Genentech Announces Positive Results for First Global Phase III Study Investigating One-dose XOFLUZA (Baloxavir Marboxil) in Children With the Flu

August 2, 2019  Source: drugdu 458



– The MINISTONE-2 study showed XOFLUZA, given as a new oral suspension, is a well-tolerated and effective potential treatment for the flu in otherwise healthy children aged one to less than 12 years –

– Approximately one in three children develop the flu every year and they are often contagious longer than adults – treating children may therefore help reduce symptoms and prevent the spread of the flu to the wider community –

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Jul. 3, 2019-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the Phase III MINISTONE-2 study met its primary endpoint, demonstrating that XOFLUZA™ (baloxavir marboxil) was well-tolerated in children with the flu. The study also showed that XOFLUZA is comparable to oseltamivir – a proven effective treatment for children with the flu – at reducing the duration of flu symptoms, including fever. The study assessed XOFLUZA versus an active comparator (oseltamivir) in children aged between one and less than 12 years old with the flu. Full results from MINISTONE-2 will be presented at an upcoming medical meeting.

“Children need new medicines for the flu because they are at higher risk of developing the flu and more likely to have complications such as breathing problems and pneumonia. These flu complications, which in some cases can be fatal, lead to approximately one million children under five being admitted to the hospital globally every year,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “As a one-dose oral suspension medicine, XOFLUZA could potentially provide a convenient treatment option for children with the flu, and we look forward to sharing these data with health authorities around the world.”

The safety and efficacy of XOFLUZA in children with the flu under the age of one is also being studied in the global Phase III MINISTONE-1 study (NCT03653364). XOFLUZA is the first and only one-dose oral medicine approved to treat the flu and the first new flu medicine with a novel proposed mechanism of action approved by the U.S. Food and Drug Administration (FDA) in nearly 20 years. XOFLUZA is also the only flu treatment shown to be efficacious in both otherwise healthy people with the flu (CAPSTONE-1) and people at high risk of complications from the flu (CAPSTONE-2), as well as a preventive measure against developing the flu following exposure to an infected household member (BLOCKSTONE).

XOFLUZA is currently approved in several countries, including Japan for the treatment of influenza types A and B in children, adolescents and adults, and in the U.S. for the treatment of acute, uncomplicated influenza in people 12 years of age and older. In addition, the FDA recently accepted a supplemental New Drug Application (sNDA) for XOFLUZA as a one-dose oral treatment for people at high risk of complications from the flu. The FDA is expected to decide on whether to approve this additional indication by November 4, 2019.

About MINISTONE-2 (NCT03629184)

MINISTONE-2 is a Phase III, multicenter, randomized, double-blind study that evaluated the safety, pharmacokinetics and efficacy of one-dose of XOFLUZA compared with oseltamivir in otherwise healthy children aged one to less than 12 years with an influenza infection confirmed by a rapid influenza diagnostic test and displaying influenza-like symptoms (a temperature of 38°C or over, and one or more respiratory symptoms).

Participants enrolled in the study were recruited in parallel into two cohorts: patients aged five to less than 12 years and patients aged one to less than 5 years. Patients in both cohorts were randomly assigned to receive one-dose of XOFLUZA (2mg/kg for patients under 20kg or 40mg for patients 20kg or over) or oseltamivir twice a day over five days (dosing according to body weight). The primary endpoint of the study was the proportion of patients with adverse events or severe adverse events up to study day 29. Secondary endpoints include pharmacokinetics, time to alleviation of influenza signs and symptoms, and duration of symptoms, including fever.

About XOFLUZA™ (baloxavir marboxil)

XOFLUZA is a first-in-class, one-dose oral medicine with a novel proposed mechanism of action that has demonstrated efficacy in a wide range of influenza viruses, including in vitro activity against oseltamivir-resistant strains and avian strains (H7N9, H5N1) in non-clinical studies. Unlike other currently available antiviral treatments, XOFLUZA is the first in a new class of antivirals designed to inhibit the cap-dependent endonuclease protein, which is essential for viral replication.

XOFLUZA is being further studied in a Phase III development program, including children under the age of one (NCT03653364), and severely ill, hospitalized people with the flu (NCT03684044), as well as to assess the potential to reduce transmission in otherwise healthy people (NCT03969212).

XOFLUZA 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.) and Shionogi & Co., Ltd. Under the terms of this agreement, Roche holds worldwide rights to XOFLUZA excluding Japan and Taiwan, which will be retained exclusively by Shionogi & Co., Ltd.


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