Slide 1: OPGx-LCA5 Phase 1/2 Trial Results
OPGx-LCA5 Phase 1/2 Trial Results September 30, 2025 Alan,LCA5 patient Exhibit 99.2
Slide 2: Forward-looking statements
This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements concerning data from and future enrollment for our clinical trials and our pipeline of additional indications.
Slide 3: Management Team Call Participants
Management Team Call Participants George Magrath, MD, MBA, MS Chief Executive Officer Ben Yerxa, PhD President Ash Jayagopal, PhD, MBA Chief Scientific & Development Officer Sally Tucker, PhD Senior Vice President, Clinical Development 3 Rob Gagnon, CPA, MBA Chief Financial Officer
Slide 4: Introduction and Corporate Overview
4 Introduction andCorporate OverviewGeorge Magrath, MDChief Executive Officer 4
Slide 5: Leveraging an Efficient Platform for Clinical and Commercial Success
Leveraging an Efficient Platform for Clinical and Commercial Success Founded: 2018 HQ: Research Triangle Park, NC Focus: Developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders Company Snapshot No approved therapies
Slide 6: Efficient IRD Pipeline with Multiple Near-Term Value Inflection Points Anticipated
U.S. Prevalence Preclinical IND-enabling Phase 1/2 Phase 2/3 Rare Pediatric Orphan Drug Status OPGx-LCA5 LCA ~200 patients1,2 Granted 3-month pediatric & 18-month adult data in Q3 2025 Co-funded by FDA OOPD OPGx-BEST1 Bestrophinopathies ~9,000 patients1,2 Eligible Initiate clinical trial in Q4 2025
Slide 7: Inherited Retinal Diseases (IRDs) and OPGx-LCA5
7 7 Inherited Retinal Diseases (IRDs) and OPGx-LCA5
Slide 8: Limited Treatment Options Despite Key Advances in Gene Therapy
8 Over 350 genes are known to cause IRDs, which severely affect vision in more than 180,000 people in the United States1,2 Almost all IRDs lack treatment to halt progression and rescue vision2 Luxturna® is the only FDA-approved IRD gene therapy and targets the RPE65 gene mutation2 Luxturna® is a reg
Slide 9: LCA5 is an Early-Onset, Severe Hereditary Retinal Degeneration
LCA5 is an Early-Onset, Severe Hereditary Retinal Degeneration 9 Prevalence ~200 patients in the U.S.1,2 LCA5 represents ~2% of all LCA cases3 Clinical Characteristics Presentation in 1st year of life with nystagmus and vision loss3,4 Severe and early photoreceptor loss results in severely abnormal
Slide 10: OPGx-LCA5: Designed to Restore Structure and Function in Photoreceptors
OPGx-LCA5: Designed to Restore Structure and Function in Photoreceptors 10 Lebercilin is a ciliary protein critical for the function of photoreceptor inner and outer segments1 In LCA5 patients, photoreceptor function is severely impaired due to a lack of functioning lebercilin1 However, photorecepto
Slide 11: Secondary Endpoints for the OPGx-LCA5 Phase 1/2 Trial
Secondary Endpoints for the OPGx-LCA5 Phase 1/2 Trial Visual Acuity Measured by Logarithm of the Minimum Angle of Resolution (LogMAR) Used to assess visual acuity in a standardized fashion Equal distances on the scale represent multiplicative changes rather than additive ones FST Full-field Stimulus
Slide 12: OPGx-LCA5 Phase 1/2 Study Data
12 12 OPGx-LCA5 Phase 1/2 Study DataSally Tucker, PhDSenior Vice President, Clinical Development
Slide 13: Participant Demographics
Deemed study eye: OD, right eye; OS, left eye. Participant Demographics 13 Adult Participants Pediatric Participants Participant # 01-01 01-03 01-04 01-05 01-06 01-07 Age 34 26 19 17 16 17 Gender Female Male Female Female Male Female Study eye treated Left (OS) Left (OS) Right (OD) Right (OD) Right
Slide 14: OPGx-LCA5 Well-Tolerated in All 6 Treated Participants
Safety evaluated in 3 adult participants at 18 months and 3 pediatric participants at 3 months No ocular serious adverse events No observed dose-limiting toxicities All ocular AEs were mild, anticipated, and unrelated to study drug One adverse event associated with surgical procedure Pediatric parti
Slide 15: Visual Acuity
15 Visual Acuity 15
Slide 16: Visual Acuity Maintained Over Months in Adult Cohort (N=3)
Visual Acuity Maintained Over 18 Months in Adult Cohort (N=3) BL, baseline; CFB, change from baseline; IOD, interocular difference; logMAR, logarithm of the minimum angle of resolution; VA, visual acuity. Improvement Study Eyes Better Mean Change from Baseline in Visual Acuity Mean Interocular Diffe
Slide 17: Visual Acuity Improved Over Months in Pediatric Cohort (N=3)
Visual Acuity Improved Over 3 Months in Pediatric Cohort (N=3) Improvement BL, baseline; CFB, change from baseline; IOD, interocular difference; logMAR, logarithm of the minimum angle of resolution; VA, visual acuity. Study Eyes Better Mean Change from Baseline in Visual Acuity Mean Interocular Diff
Slide 18: Observed Change from Baseline in Visual Acuity (Pediatric Cohort)
BL, baseline; CFB, change from baseline; logMAR, logarithm of the Minimum Angle of Resolution; VA, visual acuity. Observed Change from Baseline in Visual Acuity (Pediatric Cohort) Participant 01-05 Participant 01-06 Participant 01-07 18
Slide 19: FST, MLoMT and Microperimetry
19 Full-field Stimulus Test (FST) Multi-Luminance Orientation and Mobility Test (MLoMT)Microperimetry 19
Slide 20: FST: Observed Vision Improvement Durable to 18 Months in Adult Cohort
BL, baseline; CFB, change from baseline; FST, full-field stimulus test. FST: Observed Vision Improvement Durable to 18 Months in Adult Cohort Participant 01-03 Participant 01-01 Participant 01-04 FST CFB – Red FST CFB – Blue 20 NOTE: Red FST values unavailable for the untreated eye of Participant 01
Slide 21: FST: Vision Improvement Observed to 3 Months in Pediatric Cohort
Participant 01-06 Participant 01-05 Participant 01-07 BL, baseline; CFB, change from baseline; FST, full-field stimulus test. FST: Vision Improvement Observed to 3 Months in Pediatric Cohort FST CFB – Red FST CFB – Blue 21
Slide 22: MLoMT: All Adult Participants Identified More Objects in the Study Eye
MLoMT: All Adult Participants Identified More Objects in the Study Eye Through 18 Months Compared to Baseline Participant 01-03 Participant 01-01 Participant 01-04 Control Eyes – Untreated 22 Objects Identified (count) Brightest Luminance (log phot cd. m-2) Objects Identified (count) Dimmest Normal-
Slide 23: MLoMT: All Pediatric Participants Identified More Objects in the Study Eye
MLoMT: All Pediatric Participants Identified More Objects in the Study Eye Through 3 Months Compared to Baseline Participant 01-06 Participant 01-05 Participant 01-07 Control Eyes – Untreated 23 Study Eyes - Treated MLoMT, Multi-Luminance orientation and Mobility Test. Objects Identified (count) Nor
Slide 24: Microperimetry Data Provides Evidence of Increased Sensitivity
24 Microperimetry Data Provides Evidence of Increased Sensitivity and Movement of Fixation Toward the Fovea Adult Participant 01-04 Pediatric Participant 01-06
Slide 25: Phase 1/2 Clinical Data Highlights in Pediatric Cohort (N=3)
25 Phase 1/2 Clinical Data Highlights in Pediatric Cohort (N=3) Visual Acuity: Observed improvement with initial gains as early as 1 month FST: Observed improvement with increased sensitivity in treated eyes and measurable photoreceptor function MLoMT: Observed improvement from baseline out to 3 mon
Slide 26: Phase 1/2 Clinical Data Highlights from 6 Treated Participants
Significant improvement observed in participants with formative vision at baseline Observed improvement in 2 adult participants out to 18 months Phase 1/2 Clinical Data Highlights from 6 Treated Participants 26 Observed improvement in visual acuity in 5 out of 6 participants Initial gains as early a
Slide 27: Summary and Next Steps
27 Summary and Next StepsGeorge Magrath, MDChief Executive Officer 27
Slide 28: Summary of positive results and next steps
Positive results observed in both adult and pediatric participants Well-tolerated in all participants with follow-up out to 18 months in adult cohort Robust biologic activity corroborated through multiple functional outcomes: Visual Acuity and Full-field Stimulus Testing improvements suggest potent
Slide 29: Powerful Participant-Reported Outcomes After OPGx-LCA5 Treatment
29 Powerful Participant-Reported Outcomes After OPGx-LCA5 Treatment Adult Participants 01-01: Reported being able to identify her children within a larger group of children 1 month after surgery which she previously could not have done. 01-03: Had no formative vision prior to treatment. Reported bei
Slide 30: Opus Genetics Nasdaq: IRD
Opus GeneticsNasdaq: IRD 30