13 March 2018 - - Cambridge, Massachusetts-based neurological and autoimmune disease therapies specialist Biogen (NASDAQ: BIIB) has released interim Phase 2 results from NURTURE, the ongoing open-label, single-arm study evaluating the efficacy and safety of Spinraza (nusinersen) among pre-symptomatic infants with spinal muscular atrophy (SMA), the company said.
In the NURTURE study, Spinraza was administered to infants six weeks old or younger, who were in the pre-symptomatic stage, genetically-diagnosed with SMA and had two or three copies of the SMN2 gene.
All infants treated with Spinraza were alive, did not require permanent ventilation, and showed improvement in motor function and motor milestone achievements compared to the disease's natural history.
This study, along with a case series demonstrating Spinraza's effectiveness among teens and young adults, is being presented at the 2018 Muscular Dystrophy Association Clinical Conference in Arlington, Virginia.
In the case series, participants were 14 to 15 years old at the start of Study CS2, and 17 to 19 years old at the time of their last visit in the extension Study CS12. One participant was Type 2 and four were Type 3, and all received multiple doses of Spinraza over 2.5 years of observation.
The results included improvement on the Hammersmith functional motor scale–expanded (HFMSE); stabilization on the upper limb module; improvement in the six-minute walk test; and stable or improved scores on the assessment of caregiver experience with neuromuscular disease (ACEND).
The most common adverse reactions reported for Spinraza were upper respiratory infection, lower respiratory infection, and constipation. Serious adverse reactions of atelectasis were more frequent in Spinraza-treated patients.
Spinraza, an antisense oligonucleotide licensed from Ionis Pharmaceuticals (NASDAQ: IONS), is designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q that leads to SMN protein deficiency. Spinraza alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein.
Biogen discovers, develops, and delivers therapies for people living with serious neurological and neurodegenerative diseases.
The company has a portfolio of medicines to treat multiple sclerosis and spinal muscular atrophy, and is at the forefront of neurology research for conditions including Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis (ALS). Biogen also manufactures and commercializes biosimilars of advanced biologics.