Dear Members of the SMA Community,

As part of Biogen’s ongoing commitment to individuals living with, and caregivers affected by, spinal muscular atrophy (SMA), we are pleased to announce updates to our SPINRAZA® (nusinersen) clinical development program – an extensive clinical data set in the treatment of SMA to date that includes more than 300 patients, treated for up to 6 years.

SPINRAZA is approved to treat infants, children, and adults with SMA. As of September 2019, more than 9,300 patients have been treated with SPINRAZA in over 40 countries.[i]*

We are pleased to share the following details with the community in this statement:

  • The initiation of DEVOTE, a new global clinical trial to examine the safety and tolerability of SPINRAZA when administered at a higher dose.
  • New data from two open-label studies: the SHINE study (for individuals with SMA up to age 21 years) and the NURTURE study (for pre-symptomatic infants).

We are grateful for this opportunity to continue investing in research that may help improve the lives of those with SMA, their caregivers, and families.

New Study, DEVOTE, to Explore if Administering SPINRAZA at Higher Doses Can Further Improve Patient Outcomes[ii]

DEVOTE is a Phase 2/3 randomized, controlled dose-escalating study that will be conducted at 50 sites around the world with a projected enrollment of 126 individuals with infantile and later-onset SMA of all ages, including adults. The three-part trial will include:

  • Part A – An open-label safety evaluation.
  • Part B – A double-blind, active control, randomized treatment period comparing two loading doses of 50 milligrams (mg) 15 days apart, followed by a maintenance dose of 28 mg every four months with the current U.S. Food and Drug Administration-approved administration of SPINRAZA, which uses four loading doses with 12 mg maintenance doses every four months.
  • Part C – An open-label treatment period.

DEVOTE will assess a variety of measures for clinical efficacy based on which type of SMA the trial participants have, including motor developmental for infantile-onset SMA and motor function for later-onset SMA.

More information on the trial is available at clinicaltrials.gov/. For questions about SPINRAZA treatment or participation in ongoing clinical trials, patients or caregivers should contact their treating physician directly. This includes asking if they (or their loved one) would be eligible for enrollment in the DEVOTE trial.

Long-term Data from the SHINE Study Presented at European Paediatric Neurology Society (EPNS)

In addition, new data demonstrating the safety and efficacy of treatment with SPINRAZA in individuals with later-onset SMA were recently featured in a podium presentation at the 13th Congress of the EPNS in Athens (September 17-21).

An integrated analysis from SHINE (clinicaltrials.gov/), an open-label extension study for individuals with SMA who participated in prior SPINRAZA studies, found that children with later-onset SMA (Type 2 or Type 3) experienced improvements or stabilization in one or more measures of motor function for up to nearly 6 years, in contrast to the expected decline observed in natural history cohorts. Patients in the study were between 7 and 21 years old at the last study visit.

The SHINE study followed 24 individuals aged 2-15 years at treatment initiation (SMA Type 2; n=10 and SMA Type 3; n=14) who transitioned from the CS2/CS12 studies, which previously showed that individuals with later-onset SMA who were treated with SPINRAZA demonstrated improvements in motor function and disease stabilization over approximately 3 years, that were not observed in natural history cohorts.

No participants discontinued treatment due to adverse events, and side effects were consistent with those in the SPINRAZA Prescribing Information (see below for additional safety information). These data are important to our understanding of the impact of long-term SPINRAZA treatment in individuals with later-onset SMA.

NURTURE Study of Infants with SMA Treated Pre-Symptomatically with SPINRAZA Published in Journal of Neuromuscular Disorders[iii]

Finally, interim study results from NURTURE, an ongoing, Phase 2, open-label study of 25 infants with SMA (most likely to develop SMA Type 1 or Type 2) who initiated treatment with SPINRAZA prior to the onset of clinical symptoms attained impressive results in comparison to the natural history of the disease.

As of March 2019, all infants in the study were aged 25 months or older, past the age of symptom onset, and were alive, without the need for permanent ventilation. In comparison to the natural history of SMA, infants with Type 1 SMA would likely have passed away or required permanent ventilation, on average, by 13.5 months of age. Motor milestones achieved included 100 percent of participants sitting without support and 88 percent walking independently (evaluated as secondary endpoints).

NURTURE, the first study of infants with SMA treated pre-symptomatically, demonstrated the critical importance of newborn screening and early treatment of SMA.

Moving Forward Together

We are proud of the momentum in our clinical program and our progress in adding to the largest and longest clinical data set in the treatment of SMA to date. We continue to focus on supporting the SMA community through our robust ongoing clinical program, including studies to evaluate the safety and efficacy of a higher dose of SPINRAZA.

We look forward to continuing to share these ongoing updates with the SMA community.

Best regards,

The Biogen SMA Team

*Based on commercial patients, early access patients and clinical trial participants as of October, 2019.

INDICATION

SPINRAZA® (nusinersen) is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients. 

IMPORTANT SAFETY INFORMATION 

Increased risk of bleeding complications has been observed after administration of some similar medicines. Your healthcare provider should perform blood tests before you start treatment with SPINRAZA and before each dose to monitor for signs of these risks. Seek medical attention if unexpected bleeding occurs.

Increased risk of kidney damage, including potentially fatal acute inflammation of the kidney, has been observed after administration of similar medicines. Your healthcare provider should perform urine testing before you start treatment with SPINRAZA and before each dose to monitor for signs of this risk.

The most common possible side effects of SPINRAZA include lower and upper respiratory infections, constipation, headache, back pain, and post-lumbar puncture syndrome. These are not all of the possible side effects of SPINRAZA. Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Before taking SPINRAZA, tell your healthcare provider if you are pregnant or plan to become pregnant.

Please see full Prescribing Information.

This information is not intended to replace discussions with your healthcare provider.

References

  1. Data on file. Biogen, Cambridge, MA.
  2. Biogen. Biogen Advances Spinal Muscular Atrophy (SMA) Clinical Research with New Study Evaluating a Higher Dose of SPINRAZA® (nusinersen) and Additional Data in a Broad Range of Patients [Press release]. 18 Sep. 2019.
  3. Biogen. Landmark NURTURE Study of Infants with Spinal Muscular Atrophy (SMA) Treated Pre-Symptomatically with SPINRAZA® (nusinersen) Published in Neuromuscular Disorders [Press release]. 2 Oct. 2019.