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NTLA-2002 Reduces Hereditary Angioedema Attacks and Plasma Kallikrein

The first 3 patients dosed have been attack-free for up to 10.5 months.

Intellia Therapeutics’ CRISPR-based therapy NTLA-2002 has continued to show efficacy in hereditary angioedema (HAE) in updated data froma phase 1/2 study (NCT05120830) presented at the American College of Allergy, Asthma & Immunology (ACAAI) 2022 Annual Scientific Meeting, held November 10 – 14 in Louisville, Kentucky.

“We see early evidence that our one-time CRISPR-based investigational therapy may offer patients suffering from hereditary angioedema a functional cure for their disease,” John Leonard, MD, president and chief executive officer, Intellia Therapeutics, said in a statement. “Based on the extended data across multiple dose cohorts, we are strongly encouraged that all patients who received a single dose of NTLA-2002 subsequently became attack-free. In the patients with the longest follow-up to date, their attack-free interval has been maintained 5 to 10 months from their last attack. Importantly, the safety data from all 10 patients are highly encouraging, further supporting NTLA-2002’s potential to change the future HAE treatment paradigm.

The data presented, updated as of September 28, 2022, were from 10 participants with HAE from the dose-escalation phase 1 portion of the study. Participants received single doses of 25 mg (n = 3), 50 mg (n = 4), or 75 mg (n = 3) of NTLA-2002 via intravenous infusion. At latest follow-up, the 25 mg cohort had a mean 4% plasma kallikrein reduction as of week 32, the 50 mg cohort had a mean 81% plasma kallikrein reduction as of day 22, and the 75 mg cohort had a mean 92% plasma kallikrein reduction as of week 16.

READ MORE: FDA Recognizes CRISPR Therapy for Treatment of Hereditary Angioedema

Investigators also assessed HAE attack rates beginning at the end of the pre-specified 16-week primary observation period. The 25 mg cohort, which had a baseline attack rate of 1.1 to 7.2 attacks per month, has been attack free for 5.5 to 10.6 months. This cohort had a 91% mean HAE attack rate reduction from weeks 1 to 16 and an 89% reduction from weeks 5 to 16. The 75 mg cohort, which had a baseline attack rate of 4.0 to 5.9 attacks per month, has been attack-free for 2.3 to 4.2 months, and had a 78% mean HAE attack rate reduction from weeks 1 to 16 and an 89% reduction from weeks 5 to 16.

In terms of safety, NTLA-2002 was well-tolerated, with mostly mild adverse events (AEs). AEs were mostly grade 1 infusion-related reactions which resolved within 1 day. There were no dose-limiting toxicities, no serious AEs, or no clinically significant laboratory abnormalities observed.

“As the second clinical program from our in vivo pipeline to demonstrate deep and consistent protein reduction following a one-time administration, the latest interim data further reinforce the enormous potential of our modular CRISPR genome editing platform to treat a host of genetic diseases,” Leonard added.

Intellia plans to evaluate 2 additional doses of NTLA-2002 in the dose-expansion, phase 2, placebo-controlled portion of the study, which is expected to begin in the first half of 2023. The study will expand to more US and international sites.

REFERENCE
Intellia Therapeutics Presents New Interim Data from First-in-Human Study of NTLA-2002 for the Treatment of Hereditary Angioedema (HAE) at the American College of Allergy, Asthma & Immunology 2022 Annual Scientific Meeting/ November 12, 2022. https://www.einpresswire.com/article/600986799/intellia-therapeutics-presents-new-interim-data-from-first-in-human-study-of-ntla-2002-for-the-treatment-of-hereditary-angioedema-hae-at-the