Baijian SPINRAZA improves exercise capacity in infants and adolescents with spinal muscular atrophy

Baijian SPINRAZA improves exercise capacity in infants and adolescents with spinal muscular atrophy

March 13, 2018 Source: Sina Pharmaceutical

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On March 12, Biogen announced the interim data of the clinical phase 2 study NURTURE, a one-arm study of the open label for SPINRAZA® (nusinersen) for the treatment of spinal muscular atrophy (SMA) in children before symptom onset The effectiveness and safety of the assessment were evaluated. The interim analysis entitled "Nusinersen for the treatment of infants in pre-symptomatic SMA conditions: mid-term efficacy and safety outcomes of clinical phase 2 NURTURE studies" showed that all babies were alive, no patients required tracheotomy or permanent Sexual ventilation. As of July 5, 2017, the patient's motor function and athletic milestones have improved compared to the natural history of the disease. The study, along with the effectiveness of SPINRAZA in adolescents and young adults, will be presented at the Muscular Dystrophy Association Clinical Conference, March 11-14, 2018, in Arlington, Virginia.

Dr. Alfred Sandrock, Senior Vice President and Chief Medical Officer of Baijian, said: "These results enhance the effectiveness of SPINRAZA as the first and only drug approved for the treatment of SMA, once again proving that it can be given to individuals who use SMA (including babies, Benefits for adolescents and young adults. SPINRAZA is supported by the largest SMA clinical development project to date, and Baijian looks forward to working with healthcare providers and healthcare providers to provide significant improvements in patient movement for SMA teens and young adult patients. The ability of SPIRAZA."

In the NURTURE study, SPINRAZA was administered to infants (n=25) of six weeks or younger, with these children in the pre-symptomatic phase, genetically diagnosed as SMA, and 2 to 3 copies of the SMN2 gene (n=15 Two copies; n=10 for three copies). During this interim analysis, the children underwent follow-up for up to 25.6 months, far exceeding the typical time that most type 1 SMA infants require permanent ventilation or death.

Dr. Darryl C. De Vivo, chief researcher at Columbia University Medical Center, said: "The findings of the NURTURE trial provide evidence of the continued benefits of SPINRAZA. The hereditary SMA infants treated in the NURTURE study (clinically may develop type 1 or Type 2), as long as there is enough observation time, they can sit independently, and most people have the ability to walk."

Participants in the NURTURE trial also achieved an average CHOP INTEND score of 58.4 with a maximum score of 64. The CHOP INTEND indicator was used to measure the general motor function of children with SMA. Many children can continue to improve and maintain these scores beyond a point in time at which point untreated patients with type 1 SMA experience a significant decline. Overall, studies have shown that SPINRAZA is well tolerated and no new safety concerns have been identified.

In addition, the published results will include the stabilization or improvement of SPINRAZA's condition in adolescents and young adults with type 2 or 3 SMA. In this case analysis, participants (n=5) were between the ages of 14 and 15 at the start of the CS2 study, and the age of patients in the expanded study CS12 was 17-19 years. One participant was type 2 and 4 was type 3. During the 2.5 years of observation, all participants received multiple doses of SPINRAZA. Results included: improvement of the Hammersmith Motor Function Rating Scale (HFMSE); stabilization of the upper limb module (ULM); improvement of the 6-minute walk test (6MWT); and assessment of neuromuscular disease caregiver experience (ACEND) scores.

Dr. John Day, Stanford University Medical Center, said: "This case analysis shows the efficacy of SPINRAZA in adolescents and young adults with type 2 or 3 SMA. Study participants in two years of treatment, even after treatment, motor function and quality of life There is a steady or slight improvement, unlike adolescents and young people with SMA who are not treated, their motor function declines, especially walking distance and lower limb activity, as well as health-related quality of life. Falling." (Sina Pharmaceutical Compilation / David)

Article Reference Source: New Data Presented at MDA Clinical Conference Show Benefit in Motor Function for Infants, Teens and Young Adults Treated With SPINRAZA® (nusinersen)

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