Ranibizumab (Genentech)

Australia-based Filamon Limited has registered a Phase Ib/IIa first-in-human clinical trial of BETA-TT8 Ophthalmic Gel 3.8%, a topical small-molecule MEK/ERK (mitogen-activated protein kinase) pathway inhibitor, in patients with neovascular age-related macular degeneration (wetAMD). The study, listed on ClinicalTrials.gov as NCT07766473 with a status of "not yet recruiting," is expected to open at four Australian sites in Q3 2026, with primary completion targeted for Q1 2027. The open-label, randomized study will enroll 24 patients with stable wetAMD who have previously received at least three intravitreal aflibercept (Eylea) injections. Participants will be assigned in parallel groups of eight to receive BETA-TT8 at one of three dosing frequencies — once, twice, or three times daily — for 12 weeks. The dose per administration is identical across all arms; frequency is the only variable. Primary endpoints cover treatment-emergent adverse events, corneal epithelial safety, intraocular pressure changes, and dose-limiting toxicities. Exploratory secondary endpoints include aflibercept rescue injection frequency, best-corrected visual acuity change, and central subfield thickness on optical coherence tomography. Patients meeting pre-specified criteria at Week 12 may enter a 12-month open-label extension. The topical gel format is the asset's primary differentiating feature: current standard-of-care anti-VEGF agents — including aflibercept (Eylea, Eylea HD), ranibizumab, and faricimab — require repeated intravitreal injections, which carry procedural burden and cumulative risk. A self-administered eye drop or gel that could extend injection intervals or reduce rescue injection frequency would represent a meaningful change in the treatment paradigm, though whether BETA-TT8 achieves this remains entirely unproven at this stage. No peer-reviewed preclinical studies of BETA-TT8 were identified. Filamon has disclosed company-generated animal data, including a claim that BETA-TT8 outperformed aflibercept in a wet AMD model, but the company labels the underlying results as data on file. Filamon's corporate website lists BETA-TT8 as part of its BETA platform, which the company states targets MEK/ERK signaling in inflamed tissue across ophthalmic, oncology, and cardiovascular indications, but independent corroboration of these claims was not available. The wetAMD space is heavily contested at the anti-VEGF level, with approved agents from Regeneron, Roche, and Novartis dominating treatment. No confirmed comparator programs targeting MEK/ERK via topical ophthalmic delivery were identified in available sources. The absence of public preclinical data and Filamon's early-stage profile mean the key questions — whether the gel achieves sufficient ocular penetration to suppress subretinal neovascularization, and whether MEK/ERK inhibition adds meaningful benefit on top of anti-VEGF therapy — remain entirely open. Safety data from the 12-week treatment period will inform whether BETA-TT8 can progress to a powered efficacy study. The exploratory efficacy endpoints will provide an initial clinical signal of whether topical MEK/ERK inhibition affects wetAMD disease activity, although the study's small size and lack of a control arm will limit interpretation. Access the AllSci platform to explore the science behind the news. Spot something wrong? Report an issue with this article

EyePoint, a Boston-area biotechnology company, lost nearly a billion dollars in market value Monday after its main drug failed to hit the central goal of a key clinical trial.Titled LUGANO, the trial enrolled roughly 400 people with the less common wet form of age-related macular degeneration, meaning they have impaired vision because of abnormal, fluid-leaking blood vessels in their eyes. They received inside-the-eye injections of either aflibercept, the active ingredient in Regeneron Pharmaceuticals blockbuster medicine Eylea, or EyePoints drug, which the company has branded as Durayvu.Researchers then evaluated these participants for a little over a year, with a particular focus on any changes in average best-corrected visual acuity a measure of how clearly a person, when outfitted with optimal prescription lenses, can read the kind of standard eye chart found in an optometrist office. EyePoint has now revealed that, on that measure, across the trials full dataset, its drug didnt prove non-inferior to aflibercept.The company largely blamed the failure on an asymmetric cohort of nine patients in the Durayvu-treated group, who had vision loss or a dropoff of at least 15 letters on the eye exam that was unrelated to wet AMD. According to EyePoint, an after-the-fact analysis found the drug would have achieved non-inferiority had that group been excluded.Additionally, no patients in the aflibercept group experienced vision loss that was on that scale and unrelated to wet AMD. EyePoint said this indicates meaningful overperformance in the control arm, since other, similarly designed studies have shown around 3-5% of patients on aflibercept losing 15 or more letters when tested.Those issues aside, EyePoint maintained the trial produced compelling data that represent a meaningful improvement to current wet AMD standard of care. The company highlighted how Durayvu demonstrated favorable safety, reduced the burden of treatment and allowed three-fourths of patients to go about eight months without supplemental injections of anti-VEGF therapies a class of drugs that includes Eylea as well as Lucentis, Vabysmo and Beovu.While the primary endpoint result for the full dataset was unexpected, the consistently positive results from the pre-specified secondary endpoints and the ad hoc analysis on the primary endpoint present a compelling case for [our drug] as a new potential therapeutic option for wet AMD, Jay Duker, EyePoints CEO, said in a statement.Duker added that his company may file for Food and Drug Administration approval in the first half of next year, depending on how a second late-stage trial, LUCIA, shakes out. Results from that experiment should come sometime between October and the end of December.Investors, meanwhile, appear to have lost confidence in EyePoint and its program, as shares were down 70% by mid-morning Monday.Lisa Walter, an analyst at RBC Capital Markets, wrote in a note to clients that the LUCIA results will be needed to paint a clearer picture of EyePoints future in wet AMD. Yet, even if the study is successful, we think it will be more challenging for the FDA to approve Durayvu without two positive non-inferior trials.The primary endpoint miss is a genuine overhang, Mizuho Securities analyst Graig Suvannavejh wrote in his own note. LUCIA is now the swing factor, as a clean win would substantially de-risk the story and approval plans around Durayvu, whereas a second miss would be far more problematic.To Stifel analyst Annabel Samimy, the findings from LUGANO, while tricky, still offer reasons to be optimistic about Durayvu.Optically, this imbalance is disappointing given the competitive landscape, but it is clear to us that Duravyu is having clinical benefit and has the potential to extend treatment intervals and reduce injection frequency by a significant amount in a maintenance setting, Samimy wrote. '