Iridocorneal Endothelial Syndrome

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.

Efficacy data from Escalón study shows improvements in central corneal thickness and best corrected visual acuity SEATTLE & CAMBRIDGE, Mass. & TOKYO--(BUSINESS WIRE)-- Aurion Biotech, whose mission is to restore vision to millions of patients with life-changing regenerative therapies, today announced several presentations during the 2023 American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting to be held May 5-8, 2023 in San Diego, California. At Eyecelerator and ASCRS 2023, data will be presented from the Company’s Escalón study, which treated subjects with its novel cell therapy, AURN001. In this study, 22 subjects diagnosed with corneal edema secondary to corneal endothelial disease were treated intracamerally with a consistent dose of endothelial cells and three different doses of Y-27632, a rho kinase inhibitor. “We are thrilled to share Escalón findings in multiple sessions at the ASCRS 2023 and Eyecelerator meetings,” said Michael Goldstein, president and chief medical officer, Aurion Biotech. “Escalón data are consistent with our previous trials showing clinically meaningful improvements in central corneal thickness and best corrected visual acuity. We believe these presentations will be of significant interest to the ophthalmology community and will help to highlight the exciting potential of AURN001 to treat patients with corneal endothelial disease.” The schedule of corporate presentations is as follows: Transformational Cell Therapy for Corneal Endothelial Disease Thursday, May 4, 2023 1:00 p.m. – 2:30 p.m. PDT Eyecelerator, Marriott Marquis San Diego Marina Presenter: Michael Goldstein, President and Chief Medical Officer, Aurion Biotech If Not First, You’re Last: First-in-Human Studies Thursday, May 4, 2023 3:45 p.m. - 4:15 p.m. PDT Eyecelerator, Marriott Marquis San Diego Marina Panelist: Greg Kunst, Chief Executive Officer, Aurion Biotech Aurion Biotech and its cell therapy will also be mentioned in the following ASCRS scientific presentations: Overview of Injectable Endothelial Cell Therapy Friday, May 5, 2023 8:54 a.m. – 9:01 a.m. PDT San Diego Convention Center - Upper Level, Room 6B Presenter: Deepinder Dhaliwal, MD Update on Endothelial Cell Therapy Clinical Trials Friday, May 5, 2023 9:01 a.m. - 9:08 a.m. PDT San Diego Convention Center - Upper Level, Room 6B Presenter: John P. Berdahl, MD Interim Assessment of Safety & Efficacy of Human Corneal Endothelial Cell Therapy for Corneal Edema Secondary to Endothelial Dysfunction Sunday, May 7, 2023 10:15 a.m. - 10:20 a.m. PDT San Diego Convention Center - Upper Level, Room 4 Presenter: Matthew Giegengack, MD, ABO Aurion Biotech’s combination therapy recently received regulatory approval from Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), for the treatment of bullous keratopathy, marking the first-ever regulatory approval in the world for an allogeneic cell therapy to treat corneal endothelial disease. About Aurion Biotech With offices in Seattle, Cambridge and Tokyo, Aurion Biotech is a clinical-stage biotech company, whose mission is to restore vision to millions of patients with life-changing regenerative therapies. Aurion Biotech is the recipient of the prestigious Prix Galien award for best start-up in biotech. The Company’s first candidate is for the treatment of corneal edema secondary to corneal endothelial disease, and is the first off-the-shelf, clinically validated cell therapy for corneal care, having received regulatory approval in Japan. The Company is preparing for clinical trials in the U.S. Privately held, Aurion Biotech is backed by leading investors that include Deerfield, Alcon, Petrichor, Flying L Partners, Falcon Vision / KKR, and Visionary Ventures. To learn more about Aurion Biotech, visit View source version on businesswire.com: Contacts Judith McGarry (corporate communications) 415-971-2900 judith.mcgarry@aurionbiotech.com Michele Gray (ophthalmology media) 917-449-9250 michele@mgraycommunications.com Beth Keshishian (biotech & business media) 917-912-7195 beth@bethkeshishian.com Source: Aurion Biotech View this news release online at:

SAN DIEGO--( BUSINESS WIRE )-- Trefoil Therapeutics , a clinical-stage biotechnology company focused on developing novel new therapeutic agents to treat corneal diseases, today announced a series of data presentations demonstrating the potential use of TTHX1114 to protect endothelial cells at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in New Orleans. The company also announced the completion of the Phase 1 epithelial study designed to assess the safety and dosing profile of TTHX1114 in a topical eye drop formulation for epithelial conditions. David Eveleth, Ph.D., President and CEO of Trefoil Therapeutics, presented a data update from the Phase 2 STORM study , which evaluated the potential use of TTHX1114 as a way to protect endothelial cells from damage due to cataract surgery in Fuchs endothelial corneal dystrophy (FECD) patients undergoing Descemet Stripping Only (DSO). New findings included: Across all patients, TTHX1114 produced a dose-dependent response in resolution of corneal edema and improvement of best corrected visual acuity (BCVA) following surgery. The proportion of patients with complete resolution of corneal edema in the high-dose groups at 28 days after surgery was similar in patients with DSO vs. those with DSO and cataract surgery (25.9% vs 17.4%). The proportion of patients recovering adequate BCVA (seeing 69 letters or better) was also similar between these groups, with 58% of the DSO only patients vs. 61% of the DSO and cataract surgery patients at 28 days after surgery. “Corneal endothelial diseases, like FECD, affect millions of Americans, and today there are limited treatment options. Therefore, it is imperative that we continue to explore new therapies to allow these patients to retain functional vision. It is exciting to see TTHX1114 perform well in controlling post-surgical edema in at-risk patients undergoing cataract surgery,” said Eveleth. In the poster presentation “Factors affecting recovery following Descemet Stripping Only (DSO) with TTHX1114,” recovery following DSO was evaluated in relation to three variables. Smaller descemetorhexis (surgical removal of Descemet membrane) size was associated with faster recovery while comorbid diabetes appeared to delay recovery. However, visual outcomes for patients undergoing DSO did not appear to be adversely affected by concurrent cataract surgery. The poster presentation “The engineered fibroblast growth factor TTHX1114 mediates enrichment of proliferative and wound healing pathways in wounded human corneal endothelium,” followed up on Trefoil’s publication earlier this year demonstrating that TTHX1114 accelerates wound healing in the corneal endothelium. RNA-sequencing analysis was performed to identify changes in gene expression and pathway enrichment resulting from treatment with TTHX1114. These results confirmed that treatment of wounded corneal endothelium with TTHX1114 generates a statistically significant shift in gene expression, and specifically produces a response in fibroblast growth factor (FGF) signaling, potentially connected to the previously reported 2.5 times greater wound healing in treated corneas. Protecting the corneal endothelium from further damage due to oxidative stress is beneficial for FECD patients and cataract patients as the two are closely linked. In the poster presentation, “TTHX1114 protects and rescues primary rabbit corneal endothelial cells from oxidative stress,” data show that TTHX1114 protects and rescues rabbit corneal endothelial cell viability against tBHP induced oxidative stress, supporting TTHX1114 as a promising approach to mitigate endothelial damage. The abstracts are available at the ARVO 2023 program here . 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 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. The topical formulation of TTHX1114 is designed to accelerate corneal ulcer wound healing by stimulating corneal epithelial cell growth, potentially reducing common complications associated with corneal ulcers, such as pain, inflammation, and loss of vision due to corneal scarring. The Phase 1 safety study for the topical product was completed in March of 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, thereby reversing vision loss. 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 www.trefoiltherapeutics.com . 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.