Today, a brief rundown of news from Merck & Co. and Cabaletta Bio, as well as updates from Intellia Therapeutics, Vertex Pharmaceuticals and Novartis that you may have missed.
An experimental Merck & Co. drug aimed at the cancer immunotherapy target TIGIT has failed another clinical trial, the company said Thursday. Independent trial monitors advised Merck to stop a Phase 3 study testing a co-formulation of the medicine, vibostolimab, and its drug Keytruda in advanced small-cell lung cancer because treatment was unlikely to extend survival compared to an immunotherapy and chemotherapy regimen and was associated with higher rates of side effects, Merck said. The vibostolimab-Keytruda combination has already fallen short in studies for lung and skin cancer. — Ben Fidler
Shares of cell therapy developer Cabaletta Bio fell 34% Thursday after the company disclosed a clinical trial participant with lupus who received its autoimmune disease cell therapy experienced a life-threatening neurological side effect. Cabaletta said the adverse event, a known cell therapy complication called ICANS, resolved quickly following treatment with an antiseizure medication. A committee of investigators has recommended the study continue at the current dose level. Nonetheless, the incident will “fuel the commercial debate on the feasibility” of broadly using cell therapies on autoimmune conditions, wrote Stifel analyst Benjamin Burnett. — Ben Fidler
A gene editing therapy from Intellia Therapeutics met the primary and secondary goals of a Phase 2 trial testing it in people with hereditary angioedema, the company said Thursday, without disclosing specific data. According to Intellia, both tested doses of its medicine, dubbed NTLA-2002, led to “deep reductions” in the swelling attacks that characterize HAE. The data build on prior results from Phase 1 testing, and led Intellia to choose a 50 milligram dose for a Phase 3 study it plans to start later this year. The company will disclose the full Phase 2 findings in the fourth quarter. — Ned Pagliarulo
Vertex Pharmaceuticals and the National Health Service in England have reached an agreement on reimbursement for the company’s CRISPR therapy Casgevy in people with transfusion-dependent beta thalassemia. Vertex did not disclose the agreed-upon price, but noted the funding for patient access comes from NHS England’s Innovative Medicines Fund. Discussions on reimbursement of Casgevy for sickle cell disease are ongoing, the company said. Casgevy has a $2.2 million list price in the U.S. — Ned Pagliarulo
The Food and Drug Administration has granted accelerated approval to Novartis’ drug Fabhalta to treat people with primary IgA nephropathy, a rare disease in which the body’s immune system attacks the kidneys. It’s the second U.S. approval for Fabhalta, which secured an initial clearance in paroxysmal nocturnal hemoglobinuria late last year. The OK in IgAN was based on data showing that treatment lowered urine protein levels, a surrogate marker of organ benefit. Novartis is now evaluating whether that finding translates to a slower decline in kidney function. — Ned Pagliarulo
