NTLA-2002

A single dose of CRISPR-based NTLA-2002 reduced angioedema attacks by 75-77% in patients with hereditary angioedema, with 80% of patients in the 50 mg group experiencing complete response.

Intellia Therapeutics' stock dropped 20% after Phase 2 results of NTLA-2002 showed 75-81% reduction in HAE attacks, with 8/11 patients in the 50 mg dose group remaining attack-free for up to 8 months. Despite the therapy being well-tolerated, analysts lowered Intellia's price target to $18 due to concerns over in vivo gene editing risks and market competition.

Intellia Therapeutics reports positive Phase 2 data for NTLA-2002, showing deep attack rate reductions and potential for a functional cure for hereditary angioedema (HAE). Eight of 11 patients in the 50 mg arm were completely attack-free post-infusion. Data published in The New England Journal of Medicine and to be presented at the 2024 ACAAI Scientific Meeting. Intellia is actively screening patients for the global pivotal Phase 3 HAELO study.

Intellia Therapeutics announces positive Phase 2 data for NTLA-2002, a gene editing therapy for hereditary angioedema (HAE), showing significant attack rate reductions and potential for a functional cure. The company selects the 50 mg dose for further evaluation in the Phase 3 HAELO study and aims to redefine HAE treatment. Intellia also reports progress in other clinical programs and a strong financial position.

BMO Capital's Kostas Biliouris maintains Buy rating on Intellia Therapeutics (NTLA) with $70.00 target, citing NTLA-2002's Phase II success, expected Oct 24th data release, and long-term HAE attack elimination potential.

Intellia Therapeutics initiated a Phase 3 trial, HAELO, to assess NTLA-2002, a single-dose gene-editing therapy for hereditary angioedema (HAE). The trial follows promising Phase 1/2 data showing a 98% reduction in swelling attacks. HAELO will evaluate NTLA-2002 in 60 HAE patients, aiming to confirm its efficacy in preventing attacks over six months.

Intellia Therapeutics initiates Phase 3 trial of NTLA-2002 for hereditary angioedema; Poseida Therapeutics reports positive Phase 1 data for P-BCMA-ALLO1 in multiple myeloma; YolTech Therapeutics receives FDA orphan drug designation for YOLT-203 to treat primary hyperoxaluria type 1; KSQ Therapeutics gets FDA clearance for Phase 1/2 trial of CRISPR-edited TIL therapy KSQ-004EX.

Intellia Therapeutics initiates Phase III HAELO trial of NTLA-2002, an in-vivo gene editing therapy for hereditary angioedema, with a placebo-controlled study assessing efficacy and safety in 60 adult subjects.

Intellia Therapeutics initiates Phase 3 HAELO trial for NTLA-2002, a CRISPR gene editing treatment targeting hereditary angioedema, aiming to reduce attacks and kallikrein protein levels. The trial will involve 60 adults, with a 2-to-1 randomization to NTLA-2002 or placebo, followed by a crossover phase. Intellia seeks to compete with Pfizer, BridgeBio Pharma, and Alnylam in rare disease treatments.

Intellia Therapeutics initiates HAELO Phase 3 study of NTLA-2002, an in vivo CRISPR gene editing treatment for hereditary angioedema (HAE), designed as a single-dose to prevent life-threatening swelling attacks. The study will evaluate efficacy and safety in 60 adults with Type I or II HAE, randomized 2:1 to receive a 50 mg infusion of NTLA-2002 or placebo, with primary endpoint being the change in HAE attacks from week 5 to 28.