Financial Data and Key Metrics - Cash, cash equivalents, and marketable securities were approximately $944.7 million as of September 30, 2024, compared to $1 billion as of December 31, 2023, driven by $335 million used to fund operations [24] - Collaboration revenue was $9.1 million in Q3 2024, down from $12 million in Q3 2023, primarily due to reduced revenue from the AvenCell license and collaboration agreement [25] - R&D expenses increased to $123.4 million in Q3 2024 from $113.7 million in Q3 2023, driven by the advancement of lead programs [26] - G&A expenses rose to $30.5 million in Q3 2024 from $29.4 million in Q3 2023, primarily due to stock-based compensation [26] Business Line Updates - NTLA-2002 for Hereditary Angioedema (HAE): Phase II results showed 8 out of 11 patients in the 50 mg arm were attack-free for 16 weeks post-treatment, with 80% of patients in the Phase I/II study appearing functionally cured [11][12] - NEX-Z (NTLA-2001) for ATTR Amyloidosis: FDA cleared the IND for the Phase III MAGNITUDE-2 trial, with enrollment expected to begin soon [9][15] - NTLA-3001 for Alpha-1 Antitrypsin Deficiency: First patient dosing in the Phase I/II study is expected by year-end, with non-human primate data showing durable alpha-1 protein levels for 2 years [21] Market and Strategic Direction - The company is leading the field of in vivo CRISPR-based medicines with three active Phase III studies expected by year-end [9] - The emerging product profile of NTLA-2002 aligns with patient and physician needs, driving rapid enrollment in the Phase III HAELO study [13] - The company is advancing gene editing programs in five different tissues, expanding its platform and pipeline [22] Management Commentary - The company is encouraged by the Phase II results for NTLA-2002 and expects to submit a BLA in 2026 [8] - Management highlighted the potential of NEX-Z to significantly reduce TTR levels, which could improve clinical outcomes for ATTR Amyloidosis patients [17] - The company is focused on advancing its in vivo gene insertion programs, with NTLA-3001 representing a major step forward for alpha-1 patients [21] Q&A Session Enrollment in MAGNITUDE-1 - Enrollment is ahead of projections, with 765 patients targeted globally [28] Capital Allocation Priorities - The company has $945 million in cash and is focused on three Phase III studies, with cash expected to fund operations until late 2026 [32] MAGNITUDE-2 Trial Design - The trial is placebo-controlled with 50 patients, designed to provide a solid readout on drug performance and safety [34][35] HAELO Study Randomization - The 2:1 randomization in the HAELO study is designed to accelerate enrollment and provide more data on the active arm [39] AATD Program - The Phase I/II study for NTLA-3001 aims to normalize alpha-1 protein levels, with initial data expected when meaningful [64] Ex-U.S. Strategy for NTLA-2002 - The company plans to launch NTLA-2002 in the U.S. and expand to Europe, with potential partnerships to extend reach [70] ATTR Therapy Excitement - Investigators are excited about the deep and rapid TTR reduction achieved with NEX-Z, which could outperform silencers [80] MAGNITUDE-2 U.S. Initiation - The trial will be conducted ex-U.S. to facilitate a placebo-controlled study, with FDA agreement on the design [74] AHD Program Expansion - Additional sites beyond New Zealand are planned, with data potentially available in 2025 [76]

Received IND clearance from the U.S. FDA to initiate MAGNITUDE-2 Phase 3 trial of nexiguran ziclumeran (nex-z) in patients with hereditary transthyretin (ATTR) amyloidosis with polyneuropathy; on track to initiate study by year-endStrong patient enrollment continues in the MAGNITUDE Phase 3 study of nex-z for ATTR amyloidosis with cardiomyopathy, tracking ahead of plans Plan to present new clinical data from the ongoing nex-z Phase 1 study at upcoming 2024 American Heart Association Scientific SessionsActiv ...

There's nothing bearish at all about what it reported recently.Sometimes with biotech stocks, what a company considered to be good news clashes with what the market is expecting. That was true on Oct. 24 when Intellia Therapeutics (NTLA 2.56%) announced some positive new clinical trial data only to see its stock slide by 18% -- and the price hasn't fully recovered as of yet.What's causing this disconnect, and could Intellia Therapeutics be a buy now? We'll start by answering the first question.A competitor' ...

Third quarter 2024 financial results – November 7, at 8 a.m. ETNew clinical data from the Phase 1 study of nexiguran ziclumeran (nex-z) for the treatment of transthyretin (ATTR) amyloidosis – November 16, at 11 a.m. CT / 12 p.m. ET CAMBRIDGE, Mass., Oct. 31, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced it will be hosting two virtual investor events in November ...

Intellia Therapeutics, Inc. (NASDAQ:NTLA) Call to Discuss NTLA-2002 Phase 2 Study Results Call October 24, 2024 11:00 AM ET Company Participants Lina Li - Senior Director, Investor Relations and Corporate Communications John Leonard - Chief Executive Officer David Lebwohl - Chief Medical Officer. Danny Cohn - Internist, Department of Vascular Medicine, Amsterdam University Medical Center Paula Busse - Professor of Medicine, Division of Clinical Immunology Jim Butler - General Manager, NTLA-2002 Program, Con ...

The ever-volatile biotech sector is making noise today. Viking Therapeutics Inc (NASDAQ:VKTX) is soaring up the charts, while Intellia Therapeutics Inc (NASDAQ:NTLA) stumbles.Viking Could Burn Shorts' ShipVKTX is up 24% to trade at $74.75 today, after the company's third-quarter loss came in below analyst estimates. The big driver though is the encouraging pipeline update on its obesity drug, which showed promising results in clinical studies. This is even more notable considering Viking doesn't have an app ...

Core Viewpoint - Intellia Therapeutics is set to hold an investor webcast on October 24, 2024, to discuss Phase 2 data for its gene editing therapy NTLA-2002, which aims to treat hereditary angioedema (HAE) [1][2]. Group 1: Company Overview - Intellia Therapeutics, Inc. is a clinical-stage gene editing company focused on CRISPR-based therapies, with programs designed for precise editing of disease-causing genes both in vivo and ex vivo [5]. - The company has developed NTLA-2002, which is based on CRISPR/Cas9 technology and aims to be the first one-time treatment for hereditary angioedema by inactivating the KLKB1 gene [3]. Group 2: Product Information - NTLA-2002 has shown promising interim Phase 1 clinical data, demonstrating significant reductions in attack rates and consistent decreases in kallikrein levels [3]. - The therapy has received multiple regulatory designations, including Orphan Drug and RMAT Designation from the U.S. FDA, Innovation Passport from the U.K. MHRA, and PRIME Designation from the European Medicines Agency [3]. Group 3: Industry Context - Hereditary angioedema is a rare genetic disease affecting approximately 1 in 50,000 people, characterized by severe and unpredictable inflammatory attacks [4]. - Current treatment options for HAE often require lifelong therapies, which may involve chronic administration and still result in breakthrough attacks, highlighting the need for innovative solutions like NTLA-2002 [4].

New data to be presented will include biomarkers of disease progression and functional capacity from the ongoing Phase 1 study of nex-z, an investigational in vivo CRISPR gene editing therapy for ATTR amyloidosis CAMBRIDGE, Mass., Oct. 01, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that interim data from the ongoing Phase 1 study of nexiguran ziclumeran (nex ...

Group 1: Ark Invest Portfolio Strategy - Ark Invest, led by Cathie Wood, focuses on aggressive investments in growth businesses pursuing disruptive innovation [1][2] - The portfolio recently increased its holdings in two biotech stocks, indicating a belief in their potential for success despite inherent risks [1][2] Group 2: Intellia Therapeutics - Intellia Therapeutics is a gene-editing biotech aiming to treat inherited rare diseases by editing dysfunctional genes [3][4] - The company has a late-stage program for transthyretin (ATTR) amyloidosis entering phase 3 trials and a mid-stage program for hereditary angioedema (HAE) with upcoming data [4][5] - Financially, Intellia reported approximately $940 million in cash and equivalents, with R&D expenses of about $449 million, providing a runway until late 2026 [5] - Risks include potential scientific or regulatory hurdles that could significantly impact the company's value [6][7] Group 3: Recursion Pharmaceuticals - Recursion Pharmaceuticals holds a focus on using artificial intelligence (AI) in drug development, with a pipeline of therapies for rare diseases [8][9] - The company is merging with Exscientia, expected to close in early 2025, which will enhance its cash position and oncology pipeline [9][10] - The merged entity will have around $850 million in cash and is projected to achieve $100 million in efficiencies post-transaction [10] - The new company anticipates up to 10 clinical data readouts in the next 18 months, presenting potential catalysts for stock movement [10][11] - Collaborations with major biopharma companies like Merck, Roche, and Nvidia suggest confidence in Recursion's future prospects [12][13]

If you like what you have just read and want to receive at least 4 exclusive stock tips every week focused on Pharma, Biotech and Healthcare, then join me at my marketplace channel, Haggerston BioHealth . Invest alongside the model portfolio or simply access the investment bank-grade financial models and research. I hope to see you there. Intellia Therapeutics (NASDAQ: NTLA ) is one of the vanguard of companies that is attempting to convert the amazing and Nobel Prize winning technology of CRISPR/Cas9 into ...