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

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.

REFERENCES

1. Intellia Therapeutics to Present New Clinical Data from the Phase 1 Study of nexiguranziclumeran (nex-z) for the Treatment of Transthyretin (ATTR) Amyloidosis at the 2024 AHA Scientific Sessions. News release. Intellia Therapeutics. October 1, 2024. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-present-new-clinical-data-phase-1-study

2. Intellia Therapeutics Announces First Patient Dosed in the Phase 3 MAGNITUDE Study of NTLA-2001 as a Single-Dose CRISPR-Based Treatment for Transthyretin Amyloidosis with Cardiomyopathy. News release. March 18, 2024. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-first-patient-dosed-phase-3