This presentation contains forward-looking statements that reflect Applied Genetic Technologies Corporation's ("AGTC" or the "Company") plans, estimates, assumptions and beliefs. Forward-looking statements include statements regarding AGTC's proposed Phase 2/3 trial design for XLRP and the proposed Phase 1/2 expansion trial for XLRP, including the effectiveness of
AGTC's pre-selection methodology for pre-specifying loci, its anticipated timeline and potential to receive FDA approval to begin the XLRP pivotal trial, the timing for completing and reporting data in its XLRP and ACHM clinical programs, its ability to enroll patients for its clinical trials, the expected costs of its clinical programs and its planned manufacturing activities, 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 continue to progress
its clinical trials, 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 trial enrollment; 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, AGTC assumes no obligation to update or revise these forward-looking statements publicly, whether as a result of new information, new events or otherwise.
Science
Differentiated With
Demonstrated
Capabilities
Broad pre-clinical data
Robust use of disease- specific animal modeling
Manufacturing XLRP
Unmatched | Potential Best-in-Class |
Productivity, | Late-Stage |
Scalability & Quality | Asset |
10-fold reduction in | Improvements in visual |
process residuals and | function sustained for at |
10-fold increase in | least six months |
productivity | No product related |
At commercial scale: 40 L | SAEs across 100-fold |
≥ 2000 ophthalmic doses | dose range |
Diversified
Pipeline
High-Value
Programs
1Q 21 ACHM data
shows visual sensitivity improvements support continued clinical development
Diversified programs across ophthalmology, neurodegenerative and otology indications
IND ENABLING | PHASE 1/2 | PHASE 2/3 | NEXT MILESTONE* | KEY CAPABILITY | |
OCULAR | |||||
XLRP | RPGR (ORPHAN DRUG) | 3-month data Skyline 4Q 21 | |||
6-month data Vista 3Q 22 | |||||
ACHM | CNGB3 (ORPHAN DRUG) | 12-month Adult data both trials 2Q 21 | |||
3-month Pediatric data both trials 4Q 21 | |||||
CNGA3 (ORPHAN DRUG) | |||||
Retinitis Pigmentosa | UNIQUE CHR & NEUROPROSTHETIC | Partnership with Bionic Sight; complete dosing | |||
Dry AMD | COMPLEMENT FACTOR H | IND filing | |||
Stargardts | ABCA4 | IND filing | |||
NEURODEGENERATIVE | |||||
FTD | PROGRANULIN | IND filing; partnering effort | |||
ALS | C9ORF72 | Development candidate; partnering effort | |||
OTOLOGY |
Non-Syndromic | GJB2 | Partnership with Otonomy; IND filing | ||
Hearing Loss | ||||
*Anticipated milestones based on calendar year, not AGTC's fiscal year | ||||
This is an excerpt of the original content. To continue reading it, access the original document here.
Attachments
- Original document
- Permalink
Disclaimer
Applied Genetic Technologies Corp. published this content on 01 February 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 01 February 2021 21:27:04 UTC.