A groundbreaking gene therapy trial for cystic fibrosis (CF) was announced inUKrecently. This serves as yet another strong indicator that pharmaceutical and biotech companies are increasingly moving into the market with treatments that target the underlying cause of the disease, rather than just the symptoms.
This announcement was made by the UK Cystic Fibrosis Gene Therapy Consortium, a nonprofit research group that is coordinating the trial. It is the largest trial of its kind to date, enrolling 130 adults and children with CF. The trial will be funded by a £3.1 million ($4.9 million) grant from the National Institute for Health Research (NIHR) and the Medical Research Council (MRC). Patients in the trial will receive either a dose of the inhaled gene therapy or a placebo once monthly during the course of a year. The active treatment will be administered by inhaling molecules of DNA into the cells of the lining of the lungs, in order to replace the defective gene that causes CF. The goal of the study is to determine whether this therapy can improve symptoms in patients with CF.
The Consortium, which has been working for more than a decade on its gene therapy, expects results of the trial to be released in the spring of 2014. The MRC also plans to fund another study of an advanced version of the therapy, which uses a modified virus as a vehicle to deliver the replacement gene into the lungs. This, in turn, could lead to an improved delivery mechanism in the future.
CF is an inherited chronic disease that affects approximately 30,000 children and adults in theUnited States, and about 70,000 individuals worldwide. It is caused by a defective gene that results in the buildup of abnormally thick and sticky mucus in the lungs, digestive system, and other organs. This leads to life-threatening lung infections and serious digestion problems. Back in the 1950s, few children with CF even lived long enough to begin school. Since then, advances in treatment have both extended the lifespan and improved the quality of life of people with CF, who can now expect to live well into their thirties, forties, and beyond.
Conventional products used to treat CF include inhaled medications that break up mucus (mucolytics), antibiotics to fight infections, and saline solutions that make it easier to cough up mucus. These products, however, provide only symptomatic relief. This has created a large unmet need for disease-modifying therapies to address the underlying genetic cause of the disease. Vertex Pharmaceuticals of Cambridge, MA was the first company to introduce a gene therapy for CF, with the launch of Kalydeco (ivacaftor) in January 2012. The downside, however, is that Kalydeco – which has an annual price tag of $294,000 – can only be used by 4% of CF patients in theUS, who have a specific genetic defect known as a G551D mutation. Vertex is continuing to work on developing other targeted gene therapies, and Ataluren (PTC124) is another gene therapy currently undergoing clinical investigation with a launch expectedin 2014.
Due to the significant unmet clinical needs associated with conventional symptomatic treatments, there is a huge market potential for genetic therapies for CF. According to GlobalData, the size of the global CF market was $1.1 billion in 2010. We believe that this market will reach $2.5 billion by 2018, at a combined annual growth rate (CAGR) of 10.7%. Although the global therapeutics market is currently dominated by Novartis and Roche, GlobalData believes that companies such as Vertex, PTC Therapeutics, and Cystic Fibrosis Foundation Therapeutics (CFFT) with advanced therapeutic products in their pipeline will attract the majority of investors’ attention in the near future.
These novel therapies will undoubtedly spur increased competition in a market that has until recently been considered to be weak.