Roche Scores Wins in AMD & COVID-19 in Big Week
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Wet age-related macular degeneration is a leading cause of blindness in people over the age of 60. Genentech believes its investigational Port Delivery System with a ranibizumab (PDS) could be the first implant treatment for Wet AMD patients that provides continual drug delivery without the need for frequent injections.
On Thursday, Genentech, a Roche subsidiary, announced the U.S. Food and Drug Administration (FDA) accepted the company’s Biologics License Application under Priority Review. The regulatory agency is eying a decision by Oct. 23.
If approved, Genentech said PDS would reduce the need for doctor’s visits for patients who require an injection once per month. Citing a survey from patients who participated in the Phase III ARCHWAY trial the BLA is based upon, Genentech said 93.2% of patients preferred PDS over intravitreal injections, with the most common reasons being fewer treatments and less discomfort.
PDS is a permanent refillable eye implant that is designed to continuously deliver a customized formulation of ranibizumab over a period of several months, intending to reduce the treatment burden associated with frequent eye injections. Ranibizumab is a vascular endothelial growth factor (VEGF) inhibitor designed to bind to VEGF-A, a protein believed to play a critical role in forming new blood vessels.
Ranibizumab intravitreal injection, marketed as Lucentis, is the current standard of care for Wet AMD, caused by abnormal blood vessel growth. Levi Garraway, chief medical officer and head of Global Product Development at Roche, said if approved, PDS will revolutionize wet AMD treatment by delivering uninterrupted therapy for a six month period. Garraway said this could potentially improve vision outcomes in the 1.1 million Wet AMD patients in the United States.
“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,” Garraway said in a statement.
The BLA is based on positive results from the ARCHWAY study that showed more than 98% of Wet AMD patients who received the PDS implant were able to go six months without needing additional treatment. Genentech said patients who received the PDS implant experienced vision outcomes equivalent to patients receiving monthly ranibizumab eye injections.
PDS also controlled retinal thickness as effectively as monthly ranibizumab. PDS was generally well-tolerated, with a favorable benefit-risk profile. According to pre-specified study criteria, PDS was shown to be non-inferior and equivalent to monthly ranibizumab injections.
The PDS announcement was not Roche’s only news Thursday. The company also announced that the FDA granted Emergency Use Authorization for intravenous Actemra, an IL-6 inhibitor, as a treatment for COVID-19 patients ages two and up who are hospitalized and receiving systemic corticosteroids and require supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO).
The EUA is based on four randomized, controlled studies that assessed Actemra, a rheumatoid arthritis drug, in COVID-19. Those results suggest that Actemra may improve outcomes in patients who are in the previously defined treatment population.
One of those studies, EMPACTA, demonstrated that Actemra was effective in minority patients with COVID-19-associated pneumonia. In addition, the data showed that Actemra-treated patients were 44% less likely to progress to mechanical ventilation or death compared to patients who received placebo plus standard of care.
The positive EMPACTA results contrast to the disappointing Phase III COVACTA study that assessed Actemra as a treatment for hospitalized patients with severe COVID-19 associated pneumonia. The COVACTA study failed to hit both primary and secondary endpoints in that patient population.
Also this week, Genentech announced it was launching the Advancing Inclusive Research Site Alliance, an initiative aimed at improving the representation of diverse patient populations in clinical trials. The company said the effort will establish best practices to help achieve health equity for people with cancer.
In its announcement, Genentech said fewer than 10% of the U.S. patient population participates in clinical trials. Of those, between 5% and 15% are minorities, despite making up more than 40% of the national population.
Genentech noted that African American women breast cancer patients have a 40% higher mortality rate compared to Caucasian women. However, black women only represent about 6% of clinical patients.
There are numerous reasons for the lack of minority participants, but the primary drivers for these disparities include distrust in the healthcare system, lack of access to nearby trial sites, and insufficient engagement with underserved communities.
Quita Highsmith, chief diversity officer at Genentech, said the company must engage with these disenfranchised patient communities to “ensure the most representative and effective clinical research and achieve optimal treatment outcomes for all.”
Lastly, Roche is stepping away from its gene therapy partnership with 4D Molecular Therapeutics. The Swiss pharma giant sent a notice of termination without cause to California-based 4D to end its involvement in the 4D-110 trial in advanced choroideremia patients. In its announcement, 4D said the company stepped away following an assessment of a change in the risk-benefit profile. Shares of 4D fell more than 10% Thursday after the revelation.