UNITED STATES
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CURRENT REPORT
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Item 7.01. | Regulation FD Disclosure. |
On February 8, 2022, Applied Genetic Technologies Corporation (the “Company”) issued a press release reporting updated interim results from its ongoing Phase 1/2 dose escalation studies for Achromatopsia (ACHM). A copy of this press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K.
The Company will have a call to discuss the data at 8:00 a.m. on February 8, 2022. A copy of the slide presentation that will be used by officers of the Company in connection with the 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 February 8, 2022, and the Company disclaims any obligation to correct or update this material in the future.
The information in this Item 7.01 and Exhibits 99.1 and 99.2 attached hereto are intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall they be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, 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 February 8, 2022. | |
99.2 | Corporate Presentation current as of February 8, 2022. | |
104 | The cover page from this Current Report on Form 8-K, formatted in Inline XBRL. |
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/ Jonathan I. Lieber | |
Jonathan I. Lieber | ||
Chief Financial Officer |
Date: February 8, 2022
Exhibit 99.1
AGTC Announces Robust Improvements in Visual Sensitivity in Adult and Pediatric Patients, and Plans to Continue Clinical Development of AGTC-401 in Patients with CNGB3 Achromatopsia
February 8, 2022, at 7:00 a.m. EDT
| AGTC-401 demonstrated a favorable safety profile through second-highest dose (1.1E+12 vg/mL) in ACHMB3 pediatric patients |
| At the dose of 1.1E+12 vg/mL greater than 50% of the combined adults and children that received AGTC-401 were responders based on improvements in visual sensitivity |
| Totality of data from adult and pediatric patients indicate biologic activity and reaffirm previous positive safety findings in adults |
| Company to host conference call to review data today at 8:00 a.m. ET |
GAINESVILLE, Fla., and CAMBRIDGE, Mass., Feb.8, 2022 (GLOBE NEWSWIRE) Applied Genetic Technologies Corporation (Nasdaq: AGTC), a clinical stage biotechnology company focused on the development and commercialization of adeno-associated virus (AAV)-based gene therapies for the treatment of rare and debilitating diseases with an initial focus on inherited retinal diseases, today reported updated interim three- month pediatric results and additional adult safety results out to as long as 24 months from its ongoing Phase 1/2 dose escalation study of AGTC-401. AGTC-401 is a recombinant AAV viral vector-based gene therapy targeting mutations in the CNGB3 gene in patients with achromatopsia (ACHMB3). Data from seven pediatric patients support previously reported findings from adults treated with AGTC-401, which demonstrated encouraging biologic activity and a favorable safety profile. There were no new Suspected Unexpected Serious Adverse Reactions (SUSARs) and for the three previously reported SUSARs, the inflammation continues to improve with steroid treatment. Based on the totality of the ACHMB3 data generated to date from 31 patients over as long as 24 months, AGTC plans to advance the clinical development of AGTC-401 subject to consultation with the U.S. Food and Drug Administration (FDA) at an End-of-Phase 2 (EOP2) meeting in the first half of 2022.
The Company also reported updated results from a parallel study of AGTC-402 targeting CNGA3 mutations in patients with achromatopsia that are consistent with previously reported results in adults, provide no indication of clinical improvements, and do not support further clinical development. Most patients with CNGA3 mutations express a mutant protein that is not typically found in patients with CNGB3 mutations, which the Company believes may have impacted results seen in patients that received AGTC-402. AGTC will continue to follow the ACHMA3 patients for long-term safety observations.
The three-month pediatric findings provide further evidence for the strong potential of our product candidate for patients with ACHMB3, and we look forward to continuing our discussions with the regulatory agencies to determine the best path forward to bring this important therapy to patients, said Sue Washer, President and Chief Executive Officer of AGTC. We understand the challenges that patients with achromatopsia face in their daily lives and our team remains dedicated to advancing this program toward commercialization. These additional data provide us the opportunity to help inform the next phase of clinical development and we thank the patients and investigators for their participation in this important clinical trial.
In the Phase 1/2 dose escalation study of AGTC-401 in ACHMB3 patients, a total of 21 adults were treated over a 100-fold dose range in five groups and a total of ten pediatric patients were treated at the three highest dose groups. The primary purpose of any Phase1/2 clinical trial is to identify a well-tolerated dose that provides clinical benefit to patients. The Company believes that the data to date support that the 1.1E+12vg/mL dose is well tolerated and provides clinical benefit in both adult and pediatric patients.
Were pleased to have identified a generally safe and well tolerated dose of AGTC-401 for both adults and pediatric patients when inflammation is controlled, said Dr. Robert Sisk, MD, Director of Pediatric Vitreoretinal Surgery and Director of Ophthalmic Genetics Cincinnati Childrens Hospital and the Cincinnati Eye Institute and an investigator in the ongoing AGTC achromatopsia Phase 1/2 trials. Based on the available pediatric data at three months, which are consistent with previously reported results in adults with ACHMB3, we are encouraged by the biologic activity observed in patients treated with AGTC-401. The achromatopsia community currently faces a significant unmet need and the totality of this data reinforces the promise of AGTC-401 as a potential treatment option if confirmed in future studies.
As previously reported, in both the ACHMB3 and ACHMA3 trials, treatment with the highest doses of AGTC-401 and AGTC-402, respectively, led to three cases of severe ocular inflammation in pediatric patients, which were reported as SUSARs. No new additional SUSARs have been reported and the inflammation in all previously reported SUSARs improved with an adjusted steroid regimen. Two SUSARs (one in ACHMA3 and one in ACHMB3) have since fully resolved and one (ACHMA3) continues to resolve, with all three patients best corrected visual acuity returning to baseline.
Secondary outcome measures evaluating efficacy were assessed by standard visual function tests such as perimetry. We defined two pediatric patients (17 and 7 years old) in the 1.1E+12 vg/mL dose group as responders based on improvements in visual sensitivity. Therefore, of the three adults and four children (total n=7) in the 1.1E+12 vg/mL dose group, four (>50%) are visual sensitivity responders. These patients also had improvements in quality of life as measured by a patient reported outcome survey developed specifically for patients with achromatopsia.
The two other pediatric patients in the 1.1E+12 vg/mL dose group and three pediatric patients ages 7 years and younger in the 3E+12vg/mL dose group (total n=5) could not sufficiently concentrate and consistently complete the visual sensitivity testing. Similar to other trials where endpoints are adapted for young children, AGTC plans to work closely with clinicians and regulators to develop potential adaptations for younger patients for visual sensitivity testing.
The Company is currently working on an EOP2 meeting package for the FDA and expects to receive feedback in the first half of 2022. Subject to these discussions with FDA, the Company plans to initiate the next phase of AGTC-401 clinical development. The clinical program will be based on the favorable risk benefit profile of the 1.1E+12 vg/mL dose, which had a generally safe and well tolerated safety profile across all patients and signs of biologic response based on improvements in visual sensitivity and other measures of visual function.
Final data and analysis of both ACHMA3 and ACHMB3 studies will be presented at relevant conferences and published for the ophthalmology community.
Conference Call and Webcast
AGTC will host a conference call and webcast to review the updated and previously reported interim study results today at 8:00 a.m.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 AGTCs 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 Companys website.
About Achromatopsia (ACHM)
Achromatopsia (ACHM) is an inherited condition caused by mutations in one of several genes, with the two most common being mutations in either the CNGB3 or CNGA3 genes. ACHM is associated with extremely poor visual acuity (most affected individuals are legally blind), extreme light sensitivity resulting in daytime blindness, and complete loss of color discrimination. AGTC is currently developing two separate AAV gene therapy product candidates for the two most prevalent forms of ACHM, caused by either a genetic mutation in the CNGB3 or CNGA3 genes. Together, these two genetic mutations account for up to 75% of the ACHM patient population.
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 with the potential to address unmet patient needs. AGTCs most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with inherited retinal diseases. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and 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 needs in optogenetics, otology and CNS disorders, and has entered strategic collaborations with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding. For more information, please visit https://agtc.com/.
Forward-Looking Statements
This release contains forward-looking statements that reflect AGTCs plans, estimates, assumptions and beliefs, including statements about the potential of the Companys late-stage development program in Achromatopsia (ACHM), including the potential for the clinical development of AGTC-401 at the second highest dose, the continued favorable safety profile, the timing of any discussions with the FDA, and its ability to continue the clinical development of AGTC-401. 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; risks and uncertainties related to funding sources for our development programs; 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 our 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 managements 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.
PR Contact:
Kerry Sinclair
Spectrum Science Communications
ksinclair@spectrumscience.com
Corporate Contact:
Jonathan Lieber
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5778
jlieber@agtc.com
Exhibit 99.2
ACHM Phase 1/2 Clinical Trial: 3-month pediatric data analysis
Forward-Looking Statements This release contains forward-looking statements that reflect AGTCs plans, estimates, assumptions and beliefs, including statements about the potential of the Companys late-stage development program in Achromatopsia (ACHM), including the potential for the clinical development of AGTC-401 at the second highest dose, the continued favorable safety profile, the timing of any discussions with the FDA, and its ability to continue the clinical development of AGTC-401. 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; risks and uncertainties related to funding sources for our development programs; 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 our 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 managements 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.
Summary 3 ACHMB3 data continues to support advancement of clinical development Full data analysis follows ACHMA3 data does not support advancement of clinical development even in pediatric patients
Key Takeaways 4 Safety: Both ACHMA3 and ACHMB3 Adults: favorable safety profile through the highest dose (3.2E+12 vg/ml) in all patients across trials Peds: favorable safety profile through the 2nd highest dose (1.1E+12 vg/ml) Highest dose led to severe ocular inflammation in both trials with three previously reported SUSARs Inflammation has resolved in two of the SUSARS and is resolving in the third with BCVA back to baseline for all three Efficacy: ACHMB3 Adults: Consistent with previous reports there are responders in visual sensitivity, light discomfort, and Quality of Life Survey; especially in the 1.1E+12 vg/ml dose, Group 5 In responders, the improvement in visual sensitivity was robust and durable Pediatrics: In the relevant Group 5, two pediatric patients had robust improvements in visual sensitivity Consistent with adult data, improvements in BCVA and light discomfort may improve with time agtc
Achromatopsia Overview (ACHM) OVERVIEW Approximately 27,000 patients in US and EU affected AGTC focused on A3 and B3 gene mutations B3 accounts for approximately 50% or 14,000 patients A3 accounts for approximately 25% or 7,000 patients loss Severely of cone impaired photore vision ceptor and function day blindness due to No current treatments IMPACT Extremely poor vision, legally blind Extreme light sensitivity (day blind) Complete loss of color discrimination The bright light makes it really hard for him, so he tends to freeze up and doesnt want to walk anywhere because hes afraid he might injure himself He . gets more cautious in new or bright environments.
ACHMB3 Trial Overview: Dose Escalation and Age De-escalation DOSE LEVEL VG/ML Group 6 Group 6a 3.2E+12 PAUSE PAUSE N = 4 Pediatric: 3 Group 5 Group 5a 1.1E+12 PAUSE PAUSE N = 3 Pediatric: 4 Group 0 Group 3 Group 4 3.6E+11 PAUSE PAUSE N= 4 N= 4 Pediatric: 3 Group 2 1.2E+11 PAUSE N=4 Group 1 4.0E+10 N= 2 Total number of patients: 21 adults, 10 pediatrics PAUSE DSMC Review
ACHMB3 Baseline Patient Demographics N = 31 Male Female 15 16 48% 52% <18 yrs ≥18 yrs 10 21 32% 68% Baseline, mean (SD) Study Eye Fellow Eye BCVA 40.0 (8.4) 41.5 (8.5) Mean Sensitivity1 5.3 (5.2) 5.0 (3.6) Light Discomfort2 0.9 (0.7) 0.9 (0.7)
Safety Summary Across Both ACHMB3 and ACHMA3 8 Favorable vg/ml) and safety in pediatric profile patients in all adult through patients the through second highest the highest dose dose (1.1E+12 (3.2E+12 vg/ml) No dose limiting toxicities (DLTs) observed in adult patients at any dose DLTs only observed in the highest dose in pediatric patients 1 SUSAR in B3, 2 in A3 (all previously reported) Response to adjusted steroid regimen reported with intraocular inflammation resolved or significantly improving and BCVA returning to baseline Below the DLT dose level No endophthalmitis No drug-related SAEs reported in B3 or A3 No injection-related SAEs in B3; one reported in A3 (macular hole), resolved Similar safety profiles observed in pediatric and adult patients Most frequently reported ocular AE below the DLT dose level, related to study agent, was vitreal cells (3/7 peds vs 11/21 adults in B3; 2/6 peds vs 7/16 adults in A3), Grade 1-2 and non-serious agtc
Group 5a and 6a Pediatrics: Ability to Complete Assessments ACHMB3 (n=7) Test performed (Yes or No) Dose Subject Age BCVA Visual Sensitivity Light Discomfort Patient 27 7 Y Y Y Patient 28 5 Y N Y 1.1E+12 vg/ml Patient 29 5 Y N Y Patient 26 17 Y Y Y Patient 30 7 Y Y Y 3.2E+12 vg/ml Patient 31 5 Y N Y Patient 32 6 Y N Y
Efficacy Endpoint Discussion
ACHMB3 Overall Efficacy Data Summary for Top Doses Dose (vg/mL) Subject ID Age** BCVA Visual Sensitivity* Light Discomfort Quality of Life 3.6E+11; Group 4 Patient 11 21 R Patient 12 21 Patient 13 61 R Patient 14 57 Patient 18 17 0 Patient 21 16 R 3 Patient 24 12 R R 0 1.1E+12; Patient 16 53 R R 5 Group 5 Patient 17 39 R 0 Patient 19 27 R 3 Patient 26 17 R 3 Patient 27 7** R 1 Patient 28 5** Unable to complete test 3 Patient 29 5** R Unable to complete test 2 3.2E+12; Group 6 Patient 20 18 R 0 Patient 22 37 R 1 Patient 23 27 R R 3 Patient 25 52 1 Patient 30 7** 1 Patient 31 5 ** R NA 1 Patient 32# 6 ** NA 2 *As assessed by improvement in mean sensitivity across treated area of these 5 where also responders of at least 7 dcb at 4-5 loci. **Six pediatric patients with 3 month data, remainder of patients with 12 month data. # Only 31 patients were actually dosed, but one patient, number 5, was enrolled but not dosed.
ACHMB3 Overall Data Summary for Top Doses 12 Dose (vg/mL) Subject ID Age BCVA Visual Sensitivity* Light Discomfort Quality of Life 3.6E+11; group 4 Patient 11 21 R Patient 12 21 Dose (vg/mL) Subject ID Age BCVA Visual Sensitivity* Light Discomfort Quality of Life 1.1E+12; Patient 16 53 R R 5 Group 5 Patient 17 39 R 0 Patient 19 27 R 3 Patient 26 17 R 3 Patient 27 7 R 1 Patient 28 5 Unable to complete test 3 Patient 29 5 R Unable to complete test 2 Patient 25 52 1 Patient 30 7 1 Patient 31 5 R NA 1 Patient 32 6 NA 2 *As assessed by improvement in mean sensitivity across treated area of these 5 where also responders of at least 7 dcb at 4-5 loci.
Patient Anecdotes Reinforce 13 Response in Dose Group 5 Patient is loving soccer and their foot coordination has really taken off recently. The patient pointed out some boats far away in the water at the beach. The patient has been taking off their red Mom mentioned that they have been tinted glasses at dusk. 7yr old. practicing matching color cards and he has been able to identify the colors 50% more in his treated eye vs the untreated eye. Mom says he squints less outdoors now. 5yr old. Patient would love to get a No improvements yet. 27 yr old. second eye treated, went to a hockey game and from upper stadium noticed that he could see the player numbers on the jerseys. 17 yr old. No more tinted contacts and dark tinted sunglasses outside, I can see At three different visits the parents stated: boats in bright light on the water, I notices better coordination of stairs and can see bicycles coming, Its easier to curbs even in bright sunlight and in distinguish different colors in my unfamiliar places. 5yr old. closet. 53 yr old. Recent Patient Anecdotes as Reported by Principal Investigators; Anecdotes not indicative of entire patient population. agtc
VS: Mean Change Group 5 Responders Remains Compelling Patients 16, 19, 26, 27 Pediatric and Adult Dose (1e12vg/ml)CFB Mean Sensitivity Responders Based on RC (2.2) 10 8 6 Baseline Treated from 4 Untreated Threshold Change 2 0 n = 4 n = 4 n = 4 n = 4 n = 2 n = 3 n = 3 -2 Baseline Month Month Month Month 3 6 9 12 Patients 16, 26, 27* Pediatric and Adult Dose (1e12vg/ml)CFB Mean Sensitivity Responders Based on 7dB/5 loci 10 8 6 Baseline Treated from 4 Untreated Threshold Change 2 0 n = 3 n =3 n = 3 n = 3 n = 1 n = 2 n = 3 -2 Baseline Month Month Month Month 3 6 9 12 *also includes Patient 27, which responded at 7dB/4 loci Note: does not include responders from group 4 (n=1) and group 6 (n=1)
Group 5 Responders Patient 16, 19, 26 and 27
Static Perimetry Light Discomfort Assessment Line plot of mean within bleb loci sensitivity LDT2 20 3 2 10 1 0 10lux Log 0 -10 Treated Treated -1 Untreated Untreated Threshold -20 -2 Baseline Month 3 Month 6 Month 9 Month 12 Baseline Month 3 Month 6 Month 9 Month 12 Histogram plots of visual field change in loci sensitivity LDT1 3 20 Treated 2 15 Untreated Loci Threshold 1 of 10lux Number 10 Log 0 Treated 5 -1 Untreated Threshold -2 0 -7db -5db -3db -1db 1db 2db 3db 4db 5db 6db 7db Baseline Month 3 Month 6 Month 9 Month 12
Patient 16: Increase in VS and size of field 17 Baseline Month 3 Month 9 Month 12 EYE TREATED EYE UNTREATED
Patient 19: Visual Field Responders (RC) and QOL Survey QOL Survey: 3 of 8 questions improved from baseline Age 27 Dose Group 1.1E+12 Study Eye OS OD: 38 Baseline VA OS: 41 Static Perimetry Line plot of mean within bleb loci sensitivity 10 0 -10 Treated Untreated -20 Baseline Month 3 Month 6 Month 9 Month 12 Histogram plots of visual field change in loci sensitivity 20 Treated 15 Untreated Loci Threshold of Number 10 0 -7db -5db -3db -1db 1db 2db 3db 4db 5db 6db 7db 18 Light Discomfort Assessment LDT2 3 Treated 2 Untreated Threshold 10lux 1 Log 0 -1 -2 Baseline Month 3 Month 6 Month 9 Month 12 LDT1 3 Treated 2 Untreated Threshold 10lux 1 Log 0 -1 -2 Baseline Month 3 Month 6 Month 9 Month 12
Patient 19: Increase in VS Baseline Month 3 Month 6 Month 12 EYE TREATED EYE UNTREATED
Patient 26: Visual Field Responder Static Perimetry Light Discomfort Assessment Line plot of mean within bleb loci sensitivity LDT2 7dB/5loci, RC and 20 3 Treated 2 Untreated QOL Surve y 10 Threshold 1 0 10lux QOL Survey: 3 of 8 Log 0 questions improved -10 Treated -1 Untreated from baseline -20 -2 Baseline Month 3 Month 6 Month 9 Month 12 Baseline Month 3 Month 6 Month 9 Month 12 Histogram plots of visual field change in loci sensitivity LDT1 3 Age 17 Treated 20 Treated Untreated 2 Threshold Dose Group 1.1E+12 15 Untreated Loci Threshold 1 of 10lux Study Eye OD Number 10 Log 0 OD: 46 Baseline VA OS: 46 5 -1 -2 0 -7db -5db -3db -1db 1db 2db 3db 4db 5db 6db 7db Baseline Month 3 Month 6 Month 9 Month 12
Patient 26: Increase in VS and size of field 21 Baseline Month 3 Month 6 Month 12 EYE TREATED EYE UNTREATED
Patient 27: Visual Field Responder Static Perimetry Light Discomfort Assessment Line plot of mean within bleb loci sensitivity LDT2 7dcb/4 loci, RC, 20 3 Treated Untreated 2 and QOL Survey 10 Threshold 1 0 10lux QOL Survey: 1 of 8 Log 0 questions improved -10 Treated -1 Untreated from baseline -20 -2 Baseline Month 3 Month 6 Month 9 Month 12 Baseline Month 3 Month 6 Month 9 Month 12 Histogram plots of visual field change in loci sensitivity LDT1 3 Age 7 20 Treated Treated Untreated 2 Untreated T hreshold Dose Group 1.1E+12 Loci 15 Threshold 1 of 10lux Study Eye OD Numb er 10 Log 0 OD: 42 5 -1 Baseline VA OS: 40 -2 0 -7db -5db -3db -1db 1db 2db 3db 4db 5db 6db 7db Baseline Month 3 Month 6 Month 9 Month 12
Patient 27: Increase in VS and size of field Baseline Month 3 EYE TREATED Note: The kind of filter used d uring this testing procedure was not the same as used for all other patients resulting in the muc h darker colors. EYE UNTREATED
Conclusions This interim analysis provides additional support of a favorable risk/benefit profile A favorable safety profile was seen in adults through the highest dose (3.2E+12 vg/ml) in all patients and through the second highest dose in pediatrics (1.1E+12 vg/ml) Significant intraocular inflammation was seen at the highest dose in pediatrics that was controllable with steroids A higher response rate was seen in higher dose levels with robust improvements in visual sensitivity among responders Consistent with previous reports in ACHMB3 adults, we saw responders in visual sensitivity, light discomfort, and Quality of Life survey, especially in Group 5 (1.1E+12 vg/ml) In responders, improvement in visual sensitivity was robust and durable In the 1.1E+12 vg/ml dose group in ACHMB3 pediatrics, 2 patients had robust improvements in visual sensitivity, with very young patients having difficulty completing this testing Consistent with adult data, improvements in BCVA and light discomfort may improve with time Insights gained to be incorporated into next steps in clinical development
Question and Answer Period
Summary Post Q&A We and have shows identified improvements a dose, in 1 .visual 1 E+12 function vg/ml that across is generally several measures safe and well tolerated We expect feedback from the FDA in the 1H of 2022 We thank the patients, investigators and the entire AGTC team for their support
ACHM Phase 1/2 Clinical Trial: 3-month pediatric data analysis
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