filing; novel capsid tropism profile; partnered with Otonomy ALD ABCD1 IND Enabling Tox Studies Frontotemporal Dementia, ALS Progranulin, c9orf72 Final product construct testing; Novel trivalent cassette components Exciting Preclinical Pipeline Advancing multiple important pipeline candidates in ophthalmology, otology and central nervous system disorders Manageable short-term capital investment for potential long-term significant value creation


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Proprietary Pivotal-Stage Manufacturing Platform Enhanced Productivity & Quality Unmatched productivity, characterization Highly scalable, extensive regulatory vetting Up to 2000 ophthalmic doses from 50 L Most process residuals below level of detection for the current assay Compared to early phase AAV manufacturing method Significant Productivity Increase COGs Decrease 90X Achievable with aggressive scaling Fold Decrease in Estimated COGs Sedimentation Coefficient and Peak Area Determination: Nearly 90%, full-length genome final product encapsidation ~90% Full AAV Volumetric Productivity 70x 60x 50x 40x 30x 20x 10x N=3 N=4 N=6 N=3


Slide 21

Q4 Financial Results


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Financial Results 4Q 2020 net loss of $14.5 million compared to net loss of $10.5 million in 4Q 2019 Strong balance sheet $80.5 million in cash, cash equivalents, and investments as of June 30, 2020 Runway into 4Q 2021 The company believes these funds will be sufficient to allow AGTC to: Generate data from ongoing clinical programs Initiate a Phase 2/3 registration trial in XLRP Fund currently planned prioritized preclinical programs


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Q&A


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Upcoming Virtual Investor Conferences 2020 Wells Fargo Virtual Healthcare Conference   Ms. Washer will participate in a fireside chat at 9:20 a.m. ET on Thursday, September 10 H.C. Wainwright 22nd Annual Global Investment Virtual Conference AGTC management will participate in investor 1x1 meetings on Monday, September 14 and Tuesday, September 15 Cantor Fitzgerald Virtual Global Healthcare Conference 2020 Ms. Washer will participate in a fireside chat at 2:40 p.m. ET on Wednesday, September 16


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2020 10-K Conference Call September 9, 2020