Retinal Physician eUpdate - February 16, 2024

This article was originally published in a sponsored newsletter.

EyeBio Announces Positive Visual, Anatomic, and Safety Data from AMARONE Trial of Restoret

EyeBio has announced in a press release the presentation of Week 12 data from its first-in-human Phase 1b/2a AMARONE trial of Restoret in patients with treatment-naïve diabetic macular edema (DME) and treatment-naïve neovascular age-related macular degeneration (nAMD). According to the release, the data demonstrated that Restoret was well tolerated, with no drug-related adverse events, drug-related serious adverse events, or intraocular inflammation reported. Patients with DME (n=26) received Restoret as monotherapy, manifesting a mean improvement in best-corrected visual acuity of +11.2 letters and a mean reduction in retinal thickness of -143 microns. Similar outcomes were observed in patients with nAMD (n=5), who received Restoret in combination with aflibercept. The data demonstrated that multiple monthly doses of Restoret, as both monotherapy and in combination with aflibercept, were well tolerated. CEO David Guyer, MD, was quoted in the release saying, “The AMARONE trial represents the first ever clinical use of a Wnt pathway agonist to address retinal disease, and we are encouraged by the preliminary safety and efficacy data we’ve seen thus far.” More information can be found at https://www.businesswire.com/news/home/20240212626117/en/EyeBio-Announces-Positive-Visual-Anatomic-and-Safety-Data-from-First-in-Human-Ph1b2a-AMARONE-Trial-of-Restoret-at-Macula-Society-Annual-Meeting.

New Long-Term Data for Vabysmo Show Sustained Retinal Drying and Vision Improvements in RVO

Genentech, a member of the Roche Group, has announced new 72-week data from two global Phase III studies, BALATON and COMINO, evaluating Vabysmo (faricimab-svoa) in macular edema due to branch and central retinal vein occlusion (BRVO and CRVO). A press release says that whereas available RVO treatments are typically given every one to two months, the data showed nearly 60% of people receiving Vabysmo in BALATON and up to 48% of people in COMINO were able to extend their treatment intervals to three or four months apart. In addition, patients in the studies maintained vision gains and robust retinal drying achieved in the first 24 weeks of the studies for more than one year. In both studies, Vabysmo was well tolerated, and the safety profile was consistent with previous studies. More information can be found at https://www.gene.com/media/press-releases/15017/2024-01-31/new-long-term-data-for-genentechs-vabysm.

4DMT’s Experimental Gene Therapy Cuts Eylea Use in Phase II Wet AMD Trial

4D Molecular Therapeutics has unveiled topline results from its Phase II PRISM study, showing that its investigational gene therapy 4D-150 can significantly ease treatment burden in patients with wet AMD. According to a press release, over 24 weeks of follow-up, 84% of patients treated with 4D-150’s higher 3E10 vg/eye dose did not need supplemental injections with Regeneron and Bayer’s Eylea (aflibercept). At the lower dose group of 1E10 vg/eye, only one patient needed supplemental Eylea. The release said 63% and 50% of patients in the high- and low-dose groups, respectively, did not need supporting Eylea treatment, and overall, the high dose level of 4D-150 reduced annual Eylea injections by 89%, while the lower dose cut Eylea rates by 85%. PRISM found no instance of significant inflammation, even in the high-dose group. 4D-150 is an investigational gene therapy that delivers a transgene cassette that expresses both aflibercept and an RNA interference molecule that blocks the VEGF-C protein. The double-payload approach allows 4D-150 to inhibit four VEGF family members—VEGF A, B, C and PIGF. More information can be found at https://www.biospace.com/article/4dmt-s-experimental-gene-therapy-cuts-eylea-use-in-phase-ii-wet-amd-trial/.

Kiora and Théa Open Innovation Enter Strategic Agreement to Develop and Commercialize KIO-301 for the Treatment of Inherited Retinal Diseases

Kiora Pharmaceuticals has entered a strategic development and commercialization agreement with Théa Open Innovation (TOI), a sister company of the global ophthalmic specialty company Laboratoires Théa (Théa). Under the agreement, Kiora granted TOI exclusive worldwide development and commercialization rights, excluding Asia, to KIO-301 for the treatment of degenerative retinal diseases. In exchange, Kiora will receive an upfront payment of $16 million; up to $285 million upon achievement of pre-specified clinical development, regulatory, and commercial milestones; tiered royalties of up to low 20% on net sales; and reimbursement of KIO-301 research and development expenses. The partnership covers retinitis pigmentosa and potentially other indications in ophthalmology across all global geographies, excluding China, Japan, and certain other countries in Asia. Kiora is primarily responsible for the design and implementation of clinical development through phase 2 whereas Théa will assume primary responsibility for phase 3 clinical trials as well as for securing regional marketing authorizations. Upon approval in respective regions, Théa will be responsible for all commercial activities including sales, marketing, and market access. KIO-301 is a small molecule, referred to as a molecular photoswitch, designed to confer light-sensing capabilities to retinal ganglion cells. More information can be found at https://www.biospace.com/article/kiora-pharmaceuticals-and-thea-open-innovation-enter-strategic-agreement-to-develop-and-commercialize-kio-301-for-the-treatment-of-inherited-retinal-diseases/.

Foundation Fighting Blindness Hosting Webinar on Best Clinical Practices for Patients with Inherited Retinal Diseases

The Foundation Fighting Blindness will host a webinar for eye care professionals on the best practices for managing their patients with inherited retinal diseases (IRDs), including retinitis pigmentosa, Usher syndrome, Stargardt disease, and Leber congenital amaurosis. According to a press release the webinar will be part of the Envisioning a Path to Hope initiative, raising awareness among eye care professionals for the valuable resources the Foundation provides IRD patients and caregivers. The release says the free online course will review several topics related to the care and management of IRD patients, including recommended functional and structural tests, potential manageable complications, genetic testing and why it is a critical element in IRD care, clinical trials for emerging therapies, IRD patient registries, low vision clinics, and additional resources. The hour-long event will take place Wednesday, March 20, at 7:00 p.m. ET. More information can be found at https://www.prnewswire.com/news-releases/foundation-fighting-blindness-hosting-webinar-for-eye-care-professionals-best-clinical-practices-for-patients-with-inherited-retinal-diseases-302059690.html.

Ocuphire Pharma Announces Presentation on APX3330 at Angiogenesis, Exudation, and Degeneration 2024

Ocuphire Pharma has announced that clinical data from its ZETA-1 trial on a person-level scale evaluating APX3330 in diabetic retinopathy (DR) were presented at the recent Angiogenesis, Exudation, and Degeneration conference. According to a press release, ZETA-1 was a multi-center, randomized, double-masked, placebo-controlled Phase 2 trial of oral APX3330 in DR that enrolled 103 subjects. Analysis of ZETA-1 Phase 2 results using the person-level scale showed only 5% of subjects treated with APX3330 had a clinically meaningful ≥ 3-step worsening in DRSS at Week 24 on this binocular person-level scale, compared with 13% of placebo patients (p=0.18). APX3330 showed favorable safety and tolerability in diabetic patients that continued dosing their medications through the study durations to manage their diabetic comorbidities. More information can be found at https://ir.ocuphire.com/press-releases/detail/427/ocuphire-pharma-announces-presentation-on-apx3330-at-the.

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