A host of exciting data and potentially ground-breaking results which could make a real impact on clinical care for a wide range of retinal diseases were featured at the Late Breaking News session at EURETINA.
Global Survey on Management of DME and DR
Chaired by Ramin Tadayoni, Jose García Arumí and Kourous Rezaei, the session commenced with Anat Loewenstein MD, PhD who presented the interim results from a global survey to better understand the management of diabetic macular oedema (DME) and diabetic retinopathy (DR) from the perspectives of patients, physicians, and clinic staff. 80 clinics in 24 countries have been enrolled to date, with 37 clinics in 16 countries included in the interim analysis. The survey found that patients with DME did not fully understand their disease and treatment, with 55% saying they did not know how many treatments they would have in the next 12 months and 50% saying that they did not know how long the treatment would last for. Furthermore, 41% of patients with DR said that they had not received any information about what to expect with treatment over time, while 53% of clinics said they did not have sufficient educational material for their patients. “We hope that the final analysis of this global survey will generate a wealth of detailed and systematic information to facilitate the enhancement of care and improve treatment outcomes for our patients,” said Prof Loewenstein.
Arshad Khanani MD then presented 12-week data from the BEHOLD Trial, a phase 2 study of UBX1325, a novel senolytic agent for patients with advanced diabetic macular oedema (DME). The early results showed that the treatment was well tolerated with no serious adverse events and no intraocular inflammation. Patients showed improvement in best-corrected visual acuity (BCVA) through to 18 weeks and the gains were robust across a range of disease severity. Retinal structure was maintained in treated patients compared to sham treated patients. Summing up, Dr Khanani said that UBX1325 could potentially provide an important benefit as a stand-alone treatment, in combination regimen, or for use in patients who have a suboptimal response to current standard of care treatments for diabetic macular oedema.
Dr Khanani also presented 24-week efficacy and safety outcomes of the BEACON phase 3 pivotal study of the anti-VEGF antibody biopolymer conjugate for retinal vein occlusion (RVO). He said that the study of tarcocimab tedromer (KSI-301; tarcocimab) met the primary endpoint of non-inferior change from baseline in visual acuity at week 24 compared to aflibercept in patients with macular oedema due to retinal vein occlusion. Tarcocimab also demonstrated robust anatomic responses and a favourable safety profile. “Tarcocimab is the first anti-VEGF therapy to show comparable visual acuity outcomes to monthly aflibercept while doubling the treatment interval,” he said.
Gene Therapy for LHON
Qiutang Li PhD presented the first clinical results at 12 months for post intravitreal administration of NFS-02 (rAAV2-ND1) for gene therapy treatment of Leber’s hereditary optic neuropathy with ND1 G3460A mutation. Dr Li noted that Leber’s hereditary optic neuropathy (LHON) is a rare genetic disease caused by mitochondrial gene mutations, such as MT-ND1, MT-ND4, and MT-ND6. It causes sudden progressive vision loss in the affected patients, and there is no approved treatment currently available for LHON. Summing up the interim results in 10 patients, Dr Li said that the gene therapy was well tolerated by patients and no serious adverse events have been observed thus far. Significant best-corrected visual acuity gains were also observed in 6 out of 10 patients at 12 months in the treated eye.
OAKS and DERBY phase 3 Trials
Frank Holz MD, PhD, presented the latest update on the OAKS and DERBY phase 3 trials, with analyses of pegcetacoplan efficacy in geographic atrophy (GA) due to AMD at 18 and 24 months using three different statistical models. The pegcetacoplan treatment effect accelerated between months 18-24, demonstrating a robust reduction of GA lesion growth compared to sham. The drug also continued to demonstrate a favourable safety profile. “This is the first agent to show clinically meaningful reduction of GA lesions in two pivotal phase 3 clinical trials. Overall, the safety profile is favourable at 24 months. As this is a chronic disease, long-term data will be very important and informative. The GALE extension study will show the impact of treatment over a longer period of time,” said Prof Holz.
Peter Kertes MD then discussed the 32-week results from TALON, a phase 3b study of brolucizumab versus aflibercept in a matched (treat-and-extend) regimen in patients with neovascular age-related macular degeneration. Brolucizumab met both primary end points of the study. It was found to be superior to aflibercept in the distribution of the last treatment interval and non inferior in visual outcome. Additionally, brolucizumab achieved greater reduction in central subfield thickness from baseline at weeks 28 and 32 compared to aflibercept.
Veeral Sheth MD, FACS, presented data looking at the real-world efficacy, durability and safety of faricimab in neovascular AMD. In the ongoing collaborative TRUCKEE study, investigators analysed the patient demographic data, previous treatment history, efficacy, and safety of patients’ treatment with faricimab (Vabysmo, Roche) who had in most cases been treated with another drug for neovascular AMD. The results showed that treatment with faricimab in patients with neovascular AMD achieved improvements in visual acuity (VA), central subfield thickness (CST), and pigment epithelial detachments (PEDs).
United States-Europe Awareness Study
Ramin Tadayoni MD rounded off the session presenting on behalf of Ashish Sharma MD with the latest update on biosimilars for retinal diseases focusing on the United States-Europe Awareness Survey (Bio-USER –Survey). The survey found that the majority of retinal physicians in the United States and Europe are familiar with anti-VEGF biosimilars, but most felt that additional information regarding their safety, efficacy and immunogenicity was needed in order to make informed clinical decisions regarding their use. The survey also revealed that retinal physicians from the United States were more comfortable in using off-label bevacizumab compared to their counterparts in Europe.
All registered attendees will be able to view this session via playback on the virtual platform.