December 22, 2016
By Alex Keown, BioSpace.com Breaking News Staff
SOUTH SAN FRANCISCO, Calif. – Genentech and its parent company Roche received an early Christmas present after the company announced emicizumab, its experimental Phase III hemophilia A drug, achieved its trial endpoints.
This morning the company announced use of the drug in people 12 years of age or older with hemophilia A and inhibitors to factor VIII showed a statistically significant reduction in the number of bleeds over time compared to those receiving no prophylactic treatment. Not only did the drug meet its primary objectives, but Genentech said secondary endpoints, including a statistically significant reduction in the number of bleeds over time with emicizumab prophylaxis treatment in an intra-patient comparison in people who had received prior bypassing agent prophylaxis treatment, were also met.
“The development of inhibitors that render factor VIII replacement less effective, or ineffective, is one of the greatest challenges in the treatment of hemophilia A today, putting patients at high risk for life-threatening bleeds and repeated bleeds that may cause long-term joint damage,” Sandra Horning, Genentech’s chief medical officer and head of global product development, said in a statement. “We are pleased to see that, in our first pivotal trial, emicizumab prophylaxis significantly reduced the number of bleeds over time in people in this difficult-to-treat setting. We look forward to working with health authorities to bring this treatment to the hemophilia community as soon as possible.”
Emicizumab is a bispecific antibody engineered to bind both factors IXa and X, replacing the function of the missing factor VIII to improve clotting function and prevent spontaneous bleeding, according to Roche. Additionally, the therapy is designed to promote blood coagulation in hemophilia A patients, regardless of whether they have developed inhibitors to factor VIII, the company said. The drug earned the Breakthrough Therapy Designation from the U.S. Food and Drug Administration.
Hemophilia A affects around 320,000 people worldwide, approximately 50-60 percent of whom have a severe form of the disorder. People with hemophilia A either lack or do not have enough of a clotting protein called factor VIII.
The positive news comes nearly two months after it was reported that four patients taking part in the trial suffered serious adverse events. The patients were taking emicizumab, which is also called ACE910, and developed serious cases of blood clots. Roche disclosed two of the patients developed thromboembolic events and two patients developed thrombotic microangiopathy (TMA).
In its statement this morning, Genentech said the common aspect between all cases of thromboembolic events and TMA is that they occurred in patients who were on emicizumab prophylaxis and in addition received activated prothrombin complex concentrate to treat breakthrough bleeds. Neither thromboembolic event required anti-coagulation therapy and one patient restarted emicizumab. Both cases of TMA have completely resolved, and one patient restarted emicizumab, Genentech said this morning.
The news puts emicizumab one step closer to battling out rival drugs FEIBA and NovoSeven, developed by Shire Pharmaceuticals and Novo Nordisk respectively, for a share of the market.
Biogen is also planning to make a splash in the hemophilia market with its new dedicated spinoff company, Bioverativ. Bioverativ will be focused on the discovery, research, development and commercialization of treatments for hemophilia and other blood disorders. The company will launch with two approved hemophilia drugs, Alprolix and Electate, which generated $500 million in revenue in 2015. Analysts have projected the two drugs could ultimately hit $1 billion in annual revenue. Both drugs were approved in 2014 for the treatments of hemophilia B and A, respectively.
