This presentation contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs. Forward-looking statements include statements regarding the company's (i) clinical and pre-clinical programs, including the proposed Phase 2/3 (Vista) trial design for XLRP, the anticipated timeline for initiating and reporting data in the Vista trial and the Phase 1/2 expansion (Skyline) trial for XLRP, (ii) ability to enroll patients (ii) regulatory progress with FDA, (iii) potential growth and market opportunities, and (iv) ability to be competitive. 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, which speak 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, whether as a result of

new information, new events or otherwise.

  • Attendees
    • Sue Washer CEO
    • Matthew Feinsod, Executive VP of Global Strategy and Development
    • Mark Shearman CSO
  • Agenda
    • Introductions and Overview
    • Update on XLRP product candidate clinical development
    • Q&A
    • Closing Remarks
  • Group 5 & Group 6 doses have responders at Month 12
    • 50% response rate (4/8) in patients who met Phase 2/3 inclusion criteria
      • Vista trial was powered to be statistically significant for a 50% response rate
    • One Group 5 patient who was a responder at Month 6, not a responder at Month 12
  • BCVA continues to show supportive evidence of a biological response at Month 12
    • Clear difference between treated eye and untreated eye
  • Early-lookdata shows two Group 4 patients with continued response at Month 24
    • Evidence for continued durability of response to treatment
    • Only three patients have reached Month 24; third patient was never a responder

Dose

C36 Change ≥7db

Number of

C36 Change ≥7db

Number of

@ ≥5 Loci at

@≥5 Loci at

Group

Responders

Responders

Month 6

Month 12

2#

Yes

1/1

No

0/1

No

No

Yes

Yes

No

No

4

No

2/7

No

2/7

Yes

Yes

No

No**

No

No

No

No

Yes

No

Yes

Yes

5

No**

3/7

No**

2/7

Yes

Yes

No

No

No

No

No**

No**

6

No**

2/4

No**

2/4

Yes

Yes

Yes

Yes

**One patient does not meet new inclusion criteria; so responders are 2/7 or 28%

Additional two patients do not meet strict criteria but treated eye is statistically better than untreated eye

**Three patients do not meet new inclusion criteria; so responders are 4/8 or 50%

Additional patient does not meet strict criteria but treated eye is statistically better than untreated eye

# There is an additional Group 2 patient with no microperimetry data available

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Applied Genetic Technologies Corp. published this content on 06 May 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 06 May 2021 12:36:06 UTC.