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]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 Commission File Number: 001-37766 INTELLIA THERAPEUTICS, INC. (Exact Name of Registrant as Specified in its Charter) FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to (State or Other Jurisdiction of (I.R.S. Employer I ...

Financial Data and Key Metrics Changes - As of June 30, 2025, the company's cash, cash equivalents, and marketable securities were approximately $630.5 million, down from $861.7 million as of December 31, 2024 [27] - Collaboration revenue increased to $14.2 million in Q2 2025 from $6.9 million in the prior year quarter, driven by cost reimbursements related to collaboration with Regeneron Pharmaceuticals [28] - R&D expenses decreased to $97 million in Q2 2025 from $114.2 million in the prior year quarter, primarily due to lower employee-related expenses and stock-based compensation [28] - G&A expenses decreased to $27.2 million in Q2 2025 from $31.8 million in the prior year quarter, mainly due to lower stock-based compensation [29] Business Line Data and Key Metrics Changes - The company is increasing enrollment in the Phase 3 study evaluating Nexi in ATTR cardiomyopathy to approximately 1,200 patients, up from the previous target of 765 patients [7][20] - Enrollment in the HALO study for LONVOZIE in HAE has exceeded expectations, with a notable screening of 41 patients in April alone [14][15] - The company anticipates completing enrollment in the HAE and ATTR polyneuropathy programs earlier than previously thought [6] Market Data and Key Metrics Changes - The company noted strong interest from both patients and physicians in its clinical trials, which has led to faster-than-expected enrollment [6][10] - The competitive landscape for TTR therapies is evolving, with the company positioning Nexi as a formidable competitor in a growing market [20][105] Company Strategy and Development Direction - The company is focused on transforming the treatment landscape for HAE and ATTR amyloidosis, with plans to launch LONVOZIE for HAE by 2027 [5][30] - The company is building its commercial and medical teams to prepare for a successful product launch, having filled key leadership positions [12] - The company aims to demonstrate a significant clinical benefit over existing therapies, particularly in the context of combining Nexi with stabilizers [62] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's plans and execution, highlighting the strong demand for its clinical trials and the potential for significant clinical benefits from its therapies [12][30] - The company is committed to maintaining a solid cash runway to support its operating plans into 2027, despite the increased enrollment in clinical trials [29][40] Other Important Information - The Chief Medical Officer announced plans for retirement in August 2026, with a search for a successor already underway [13] - The company is focused on ensuring a seamless transition in leadership while continuing to advance its clinical pipeline [13] Q&A Session Summary Question: What is the target proportion of patients on stabilizers in the ATTR study? - The company estimates that approximately 70% of patients will be on stabilizers, which is higher than the initial estimate of 50-60% [35] Question: How will cash runway be affected by the trial expansion? - The company indicated that the increase in enrollment to 1,200 patients will not significantly impact cash runway or net cash burn guidance [40] Question: What is the latest regarding Phase 3 events accrual rate for Nexe? - The company is not disclosing specific event rates but is optimistic about the potential for robust outcomes based on emerging data [42] Question: How does the enrollment pace compare to competitors? - The company noted that despite the availability of new therapies, enrollment has increased, indicating strong interest from patients and physicians [49] Question: Will the study be powered to show a statistically significant difference in the subset on stabilizer background? - Yes, the study is now powered to show a statistically significant difference in that subset [76] Question: What are the qualities of patients enrolling in gene editing studies? - Patients are motivated to find permanent solutions for their diseases, as evidenced by their willingness to wash out from standard therapies to participate in trials [80] Question: How does the company view the competitive landscape for Nexi? - The company believes Nexi will be a formidable competitor in the market, particularly due to its unique profile and the unmet needs in the treatment of ATTR amyloidosis [105]

[Q2 2025 Earnings Release Overview](index=1&type=section&id=Intellia%20Therapeutics%20Announces%20Second%20Quarter%202025%20Financial%20Results%20and%20Highlights%20Recent%20Company%20Progress) Intellia Therapeutics reported accelerated clinical trial progress for ATTR-CM and HAE, with $630.5 million cash on hand, extending its financial runway into H1 2027 [Second Quarter 2025 Highlights](index=1&type=section&id=Second%20Quarter%202025%20Highlights) Q2 2025 highlights include accelerated enrollment in Phase 3 trials for ATTR-CM and HAE, an expanded MAGNITUDE trial, and a strong cash position of $630.5 million - Enrollment for the Phase 3 MAGNITUDE trial (ATTR-CM) and Phase 3 HAELO study (HAE) is tracking ahead of schedule, leading to an accelerated timeline for the HAELO study and an expansion of the MAGNITUDE trial[3](index=3&type=chunk)[4](index=4&type=chunk) - The company plans to expand the MAGNITUDE study enrollment from 765 to approximately **1,200 patients** to provide a more robust dataset, with no expected impact on projected timelines or financial runway[4](index=4&type=chunk)[9](index=9&type=chunk) - Ended Q2 2025 with approximately **$630.5 million** in cash, cash equivalents, and marketable securities, which is expected to fund operations into H1 2027 and through the first anticipated commercial launch[4](index=4&type=chunk)[14](index=14&type=chunk) [Clinical Program Updates](index=2&type=section&id=Clinical%20Program%20Updates) The company provided updates on its HAE and ATTR amyloidosis programs, detailing accelerated trial progress and positive long-term efficacy data [Hereditary Angioedema (HAE) Program: Lonvoguran ziclumeran (lonvo-z)](index=2&type=section&id=Hereditary%20Angioedema%20%28HAE%29) The HAE program for lonvo-z shows accelerated Phase 3 trial progress, with long-term data demonstrating a 98% reduction in HAE attacks and a BLA submission targeted for H2 2026 - Randomization in the global Phase 3 HAELO study is expected to be completed in **Q3 2025**, earlier than originally planned[6](index=6&type=chunk) - Three-year follow-up data from the Phase 1/2 study showed a single dose of lonvo-z resulted in a **98% mean reduction** in monthly HAE attack rates, with all 10 patients attack-free and treatment-free for a median of nearly two years[6](index=6&type=chunk) - The company remains on track to submit a Biologics License Application (BLA) for lonvo-z in the **second half of 2026**[6](index=6&type=chunk) [Transthyretin (ATTR) Amyloidosis Program: Nexiguran ziclumeran (nex-z)](index=2&type=section&id=Transthyretin%20%28ATTR%29%20Amyloidosis) The nex-z program for ATTR amyloidosis is rapidly advancing, with accelerated enrollment in both ATTR-CM and ATTRv-PN Phase 3 trials and strong TTR reduction data - Nex-z is an investigational in vivo CRISPR-based therapy designed to inactivate the TTR gene, aiming for a lifelong reduction in TTR protein after a single dose to halt and reverse ATTR amyloidosis[6](index=6&type=chunk)[7](index=7&type=chunk) [ATTR Amyloidosis with Cardiomyopathy (ATTR-CM)](index=3&type=section&id=ATTR%20Amyloidosis%20with%20Cardiomyopathy%20%28ATTR-CM%29) Enrollment for the Phase 3 MAGNITUDE trial in ATTR-CM is ahead of schedule, leading to an expansion of the study size for a more robust dataset - Enrollment in the Phase 3 MAGNITUDE trial is ahead of projections, with plans to expand total enrollment from 765 to approximately **1,200 patients** to generate a more robust dataset[9](index=9&type=chunk) - Phase 1 data presented in May 2025 showed nex-z effectively reduced TTR production and showed promise for treating both wild-type (ATTRwt) and variant (ATTRv) forms of ATTR-CM with a favorable safety profile[9](index=9&type=chunk) [Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN)](index=3&type=section&id=Hereditary%20ATTR%20Amyloidosis%20with%20Polyneuropathy%20%28ATTRv-PN%29) The Phase 3 MAGNITUDE-2 study for ATTRv-PN is enrolling ahead of schedule, with two-year follow-up data showing significant TTR reduction and neurological improvement - Enrollment in the global Phase 3 MAGNITUDE-2 study is ahead of schedule, with completion now expected in the **first half of 2026**[9](index=9&type=chunk) - Two-year follow-up data from Phase 1 showed a mean serum TTR reduction of **90% by Day 28**, which remained durable through 24 months, with 13 of 18 patients showing clinically meaningful neurological improvement (≥ 4 point improvement in mNIS+7)[9](index=9&type=chunk)[10](index=10&type=chunk) [Platform and Company Updates](index=4&type=section&id=Platform%20and%20Company%20Updates) Intellia is advancing its CRISPR platform with gene writing and extrahepatic delivery technologies while building commercial infrastructure for future product launches [Platform Technology and Commercial Readiness](index=4&type=section&id=Platform%20Technology%20and%20Commercial%20Readiness) Intellia is developing advanced CRISPR technologies and expanding its commercial and medical affairs teams to prepare for future product launches - The company is developing novel CRISPR technologies like gene writing and extrahepatic lipid nanoparticle (LNP) delivery to create differentiated in vivo and ex vivo product candidates[12](index=12&type=chunk) - Intellia has expanded its commercial and medical affairs teams, including key leadership hires in U.S. sales and commercial operations, to build a strong foundation for commercial readiness[12](index=12&type=chunk) [Upcoming Events](index=4&type=section&id=Upcoming%20Events) Intellia will participate in several key industry and medical conferences in Q3 2025, including events hosted by Citi, Wells Fargo, Bernstein, and the International ATTR Amyloidosis Meeting - The company will present at four conferences in September 2025: - Citi 2025 Biopharma Back to School Conference (Sept. 3) - Wells Fargo Health Care Conference (Sept. 4) - Bernstein Healthcare Forum (Sept. 23) - 5th International ATTR Amyloidosis Meeting (Sept. 25-26)[12](index=12&type=chunk) [Second Quarter 2025 Financial Results](index=5&type=section&id=Second%20Quarter%202025%20Financial%20Results) The company reported its Q2 2025 financial performance, detailing collaboration revenue, operating expenses, net loss, and cash position [Financial Summary](index=5&type=section&id=Financial%20Summary) Q2 2025 saw increased collaboration revenue, decreased operating expenses, a reduced net loss of $101.3 million, and a strong cash position of $630.5 million Q2 2025 Key Financial Metrics | Financial Metric | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | Collaboration Revenue | $14.2M | $6.9M | +$7.3M | | R&D Expenses | $97.0M | $114.2M | -$17.2M | | G&A Expenses | $27.2M | $31.8M | -$4.6M | | Net Loss | $101.3M | $147.0M | -$45.7M | | Cash Position (as of quarter end) | $630.5M | N/A | N/A | - The increase in collaboration revenue was mainly driven by cost reimbursements from the collaboration with Regeneron Pharmaceuticals, Inc[14](index=14&type=chunk) - The decrease in R&D and G&A expenses was primarily driven by lower employee-related costs and stock-based compensation, partially offset by increased spending on lead programs and commercial infrastructure[14](index=14&type=chunk) [Consolidated Statements of Operations](index=8&type=section&id=Consolidated%20Statements%20of%20Operations) The unaudited consolidated statements of operations detail the company's revenues, expenses, and net loss for the three and six months ended June 30, 2025, compared to the same periods in 2024 Consolidated Statements of Operations (Unaudited, in thousands, except per share data) | | Three Months Ended June 30, | Six Months Ended June 30, | | :--- | :--- | :--- | | | **2025** | **2024** | **2025** | **2024** | | **Collaboration revenue** | **$14,245** | **$6,957** | **$30,872** | **$35,892** | | **Operating expenses:** | | | | | | Research and development | 97,035 | 114,207 | 205,462 | 226,054 | | General and administrative | 27,206 | 31,793 | 56,213 | 62,884 | | Total operating expenses | 124,241 | 146,000 | 261,675 | 288,938 | | **Operating loss** | **(109,996)** | **(139,043)** | **(230,803)** | **(253,046)** | | **Net loss** | **$(101,255)** | **$(146,975)** | **$(215,584)** | **$(254,411)** | | **Net loss per share, basic and diluted** | **$(0.98)** | **$(1.52)** | **$(2.08)** | **$(2.64)** | [Consolidated Balance Sheet Data](index=8&type=section&id=Consolidated%20Balance%20Sheet%20Data) The unaudited consolidated balance sheet data provides a snapshot of the company's financial position as of June 30, 2025, compared to December 31, 2024, highlighting key assets, liabilities, and stockholders' equity Consolidated Balance Sheet Data (Unaudited, in thousands) | | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents and marketable securities | $630,506 | $861,730 | | Total assets | $898,894 | $1,191,015 | | Total liabilities | $183,639 | $319,059 | | Total stockholders' equity | $715,255 | $871,956 |

Core Insights - Intellia Therapeutics, Inc. is a leading clinical-stage gene editing company focused on CRISPR-based therapies aimed at revolutionizing medicine [2] Group 1: Financial Results and Operational Highlights - Intellia will present its second quarter 2025 financial results and operational highlights on August 7, 2025, at 8 a.m. ET [1] - A replay of the conference call will be available on Intellia's website starting August 7, 2025, at 12 p.m. ET [1] Group 2: Company Overview - Intellia is dedicated to developing novel, first-in-class medicines that address significant unmet medical needs and advance treatment paradigms [2] - The company is expanding its CRISPR-based platform with novel editing and delivery technologies to harness the full potential of gene editing [2]

Market Overview - The U.S. markets have shown an upward trend, with the S&P 500 and Nasdaq Composite reaching all-time highs, increasing by 1.32% and 0.64% respectively last week [1] - Positive corporate earnings and expectations of easing tariffs and trade policies have bolstered investor confidence [1] - Concerns over inflation and signs of a potential economic slowdown are influencing the Federal Reserve's cautious stance on interest rates [1] Economic Indicators - The Conference Board's Leading Economic Indicator fell by 0.3% in June, indicating early signs of a slowdown, with weakened consumer expectations and fewer manufacturing orders [2] - The S&P Global's U.S. flash PMI data for July indicated an acceleration in business activity driven by the services sector, while the manufacturing PMI dropped to 49.5 [2] - Initial jobless claims decreased by 4,000 to 217,000 for the week ending July 19, suggesting a resilient labor market with limited layoffs [2] Stock Performance - AngloGold Ashanti plc's shares increased by 23.1% since its upgrade to Zacks Rank 1 on May 19, outperforming the S&P 500's 7.2% increase [3][4] - NN Group N.V. saw a return of 12.1% since its upgrade to Zacks Rank 1 on May 29, compared to the S&P 500's 8.4% increase [4] - Zacks Rank 1 stocks returned +6.51% in May 2025, outperforming the S&P 500's +4.47% [5] Portfolio Performance - A hypothetical portfolio of Zacks Rank 1 stocks has outperformed the S&P 500 index by more than 12 percentage points since 1988 [6] - The Zacks Focus List portfolio returned +8.84% in 2025 (through June 30) compared to +6.21% for the S&P 500 [14] - The Zacks Earnings Certain Admiral Portfolio (ECAP) returned +3.20% in Q1 2025, outperforming the S&P 500's -4.30% decline [18] Notable Stocks - Intellia Therapeutics, Inc. gained 60.3% over the past 12 weeks, while Caterpillar Inc. returned 41.3% in the same period [13][14] - OptimizeRx Corporation and Artisan Partners Asset Management saw increases of 18.2% and 14.6% respectively since their upgrades to Outperform [10] - Hershey Company returned 17.8% over the past 12 weeks, benefiting from investor interest in quality dividend stocks [20]

Core Insights - The individual has retired after over 43 years in investment research, now operating independently to provide actionable investment insights [1] - The focus is on rules and factor-based equity investing strategies, emphasizing the use of numbers to inspire human intelligence-driven investment stories rather than solely relying on statistical studies [1] - The individual combines factor analysis with classic fundamental analysis to uncover the true story of companies and their stocks, highlighting the importance of future potential over past performance [1] Experience and Background - The individual has extensive experience covering a wide range of stocks, including large cap, small cap, micro cap, value, growth, and income [1] - Previous roles include managing a high-yield fixed-income fund and conducting research on quantitative asset allocation strategies, contributing to the development of Robo Advising [1] - The individual has authored two books on stock selection and analysis and has a passion for investor education, having conducted numerous seminars [1]

Market Outlook - Goldman Sachs raised its year-end forecast for the S&P 500 (SPX) to 6,900, up from 6,500, highlighting three stocks for investors to consider [1] - Other major investment banks, including Bank of America, are also increasing their S&P 500 forecasts, reflecting optimism around economic resilience and stabilizing inflation [2][3] - The current SPX stands at 6,263, with a year-to-date increase of 6.49% and a 14.8% rise over the past three months [3] Earnings Projections - Goldman projects S&P 500 earnings-per-share (EPS) to grow by 7% for both this year and next, driven by strong consumer demand and margin expansion [4] - The Federal Reserve is expected to support this growth through earlier and deeper interest rate cuts [4] Stock Recommendations - Goldman highlighted three stocks to watch: Kohl's, Intellia Therapeutics, and Gogo Inc., each linked to structural trends that could drive outperformance [6] Kohl's - Kohl's is viewed as a deep value play with a turnaround catalyst, currently trading down 33.40% for the year but up 20.5% over the past 90 days [7][8] - The company is focusing on inventory discipline, cost-cutting, and enhancing its loyalty program to stabilize revenue [9] - Kohl's is expected to benefit from lower bond yields when the Federal Reserve cuts rates, making it attractive for income-minded investors [10] Intellia Therapeutics - Intellia has seen a 45.3% increase in share price over the past month, focusing on CRISPR-based therapies for rare genetic disorders [11][12] - The company is recognized for its strong intellectual property portfolio and is positioned to benefit from the growing importance of gene therapies [13] Gogo Inc. - Gogo, a leader in business aviation connectivity, has seen its stock price rise 117% over the past three months, driven by strong recurring revenues and a 5G rollout [15][16] - The company has received FAA certification for 42 aircraft types, which covers 70% of its current North American customer base [16] - Gogo's growth is supported by a favorable capital markets backdrop and a strong recurring revenue model [19]

Market Overview - The three major U.S. stock indexes closed lower last week, with the Dow Jones Industrial Average down 1%, the S&P 500 down 0.3%, and the Nasdaq Composite down 0.1% [1] - The market pullback was influenced by renewed U.S. tariff threats, particularly higher levies on Canada and the European Union, raising concerns about economic growth and inflation [2] Earnings Performance - Despite tariff concerns, many investors focused on strong earnings reports from airlines and consumer stocks, indicating a resilient economy [2] - The second-quarter earnings season is critical for assessing the impact of tariffs on corporate profits [3] Zacks Research Performance - Mogo Inc. shares surged 81.8% since being upgraded to Zacks Rank 2 (Buy) on May 8, significantly outperforming the S&P 500's 11.1% increase [4] - ATI Inc. also saw a 32.6% return since its upgrade to Zacks Rank 2 on May 6, compared to the S&P 500's 10.7% increase [5] - A hypothetical portfolio of Zacks Rank 1 (Strong Buy) stocks returned +6.51% in May 2025, outperforming the S&P 500's +4.47% [5] Zacks Recommendations - Amarin Corporation and European Wax Center shares increased by 62.6% and 49.9%, respectively, after being upgraded to Outperform on May 12, against the S&P 500's 10.5% rise [8] - The Zacks Focus List portfolio returned 8.84% in 2025 (through June 30) compared to the S&P 500's +6.21% [12] Portfolio Performance - The Zacks Earnings Certain Admiral Portfolio (ECAP) returned +3.20% in Q1 2025, outperforming the S&P 500's -4.30% decline [16] - The Zacks Earnings Certain Dividend Portfolio (ECDP) returned +5.74% in Q1 2025, compared to the S&P 500's -2.41% [20] - The Zacks Top 10 Stocks portfolio delivered +11.8% year-to-date through June 2025, outperforming the S&P 500's +6.2% [22] Long-term Performance - Since 2004, the Zacks Focus List portfolio has produced an annualized return of +11.60%, compared to +10.22% for the S&P 500 [15] - The Top 10 portfolio has generated a cumulative return of +2,246.8% since 2012, significantly outperforming the S&P 500's +502.3% [24]