Intellia Therapeutics, Inc. reports clinical, regulatory and financing developments as a clinical-stage biopharmaceutical company using CRISPR gene editing and other core technologies. Its recurring updates center on in vivo and ex vivo therapeutic approaches for genetically defined diseases, including lonvoguran ziclumeran, or lonvo-z, for hereditary angioedema and nexiguran ziclumeran, or nex-z, for ATTR amyloidosis with cardiomyopathy and polyneuropathy.
Company announcements also cover Phase 3 clinical data, FDA interactions, biologics license application activity, quarterly financial results, common stock offerings, equity incentive grants and investor conference participation. These updates reflect Intellia's progress from gene-editing research and clinical development toward regulatory and commercial planning for its lead programs.
Intellia Therapeutics (NASDAQ:NTLA) announced that its investigational therapy nexiguran ziclumeran (nex-z) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for treating hereditary transthyretin amyloidosis with polyneuropathy. This single-dose CRISPR-based treatment aims to inactivate the TTR gene to prevent TTR protein production. The designation was granted based on promising Phase 1 data showing rapid, deep, and durable TTR reduction. This marks the third special regulatory designation for nex-z, following Orphan Drug Designations from both the FDA and European Commission.
Intellia Therapeutics announced positive clinical data from its ongoing Phase 1 trial of nexiguran ziclumeran (nex-z), a CRISPR-based gene editing therapy for ATTR amyloidosis. The one-time treatment showed consistently rapid, deep, and durable reduction in serum TTR, with evidence of disease stabilization or improvement. In the ATTR-CM arm, patients demonstrated favorable trends across cardiac disease markers at month 12, with a mean 90% serum TTR reduction. The ATTRv-PN arm showed similar positive trends with 91% TTR reduction at month 12. The therapy maintained a favorable safety profile, with mainly mild to moderate infusion-related reactions reported.
Intellia Therapeutics (NASDAQ:NTLA) reported Q3 2024 financial results and pipeline progress. Key highlights include FDA clearance for MAGNITUDE-2 Phase 3 trial of nexiguran ziclumeran in ATTR amyloidosis, strong enrollment in MAGNITUDE Phase 3 study, and active patient screening in HAELO Phase 3 study for hereditary angioedema. The company ended Q3 with $944.7 million in cash. Financial results show $9.1 million in collaboration revenue, R&D expenses of $123.4 million, and a net loss of $135.7 million. Cash runway extends into late 2026.
Intellia Therapeutics (NASDAQ:NTLA) has granted inducement awards to ten new employees under its 2024 Inducement Plan. The grants include 73,459 restricted stock units (RSUs) of common stock, which will vest in three equal installments on November 1, 2025, 2026, and 2027. These awards were granted outside the company's stockholder-approved equity incentive plans and were approved by Intellia's compensation committee in accordance with Nasdaq Listing Rule 5635(c)(4).
Intellia Therapeutics (NASDAQ:NTLA) has announced two upcoming virtual investor events in November 2024. The first event, scheduled for November 7 at 8 a.m. ET, will present third quarter 2024 financial results and operational highlights. The second event, on November 16 at 11 a.m. CT/12 p.m. ET, will showcase new clinical data from the Phase 1 study of nexiguran ziclumeran (nex-z), their investigational CRISPR-based gene editing therapy for transthyretin (ATTR) amyloidosis. The nex-z data presentation follows a late-breaking oral presentation at the 2024 American Heart Association Scientific Sessions in Chicago. This therapy is being developed in collaboration with Regeneron as a single-dose treatment.
Intellia Therapeutics announced positive Phase 2 results for NTLA-2002, their investigational CRISPR gene editing treatment for hereditary angioedema (HAE). The study showed that a single 50 mg dose resulted in a 77% and 81% mean monthly attack rate reduction compared to placebo during weeks 1-16 and 5-16, respectively. Eight of 11 patients in the 50 mg arm remained completely attack-free following a one-time infusion. The treatment demonstrated an encouraging safety profile, with most adverse events being Grade 1 or 2. Based on these results, Intellia has selected the 50 mg dose for their global pivotal Phase 3 HAELO study.
Intellia Therapeutics (NASDAQ:NTLA) has announced a new date for its investor webcast to review NTLA-2002 Phase 2 data. The webcast will now be held on Thursday, October 24 at 8:30 a.m. ET, instead of the previously announced date of October 28, 2024.
This change does not affect the planned oral presentation at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting in Boston, Massachusetts, scheduled for October 24 – 28.
Investors can join the webcast via a provided link or through the Events and Presentations page on Intellia's website. A replay of the webcast will be available on the company's website for at least 30 days following the call.
Intellia Therapeutics (NASDAQ:NTLA) has initiated HAELO, a global Phase 3 study of NTLA-2002, an investigational in vivo CRISPR gene editing treatment for hereditary angioedema (HAE). NTLA-2002 is designed as a single-dose treatment to prevent potentially life-threatening swelling attacks in HAE patients. The study will evaluate the efficacy and safety of NTLA-2002 in 60 adults with Type I or Type II HAE, with patients randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo.
The primary endpoint is the change in number of HAE attacks from week 5 through week 28. This initiation follows positive safety and efficacy data from the ongoing Phase 1/2 study, which showed dramatic reductions in attack rate and consistent, deep, and durable reductions in kallikrein levels. Detailed results from the Phase 2 portion will be presented at the 2024 ACAAI Annual Scientific Meeting in October.
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on CRISPR-based therapies, has announced the granting of inducement awards to nine new employees under its 2024 Inducement Plan. The awards, granted on October 1, 2024, consist of time-based restricted stock units (RSUs) for 54,944 shares of Intellia's common stock.
The RSUs will vest in three equal installments on October 1 of 2025, 2026, and 2027, subject to the employees' continued service. These awards were granted outside of Intellia's stockholder-approved equity incentive plans, as part of the 2024 Inducement Plan adopted by the board of directors in June 2024. The compensation committee approved the awards as a material inducement for employment, in accordance with Nasdaq Listing Rule 5635(c)(4).
Intellia Therapeutics (NASDAQ:NTLA) announced that new interim data from the ongoing Phase 1 study of nexiguran ziclumeran (nex-z) will be presented at the 2024 American Heart Association (AHA) Scientific Sessions in Chicago. Nex-z is an investigational in vivo CRISPR-based gene editing therapy designed as a single-dose treatment for transthyretin (ATTR) amyloidosis.
The presentation will include safety data, reduction in serum TTR, biomarkers of disease progression, and functional capacity data in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). Dr. Marianna Fontana from University College London will present the findings on November 16, 2024, as part of the Late-Breaking Science session.
Intellia is developing nex-z in collaboration with Regeneron as part of a multi-target discovery, development, and commercialization partnership.