Biogen Announces First Patient Treated With Higher Dose of SPINRAZA

Biogen announced that the first patient has been treated in the global clinical study, DEVOTE. The study is designed to evaluate the safety, tolerability and potential for even greater efficacy of SPINRAZA when administered at a higher dose than currently approved for the treatment of spinal muscular atrophy. The Phase 2/3 randomized, controlled, dose-escalating study will be conducted at approximately 50 sites around the world and aims to enroll individuals of all ages with SMA.

“Today, people of all ages with SMA are achieving better outcomes related to survival, mobility and independence, yet there is still no cure and unmet needs remain for many,” said Kenneth Hobby, President of Cure SMA, a patient advocacy organization dedicated to the treatment and cure of SMA. “Our community is energized by the advances we’ve made and believes that ongoing research like the DEVOTE study is instrumental to help deepen our understanding and potentially further transform and improve what it means to live with SMA.”

The three-part study will include an open-label safety evaluation cohort and a pivotal, double-blind, active control randomized treatment cohort followed by an open-label treatment cohort. After the safety evaluation cohort completes, the pivotal cohort will begin and include a treatment group receiving two loading doses of 50 milligrams (mg) 14 days apart, followed by a maintenance dose of 28 mg every four months. A second treatment group will receive the current U.S. Food and Drug Administration-approved administration of SPINRAZA, which is four loading doses with 12 mg maintenance doses every four months. The third cohort will be an open-label evaluation to assess the safety and tolerability of transitioning patients from the currently approved dose of SPINRAZA to the higher dose being tested in the study.

“As the first treatment for SMA, SPINRAZA fundamentally shifted the disease course compared to natural history, allowing many individuals to reach milestones that may have been previously unattainable,” said Alfred Sandrock Jr., M.D., Ph.D., Executive Vice President, Research and Development at Biogen. “With the success we’ve seen to date with the 12 mg dose, we are exploring a higher dose of SPINRAZA for the potential to deliver even better clinical outcomes.”

SPINRAZA is the first therapy approved to treat infants, children and adults with spinal muscular atrophy (SMA) and is approved in more than 50 countries. As of December 31, 2019, more than 10,000 individuals have been treated with SPINRAZA. It is the only SMA treatment to combine unsurpassed real-world experience with a robust level of clinical evidence across a broad spectrum of patient populations.

SMA is a rare, genetic, neuromuscular disease that is characterized by a loss of motor neurons in the spinal cord and lower brain stem that can result in severe, progressive muscle atrophy and weakness. Approximately one in 10,000 live births have a diagnosis of SMA, and people of all ages are impacted by the disease. It is a leading genetic cause of infant mortality.

SPINRAZA, a foundation of care in SMA, is an antisense oligonucleotide (ASO) developed using Ionis Pharmaceuticals’ proprietary technology that is designed to target a root cause of SMA by increasing the amount of full-length survival motor neuron (SMN) protein, which is critical to maintaining motor neurons. It is administered by intrathecal injection into the fluid surrounding the spinal cord where motor neurons reside to deliver the treatment where the disease starts.

SPINRAZA currently maintains a robust clinical data set in SMA, based on data from approximately 300 patients across a broad range of SMA populations, demonstrating a favorable benefit:risk profile. SPINRAZA was evaluated in two randomized, double-blind, sham-controlled studies of infantile and later-onset SMA (ENDEAR and CHERISH, respectively) and supported by open-label studies that include pre-symptomatic infants (NURTURE), individuals with later-onset SMA (CS2/CS12), and an extension study of individuals who previously participated in the clinical development program (SHINE). The most common adverse events observed were respiratory infection, fever, constipation, headache, vomiting and back pain. Hypersensitivity, meningitis and hydrocephalus have been observed in the post-marketing setting. Renal toxicity and coagulation abnormalities, including acute severe low platelet counts, have been observed after administration of some ASOs. Laboratory tests can monitor for these signs.

Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), a leader in antisense therapeutics. Biogen and Ionis conducted an innovative clinical development program that moved SPINRAZA from its first dose in humans in 2011 to its first regulatory approval in five years.

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