Roche’s Fenebrutinib Demonstrates Promising Results with Low Relapse Rates and No Disability Progression in Phase II Multiple Sclerosis Study

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(IN BRIEF) Roche presented promising 96-week data from the Phase II FENopta study, showing that its investigational drug, fenebrutinib, significantly reduced relapse rates and prevented disability progression in patients with relapsing multiple sclerosis (RMS). The study revealed an annualized relapse rate of 0.06, equivalent to one relapse every 17 years, with no new T1 gadolinium-enhancing lesions detected. The drug also demonstrated a decrease in chronic disease burden, as shown by fewer new or enlarging T2 lesions. Fenebrutinib was well tolerated, with no new safety concerns. Phase III studies are ongoing, with results expected by the end of 2025.

(PRESS RELEASE) BASEL, 30-May-2025 — /EuropaWire/ — Roche (SIX: RO, ROG; OTCQX: RHHBY) has revealed promising new data for its investigational drug, fenebrutinib, from the Phase II FENopta open-label extension (OLE) study. These results demonstrate that patients with relapsing multiple sclerosis (RMS) who were treated with fenebrutinib showed a significant reduction in relapse rates and maintained a stable level of disability over the course of up to two years. The data, presented at the Consortium of Multiple Sclerosis Centers (CMSC) Annual Meeting in Phoenix, Arizona, highlight fenebrutinib’s potential in treating RMS.

The data from the 96-week study show that patients on fenebrutinib experienced an annualized relapse rate (ARR) of just 0.06, which equates to a relapse every 17 years, and no disability progression as measured by the Expanded Disability Status Scale (EDSS). MRI scans also demonstrated the suppression of disease activity in the brain, with no new T1 gadolinium-enhancing (T1-Gd+) lesions—indicative of active inflammation—detected at 96 weeks. Additionally, patients who switched from placebo to fenebrutinib showed a marked decrease in the annualized rate of new or enlarging T2 lesions, further indicating a reduction in chronic disease burden.

“This data is encouraging as it suggests that fenebrutinib can significantly reduce disease activity and prevent disability progression in RMS,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “Fenebrutinib is the only reversible Bruton’s tyrosine kinase (BTK) inhibitor currently in Phase III trials for multiple sclerosis, and we are eager to see the results of these trials later this year.”

Fenebrutinib was well tolerated in both AERIFY-1 and AERIFY-2 studies, with no new safety concerns emerging over the 96 weeks of treatment. The most common adverse events reported were COVID-19 (10%), urinary tract infections (10%), and respiratory tract infections (5%). Serious adverse events were low, with two patients in the study group experiencing such incidents.

The ongoing Phase III trials, including FENhance 1 and 2 in RMS and FENtrepid in primary progressive multiple sclerosis (PPMS), are expected to provide further insights into fenebrutinib’s potential across the full spectrum of multiple sclerosis. Initial data from these studies is expected by the end of 2025.

About fenebrutinib
Fenebrutinib is an investigational oral, reversible and non-covalent Bruton’s tyrosine kinase (BTK) inhibitor that blocks the function of BTK. BTK, also known as tyrosine-protein kinase BTK, is an enzyme that regulates B-cell development and activation and is also involved in the activation of innate immune system myeloid lineage cells, such as macrophages and microglia. Preclinical data have shown fenebrutinib to be potent and highly selective, and it is the only reversible BTK inhibitor currently in Phase III trials for multiple sclerosis. Fenebrutinib has been shown to be 130 times more selective for BTK vs. other kinases.

Fenebrutinib is a dual inhibitor of both B-cell and microglia activation. This dual inhibition may be able to reduce both multiple sclerosis disease activity and disability progression, thereby potentially addressing the key unmet medical need of disability progression in people living with multiple sclerosis. The fenebrutinib Phase III programme includes two identical trials in relapsing multiple sclerosis (RMS) (FENhance 1 & 2) with active comparator teriflunomide and the only trial in primary progressive multiple sclerosis (PPMS) (FENtrepid) in which a BTK inhibitor is being evaluated against OCREVUS. To date, more than 2,700 patients and healthy volunteers have been treated with fenebrutinib in Phase I, II and III clinical programmes across multiple diseases, including multiple sclerosis and other autoimmune disorders.

About the FENopta study
The FENopta study was a global Phase II, randomised, double-blind, placebo-controlled 12-week study to investigate the efficacy, safety and pharmacokinetics of fenebrutinib in 109 adults aged 18-55 years with relapsing multiple sclerosis (RMS). The primary endpoint was the total number of new T1 gadolinium-enhancing (T1-Gd+) lesions as measured by MRI scans of the brain at 4, 8 and 12 weeks. Secondary endpoints included the number of new or enlarging T2-weighted lesions as measured by MRI scans of the brain at 4, 8 and 12 weeks, and the proportion of patients free from any new T1-Gd+ lesions and new or enlarging T2-weighted lesions as measured by MRI scans of the brain at 4, 8 and 12 weeks. The goal of the FENopta study was to characterise the effect of fenebrutinib on MRI and soluble biomarkers of disease activity and progression, and it included an optional substudy to measure cerebrospinal fluid fenebrutinib levels and biomarkers of neuronal injury.

Data from the 12-week study showed that fenebrutinib is central nervous system (CNS) penetrant (crosses the blood-brain barrier) and has the potential to impact mechanisms underlying chronic progressive disease biology in multiple sclerosis patients. Fenebrutinib significantly reduced new T1-Gd+ lesions and new/enlarging T2 lesions compared to placebo. The safety profile of fenebrutinib was consistent with previous and ongoing fenebrutinib clinical trials and there were no new safety concerns identified.

Patients who completed the FENopta study were given the option to take part in an open-label extension (OLE) study, in which all patients receive fenebrutinib up to 192 weeks.

About multiple sclerosis
Multiple sclerosis is a chronic disease that affects more than 2.9 million people worldwide. Multiple sclerosis occurs when the immune system abnormally attacks the insulation and support around nerve cells (myelin sheath) in the central nervous system (brain, spinal cord and optic nerves), causing inflammation and consequent damage. This damage can cause a wide range of symptoms, including weakness, fatigue and difficulty seeing, and may eventually lead to disability. Most people with multiple sclerosis experience their first symptom between 20 and 40 years of age, making the disease the leading cause of non-traumatic disability in younger adults.

People with all forms of multiple sclerosis experience disease progression from the beginning of their disease. Therefore, delays in diagnosis and treatment can negatively impact people with multiple sclerosis, in terms of their physical and mental health, and contribute to the negative financial impact on the individual and society. An important goal of treating multiple sclerosis is to slow, stop and ideally prevent progression as early as possible.

Approximately 85% of people with multiple sclerosis have a relapsing form of the disease (RMS) characterised by relapses and also worsening disability over time. Primary progressive multiple sclerosis (PPMS) is a debilitating form of the disease marked by steadily worsening symptoms but typically without distinct relapses or periods of remission. Approximately 15% of people with multiple sclerosis are diagnosed with the primary progressive form of the disease. Until the FDA approval of OCREVUS, there had been no FDA-approved treatments for PPMS and OCREVUS is still the only approved treatment for PPMS.

About Roche in Neuroscience
Neuroscience is a major focus of research and development at Roche. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases.

Roche is investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today.

About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.

For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045.

Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan.

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SOURCE: Roche