RegulationOverseas New Drugs Urgently Needed in Clinical Settings (CN), Fast Track (US), Priority Review (AU), Breakthrough Therapy (US), Orphan Drug (US)

Gene Sequence

Sequence Code 28791467

Source: *****

Clinical Trials associated with Cenegermin-BKBJ

NCT06411145 / Active, not recruitingPhase 4

A Prospective, Multicenter Trial to Evaluate Efficacy and Safety of Cenegermin-bkbj (20 mcg/mL) Ophthalmic Solution on Corneal Thickness Via Anterior Segment Optical Coherence Tomography (AS-OCT) in Stage 3 Neurotrophic Keratitis

Primary Objective:
To assess the efficacy of cenegermin-bkbj (20 mcg/mL) ophthalmic solution on overall corneal thickness via AS-OCT in patients with stage 3 neurotrophic keratitis with respect to change from baseline at weeks 4, 8, and 16.
Secondary Objectives:
To assess the effects of cenegermin-bkbj (20 mcg/mL) ophthalmic solution on corneal stromal thickness via AS-OCT in patients with stage 3 neurotrophic keratitis with respect to change from baseline at weeks 4, 8, and 16.
To assess the effects of cenegermin-bkbj (20 mcg/mL) ophthalmic solution on corneal stromal reflectivity via AS-OCT in patients with stage 3 neurotrophic keratitis with respect to change from baseline at weeks 4, 8, and 16.
To assess the effects of cenegermin-bkbj (20 mcg/mL) ophthalmic solution on corneal sensitivity via Cochet-Bonnet aesthesiometer in the center of the lesion with respect to change from baseline at weeks 4, 8, and 16.

Start Date26 Apr 2024

Sponsor / Collaborator

Dompe Farmaceutici SpA

NCT06244316 / RecruitingPhase 2

A 4-Week, Phase II, Multicenter, Randomized, Double-Masked, Vehicle-Controlled, Parallel Group Study With 4 Weeks of Follow-Up to Evaluate Safety and Efficacy of a New Formulation of Recombinant Human Nerve Growth Factor (rhNGF) Eye Drop Solution at Two Different Concentrations in Patients With Dry Eye Disease

The purpose of this study is to evaluate the safety and efficacy of two different concentrations of the new formulation of rhNGF ophthalmic solution versus vehicle, in order to demonstrate superiority of at least one of the two concentrations over vehicle in the improvement of ocular symptoms of dry eye in participants with dry eye disease (DED). The rhNGF ophthalmic solution, or vehicle, will be administered as one drop in each eye, three times a day, for 4 weeks. Participants will attend a total of 5 study visits from screening through end of the study (Week 8), which will include eye exams and questionnaires.

Start Date22 Jan 2024

Sponsor / Collaborator

Dompe Farmaceutici SpA

NCT05552261 / RecruitingNot Applicable

A Long-term Extension Study to Evaluate the Safety and Efficacy of OXERVATE 0.002% (20 mcg/mL) Cenegermin-bkbj Ophthalmic Solution in Patients With Stage 1 Neurotrophic Keratitis Who Enrolled in the DEFENDO Study

The primary objectives are to evaluate the long-term safety and efficacy of OXERVATE™ 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution in Stage 1 Neurotrophic Keratitis (NK) patients who enrolled in the DEFENDO Study.

Start Date20 Dec 2022

Sponsor / Collaborator

Dompe Farmaceutici SpA

100 Clinical Results associated with Cenegermin-BKBJ

100 Translational Medicine associated with Cenegermin-BKBJ

100 Patents (Medical) associated with Cenegermin-BKBJ

207

Literatures (Medical) associated with Cenegermin-BKBJ

01 Apr 2024·Journal of Pediatric Surgery

Superiority of Intestinal Adaptation by Hepatocyte Growth Factor in the Jejunum: An Experimental Study in a Short-Bowel Rat Model

Article

Author: Harumatsu, Toshio ; Muto, Mitsuru ; Kawano, Takafumi ; Onishi, Shun ; Iwamoto, Yumiko ; Sugita, Koshiro ; Kumagai, Kotaro ; Masakazu, Murakami ; Ido, Akio ; Yano, Keisuke ; Ieiri, Satoshi ; Kedoin, Chihiro ; Takada, Lynne ; Kaji, Tatsuru ; Ogata, Masato

BACKGROUNDWe evaluated the effect of recombinant human hepatocyte growth factor (rh-HGF) on intestinal adaptation in a rat model of short-bowel syndrome (SBS).METHODSSprague-Dawley rats underwent jugular vein catheterization for continuous total parenteral nutrition (TPN) and 90 % small bowel resection. The animals were divided into 3 groups: TPN/SBS (control group, n = 7), TPN/SBS/intravenous recombinant human hepatocyte growth factor (HGF) (0.3 mg/kg/day) (HGF group, n = 7), and TPN/SBS/intravenous c-Met inhibitor (0.3 mg/kg/day) (anti-HGF group, n = 5). On day 7, rats were euthanized and histologically evaluated. Serum diamine oxidase (S-DAO) levels were evaluated using an enzyme-linked immunosorbent assay. The nutrient transporter and glucagon-like peptide-2 (GLP-2) receptor expression were evaluated using real-time polymerase chain reaction.RESULTSThe jejunal and ileal villus heights were higher and the S-DAO concentrations significantly higher (p = 0.04) in the HGF group than in the control and anti-HGF groups. The sodium-dependent glucose transporter 1 expression in the HGF group was significantly higher than in the control group and significantly suppressed in the anti-HGF group (p < 0.01). The peptide transporter 1 expression in the jejunum was higher in the HGF group than in the other groups and significantly suppressed in the anti-HGF group (p < 0.01). The GLP-2 receptor expression in the jejunum was higher in the HGF group than the other groups, and it was significantly suppressed in the anti-HGF group (p < 0.01). These jejunal results regarding nutrient transporter an GLP-2 receptor were not found in the ileum.CONCLUSIONSThe administration of rh-HGF appears to be more effective in the jejunum than in the ileum.TYPE OF STUDYExperimental Research.LEVEL OF EVIDENCEN/A.

03 Mar 2024·Expert Opinion on Drug Safety

Ocular adverse events of cenegermin used in neurotrophic keratopathy: an analysis of the FDA adverse event reporting system database

Article

Author: He, Weimin ; Li, Yunfei ; Yang, Haiyun ; Gao, Yuan

BACKGROUNDCenegermin, a recombinant human nerve growth factor, is an orphan drug approved for neurotrophic keratitis. The safety information on the label is incomplete, and the adverse reactions noted are mostly mild and tolerable. However, the occurrence of painful epithelial plagues and irreversible corneal deposits after cenegermin usage have been reported. Real-world data on long-term ocular safety are lacking. We aimed to assess the cenegermin-associated eye safety profile in the FDA pharmacovigilance database.METHODSThe signals of cenegermin-related ocular adverse events (AEs) from 2018 to 2022 were quantified using the reporting odds ratio (ROR) and information component (IC). The grading system was used to prioritize the signals.RESULTSWe identified 3288 cases of cenegermin-related ocular AEs and 56 positive ocular-related signals. Fifty unexpected signals of ocular AE were identified. Eye ulcer was classified as a designated medical event. Twenty AEs, including corneal perforation, eye infection, corneal deposits, and eye inflammation, were recognized as important medical event. The median onset time for ocular AEs was 6 days (interquartile range [IQR]: 1-29 days).CONCLUSIONThis study revealed new cenegermin-related ocular AE signals. Clinical practice requires close monitoring to early identify and manage adverse reactions that may cause occurrence of serious irreversible consequences.

01 Feb 2024·Ophthalmology and Therapy

Phase IV Multicenter, Prospective, Open-Label Clinical Trial of Cenegermin (rhNGF) for Stage 1 Neurotrophic Keratopathy (DEFENDO)

Article

Author: Schanzlin, David ; Mantelli, Flavio ; Massaro-Giordano, Mina ; Goisis, Giovanni ; Berdy, Gregg ; Pasedis, Georgea ; Hamrah, Pedram ; Holland, Edward

INTRODUCTIONCenegermin is approved for treatment of neurotrophic keratopathy (NK) and has been studied in patients with stage 2 or 3 NK. This study evaluated the efficacy and safety of cenegermin in adults with stage 1 NK.METHODSThis was a phase IV, multicenter, prospective, open-label, uncontrolled trial. Adults with stage 1 NK (Mackie criteria) and decreased corneal sensitivity (≤ 4 cm) received 1 drop of cenegermin 20 mcg/ml in the affected eye(s) 6 times/day for 8 weeks with a 24-week follow-up.RESULTSOf 37 patients, corneal epithelial healing was observed in 84.8% (95% confidence interval [CI] 68.1-94.9%; P < 0.001) at week 8; 95.2% (95% CI 76.2-99.9%; P < 0.001) of those patients remained healed at the end of the 24-week follow-up (week 32). At week 8, 91.2% (95% CI 76.3-98.1%; P < 0.001) of patients experienced improved corneal sensitivity; this improvement was observed in 82.1% (95% CI 63.1-93.9%; P < 0.001) of patients at week 32. Mean best-corrected distance visual acuity change from baseline at week 8 was - 0.10 logMAR (standard deviation [SD], 0.15; 95% CI - 0.16 to - 0.05; P < 0.001) and at week 32 was - 0.05 logMAR (SD, 0.16; 95% CI - 0.11 to 0.01; P = 0.122). At weeks 8 and 32, 15.2% (95% CI 5.1-31.9%; P < 0.001) and 10.7% (95% CI 2.3-28.2%; P < 0.001) of patients, respectively, had a 15-letter gain from baseline. At least one adverse event (AE) was reported by 73.0% and 45.7% of patients during the treatment and follow-up periods, respectively. The most common treatment-related, treatment-emergent AEs were eye pain (37.8%), blurred vision (10.8%), and eyelid pain (8.1%); these were mostly mild or moderate and were only reported during the treatment period.CONCLUSIONSThese results support the potential use of cenegermin for treating patients with stage 1 NK, and future confirmatory studies would be beneficial to elaborate on these findings.TRIAL REGISTRATIONDEFENDO; NCT04485546.

News (Medical) associated with Cenegermin-BKBJ

23 May 2023

·www.prnewswire.com

DelveInsight Evaluates a Robust Dry Eye Disease Clinical Trial Pipeline as 60+ Influential Pharma Players to Set Foot in the Domain

The prevalence of dry eye disease has been rising over the past few years, which prompts the growing demand for treatment options. The increasing prevalence of dry eye disease and the growing research and development activities to develop novel therapies to treat dry eye disease to drive the market. The companies developing the potential therapies in the last stage of development include Aldeyra Therapeutics, RegeneRx Biopharmaceuticals, Mitotech, and several others. LAS VEGAS, May 23, 2023 /PRNewswire/ -- DelveInsight's ' Dry Eye Disease Pipeline Insight – 2023 ' report provides comprehensive global coverage of pipeline dry eye disease therapies in various stages of clinical development, major pharmaceutical companies working to advance the pipeline space, and the future growth potential of the dry eye disease pipeline domain. Key Takeaways from the Dry Eye Disease Pipeline Report DelveInsight's dry eye disease pipeline report depicts a robust space with 50+ active players working to develop 60+ pipeline therapies for dry eye disease treatment. Key dry eye disease companies such as TearSolutions, Novaliq, HanAll Biopharma, Aldeyra Therapeutics, RegeneRx Biopharmaceuticals, Mitotech, Aerie Pharmaceuticals, Azura Ophthalmics, Stuart Therapeutics, Surface Ophthalmics, Glaukos Corporation, BRIM Biotechnology Inc., Dompé Farmaceutici S.p.A, Okyo Pharma Ltd, AxeroVision, Inc., Palatin Technologies, Seikagaku Corporation, Invirsa, Inc., Allysta Pharmaceutical, Taejoon Pharmaceutical Co., Ltd., Ocular Therapeutix, Inc., Dreamhawk Vision Biotech, Inc., Yuyu Pharma, Inc., Novartis Pharmaceuticals, VivaVision Biotech, Inc, Huons Co., Ltd., and others are evaluating new dry eye disease drugs to improve the treatment landscape. Promising dry eye disease pipeline therapies in various stages of development include Reproxalap, Timbetasin, Visomitin, HL036, CyclASol, Lacripep, AR15512, AZR MD 001, ST-100, SURF-100, SURF-200, GLK-301, BRM421, Oxervate, OK-101, AXR 159, AXR 270, PL9643, SI-614, INV-102, ALY688, TJO-083, OTX-CSI, OTX-DED, SHJ002, YP-P10, ECF843, VVN001, HU007 and others. In March 2023, Selagine, Inc., a spin-out company from the University of Illinois at Chicago (UIC), announced that it has entered into a research, development, and sublicense agreement with Grifols (Nasdaq: GRFS), a global leader in plasma-derived medicines, for the development and commercialization of immunoglobulin eye drops for dry eye disease.Under the agreement, Selagine will receive an upfront payment and annual collaboration fee. In addition, Grifols has committed to fund the development of immunoglobulin eye drops through FDA approval, which will be managed collaboratively by Grifols and Selagine, including the clinical, manufacturing and regulatory activities required for FDA approval for dry eye disease indication. In February 2023, Aldeyra Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for topical ocular reproxalap, a first-in-class investigational new drug candidate, for the treatment of the signs and symptoms of dry eye disease. The FDA assigned a Prescription Drug User Fee Act (PDUFA) date of November 23, 2023. The FDA noted that no potential filing review issues have been identified, and that an advisory committee meeting is not currently planned. In February 2023, Novaliq, announced its plans for filing a Marketing Authorisation Application (MAA) for CyclASol® (ciclosporin ophthalmic solution), a first-of-its-kind anti-inflammatory product for the treatment of dry eye disease (DED) in the European Union. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) determined that CyclASol® is eligible to be reviewed in a centralized procedure to receive a Union Marketing Authorisation for the European Economic Area (EEA). In January 2023, Bausch + Lomb Corporation and Novaliq GmbH, announced that Ophthalmology, the peer-reviewed journal of the American Academy of Ophthalmology, has published results from the pivotal Phase 3 trial GOBI, which is one of two pivotal Phase 3 trials for NOV03 (perfluorohexyloctane). NOV03 is being investigated to treat the signs and symptoms of dry eye disease (DED) associated with Meibomian gland dysfunction (MGD).In the study, NOV03 was well tolerated with few subjects experiencing ocular adverse events (AEs) (9.6% NOV03 group, 7.5% control group) or treatment-related ocular AEs (6.3% NOV03 group, 3.1% control group). In August 2022, Alcon and Aerie Pharmaceuticals, Inc. announced the companies have entered into a definitive merger agreement through which Alcon acquired Aerie. The transaction affirmed Alcon's commitment to the ophthalmic pharmaceutical space and is expected to add broader pharmaceutical R&D capabilities to Alcon's existing commercial expertise, maximizing the value of its diversified portfolio. Through the transaction, Alcon added the commercial products Rocklatan® (netarsudil and latanoprost ophthalmic solution) 0.02%/0.005% and Rhopressa® (netarsudil ophthalmic solution) 0.02%, as well as AR-15512, a Phase 3 product candidate for dry eye disease, and a pipeline of several clinical and preclinical ophthalmic pharmaceutical product candidates. In May 2022, Yuyu Pharmaceutical presented a poster study for YP-P10, its new dry eye syndrome treatment, during the 2022 ARVO (Association for Research in Vision and Ophthalmology) conference. YP-P10 is a new biopharmaceutical using synthetic peptides, aiming to relieve signs and symptoms of dry eye syndrome caused by inflammation by administering the treatment twice a day. According to preclinical trial results unveiled at the conference, Yuyu confirmed that the candidate had an excellent anti-inflammatory mechanism and corneal epithelial cell healing effect compared to existing drugs. Request a sample and discover the recent advances in dry eye disease drug treatment @ Dry Eye Disease Pipeline Report The dry eye disease pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage dry eye disease drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the dry eye disease clinical trial landscape. Dry Eye Disease Overview Dry eye disease is characterized as a multifactorial disease of the tears and ocular surface that causes symptoms such as pain, visual disruption, and tear film instability, as well as potential ocular surface damage. It is accompanied by increased tear osmolarity and transient ocular surface irritation. When tear production and drainage are out of balance, dry eyes can result. People with dry eyes either do not produce enough tears or have poor-quality tears. Dry eyes can occur for various reasons, including age, gender, drugs, and medical disorders. Dry eye disease symptoms include redness, stinging, itching, or burning sensations, light sensitivity, watery eyes, stringy mucus near the eye, and blurred vision. A thorough eye examination can detect dry eyes. Dry eye disease treatments seek to restore or maintain the usual number of tears in the eye in order to reduce dryness and associated discomfort and to maintain eye health. The basic approaches used to manage and treat dry eyes include adding tears with over-the-counter artificial tear solutions, conserving tears, boosting tear production, and treating the inflammation of the eyelids or eye surface that contributes to dry eyes. Find out more about drugs for dry eye disease @ New Dry Eye Disease Drugs A snapshot of the Dry Eye Disease Pipeline Drugs mentioned in the report: Learn more about the emerging dry eye disease pipeline therapies @ Dry Eye Disease Clinical Trials Dry Eye Disease Therapeutics Assessment The dry eye disease pipeline report proffers an integral view of dry eye disease emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Dry Eye Disease Pipeline Report Coverage: Global Dry Eye Disease Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Dry Eye Disease Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Dry Eye Disease Therapeutics Assessment By Route of Administration: Intra-articular, Intraocular, Intrathecal, Intravenous, Ophthalmic, Oral, Parenteral, Subcutaneous, Topical, Transdermal Dry Eye Disease Therapeutics Assessment By Molecule Type: Oligonucleotide, Peptide, Small molecule Dry Eye Disease Therapeutics Assessment By Mechanism of Action: Malondialdehyde inhibitors, Actin modulators, Amyloid inhibitors, Angiogenesis inducing agents, Apoptosis inhibitors, Cell movement activators, Collagen stimulants, Heat-shock protein modulators, MicroRNA modulators, Nerve growth factor stimulants, NF-kappa B inhibitors, Stem cell stimulants, Antioxidants, Electron transport chain complex protein modulators, Lipid modulators, Tumour necrosis factor alpha inhibitors, Eye protein modulators, TRPM8 protein stimulants, Glucocorticoid receptor agonists, Inosine monophosphate dehydrogenase inhibitors, Muscarinic receptor agonists Key Dry Eye Disease Companies: TearSolutions, Novaliq, HanAll Biopharma, Aldeyra Therapeutics, RegeneRx Biopharmaceuticals, Mitotech, Aerie Pharmaceuticals, Azura Ophthalmics, Stuart Therapeutics, Surface Ophthalmics, Glaukos Corporation, BRIM Biotechnology Inc., Dompé Farmaceutici S.p.A, Okyo Pharma Ltd, AxeroVision, Inc., Palatin Technologies, Seikagaku Corporation, Invirsa, Inc., Allysta Pharmaceutical, Taejoon Pharmaceutical Co., Ltd., Ocular Therapeutix, Inc., Dreamhawk Vision Biotech, Inc., Yuyu Pharma, Inc., Novartis Pharmaceuticals, VivaVision Biotech, Inc, Huons Co., Ltd., and others. Key Dry Eye Disease Pipeline Therapies: Reproxalap, Timbetasin, Visomitin, HL036, CyclASol, Lacripep, AR15512, AZR MD 001, ST-100, SURF-100, SURF-200, GLK-301, BRM421, Oxervate, OK-101, AXR 159, AXR 270, PL9643, SI-614, INV-102, ALY688, TJO-083, OTX-CSI, OTX-DED, SHJ002, YP-P10, ECF843, VVN001, HU007, and others. Dive deep into rich insights for new drugs for dry eye disease treatment; visit @ Dry Eye Disease Medications Table of Contents For further information on the dry eye disease pipeline therapeutics, reach out @ Dry Eye Disease Drug Treatment Related Reports Dry Eye Disease Market Dry Eye Disease Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key dry eye disease companies, including Oyster Point Pharma, Aerie Pharmaceuticals, Azura Ophthalmics, Stuart Therapeutics, Surface Ophthalmics, Glaukos Corporation, among others. Dry Eye Disease Epidemiology Forecast Dry Eye Disease Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and dry eye disease epidemiology trends. Mild Dry Eye Market Mild Dry Eye Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key mild dry eye companies, including Allergan Plc., Novartis AG., Otsuka Pharmaceutical Co. Ltd., Santen Pharmaceutical Co. Ltd., among others. Moderate Dry Eye Market Moderate Dry Eye Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key moderate dry eye companies, including Santen Pharmaceutical Co. Ltd., OASIS Medical, Alcon Inc., Bausch Health Companies Inc., AbbVie Inc., among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

Phase 3Clinical ResultAcquisition

04 May 2023

·www.biospace.com

EyeCare Partners’ Leadership and Clinical Research to be Showcased at American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting

ST. LOUIS--(BUSINESS WIRE)-- EyeCare Partners (ECP), the nation’s leading provider of clinically integrated eye care, today announced more than 20 of its physicians will be presenting the latest innovative modalities and mechanisms across a wide range of ophthalmic disciplines in 90 sessions at the American Society of Cataract and Refractive Surgery (ASCRS). The annual meeting will take place May 5-8, 2023, in San Diego. The breadth of topics being presented at ASCRS by ECP physicians emphasizes the company’s culture of innovation and continuous learning. It also underscores the possibility of a career “third pathway” which allows physicians to conduct industry-leading research while also continuing to build a clinical practice. “EyeCare Partners has a strong representation of ophthalmologists presenting research at ASCRS this year, in addition to one of our specialists, Dr. Elizabeth Yeu, serving as the incoming president of ASCRS,” said David Clark, CEO of EyeCare Partners. “We are committed to elevating eye care, and we are proud to work with many ophthalmologists who are leading educators in their field and committed to advancing eye care through innovation and clinical research.” Select ECP physician presentations at ASCRS include: “Prospective Evaluation of Eye Drop Self-Administration Accuracy in a Real-World Patient Population” – Alison Early, M.D., Cincinnati Eye Institute “Cardiovascular Effects of Immunosuppression for Ocular Surface Stem Cell Transplantation” – Albert Y. Cheung, M.D., Virginia Eye Consultants “Treatment of Corneal Neovascularization Prior to Deep Anterior Lamellar Keratoplasty” – Albert Cheung, M.D., Virginia Eye Consultants “Corneal Healing after Cenegermin Treatment in Patients with Stage 1 Neurotrophic Keratopathy” – Gregg Berdy M.D., Ophthalmology Associates “Cryopreserved Amniotic Membrane during Pterygium Excision: Health Economic Opportunity Analysis” – Neel Desai, M.D., Eye Institute of West Florida “Surgeon and Patient Experience Following Toric IOL Alignment with and without a Novel Ocular Registration-Guided Graphical Overlay System” – Robert Weinstock, M.D., Eye Institute of West Florida “Retrospective Assessment of the Routine Use of Loteprednol Etabonate Gel 0.38% Following LASIK and PRK Surgery” – Joseph Gira, M.D., Ophthalmology Associates “Interim Assessment of Safety & Efficacy of Human Corneal Endothelial Cell Therapy for Corneal Edema Secondary to Endothelial Dysfunction” – Edward Holland, M.D., Cincinnati Eye Institute “A Trifocal Lens with New Hydrophobic Acrylic Lens Material: A Study of Patient Satisfaction and Visual Performance” – Michael Jones, M.D., Quantum Vision Centers “EW-6 Be part of History in the making…The Big Bang in surgical visualization systems has arrived” – Caroline Watson, M.D., Alabama Vision Center, John Kitchens, M.D., Retina Associates of Kentucky “Axial Length Related Visual Outcomes in an Extended Depth of Focus Lens Implantation” – Rohit Krishna, M.D., Sabates Eye Centers “Sublingual Midazolam and Ketamine for Surgical Sedation, a Multicenter, Double Masked, Randomized Controlled Trial” – Michael Nordlund, M.D., Ph.D., Cincinnati Eye Institute “Visual Outcomes of the Tecnis Symfony Lens in Patients with Ocular Hypertension and Mild to Moderate Glaucoma” – Constance Okeke, M.D., Virginia Eye Consultants “IC-124 Management of Complications Encountered during Cataract Surgery” – Robert Osher, M.D., Cincinnati Eye Institute “STL-2 Iris Prothesis” – Samantha Schockman, M.D., Cincinnati Eye Institute “Real-World Outcomes of Intranasal Varenicline for the Treatment of Dry Eye Disease” – Amar Shah, M.D., Cincinnati Eye Institute “Sterile and Infectious Infiltrates after Epithelial Defect Treatment with Cryopreserved and Dehydrated Amniotic Membrane: A Case Series” – John D. Sheppard, M.D., Virginia Eye Consultants “Refractive impact of topical meds” – Kavitha Sivaraman, M.D., Cincinnati Eye Institute “IC-001 The Wonderful World of Iris Prostheses: A Case-Based Compendium” – Michael Snyder, M.D., Cincinnati Eye Institute “Eyeninja - Danger in the Dark: Introducing the Eidolon Handheld Slit Beam” – Lawrence Tenkman, M.D., John-Kenyon Eye Centers “Can't Touch That!” – Megan Tuohy, M.D., Cincinnati Eye Institute, Michael Snyder, M.D., Cincinnati Eye Institute “Effectiveness of Femtosecond Laser Assisted Cataract Surgery and Manual Phacoemulsification during the Learning Curve of Fellows in Training” – Robert Weinstock, M.D., Eye Institute of West Florida “Safety & Efficacy of Lotilaner Ophthalmic Solution, 0.25% in Treating Demodex Blepharitis: Combined Analysis of Two Pivotal Trials” – Elizabeth Yeu, M.D., Virginia Eye Consultants “CSF-1 (0.4% Pilocarpine HCl Ophthalmic Solution) for the Treatment of Presbyopia: Near-1 and Near-2 Phase 3 Clinical Trials” – Edward J. Holland, M.D., Cincinnati Eye Institute “Effects of Uncrosslinking Ocular Rigidity in Aging Eyes: Rejuvenation of Visual Age Utilizing a New Microporation Therapy” – Elizabeth Yeu, M.D., Virginia Eye Consultants “Prospective, Randomized, Contralateral Eye Study of Femtosecond Laser Assisted Cataract Surgery and Manual Phacoemulsification” – Robert Weinstock, M.D., Eye Institute of West Florida “Interim Assessment of Safety & Efficacy of Human Corneal Endothelial Cell Therapy for Corneal Edema Secondary to Endothelial Dysfunction” – Elizabeth Yeu, M.D., Virginia Eye Consultants “Efficiency of a water-free topical cyclosporine in the treatment of dry eye disease (DED) in patients with or without cataract” – John D. Sheppard, M.D., Virginia Eye Consultants “Changes in Creatinine Levels Related to Tacrolimus Used for Ocular Surface Stem Cell Transplantation” – Albert Y. Cheung, M.D., Virginia Eye Consultants About EyeCare Partners EyeCare Partners is the nation’s leading provider of clinically integrated eye care. Our national network of over 300 ophthalmologists and 700 optometrists provides a lifetime of care to our patients with a mission to enhance vision, advance eye care and improve lives. Based in St. Louis, Missouri, over 700 ECP-affiliated practice locations provide services that span the eye care continuum in 18 states and 30 markets. For more information, visit and follow us on LinkedIn.

Phase 3Cell Therapy

13 Apr 2023

·www.biospace.com

First Patient Enrolled into U.S. Phase 3 Neurotrophic Keratitis Clinical Trial with RGN-259

ROCKVILLE, Md., April 13, 2023 /PRNewswire/ -- RegeneRx Biopharmaceuticals, Inc. (OTCQB: RGRX) ("RegeneRx" or "Company"), a clinical-stage drug development company focused on tissue protection, repair, and regeneration, has announced that on April 12, the first patient of the Phase 3 clinical trial (SEER-2) of RGN-259, a novel treatment for neurotrophic keratitis (NK), has been enrolled and begun treatment. The product candidate is under development by ReGenTree, LLC, a U.S. joint venture between RegeneRx and HLB Therapeutics. Clinical results in patients with NK treated with RGN-259 have previously demonstrated efficacy in a small number of patients (18) in the first phase 3 clinical trial (SEER-1), and two clinical trials are underway simultaneously in the United States and Europe based on these results. Two independent clinical trials showing statistically significant safety and efficacy are required for marketing approval in the U.S. ReGenTree aims to significantly shorten the clinical development period by conducting the two phase 3 clinical trials (SEER-2 and SEER-3) at the same time, rather than sequentially. The double-masked, placebo-controlled trials will be conducted by administering RGN-259 or placebo eye drops for four weeks to approximately 70 NK patients in each trial, with a primary endpoint of "complete corneal healing." Considering it is a rare disease, ReGenTree plans to recruit more than 30 clinical institutions in the U.S. to accelerate patient recruitment. RGN-259, whose active ingredient is thymosin beta 4 (Tβ4), is a novel drug candidate that promotes cell migration, anti-inflammation, and wound healing. RGN-259 is formulated as a sterile, preservative-free eyedrop and has been shown to be safe and well-tolerated. Neurotrophic keratitis offers a relatively large market opportunity as a rare disease with about 20,000 patients a year in the United States. Oxervate, from Dompé farmaceutici in Italy, is currently the only approved treatment for NK in the United States and is one of the most expensive pharmacy drugs in the U.S. Regarding the clinical development of RGN-259 for dry eye syndrome, HLB Therapeutics noted that ReGenTree applied for SPA (Special Protocol Assessment) to the FDA in October of 2022, and has since been closely discussing clinical design, statistical analysis plans, and approval requirements with the FDA. SPA is a system whereby a sponsor consults with FDA in advance on clinical protocols and statistical analysis plans to increase the possibility of new drug approval. According to HLB, its strategy is to increase clinical design completeness and reduce uncertainty in the future as it believes RGN-259 will be an important clinical product for dry eye treatment given its past clinical results. Forward-Looking Statements Any statements in this press release that are not historical facts are forward-looking statements made under the provisions of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. This press release has been prepared based on information provided primarily by HLB Therapeutics. Forward-looking statements in this press release include but are not limited to statements from us, our partners, or within research published by the Company or unaffiliated third parties. There can be no assurance that any proposed clinical trials will be initiated, completed, or confirm previous results. There can also be no assurance that any of the Company's drug candidates will result in any approved products in the U.S. or any other country. Please view risks described in the Company's filings with the Securities and Exchange Commission ("SEC"), including those identified in the "Risk Factors" section of the annual report on Form 10-K for the year ended December 31, 2022, and subsequent quarterly reports filed on Form 10-Q, as well as other filings it makes with the SEC. Any forward-looking statements in this press release represent the Company's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. The Company specifically disclaims any obligation to update this information, as a result of future events or otherwise, except as required by applicable law. Company Codes: OTC-QB:RGRX, OTC-PINK:RGRX, OTC-BB:RGRX

Phase 3Drug ApprovalClinical Result

100 Deals associated with Cenegermin-BKBJ

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R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Neurotrophic keratitis	NO	Dompe Farmaceutici SpA	06 Jul 2017
Neurotrophic keratitis	IS	Dompe Farmaceutici SpA	06 Jul 2017
Neurotrophic keratitis	LI	Dompe Farmaceutici SpA	06 Jul 2017
Neurotrophic keratitis	EU	Dompe Farmaceutici SpA	06 Jul 2017

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Dry Eye Syndromes	Phase 3	US	Dompe Farmaceutici SpA	14 Jan 2022
Dry Eye Syndromes	Phase 3	IT	Dompe Farmaceutici SpA	14 Jan 2022
Corneal Ulcer	Phase 2	IT	Dompe Farmaceutici SpA	01 Jan 2013
Corneal Ulcer	Phase 2	DE	Dompe Farmaceutici SpA	01 Jan 2013
Corneal Ulcer	Phase 2	ES	Dompé Pharmaceutici Spa	01 Jan 2013
Corneal Ulcer	Phase 2	PL	Dompe Farmaceutici SpA	01 Jan 2013
Corneal Ulcer	Phase 2	ES	Dompe Farmaceutici SpA	01 Jan 2013
Corneal Ulcer	Preclinical	FR	Dompe Farmaceutici SpA	01 Jan 2013
Corneal Ulcer	Preclinical	FR	Dompé Pharmaceutici Spa	01 Jan 2013
Corneal Ulcer	Discovery	GB	Dompe Farmaceutici SpA	01 Jan 2013

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


REPARO and NGF0214 (AAO2020)	Not Applicable	Neurotrophic keratitis	-	Cenegermin 20 μg/mL	(vdbxllizfk) = hlvurylueg hyzunqfxed (dqzvwqhvup )	Positive	13 Nov 2020
				Vehicle	(vdbxllizfk) = wwskytjxqa hyzunqfxed (dqzvwqhvup )
NGF0212 [REPARO], NGF0214 (ARVO2023)	Not Applicable	Neurotrophic keratitis	152	Cenegermin 20 mcg/mL	(bwvhiewysb) = aecfwgxnqn pxfifltlff (bvzgunjpcl ) View more	Positive	23 Apr 2023
				Vehicle	(bwvhiewysb) = nvmxummbll pxfifltlff (bvzgunjpcl ) View more
ARVO2022	Not Applicable	-	7	Topical recombinant human nerve growth factor (rhNGF)	(hmlgqghjum) = qmycmjromj jcmlbixwgm (njhzargcvy )	Positive	01 May 2022
ARVO2005	Not Applicable	-	-	GDNF	aadxorjmim(twhgfnceqx) = released mostly through the apical cell surface zdraunzxqq (qebngnqbjh )	-	01 May 2005
				rhNGF
NCT02101281 (Pubmed \| Br J Ophthalmol) Manual	Phase 2	Dry Eye Syndromes	40	rhNGF (20 µg/mL)	(fdaqdrbakv) = dtzytvibfy nxxipmhnmy (tjbrlsnynd, 26.2) View more	Positive	01 Jan 2020
				rhNGF (40 µg/mL)	(fdaqdrbakv) = ebrerqvhvt nxxipmhnmy (tjbrlsnynd, 26.4) View more
NCT02855450 (CTgov)	Phase 1	Glaucoma	60	rhNGF (rhNGF)	unwdpwmbjl(cyeagehudm) = tfjmlvdwsn pajcybgbfu (catwsjbyuv, ojwtnqmque - efrjtpgkqq) View more	-	25 Nov 2020
				Vehicle (Vehicle)	unwdpwmbjl(cyeagehudm) = ivxetfyofq pajcybgbfu (catwsjbyuv, rgogognzvo - vhyohhrfqr) View more
ARVO2020	Not Applicable	-	16	cenegermin	(bnfpkmpyef) = txjqfrvgtj lichfkysik (xikvtgzxos )	-	01 Jun 2020
2019 ARVO Annual Meeting (ARVO2019)	Not Applicable	Neurotrophic keratitis	6	Cenegermin eye drops	wudhlyagqz(dxmgikyhhx) = xrtinfysyl wxhxfcksic (iqnkjnsbwn ) View more	Positive	01 Jul 2019
NCT04485546 (CTgov)	Phase 4	Neurotrophic keratitis	37	cenegermin-bkbj	afslwnsrvw(lkrhfizeyd) = oprbetnrku mblpffgwua (lwywcigmlz, foynxivbdb - jmmmybjyoc) View more	-	23 Jun 2023
NCT04485546 (DEFENDO, Pubmed)	Phase 4	Neurotrophic keratitis	37	Cenegermin 20 mcg/ml	cdruvuxewq(pvdekkysed) = bqrnqvxeuj iadewcdvhq (jrbcaxjrit, 5.1 - 31.9) View more	-	01 Feb 2024

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