RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-MASKED STUDY OF THE SAFETY AND EFFICACY OF POS (0.75% PHENTOLAMINE OPHTHALMIC SOLUTION) IN PARTICIPANTS WITH PREVIOUS KERATOREFRACTIVE SURGERY WITH DECREASED VISUAL ACUITY UNDER MESOPIC CONDITIONS

The goal of this clinical trial is to learn if Phentolamine Ophthalmic Solution works to treat adults that have had keratorefractive surgery and have decreased visual acuity under mesopic conditions. It will also learn about the safety of Phentolamine Ophthalmic Solution. The main questions it aims to answer are:
Researchers will compare Phentolamine Ophthalmic Solution to a placebo (a look-alike substance that contains no drug) to see if Phentolamine Ophthalmic Solution works to improve vision in low light conditions.
Participants will:
Take Phentolamine Ophthalmic Solution or a placebo drop every day for 2 weeks Visit the clinic once every week for 2 weeks for checkups and tests Keep a diary of when they instill the study medication each evening

Start Date02 Sep 2025

Sponsor / Collaborator

Opus Genetics, Inc.

NCT06787066

/ RecruitingEarly Phase 1IIT

Sympathetic Vascular Transduction Across the Menopause Transition: Contributing Mechanisms

The investigators aim to assess the interactions between the sympathetic nervous system and the vasculature in women across the menopause transition to better understand why aging women have a higher risk of cardiovascular disease.

Start Date10 Jul 2025

Sponsor / Collaborator

University of Missouri

NCT05448807

/ RecruitingPhase 3IIT

A Randomized Effectiveness of Phentolamine Mesylate as a Reversing Agent for Local Anesthesia in Decreasing Self- Inflicted Soft Tissue Trauma Following Local Anaesthesia Injection for Dental Procedure in Children: Randomized Clinical Trial

Local anaesthesia is considered one of the most alarming and non-comfortable dental procedures for children. This goes back to the persisting effect of the local anaesthesia after finishing dental procedures. The effect of Local anaesthesia can last from 3 to 5 hours due to the presence of vasoconstrictor. The U.S Food and Drug Administration (FDA approved the use of phentolamine mesylate (Oraverse) in May 2008 to be used as a drug in reversing the numbing effect of Local anaesthesia by decreasing the time needed to restore the normal functions of the mouth

Start Date05 Jul 2025

Sponsor / Collaborator

Cairo University

100 Clinical Results associated with Phentolamine Mesylate

100 Translational Medicine associated with Phentolamine Mesylate

100 Patents (Medical) associated with Phentolamine Mesylate

11,901

Literatures (Medical) associated with Phentolamine Mesylate

01 Jan 2026·MICROBIAL PATHOGENESIS

A QseBC-like system is involved in motility and biofilm formation responses to catecholamines in Pseudomonas aeruginosa PAO1

Article

Author: André, Gwenaëlle ; Boukerb, Amine M ; Connil, Nathalie ; Jeannot, Katy ; Boujnane, Meryem

Catecholamines hormones, including epinephrine (Epi), norepinephrine (NE), and dopamine (Dopa), are known to enhance Pseudomonas aeruginosa PAO1 growth, motility, and biofilm formation. However, the bacterial sensing mechanisms underlying these responses remain poorly understood. In this study, adrenergic and dopaminergic inhibitors (phentolamine, propranolol and chlorpromazine) were used to investigate how P. aeruginosa PAO1 recognizes host-derived catecholamines. We found that phentolamine and propranolol inhibited Epi/NE-induced bacterial growth, motility and biofilm formation. In contrast, chlorpromazine selectively abolished Dopa-induced biofilm formation. These results suggest the involvement of at least one putative adrenergic/dopaminergic-like sensor in P. aeruginosa PAO1. Bioinformatics analysis identified PmrB, a QseC-like histidine sensor kinase in PmrAB two-component system (TCS), as a plausible candidate. Molecular docking using an AlphaFold3 model of PmrB, showed that catecholamines can bind to a cavity at the dimer interface. A ΔpmrAB mutant failed to respond to catecholamine exposure in motility and biofilm experiments, supporting a direct role of the PmrAB TCS in mediating PAO1 responses to host-derived catecholamines.

01 Nov 2025·POULTRY SCIENCE

Effects of pectic oligosaccharide on growth performance, organ indexes and intestinal health in broilers exposed to aflatoxin B1

Article

Author: Ye, Hui ; Zhang, Changming ; Wang, Weiwei ; Qin, Yongzhi ; Zuo, Jianjun ; Dong, Zemin ; Ye, Zhengqi ; Zhang, Haijun ; Cao, Qingyun ; Xian, Meitian

This study aimed at investigating the influences of pectic oligosaccharide (POS) on growth performance, organ indexes and intestinal health in broilers exposed to aflatoxin B₁ (AFB₁). In vitro experiment was conducted to assess reactive oxygen species (ROS)-scavenging ability of POS. In vivo experiment was then implemented by allocating 320 one-day-old yellow-feathered broilers to 4 groups (8 replicates/group), according to a 2 × 2 factorial arrangement with POS addition (0 or 400 mg/kg) and AFB₁ exposure (with or without) as the two factors. All parameters were determined on d 21. The results revealed an in vitro ability of POS to scavenge ROS. AFB₁ exposure tended to decrease (P < 0.10) average daily gain (ADG) and average daily feed intake (ADFI), as well as caused abnormities in liver, thymus and bursal indexes. It also elevated (P < 0.05) intestinal ROS and malondialdehyde contents but reduced (P < 0.05) intestinal catalase and glutathione peroxidase activities, as well as caused intestinal injuries including the decreasing trends (P < 0.10) of ileal villus height:crypt depth ratio and the relative mRNA expression of claudin-1 together with the increasing trend (P < 0.10) of serum diamine oxidase (DAO) activity. POS addition tended to elevate (P < 0.10) ADG and ADFI, as well as mitigated AFB₁-induced elevation (P < 0.10) in spleen index and reduction (P < 0.05) of bursal index. Moreover, POS addition tended to enhance (P < 0.10) ileal total antioxidant capacity in AFB₁-exposed broilers. Besides, POS addition lowered (P < 0.05) ROS level and increased (P < 0.05) glutathione content in ileum, as well as showed a tendency (P < 0.10) to elevate ileal claudin-1 expression and decrease serum DAO activity of broilers regardless of AFB₁ exposure. Collectively, supplemental POS benefited growth performance and organ indexes of broilers exposed to AFB₁, which could attribute to its ability to ameliorate intestinal redox state and integrity. These findings underscore a potential of POS to attenuate the detriments of AFB₁ contamination in diets.

01 Nov 2025·INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES

Degradation of pectin with probiotic candidate Bacillus subtilis HA1 to oligosaccharides with enhanced anticancer and antioxidant properties

Article

Author: Taherzadeh, Mohammad J. ; Hosseini-Abari, Afrouzossadat ; Amini-Rourani, Hamed

Upcycling fruit waste into health-promoting ingredients is an urgent sustainability challenge. In this work, a microbial degradation is described that converts apple pectin into bioactive pectic oligosaccharides (POS) using Bacillus subtilis HA1, a strain isolated from traditional yogurt. HA1 is γ-hemolytic, lecithinase-negative, and free of nhe/hbl enterotoxin genes, yet endures acid/bile and adheres to intestinal cells, confirming its safety and probiotic aptitude. The bacterium cleaves pectin within 6 h of incubation, and under optimum conditions (50 °C, pH 6), the extracellular pectinase showed a maximum activity of ≈18 IU/mL. TLC, LC-ESI-MS, and FTIR verify the formation of low-methylated mono- to tri-galacturonic acids. Crude POS scavenge up to 90 % of DPPH radicals at 10 mg/mL, which is five-fold higher than untreated pectin. POS also act selectively against tumor cells: MCF-7 breast cancer viability drops to 17 %, while healthy L-929 and HUVEC cells remain ≥95 % viable. Flow cytometry and qRT-PCR confirm apoptosis via Bax up-regulation and galectin-3 suppression. Altogether, probiotic candidate strains belonging to the B. subtilis afford a safe, eco-friendly route to high-value POS with potent antioxidant and anticancer activities, opening avenues for functional foods, nutraceuticals, and sustainable pectin valorization.

166

News (Medical) associated with Phentolamine Mesylate

08 Jan 2026

·www.biospace.com

Opus Genetics Highlights 2025 Progress and Upcoming 2026 Catalysts

- Multi-asset pipeline with significant targeted data readouts and milestones - - Two lead ophthalmic gene therapy programs in clinical trials with new data anticipated in 2026 - - Supplemental New Drug Application (sNDA) submitted for partnered presbyopia treatment - - Corporate presentation at J.P. Morgan Healthcare Conference on Thursday, January 15, at 8:15 a.m. PT - RESEARCH TRIANGLE PARK, N.C., Jan. 08, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc . (Nasdaq: IRD) (the “Company,” “Opus,” or “Opus Genetics”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today highlighted its progress achieved during 2025 and upcoming catalysts in 2026. In 2026, Opus is expected to announce clinical data from its BEST1 program and accelerate its LCA5 program into pivotal testing and toward potential U.S. Food and Drug Administration (FDA) approval. Additional programs are also expected to advance into the clinic. Most of Opus’ pipeline programs have the potential to qualify for Rare Pediatric Disease designation, which could result in Priority Review Vouchers (PRV), providing future non-dilutive capital. “2025 was a year defined by strong execution at Opus,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “We achieved aggressive milestones across multiple ophthalmic programs – advancing two gene therapies in clinical trials and submitting an application to the FDA for approval of our second partnered commercial product, which could potentially provide a large new market opportunity. We secured capital from leading institutional investors and through non-dilutive sources such as patient advocacy grant funding. We believe that the progress we’ve made with our LCA5 and BEST1 programs is proof of what’s possible and our broader pipeline is poised to follow. We enter 2026 with confidence, momentum, and an unwavering commitment to patients who inspire everything we do.” Corporate Updates OPGx-BEST1 – Gene Therapy for BEST1-Related IRD Potential treatment for both the dominant and recessive forms of BEST1 disease. First participant dosed in Phase 1/2 trial (BIRD-1) with enrollment ongoing. Initial data expected this quarter at Macula Society with 3-month results from the entire Cohort 1 expected in mid-2026. OPGx-BEST1 is potentially eligible for multiple regulatory designations which the Company expects to in 2026. OPGx-LCA5 – Gene Therapy for Leber Congenital Amaurosis (LCA) Multiple regulatory designations granted including Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy (RMAT) with potential eligibility for Priority Review Voucher upon approval. Positive Phase 1/2 safety and efficacy data reported in adults and pediatric participants. Enrollment ongoing in run-in period for planned, adaptive pivotal Phase 3 trial. Dosing with OPGx-LCA5 in the Phase 3 trial expected in the second half of 2026. Pre-Clinical Gene Therapy Pipeline Multiple partnerships with patient advocacy organizations are increasing awareness in the IRD community and providing non-dilutive funding for pre-clinical testing of Opus Genetics gene therapy pipeline. Promising programs for IRDs related to genetic mutations in RHO, CNGB1, MERTK, RDH12-LCA, and NMNAT1 with one to two programs targeted to enter clinical testing this year. Phentolamine Ophthalmic Solution 0.75% (PS) Supplemental New Drug Application (sNDA) submitted for the treatment of presbyopia, with an anticipated regulatory decision by the end of 2026. LYNX-3, the second pivotal Phase 3 trial in keratorefractive participants with visual disturbances under mesopic, low-contrast conditions, is ongoing with topline results expected in the first half of 2026. Financial Outlook As of September 30, 2025, Opus Genetics had cash and cash equivalents of $30.8 million. Subsequent to the end of the third quarter of 2025, the Company raised approximately $23.0 million in gross proceeds through a registered direct offering of equity securities, resulting in a total cash position of over $50 million. Based on current operating plans, the Company expects its existing cash resources will fund operations into the second half of 2027, excluding any potential proceeds from callable warrants or future milestone payments. About OPGx-BEST1 and the Phase 1/2 Trial OPGx-BEST1 leverages Opus Genetics’ proprietary AAV-based gene therapy platform, designed to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium (RPE) cells where the defective gene resides. The program builds on extensive preclinical work demonstrating restoration of BEST1 protein expression and improved retinal function in relevant disease models. The multi-center, adaptive, open-label, dose-exploring Phase 1/2 trial, known as BIRD-1, will evaluate the safety, tolerability, and preliminary efficacy of OPGx-BEST1 in participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB). Treatment will be administered via a single subretinal injection in one eye of each participant with two dosing cohorts. The trial will also explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure. About OPGx-LCA5 OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being advanced into a pivotal Phase 3 trial. Data from pediatric participants demonstrated large gains in cone-mediated vision, and the therapy remains well tolerated with no ocular serious adverse events or dose-limiting toxicities. The adult cohort showed durable improvements in cone sensitivity and visual function out to 18 months. OPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. 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 RHO, CNGB1, RDH12, NMNAT1, and MERTK. Opus Genetics is also advancing Phentolamine Ophthalmic Solution 0.75%, an approved small-molecule therapy for pharmacologically induced mydriasis, with additional potential indications in presbyopia and low-light visual disturbances following keratorefractive surgery. The Company is based in Research Triangle Park, NC. For more information, visit . Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements related to cash runway, the clinical development, clinical results, preclinical data, and future plans for Phentolamine Ophthalmic Solution 0.75%, OPGx-LCA5, OPGx-BEST1, RDH12, and earlier stage programs, and expectations regarding us, our business prospects, and our results of operations and 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, those described under the heading “Risk Factors” included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024, our subsequent Quarterly Report on Form 10-Q, and our 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 our 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. 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. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise. 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.

Phase 3Gene TherapyOrphan DrugBreakthrough TherapyPriority Review

09 Dec 2025

·www.globenewswire.com

Opus Genetics Announces Positive Recommendation from Independent Data Monitoring Committee for Phase 1/2 Trial in Best Disease

Initial favorable safety profile demonstrated in OPGx-BEST1 clinical trialRESEARCH TRIANGLE PARK, N.C., Dec. 09, 2025 (GLOBE NEWSWIRE) -- Opus Genetics (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), today announced that the Independent Data Monitoring Committee (IDMC) issued a positive recommendation to continue as planned in the Company’s Phase 1/2 BEST1 clinical trial (BIRD-1), which is a multi-center, adaptive, open-label, dose-exploring study evaluating OPGx-BEST1 in participants with Best disease. The IDMC overseeing the trial completed its pre-specified safety review of the one-month data from the sentinel participant and recommended advancing enrollment and dosing of additional participants in the trial, without modification. “We are thrilled with this outcome from the first participant, whose encouraging safety results at one month enable us to proceed with dosing the next four participants in our BEST1 Phase 1/2 trial,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “The IDMC’s safety review and recommendation to continue the trial reinforces our confidence as we advance this program. BEST1-associated retinal diseases constitute a substantial unmet medical need, and this progress with OPGx-BEST1 represents an important step toward potentially preserving and restoring visual function for patients with Best disease.” About OPGx-BEST1 and the Phase 1/2 Trial OPGx-BEST1 leverages Opus Genetics’ proprietary AAV-based gene therapy platform, designed to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium (RPE) cells where the defective gene resides. The program builds on extensive preclinical work demonstrating restoration of BEST1 protein expression and improved retinal function in relevant disease models. The multi-center, adaptive, open-label, dose-exploring study, known as BIRD-1, will evaluate the safety, tolerability, and preliminary efficacy of OPGx-BEST1 in participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB). Treatment will be administered via a single subretinal injection in one eye of each participant with two dosing cohorts. The trial will also explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure. About BEST1 Inherited Retinal Disease Best disease, or vitelliform macular dystrophy, is a rare inherited retinal condition caused by mutations in the BEST1 gene, leading to impaired retinal pigment epithelium (RPE) function, progressive vision loss, and, in some cases, blindness. The BEST1 gene is responsible for providing instructions to produce bestrophin, a protein that acts as a channel to manage the movement of charged chloride ions in and out of retinal cells. Variants (mutations) in the BEST1 gene, as well as the PRPH2 gene, can result in the formation of abnormally shaped channels that cannot properly control chloride flow. BEST1 plays a key role in the RPE, which is essential for healthy vision, and such mutations can lead to BEST1-related inherited retinal diseases (bestrophinopathies). These rare conditions affect an estimated 9,000 patients across the United States and can lead to progressive vision loss and blindness. 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 RHO, RDH12, and MERTK. Opus Genetics is also advancing Phentolamine Ophthalmic Solution 0.75%, an approved small-molecule therapy for pharmacologically induced mydriasis, with additional indications in late-stage development for presbyopia and low-light visual disturbances following keratorefractive surgery. The Company is based in Research Triangle Park, NC. For more information, please visit www.opusgtx.com. Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements related to the clinical development, clinical results, preclinical data, and future plans for Phentolamine Ophthalmic Solution 0.75%, OPGx-LCA5, OPGx-BEST1, RDH12, and earlier stage programs, and expectations regarding us, our business prospects, and our results of operations and 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, those described under the heading “Risk Factors” included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our 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 our 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. 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. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise. Contacts InvestorsJenny KobinRemy BernardaIR Advisory Solutionsir@opusgtx.com MediaKimberly HaKKH Advisors917-291-5744kimberly.ha@kkhadvisors.com Source: Opus Genetics, Inc.

Gene TherapyClinical StudyClinical Result

15 Nov 2025

·www.globenewswire.com

Opus Genetics Announces Dosing of First Participant in OPGx-BEST1 Phase 1/2 Gene Therapy Clinical Trial for Best Disease

Emerging gene therapy administered through a one-time subretinal injection marks a major milestone for the inherited retinal disease community Initial data expected in Q1 2026 Nov. 13, 2025 -- Opus Genetics, (Nasdaq: IRD) (the “Company” or “Opus Genetics”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced that the first participant has been dosed in the Company’s OPGx-BEST1 Phase 1/2 clinical trial for Best disease (BEST1). Best disease, or vitelliform macular dystrophy, is a rare, inherited retinal condition causing macular degeneration by mutations in the BEST1 gene, leading to progressive vision loss and, in some cases, blindness. OPGx-BEST1 is an emerging gene therapy administered through a one-time subretinal injection designed to restore function to the retinal pigment epithelium (RPE) cells affected by mutations in the BEST1 gene. The Phase 1/2 trial is being conducted by Dr. Mark Pennesi of the Retina Foundation of the Southwest and the surgical team of Drs. Kenneth Fan and Charles Wykoff at Retina Consultants of Texas. “Dosing the first participant in our OPGx-BEST1 program is a historic moment for the BEST disease community and for our team at Opus,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “This milestone reinforces our mission to develop one-time gene therapies for inherited retinal diseases that previously had no treatment options. It’s a privilege to collaborate with leaders in the field like Drs. Pennesi, Fan, and Wykoff. We are deeply thankful to the Retina Foundation of the Southwest, Retina Consultants of Texas, and most importantly, the patients and families who place their trust in us. Together, we are striving to build a future where no one loses their sight to inherited retinal disease.” “This milestone highlights the significant progress being made in ophthalmic gene therapy and the potential power of collaboration between industry and academia to bring new hope to families affected by inherited retinal diseases,” said Dr. Mark Pennesi, Director of the Inherited Retinal Degeneration Clinic at the Retina Foundation of the Southwest. “For patients with Best disease, this represents an important step toward potentially preserving and restoring vision.” “The successful dosing of the first patient underscores both the potential promise of gene therapy in ophthalmology and the dedication of the entire community working to make these treatments a reality,” said Dr. Charles Wykoff, Surgical Retina Specialist and Ophthalmologist at the Retina Consultants of Texas. “It’s inspiring to see years of scientific progress translate into potentially meaningful advances for patients and families.” OPGx-BEST1 leverages Opus Genetics’ proprietary AAV-based gene therapy platform, designed to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium (RPE) cells where the defective gene resides. The program builds on extensive preclinical work demonstrating restoration of BEST1 protein expression and improved retinal function in relevant disease models. The multi-center, adaptive, open-label, dose-exploring study, known as BIRD-1, will evaluate the safety, tolerability, and preliminary efficacy of OPGx-BEST1 in participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB). Treatment will be administered via a single subretinal injection in one eye of each participant with two dosing cohorts. The trial will also explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure. Initial data from the trial is expected in the first quarter of 2026. The BEST1 gene is responsible for providing instructions to produce bestrophin, a protein that acts as a channel to manage the movement of charged chloride ions in and out of retinal cells. Variants (mutations) in the BEST1 gene, as well as the PRPH2 gene, can result in the formation of abnormally shaped channels that cannot properly control chloride flow. BEST1 plays a key role in the retinal pigment epithelium (RPE), which is essential for healthy vision, and such mutations can lead to BEST1-related inherited retinal diseases (bestrophinopathies). These rare conditions affect an estimated 9,000 patients across the United States and can lead to progressive vision loss and blindness. 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 RHO, RDH12, and MERTK. Opus Genetics is also advancing Phentolamine Ophthalmic Solution 0.75%, an approved small-molecule therapy for pharmacologically induced mydriasis, with additional indications in late-stage development for presbyopia and low-light visual disturbances following keratorefractive surgery. The Company is based in Research Triangle Park, NC. The content above comes from the network. if any infringement, please contact us to modify.

Gene TherapyClinical Study

100 Deals associated with Phentolamine Mesylate

External Link

KEGG	Wiki	ATC	Drug Bank
D00509	Phentolamine Mesylate	V03AB36 C04AB01	DB00692

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Mydriasis	United States	Famy Life Sciences Pvt Ltd.	25 Sep 2023
Erectile Dysfunction	China	Shanghai Fudan Forward Pharmaceutical Co. Ltd.	01 Jan 1989
Anesthesia	United States	Novartis Pharmaceuticals Corp.	30 Jan 1952

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Night Blindness	Phase 3	United States	Opus Genetics, Inc.	27 Apr 2023
Presbyopia	Phase 3	United States	Opus Genetics, Inc.	22 Dec 2022
Vision, Low	Phase 3	United States	Opus Genetics, Inc.	30 Dec 2020
Glaucoma, Open-Angle	Phase 2	United States	Opus Genetics, Inc.	28 May 2019
Blindness	Phase 2	United States	Opus Genetics, Inc.	01 Aug 2007

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06542497 (VEGA-3, Company_Website) Manual	Phase 3	Presbyopia	545	Phentolamine Ophthalmic Solution 0.75%	fmfidjvjik(fqqqkxsuoh) = pqcjqqaboc jgeyhqgjde (sxbuxvwyib ) Met View more	Positive	26 Jun 2025
				Placebo	fmfidjvjik(fqqqkxsuoh) = qffnjkguyz jgeyhqgjde (sxbuxvwyib ) Met View more
NCT05646719 (VEGA-2, CTgov)	Phase 3	Presbyopia	333	Phentolamine Opthalmic Solution 0.75%+Low dose pilocarpine (Nyxol + Low Dose Pilocarpine)	opvnhbtbir = yesdhgsgre rcqsmvdshe (teexeqdztq, oskzygbhei - puhlkihgid) View more	-	03 Jun 2025
				Low dose pilocarpine vehicle+Phentolamine Opthalmic Solution 0.75% (Nyxol + Low Dose Pilocarpine Vehicle)	opvnhbtbir = mvifoxchny rcqsmvdshe (teexeqdztq, kogfqpxmka - cmhxuhbwkf) View more
NCT04620213 \| NCT05134974 (MIRA-3, Literature) Manual	Phase 3	Mydriasis	553	phentolamine	fpahqbwsmu(bmvztaaqwz) = wvfylranew dbmnhomzhd (aqqbuftogu )	Positive	16 Sep 2024
				placebo	fpahqbwsmu(bmvztaaqwz) = ddxmnoboth dbmnhomzhd (aqqbuftogu )
NCT04004507 (SNV, CTgov)	Phase 2	Night Blindness \| Blindness	24	Phentolamine Mesylate Ophthalmic Solution 1% (Phentolamine Mesylate Ophthalmic Solution 1%)	fbbxfqksur(qnzeruxbpw) = vyueuxcvuy taudqmlgpw (imubsbnqln, 1.8) View more	-	15 Feb 2024
				Phentolamine Mesylate Ophthalmic Solution Vehicle (Placebo) (Phentolamine Mesylate Ophthalmic Solution Vehicle)	fbbxfqksur(qnzeruxbpw) = zlywcsxmdx taudqmlgpw (imubsbnqln, 1.5) View more
NCT04620213 \| NCT05134974 (MIRA-3, FDA) Manual	Phase 3	Mydriasis	553	Ryzumvi (MIRA-2)	oiitqzehzz(nlhntfcyjd) = ldkztdueit ekymmqlyoj (rfuiebnyxp )	Positive	25 Sep 2023
				Placebo (MIRA-2)	oiitqzehzz(nlhntfcyjd) = kmaqpspmzs ekymmqlyoj (rfuiebnyxp )
NCT04620213 (MIRA-2, CTgov)	Phase 3	Mydriasis	185	Phentolamine Ophthalmic Solution 0.75% (Phentolamine Ophthalmic Solution 0.75%)	wtflacmvpi = gtadsawzkv ndbelhgamx (fnbfkqbazh, ytcvfmeaxm - esxmhwgypf) View more	-	14 Aug 2023
				Phentolamine Ophthalmic Solution Vehicle (Placebo) (Phentolamine Ophthalmic Solution Vehicle)	wtflacmvpi = eerizrexwo ndbelhgamx (fnbfkqbazh, ocdlbnsguu - wpelsxsxbk) View more
NCT04638660 (LYNX-1, CTgov)	Phase 3	Vision, Low	144	Phentolamine Ophthalmic Solution 0.75% (Phentolamine Ophthalmic Solution 0.75%)	mcabwtcgus = sjkmqyaiki mfldgtmsdz (fcvdnggwqk, typmlitobk - eehvnvpkcv) View more	-	09 Aug 2023
				Phentolamine Ophthalmic Solution Vehicle (Placebo) (Phentolamine Ophthalmic Solution Vehicle)	mcabwtcgus = decdnfobhk mfldgtmsdz (fcvdnggwqk, bfrpakxpfe - gbwxcklray) View more
NCT05223478 (MIRA-4, CTgov)	Phase 3	Mydriasis	23	Phentolamine Ophthalmic Solution 0.75% (Phentolamine Ophthalmic Solution 0.75%)	nhfynlpphw(cycgsurgev) = gseinkxfbv ixwgrvwrfh (dplvotizgx, 0.65) View more	-	02 Aug 2023
				Phentolamine Ophthalmic Solution Vehicle (Phentolamine Ophthalmic Solution Vehicle)	nhfynlpphw(cycgsurgev) = knaluyqgbp ixwgrvwrfh (dplvotizgx, 0.58) View more
NCT04675151 (CTgov)	Phase 2	Presbyopia	150	LDP+POS (POS 0.75% First, Then LDP 0.4%)	pwosnskkgo = kggnroanha wjfyoswafd (ktlfbvtjvn, ygvputtsij - usmabqqbeu) View more	-	02 Aug 2023
				LDP+POS (POS 0.75% First, Then LDP Vehicle)	pwosnskkgo = bujqkqhgtz wjfyoswafd (ktlfbvtjvn, hzhjdcvfvj - fivqmyoojm) View more
NCT05134974 (MIRA-3, CTgov)	Phase 3	Mydriasis	368	Phentolamine Ophthalmic Solution 0.75% (Phentolamine Ophthalmic Solution 0.75%)	umwpsuhbtb(aufwycztft) = mdzpbpknth kohktwcyws (tvtczwduuy, 0.7104) View more	-	01 Aug 2023
				Phentolamine Ophthalmic Solution Vehicle (Phentolamine Ophthalmic Solution Vehicle)	umwpsuhbtb(aufwycztft) = kyhgkwndml kohktwcyws (tvtczwduuy, 0.5335) View more

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