By Christy Santhosh
March 2 (Reuters) – Intellia Therapeutics said on Monday the U.S. Food and Drug Administration has lifted the clinical hold on a late-stage trial of its gene therapy for a heart disease.
The gene-editing therapy, nexiguran ziclumeran (nex-z), is being tested separately for a heart condition and nerve damage caused by transthyretin amyloidosis, a rare and progressive disease resulting from the buildup of a faulty protein in organs.
Last year, the FDA paused Intellia’s two trials of the gene-editing treatment after a patient in the heart disease study was hospitalized with severe liver complications and later died.
Monday’s decision allows for resumption for the trial in the cardiomyopathy patients, sending shares of Intellia up 2% in morning trade. The company’s shares had fallen more than 40% after the trials were halted.
In January, the FDA removed its clinical hold on a trial for patients with nerve damage, or polyneuropathy.
Evercore analyst Jon Miller said Intellia came off the hold relatively quickly by historical standards, suggesting the overall clinical delay may be limited to roughly three to four months, and called it a “great result”.
The company said it has taken mitigation measures for both trials, such as enhanced liver test monitoring, short-term steroid use for early liver issues post-dosing and excluding patients with certain liver abnormalities.
For the heart disease trial, Intellia said it is adding new exclusion criteria for patients with recent cardiovascular instability or severely reduced ability to pump blood.
Current treatments for the heart-related form of the disease include Alnylam Pharmaceuticals’ injectable drug Amvuttra, Pfizer’s blockbuster Vyndaqel and BridgeBio Pharma’s Attruby.
(Reporting by Christy Santhosh in Bengaluru; Editing by Leroy Leo and Vijay Kishore)
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