FDA Accepts Application for Genentech’s Vabysmo for the Treatment of Retinal Vein Occlusion (RVO)

‒ Acceptance based on two Phase III studies that demonstrated early and sustained vision improvement with Vabysmo, meeting primary endpoint of non-inferiority compared to aflibercept –

‒ Application was further supported by data showing Vabysmo achieved rapid and robust drying of retinal fluid –

‒ If approved, RVO would be the third indication for Vabysmo in addition to wet age-related macular degeneration (AMD) and diabetic macular edema (DME) –

‒ Vabysmo is currently approved in 60 countries to treat wet AMD and DME, with nearly one million doses distributed globally –

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) for Vabysmo^® (faricimab-svoa) for the treatment of macular edema following retinal vein occlusion (RVO). The sBLA is based on results from the Phase III BALATON and COMINO studies that demonstrated treatment with Vabysmo provided early and sustained improvement in vision, meeting the primary endpoint of non-inferior visual acuity gains at 24 weeks compared to aflibercept. Vabysmo’s safety profile was consistent with previous trials.

“This acceptance brings us one step closer to delivering Vabysmo as a treatment for retinal vein occlusion, a disease that affects more than one million people in the United States and can cause severe and sudden vision loss,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “If approved, this would be the third indication for Vabysmo, the first bispecific antibody available for the treatment of retinal conditions that can cause blindness.”

The data from the BALATON and COMINO studies will be submitted to other health authorities around the world, including the European Medicines Agency, for approval for the treatment of macular edema following RVO. The studies are ongoing, and data from weeks 24 to 72 will assess the potential of Vabysmo to extend dosing intervals up to every four months.

Vabysmo is the first bispecific antibody approved for the eye and was approved in the United States for the treatment of wet, or neovascular, age-related macular degeneration (AMD) and diabetic macular edema (DME) in January 2022. Vabysmo is approved in 60 countries, including Japan, the United Kingdom, and in the European Union, for wet AMD and DME. Wet AMD, DME, and RVO together affect around three million people in the United States and are among the leading causes of vision loss.

Vabysmo’s efficacy and safety profile in wet AMD and DME is supported by four large, global studies involving more than 3,000 participants and extensive real world experience, with nearly one million doses distributed globally. Vabysmo is the only injectable eye medicine approved for wet AMD and DME by the U.S. Food and Drug Administration (FDA) with the option for treatments from one to four months apart in the first year following four initial monthly loading doses, based on evaluation of the patient’s anatomy and vision outcomes. It targets and inhibits two disease pathways linked to a number of vision-threatening retinal conditions by neutralizing angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A).

About the BALATON and COMINO Studies

BALATON (NCT04740905) and COMINO (NCT04740931) are two randomized, multicenter, double-masked, global Phase III studies evaluating the efficacy and safety of Vabysmo^®️ (faricimab-svoa) compared to aflibercept. For the first 20 weeks, patients are randomized 1:1 to receive six monthly injections of either Vabysmo (6.0 mg) or aflibercept (2.0 mg). From weeks 24 to 72, all patients receive Vabysmo (6.0 mg) up to every four months – according to a personalized treatment interval dosing regimen – using a treat-and-extend approach.

The BALATON study is being conducted in 553 patients with branch retinal vein occlusion. The COMINO study is being conducted in 729 patients with central retinal or hemiretinal vein occlusion.

The primary endpoint of each study is the change in best-corrected visual acuity (BCVA) from baseline at 24 weeks. Secondary endpoints include change in central subfield thickness (CST) from baseline over time up to 24 weeks.

Both studies met their primary endpoint, with Vabysmo showing non-inferior visual acuity gains compared to aflibercept. The average vision gains from baseline were comparable between the two treatments in both studies. In BALATON, vision gains were +16.9 eye chart letters in the Vabysmo arm and +17.5 letters in the aflibercept arm at 24 weeks. In COMINO, vision gains were +16.9 letters in the Vabysmo arm and +17.3 letters in the aflibercept arm at 24 weeks.

A secondary endpoint showed that Vabysmo achieved rapid and robust drying of retinal fluid, as measured by reduction in CST from baseline. In both studies, reductions in CST were comparable across treatment arms. In BALATON, CST reductions from baseline at 24 weeks were 311.4 μm in the Vabysmo arm and 304.4 μm in the aflibercept arm. In COMINO, CST reductions from baseline at 24 weeks were 461.6 μm in the Vabysmo arm and 448.8 μm in the aflibercept arm.

Additionally, both studies showed that more Vabysmo patients had an absence of blood vessel leakage in the retina compared to aflibercept patients as seen in a pre-specified exploratory endpoint. Blood vessel leakage in the macula may lead to more retinal fluid, which can cause swelling and blurry vision.

In BALATON, one-third of patients (34%) treated with Vabysmo had an absence of macular leakage compared to one-fifth (21%) of aflibercept patients at 24 weeks. In COMINO, the rates were 44% for Vabysmo patients versus 30% for aflibercept patients at 24 weeks.

In both studies, Vabysmo’s safety profile was consistent with previous trials. The most common adverse reaction was conjunctival hemorrhage (3%). Safety results were consistent across study arms.

The studies are ongoing, and data from weeks 24 to 72 will assess the potential of Vabysmo to extend dosing intervals up to every four months.

About Retinal Vein Occlusion

Retinal vein occlusion (RVO) is the second most common cause of vision loss due to retinal vascular diseases. It affects more than one million people in the U.S., mainly those aged 50 or older, and can lead to severe and sudden vision loss. RVO typically results in sudden, painless vision loss in the affected eye because the vein blockage restricts normal blood flow in the affected retina, resulting in ischemia, bleeding, fluid leakage, and retinal swelling called macular edema. Currently, macular edema due to RVO is typically treated with repeated intravitreal injection of anti-vascular endothelial growth factor therapies. There are two main types of RVO: branch retinal vein occlusion (BRVO), which affects an estimated 887,000 people in the U.S. and occurs when one of the four smaller “branches” of the main central retinal vein becomes blocked; and central retinal vein occlusion (CRVO), which is less common, affecting an estimated 265,000 people in the U.S., and occurs when the eye’s central retinal vein becomes blocked.

About the Vabysmo^® (faricimab-svoa) Clinical Development Program

Genentech has a robust Phase III clinical development program for Vabysmo. The program includes AVONELLE-X, an extension study of TENAYA and LUCERNE evaluating the long-term safety and tolerability of Vabysmo in wet, or neovascular, macular degeneration (AMD), and RHONE-X, an extension study of YOSEMITE and RHINE evaluating the long-term safety and tolerability of Vabysmo in diabetic macular edema (DME). In addition, Genentech is investigating the efficacy and safety of Vabysmo in people with macular edema following retinal vein occlusion (RVO) in two Phase III studies, BALATON and COMINO. Genentech has also initiated several Phase IV studies, including the Elevatum study of Vabysmo in underrepresented patient populations with DME, the SALWEEN study of Vabysmo in a subpopulation of wet AMD highly prevalent in Asia, as well as the VOYAGER study, a global real-world data collection platform.

About Vabysmo^® (faricimab-svoa)

Vabysmo (faricimab-svoa) is the first bispecific antibody approved for the eye. It targets and inhibits two disease pathways linked to a number of vision-threatening retinal conditions by neutralizing angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A). While research is underway to better understand the role of the Ang-2 pathway in retinal disease, Ang-2 and VEGF-A are thought to contribute to vision loss by destabilizing blood vessels, which may cause new leaky blood vessels to form and increase inflammation. By blocking pathways involving Ang-2 and VEGF-A, Vabysmo is designed to stabilize blood vessels.

Vabysmo U.S. Indications

Vabysmo (faricimab-svoa) is a prescription medicine given by injection into the eye, used to treat adults with neovascular (wet) age‑related macular degeneration (AMD) and diabetic macular edema (DME).

Important Safety Information

Contraindications

Vabysmo is contraindicated in patients who have an infection in or around their eye, have active swelling around their eye that may include pain and redness, or are allergic to Vabysmo or any of the ingredients in Vabysmo.

Warnings and Precautions

• Injections like the one for Vabysmo can cause an eye infection (endophthalmitis) or separation of layers of the retina (retinal detachment). Patients should seek medical care if they experience increasing eye pain, vision loss, sensitivity to light, or redness in the white of the eye.
• Vabysmo may cause a temporary increase in pressure in the eye (intraocular pressure), which occurs 60 minutes after the injection.
• Although not common, Vabysmo patients have had serious, sometimes fatal, problems related to blood clots, such as heart attacks or strokes (thromboembolic events). In clinical studies for wet AMD during the first year, 7 out of 664 patients treated with Vabysmo reported such an event. In DME studies from baseline to week 100, 64 out of 1,262 patients treated with Vabysmo reported such an event.

Adverse Reactions

The most common adverse reactions (≥5%) reported in patients receiving Vabysmo were cataract (15%) and blood on the white of the eye (conjunctival hemorrhage, 7%). These are not all the possible side effects of Vabysmo.

Pregnancy, Lactation, Females and Males of Reproductive Potential

• Based on how Vabysmo interacts with your body, there may be a potential risk to an unborn baby. Patients should use birth control before their first injection, during their treatment with Vabysmo, and for 3 months after their last dose of Vabysmo.
• It is not known if Vabysmo passes into breast milk. Patients should talk to their healthcare provider about the best way to feed their baby if they receive Vabysmo.

Patients may report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. Patients may also report side effects to Genentech at (888) 835-2555.

Please see additional Important Safety Information in the full Vabysmo Prescribing Information or visit https://www.Vabysmo.com.

About Genentech in Ophthalmology

Genentech is researching and developing new treatments for people living with a range of eye diseases that cause significant visual impairment and blindness, including wet age-related macular degeneration (AMD), diabetic macular edema (DME), diabetic retinopathy (DR), geographic atrophy (GA) and other retinal diseases.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

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