Phase 3 MAGNITUDE Trial Initiates with First Patient Dosed in NTLA-2001 Study for ATTR Cardiomyopathy

Intellia Therapeutics, a pioneering company in the gene editing field, has reached a significant milestone with the commencement of the Phase 3 MAGNITUDE trial for their investigational treatment, NTLA-2001. This CRISPR-based therapy is intended to provide a one-time, in vivo treatment to combat transthyretin (ATTR) amyloidosis by deactivating the TTR gene, thus preventing the synthesis of the TTR protein, which is linked to the disease's progression.
The MAGNITUDE trial, a global, pivotal study, aims to assess the efficacy and safety of NTLA-2001 in approximately 765 patients diagnosed with ATTR amyloidosis with cardiomyopathy. The trial is structured as a randomized, double-blind, placebo-controlled study, with a primary focus on cardiovascular-related mortality and events as the main endpoints. Participants will be administered either a single 55 mg infusion of NTLA-2001 or a placebo, in a 2:1 ratio.
John Leonard, M.D., Intellia's President and CEO, expressed optimism about the potential of NTLA-2001 as a transformative treatment for ATTR amyloidosis. He highlighted the positive interim Phase 1 results, which demonstrated significant and lasting reductions in TTR protein levels following a single dose of the therapy. Dr. Julian Gillmore, a leading expert in ATTR amyloidosis and the national coordinating investigator for the Phase 3 study, echoed this enthusiasm, noting the high expectations within the patient community for a one-time treatment option.
ATTR amyloidosis is a severe, progressive disease without a cure, characterized by the misfolding and accumulation of TTR protein in various tissues, leading to heart, nerve, and digestive system complications. The disease can be hereditary or occur in individuals without genetic mutations, affecting an estimated 50,000 to 500,000 people worldwide. Current treatments are limited to slowing the protein's accumulation.
Intellia Therapeutics, listed on NASDAQ, is at the forefront of gene editing innovation, utilizing CRISPR technology to edit disease-causing genes within the body and to engineer human cells for the treatment of cancer and autoimmune diseases. The company's development of NTLA-2001 represents a significant step towards a novel class of medicine, with the potential to dramatically improve the standard of care for ATTR amyloidosis patients.