Clinical trials paused due to heart patient’s hospitalization
Intellia Therapeutics, a Massachusetts-based genome editing company, has paused two clinical trials after a patient was hospitalized due to a significant liver injury.
The MAGNITUDE and MAGNITUDE-2 clinical trials are focused on tracking the safety and efficacy of nex-z, an investigational therapy, in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN), respectively. When a patient participating in the MAGNITUDE trial was hospitalized due to grade 4 liver transaminases and elevated levels of bilirubin, the decision was made to temporarily put both trials on hold.
“In line with our commitment to patient safety, we have taken immediate action to temporarily pause enrollment in MAGNITUDE and MAGNITUDE-2 as we investigate this recent event,” John Leonard, MD, Intellia Therapeutics president and CEO, said in a statement. “As we focus on ensuring the health of this patient, we also are engaging with regulatory authorities and other stakeholders globally to develop a strategy to resume enrollment as soon as appropriate.”
Before this incident, the MAGNITUDE trial had enrolled more than 650 ATTR-CM patients. MAGNITUDE-2, meanwhile, had enrolled nearly 50 patients with ATTR-PN. In total, approximately 450 patients have been dosed with nex-z.
As one might expect, Intellia Therapeutics saw its stock price drop after the company shared this update. Crispr Therapeutics, a separate biotech company also focused on gene-editing therapies, also saw its stock price take a hit in the aftermath of this announcement.
Multiple ATTR-CM treatment options already available
Pfizer’s tafamidis (Vyndaqel, Vyndamax) was the only FDA-approved treatment available for ATTR-CM for years before BridgeBio’s acoramidis (Attruby) gained approval in November 2024. Alnylam Pharmaceuticals then gained FDA approval for vutrisiran (Amvuttra) back in March, bringing the total number of available therapies to three.
There are concerns, however, that the costs of these treatments could limit access for many patients.
Michael has more than 19 years of experience as a professional writer and editor. He has written at length about cardiology, radiology, artificial intelligence and other key healthcare topics.