Positive Phase III Results for Baloxavir Marboxil in People at High Risk of Complications From Influenza to be Presented at IDWeek 2018
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Oct 4, 2018--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the Phase III CAPSTONE-2 study showed treatment with baloxavir marboxil significantly reduced the time to improvement of influenza symptoms versus placebo (median time of 73.2 hours versus 102.3 hours; p<0.0001) in people at high risk of serious complications from the flu, which includes adults 65 years of age or older, or those who have conditions such as asthma, chronic lung disease, morbid obesity, or heart disease. Baloxavir marboxil was well-tolerated and no new safety signals were identified. Results of the study will be presented as a late-breaking oral presentation during IDWeek 2018 in San Francisco, California on Saturday, October 6, 2018 (Abstract #LB16). Baloxavir marboxil was discovered and developed by Shionogi & Co., Ltd., and is sold in Japan under the trade name Xofluza ®.
“This is the first Phase III trial to demonstrate a significant, clinically meaningful benefit in people at high risk for complications from the flu for which there are no currently approved medicines,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “This study adds to the growing body of evidence supporting baloxavir marboxil as a potential first-in-class antiviral flu treatment, and we plan to discuss these data with health authorities around the world.”
The CAPSTONE-2 study also showed that baloxavir marboxil demonstrated efficacy (reduced time to improvement of influenza symptoms) in influenza type A/H3N2 (median time of 75.4 hours and 100.4 hours; p<0.05) and type B (median time of 74.6 hours and 100.6 hours; p<0.05) versus placebo. In addition, results for the overall patient population of the study showed numerically shorter time to improvement of influenza symptoms of baloxavir marboxil versus oseltamivir with a median time to improvement of symptoms of 73.2 hours for baloxavir marboxil compared with 81.0 hours for oseltamivir (p=0.8347). In the subpopulation of people with influenza type B, a subgroup where some antiviral treatments have shown only limited efficacy or inconclusive data, baloxavir marboxil was significantly more efficacious than oseltamivir in reducing the time to improvement of symptoms (median time of 74.6 hours versus 101.6 hours; p<0.05).
Baloxavir marboxil demonstrated efficacy compared to placebo and oseltamivir for key secondary endpoints, including reducing the time that the virus continued to be released from the body (viral shedding; median time of 48.0 hours for baloxavir marboxil versus 96.0 hours for both placebo and oseltamivir; p<0.0001). Baloxavir marboxil also reduced the use of antibiotics and incidence of influenza-related complications (3.4 percent and 2.8 percent respectively) compared to placebo (7.5 percent and 10.4 percent; p=0.01 and p<0.05). Baloxavir marboxil had a numerically lower overall incidence of reported adverse events (25.1 percent) compared with placebo (29.7 percent) or oseltamivir (28.0 percent).
Baloxavir marboxil is a single-dose, investigational oral medicine that represents the first of a new class of antivirals with a novel proposed mechanism of action that differs from other currently available treatments. Baloxavir marboxil has already demonstrated a clinically significant benefit over placebo in otherwise healthy people with influenza in the Phase III CAPSTONE-1 study. The U.S. Food and Drug Administration (FDA) accepted a New Drug Application (NDA) and granted Priority Review to baloxavir marboxil as a single-dose, oral treatment for acute, uncomplicated influenza in people 12 years and older based on the results from CAPSTONE-1 and an earlier Phase II study. The FDA is expected to make a decision on approval by December 24, 2018. If approved, baloxavir marboxil will be the first single-dose oral antiviral, and the first medicine with a novel proposed mechanism of action to treat the flu in nearly 20 years.
Genentech also announced that they have entered into a private-public partnership with the Biomedical Advanced Research and Development Authority (BARDA) of the U.S. Department of Health and Human Services (HSS), to advance the development of medicines for infectious diseases for which there is a significant unmet need. As part of the agreement, BARDA will provide funding that will support the development of baloxavir marboxil for severely ill hospitalized people with influenza, with the potential for funding of other studies.
CAPSTONE-2 is a Phase III, multicenter, randomized, double-blind study that evaluated the efficacy and safety of a single dose of baloxavir marboxil compared with placebo and oseltamivir in people 12 years or older who are at a high risk of complications from the flu. The Centers for Disease Control and Prevention (CDC) defines people at high risk for serious flu complications to include adults 65 years of age or older, or those who have conditions such as asthma, chronic lung disease, morbid obesity or heart disease. The study was conducted globally by Shionogi & Co., Ltd.
A total of 2,184 participants enrolled in the study and were randomly assigned to receive a single, oral dose of 40 mg or 80 mg of baloxavir marboxil (according to body weight), placebo or 75 mg of oseltamivir twice daily for five days. Among them, 1,163 people (53 percent) were confirmed to have influenza virus infection with RT-PCR (influenza virus subtype: 47.9 percent for A/H3N2, 6.9 percent for A/H1N1, 41.6 percent for B). The most common risk factors were asthma or chronic lung disease (39.2 percent), age ≥65 years (27.4 percent), endocrine disorders (32.8 percent), metabolic disorders (13.5 percent), heart disease (12.7 percent) and morbid obesity (10.6 percent). The primary objective of the study evaluated the efficacy of a single dose of baloxavir marboxil compared with placebo by measuring the time to improvement of influenza symptoms. Key secondary endpoints compared outcomes in baloxavir marboxil versus placebo or oseltamivir – these included time to resolution of fever, time to cessation of viral shedding, infectious virus detection in swabs of the nose and throat, prescription of antibiotics and influenza-related complications.
About baloxavir marboxil
Baloxavir marboxil is a first-in-class, single-dose investigational oral medicine with a novel proposed mechanism of action designed to target the influenza (“flu”) A and B viruses, including oseltamivir-resistant strains and avian strains (e.g. H7N9, H5N1). Unlike other currently available antiviral treatments, baloxavir marboxil is the first in a new class of antivirals designed to inhibit the cap-dependent endonuclease protein within the influenza virus, preventing the virus from replicating in the body early in its lifecycle.
In the Phase III CAPSTONE-1 study in otherwise healthy people with flu, conducted globally by Shionogi & Co., Ltd., baloxavir marboxil demonstrated a clinically significant benefit over placebo. Baloxavir marboxil will also be further studied in a Phase III development program including pediatric populations and severely ill hospitalized people with influenza.
Baloxavir marboxil was discovered by Shionogi & Co., Ltd. and is being further developed and commercialized globally in collaboration with the Roche Group (which includes Genentech in the U.S.). Under the terms of this agreement, Roche holds worldwide rights to baloxavir marboxil excluding Japan and Taiwan, which will be retained exclusively by Shionogi & Co., Ltd. Baloxavir marboxil was approved in February 2018 by the Japanese Ministry of Health, Labour and Welfare for the treatment of influenza types A and B in adult and pediatric populations, and is being commercialized in Japan and marketed under the brand name Xofluza ®.
About Genentech in influenza
Influenza, or flu, is one of the most common yet serious infectious diseases. Globally, seasonal epidemics result in 3 to 5 million cases of severe disease, millions of hospitalizations and up to 650,000 deaths worldwide. Although vaccines are an important first line of defense in preventing the flu, there is a need for new medical options for prophylaxis and treatment. Current antiviral drugs have limitations with respect to efficacy, convenience of dosing and resistance. Genentech is committed to addressing the unmet need in this area through its agreement with Shionogi to develop and commercialize baloxavir marboxil.
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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PUB: 10/04/2018 01:00 AM/DISC: 10/04/2018 01:01 AM