A new corporate presentation from one of the world's leading gene therapy companies has once again highlighted the commercial challenge confronting companies with the new technologies.
US company bluebird bio is currently in the process of planning the launch of gene therapy, ZYNTEGLO, for transfusion-dependent β-thalassemia.
It also recently announced positive results from a trial of its investigational Lenti-D gene therapy in patients with cerebral adrenoleukodystrophy.
ZYNTEGLO has been approved in Europe and the company has also disclosed launch plans for the US. It has not yet announced any plans to launch its therapies in Australia and the ZYNTEGLO launch in Europe has been delayed until 2020 due to issues with manufacturing.
bluebird bio has a range of other products in development and has a self-described vision of being recognised as 'the gene therapy' company by 2022.
In its most recent corporate presentation, the company has disclosed a US$1.5 billion cash position, which it says provides a 'runway' into 2022, based on half-year losses of around US$300 million.
The company's cash position has concerned some investors with its share price almost halving in the past six months. It still has a current market capitalisation of almost US$5 billion.
However, with a product approval and healthy pipeline, bluebird bio is considered amongst the most likely of the small to mid-size gene therapy companies to achieve successful commercialisation.
Previous products commercialised by companies focused exclusively on gene therapy, such as uniQure's GLYBERA (alipogene tiparvovec), have struggled. GLYBERA was ultimately taken off the market due to high costs and payer reluctance to agree to its high price.
GLYBERA (alipogene tiparvovec), which treated an ultra-rare genetic fat disorder called lipoprotein lipase deficiency, was priced at around US$1 million.
bluebird bio is pricing ZYNTEGLO at around US$1.8 million - to be paid for in instalments over five years, with no cost to the health system afterwards, if successful.
Larger biopharmaceutical companies, such as Novartis and Roche, are also working to commercialise gene therapies but have the advantage of 'scale underpinned by large, diverse and commercially successful product portfolios.
Australia is likely to benefit from the global scale of companies like Novartis and Roche when it comes to accessing their gene therapies. However, it be might be towards to end of the country launch queue for small to mid-size companies like bluebird bio.