Charles Moore,  —

Ionis Pharmaceuticals has received $40 million in milestone earnings from Biogen following Japanese regulatory approval of the price of  Spinraza, Biogen's treatment for spinal muscular atrophy. So far, Ionis has earned more than $435 million from Biogen in Spinraza-related regulatory approvals and sales royalties, it said in a press release. The market performance speaks to the overwhelming success that the drug is having in treating the SMA patient population. Spinraza has been approved by U.S, European, Canadian, Brazilian and now Japanese regulatory agencies. Ionis is now working to bring the treatment to other countries including Switzerland, Israel, South Korea and Australia, where it is now under review by regulatory agencies Biogen licensed global development, manufacturing and commercialization rights to Spinraza from Ionis in August 2016. In so doing, it assumed responsibility for all activities and costs associated with the drug. Ionis is eligible to receive tiered royalties on Spinraza sales up to a percentage in the mid-teens, in addition to up to $150 million in milestone payments based on regulatory approvals. Spinraza became the first approved SMA treatment in December 2016, when the U.S. Food and Drug Administration supported its use in children and adults. The FDA made that decision within three months of Biogen's regulatory filing. Biogen sponsors one of the largest Expanded Access Programs worldwide, offering Spinraza free of charge to patients requesting it in countries that have not yet approved its use. About 600 children with infantile-onset SMA in 24 countries began treatment under such programs, the release said.

The national nonprofit spinal muscular atrophy (SMA) support and advocacy organization Cure SMA has launched a grassroots campaign to have required newborn-screening for SMA in every state. The campaign was announced at the 2017 Annual SMA Conference held June 29-July 2 in Orlando, Florida. It was attended by…

Vivonoetics is partnering with Great Lakes NeuroTechnologies to provide advanced respiratory assessment to the Roche FIREFISH Phase 2 clinical trial in infants with type 1 spinal muscular atrophy type 1 (SMA-1). Vivonoetics is the developer of VivoSense data analysis and software and wearable monitoring devices that have supported research…

The EU Joint Programme —Neurodegenerative Disease Research (JPND) is calling for proposals for pathway analysis of multinational research projects, focusing on neurodegenerative diseases such as spinal muscular atrophy (SMA). According to a JPND news piece, neurodegenerative disorders such as Alzheimer’s disease and other dementias and Parkinson’s disease are…

Spotlight Innovation announced that it has acquired an exclusive, worldwide license to commercialize STL-182, an orally administered small molecule under investigation as a potential treatment for spinal muscular atrophy (SMA). SMA is an autosomal recessive disorder. Between 1 in 40 and 1 in 50 adults have only a single intact…

In a late-breaking session at the 2016 World Muscle Society Congress in Granada, Spain, Biogen and Ionis Pharmaceuticals presented new data from clinical trials for their co-developed drug candidate nusinersen for treating spinal muscular atrophy (SMA). Nusinersen, a potentially disease-modifying SMA therapy, is an antisense oligonucleotide (ASO) designed to…

International pharmaceutical company Roche, with its SMA drug development collaboration partners PTC Therapeutics and the SMA Foundation, said a clinical study called Moonfish (NCT02240355) that was investigating a compound and investigational medicine known as RG7800 for people with spinal muscular atrophy (SMA) was placed on clinical hold in April 2015 after…

Cure SMA-funded researcher Dr. Christian L. Lorson and his colleagues have published a paper in the journal Molecular Therapy describing a promising therapeutic prospect in a mouse model with severe spinal muscular atrophy (SMA). The article, “Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal…

August, proclaimed SMA Awareness Month, is when advocacy and research organizations like CureSMA and the SMA Foundation and supporters seek to raise public consciousness of the genetic disorder spinal muscular atrophy (SMA). While the SMA support community works year-round to raise awareness of and funds for…

The Spanish National Research Council (CSIC) recently unveiled a prototype of what it believes is the world’s first assistive exoskeleton designed to enhance mobility for children with the degenerative illness spinal muscular atrophy (SMA), a rare disease that affects 1 in 10,000 babies born in Spain. The 26-pound device, made primarily of…