This presentation contains forward-looking statements that reflect AGTC's plans, estimates and beliefs, including statements regarding the timing for
reporting data in both its XLRP and ACHM clinical programs, the timing and potential of the Company's pre-clinical programs, the potential of its
partnered programs and collaborations, the potential to partner future programs, and the timing for an impact of its planned manufacturing facilities and the funding needs for these programs. Forward-looking statements include information concerning our possible or assumed future results of operations, including results and timing of our clinical trials and planned clinical trials, business strategies and operations, financing plans, 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 AGTC's 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," "hopes,"
"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 report on Form 10-K filed with the Securities and Exchange Commission as supplemented by subsequently filed quarterly reports on Form 10-Q. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent our 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.
XLRP: Lead Phase 2/3
Ophthalmology
Candidate
Potential Best-in-Class
Late-Stage Asset
Improvements in visual sensitivity and acuity sustained for at least twelve months
No product-related SAEs
across 100-fold dose
range
Key clinical data readouts
expected in 2022
High-Value
Clinical Programs
ACHMB3 Data
Visual sensitivity and light
discomfort improvements support continued clinical development
Bionic Sight
Partnership
Encouraging signs of
significant improvements in
function vision
Integrated Gene Therapy Platform
Differentiated with
Demonstrated
Capabilities
Broad pre-clinical data
Robust use of disease- specific animal modeling
Novel, patent protected
vector components
Manufacturing
Unmatched
Productivity,
Scalability & Quality
10-fold reduction in
process residuals and
10-fold increase in
productivity
At commercial scale:
40 L ≥ 2000 ophthalmic
doses
IND ENABLING | PHASE 1/2 | PHASE 2/3 | NEXT MILESTONE* | Patient Population (approx.) | |||
OCULAR | |||||||
XLRP | RPGR (ORPHAN DRUG) | Skyline 3-month data 2Q 22 | 20,000 (US and EU) | ||||
Vista interim analysis 1H 23 | |||||||
ACHM | CNGB3 (ORPHAN DRUG) | 3-month Pediatric data both trials 1Q 22 | |||||
27,000 (US and EU) | |||||||
CNGA3 (ORPHAN DRUG) | EOP2 Feedback 1H 22 | ||||||
Retinitis Pigmentosa | UNIQUE CHR & NEUROPROSTHETIC | Partnership with Bionic Sight; complete dosing | 20,000 (US and EU) | ||||
Dry AMD | COMPLEMENT FACTOR H | IND filing | 1,000,000 GA (US) | ||||
NEURODEGENERATIVE
FTD
ALS
PROGRANULIN | IND filing; partnering effort | 50,000 (US) | |
Development candidate; partnering effort | 30,000 (US) | ||
C9ORF72 | |||
OTOLOGY
Non-Syndromic | GJB2 | Partnership with Otonomy; IND 1H 23 | 200,000 (US and EU) |
Hearing Loss | |||
*Anticipated milestones based on calendar year, not AGTC's fiscal year
Capsid Diversity | Promoter | Gene Cassette | Manufacturing | Clinical Development |
Targeted capsid | Optimized | Expression of | Unmatched | Patient focus |
for each cell | expression in | high-fidelity | productivity and | enhances |
type | target cells | gene products | quality | outcomes |
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Applied Genetic Technologies Corp. published this content on 11 January 2022 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 11 January 2022 13:17:07 UTC.