More than 90% of patients had absence of DME after four years in a pre-specified exploratory endpoint

People treated with Vabysmo sustained vision gains and anatomical improvements, with almost 80% receiving treatment at intervals of three or four months, in an exploratory analysis

The study met all primary endpoints, showing safety data were consistent with Vabysmo’s known safety profile

This is the largest long-term extension dataset in DME to-date, demonstrating consistent positive results in a highly prevalent eye condition

Basel, 17 July 2024 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new, four-year data from the RHONE-X extension study.1 The study met all primary endpoints, showing that Vabysmo® (faricimab) was well tolerated in people with diabetic macular edema (DME) who received treatment for up to four years.2 Exploratory results from the long-term study showed that Vabysmo continued to preserve vision, dry retinal fluid that can impair sight, and allow extended time between treatments in people with DME.2 These data were presented in a late-breaking oral presentation at The American Society of Retina Specialists (ASRS) 2024 Annual Meeting in Stockholm, Sweden.

“These four-year data build on our pivotal studies and reinforce Vabysmo’s potential to become standard of care treatment for diabetic macular edema (DME), which affects 29 million people worldwide,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “We are especially pleased to see that 9 out of 10 patients showed no sign of DME after four years of treatment with Vabysmo, which is an incredible long-term outcome for people living with this condition.”

The RHONE-X study is the largest long-term extension dataset in DME, a leading cause of vision loss in people with diabetes.

“I have been using Vabysmo as a first-line treatment for all the approved indications, including diabetic macular edema, and the positive long-term safety and efficacy results from the RHONE-X study are consistent with my clinical experience for over two years,” said study investigator Arshad M. Khanani, M.D., Director of Clinical Research at Sierra Eye Associates and Clinical Professor at the University of Nevada, Reno, who presented the data at the ASRS.

During RHONE-X, all participants were treated with Vabysmo on a personalised treat-and-extend regimen, where the time between Vabysmo treatments could be increased based on retinal fluid levels and visual acuity. Results of the exploratory analysis showed that at the end of four years, nearly 80% of participants treated with Vabysmo had extended their treatment intervals to every three or four months. Additionally, people treated with Vabysmo maintained the vision improvements and sustained the drying of retinal fluid they achieved during the initial Phase III studies (YOSEMITE and RHINE). In a pre-specified exploratory endpoint, more than 90% of people treated with Vabysmo achieved absence of DME, defined as central subfield thickness (CST) less than 325 microns.2 CST is a measure of swelling from fluid in the back of the eye caused by unstable, leaky blood vessels; reducing CST indicates retinal drying.

To date, Vabysmo is approved in nearly 100 countries for DME and neovascular or ‘wet’ age-related macular degeneration (nAMD).3,5-8 It is also approved in several countries, including the United States and Japan, for the treatment of macular edema following retinal vein occlusion.3,5,9 More than four million doses of Vabysmo have been distributed worldwide since its initial US approval in 2022

About RHONE-X

RHONE-X is a multicentre two-year extension study designed to evaluate the long-term safety and tolerability of Vabysmo® (faricimab) in 1,474 patients with diabetic macular edema who completed one of the two Phase III studies, YOSEMITE (NCT03622580) or RHINE (NCT03622593).10,11 Patients in YOSEMITE and RHINE were treated with either Vabysmo or 2 mg aflibercept. Patients in RHONE-X were all treated with Vabysmo according to a personalised treatment interval.

The primary objectives were to evaluate the long-term safety and tolerability of Vabysmo, including ocular adverse events (AEs), non-ocular AEs and presence of anti-drug antibodies. The study also had an exploratory objective to assess the long-term efficacy of Vabysmo.

AAbout diabetic macular edema

Affecting around 29 million people globally, diabetic macular edema (DME) is a vision-threatening retinal condition associated with blindness and decreased quality of life when left untreated.12,13 DME occurs when damaged blood vessels leak into and cause swelling in the macula – the central area of the retina responsible for the sharp vision needed for reading and driving.14,15 The number of people with DME is expected to grow as the prevalence of diabetes increases.

About the Vabysmo® (faricimab) clinical development programme

Roche has a robust Phase III clinical development programme for Vabysmo. The programme includes AVONELLE-X (NCT04777201), an extension study of TENAYA (NCT03823287) and LUCERNE (NCT03823300), evaluating the long-term safety and tolerability of Vabysmo in neovascular or ‘wet’ age-related macular degeneration (nAMD).17 Roche has also initiated several Phase IV studies, including the ELEVATUM (NCT05224102) study of Vabysmo in underrepresented patient populations with diabetic macular edema, the SALWEEN study of Vabysmo in a subpopulation of nAMD highly prevalent in Asia, and the POYANG (NCT06176352) study of Vabysmo in adult treatment-naive patients with choroidal neovascularisation secondary to pathologic myopia.18-20 Roche has also initiated the VOYAGER (NCT05476926) study, a global real-world data collection platform, and supports several other independent studies to further understand retinal conditions with a high unmet need.

About Vabysmo® (faricimab)

Vabysmo is the first bispecific antibody approved for the eye.5,22 It targets and inhibits two signalling pathways linked to a number of vision-threatening retinal conditions by neutralising angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A). Ang-2 and VEGF-A contribute to vision loss by destabilising blood vessels, causing new leaky blood vessels to form and increasing inflammation. By blocking pathways involving Ang-2 and VEGF-A, Vabysmo is designed to stabilise blood vessels.22,23 Vabysmo is approved in nearly 100 countries around the world, including the United States (US), Japan, the United Kingdom and the European Union, for people living with neovascular or ‘wet’ age-related macular degeneration and diabetic macular edema, and in several countries, including the US and Japan, for the treatment of macular edema following retinal vein occlusion.3,5,9 Review by other health authorities is ongoing.

About Roche in ophthalmology

Roche is focused on saving people’s eyesight from the leading causes of vision loss through pioneering therapies. Through our innovation in the scientific discovery of new potential drug targets, personalised healthcare, molecular engineering, biomarkers and continuous drug delivery, we strive to design the right therapies for the right patients.

We have the broadest retina pipeline in ophthalmology, which is led by science and informed by insights from people with eye diseases. Our pipeline includes gene therapies and treatments across multiple vision-threatening conditions, including diabetic eye diseases, geographic atrophy and autoimmune conditions, such as thyroid eye disease and uveitic macular edema.

Applying our extensive experience, we have already brought breakthrough ophthalmic treatments to people living with vision loss. Susvimo® (previously called Port Delivery System with ranibizumab) 100 mg/mL for intravitreal use via ocular implant is the first United States (US) Food and Drug Administration-approved refillable eye implant for neovascular or ‘wet’ age-related macular degeneration (nAMD) that continuously delivers a customised formulation of ranibizumab over a period of months.24,25 Vabysmo® (faricimab) is the first bispecific antibody approved for the eye, which targets and inhibits two signalling pathways linked to a number of vision-threatening retinal conditions by neutralising angiopoietin-2 and vascular endothelial growth factor-A.5,22,23 Vabysmo is approved around the world for people living with nAMD and diabetic macular edema, and in several countries, including the US and Japan, for macular edema following retinal vein occlusion.3,5-9 Lucentis® (ranibizumab injection)* was the first treatment approved to improve vision in people with certain retinal conditions

About Roche

Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the twelfth consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2020 employed more than 100,000 people worldwide. In 2020, Roche invested CHF 12.2 billion in R&D and posted sales of CHF 58.3 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan