FDA Accepts Application for Genentech’s Port Delivery System With Ranibizumab (PDS) for Treatment of Wet Age-Related Macular Degeneration (AMD)

‒ If approved, PDS would be the first and only eye implant with continuous drug delivery that offers people living with wet AMD an alternative to frequent eye injections –

‒ A pivotal study showed PDS extends time between treatments up to six months for more than 98% of patients and provides vision outcomes equivalent to monthly ranibizumab injections –

‒ The European Medicines Agency has also validated the PDS Marketing Authorization Application in wet AMD –

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 Biologics License Application (BLA), under Priority Review, for Port Delivery System with ranibizumab (PDS) for the treatment of wet, or neovascular, age-related macular degeneration (AMD). Wet AMD is a leading cause of blindness for people aged 60 and over and impacts approximately 1.1 million people in the United States. If approved, PDS would be a first-of-its-kind therapeutic approach, offering people living with wet AMD an alternative to frequent eye injections of anti-vascular endothelial growth factor (VEGF), the current standard of care. The FDA is expected to make a decision on approval by Oct. 23, 2021.

“Anti-VEGF therapy brings significant benefit to people with wet AMD, but optimal results require frequent trips to the doctor’s office for eye injections. This burden leaves many people under-treated and susceptible to vision loss,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “If approved, PDS would transform wet AMD treatment by providing up to six months of uninterrupted therapy that could potentially improve vision outcomes compared to what is currently achieved in the clinic.”

PDS is a permanent refillable eye implant, approximately the size of a grain of rice, designed to continuously deliver a customized formulation of ranibizumab over a period of months, potentially reducing the treatment burden associated with frequent eye injections.

The BLA submission is based on positive results from the Phase III Archway study primary analysis, which showed that of those wet AMD patients being treated with PDS, more than 98% were able to go six months without needing additional treatment prior to the refill exchange. In addition, these patients achieved vision outcomes equivalent to patients receiving monthly ranibizumab eye injections. In the study, PDS was generally well-tolerated, with a favorable benefit-risk profile. The safety profile of PDS in the clinical trial setting is well understood and will continue to be closely monitored. If approved, PDS would be the first and only wet AMD therapy indicated to allow six months between treatments.

Genentech has a robust Phase III clinical development program underway for PDS, including the Portal, Pagoda and Pavilion studies. Portal is an extension study evaluating the long-term safety and efficacy of PDS in wet AMD. Pagoda is evaluating PDS for the treatment of diabetic macular edema (DME), while Pavilion is a study of PDS in diabetic retinopathy without DME. Both the Pagoda and Pavilion trials are actively recruiting participants.

The PDS Marketing Authorization Application has also been validated by the European Medicines Agency and is currently under review.

About the Archway Study

Archway (NCT03677934) is a randomized, multicenter, open-label Phase III study evaluating the efficacy and safety of Port Delivery System with ranibizumab (PDS), refilled every six months at fixed intervals, compared to monthly intravitreal injections of ranibizumab 0.5 mg in 418 people living with wet age-related macular degeneration (AMD). Patients enrolled in Archway were responders to prior treatment with anti-vascular endothelial growth factor (VEGF) therapy. In both study arms, patients were treated with at least three anti-VEGF injections within the six months prior to their Archway screening visit. The primary endpoint of the study is the change in best-corrected visual acuity (BCVA) score (the best distance vision a person can achieve – including with correction such as glasses – when reading letters on an eye chart) from baseline at the average of Week 36 and Week 40. Secondary endpoints include safety, overall change in BCVA from baseline and change from baseline in center point thickness over time.

According to pre-specified study criteria, PDS was shown to be non-inferior and equivalent to monthly ranibizumab injections. On average, patients had received five prior ranibizumab injections before their first Archway visit. In the PDS arm of the study, patients gained an average of 0.2 eye chart letters in visual acuity from baseline compared with 0.5 eye chart letters for the monthly ranibizumab arm. During the first treatment interval, before the first scheduled refill, 1.6% of PDS patients assessed (n=4/246) received supplemental treatment, and 98.4% of patients (n=242/246) did not receive supplemental treatment.

In addition, PDS controlled retinal thickness as effectively as monthly ranibizumab, with patients in both arms achieving a mean change in center point thickness within 10 μm from baseline at Week 36. In the study, PDS was generally well-tolerated, with a favorable benefit-risk profile. The safety profile of PDS in the clinical trial setting is well understood and will continue to be closely monitored.

About Wet Age-Related Macular Degeneration

Age-related macular degeneration (AMD) is a condition that affects the macula, the part of the eye that provides sharp, central vision needed for activities like reading, and is a leading cause of blindness for people aged 60 and over in the United States. Wet, or neovascular, AMD is an advanced form of the disease that can cause rapid and severe vision loss. Approximately 11 million people in the United States have some form of AMD, and of those, about 1.1 million have wet AMD.

Wet AMD is caused by growth of abnormal blood vessels, also referred to as choroidal neovascularization (CNV), into the macula. These vessels leak fluid and blood and cause scar tissue that destroys the central retina. This process results in a deterioration of sight over a period of months to years.

About Port Delivery System with ranibizumab (PDS)

Port Delivery System with ranibizumab (PDS) is a permanent refillable eye implant, approximately the size of a grain of rice, which is designed to continuously release a customized formulation of ranibizumab into the eye over time. Ranibizumab is a vascular endothelial growth factor (VEGF) inhibitor designed to bind to and inhibit VEGF-A, a protein that is believed to play a critical role in the formation of new blood vessels and the leakiness of the vessels. PDS contains a customized formulation of ranibizumab not approved by the U.S. Food and Drug Administration (FDA). It is different from the ranibizumab intravitreal injection, a medicine marketed as Lucentis^® (ranibizumab injection), which is FDA-approved to treat wet age-related macular degeneration (AMD) and other retinal diseases.

By maintaining therapeutic drug concentration levels of ranibizumab with two refills per year, PDS may offer greater outcomes certainty in terms of vision gains and maintaining those gains for people living with retinal diseases, including wet AMD. Additionally, by decreasing the need for frequent injections and physician visits, PDS may reduce the burden of treatment associated with standard anti-VEGF treatments.

About Lucentis^® (ranibizumab injection)

Lucentis is a vascular endothelial growth factor (VEGF) inhibitor designed to bind to and inhibit VEGF-A, a protein that is believed to play a critical role in the formation of new blood vessels (angiogenesis) and the hyperpermeability (leakiness) of the vessels.

Lucentis is FDA-approved for the treatment of patients with wet age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), diabetic retinopathy (DR) and myopic choroidal neovascularization (mCNV).

Lucentis was developed by Genentech, a member of the Roche Group. The company retains commercial rights in the United States and Novartis has exclusive commercial rights for the rest of the world.

Outside the United States, Lucentis is approved in more than 120 countries to treat adult patients with wet AMD, for the treatment of visual impairment due to DME, due to macular edema secondary to both branch retinal vein occlusion (BRVO), central retinal vein occlusion (CRVO) and due to choroidal neovascularization (CNV).

Lucentis Important Safety Information

Patients should not use Lucentis if they have an infection in or around the eye or are allergic to Lucentis or any of its ingredients.

Lucentis is a prescription medication given by injection into the eye, and it has side effects. Some Lucentis patients have had detached retinas and serious infections inside the eye. If your eye becomes red, sensitive to light, or painful, or if there is a change in vision, call or visit your eye doctor right away.

Some patients have had increased eye pressure before and within 1 hour of an injection.

Uncommonly, Lucentis patients have had serious, sometimes fatal, problems related to blood clots, such as heart attacks or strokes.

Fatal events were seen more often in patients with DME and DR with Lucentis compared with patients who did not receive Lucentis. Although there were only few fatal events which included causes of death typical of patients with advanced diabetic complications, these events may be caused by Lucentis.

Some Lucentis patients have serious side effects related to the injection. These include serious infections inside the eye, detached retinas, and cataracts. The most common eye-related side effects are increased redness in the white of the eye, eye pain, small specks in vision, and increased eye pressure. The most common non–eye related side effects are nose and throat infections, anemia, nausea and cough.

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.

For additional safety information, please see Lucentis full Prescribing Information, available here: http://www.gene.com/download/pdf/lucentis_prescribing.pdf

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. The company is also investigating platforms for sustained ocular drug delivery, including Port Delivery System with ranibizumab (PDS).

Genentech’s parent company, Roche, is investigating a bispecific antibody for the treatment of retinal eye 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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