Longer-term data to be presented at the 5th International ATTR Amyloidosis Meeting for Patients and Doctors on September 25 highlighting up to three years of patient follow-up and additional insight into ATTRv-PN disease-relevant measuresCAMBRIDGE, Mass., Sept. 22, 2025 (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 longer-term data from the ongoing Phase 1 tria ...

If you are interested in keeping up to date with stocks making moves within the biotech, pharma and healthcare industries, and understanding the key trends and catalysts driving valuations ahead of the market, why not subscribe to my weekly newsletter via my Investing Group, Haggerston BioHealth ?My Buy call on Intellia Therapeutics, Inc. (NASDAQ: NTLA ) stock made in a Seeking Alpha post published last month—August 15th, to be precise—has already paid off. Shares have been soaring in value thisThe group is ...

Group 1 - The article highlights the success of a buy call on Intellia Therapeutics, Inc. (NASDAQ: NTLA) made on August 15, which has resulted in a significant increase in the stock's value [1] - The investing group Haggerston BioHealth offers insights for both novice and experienced biotech investors, including catalysts to monitor and buy/sell ratings [1] - The group provides comprehensive financial analyses, including product sales forecasts, integrated financial statements, discounted cash flow analysis, and market-by-market analysis for major pharmaceutical companies [1] Group 2 - The author, Edmund Ingham, has over five years of experience in covering biotech, healthcare, and pharma sectors, having prepared detailed reports on more than 1,000 companies [1]

Core Insights - Intellia Therapeutics' shares increased by 29.8% following the completion of enrollment in the phase III HAELO study for its investigational candidate lonvoguran ziclumeran (lonvo-z) aimed at treating hereditary angioedema (HAE) [1][8] - Top-line data from the pivotal HAELO study is anticipated in the first half of 2026, with a potential biologics license application submission planned for the second half of 2026 [2][8] - The company aims to launch lonvo-z in the U.S. in the first half of 2027 [2] Company Developments - Intellia began dosing the first patient in the phase III HAELO study in January 2025, with full enrollment completed within nine months [6][7][8] - The study focuses on the safety and efficacy of lonvo-z in adults with type I or type II HAE, measuring the number of HAE attacks and the number of patients achieving attack-free status [6][8] - Lonvo-z is designed to prevent HAE attacks by suppressing plasma kallikrein activity, addressing the limitations of current chronic treatment options [9] Additional Candidate Development - Intellia is also developing nexiguran ziclumeran (nex-z) in collaboration with Regeneron Pharmaceuticals, targeting ATTR amyloidosis with polyneuropathy (ATTRv-PN) and ATTR amyloidosis with cardiomyopathy (ATTR-CM) [10][11] - The phase III MAGNITUDE study for nex-z in treating ATTR-CM is currently enrolling patients, with the first patient in the MAGNITUDE-2 study for ATTRv-PN dosed in April 2025 [11] - Positive data from the MAGNITUDE-2 study could lead to global regulatory filings for nex-z [12] Stock Performance - Year-to-date, Intellia's shares have risen by 38.2%, significantly outperforming the industry average increase of 3.3% [5]

Core Points - Intellia Therapeutics, Inc. (NTLA) shares increased by 29.8% to $16.11, with a trading volume higher than average, following a 21.6% gain over the past four weeks [1][2] - The stock's rally was driven by the completion of enrollment in the pivotal phase III HAELO study for its investigational candidate, lonvoguran ziclumeran (lonvo-z), aimed at treating hereditary angioedema (HAE) [2] - Top-line data from the HAELO study is anticipated in the first half of 2026, with a potential biologics license application submission planned for the second half of 2026 [3] - If approved, the company intends to launch lonvo-z in the United States in 2027 [3] - The company is projected to report a quarterly loss of $1.01 per share, reflecting a year-over-year increase of 24.6%, while revenues are expected to reach $14.38 million, up 57.8% from the previous year [3] - The consensus EPS estimate for the quarter has been revised 0.5% higher in the last 30 days, indicating a positive trend that may lead to price appreciation [5] - Intellia Therapeutics holds a Zacks Rank of 3 (Hold), indicating a neutral outlook [6] Industry Context - Intellia Therapeutics is part of the Zacks Medical - Biomedical and Genetics industry, which includes other companies like CG Oncology, Inc. (CGON) [6] - CGON's stock closed 2.9% higher at $38.82, with a 47.8% return over the past month, while its consensus EPS estimate has remained unchanged at -$0.57, representing a 90% decrease compared to the previous year [6][7]

Core Insights - Intellia Therapeutics has completed enrollment in the global Phase 3 HAELO study for lonvoguran ziclumeran (lonvo-z) targeting hereditary angioedema (HAE) with topline data expected in the first half of 2026 [1][2] - The company plans to submit a biologics license application (BLA) in the second half of 2026, aiming for a U.S. launch in the first half of 2027 [1][6] Company Overview - Intellia Therapeutics is a clinical-stage gene editing company focused on CRISPR-based therapies [1][7] - The company aims to address unmet medical needs through innovative gene editing technologies [7] Study Details - The Phase 3 HAELO study is a randomized, double-blind, placebo-controlled trial involving at least 60 adults and adolescents aged 16 years and older with Type I or Type II HAE [2][4] - Key endpoints include the number of HAE attacks and the number of patients achieving attack-free status from week 5 through week 28 [2][4] Treatment Potential - Lonvo-z is designed to be a one-time treatment for HAE, utilizing CRISPR technology to inactivate the KLKB1 gene, which is crucial for preventing HAE attacks [4][5] - Interim Phase 1/2 clinical data indicated significant reductions in attack rates and consistent decreases in kallikrein levels [4] Regulatory Designations - Lonvo-z has received multiple regulatory designations, including Orphan Drug and RMAT Designation from the FDA, Innovation Passport from the U.K. MHRA, and PRIME Designation from the European Medicines Agency [4]

Investment Opportunities - Identifying stocks that can rise 1,000% or more is essential for building significant wealth, as these investments can dramatically increase portfolio value [1][2] - Tronox Holdings Plc (TROX), a major producer of titanium dioxide, is highlighted as a potential investment with a projected return to the $20 range, representing a 4X return from current levels [3] - Intellia Therapeutics Inc. (NTLA) has seen its shares drop significantly but shows potential for a 1,000% upside due to promising drug trials and significant backing from Regeneron Pharmaceuticals Inc. [11][12] - WeRide Inc. (WRD), a leading robotaxi firm in China, is positioned for substantial growth with a potential 1,000% upside through 2030 as it expands into international markets [18] Market Trends - The Chinese auto market is evolving rapidly, with local manufacturers producing advanced vehicles that compete with Western models, particularly in the robotaxi sector [13][14] - The number of self-driving taxis in China is expected to grow significantly, with estimates suggesting up to 4 million robotaxis by 2030 [14] Investment Strategies - A quantitative system called Apogee has been developed to identify high-quality stocks that have fallen significantly but show signs of recovery, focusing on the "down a lot, up a little" strategy [3][21] - The system has already identified several companies with potential for substantial gains, emphasizing the importance of recognizing turnaround signals in stock performance [21]

Group 1: Cathie Wood's Investment Strategy - Cathie Wood focuses on seizing opportunities, viewing stock price drops as chances to increase investments at favorable prices, resulting in an 80% increase in her Ark Innovation fund over the past year [1][2] - Wood invests in innovative technology companies, regardless of their current product launches or revenue streams, demonstrating patience for long-term growth [2] Group 2: CRISPR Therapeutics - CRISPR Therapeutics specializes in CRISPR gene editing technology, which addresses faulty genes related to diseases by cutting DNA at specific locations [5] - The company achieved a significant milestone with the approval of its first treatment, Casgevy, for blood disorders, although the treatment process is complex and requires time to generate significant revenue [6][7] - CRISPR Therapeutics has launched 75 treatment centers and collected cells from 115 patients, and it has $1.7 billion in cash to support its ongoing programs [7][8] - The stock has decreased by 20% from its 2025 high, and Wall Street predicts a potential 50% increase in the next 12 months, positioning the company for long-term gains [9] Group 3: Intellia Therapeutics - Intellia Therapeutics also develops CRISPR gene editing candidates, with its lead project NTLA-2002 targeting hereditary angioedema (HAE) [10] - Earlier trials of NTLA-2002 showed a remarkable 98% reduction in the monthly HAE attack rate after three years [11] - The company plans to submit NTLA-2002 for regulatory review in the second half of next year and has over $630 million in cash to support operations through the first half of 2027 [12] - Wall Street forecasts a potential 190% gain for Intellia's stock, indicating strong bullish sentiment despite the absence of a market product [13]

Group 1 - The article discusses the performance of Intellia Therapeutics, Inc. (NASDAQ: NTLA), a company focused on developing gene therapies using CRISPR technology, highlighting a significant decline in its share price as of late May [1] - The Haggerston BioHealth investing group provides insights for both novice and experienced biotech investors, including catalysts to monitor, buy and sell ratings, and forecasts for major pharmaceutical companies [1] - The article emphasizes the importance of understanding key trends and catalysts that drive valuations in the biotech, pharma, and healthcare industries [1]

Core Insights - Intellia Therapeutics reported a second-quarter 2025 loss of $0.99 per share, which is an improvement from a loss of $1.31 per share in the same quarter last year and better than the Zacks Consensus Estimate of a loss of $1.03 [1][4] - Total revenues for the second quarter of 2025 were $14.2 million, exceeding the Zacks Consensus Estimate of $13 million, and showing significant year-over-year growth driven by collaboration revenues with Regeneron Pharmaceuticals [2][4] - Research and development expenses decreased by 15% year-over-year to $97 million, attributed to lower employee-related expenses and stock-based compensation [4][5] - As of June 30, 2025, Intellia had cash and marketable securities totaling $630.5 million, down from $707.1 million as of March 31, 2025, which is expected to fund operations into the first half of 2027 [6] Pipeline Developments - Intellia is advancing its investigational in vivo genome-editing candidate, nexiguran ziclumeran (nex-z, also known as NTLA-2001), for two indications: ATTR amyloidosis with polyneuropathy and ATTR amyloidosis with cardiomyopathy [7][8] - The Phase III MAGNITUDE study is currently evaluating the safety and efficacy of nex-z, with ongoing enrollment; however, a participant experienced grade 4 liver transaminase elevations, raising safety concerns [9] - Intellia has also initiated the Phase III HAELO study for NTLA-2002, targeting hereditary angioedema, with patient randomization expected to be completed in the third quarter of 2025 [11] Financial Performance - Year-to-date, Intellia's stock has declined by 1.7%, compared to a 1.2% decline in the industry [2] - General and administrative expenses decreased by 14% year-over-year to $27.2 million, primarily due to reduced stock-based compensation [5]