Corneal fibrosis

SAN DIEGO, CA / ACCESSWIRE / August 18, 2023 / Amber Ophthalmics, Inc. (Amber), a privately held, clinical stage biopharmaceutical company, announced today that Dr. Mark S. Gorovoy from Gorovoy M.D. Eye Specialists has enrolled the first patient in the AMB-01-006 (NEXPEDE-1) study. The NEXPEDE-1 study is a randomized, double-masked, vehicle-controlled Phase 2/3 clinical trial designed to evaluate two concentrations of Nexagon® (lufepirsen ophthalmic gel) for the treatment of persistent corneal epithelial defects (PCED) (ClinicalTrials.gov identifier: NCT05966493). This study evaluates the safety and efficacy of Nexagon, in a dosing regimen that utilizes as few as five topical in-office administrations for subjects with confirmed non-infectious PCED. Subjects who do not re-epithelialize within the first four weeks of treatment will continue to receive weekly administrations until subjects either achieve re-epithelialization or complete eight weeks of therapy. The primary endpoint for the NEXPEDE-1 study will be complete corneal healing as determined by corneal fluorescein staining. "We are excited to have enrolled the first subject into the NEXPEDE-1 study and are fortunate to have a strong contingency of clinical sites dedicated to the treatment of PCED," said Dr. Shawn A. Scranton, President and CSO of Amber. "We look to expeditiously complete this study, as it will further guide the development of this first-in-class PCED therapy that we believe can address a variety of the underlying PCED etiologies." LUFEPIRSEN OPHTHALMIC GEL (NEXAGON) is a patent-protected, unmodified antisense oligonucleotide that inhibits connexin43 protein translation, a cell membrane hemichannel forming protein associated with ocular inflammation and progression of PCED disease pathology. As a result of Nexagon's unique mechanism of action, the medication may be administered topically in the office by the physician at a frequency far less than the current treatment paradigm consisting of multiple daily patient-administered drops. ABOUT AMBER OPHTHALMICS Amber Ophthalmics, Inc. is a privately held, clinical-stage biopharmaceutical company focused on innovative therapies for rare ophthalmic diseases. We are committed to addressing unmet medical needs for the restoration, preservation and improvement of patient vision and quality of life. For more information, visit amberophthalmics.com or LinkedIn for updates. ABOUT PERSISTENT CORNEAL EPITHELIAL DEFECTS (PCED) PCED is a rare and progressive disease impacting ~100,000 patients in the United States per year that arises from the failure of corneal re-epithelialization within 10-14 days of a corneal insult, even with the use of standard supportive treatment. Despite treatment, and regardless of underlying etiology if progression is not halted, PCED can result in corneal scarring, vascularization and/or severe vision loss. There are currently no FDA-approved therapies for the treatment of PCED, with many patients progressing to corneal transplant. Contact Information Jill Jene, PhD CBO, Amber Ophthalmics bizdev@amberophthalmics.com SOURCE: Amber Ophthalmics View source version on accesswire.com:

Additional data from the STORM study demonstrate vision quality to be a major driver of the patient decision to undergo surgery SAN DIEGO--(BUSINESS WIRE)-- Trefoil Therapeutics, a clinical-stage biotechnology company focused on developing novel new therapeutic agents to treat corneal diseases, today announced that TTHX1114 was found to improve the rate of visual recovery and corneal deturgescence (state of relative dehydration maintained by the normal cornea that is necessary for transparency) after Descemet Stripping Only (DSO) in a dose-responsive manner in the Phase 2 STORM study. A separate STORM analysis assessed visual morbidity in patients with Fuchs endothelial corneal dystrophy (FECD) prior to surgery using the V-Fuchs Questionnaire (V-FUCHS). The data were presented at the American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting in San Diego. Brandon J. Baartman, M.D., lead surgeon, Vance Thompson Vision (Omaha, NE), presented data from the Phase 2 STORM study designed to evaluate the safety and efficacy of TTHX1114, an engineered form of Fibroblast Growth Factor (FGF1), delivered as an intracameral injection as an adjunct to DSO (with or without combination cataract surgery). The study results showed TTHX1114 improved best-corrected visual acuity (BCVA) and central corneal thickness (CCT) after DSO in the 49 subjects/64 eyes. Faster recovery was associated with smaller descemetorhexis (surgical removal of Descemet membrane), while comorbid diabetes appeared to delay recovery. No significant differences were noticed in the recovery of subjects undergoing combination cataract surgery compared to those undergoing DSO alone. “Our increasing body of clinical evidence continues to support our hypothesis that FGF1 leverages the inherent regenerative properties of corneal endothelial and epithelial cells with TTHX1114 to restore vision. We are excited to continue to build on our research demonstrating the safety and efficacy of TTHX1114 in scenarios where patients with endothelial compromise undergo ocular surgery,” said David Eveleth, Ph.D., President and CEO of Trefoil Therapeutics. Russell Swan, M.D., presented “Assessing Visual Morbidity in Patients with FECD Prior to Surgery Using the V-Fuchs Questionnaire.” In this study data from the Phase 2 STORM study was utilized to assess the baseline characteristics of subjects and identify quality of vision characteristics that may motivate patients to seek DSO surgery. Despite baseline assessments showing reasonably good vision, even in the presence of disease, results from the V-FUCHS suggest moderate vision interference in daily activities, difficulty in seeing fine details, and issues with glare and halos, particularly at night. The V-FUCHS questionnaire is a 15 item, two domain self-report measure of vision function developed by Mayo Clinic. “Vision function from the perspective of the patient is an important consideration as quantitative measures of vision often do not capture the totality of how quality of vision affects daily activities. This indicates a need for providers to consider the lifestyle and visual requirements for patients and how quality of life may be impaired by corneal diseases when discussing treatment options,” said Dr. Eveleth. “Protection of the corneal endothelium in patients with Fuchs dystrophy undergoing surgery represents a significant unmet medical need,” said Mark Packer, M.D., Chief Medical Officer of Trefoil Therapeutics. “There is now a promising therapeutic agent that speeds recovery of the endothelium following surgery. TTHX1114 provides hope that patients at risk of corneal decompensation may maintain good vision and avoid the risks of corneal transplantation.” The abstracts can be found online here. All educational content of the ASCRS Annual Meeting is planned by its program committee, and ASCRS does not endorse, promote, approve, or recommend the use of any products, devices, or services. About TTHX1114 TTHX1114 is an investigational engineered variant of the naturally occurring molecule Fibroblast Growth Factor-1 (FGF1), which stimulates corneal endothelial and epithelial cell proliferation and migration as well as protecting these cells from stress and injury. Trefoil is conducting clinical trials for TTHX1114 as an intracameral injection in patients susceptible to corneal edema due to endothelial damage, including Fuchs endothelial corneal dystrophy (FECD) and other corneal endothelial dystrophies (CED) and patients with other risk factors undergoing ocular surgeries. The drug is aimed at protecting and regenerating corneal endothelial cells lost due to disease and thereby improving vision. Trefoil is also developing a topical formulation of TTHX1114 which stimulates corneal epithelial cell growth, potentially reducing common complications associated with ulcerative corneal conditions, such as pain, inflammation, and loss of vision due to corneal scarring. The Phase 1 safety study for the topical product was completed in April 2023. About Trefoil Therapeutics San Diego-based Trefoil Therapeutics is a clinical-stage biotechnology company focused on leveraging its engineered FGF1 protein technology platform to develop first-in-class pharmacologic treatments for serious corneal endothelial diseases and epithelial disorders. Trefoil’s lead product candidate is TTHX1114, an investigational engineered form of naturally occurring FGF1 designed to stimulate corneal cell protection, proliferation and migration, which will treat a number of corneal conditions. The technology underlying Trefoil’s platform was developed by co-founders Michael Blaber, Ph.D., and David Eveleth, Ph.D., and is licensed from Florida State University. Trefoil investors include Bios Partners, Hatteras Venture Partners, Access Biotechnology, Solasta Ventures, InFocus Capital Partners, Correlation Ventures, ExSight Ventures and FemHealth Ventures. Learn more at . Safe Harbor Statement The clinical research discussed in this press release is preliminary and the outcome of such studies may not be predictive of the outcome of later clinical trials. Future clinical trial results may not demonstrate safety and efficacy sufficient to obtain regulatory approval related to the clinical research findings discussed in this press release.

Data presented at Association for Research in Vision and Ophthalmology Annual Meeting on April 23, 2023Topical application of B-VEC to the eye was well tolerated and patient showed significant improvement of visual acuity and complete corneal re-epithelization PITTSBURGH, April 24, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a biotechnology company focused on developing and commercializing genetic medicines for patients with rare diseases, announced today that the Company presented new data on the compassionate use of topical beremagene geperpavec (B-VEC) to treat a patient with dystrophic epidermolysis bullosa (DEB) with recurrent cicatrizing conjunctivitis at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting on April 23, 2023. “DEB is a devastating disease with limited treatment options, and there is a substantial population of DEB patients with ocular complications for which treatment options are limited and often include surgery,” said Alfonso L. Sabater, M.D., PhD, Assistant Professor of Clinical Ophthalmology at the Bascom Palmer Eye Institute at the University of Miami Miller School of Medicine. “It is exciting to potentially advance a topical treatment for patients with ocular complications associated with DEB.” The data presented describes the first application of B-VEC to treat ocular complications in a patient with DEB under a compassionate use program. The patient presented with cicatrizing conjunctivitis and underwent surgical symblepharon lysis with pannus removal in the right eye. B-VEC was administered to the patient’s right eye at regular intervals following surgery in addition to routine post-surgical management. B-VEC was well tolerated and associated with full corneal healing by 3 months as well as significant visual acuity improvement from hand motion to 20/40 at 7 months, the latest time point of the on-going treatment effect evaluation. A B Figure 1: Slit lamp pictures of the right eye. A: Baseline ankyloblepharon. The visual acuity was hand motion (HM) B: Ocular surface of the right eye 6 months after the surgery after B-VEC applications. No drug-related adverse events (AE) have been observed. Two non drug related, serious AEs were reported: 1) Prolonged hospitalization due to complications post-gastrointestinal surgery, and 2) Prolonged hospitalization due to complications post-esophageal dilation. B-VEC treatment was not interrupted during either event. Ocular complications are common in patients with DEB, with over half of the patients diagnosed with recessive DEB potentially affected. Typical ocular manifestations include corneal abrasion, as well as corneal scarring, pannus, eyelid ectropions and blisters.1,2 There are no specific FDA-approved treatment options for ocular manifestations of DEB.3 “Ocular complications impose a heavy burden on DEB patients. Based on this promising initial data, we plan to engage with regulatory authorities and explore how we can expand the utility of B-VEC to address this urgent need,” said Suma Krishnan, President, Research & Development, Krystal Biotech. “We are also excited about the implications for our platform as this clinical data, together with ongoing preclinical studies evaluating intravitreal and subretinal routes of delivery to the eye, suggests significant potential to treat multiple ocular diseases with few or no treatment options.” The poster was available to conference attendees and is available on the Investor section of the Company’s website. About Dystrophic Epidermolysis Bullosa (DEB)DEB is a rare and severe disease that affects the skin and mucosal tissues. It is caused by one or more mutations in a gene called COL7A1, which is responsible for the production of the protein type VII collagen (COL7) that forms anchoring fibrils that bind the dermis (inner layer of the skin) to the epidermis (outer layer of the skin). The lack of functional anchoring fibrils in DEB patients leads to extremely fragile skin that blisters and tears from minor friction or trauma. DEB patients suffer from open wounds, which leads to skin infections, fibrosis which can cause fusion of fingers and toes, and ultimately an increased risk of developing an aggressive form of squamous cell carcinoma which, in severe cases, can be fatal. About B-VEC B-VEC is an investigational non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. B-VEC was designed to treat DEB at the molecular level by providing the patient’s skin cells the template to make normal COL7 protein, thereby addressing the fundamental disease-causing mechanism. The FDA and EMA have each granted B-VEC orphan drug designation for the treatment of DEB, and the FDA has granted B-VEC fast track designation and rare pediatric designation for the treatment of DEB. In addition, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) to B-VEC for the treatment of DEB and the EMA granted PRIority MEdicines (PRIME) eligibility for B-VEC to treat DEB. About Krystal Biotech, Inc.Krystal Biotech, Inc. (NASDAQ: KRYS) is a biotechnology company focused on developing and commercializing genetic medicines for patients with rare diseases. The Company’s wide-ranging pipeline is based on its proprietary redosable HSV vector. Headquartered in Pittsburgh, Pennsylvania, the Company is led by an experienced management team, is fully-integrated and has core capabilities in viral vector design, vector optimization, gene therapy manufacturing and commercialization. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and Twitter. Forward-Looking StatementsAny statements in this press release about future expectations, plans and prospects for Krystal Biotech, Inc., including statements about our plans to engage with regulatory authorities to explore how we can expand the utility of B-VEC; the significant potential of our platform to treat multiple ocular diseases with few or no treatment options; and other statements containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “likely,” “will,” “would,” “could,” “should,” “continue,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties associated with regulatory review of clinical trials and applications for marketing approvals, the availability or commercial potential of B-VEC, the sufficiency of cash resources and need for additional financing and such other important factors as are set forth under the caption “Risk Factors” in the Company’s annual and quarterly reports on file with the U.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date of this release. The Company anticipates that subsequent events and developments will cause its views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this release. CONTACTInvestors and Media: Meg Dodge Krystal Biotech mdodge@krystalbio.com 1. Tang JY, Marinkovich MP, Lucas E, et al. A systematic literature review of the disease burden in patients with recessive dystrophic epidermolysis bullosa. Orphanet J Rare Dis. 2021 Apr 13; 16(1): 175. doi: 10.1186/s13023-021-01811-7. 2. Tong L, Hodgkins PR, Denyer J, et al. The eye in epidermolysis bullosa. Br J Ophthalmol. 1999 Mar; 83(3): 323-6. doi:10.1136/bjo.83.3.323. 3. Chen VM, Mehta N, Robbins CC, et al. Anterior-segment spectral domain optical coherence tomography in epidermolysis bullosa. Ocul Surf. 2020 Oct; 18(4): 912-919. doi: 10.1016/j.jtos.2020.08.010 Photos accompanying this announcement are available at https://www.globenewswire.com/NewsRoom/AttachmentNg/dd1859a6-21d6-4bee-84d1-1cd6b7052ea5 https://www.globenewswire.com/NewsRoom/AttachmentNg/0ef5004a-2f1b-4cc7-98ec-d8fda41d606c