New SPINRAZA® (nusinersen) Data Reinforce Sustained Efficacy and Longer-Term Safety Across Broad Range of Spinal Muscular Atrophy (SMA) Patients
CAMBRIDGE, Mass., May 18, 2020 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced additional data from the SPINRAZA (nusinersen) clinical development program that further demonstrate the sustained efficacy and longer-term safety of SPINRAZA in a broad range of patients with spinal muscular atrophy (SMA). These new data were selected for presentation at the 72nd American Academy of Neurology (AAN) annual meeting and will be available online via the 2020 AAN Science Highlights virtual platform.
“As the first approved treatment for SMA, SPINRAZA offers a significant data set that allows us to uniquely assess the safety and durability of repeated doses over time in individuals across age groups and varying disease severity,” said Alfred Sandrock, Jr., M.D., Ph.D., Executive Vice President, Research and Development at Biogen. “New data show that continuous treatment with SPINRAZA for up to six and a half years improved or stabilized motor function and disease activity in a broad spectrum of patients with SMA. These results are in stark contrast to the expected natural history of the disease. Further, in a progressive disease like SMA, stabilization is an important measure of treatment success, allowing patients to retain motor function that may otherwise be lost.”
New Data Reinforce Sustained Efficacy and Longer-Term Safety of SPINRAZA Across Age Groups and SMA Types
Key highlights include:
AAN data presentations highlighted in this release include:
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 March 31, 2020, 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.
We routinely post information that may be important to investors on our website at www.biogen.com. To learn more, please visit www.biogen.com and follow us on social media – Twitter, LinkedIn, Facebook, YouTube.
Biogen Safe Harbor
These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation the occurrence of adverse safety events; risks of unexpected costs, delays or other unexpected hurdles; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; regulatory authorities may require additional information or further studies; failure to obtain regulatory approvals in other jurisdictions; product liability claims; third party collaboration risks; and the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations and financial condition. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this news release. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.