NTLA-2002

Phase 3 study evaluating NTLA-2002 efficacy & safety vs. placebo in HAE adults. Multinational, multicenter, double-blind, placebo-controlled. 60 participants randomized 2:1. Primary Observation Period: Week 1-28. Long-Term Observation Period: 76 weeks. Total study duration: ~28 months.

CGTLive’s Weekly Rewind highlights gene and cell therapy advances, including improved myelination, adeno-associated virus for muscular dystrophies, FDA resubmission, LX1001 trial for Alzheimer disease, and NTLA-2002 dosing.

The cell and gene therapy sectors are rapidly advancing, with significant developments including Sangamo's FDA alignment for Fabry disease therapy, Ocugen's phase 2 trial approval for Stargardt disease, Intellia's CRISPR therapy showing promise for hereditary angioedema, Immatics' TCR-T therapy demonstrating safety and efficacy, March Biosciences securing funding for CAR-T therapy development, Abeona resubmitting BLA for epidermolysis bullosa therapy, Capsida receiving orphan drug designation for encephalopathy therapy, and RiboX's circular RNA therapy cleared for clinical trials.

A Phase 2 clinical trial of gene-editing therapy NTLA-2002 showed that 50 mg doses led to an 80% reduction in swelling attacks over 16 weeks, with 8 out of 11 patients free from attacks. The 25 mg dose also showed positive results, with 40% of patients experiencing no attacks. Intellia Therapeutics, the developer, plans to advance the 50 mg dose to Phase 3 trials.

Sangamo Therapeutics' shares surged 69% after FDA alignment on an Accelerated Approval pathway for its Fabry disease gene therapy, isaralgagene civaparvovec (ST-920), using data from its Phase I/II STAAR trial. The company plans to submit a BLA in the second half of 2025, three years ahead of previous estimates.

Intellia Therapeutics reports positive Phase II results for NTLA-2002, a CRISPR-based gene editing therapy for hereditary angioedema (HAE), showing significant reduction in attack rates and well-tolerated doses, leading to selection of the 50mg dose for further Phase III evaluation.

Intellia Therapeutics unveiled Phase II data for NTLA-2002, showing 80-81% reduction in monthly attacks with 25-50 mg doses in hereditary angioedema patients. Eight of 11 patients given 50 mg reached complete response. NTLA-2002 is well-tolerated, with no serious toxicities. Intellia plans to advance 50 mg dose to Phase III. Analysts are cautiously optimistic, though stock dropped 9% due to investor overreaction.

Chardan Capital's Geulah Livshits maintains Buy rating on Intellia Therapeutics (NTLA) with a $88.00 price target, citing NTLA-2002's phase II trial data showing 77% reduction in HAE attacks. Despite high expectations not fully met, one-time dosing potential supports the rating. Livshits anticipates positive phase III results and differentiates NTLA-2002 from other treatments.

Intellia advances NTLA-2002 to Phase III, achieving functional cures in 8/11 patients at 50mg, though investor expectations were not fully met.