Intellia is set to report Phase 3 trial results for a potential treatment for hereditary angioedema in the first half of 2026, amidst a backdrop of significant clinical trial activity in rare diseases. Despite recent challenges, including a study participant's death and program suspensions, the biotech sector is regaining momentum with key readouts expected across various conditions.
After a lengthy downturn, the biotech industry finally gathered momentum in 2025. Key readouts in obesity, infectious disease and many rare conditions could help it continue. But, while Intellia’s clinical progress has at times displayed CRISPR’s massive therapeutic potential, the company has burned through more than $2 billion since inception and twice laid off staff amid shifting research plans. Its share price sits near record lows, too, following the death of a study participant and suspension of one of its top programs. Intellia still has a shot at success, though. In the first half of 2026, it’s expecting results from a Phase 3 trial testing a possible treatment for a rare condition called hereditary angioedema. Clinical trial results have fueled many of these outcomes, spurring interest from acquirers or investors. This year, other important readouts lay ahead in obesity, infectious diseases, rare disorders and more. Miller, of Evercore, projects sales would top out at $500 million annually in peak years, given the way gene therapy launches have gone so far. — Ben Fidler ... Vertex Pharmaceuticals is best known for a cystic fibrosis drug business that now regularly brings in more than $10 billion each year. But pressure to recreate that success elsewhere is intensifying, as a pain medicine it recently brought to market may not be the blockbuster it once hoped. One drug Vertex is developing for a rare kidney disease could be part of the solution. The second is in people with obesity and diabetes, while the third is in those with obesity and established cardiovascular disease, as defined by a previous heart attack or stroke. Combined, the trials have enrolled more than 5,000 people. Success in these studies would keep Lilly ahead of a raft of emerging competitors — among them chief rival Novo Nordisk, which is currently trying to win approval of a dual-acting therapy.
Original title: “10 clinical trials to watch in the first half of 2026 | BioPharma Dive”