New Results From Landmark NURTURE Study Show That Pre-Symptomatic SMA Patients Treated With SPINRAZA (nusinersen) Continue to Demonstrate Sustained…

By daniellenierenberg

DetailsCategory: DNA RNA and CellsPublished on Wednesday, 10 June 2020 16:58Hits: 131

CAMBRIDGE, MA, USA I June 10, 2020 I Biogen Inc.(Nasdaq: BIIB) today announced new results from NURTURE, the longest study of pre-symptomatic patients with spinal muscular atrophy (SMA) that is transforming expectations of early treatment with SPINRAZA (nusinersen). In infants genetically diagnosed with SMA, new data demonstrate that early and sustained treatment with SPINRAZA for up to 4.8 years enabled unprecedented survival. Patients continued to maintain and make progressive gains in motor function compared to the natural course of the disease. These results are being presented at the virtual Cure SMA Research & Clinical Care Meeting taking place June 10-12, 2020.

The new data include nearly a year of additional follow-up for NURTURE study participants. As of February 2020, all patients treated (n=25; median age of 3.8 years old) were alive and remained free of permanent ventilation. In the absence of treatment, the majority of children with SMA Type 1 would, on average, not reach their second birthday. Additionally, all children who achieved the motor milestone of being able to walk independently (many within a normal timeframe) have maintained that ability from the first occurrence until the last visit.

The impact of early and sustained SPINRAZA treatment on these infants and their families is remarkable. Ive had the privilege to watch them grow into active young children, many of whom have experienced progress in motor function consistent with children their age who do not have SMA, said Kathryn Swoboda, M.D., the Katherine B. Sims, M.D., Endowed Chair in Neurogenetics and Director of the Neurogenetics Program, Massachusetts General Hospital. The new results from NURTURE continue to bolster the substantial benefit of both prompt diagnosis and early and longer-term treatment with SPINRAZA.

NURTURE is an ongoing, Phase 2, open-label study of 25 pre-symptomatic patients with the genetic diagnosis of SMA (most likely to develop SMA Type 1 or 2) who received their first dose of SPINRAZA before 6 weeks old. The study has been extended by an additional three years, enabling Biogen to evaluate the longer-term efficacy and safety of SPINRAZA through 8 years of age and further understand the impact of early treatment. More information on the NURTURE study (NCT02386553) is available

Additional results from the updated interim analysis as of February 2020 show:

About SPINRAZA (nusinersen)2-4 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.

About BiogenAt Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the worlds first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics and is focused on advancing research programs in multiple sclerosis and neuroimmunology, Alzheimers disease and dementia, neuromuscular disorders, movement disorders, ophthalmology, immunology, neurocognitive disorders, acute neurology and pain.

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SOURCE: Biogen

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New Results From Landmark NURTURE Study Show That Pre-Symptomatic SMA Patients Treated With SPINRAZA (nusinersen) Continue to Demonstrate Sustained...

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