Europe has launched the most clinical trials for cystic fibrosis (CF) treatments, while North America leads in those for acute myeloid leukemia (AML), according to a pair of new global trial landscape reports announced April 10 by contract research organization Novotech. Global venture funding for R&D on treatments for both diseases topped $1 billion each over the past five years.
CF is caused by an inherited mutation in the gene CFTR that damages mucus-producing cells in the lungs, pancreas and other organs. Though there is currently no cure, the past four decades have seen significant progress on treatments. Before the 1980s, half of CF patients survived into their 20s, the Novotech report noted. By 2025, 75% of patients with CF are expected to live to adulthood.
Drugmakers since 2018 have launched 450 clinical trials for CF drugs worldwide, with 37% in Europe and 35% in North America, the report said. The field includes Vertex Pharmaceuticals, the creator of a powerful but pricey drug trio called Trikafta that in 2019 became the first FDA-approved triple combination therapy for the most common genetic mutation in CF. The company has several investigational CF treatments currently in late-stage clinical trials on top of four therapies already on the market.
Worldwide venture capital funding for CF research totaled just over $1 billion between 2019 and 2023, the report said, with the U.S. leading at $919 million followed by the Netherlands, Israel and Italy. ReCode Therapeutics, Adaptive Phage Therapeutics, Metagenomi and Carmine collected the most VC dollars. ReCode, Metagenomi and Carmine are all working on genetic medicines, while Adaptive Phage (recently acquired by BiomX) is studying whether viruses called bacteriophages can clear dangerous bacteria from the lungs of those with CF.
Another Novotech report highlights the landscape for AML, a type of blood cancer that begins in the blood stem cells in the bone marrow. It’s the second most prevalent blood cancer in adults and children but has the lowest survival rate. Fewer than half of patients live beyond five years of diagnosis.
Pharma companies have attempted to improve the odds by launching more than 1,000 trials for AML treatments globally since 2019, according to Novotech. North American trials account for about 40% of those trials, with another third in the Asia-Pacific region and 22% in Europe. Venture funding for AML R&D from U.S. firms totaled about $1.8 billion between 2019 and 2023, with another $1.2 billion in China over the same period. The top-funded companies for AML drugs within the same time span were Opna Bio, Shorla Oncology and OncoVerity.
Novotech runs clinical trials for clients in the APAC region, the U.S. and Europe. The CRO is based in Australia and has more than 3,000 staff worldwide, according to the company.