Opus Genetics: 3 of 4 show interim retinal sensitivity gains
The OPGx-LCA5 Phase 3 program is proceeding at the low dose, with dosing initiation expected in the fourth quarter of 2026.
Sentiment and the balance of points
Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.
Form Type
8-K
Rhea-AI Filing Summary
Opus Genetics, Inc. (IRD) presented interim clinical findings for its OPGx-BEST1 and OPGx-LCA5 gene therapy programs. In low-dose Cohort 1 of the ongoing Phase 1/2 OPGx-BEST1 trial, five participants were treated; among four evaluable for retinal sensitivity testing, three (75%) improved in the treated transitional zone, with follow-up of three to six months. The company also reported imaging analyses associating retinal structure with visual function.
In the OPGx-LCA5 Phase 1/2 study, three adults had reported improvements in visual acuity and light sensitivity through 24 months, and three pediatric participants through six months. The low dose was 1E10 vector genomes per eye. The Phase 3 trial advanced using the low dose; its natural-history and treatment cohorts are fully enrolled, and dosing initiation is expected in the fourth quarter of 2026. The company said trial data are interim, subject to change, and may not predict final outcomes.
Positive
- Minor pointOPGx-BEST1 retinal sensitivity improved in 3 of 4 evaluable participants.
- Minor pointOPGx-LCA5 visual-acuity and light-sensitivity improvements were reported through 24 months in three adults.
Negative
- None.
Filing Explained
BEST1’s high-dose enrollment is complete; LCA5 Phase 3 has a specified retinal-sensitivity bar for assessing efficacy.
The conference presentations add that OPGx-BEST1’s high-dose Cohort 2 is fully enrolled with 11 participants, and specify the primary efficacy measure for OPGx-LCA5 Phase 3.
That measure is mean retinal-sensitivity improvement of more than 7 dB across 16 central test locations, defining what the trial will assess.
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
OPGx-BEST1 participants with improved retinal sensitivity: 3 of 4 participants (75%)
OPGx-BEST1 participants treated: 5 participants
OPGx-LCA5 low dose: 1E10 vector genomes per eye
+4 more
7 metrics
OPGx-BEST1 participants with improved retinal sensitivity
3 of 4 participants (75%)
Evaluable participants in low-dose Cohort 1
OPGx-BEST1 participants treated
5 participants
Low-dose Cohort 1; follow-up ranged from three to six months
OPGx-LCA5 low dose
1E10 vector genomes per eye
Dose used in the Phase 1/2 study
Adult OPGx-LCA5 follow-up
24 months
Visual-acuity and light-sensitivity improvements reported in three adults
Pediatric OPGx-LCA5 follow-up
Six months
Visual-acuity and light-sensitivity improvements reported in three pediatric participants
OPGx-LCA5 Phase 3 dosing initiation
Fourth quarter of 2026
Expected timing
OPGx-BEST1 high-dose Cohort 2 enrollment
11 participants
Enrollment completed
Key Terms
transitional zone, microperimetry, full-field stimulus testing, AAV8-based gene therapy
4 terms
transitional zone medical
"improvements in the treated transitional zone"
microperimetry medical
"measured using microperimetry"
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.
full-field stimulus testing medical
"as measured by full-field stimulus testing"
A psychophysical eye test that measures the eye's overall light sensitivity by presenting full-field (whole-retina) flashes of light and recording whether the patient perceives them. Unlike spatial visual field tests that map sensitivity across locations, full-field stimulus testing (FST) provides a single global threshold (often across different light intensities and colors) and is used when fixation or localized responses are unreliable, for example in very low-vision or widespread retinal disease. Limitation: FST does not give information about where on the retina loss occurs, only how much light is needed for a detectable response.
AAV8-based gene therapy technical
"adeno-associated virus serotype 8 (AAV8)-based gene therapy"
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What did IRD report for OPGx-BEST1?
In low-dose Cohort 1, 3 of 4 evaluable participants (75%) showed improved retinal sensitivity in the treated transitional zone. Five participants were treated, and follow-up ranged from three to six months.
How long did OPGx-LCA5 improvements last in the reported study?
The study reported improvements in visual acuity and light sensitivity through 24 months in three adults and through six months in three pediatric participants.
What is the primary efficacy endpoint for IRD’s OPGx-LCA5 Phase 3 trial?
The presentation described the primary efficacy endpoint as a mean retinal sensitivity improvement of more than 7 dB across 16 central test locations.
AI-generated analysis. How Rhea-AI works. Not financial advice.
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): October 14, 2026
(Exact name of registrant as specified in its charter)
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(IRS Employer Identification No.)
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(Address of principal executive offices)
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(Zip Code)
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(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:
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR
240.14d-2(b))
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR
240.13e-4(c))
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Securities registered pursuant to Section 12(b) of the Act:
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Title of each class
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Trading Symbol(s)
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Name of each exchange on which registered
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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.
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On October 15, 2026, Opus Genetics, Inc. (the “Company”) issued a press release regarding the Company’s October 14, 2026
presentations at the Retina in Rio 2026 conference held in Rio de Janeiro, Brazil. The presentations covered clinical data from Cohort 1 of the Company’s ongoing Phase 1/2 clinical trial of OPGx-BEST1 (the “OPGx-BEST1 Presentation”) and clinical
data from the Company’s ongoing Phase 1/2/3 clinical trial of OPGx-LCA5 (the “OPGx-LCA5 Presentation”).
A copy of the press release is furnished as Exhibit 99.1 to
this Current Report on Form 8-K. A copy of the OPGx-BEST1 Presentation is furnished as Exhibit 99.2 to this Current Report on Form 8-K. A
copy of the OPGx-LCA5 Presentation is furnished as Exhibit 99.3 to this Current Report on Form 8-K.
The information in this report is furnished pursuant to Item 7.01, including Exhibit 99.1 attached hereto, 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 it 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.
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Financial Statements and Exhibits.
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Exhibits
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Exhibit No.
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Description
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99.1
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Press release issued by Opus Genetics, Inc. on October 15, 2026, furnished herewith
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99.2
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Retina in Rio 2026 – OPGx-BEST1 Presentation, furnished herewith
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99.3
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Retina in Rio 2026 – OPGx-LCA5 Presentation, furnished herewith
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104
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Cover page from this Current Report on Form 8-K, formatted in Inline XBRL
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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.
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OPUS GENETICS, INC.
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Date:
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October 15, 2026
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By:
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/s/ Dr. George Magrath
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Dr. George Magrath
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Chief Executive Officer
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Exhibit 99.1
Opus Genetics Presents Positive Clinical Data from OPGx-BEST1 and OPGx-LCA5 Gene Therapy Programs at Retina in Rio 2026
OPGx-BEST1 showed improved retinal sensitivity in 75% of evaluable participants in the initial low-dose Cohort 1 of the Company’s Phase 1/2 clinical
trial, supporting further clinical development
New BEST1 data analyses link retinal structure to visual function, enabling focus on regions most likely to respond to treatment
OPGx-LCA5 demonstrated sustained improvements in visual acuity and light sensitivity through 24 months in adults, with encouraging results in
pediatric participants through six months in the Company’s Phase 1/2/3 clinical trial
Phase 3 clinical trial dosing for OPGx-LCA5 expected in the fourth quarter of 2026
RESEARCH TRIANGLE PARK, N.C. – October 15, 2026 - Opus Genetics, Inc. (Nasdaq: IRD) (“Opus Genetics” or the “Company”), a clinical-stage biopharmaceutical company
developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), presented clinical findings from its OPGx-BEST1 and OPGx-LCA5 gene therapy programs at Retina in Rio 2026 in Rio de Janeiro,
Brazil.
The Company presented findings from the low-dose Cohort 1 of its Phase 1/2 BIRD-1 trial of OPGx-BEST1, which showed improved retinal sensitivity in three of four
evaluable participants (75%). Additional analyses identified retinal structural and imaging characteristics associated with visual function and treatment response, providing insights that may help guide future clinical development. Additional
findings from the ongoing Phase 1/2 trial of OPGx-BEST1 demonstrated encouraging improvements in retinal sensitivity in participants with BEST1-associated retinal diseases, along with new insights into imaging markers that may help predict
treatment response.
The Company also presented findings from the Phase 1/2 clinical trial of OPGx-LCA5, which demonstrated sustained improvements in visual function among adults and children
treated with OPGx-LCA5 for Leber congenital amaurosis type 5 (LCA5), a rare inherited retinal disease that causes severe vision loss beginning in childhood. In the OPGx-LCA5 Phase 1/2 study, improvements in visual acuity and light sensitivity were
observed in three adult participants through 24 months and three pediatric participants through six months. Participants also reported improvements in everyday activities, including navigating independently, recognizing family members, and
identifying objects and seeing colors.
“The data presented at Retina in Rio highlight meaningful progress across our gene therapy pipeline, with sustained improvements in visual function observed through 24
months in adults treated with OPGx-LCA5 and encouraging early evidence of improved retinal sensitivity in participants receiving OPGx-BEST1,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “Importantly, these findings strengthen
our understanding of how to measure treatment response in IRDs and support the continued advancement of both programs. Our OPGx-BEST1 Phase 1/2 trial has completed enrollment in the high-dose Cohort 2 with 11 participants, and with our OPGx-LCA5
Phase 3 trial progressing toward dosing initiation later this year, we remain focused on bringing potentially transformative gene therapies to patients living with rare inherited retinal diseases.”
OPGx-BEST1
Opus presented findings from the ongoing Phase 1/2 BIRD-1 trial of OPGx-BEST1, an investigational gene therapy designed to restore bestrophin-1 function in patients with
IRDs caused by mutations in the BEST1 gene, including Best vitelliform macular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB).
The presentation included results from five participants treated in the initial low-dose cohort, with follow-up ranging from three to six months. Among four participants
who could complete retinal sensitivity testing, three (75%) demonstrated improvements in the treated transitional zone, an area surrounding damaged retinal tissue where light-sensitive cells remain and may be responsive to treatment. Improvements
were measured using microperimetry, a test that evaluates the retina's ability to detect light at specific locations. In responding participants, increases in retinal sensitivity were also associated with reductions in intraretinal fluid
characteristic of BEST1-associated disease.
Additional analyses examined the relationship between retinal structure and function, suggesting that measurements obtained through optical coherence tomography (OCT) and
other imaging techniques may help identify retinal regions most likely to respond to treatment. The presentation also highlighted natural history findings demonstrating that retinal sensitivity declines most noticeably in the transitional zone
surrounding damaged tissue, even when measurements across the entire retina appear relatively stable.
Together, these findings are informing future clinical development of OPGx-BEST1, including more targeted retinal sensitivity assessments and imaging approaches designed
to improve the measurement of treatment response.
OPGx-LCA5
Rodrigo Jorge, M.D., of Ribeirão Preto Medical School at the University of São Paulo, presented findings from the ongoing Phase 1/2/3 trial of OPGx-LCA5, an
investigational gene therapy designed to restore production of lebercilin, a protein essential for normal retinal function. The presentation included data from six participants treated at the low dose of 1E10 vector genomes per eye, including three
adults and three adolescents. Adults demonstrated sustained improvements in visual acuity through 24 months, while pediatric participants demonstrated restoration of cone-mediated vision with sensitivity improvements reaching normal ranges through six months. Both groups also demonstrated improved light sensitivity, as measured by full-field stimulus testing (FST), compared with
their untreated eyes.
Additional retinal imaging using microperimetry showed increased retinal sensitivity and movement of visual fixation toward the central retina, which is responsible for
sharp, detailed vision. Participants also reported meaningful improvements in everyday activities, including recognizing family members, navigating independently without a cane, identifying objects and participating in activities that had
previously been difficult because of severe vision loss.
Based on the encouraging Phase 1/2 findings, Opus advanced OPGx-LCA5 into Phase 3 development using the low dose without requiring further dose escalation. The Phase 3
clinical trial’s natural history and treatment cohorts are fully enrolled, with dosing initiation expected in the fourth quarter of 2026. The clinical trial’s primary efficacy endpoint will assess improvements in retinal sensitivity across 16
central retinal test locations.
OPGx-LCA5 is an investigational adeno-associated virus serotype 8 (AAV8)-based gene therapy designed to deliver a functional copy of the LCA5 gene to retinal
photoreceptors. Mutations in LCA5 disrupt production of lebercilin, a protein critical for normal retinal function, resulting in severe visual impairment beginning early in life.
The full presentations and a video recording of the BEST1 slides will be available on the Opus Genetics website in the Publications and Presentations section.
Forward-Looking Statements
This press release contains certain statements that are not statements of historical fact and are forward-looking statements. 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,” “strive,” “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 the Company’s continued clinical development, clinical results,
preclinical data, and future plans for OPGx-BEST1 and OPGx-LCA5, including the anticipated timing of dosing initiation for the Company’s Phase 3 clinical trial of OPGx-LCA5; the Company’s ability to use insights from Cohort 1 of the Phase 1/2
OPGx-BEST1 clinical trial to help guide future clinical development and predict treatment response; the Company’s expectations regarding the clinical and therapeutic potential of OPGx-BEST1 and OPGx-LCA5, including with respect to the ability of
OPGx-BEST1 to improve retinal sensitivity and the ability of OPGx-LCA5 to enable sustained improvements in visual function; the Company’s participant-selection, treatment response assessment and development strategy for subsequent clinical trials
of OPGx-BEST1 and OPGx-LCA5; the Company’s expectations regarding the design of, and potential endpoints for, current and future clinical trials of OPGx-BEST1 and OPGx-LCA5; and the Company’s expectations regarding its business prospects and
results of operations. The OPGx-BEST1 and OPGx-LCA5 trials referenced in this press release are ongoing, and the data described are interim, subject to change, and based on data available as of a specified date. As participant monitoring and
enrollment in these trials continue and additional follow-up data is obtained, the reported data and other clinical outcomes may change materially. There can be no assurance that the initial results will be predictive of final clinical trial
results or that additional data will confirm or support these observations. The forward-looking statements contained herein are subject to certain risks and uncertainties posed by many factors and events that could cause the Company’s 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, those described under the
heading “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K for the fiscal year ended December 31, 2025, its Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, and in the Company’s other filings with the
U.S. Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. These forward-looking statements are based upon the Company’s
current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various
risks and uncertainties. The Company undertakes no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.
About Opus Genetics
Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases
(IRDs). The Company is developing durable, one-time treatments designed to address the underlying genetic causes of severe retinal disorders. The Company’s pipeline includes seven AAV-based programs, led by OPGx-LCA5 for LCA5-related mutations and
OPGx-BEST1 for BEST1-related retinal degeneration, with additional candidates targeting RDH12, MERTK, RHO, CNGB1 and NMNAT1. The Company is based in Research Triangle Park, NC. For more information, visit www.opusgtx.com.
Contacts:
Investors
Jenny Kobin
Remy Bernarda
IR Advisory Solutions
ir@opusgtx.com
Media
Kimberly Ha
KKH Advisors
917-291-5744
kimberly.ha@kkhadvisors.com
Source: Opus Genetics, Inc.
Exhibit 99.2
Exhibit 99.3
