Bluebird bio (BLUE) is a company whose main focus is on gene-therapy against severe genetic diseases and cancers. Their recent potential blockbuster therapy is called Zynteglo and is used against beta-thalassemia. Beta-thalassemia is a blood disorder that reduces the production of beta-globin, a component of haemoglobin, a protein necessary for the transport of oxygen to cells throughout the body. This lower production results in a shortage of oxygen in cells causing fatigue, dizziness, low concentration, an irregular and/or rapid heartbeat and many other severe symptoms. Zynteglo is used in patients who do not completely lack beta-globin and who are eligible for stem cell transplantation but do not have a matching related donor. Zynteglo contains as its active substance stem cells taken from the patients that have been genetically modified to contain a working gene for beta-globin.
Zynteglo is the first ever approved product in this category, surprisingly it was first approved by The European Medicine Agency. FDA-approval will likely happen in the near future, bluebird filed their application at the end of last year. The costs of Zynteglo will be 1.8 million dollars with the option to spread the costs over five years. The first patients to use Zynteglo will only have to pay if the therapy actual works. The present management of the sickness includes regular blood transfusions every two to four weeks and daily iron chelation therapy, this all can be avoided using Zynteglo. The major part of the patients using Zynteglo in the Phase-III trial have been transfusion-free for up to 56 months.
The biggest challenge for bluebird bio is to justify their high price-tag. Because of the complexity of the treatment only a small number of patients have been examined. With Phase-III results looking better than the Phase-I/II results this is nevertheless looking promising. However the long term durability of the treatment is uncertain. The treatment could potentially last a lifetime although bluebird bio is claiming 22 years of quality-adjusted lifestyle years for the most successful patients.
Currently bluebird bio is doing Phase I and II studies to the effect of LentiGlobin in the treatment of sickle-cell disease. Normally red blood cells are flexible and round allowing them to move easily through blood vessels. With sickle cell disease, those red blood cells are instead sticky and rigid which results in various intense symptoms. The blockage of blood vessels can cause strokes and infections. Moreover repeated blockages can lead to damage of organ systems and are the primary cause of a shortened lifespan in patients with sickle cell disease. Sickle cell disease is an inherited disease caused by a defect, a single mutation, in the beta globin gene that helps make adult hemoglobin. Bluebird bio is doing a study to determine if stem cells taken from the patients that have been genetically modified to contain a working gene for beta-globin works for these patients too. This could potentially be a big blockbuster too. The main problem here is again the probable high price-tag of this again relatively rare disease.
Overall gene-therapy is a hot topic in the biotech industry and we must keep our eyes on the developments. It will be interesting too see if bluebird manages to attract patients who are willing and able to afford this therapy. With its 1.8 million dollar price-tag it’s the second most expensive therapy in the world. If results end up being positive, bluebird bio (BLUE) stock could rise sky high and potentially be taken over by one of the big pharma companies.
Disclaimer: The writer of this article owns bluebird bio (BLUE) stock, this article shouldn’t be interpreted as stock investment advice or anything like that.