This section highlights FDA-related milestones and regulatory updates for drugs developed by Ocuphire Pharma (OCUP).
Over the past two years, Ocuphire Pharma has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
OPGx-LCA5, VEGA-3, LYNX-2, RYZUMVI, and APX3330. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
OPGx-LCA5 FDA Regulatory Timeline and Events
OPGx-LCA5 is a drug developed by Ocuphire Pharma for the following indication: For LCA5.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- OPGx-LCA5
- Announced Date:
- September 30, 2025
- Indication:
- For LCA5
Announcement
Opus Genetics, Inc. announced positive three-month data from the pediatric cohort of its ongoing Phase 1/2 clinical trial (OPGx-LCA5-1001) evaluating OPGx-LCA5, an investigational gene augmentation therapy for Leber congenital amaurosis type 5 (LCA5).
AI Summary
Opus Genetics reported positive three-month results from the pediatric cohort in its ongoing Phase 1/2 trial of OPGx-LCA5, a gene therapy for Leber congenital amaurosis type 5. In three teens (ages 16–17) with severe vision loss, the therapy improved cone-mediated vision and was well tolerated with no serious eye adverse events.
On average, visual acuity improved by 0.3 logMAR, with individual gains of 0.5, 0.2, and 0.7 logMAR. Full-Field Stimulus Testing showed more than a 1-log unit improvement in cone sensitivity to red and blue light. All participants identified more objects in the Multi-Luminance Orientation and Mobility Test, and one showed better fixation stability on microperimetry, indicating early functional recovery.
No dose-limiting toxicities were observed, and adult participants maintained visual gains through 18 months. Opus Genetics plans an FDA meeting in Q4 2025 to discuss next steps for the LCA5 program.
Read Announcement- Drug:
- OPGx-LCA5
- Announced Date:
- May 6, 2025
- Indication:
- For LCA5
Announcement
Opus Genetics, Inc announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OPGx-LCA5, its investigational gene therapy for the treatment of Leber Congenital Amaurosis (LCA) due to genetic variations in the LCA5 gene.
AI Summary
Opus Genetics, Inc. announced that its investigational gene therapy, OPGx-LCA5, has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. This designation comes as a result of promising early clinical data from the ongoing Phase 1/2 open-label, dose-escalation trial. The trial is evaluating the safety and potential effectiveness of OPGx-LCA5 in patients with severe vision loss caused by mutations in the LCA5 gene, which leads to Leber Congenital Amaurosis (LCA). The FDA’s decision highlights both the strength of the preliminary data and the urgent need for a treatment in this underserved patient community. With RMAT designation, development and review of this gene therapy may be expedited, providing hope for patients suffering from this rare, inherited form of blindness.
Read Announcement- Drug:
- OPGx-LCA5
- Announced Date:
- May 5, 2025
- Indication:
- For LCA5
Announcement
Opus Genetics, Inc. announced one-year results from adult patients treated in the ongoing Phase 1/2 Study of its lead gene therapy candidate OPGx-LCA5.
AI Summary
Opus Genetics, Inc. announced promising one-year results from its ongoing Phase 1/2 study of OPGx-LCA5, a gene therapy for adult patients with severe vision loss due to mutations in the LCA5 gene. The treatment, given as a subretinal injection, has shown that the improvements in vision noted at six months continue through the full year. Patients experienced clear gains in cone-mediated (daytime) vision, which helped enhance their reading abilities and object recognition. Importantly, the therapy was well tolerated with no serious adverse effects reported. These encouraging results support the continued development of OPGx-LCA5 as a potential life-changing treatment for those battling inherited retinal degeneration, offering hope for substantial advances in the treatment of these conditions.
Read Announcement - Drug:
- OPGx-LCA5
- Announced Date:
- April 8, 2025
- Indication:
- For LCA5
Announcement
Opus Genetics, Inc. announced one-month clinical data from the first pediatric patient treated with its investigational gene therapy, OPGx-LCA5, in a Phase 1/2 open-label trial for LCA5-related inherited retinal disease (IRD).
AI Summary
Opus Genetics, Inc. shared promising one-month clinical results from the first pediatric patient treated with its investigational gene therapy, OPGx-LCA5, for LCA5-related inherited retinal disease. In this Phase 1/2 open-label trial, the treated patient showed notable improvements in visual function just one month after treatment. The patient reported that objects appeared significantly brighter, and she was able to distinguish letters more clearly and navigate independently—an experience she had never had before. Importantly, no drug-related adverse events have been observed, adding to the evidence that early intervention with OPGx-LCA5 may safely restore meaningful vision in pediatric patients. The trial continues to enroll additional pediatric participants, with further data expected later this year, building on positive outcomes that were previously seen in adult patients. This early pediatric data reinforces the potential of this gene therapy in addressing progressive vision loss.
Read Announcement- Drug:
- OPGx-LCA5
- Announced Date:
- March 5, 2025
- Indication:
- For LCA5
Announcement
Opus Genetics, Inc. announced that three abstracts on its investigational gene therapy candidates have been accepted for presentation at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Meeting, to take place May 4-8, 2025 in Salt Lake City, UT.
AI Summary
Opus Genetics, Inc. announced that three abstracts on its investigational gene therapy candidates have been accepted for presentation at the ARVO 2025 Meeting. This prestigious event will take place from May 4-8, 2025 in Salt Lake City, UT. The presented abstracts will share important data, including 12-month results from the first three adult patients treated with OPGx-LCA5 in an ongoing Phase 1/2 trial. The data highlights sustained improvements in vision, suggesting that this gene therapy candidate has promising potential for treating Leber congenital amaurosis caused by LCA5 mutations.
Additionally, pre-clinical studies of OPGx-MERTK and OPGx-RDH12, which target other inherited retinal diseases, will be discussed. These presentations provide valuable insights for the global ophthalmology community and support Opus Genetics' ongoing efforts to develop potentially life-changing treatments for patients with inherited retinal diseases.
Read Announcement- Drug:
- OPGx-LCA5
- Announced Date:
- February 18, 2025
- Indication:
- For LCA5
Announcement
Opus Genetics, Inc. announced that the first pediatric patient was dosed in its ongoing Phase 1/2 clinical trial evaluating OPGx-LCA5, its investigational gene therapy for the treatment of Leber congenital amaurosis (LCA).
AI Summary
Opus Genetics, Inc. announced a key milestone in its clinical study for OPGx-LCA5, an investigational gene therapy designed to treat Leber congenital amaurosis (LCA). The company has dosed its first pediatric patient in the ongoing Phase 1/2 trial, expanding testing beyond adult subjects. Early treatment in children is seen as especially important since it may help preserve or restore visual function before the disease worsens.
Preliminary pediatric data is expected by the third quarter of 2025. The therapy, which uses an adeno-associated virus vector to deliver a functional LCA5 gene, has already shown promise in adult patients with visual improvements noted as early as one month following treatment. This progress suggests a potential life-changing therapeutic option for those affected by LCA.
Read Announcement- Drug:
- OPGx-LCA5
- Announced Date:
- February 18, 2025
- Estimated Event Date Range:
- July 1, 2025 - September 30, 2025
- Target Action Date:
- Q3 2025
- Indication:
- For LCA5
Announcement
Opus plans to share initial data from the pediatric cohort by Q3 2025.
AI Summary
Opus Genetics recently announced that the first pediatric patient in their Phase 1/2 trial for OPGx-LCA5 has been dosed. This gene therapy is being tested as a treatment for Leber congenital amaurosis, an inherited retinal disease that severely impacts vision. Early intervention in pediatric patients is especially important, given the potential to preserve or restore visual function before the disease progresses. Opus Genetics plans to share the initial data from this pediatric cohort by the third quarter of 2025, marking a significant step forward in understanding the therapy’s safety and effectiveness in younger patients. The progress in the trial builds on earlier promising results observed in adult patients and continues to offer hope for a new therapeutic option for those affected by this serious condition.
Read Announcement- Drug:
- OPGx-LCA5
- Announced Date:
- December 3, 2024
- Indication:
- For LCA5
Announcement
Opus Genetics announced that it will host a virtual key opinion leader (KOL) event on Wednesday, December 11, 2024 at 4:00 PM ET.
AI Summary
Opus Genetics, a clinical-stage ophthalmic biotechnology company, announced it will host a virtual key opinion leader (KOL) event on Wednesday, December 11, 2024 at 4:00 PM ET. The session will feature expert speakers, including Jean Bennett, MD, PhD, and Tomas Aleman, MD, from the University of Pennsylvania, as well as Christine Kay, MD, and Arshad Khanani, MD, MA, FASRS. These leading professionals will present detailed six‐month efficacy and safety data for OPGx-LCA5, an adeno-associated virus (AAV)-based gene therapy aimed at treating LCA5-associated inherited retinal disease.
The event promises a comprehensive look at the latest clinical data, discussing the therapeutic potential of the treatment and the unmet needs among patients affected by this severe retinal disorder. A live question and answer session will allow participants to engage directly with the experts about the next steps in the development program.
Read Announcement
VEGA-3 FDA Regulatory Events
VEGA-3 is a drug developed by Ocuphire Pharma for the following indication: Evaluating Phentolamine Ophthalmic Solution 0.75% for Presbyopia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- VEGA-3
- Announced Date:
- June 26, 2025
- Indication:
- Evaluating Phentolamine Ophthalmic Solution 0.75% for Presbyopia
Announcement
Opus Genetics, Inc. announced positive topline results from VEGA-3, its second pivotal Phase 3 trial evaluating Phentolamine Ophthalmic Solution 0.75% for the treatment of presbyopia. Presbyopia is an ophthalmic disorder that involves the progressive loss of ability to focus on close objects that results in blurred near vision, difficulty seeing in dim light, and eye strain.
AI Summary
Opus Genetics, Inc. announced positive topline results from its second pivotal Phase 3 trial, VEGA-3, evaluating Phentolamine Ophthalmic Solution 0.75% for treating presbyopia. In this trial, 27.2% of participants receiving the drug achieved a significant improvement in near visual acuity—a gain of at least 15 ETDRS letters—while maintaining stable distance vision. This outcome was notably superior to the 11.5% observed in the placebo group, meeting the primary endpoint with strong statistical significance. The study also met key secondary endpoints, showing rapid and sustained improvement in near vision without any evidence of reduced drug response over the six-week period. Additionally, the safety profile was consistent with prior trials, with no treatment-related serious adverse events reported. These findings suggest that Phentolamine Ophthalmic Solution 0.75% could provide a beneficial treatment option for millions of adults dealing with blurred near vision, low-light difficulties, and eye strain due to presbyopia.
Read Announcement- Drug:
- VEGA-3
- Announced Date:
- September 5, 2024
- Indication:
- Evaluating Phentolamine Ophthalmic Solution 0.75% for Presbyopia
Announcement
Ocuphire Pharma, Inc. announced that the VEGA-3 Phase 3 clinical trial evaluating Phentolamine Ophthalmic Solution 0.75% for presbyopia has dosed its first participants.
AI Summary
Ocuphire Pharma, Inc. announced that the VEGA-3 Phase 3 clinical trial for its Phentolamine Ophthalmic Solution 0.75% has begun dosing its first participants. This trial is designed to assess the drug’s safety and effectiveness as a new treatment for presbyopia, a condition that causes difficulty focusing on near objects as people age. The study aims to provide an easy, non-invasive alternative to traditional corrective measures like reading glasses or bifocals.
In this randomized, double-masked, placebo-controlled trial, 545 participants from up to 40 investigational sites in the United States will take part. Each participant will receive one drop of the Phentolamine solution or a placebo every evening. The primary goal is to measure significant improvement in near visual acuity by the eighth day after the initial dose, with long-term safety data collected over 48 weeks.
Read Announcement
LYNX-2 FDA Regulatory Timeline and Events
LYNX-2 is a drug developed by Ocuphire Pharma for the following indication: For the Treatment of Decreased Visual Acuity under Dim (mesopic) Light Conditions.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- LYNX-2
- Announced Date:
- June 2, 2025
- Indication:
- For the Treatment of Decreased Visual Acuity under Dim (mesopic) Light Conditions
Announcement
Opus Genetics, Inc. announced positive topline results from LYNX-2, a pivotal Phase 3 clinical trial evaluating Phentolamine Ophthalmic Solution 0.75% for the treatment of significant, chronic night driving impairment in keratorefractive patients with reduced mesopic vision.
AI Summary
Opus Genetics, Inc. recently announced positive topline results from the pivotal Phase 3 LYNX-2 trial. This study evaluated Phentolamine Ophthalmic Solution 0.75% in keratorefractive patients experiencing significant chronic night driving impairment due to reduced mesopic vision. Patients who had undergone procedures such as LASIK and PRK often suffer from glare, halos, and other vision disturbances under low light conditions. The study met its primary endpoint, with 17.3% of patients achieving a ≥15-letter improvement in mesopic low contrast distance visual acuity after 15 days, compared to 9.2% in the placebo group.
The trial also showed notable patient-reported improvements in night driving performance, such as better visibility of oncoming headlights and reduced glare at dawn or dusk. Importantly, the safety profile was consistent with previous studies, with no new safety concerns or signs of tachyphylaxis observed over the 6-week period.
Read Announcement- Drug:
- LYNX-2
- Announced Date:
- February 26, 2025
- Indication:
- For the Treatment of Decreased Visual Acuity under Dim (mesopic) Light Conditions
Announcement
Opus Genetics, Inc. announced completion of enrollment in the VEGA-3 Phase 3 clinical trial evaluating Phentolamine Ophthalmic Solution 0.75% for presbyopia.
AI Summary
Opus Genetics, Inc. announced that it has finished enrolling patients in its VEGA-3 Phase 3 clinical trial. The trial is designed to evaluate Phentolamine Ophthalmic Solution 0.75% as a treatment for presbyopia, a common age-related condition that affects near vision. In this randomized, double-masked, placebo-controlled study, 545 participants are taking part to determine how effectively the solution improves near vision. The main goal of the trial is to assess the percentage of participants who achieve a significant 15-letter improvement in distance-corrected near visual acuity by the eighth day after treatment begins. Participants will be observed over a 48-week period to monitor safety. This trial represents an important step in developing a non-invasive treatment that may greatly improve quality of life for millions affected by presbyopia.
Read Announcement- Drug:
- LYNX-2
- Announced Date:
- September 5, 2024
- Estimated Event Date Range:
- January 1, 2025 - June 30, 2025
- Target Action Date:
- H1 2025
- Indication:
- For the Treatment of Decreased Visual Acuity under Dim (mesopic) Light Conditions
Announcement
Ocuphire Pharma, Inc. announced that the Top-line data from VEGA-3 Phase 3 trial expected in first half of 2025
AI Summary
Ocuphire Pharma Inc. recently announced that top-line data from its VEGA-3 Phase 3 trial is expected in the first half of 2025. The study is evaluating the safety and effectiveness of Phentolamine Ophthalmic Solution 0.75% for treating presbyopia, a condition that reduces the ability to focus on close objects as people age. In this randomized, double-masked, placebo-controlled trial, about 545 participants are being assessed to see if the treatment can achieve a significant improvement in near visual acuity. The primary measure will be a 15-letter gain in distance-corrected near visual acuity, recorded eight days after dosing begins. Ocuphire is hopeful that the data will support a supplemental New Drug Application with the FDA, potentially offering a non-invasive alternative for those relying on reading glasses or bifocals.
Read Announcement- Drug:
- LYNX-2
- Announced Date:
- April 11, 2024
- Indication:
- For the Treatment of Decreased Visual Acuity under Dim (mesopic) Light Conditions
Announcement
Ocuphire Pharma, Inc announced the enrollment of the first subject in the LYNX-2 Phase 3 registration study evaluating Phentolamine Ophthalmic Solution 0.75% (PS) for the treatment of decreased visual acuity under low (mesopic) light conditions following keratorefractive surgery.
AI Summary
Ocuphire Pharma, Inc. announced it has enrolled the first subject in its LYNX-2 Phase 3 registration study. The trial is assessing the safety and effectiveness of Phentolamine Ophthalmic Solution 0.75% (PS) for treating decreased visual acuity under low light conditions in patients who have undergone keratorefractive surgery. This study is being conducted under a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA), which means that the FDA has agreed that the trial protocol and statistical plans are acceptable for supporting a future marketing application if successful.
If approved, PS could become the first treatment available to improve low light vision challenges, such as glare or halos, experienced by patients following surgeries like LASIK. The trial plans to enroll 200 subjects and will measure improvements in low contrast distance vision after 15 days of use.
Read Announcement
RYZUMVI FDA Regulatory Events
RYZUMVI is a drug developed by Ocuphire Pharma for the following indication: For the treatment of pharmacologically-induced mydriasis.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- RYZUMVI
- Announced Date:
- September 30, 2024
- Indication:
- For the treatment of pharmacologically-induced mydriasis
Announcement
Ocuphire Pharma, Inc announced the publication of full results from two pivotal Phase 3 clinical trials (MIRA-2 and MIRA-3) that evaluated the safety and efficacy of RYZUMVI™ (Phentolamine Ophthalmic Solution 0.75%) for the treatment of pharmacologically-induced mydriasis in the peer-reviewed journal Ophthalmology.
AI Summary
Ocuphire Pharma, Inc. has released the full results from two pivotal Phase 3 clinical trials, MIRA-2 and MIRA-3, published in the journal Ophthalmology. These studies evaluated RYZUMVI™ (Phentolamine Ophthalmic Solution 0.75%) for reversing pharmacologically-induced mydriasis. The trials showed that a significantly higher percentage of subjects treated with RYZUMVI achieved a return to near-baseline pupil size at 90 minutes compared to those given a placebo. Notably, improvements were seen as early as 60 minutes and continued through 24 hours, with fewer subjects experiencing residual dilation. The treatment was well tolerated, with only mild and temporary side effects reported. These findings underscore the potential of RYZUMVI to provide fast and effective relief from prolonged pupil dilation, thereby enhancing patient comfort during and after routine eye examinations.
Read Announcement
APX3330 (ZETA-1) FDA Regulatory Timeline and Events
APX3330 (ZETA-1) is a drug developed by Ocuphire Pharma for the following indication: Diabetic Retinopathy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- APX3330 (ZETA-1)
- Announced Date:
- July 10, 2024
- Indication:
- Diabetic Retinopathy
Announcement
Ocuphire Pharma, Inc. announced it will participate in the Association for Research in Vision and Ophthalmology Special Interest Group (ARVO SIG) panel and the American Society of Retina Specialists (ASRS) 42nd Annual Scientific Meeting, to highlight the potential of its lead oral candidate in development for diabetic retinopathy (DR).
AI Summary
Ocuphire Pharma, Inc. announced it will take part in two important events to show the promise of its lead oral candidate for treating diabetic retinopathy (DR). The company’s CEO, Dr. George Magrath, will join a panel discussion at the Association for Research in Vision and Ophthalmology Special Interest Group (ARVO SIG). During this virtual session on July 11 at 6 p.m. ET, experts will talk about the benefits of oral medications in managing retinal diseases and how these new therapies could help solve treatment challenges found with injections.
At the same time, the American Society of Retina Specialists (ASRS) 42nd Annual Scientific Meeting will feature a presentation on a subset analysis of Ocuphire’s ZETA-1 Phase 2 clinical trial. Dr. Kareem Sioufi, along with a team of experts, will discuss how the oral candidate, APX3330, could slow the progression of DR. These presentations highlight the potential of oral APX3330 to provide an earlier, non-invasive treatment option for diabetic retinopathy.
Read Announcement- Drug:
- APX3330 (ZETA-1)
- Announced Date:
- June 6, 2024
- Indication:
- Diabetic Retinopathy
Announcement
Ocuphire Pharma, Inc announced that clinical updates on the Company's lead candidate APX3330 for diabetic retinopathy (DR) will be featured in upcoming presentations at the Clinical Trials at the Summit meeting on June 8 in Park City, Utah, and the Retinal Imaging Biomarkers & Endpoints Summit meeting June 25-27 in Boston.
AI Summary
Ocuphire Pharma, Inc. announced that it will present new clinical updates on its lead candidate, APX3330, which is being developed for diabetic retinopathy. APX3330 is an oral small molecule that targets Ref-1, a protein involved in disease pathways for non-proliferative diabetic retinopathy (NPDR), a condition affecting many diabetic patients. The clinical update titled “Clinical Update on Oral APX3330 for Diabetic Retinopathy” will be presented by Dr. Veeral Sheth at the Clinical Trials at the Summit meeting on June 8 in Park City, Utah. Additionally, Ocuphire’s Chief Scientific and Development Officer, Dr. Ashwath Jayagopal, will present “Multiplex Analysis of Clinical Imaging & Biomarker Data to Validate Novel Endpoints for Diabetic Retinopathy” at the Retinal Imaging Biomarkers & Endpoints Summit held June 25-27 in Boston.
Read Announcement- Drug:
- APX3330 (ZETA-1)
- Announced Date:
- May 6, 2024
- Indication:
- Diabetic Retinopathy
Announcement
Ocuphire Pharma, Inc announced that clinical data from its ZETA-1 trial evaluating APX3330 in diabetic retinopathy (DR) on a validated binocular person-level scale was presented yesterday at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, taking place May 5-9, 2024 in Seattle, Washington.
AI Summary
Ocuphire Pharma recently presented clinical data from its ZETA-1 trial at the ARVO Annual Meeting in Seattle. The trial evaluated the efficacy of APX3330 in slowing diabetic retinopathy (DR) progression using a validated binocular person-level scale, which assesses DR severity based on the condition of both eyes. In a subset analysis of 68 participants with moderate to severe non-proliferative DR, the results showed promising outcomes. Notably, none of the patients receiving APX3330 experienced a significant worsening (≥4-step change) on the scale at 24 weeks, compared to 15.2% in the placebo group. These findings suggest that APX3330 may offer significant potential as an oral treatment option to delay or prevent the progression of DR in patients at high risk for developing more severe, vision-threatening stages of the disease.
Read Announcement- Drug:
- APX3330 (ZETA-1)
- Announced Date:
- April 22, 2024
- Indication:
- Diabetic Retinopathy
Announcement
Ocuphire Pharma, Inc announced that Daniel Su, M.D. will deliver a paper presentation on oral APX3330 at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting to be held May 5-9, 2024 in Seattle, Washington.
AI Summary
Ocuphire Pharma, Inc. announced that Dr. Daniel Su will present a paper on oral APX3330 at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, scheduled for May 5-9, 2024, in Seattle, Washington. The presentation, titled “Oral APX3330, a Ref-1 Inhibitor, Slows Progression of Diabetic Retinopathy on a Binocular DRSS Person-Level Scale,” will be given on Sunday, May 5, 2024, from 4:00 to 4:15 pm PT at the Arch Building in Seattle Convention Center. This session is part of the diabetic retinopathy track. The study highlights the potential of APX3330 as a treatment option by targeting the Ref-1 protein, potentially slowing down disease progression in diabetic retinopathy patients. This presentation underscores Ocuphire’s commitment to advancing novel therapies for retinal disorders and broadening the options for patients with diabetic eye disease.
Read Announcement
Free OCUP Stock Alerts