Ionis Pharmaceuticals’ (IONS) nusinersen is a drug designed for the treatment of patients with spinal muscular atrophy (or SMA). SMA is a type of severe motor-neuron disease. The company has entered into a collaborative agreement with Biogen Idec (BIIB) for the development and commercialization of nusinersen.
Ionis has classified nusinersen as a drug for severe and rare therapeutic areas. It’s under Phase III clinical studies for the treatment of infants and children with spinal muscular atrophy.
What is spinal muscular atrophy?
Spinal muscular atrophy is a severe motor-neuron disease that occurs from the deletion or mutation of a gene that produces protein for normal cellular function. SMA is a leading genetic cause of infant mortality.
Other details about nusinersen
Below are a few details about the drug nusinersen:
- Nusinersen has been granted Orphan Drug Designation by the FDA (U.S. Food and Drug Administration) and the European Medicines Agency for the treatment of patients with SMA.
- Ionis reported an update on the ongoing Phase II clinical study for nusinersen in January 2016. It reported a continued increase in median event-free survival and muscle function scores.
- Presently, the company is evaluating nusinersen with Biogen Idec in a Phase III study called ENDEAR.
- ENDEAR is a double-blind, randomized, sham-procedure controlled 13-month study to evaluate the efficacy and safety of a 12mg (milligram) dose of nusinersen. The sample size is about 110 infants. The company expects to report data from this study in the first half of 2017.
To avoid risk, you can consider investing in ETFs such as the iShares Nasdaq Biotech ETF (IBB), which holds ~1.0% of its total assets in Ionis. You may also consider the Vanguard Russell 2000 Growth ETF (VTWG), which holds ~0.8% of its total assets in Ionis. IBB also holds 7.0% of its total assets in Regeneron Pharmaceuticals (REGN), 7.7% in Celgene (CELG), and 3.6% in Vertex Pharmaceuticals (VRTX).
In the next part, we’ll see what Ionis Pharmaceuticals is doing with IONIS-TTR.