Opus Genetics updates BEST1 trial data, cash into 2029

Opus Genetics, Inc. (IRD) furnished a revised investor data presentation correcting an immaterial error in converting logMAR visual acuity to ETDRS letters for its Phase 1/2 OPGx-BEST1 Cohort 1 data.

Rhea-AI Filing Summary

Opus Genetics, Inc. (IRD) furnished a revised investor data presentation correcting an immaterial error in converting logMAR visual acuity to ETDRS letters for its Phase 1/2 OPGx-BEST1 Cohort 1 data. The corrected 3‑month letter changes per participant now range from a 12‑letter gain to a 10‑letter loss across treated eyes and from a 4‑letter gain to a 8‑letter loss in fellow eyes, and high‑resolution microperimetry images were added to the appendix.

The presentation highlights that in the low‑dose Cohort 1 (5 participants) OPGx‑BEST1 was well‑tolerated with no serious adverse events, no dose‑limiting toxicities, and no intraocular inflammation. Structural improvements were seen in 80% of participants, and all participants improved in at least one functional measure, with 75% of evaluable participants reaching the FDA‑aligned microperimetry threshold of ≥3 dB improvement in ≥5 loci. Cohort 2 (8 high‑dose participants) dosing is expected to complete in Q4 2026, with 3‑month topline data targeted for Q2 2027 and a pivotal trial planned to start dosing in 2027.

Opus reports a current cash runway extending into 2029, supporting OPGx‑BEST1 and four additional gene therapy programs through multiple anticipated clinical data readouts and milestones.

Positive

• OPGx-BEST1 low-dose Cohort 1 showed a clean safety profile, with no serious adverse events, no dose-limiting toxicities, and no intraocular inflammation across 5 treated participants.
• Early efficacy signals are encouraging: 80% of participants had structural improvements, 100% improved in at least one functional measure, and 75% of evaluable participants met the FDA-aligned microperimetry threshold.
• Opus Genetics reports cash runway extending into 2029, supporting BEST1 and four additional retinal gene therapy programs through several planned clinical data readouts and pivotal-start milestones.

Negative

• None.

8-K Event Classification

2 items: 7.01, 9.01

2 items

Item 7.01 Regulation FD Disclosure Disclosure

Material non-public information disclosed under Regulation Fair Disclosure, often investor presentations or guidance.

Item 9.01 Financial Statements and Exhibits Exhibits

Financial statements, pro forma financial information, or exhibit attachments filed with this report.

Key Figures

Cohort 1 size: 5 participants Safety outcomes: 0 serious adverse events and 0 dose-limiting toxicities Structural improvement rate: 80% of participants +5 more

8 metrics

Cohort 1 size 5 participants Low-dose OPGx-BEST1 Phase 1/2 Cohort 1 at 1.5×10^9 vg/eye

Safety outcomes 0 serious adverse events and 0 dose-limiting toxicities Reported across all 5 low-dose OPGx-BEST1 Cohort 1 participants

Structural improvement rate 80% of participants 4 of 5 low-dose Cohort 1 participants had structural retinal improvements

Functional response 100% of participants All 5 Cohort 1 participants improved in at least one functional endpoint

Microperimetry success rate 75% of evaluable participants 3 of 4 evaluable Cohort 1 participants met ≥3 dB improvement in ≥5 loci

High-dose Cohort 2 size 8 participants Over-enrolled high-dose OPGx-BEST1 Cohort 2 at 4.5×10^9 vg/eye

U.S. symptomatic BEST1 prevalence 23,600 patients Estimated number of symptomatic BEST1 patients in the United States

Global BEST1 prevalence 45,400 patients Estimated symptomatic BEST1 patients across specified global regions

Key Terms

microperimetry, vitelliform lesion, intraretinal fluid, subretinal injection, +2 more

6 terms

microperimetry medical

"Measures pointwise sensitivity of the retina over the lesion"

Microperimetry is a clinical eye test that maps how well specific spots on the retina detect light while tracking their exact location on an image of the back of the eye. For investors, it matters because it provides precise, location-specific evidence of vision improvement or decline in drug and device trials, affecting regulatory decisions, market potential and reimbursement—think of it as a high-resolution measuring stick for retinal function.

vitelliform lesion medical

"BVMD is characterized by vitelliform (“egg-yolk”) lesion beneath the macula"

intraretinal fluid medical

"ARB participants had a decrease in intraretinal fluid on OCT"

Intraretinal fluid is fluid that collects inside the layers of the retina, the light‑sensitive tissue at the back of the eye, caused by leaking blood vessels or inflammation. It is a direct sign of worsening vision conditions and is routinely measured in clinical exams and trials; like water trapped between wallpaper layers, its presence and change over time can determine whether a treatment is seen as effective and therefore drive regulatory decisions, adoption and commercial value for therapies and devices.

subretinal injection medical

"Adaptive, open-label, dose-exploration study of subretinal injection of OPGx-BEST1"

A subretinal injection is a medical procedure that places a drug, gene therapy, or cells directly into the thin space beneath the retina at the back of the eye, delivering treatment precisely where damaged vision cells live. For investors, it matters because this targeted approach can increase effectiveness but also raises surgical complexity, regulatory hurdles, costs and safety considerations that affect clinical success, commercialization timeline and market adoption—think of burying fertilizer under a lawn rather than sprinkling it on top.

PDUFA regulatory

"Oct 2026 PDUFA date for Phentolamine sNDA"

PDUFA is the Prescription Drug User Fee Act, the U.S. law under which drug companies pay fees that fund the FDA's review of new medicines. In company news the term usually appears as the PDUFA date, the target deadline by which the FDA aims to decide on a drug application; that date tells investors when to expect the approval or rejection decision for the product.

Priority Review Voucher regulatory

"All gene therapy programs have the potential to qualify for a Priority Review Voucher"

A priority review voucher is a transferable regulatory incentive that lets a company move a future drug or device application to the front of the review line, shortening the review period by several months. For investors it matters because the voucher can speed up market access for a high-value product or be sold to other companies for significant cash, acting like a tradable fast-pass that can accelerate revenue or create immediate financial upside.

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did Opus Genetics (IRD) change in its September 11, 2026 OPGx-BEST1 data presentation?

Opus Genetics corrected an immaterial error in translating logMAR visual acuity into ETDRS letters, affecting the mean best-corrected visual acuity depiction through 3 months for Cohort 1. It also added high-resolution baseline and 3-month microperimetry grid images to the appendix.

What safety results did IRD report for the OPGx-BEST1 low-dose Cohort 1?

In 5 low-dose OPGx-BEST1 participants, Opus reported the therapy was well-tolerated with no serious adverse events, no dose-limiting toxicities, no intraocular inflammation, only mild or moderate ocular adverse events, and no notable systemic safety or lab issues.

What efficacy signals were seen in Opus Genetics’ OPGx-BEST1 Cohort 1?

Opus reported structural improvements in 80% of participants and that all 5 participants improved in at least one functional measure. Among evaluable eyes, 75% met the FDA-aligned microperimetry success threshold of ≥3 dB improvement in ≥5 prespecified loci in the treated transitional zone.

How many participants are in the OPGx-BEST1 Phase 1/2 cohorts and what are the doses?

Low-dose Cohort 1 includes 5 participants at 1.5×10^9 vg/eye. High-dose Cohort 2 is over-enrolled with 8 participants at 4.5×10^9 vg/eye, with dosing expected to complete in Q4 2026 and 3-month topline data targeted for Q2 2027.

What is the estimated BEST1 patient population relevant to IRD’s program?

A new epidemiology report cited by Opus estimates about 23,600 symptomatic BEST1 patients in the U.S. and a global prevalence of roughly 45,400 patients across the U.S., EU4, UK, Middle East/North Africa, and China.

What financial runway did Opus Genetics (IRD) disclose in this update?

Opus stated that its current cash runway extends into 2029, funding five clinical programs, including BEST1, LCA5, RDH12, and MERTK, through multiple expected clinical inflection points and a planned BEST1 pivotal trial start in 2027.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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Available on EDGAR 09/11/2026 - 07:48 AM Accepted by SEC EDGAR 09/11/2026 - 07:48 AM Learn about SEC filing dates

false000122862700012286272026-09-112026-09-11

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): September 11, 2026

Opus Genetics, Inc.

(Exact name of registrant as specified in its charter)

Delaware	001-34079	11-3516358
(State or other jurisdiction of incorporation)	(Commission File Number)	(IRS Employer Identification No.)

8 Davis Drive Durham, NC		27713
(Address of principal executive offices)		(Zip Code)

(984) 884-6030

(Registrant’s telephone number, including area code)

N/A

(Former name or former address, if changed since last report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

☐
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common Stock, $0.0001 par value per share	IRD	The Nasdaq Stock Market LLC

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter). Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Item 7.01 Regulation FD Disclosure.

On September 11, 2026, Opus Genetics, Inc. (the “Company”) issued a revised version of the Company’s data presentation, which was initially used in connection with its investor conference on September 9, 2026, announcing clinical data from Cohort 1 of the Company’s ongoing Phase 1/2 clinical trial of OPGx-BEST1 in patients with BEST1-related retinal diseases. The original data presentation was furnished by the Company on a Current Report on Form 8-K on September 9, 2026.

The revised version of the data presentation, which is furnished as Exhibit 99.1 to this Current Report on Form 8-K, corrects an immaterial error in the translation of logMAR visual acuity into ETDRS letters, which impacted the depiction of mean best-corrected visual acuity from baseline through 3 months in Cohort 1 on Slide 15. The correct three month change from baseline for each participant is as follows:  101-101 had a 12 letter improvement in the treated eye and a 4 letter improvement in fellow eye, 101-104 had a 1 letter improvement in the treated eye and a 7 letter loss in the fellow eye, 102-101 had a 10 letter loss in the treated eye and an 8 letter loss in the fellow eye, 102-102 had a 2 letter gain in the treated eye and a 5 letter loss in the fellow eye, and 101-106 had a 6 letter gain in the treated eye and a 2 letter gain in the fellow eye. In addition to the update on Slide 15, high-resolution images of the microperimetry grids for baseline and 3 months were added to the Appendix of the revised presentation for each participant. Individual loci data is available for the three participants in these images.  

Item 9.01 Financial Statements and Exhibits.

(d) Exhibits

Exhibit No.		Description
99.1		Data presentation issued by Opus Genetics, Inc. on September 11, 2026, furnished herewith
104		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 hereunto duly authorized.

			OPUS GENETICS, INC.
Date:	September 11, 2026	By:	/s/ Dr. George Magrath
			Dr. George Magrath
			Chief Executive Officer

Exhibit 99.1

 OPGx-BEST1 Gene Therapy Phase 1/2 Study Low Dose Cohort 1 3-month Results  September 9, 2026 

 This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “aim,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. Such statements include, but are not limited to, statements related to our continued clinical development, clinical results, preclinical data, and future plans for OPGx-BEST1, including the anticipated timing of dosing completion and topline data from Cohort 2 of the OPGx-BEST1 Phase 1/2 clinical trial; the outcome of our ongoing regulatory interactions with the U.S. Food and Drug Administration (the “FDA”) and our expectations regarding the design of, and potential endpoints for, of any pivotal clinical trial of OPGx-BEST1; our expectations regarding the clinical and therapeutic potential of OPGx-BEST1, including with respect to its ability to improve visual function and retinal structure in patients with BEST1-related retinal disease; our estimates of the BEST1 symptomatic patient population in the U.S. and globally; and our expectations regarding our company, its business prospects, and our results of operations. These forward-looking statements are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to: our clinical data related to gene therapies for the treatment of inherited retinal diseases is preliminary and related to a relatively small group of patients, and, as a result, data that initially appears promising may be revised, updated, or invalidated at a later data readout and/or may ultimately not be capable of duplication in additional patients; our gene therapy product candidates are based on a novel technology that is difficult to develop and manufacture, which may result in delays and difficulties in obtaining regulatory approval; our planned clinical trials may face substantial delays, result in failure, or provide inconclusive or adverse results that may not satisfy the FDA requirements to further develop our therapeutic products; delays or difficulties associated with patient enrollment in clinical trials may affect our ability to conduct and complete those clinical trials and obtain necessary regulatory approvals; changes in regulatory requirements could result in increased costs or delays in development timelines; we depend heavily on the success of our product pipeline; if we fail to find strategic partners or fail to adequately develop or commercialize our pipeline products, our business will be materially harmed; we have not generated significant revenue from sales of any products and expect to incur losses for the foreseeable future; our future viability is difficult to assess due to our short operating history and our future need for substantial additional capital, access to which could be limited by any adverse developments that affect the financial services markets; we rely on third parties for material aspects of our business, such as conducting our nonclinical and clinical trials and supplying and manufacturing bulk drug substances, which exposes us to certain risks; and those risks and uncertainties described under the heading “Risk Factors” included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025 and our subsequent filings with the U.S. Securities and Exchange Commission (the “SEC”). Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this presentation. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise. These forward-looking statements are based upon our current expectations and involve assumptions that may never materialize or may prove to be incorrect.  Disclosures and Forward-Looking Statements  2 

 3  Management Team and KOL Participant  Ash Jayagopal, PhD, MBA  Chief Scientific & Development Officer  Sally Tucker, MCOptom, PhD  Chief Medical Officer  Rob Gagnon, CPA, MBA  Chief Financial Officer  Mark E. Pennesi, MD, PhD, FARVO Retina Foundation of the Southwest Dallas, TX  George Magrath, MD  Chief Executive Officer  Ben Yerxa, PhD  President 

 Well-tolerated with no SAEs, no DLTs, and no intraocular inflammation  Structural and functional improvements at 3 months  FDA meeting aligned on potential pivotal endpoint  ≥3 dB microperimetry improvement in ≥5 prespecified loci  In conjunction with a RCT using the PGI-S  Phase 3 and commercial manufacturing on schedule for delivery in early 2027  Cohort 2 overenrolled at 8 participants, weighted towards earlier stage BVMD  6-month Low-Dose Cohort 1 and 3-month High-Dose Cohort 2 data expected in Q2 2027  Pivotal trial to commence dosing in 2027  New epidemiology report surveying 150 eye care professionals estimates 23,600 BEST1 patients in the U.S.  4  OPGx-BEST1 Program and Low-Dose Cohort 1 Clinical Data Highlights   BEST1, bestrophin 1; BVMD, best vitelliform macular dystrophy; dB, decibel; DLT, dose-limiting toxicity; FDA, Food and Drug Administration; PGI-S, patient global impression-severity; RCT, randomized controlled trial; SAE, serious adverse event.  OPGx-BEST1 Cohort 1 & Program Summary  T O D AY ’ S T O P I C S  01   O p u s & B E S T 1 O v e r v i e w  0 2  T r i a l D e s i g n & To p l i n e R e s u l t s  03   P a r t i c i p a n t C a s e S t u d i e s  04   P r o g r a m S u m m a r y & N e x t S t e p s  75% of evaluable participants met the FDA-aligned microperimetry of ≥3 dB improvement in ≥5 prespecified loci 

 Building a Differentiated Gene Therapy Platform  Opus Genetics owns worldwide rights to all gene therapy programs.  adRP, autosomal dominant retinitis pigmentosa; BEST1, bestrophin 1; CNGB1, cyclic nucleotide-gated channel β1; FDA OOPD, Food and Drug Administration Office of Orphan Products Development; FFB, Foundation Fighting Blindness; GTx, gene therapy; LCA5, Leber congenital amaurosis 5; NIH, National Institutes of Health; RD, retinal degeneration; RDH12, retinol dehydrogenase 12; RHO, rhodopsin; RP, retinitis pigmentosa; MERTK, MER proto-oncogene tyrosine kinase; NMNAT1, nicotinamide mononucleotide adenylyltransferase.  5  OPGx-LCA5 LCA  co-funded by FDA OOPD  OPGx-BEST1  Bestrophinopathies  OPGx-RDH12 LCA  co-funded by Global RDH12 Alliance  OPGx-MERTK RP  co-funded by FFB RD Fund & Abu Dhabi’s  Healthcare Research and Innovation Fund  OPGx-RHO adRP  co-funded by FFB & NIH  OPGx-NMNAT1 LCA  OPGx-CNGB1 RP  NIH-funded consortium  Undisclosed IRD GTx  Preclinical IND-enabling Phase 1/2  Phase 3  Approval  All gene therapy programs have the potential to qualify for a Priority Review Voucher 

 6  BEST1: Group of Inherited Retinal Diseases with a Range of Onset and Slow Rate of Progression and no Approved Treatment Options  Overview & Prevalence  Mutations in BEST1 have been associated with at least five clinically distinct retinal degenerative diseases, with onset from childhood to adulthood1  Accounts for ~3.5% of all IRDs1  Global prevalence*: ~45,400 patients2  U.S. prevalence: 23,600 patients (~23,200 BVMD and ~400 ARB)2  Clinical Features1,3  Serous retinal detachment  BVMD (most common) is characterized by vitelliform (“egg-yolk”)  lesion beneath the macula1  Macular atrophy  CNV  Symptoms3,4  Loss of central vision  Metamorphopsia (distorted vision)  Scotoma (blind spot)  Photophobia4  *Global prevalence estimate includes United States, EU4 (France, Spain, Germany, & Italy), UK, Middle East/North Africa, and China.  ARB, autosomal recessive bestrophinopathy; BEST1, bestrophin 1; BVMD, best vitelliform macular dystrophy; CNV, choroidal neovascularization; IRD, inherited retinal disease.  1. Amato A, et al. Saudi J Ophthalmol. 2023;37(4):287-295. 2. BEST1 Market Landscape Quantitative Market Research, Triangle Insights Group, Q3 2026. 3. Johnson AA, et al. Prog Retin Eye Res. 2017;58:45-69.  4. Tripathy K, et al. StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024.  BVMD  ARB  Most Common BEST1 Phenotypes  Prevalence  BVMD 23,200 (98%)2  ARB 400 (2%)2 

 BEST1 gene encodes for bestrophin-1, a homopentameric (i.e., 5 identical monomers) Ca2+-activated chloride channel required for RPE maintenance and retinal physiology  BEST1 mutations in BVMD disrupt cellular ion and fluid homeostasis resulting in electrophysiological abnormalities, RPE dysfunction, and retinal degeneration via:  Defective clearance of toxic waste products (e.g., lipid deposits)  Impaired RPE-photoreceptor interactions (e.g., defective  phagocytosis)  Build-up of vitelliform material (toxic waste products) and fluid under the retina  BEST1 Disease Biology  BEST1, bestrophin 1; BVMD, best vitelliform macular dystrophy; RPE, retinal pigment epithelium. Guziewicz KE, et al. Prog Retin Eye Res. 2017;58:70-88.  A T R O P H Y  F R A G M E N T E D  V I T E L L I F O R M MA T E R I A L  S U B R E T I N A L F L U I D  P S E U D O H Y P O P Y O N  Photoreceptors  Retinal Pigment Epithelium  Bruch’s Membrane   Choriocapillaris  V I T E L L I F O R M L E S I O N  V I T E L L I F O R M D E P O S I T S  STAGE 1  Pre-vitelliform  STAGE 2  Vitelliform  STAGE 3  Pseudohypopyon  STAGE 4  Vitelliruptive  STAGE 5  Atrophy/Fibrosis  S t a g e s o f B V M D  7  Cohort 1 Participants  Vitelliform material occurs early and is the hallmark of BVMD  Subretinal fluid occurs late in disease and pools in  atrophic areas 

 8  Trial Design &  Topline Cohort 1 Results  Sally Tucker, MCOptom, PhD Chief Medical Officer 

 OPGx-BEST1: Phase 1/2 Study Overview (BIRD-1)  D e s i g n  Adaptive, open-label, dose-exploration, safety and tolerability study of subretinal injection of OPGx-BEST1 in adult participants with BVMD or ARB  D o s i n g C o h o r t s  Cohort 1: 1.5x109 vg/eye  Cohort 2: 4.5x109 vg/eye  O b j e c t i v e s  Primary: Safety and tolerability; identify appropriate dose for Phase 3  Secondary: Efficacy  S t u d y  P o p u l a t i o n  Minimum of 5 participants at each dose level  S t a t u s o f C o h o r t 1  ( l o w d o s e )  5 participants dosed:  ARB (n=2): Data at 6 months  BVMD (n=3): Data at 3 months  S t a t u s o f C o h o r t 2  ( h i g h d o s e )  Enrollment complete with 8 participants (7 surgeries scheduled)  Dosing expected to be completed in Q4 2026  ARB, autosomal recessive bestrophinopathy; BVMD, best vitelliform macular dystrophy.  9 

 *ETDRS letters equivalent calculated from logMAR.  † Worse eye deemed study eye.  ARB, autosomal recessive bestrophinopathy; BEST, bestrophin; BVMD, best vitelliform macular dystrophy; ETDRS, Early Treatment Diabetic Retinopathy Study; RPE, retinal pigment epithelium;  VA, visual acuity.  Participant Demographics: Low-Dose Cohort 1  101-101  101-104  102-101  102-102  101-106  Age  63  59  50  45  31  Sex  Female  Female  Male  Male  Male  BEST phenotype  ARB  ARB  BVMD  BVMD  BVMD  Baseline VA*  (study eye†)  1  50  49  61  47  Baseline VA*  (fellow eye)  43  65  68  72  56  Follow-up duration  6 months  6 months  3 months  3 months  3 months  Severity of disease  End-stage with  significant atrophy  End-stage with  significant atrophy  End-stage with sub-foveal, sub-RPE scar  Advanced stage vitello-eruptive with some scarring  Earlier stage without significant atrophy or scarring  10 

 Study Endpoints  M i c r o p e r i m e t r y  Measures pointwise sensitivity of the retina over the lesion  Recent natural history data shows a steady decline over 5 years in  BVMD, with most of the decline in the area on the edge of the lesion1  B C VA & L LVA  Methods to measure central fine visual function  C o n t r a s t S e n s i t i v i t y  Measures visual function and is sensitive in patients with central atrophy (used extensively in geographic atrophy studies)  A u t o f l u o r e s c e n c e  A 2-dimensional picture of the retina highlighting vitelliform lesions  O C T  A cross-sectional view of the retina  BCVA, best-corrected visual acuity; BVMD, best vitelliform macular dystrophy; LLVA, low-luminance visual acuity; OCT, optical coherence tomography.  1. Bianco L, et al. Invest Ophthalmol Vis Sci. 2026;67(11):1.  11 

 Safety and Efficacy Summary:  Highly Encouraging Proof-of-Concept Results from Low-Dose Cohort 1  S a f e t y  OPGx-BEST1 was well-tolerated in 5/5 participants, with no SAEs or DLTs  S t r u c t u r a l  E n d p o i n t s  80% of participants (4/5) had structural improvements:  BVMD: 67% of participants (2/3) had a decrease in vitelliform material  ARB: 100% of participants (2/2) had a decrease in intraretinal fluid  F u n c t i o n a l E n d p o i n t s  100% of participants (5/5) had improvements* in at least one functional measure:  Microperimetry – 75% of participants (3/4) improved  BCVA – 60% of participants (3/5) improved  LLVA – 40% of participants (2/5) improved  Contrast Sensitivity – 40% of participants (2/5) improved  Earlier-stage participant (101-106)† showed the biggest functional gains,  suggesting a potential benefit from earlier treatment  *Improvements defined as: ≥5 letters of improvement from baseline and ≥5 letter improvement from fellow eye in BCVA or LLVA, 0.2 logMAR improvement from baseline in contrast sensitivity, and ≥5 loci cluster improvement by ≥3 decibels in RPE Transitional Zone on microperimetry.  †101-106 was the youngest participant with most recent onset of disease and less progressive disease than other participants.  ARB, autosomal recessive bestrophinopathy; BCVA, best-corrected visual acuity; BVMD, best vitelliform macular dystrophy; DLT, dose-limiting toxicity; LLVA, low-luminance visual acuity; logMAR, logarithm of the minimum angle of resolution; RPE, retinal pigment epithelium; SAE, serious adverse event.  12 

 Well-tolerated in 100% of participants  No intraocular inflammation  No serious adverse events  No dose-limiting toxicities  No treatment-related systemic AEs  All ocular treatment-related AEs were mild/moderate in severity  No vital sign issues or safety lab findings of note  OPGx-BEST1 Demonstrated a Favorable Safety Profile  Independent Data Monitoring Committee recommended the Phase 1/2 Trial advance to Cohort 2 at higher dose  No intraocular inflammation was observed in any patient at any study visit  *Based on the SUN Working Group Grading Scheme.  AE, adverse event; ARB, autosomal recessive bestrophinopathy; BVMD, best vitelliform macular dystrophy; .  101-101 101-104  (ARB) (ARB)  102-101  (BVMD)  101-106  (BVMD)  0  102-102  (BVMD)  1+ 2+  (cells in field)*  3+ 4+  13 

 Participant-Level Improvement by Endpoint*  Endpoint  101-101  ARB  101-104  ARB  102-101  BVMD  102-102  BVMD  101-106  BVMD  BCVA  ≥5 letter improvement compared to baseline and fellow eye  ✓  ✓  ✓  LLVA  ≥5 letter improvement compared to  baseline and fellow eye  ✓  ✓  Contrast Sensitivity  ≥0.2 logMAR improvement from baseline  ✓  ✓  Microperimetry  ≥5 loci cluster improvement ≥3 dB  NE  ✓  ✓  ✓  OCT  IRF / vitelliform material reduced  ✓  ✓  ✓  ✓  Improvement  NE: Not evaluable  *Improvement defined as: ≥5 letters of improvement from baseline and ≥5 letter improvement from fellow eye in BCVA or LLVA, ≥0.2 logMAR improvement from baseline in contrast sensitivity, and ≥5 loci cluster improvement by ≥3 decibels in RPE Transitional Zone on microperimetry.  ARB, autosomal recessive bestrophinopathy; BCVA, best corrected visual acuity; BVMD, best vitelliform macular dystrophy; dB, decibel; IRF, intraretinal fluid; LLVA, low luminance visual acuity; logMAR, logarithm of the minimum angle of resolution; OCT, optical coherence tomography; RPE, retinal pigment epithelium.  14 

 -4  -6  -2  0  2  4  8  6  Baseline  M1  M3  Change from Baseline in BCVA (ETDRS Letters)  All Treated Eyes (N=5)  All Fellow Eyes (N=5)  Treated Eyes with Structural Improvement (N=4)*  Corresponding Fellow Eyes (N=4)*  Mean BCVA Improved from Baseline Through 3 Months in Cohort 1  *Excludes Participant 102-101 due to significant foveal atrophy (with a subfoveal, sub-RPE scar) at baseline, likely exclusionary from the pivotal trial. Participant 102-101 had a 0.2 logMAR decrease in vision from baseline, all other participants had an improvement in vision; Participant 102-101 had a meaningful improvement in contrast sensitivity.  Error bars represent the standard error of the mean.  BCVA, best corrected visual acuity; ETDRS, Early Treatment of Diabetic Retinopathy Study; M, month; RPE, retinal pigment epithelium.  +5 Letters  -2 Letters  15 

 Defining the Transitional Zone  Border area surrounding the atrophic lesion with RPE and photoreceptors that are structurally intact but functionally compromised or at risk of imminent atrophic progression  Highest potential to be rescued with  OPGx-BEST1  Treated RPE Transitional Zone is the area of rescuable photoreceptors around the atrophic lesion where  OPGx-BEST1 treatment is administered  New natural history data1: Untreated BVMD sensitivity only declines with highest rate detected in the transition zone – No untreated eye met the 3 dB improvement threshold, consistent with fellow eyes in Cohort 1  Microperimetry Showed BVMD Improvement in the Treated RPE Transitional Zone  *All eyes includes all evaluable patients (n=4); 101-101 could not complete microperimetry due to low vision.  †Improvement is defined as ≥5 loci area within the treated transitional zone improving by ≥3 dB from baseline.  BVMD, best vitelliform macular dystrophy; dB, decibel; FDA, Food and Drug Administration; RPE, retinal pigment epithelium.  1. Bianco L, et al. Invest Ophthalmol Vis Sci. 2026;67(11):1.  3/4 evaluable participants* demonstrated retinal sensitivity  †  improvement in the Transitional Zone  75%  Treated Area  Treated RPE Transitional Zone showing all loci that improved by ≥3 dB (circled) correlate with reduced vitelliform material  Central atrophy  Autofluorescence Microperimetry  FDA aligned on ≥3 dB change from baseline in ≥5 loci in treated RPE Transitional Zone anchored to a patient reported outcome  Reduction in vitelliform  material  16 

 BVMD: 67% of participants (2/3) had reduction in vitelliform material on multimodal imaging  All three BVMD participants had improvements in visual function,  with highest gains in areas of vitelliform material reduction  ARB: 100% of participants (2/2) had reduction in intraretinal fluid on OCT  Both ARB participants had improvements in visual function in areas where intraretinal fluid decreased  Functional gains were co-localized to structural improvements  Retinal sensitivity improved in the treated transitional zone at the edge of the lesion where fluid was minimal  Fixation moved from outside the lesion to within the lesion in all  four evaluable participants  Areas of the transitional zone treated within the subretinal bleb had the highest functional gains  Structural Improvements Seen in BVMD and ARB Participants  Results I nform Future E nrollment Earlier-stage participants showed the greatest structural and functional improvements, suggesting earlier intervention may yield improved outcomes  17 ARB, autosomal recessive bestrophinopathy; BVMD, best vitelliform macular dystrophy; OCT, optical coherence tomography.  A T R O P H Y  F R A G M E N T E D V I T E L L I F O R M M A T E R I A L  S U B R E T I N A L F L U I D  P S E U D O H Y P O P Y O N  Photoreceptors  Retinal Pigment Epithelium  Bruch’s Membrane  Choriocapillaris  V I T E L L I F O R M L E S I O N  V I T E L L I F O R M D E P O S I T S  STAGE 1  Pre-vitelliform  STAGE 2  Vitelliform  STAGE 3  Pseudohypopyon  STAGE 4  Vitelliruptive  STAGE 5  Atrophy/Fibrosis  S t a g e s o f B V M D 

 Post-Treatment Participant Feedback  101-101  ARB  Able to see on the eye chart for the first time in 30 years  Wants the second eye treated  102-102  BVMD  Wants the second eye treated  Reports less eye strain after prolonged computer work; colors on TV appear brighter and clearer  Happy with the study, mostly stable vision  101-104  ARB  102-101  BVMD  101-106  BVMD  ARB, autosomal recessive bestrophinopathy; BVMD, best vitelliform macular dystrophy.  18 

 Participant Case Studies & Next Steps  Ash Jayagopal, PhD, MBA  Chief Scientific and Development Officer  19 

 Baseline  Month 3  Participant 101-106 (BVMD): Microperimetry Improvements  Treated transitional zone follows the vitelliform arc as seen on fundus autofluorescence and OCT. Microperimetry loci were not pre-specified.  BVMD, best vitelliform macular dystrophy; dB, decibel; OCT, optical coherence tomography; TTZ, treated transitional zone; TZ, transitional zone.  Threshold for success: ≥3 dB  Average improvement in TTZ = 3.125 dB  Fellow eye TZ = 0.5 dB  7 loci had ≥3 dB improvement (circled)  This participant met the microperimetry  threshold for success of ≥3 dB  20 

 Baseline  Month 3  Participant 101-106 (BVMD): Vitelliform Material Reduction Co-localized with Microperimetry Improvements  BVMD, best vitelliform macular dystrophy; dB, decibel; RPE, retinal pigment epithelium.  Lesion reduction consistent with improved function of RPE cells and treatment activity as indicated in microperimetry improvements  Vitelliform lesions  reduced  B a s e l i n e  M o n t h 3  21 

 Baseline  Month 3  Participant 102-102 (BVMD): Microperimetry Improvements  Yellow box represents the treated transitional zone. Microperimetry loci were not prespecified.  BVMD, best vitelliform macular dystrophy; dB, decibel; TTZ; treated transitional zone; TZ, transitional zone.  Threshold for success: ≥3 dB  Average improvement in TTZ = 3.5 dB  Fellow eye TZ = 0.5 dB  6 loci had ≥3 dB improvement (circled) –  clustered within the TTZ  This participant met the microperimetry  threshold for success of ≥3 dB  22 

 Baseline  Month 3  Participant 102-102 (BVMD): Microperimetry Improved in the Treated RPE Transitional Zone  Baseline  Month 3  B a s e l i n e  M o n t h 3  Vitelliform material  decreased  Yellow box represents treated transitional zone.  BVMD, best vitelliform macular dystrophy; RPE, retinal pigment epithelium.  Fixation moves to a more natural location in the fovea  23 

 Participant 102-101 (BVMD): Photoreceptor and RPE Atrophy Limit Improvement  Images are consecutive registered OCT rasters at baseline, 1 month, and 3 months.  *CS improvements defined as ≥2 dB change from baseline.  BVMD, best vitelliform macular dystrophy; CS, contrast sensitivity; OCT, optical coherence tomography; RPE, retinal pigment epithelium.  Most advanced BVMD participant  Sub-RPE atrophy and scarring, exclusionary from a potential pivotal trial  Vitelliform material changes over time  CS improvements* akin to observations in geographic atrophy  3 Months  Baseline  1 Month  24 

 P a r a f o v e a l A r e a  I n f e r i o r A r e a  Baseline  Month 3  Month 6  Participant 101-101 (ARB): Functional and Structural Outcomes Through 6 Months  Functional improvement maintained to 6 months on BCVA, CS, and LLVA  Unable to perform MP due to poor baseline vision  IRF decreased at 3 months in the parafoveal area  Fluctuations observed in both areas between 3  and 6 months  ARB, autosomal recessive bestrophinopathy; BCVA, best-corrected visual acuity; BL, baseline; CFB, change from baseline; CS, contrast sensitivity; IRF, intraretinal fluid; LLVA, low luminance visual acuity; M, month, MP, microperimetry; OD, right eye; OS, left eye.  BL M1 M2 M3 M4 M5 M6 12  9 9  0  5  10  15  BCVA CFB  (Letters equivalent)  Study Eye - OS (Treated) Fellow Eye - OD (Untreated)  25 

 Participant 101-104 (ARB): Microperimetry Improvement  Baseline  Month 6  Total macular IRF volume = 19.06 µl at baseline, 7.55 µl at 6 months.  Microperimetry loci were not prespecified.  ARB, autosomal recessive bestrophinopathy; dB, decibel; IRF, intraretinal fluid; RPE, retinal pigment epithelium; TTZ; treated transitional zone.  Threshold for success: ≥3 dB  Average improvement in area of IRF = 3.3 dB  Fellow eye corresponding area = 2.3 dB  16 loci had ≥3 dB improvement (circled) –  clustered within the TTZ  This participant met the microperimetry  threshold for success of ≥3 dB  26 

 Participant 101-104 (ARB): IRF Improvement Co-localized with Microperimetry Improvement  Baseline  Month 6  Yellow circles denote pockets of intraretinal fluid.  Total macular intraretinal fluid volume: Baseline = 19.06 µl, Month 6 = 7.55 µl. ARB, autosomal recessive bestrophinopathy; IRF, intraretinal fluid.  Fixation moves to a more natural location in the fovea  Two areas of baseline intraretinal fluid  27 

 4 milestones cleared on the path to pivotal  OPGx-BEST1 Advancing Based on Positive Cohort 1 Results  1 2  3  4  Well-tolerated with no SAEs, no DLTs, and no intraocular inflammation  Efficacy observed on both functional and structural endpoints  Positive FDA Type C meeting aligned on potential pivotal endpoint with microperimetry  28 CMC, chemistry, manufacturing, and controls; dB, decibel; DLT, dose-limiting toxicity; FDA, Food and Drug Administration; IDMC, independent data monitoring committee; SAE, serious adverse event.  75% of Cohort 1 evaluable participants met the microperimetry threshold for success of ≥3 dB 

 Program Summary and Next Steps  George Magrath, MD Chief Executive Officer  29 

 Accomplished Significant OPGx-BEST1 Program Milestones  P H 1 / 2 C O H O R T 2 S TAT U S  D o s e  4.5x109 vg/eye  E n r o l l m e n t  Already over-enrolled  Originally planned for 5 participants  Over-enrollment of 8 participants  C l i n i c a l D e v e l o p m e n t T i m e l i n e *  Dosing expected to be completed Q4 2026  Topline 3-month data expected Q2 2027   Pivotal trial planning initiated, dosing expected in 2027  R E G U L AT O R Y S TAT U S  A u g u s t 2 0 2 6 F D A Ty p e C m e e t i n g  Aligned on Phase 3 CMC   Constructive dialogue on potential endpoint options for Phase 3  P o t e n t i a l P i v o t a l E n d p o i n t s  Use of ≥3 dB microperimetry improvement in  ≥5 prespecified loci area and;  Randomized controlled trial with a patient-reported outcome  BCVA, LLVA, contrast sensitivity may also be acceptable endpoints  N e x t R e g u l a t o r y I n t e r a c t i o n  Following early data from Cohort 2  *Clinical development timelines are based on current estimates and are subject to change; data readouts are targeted for ~9-12 months after study initiation.  ARB, autosomal recessive bestrophinopathy; BCVA, best corrected visual acuity; BVMD, best vitelliform macular dystrophy; CMC, chemistry, manufacturing, and controls; dB, decibels; FDA, Food and Drug Administration; LLVA, low luminance visual acuity.  30 

 Current Cash to Support Multiple Clinical Inflection Points  2027  BEST1 Pivotal Study Start  Q1 2027  MERTK clinical study initiation  Q4 2026  RDH12 clinical study initiation  Q4 2026  LCA5 Phase 3 dosing initiation  Oct 2026  PDUFA date for  Phentolamine  sNDA  Sept 2026  BEST1 Phase 1/2 Cohort 1 3-month  results  Current cash runway extends into 2029, funding five clinical programs through multiple critical inflection points  4 Clinical Data Readouts  Expected in 2027:  BEST1, LCA5, RDH12, MERTK  Clinical development timelines are based on current estimates and are subject to change; data readouts are targeted for ~9-12 months after study initiation.  Phentolamine ophthalmic solution 0.75% is a commercial partnered program; it is FDA-approved for the treatment of pharmacologically-induced mydriasis; an sNDA has been submitted for the treatment of presbyopia. BEST1, bestrophin 1; LCA5, Leber congenital amaurosis 5; MERTK, MER proto-oncogene tyrosine kinase; PDUFA, Prescription Drug User Fee Act; PRV, Priority Review Voucher; RDH12, retinol dehydrogenase 12; RHO, rhodopsin; sNDA, supplemental New Drug Application.  Q2 2027  BEST1  Cohort 2  results  31 

 N a s d a q : I R D 

 33  Mark E. Pennesi, MD, PhD, FARVO  Leading ophthalmologist, researcher specializing in inherited retinal diseases, and pioneer in gene therapy  Current Appointments  Chief Medical Officer, Steve and Debbie Gray Inherited Retinal Degeneration Endowed Chair; Director, Inherited Retinal Degeneration Center – Retina Foundation, Dallas, Texas  Adjunct Professor of Ophthalmology – Paul H. Casey Ophthalmic Genetics Division, Casey  Eye Institute, Oregon Health & Science University, Portland, Oregon  Education  B.S. in Biomedical Engineering, University of Pennsylvania (summa cum laude); Combined MD/PhD, Baylor College of Medicine; Ophthalmology residency, UCSF; Ophthalmic genetics fellowship, Casey Eye Institute/OHSU  Research and Awards  Research focuses on developing novel treatments for inherited retinal diseases  Author of 170+ peer-reviewed publications; principal or co-principal investigator on numerous first-in-human gene therapy trials  Research to Prevent Blindness and the Foundation Fighting Blindness have recognized Dr. Pennesi with career development awards; Additionally, he was the recipient of the 2011 ARVO/Alcon Early Clinician Scientist, the Alcon Young investigator Award in 2014, and the Casey Eye Institute Resident teach award 

 34  Q&A with Opus Management and Dr. Pennesi  Ash Jayagopal, PhD, MBA  Chief Scientific & Development Officer  Sally Tucker, MCOptom, PhD  Chief Medical Officer  Rob Gagnon, CPA, MBA  Chief Financial Officer  Mark E. Pennesi, MD, PhD, FARVO Retina Foundation of the Southwest Dallas, TX  George Magrath, MD  Chief Executive Officer  Ben Yerxa, PhD  President 

 Appendix  35 

 36 Triangle Insights Group Research, Q3 2026.  BEST1 Market Landscape Quantitative Market Research  Patient Group  Definition  Est. Value  Genetic  BEST1 genotype (with or without symptoms)  ~46,000 – 87,000  patients  Symptomatic  Diagnosed + undiagnosed (with ocular symptoms)  ~23,600 patients  Diagnosed (Post-onset)  Diagnosed (with ocular symptoms)  ~13,000 patients  Diagnosed (Genetically Confirmed)  Diagnosed with genetic  confirmation  ~8,400 patients  Undiagnosed (Post-onset)  Undiagnosed (with ocular symptoms)  ~10,600 patients  BEST1 Patients: Estimated Inputs  Symptomatic  23,600 pts  Undiagnosed: 10,600 pts  Diagnosed (Post-Onset) 13,000 pts  Diagnosed  (Genetic)  8,400 pts  BEST1 Mutation: Estimated Subpopulations  Genetic 46,000 – 87,000 pts 

 37  101-106 Microperimetry  B a s e l i n e  M o n t h 3 

 38  102-102 Microperimetry  B a s e l i n e  M o n t h 3 

 39  101-104 Microperimetry  B a s e l i n e  M o n t h 6 

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