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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.

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.

SAN DIEGO--(BUSINESS WIRE)-- Trefoil Therapeutics, a clinical-stage biotechnology company focused on restoring sight to people with corneal diseases, today announced multiple presentations providing evidence for clinical and preclinical efficacy of the company’s investigational medicine TTHX1114 for improving visual outcomes and protection against corneal endothelial damage at the Annual Association for Research in Vision and Ophthalmology (ARVO) Meeting in New Orleans, April 23-27, 2023. “We aim to address an unmet clinical need with TTHX1114 by offering the potential to restore vision by stimulating and protecting the cornea’s own endothelial cells,” said David Eveleth, Ph.D., President and CEO of Trefoil Therapeutics. “We're excited to share the growing body of research that supports TTHX1114 as an important and unique approach to corneal disease.” The details of the presentations are as follows (all times local, CT): Protection of endothelial cells by TTHX1114 in patients with Fuchs endothelial corneal dystrophy (FECD) undergoing Descemet’s Stripping Only (DSO) in combination with cataract surgery: evidence from the STORM study Presenter: David Eveleth Location: Room 244 Date and Time: Monday, April 24, 2023; 11:30-11:45 am The engineered fibroblast growth factor TTHX1114 mediates enrichment of proliferative and wound healing pathways in wounded human corneal endothelium Poster Number: 618 - B0255 Date and Time: Sunday, April 23, 2023; 12:00-1:45 pm Factors Affecting Recovery Following Descemet Stripping Only (DSO) with TTHX1114 Poster Number: 631 - B0268 Date and Time: Sunday, April 23, 2023; 12:00-1:45 pm TTHX1114 Protects Primary Rabbit Corneal Endothelial Cells from Oxidative Stress Poster Number: 621 - B0258 Date and Time: Sunday, April 23, 2023; 12:00-1:45 pm David Eveleth will be a panelist in a symposium titled “Emerging Therapeutics to Maintain Corneal Health” on April 25, 2023, from 8:45-10:30 am in the La Nouvelle C room. His topic will be: The Role of Fibroblast Growth Factor derivatives in corneal endothelial regeneration which will be presented from 9:13-9:27 am. Trefoil will also be present at booth #1156. About TTHX1114 TTHX1114 is an investigational engineered variant of the naturally occurring molecule Fibroblast Growth Factor-1 (FGF1), which stimulates cell proliferation and migration as well as protecting cells from stress and injury. Trefoil is conducting clinical trials for TTHX1114 as an intracameral injection into the eye for 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. The topical formulation of TTHX1114 is designed to accelerate corneal ulcer wound healing by stimulating 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 . 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.