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 ...

Intellia Therapeutics, Inc. (NTLA) incurred second-quarter 2024 loss of $1.31 per share (excluding a one-time expense of change in fair value of investments), which was wider than the Zacks Consensus Estimate of a loss of $1.21. In the year-ago quarter, Intellia had incurred a loss of $1.40 per share. Including the above one-time expense, the company reported a loss of $1.52 per share in the second quarter of 2024. The company's total revenues currently comprise only collaboration revenues. NTLA reported re ...

Corporate Strategy & Pipeline - Intellia plans to initiate two additional in vivo Phase 3 studies in 2024[6] - Intellia expects to submit a BLA for NTLA-2002 for HAE in 2026[16] - Strategic priorities for 2024-2026 include demonstrating human proof-of-concept for targeted in vivo gene insertion and initiating clinical development for its allogeneic ex vivo program[3] NTLA-2001 (nexiguran ziclumeran) for ATTR Amyloidosis - NTLA-2001 led to a -91% change from baseline in serum TTR at day 28[34] - A Phase 3 trial is underway for ATTR-CM patients with NT-proBNP baseline ≥ 1000 pg/mL[38] - GlobalData projects an $11B+ global market size for ATTR amyloidosis by 2029[28] NTLA-2002 for Hereditary Angioedema (HAE) - NTLA-2002 demonstrated a 98% reduction in HAE attack rate, with 8 of 10 patients attack-free in the post-primary observation period[48] - NTLA-2002 continues to show dose-dependent and durable reductions in plasma kallikrein protein over time[46] - GlobalData projects a $6B+ global market size for HAE by 2029[41] NTLA-3001 for Alpha-1 Antitrypsin Deficiency (AATD) - NTLA-3001 aims to achieve normal human levels of AAT protein and halt progression of lung disease[50] - In preclinical studies, NTLA-3001 achieved durable production of physiologic levels of hAAT through one year in NHPs[51]

Intellia Therapeutics, Inc. (NTLA) came out with a quarterly loss of $1.31 per share versus the Zacks Consensus Estimate of a loss of $1.21. This compares to loss of $1.40 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of -8.26%. A quarter ago, it was expected that this company would post a loss of $1.35 per share when it actually produced a loss of $1.06, delivering a surprise of 21.48%. Over the last four quarters, the compan ...

Core Insights - Intellia Therapeutics reported positive results from the Phase 2 study of NTLA-2002 for hereditary angioedema, meeting all primary and secondary endpoints, and plans to advance to a pivotal Phase 3 trial with a selected 50 mg dose [1][2][3] - The company is on track to initiate multiple Phase 3 trials for NTLA-2001 and NTLA-3001 by the end of 2024, expanding its clinical pipeline [1][4][5] - Intellia ended Q2 2024 with approximately $940 million in cash, indicating a strong financial position to support ongoing and future clinical trials [1][9] Hereditary Angioedema (HAE) - NTLA-2002 demonstrated significant efficacy with a 98% mean reduction in monthly HAE attack rate and a high percentage of patients remaining attack-free for over 18 months after treatment [3] - The FDA has provided support for the Phase 3 plans following a successful end-of-Phase 2 meeting, with the trial expected to start in the second half of 2024 [2][3] Transthyretin (ATTR) Amyloidosis - NTLA-2001, now known as nexiguran ziclumeran (nex-z), is advancing rapidly in the Phase 3 MAGNITUDE trial for ATTR amyloidosis with cardiomyopathy, with regulatory approvals in over 12 countries [4] - A pivotal Phase 3 trial for hereditary ATTR amyloidosis with polyneuropathy is planned to begin by year-end 2024 [4] Alpha-1 Antitrypsin Deficiency (AATD) - NTLA-3001 is set to initiate a Phase 1/2 study in the second half of 2024, targeting AATD-associated lung disease through CRISPR-mediated gene insertion [5] Financial Performance - The company reported a net loss of $147 million for Q2 2024, compared to a loss of $124 million in Q2 2023, with total operating expenses slightly increasing year-over-year [9][16] - Collaboration revenue decreased to $7 million in Q2 2024 from $13.6 million in the same quarter of 2023, primarily due to reduced revenue from the AvenCell collaboration [9][16] Corporate Updates - Intellia appointed Brian Goff to its board of directors and Edward Dulac as Chief Financial Officer, enhancing its leadership team [8]