Ahmad Masri, MD, MS: Establishing Proof-of-Concept of Gene Editing in ATTR-CM

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Masri previewed data to be presented at the AHA 2024 Scientific Sessions and shared his excitement for the ongoing phase 3 trial of NTLA-2001.

Intellia Therapeutics hopes to develop NTLA-2001, or as per its new name, nexiguran ziclumeran (nex-z), a gene editing therapy, as a potential disease-modifying therapy for transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM).

Data from the phase /21 trial of nex-z are set to be presented at the American Heart Associations 2024 Scientific Sessions Meeting, to be held in Chicago, Illinois, on November 16-19.¹

“The majority of the patients, if not all of them, as far as we've seen from the data, had TTR suppression or knockdown or silencing by more than 90% and that's with a single administration, regardless of the dose that was chosen. Overall, from the data presented. it seems to [be] a safe strategy,” Ahmad Masri, MD, MS, director of the Hypertrophic Cardiomyopathy Center at Oregon Health and Science University, and investigator on the nex-z trials, told HCPLive.

In addition to the phase 1/2 trial, Intellia also recently announced that the first patient with ATTR-CM was dosed in the phase 3 MAGNITUDE study of nex-z.2

“We are very excited about the potential findings from the [phase 3] trial, and not just the fact that the drug works. I think [that's]pretty obvious, at least from the phase one trial that [was] a proof of principle. Now comes the phase3, which is looking at heart outcomes and what happens to these patients as we go through this process,” Masri said.