A potentially game-changing technology

Spinal Muscular Atrophy (SMA) is an inherited, genetic disease with no approved treatments until recently. The disease is caused by a mutation in a gene that encodes for an important protein involved in the survival of motor neurons, which control muscle function. When this protein is deficient, muscles do not form properly, and/or die prematurely. This disease affects infants, children, and even, to a lesser extent, adults. Many of these babies die before their first birthday, and many of the children cannot even sit without help.

Ionis Pharmaceuticals utilized its antisense oligonucleotide technology platform to develop Spinraza (nusinersin), a drug that creates a functional version of the deficient protein, thereby promoting survival of the neurons and muscles that would otherwise die in these patients. Spinraza is the first and only approved treatment for SMA in the US. In Europe, it is still under review. This technology can be applied to numerous genetic diseases. Furthermore, Ionis has optimized this technology and can now create drugs that can be dosed much less frequently, and at much lower concentrations, thus improving efficacy while reducing side effects. This is where the true value in Ionis lies, in our opinion. 

Spinraza is the definition of true innovation, which will offer hope to thousands of infants and children who previously had no treatment options. Not only is the drug safe, its efficacy has produced results that have never be seen before in these babies and children. While the drug will be priced at a premium, it is not a “me-too” drug, offering only incremental benefits. Spinraza will change a disease, will change lives, and offer a new treatment option where no other drugs were able to perform. The drug is priced at US$750,000 for the first year of treatment, which amounts to five or six doses. Spinraza costs US$375,000 for each additional year after that. After the approval by the FDA, multiple insurers in the US announced they would restrict coverage for Spinraza to the most severe form of SMA. However, we see value in the platform, and believe Spinraza is only the beginning for a potentially game-changing technology. With top-class partnerships (Bayer and Novartis), and a seemingly robust pipeline of therapies set up in a fairly short space of time, Ionis could finally help antisense technology live up to its long-held promise.

Dallas Webb, Investment Manager, BB Biotech AG