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Clinical Trial Efficacy
Comparison of attack reduction metrics in the Phase 3 study.
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Intellia Therapeutics says its Crispr-based treatment succeeds in ...
Intellia Therapeutics, building exterior and company sign, Cambridge, Massachusetts, USA.Spencer Grant | Universal Images Group | Getty ImagesIntellia Therapeutics said its Crispr-based treatment for a rare swelling condition met its goals in a late-stage trial, marking a milestone for the field of gene editing and putting the company on track to seek approval from the U.S. Food and Drug Administration. The company's treatment uses Nobel Prize-winning technology Crispr to edit DNA and turn off the gene that controls production of a peptide that's overactive in people with hereditary angioedema, causing them to experience potentially life-threatening swelling attacks. Intellia's treatment is administered once through an hourslong infusion, making the edits directly in the liver. Intellia said the one-time treatment reduced attacks by 87% compared with a placebo, meeting the study's main goal. Six months after treatment, 62% of patients were free from attacks and weren't using other therapies, Intellia said.The company described the safety and tolerability of the treatment as "favorable," reporting the most common side effects were infusion-related reactions, headaches and fatigue. Analysts were closely watching safety in the trial since a patient in a separate trial of a different treatment from Intellia died from liver toxicity. "When you think about where we started with Crispr, just 12 years ago with some of the fundamental insights, I think there was a lot of talk about what might be possible, and we've had reports along the way in terms of milestones, but this is the first Phase 3 data in any indication with in vivo Crispr where you're actually changing a gene that causes disease," said Intellia CEO John Leonard. The only FDA-approved Crispr-based medicine comes from Vertex Pharmaceuticals. Called Casgevy, the gene editing is done outside the body, or ex vivo. The process requires collecting a person's blood cells, making the edits outside the body, then reinfusing them back into a patient. Intellia's treatment, meanwhile, makes the edits inside the body, or in vivo. Intellia said it has started a rolling application with the FDA and plans to complete the filing in the second half of this year. The company expects to launch the treatment in the U.S. in the first half of next year, if it's approved. If approved, Intellia's treatment, lonvoguran ziclumeran, will compete with about a dozen other chronic drugs for HAE. Despite the allure of a one-time trea...
Intellia trumpets CRISPR drug data, but shares fall
Intellia has reported results from a phase 2 trial of its gene-editing drug for hereditary angioedema (HAE) that it says build the case for the drug as a phase 3 trial gets underway – but its shares fell sharply as investors reacted to the news. The CRISPR specialist said that a single 50mg infusion of NTLA-2002 – which switches off a gene involved in the potentially life-threatening inflammatory attacks that characterise HAE – resulted in an average monthly attack rate reduction of 77% over 16 weeks of follow-up in the 27-subject trial. There was an 81% reduction compared to placebo during weeks five to 16, it added, and eight of the 11 patients in the 50mg arm were completely attack-free at the end of that period, suggesting NTLA-2002 – pitching to become the first in vivo CRISPR therapy – could be a "functional cure" for HAE. Despite that positive assessment of the data, which was presented at the American College of Allergy, Asthma & Immunology (ACAAI) conference and simultaneously published in the New England Journal of Medicine, shares in Intellia fell more than 20% to $15.85, a 52-week low. The weakness came after analysts at Baird expressed the view that the results were not compelling enough to offset the perceived risks associated with in vivo gene editing, and reduced the price target for Intellia's stock from $24 to $18. They also pointed out that NTLA-2002 will enter a competitive market if approved. A series of injectable and oral treatments that require chronic dosing are already available, while others are nearing the market, including KalVista Pharma's recently filed oral plasma kallikrein inhibitor sebetralstat. The lead investigator in the phase 2 trial, Danny Cohn of Amsterdam University Medical Center in the Netherlands, said that approved HAE therapies can reduce, but frequently do not eliminate all, angioedema attacks and require lifelong administration. He said the new data is remarkable and suggests Intellia's therapy could permanently stop swelling attacks with a single infusion, a property that was highlighted by Stifel analysts who said that focusing on the attack rate data "misses the bigger picture." That view was echoed by Intellia's chief executive, John Leonard, who said the data "underscore[s] the tremendous potential of [the] in vivo CRISPR gene editing therapy" and "sets NTLA-2002 apart from other prophylaxis treatments." The company recently announced the start of patient screening for a phase 3 trial, called HAELO, whi...
Intellia Therapeutics moves HAE treatment closer to approval | STAT
Intellia's CRISPR-based treatment for hereditary angioedema dramatically reduced swelling attacks in a Phase 3 trial, setting stage for FDA approval.
Intellia Readies First Phase 3 In Vivo CRISPR Test For HAE Thesis
Intellia Therapeutics (NasdaqGM:NTLA) plans to announce topline results from the first Phase 3 trial of an in vivo CRISPR gene editing candidate for hereditary angioedema. The upcoming readout will be the first-ever Phase 3 data release for an in vivo CRISPR therapy. The results are expected to be highly relevant for Intellia's future plans and for gene editing drug development more broadly ...
