Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Investor Presentation (January 2026)
29 slides from Opus Genetics's investor presentation, Jan 12, 2026
Slide 1: Delivering on the Promise of Gene Therapy for Rare Inherited Retinal Diseases
Delivering on the Promise ofGene Therapy for Rare Inherited Retinal Diseases January 2026 Nargiza,BEST1 patient
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: The Opus Opportunity: A String of Pearls Strategy
The Opus Opportunity: A String of Pearls Strategy LCA5 RHO RDH12 CNGB1 NMNAT1 MERTK BEST1 Portfolio approach produces multiple data readouts and milestones Follow-on treatments from the first approved IRD gene therapy 2 of 7 assets in clinical trials: upside across additional patient populations Cos
Slide 4: Precision-Targeted, One-Time-Treatment for Rare Diseases
4 Precision-Targeted, One-Time-Treatment for Rare Diseases 350+ genes known to cause IRDs Proven subretinal delivery with established safety profile and clinical precedent World-class science from pioneers in gene therapy Structure-function biology well-characterized with measurable outcomes amenabl
Slide 5: Structure-Function Dissociation: The Clinical Imperative
5 Treat the function to reverse pathology and restore or preserve vision Structure-Function Dissociation: The Clinical Imperative Targeting diseases where the structure is intact Retinal structure is relatively preservedeven though visual function is already impaired This creates a “therapeutic wind
Slide 6: Building a Differentiated Pipeline
Building a Differentiated Pipeline OPGx-LCA5 LCA co-funded by FDA OOPD OPGx-BEST1 Bestrophinopathies OPGx-RHO adRP co-funded by FFB & NIH OPGx-RDH12 LCA co-funded by Global RDH12 Alliance OPGx-MERTK RP co-funded by FFB RD Fund OPGx-NMNAT1 LCA OPGx-CNGB1 RP NIH-funded consortium Commercial Partnered
Slide 7: Actively Advancing Lead Indications
7 Actively Advancing Lead Indications OPGx-LCA5 Positive Phase 1/2 safety and efficacy results observed in adult and pediatric participants Successful Type B RMAT meeting Enrollment ongoing in run-in period for planned, adaptive Phase 3 trial Multiple Regulatory Designations: Rare Pediatric Disease
Slide 8: Lead Gene Therapy Programs Target Potential $15B U.S. Market Opportunity
8 Sources: FactSet. Data as of April 7, 2025. 2. Stone et al. Ophthalmology. 2017;124:1314-1331. Triangle Insights Group market research (compilation of prevalence studies), conducted August 2023. $400 Million LCA5 ~200 patients $5.6 Billion RHO ~5,600 patients $9 Billion BEST1 ~9,000 patients $15 B
Slide 9: OPGx-BEST1
OPGx-BEST1 Juan,BEST1 patient
Slide 10: BEST1 Mutations are Associated with Retinal Degeneration
BEST1 Mutations are Associated with Retinal Degeneration 1. Triangle Insights Group market research (compilation of prevalence studies), conducted August 2023. 2. Amato A, et al. Saudi J Ophthalmol. 2023;37(4):287-295. 3. Johnson AA, et al. Prog Retin Eye Res. 2017;58:45-69. 4. Tripathy K, et al. St
Slide 11: BEST1 Disease Biology
11 BEST1 Disease Biology BEST1 gene encodes for Bestrophin-1 (basolateral RPE) 585 AA homo-pentameric transmembrane protein (5 BEST1 monomers are required for full length BEST1 channel) Ca2+-activated Chloride channel (CaCC) Transepithelial ion transport of intracellular calcium RPE cell volume Home
Slide 12: BEST1 IRDs: Clinical Staging and Pathology of Two Main Phenotypes
12 BEST1 IRDs: Clinical Staging and Pathology of Two Main Phenotypes BVMD BVMD Prevalence: 1:60,000 Macular dystrophy similar to AMD with teen onset 20/60 and worse BCVA observed beginning at Stage 3 disease, leading to choroidal neovascularization, retinal detachment, chorioretinal atrophy BVMD, be
Slide 13: Proof of Concept of OPGx-BEST1 AAV2 in a Canine Model of ARB
13 Proof of Concept of OPGx-BEST1 AAV2 in a Canine Model of ARB BSS, balanced salt solution; ERG, electroretinogram; MERTK, MER proto-oncogene, tyrosine kinase; RPE, retinal pigment epithelium. Guziewicz, et al. PNAS. 2018;115:E2839–E2848. Robust restoration of RPE-photoceptor interface demonstrated
Slide 14: OPGx-BEST1 Phase 1/2 Trial
OPGX-BEST1 Phase 1/2 Trial Title: Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB) (BIRD-1) Type: Adaptive, open-label, dose-exploring, safety and tolerability study Population: adults
Slide 15: OPGx-LCA5
OPGx-LCA5 Alan,LCA5 patient
Slide 16: LCA5 is an Early-Onset, Severe Hereditary Retinal Degeneration
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 or non-detectable visual fields3,4 Visual acuity often limited to
Slide 17: OPGx-LCA5: Designed to Restore Structure and Function in Photoreceptors
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, photoreceptors can survive through the third decade of life, suggestive of a broad wind
Slide 18: OPGx-LCA5 Phase 1/2: Well-Tolerated with Activity on Multiple Outcome Measures
OPGx-LCA5 Phase 1/2: Well-Tolerated with Activity on Multiple Outcome Measures FST, full-field stimulus test; MLoMT, Multi-Luminance orientation and Mobility Test; SAE, serious adverse events; AE, adverse event. 18 Visual Acuity Observed improvement in visual acuity in 5 out of 6 participants Initia
Slide 19: Participant Demographics in Phase 1/2 Trial
Participant Demographics in Phase 1/2 Trial Deemed study eye: OD, right eye; OS, left eye. 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) R
Slide 20: Visual Acuity Maintained in Adults and Improved in Pediatric Cohort
Visual Acuity Maintained in Adults Over 18 Months and Improved Over 3 Months in Pediatric Cohort Improvement Improvement Mean Change from Baseline (CFB) in VA Adult Cohort (N=3) Mean Change from Baseline (CFB) in VA Pediatric Cohort (N=3) VA, visual acuity; BL, baseline; M, month. 20
Slide 21: Full-Field Stimulus Test Demonstrated Durable Vision Improvement
Full-Field Stimulus Test (FST) Demonstrated Durable Vision Improvement Mean CFB in Red FST – Adult Cohort (N=3) Mean CFB in Blue FST – Adult Cohort (N=3) Mean CFB in Red FST – Pediatric Cohort (N=3) Mean CFB in Blue FST – Pediatric Cohort(N=3) FST - Measures the cone or photoreceptor sensitivity in
Slide 22: Treated Eyes Identified More Objects on the Virtual Reality Mobility Test
Treated Eyes Identified More Objects on the Virtual Reality Mobility Test Mean Change from Baseline in MLoMT Adult Cohort (N=3) Mean Change from Baseline in MLoMT Pediatric Cohort (N=3) BL, baseline; CFB, change from baseline; M, month; MLoMT, Multi-Luminance orientation and Mobility Test; VA, visua
Slide 23: Microperimetry Data Provides Evidence of Increased Sensitivity
Microperimetry Data Provides Evidence of Increased Sensitivity and Movement of Fixation Toward the Fovea Adult Participant 01-04 Pediatric Participant 01-06 Microperimetry is a detailed, eye-tracking-assisted visual field test that creates a retinal sensitivity map of the macula by testing a patient
Slide 24: OPGx-LCA5 Program Positioned for Rapid Advancement
OPGx-LCA5 Program Positioned for Rapid Advancement 24 BLA, Biologics License Application; *Potential PRV opportunities contingent on timing of FDA approval and/or congressional reauthorization of PRV program Excellent safety data in all participants with follow-up out to 18 months in adult cohort Ro
Slide 25: Partnered Asset: Phentolamine Ophthalmic Solution 0.75%
Partnered Asset: Phentolamine Ophthalmic Solution 0.75%
Slide 26: Approved, sNDA Submitted and Phase 3 Indications for Phentolamine
1 APPROVED: Pharmacologically-induced mydriasis1 100M eye dilations conducted every year2 2 3 sNDA SUBMITTED: Presbyopia Progressive loss of ability to focus on close objects results in blurred near vision and eye strain 133M presbyopes3 Phase 3 ONGOING: Dim light disturbances in keratorefractive pa
Slide 27: Differentiated MOA of Phentolamine for Presbyopia and Low Light Vision
Differentiated MOA of Phentolamine is Designed to be Well-Suited for Presbyopia and Decreased Visual Acuity Under Low Light Conditions Favorable tolerability profile, with no reported incidence of retinal tears or retinal detachment, and minimal to no headaches or dimming Fast onset of action and ex
Slide 28: Leveraging an Efficient Platform for Clinical and Commercial Success
Leveraging an Efficient Platform for Clinical and Commercial Success ^Common shares outstanding as of November 10, 2025 28 Highlights Multi-asset pipeline with significant projected data readouts and milestones Two lead gene therapy programs in clinical trials with new data expected in 2026 First mo
Slide 29: “Impossible is a dare to science.”
“Impossible is a dare to science.” – Will Reeve, Good Morning America feature story on Opus LCA5 patient Nasdaq: IRD