Biogen announced the presentation of new data from its current treatments and investigational therapies for neurological and neurodegenerative diseases at the 69th annual meeting of the American Academy of Neurology (AAN) April 22-28 in Boston. The presentations will include new data on Spinraza.
Biogen will present data further demonstrating the effectiveness and safety of Spinraza (nusinersen), the only U.S. Food and Drug Administration (FDA)-approved treatment for spinal muscular atrophy (SMA) in pediatric and adult patients.
Spinraza was designed to treat SMA caused by mutations that lead to deficiencies in the survival motor neuron (SMN) protein. The treatment aims to increase the production of the full-length SMN protein by directly targeting the RNA and regulating gene expression.
Spinraza is administered via intrathecal injection, which enables direct administration to degenerated spinal motor neurons due to insufficient levels of SMN protein.
Biogen will show data from studies evaluating the safety and effectiveness of Spinraza in people who are most likely to develop SMA Types 1, 2 or 3. These studies include the CHERISH trial (NCT02292537) with individuals with later-onset SMA (most likely to develop SMA types 2 or 3) and the NURTURE study (NCT02386553) in pre-symptomatic infants genetically diagnosed with SMA. The company states that the new results contrast with the progressive decline in motor function and failure to achieve motor milestones after symptom onset that are typically observed in SMA.
The Biogen presentation will also focus on Tecfidera (dimethyl fumarate), a prescribed oral medicine for multiple sclerosis (MS), including relapsing-remitting MS (RRMS), the most common form. Biogen will now show that, compared to other oral MS therapies, Tecfidera has strong and sustained effectiveness in patients early in the course of the disease, including newly diagnosed patients or those previously treated with another therapy.
The new data also includes Tysabri (natalizumab), a globally-approved disease-modifying therapy for patients with RRMS, including adults with a highly active form of the disease. Although the drug’s mechanisms have not been fully defined, Tysabri is thought to block the activity of inflammatory cells in MS patients.
Biogen will show “real-world” evidence supporting Tysabri’s high-efficacy in the early treatment of MS. These new results contribute to previous demonstrations of the drug’s ability to hamper the progression of disability, reduce relapse rates, and impact the number of brain lesions.
Finally, Biogen will also present data regarding aducanumab (BIIB037), an investigational therapy for early Alzheimer’s disease. The drug is currently being evaluated in two global Phase 3 studies, ENGAGE (NCT02477800) and EMERGE (NCT02484547).
“Biogen’s commitment to improving outcomes for people living with MS spans more than two decades, during which time we’ve fundamentally changed the treatment of the disease. We are now applying our expertise in neurology to discover and develop new therapies to address some of the most challenging and complex diseases of the brain,” Alfred Sandrock, MD, PhD, executive vice president and chief medical officer at Biogen, said in a press release.
“The data from our MS, SMA and Alzheimer’s disease programs reflect our desire to advance the understanding of these diseases and make a meaningful difference in the lives of patients,” he said.