Intellia begins advanced CRISPR therapy trial for rare swelling disease

Intellia Therapeutics has announced the start of a Phase 3 clinical trial aimed at testing its CRISPR gene editing drug for treating hereditary angioedema (HAE), a rare disorder characterized by recurrent swelling attacks in various parts of the body. This trial, named HAELO, will include 60 adult participants suffering from HAE, a condition that affects approximately 1 in 50,000 people. Existing treatments for HAE involve various on-demand and preventive medications.

The drug under investigation, NTLA-2002, utilizes CRISPR technology to deactivate a gene responsible for producing a protein involved in blood clotting and the manifestation of HAE. This in vivo treatment is intended to be a one-time remedy for the disorder, effectively altering the genetic code inside the patient's body.

Intellia is positioning itself to enter the competitive arena alongside Pfizer, BridgeBio Pharma, and Alnylam Pharmaceuticals, all of which are developing treatments for transthyretin amyloidosis with cardiomyopathy, another rare disease. Nearly a year ago, Intellia initiated a Phase 3 trial for NTLA-2001, marking the first late-stage trial of an in vivo CRISPR medicine. This trial, in collaboration with Regeneron Pharmaceuticals, is currently being conducted at nearly three dozen sites worldwide.

Encouraged by favorable Phase 2 results, Intellia is now advancing NTLA-2002 into late-stage testing. The initial trials demonstrated that the drug significantly reduced the frequency of HAE attacks and decreased levels of the kallikrein protein it targets.

The HAELO trial will randomize participants in a 2-to-1 ratio to receive either NTLA-2002 or a placebo. The primary measure of success will be the change in HAE attack frequency from week 5 to week 28 of the study. After this period, participants who initially received the placebo will have the option to switch to NTLA-2002 in a crossover phase.

John Leonard, CEO of Intellia, emphasized the urgency in advancing NTLA-2002 to meet the needs of those suffering from HAE. He expressed confidence that the treatment would bring substantial benefits to patients, healthcare providers, and insurers.

Despite these advancements, Intellia has faced financial challenges, with its shares declining by over 40% this year. This drop is attributed to increased investor scrutiny of genetic medicine developers and broader market difficulties affecting the biotechnology sector. Currently, Intellia's market valuation stands at approximately $1.8 billion.