It is developing new drugs based on RNA research (anti-sense technology) – and the platform it has built can be applied to treat many diseases and illnesses for which there is no cure.
But not all trials will lead to successful outcome – the stock tanked in May after one such trial with partner GSK for the treatment of Thrombocytopenia did not continue to phase 3 – the stock dropped to $21.
2 months on, and the company announced that in partnership with Biogen, they will try to commercialise Nusinersen, a drug which can be used to treat Spinal Muscular Atrophy – a horrible disease affecting over 100,000 children.
Great thing about IONIS, is that they are not tied to one drug – but a technology platform built around RNA – and this is the first drug to be potentially commercialised using this platform – if the technology is successful, it could lead to other breakthroughs.
The FDA will fast track this drug as there is no other cure.
For BIOTECHS – keep positions manageable, take profits on strength to lower cost base and weather the bad news, keep a core position and hope for the big breakthroughs – not just for the p/l, but to cure diseases which cannot be cured today.
2CentView has held a core position in IONIS pharm (ex ISIS pharm) for over 2 years and plans to keep the position.