- The Cystic Fibrosis Foundation is committing $500 million to advance drugs that would provide some form of treatment, or potentially a cure, to all patients with the genetic disease.
- Approved drugs from Vertex, a Boston biotech, can treat around 90% of cystic fibrosis patients. The remaining 10% have what are called "nonsense mutations," which cause transcription to stop prematurely and can lead to nonfunctional DNA and proteins. The foundation notes how these mutations have been "notoriously challenging targets for drug discovery and development," both in cystic fibrosis and other diseases.
- Especially of interest to the foundation are drugs that either fix the underlying genetic mutations of cystic fibrosis, repair the broken protein that results from those mutations, or make the protein in cases where there aren't functional copies. Collaborators, including pharmaceutical companies, can submit proposals to work with the foundation and its funding, which will be doled out through 2025.
Earlier this month, the Food and Drug Administration approved Vertex's fourth cystic fibrosis therapy. Branded as Trikafta, the three-drug regimen is expected to raise the percentage of the cystic fibrosis population eligible for a Vertex product from around 50% to 90%.
Such broad coverage would be a revolutionary development, particularly for a disease where the average life expectancy remains under 40 years. Vertex and its longtime partner, the Cystic Fibrosis Foundation, now have their sights set on the roughly 10% of patients who don't yet have an applicable drug. The biotech has been investigating gene therapy as one potential solution.
The foundation's research and support were integral to the development of Trikafta as well as Vertex's earlier offerings: Kalydeco, Orkambi and Symdeko. In 2014, the foundation's nonprofit affiliate sold its royalty rights to the biotech's drugs for $3.3 billion. According to its most recent quarterly filing, an amended agreement between the parties inked in 2016 gave Vertex $75 million upfront and provides $6 million in annual development funding.
The foundation says that, in 2018, it awarded $141 million to academic and industry groups for laboratory research, preclinical drug discovery, and clinical and real-world research. For this year, it expects to spend $220 million on medical awards.