Brendan, a Pennsylvanian by birth:-Completed a Ph.D. at Stanford University in the field of organic synthesis (2009). -Worked for a major pharmaceutical company (Merck, 2009-2013).-Worked in biotech including start-ups prior to co-founding 1200 Pharma at the California Institute of Technology (Caltech, 2016).-Became the first employee of 1200 Pharma as it spun out of Caltech garnering major investment (into the 8 figures)-Remains an avid investor, focused on market trends and especially biotechnology stocks ...

Core Insights - Intellia Therapeutics reported a narrower loss of $1.34 per share for Q3 2024, compared to the Zacks Consensus Estimate of a loss of $1.37 and a loss of $1.38 in the same quarter last year [1] - Total revenues for the quarter were $9.1 million, down from $12 million year-over-year, but exceeded the Zacks Consensus Estimate of $8 million [1][2] - The decline in revenues was attributed to reduced income from the AvenCell license and collaboration agreement [2] Financial Performance - Research and development expenses increased by 8.5% year-over-year to $123.4 million, driven by advancements in lead pipeline programs [3] - General and administrative expenses rose by approximately 3.7% year-over-year to $30.5 million, primarily due to increased stock-based compensation [3] - As of September 30, 2024, the company had cash, cash equivalents, and marketable securities totaling $944.7 million, slightly up from $939.9 million as of June 30, 2024 [4] Pipeline Developments - Intellia is collaborating with Regeneron Pharmaceuticals on the investigational in vivo genome-editing candidate, Nexiguran ziclumeran (nex-z), which is currently in a Phase III study for ATTR amyloidosis with cardiomyopathy [5][6] - The FDA has cleared the IND application for the pivotal Phase III MAGNITUDE-2 study for treating hereditary ATTR amyloidosis with polyneuropathy, with patient enrollment expected to begin outside the U.S. by the end of 2024 [7] - The company has initiated the pivotal Phase III HAELO study for NTLA-2002, targeting hereditary angioedema, with the primary endpoint focused on the change in the number of HAE attacks [8] - Intellia plans to dose the first patient in a Phase I/II study for NTLA-3001, aimed at treating alpha-1 antitrypsin deficiency associated lung disease, by the end of 2024 [9] Market Performance - Intellia's shares have declined by 47.6% year-to-date, contrasting with a 2.4% decline in the broader industry [2] - The company currently holds a Zacks Rank of 2 (Buy) [10]

Financing and Cash Position - The company has raised approximately $2,758.3 million to fund operations through various financing methods since inception until September 30, 2024[95]. - Net cash provided by financing activities was $183.4 million during the nine months ended September 30, 2024, including $176.9 million from at-the-market offerings[149]. - As of September 30, 2024, the company had $944.7 million in cash, cash equivalents, and marketable securities[135]. - The company expects to fund ongoing operating expenses and capital requirements into late 2026 based on current cash and collaboration funding[142]. - The company raised an aggregate of $2,758.3 million since inception through various financing methods, including public offerings and collaboration agreements[135]. - The company has $249.1 million in shares of common stock remaining eligible for sale under the amended 2022 Sale Agreement[138]. Clinical Trials and Development - The pivotal Phase 3 MAGNITUDE trial for NTLA-2001 is currently enrolling patients, with a 2:1 randomization of NTLA-2001 to placebo, and the first patients were dosed in March 2024[99]. - The MAGNITUDE-2 trial for hereditary ATTR amyloidosis (ATTRv-PN) is set to evaluate 50 adults, with primary endpoints including change in modified Neuropathy Impairment Score +7 at month 18[100]. - NTLA-2002 has shown a 98% mean reduction in monthly HAE attack rate compared to baseline in a Phase 1 study, with eight out of ten patients remaining completely attack-free for over 18 months[107]. - The company plans to submit a biologics license application for NTLA-2002 in 2026 following the ongoing Phase 3 study[104]. - The Phase 1/2 trial of NTLA-2002 met its primary efficacy and all secondary endpoints, leading to the selection of a 50 mg dose for further evaluation[106]. - The company is advancing multiple ex vivo programs for immuno-oncology and autoimmune diseases, alongside its in vivo candidates[96]. - The company is focused on developing curative CRISPR/Cas9-based medicines and expanding its gene editing capabilities with proprietary technologies[92]. - The company has received FDA clearance for the IND application to initiate the MAGNITUDE-2 trial for ATTRv-PN[100]. - The company is the clinical and commercial lead party in a co-development arrangement with Regeneron for the ATTR program, sharing approximately 25% of worldwide development costs and profits[102]. - In the Phase 1/2 study of NTLA-2002, 8 out of 11 patients in the 50 mg arm remained attack-free for 16 weeks after a single dose, indicating a potential functional cure for HAE[109]. - NTLA-3001 is set to initiate a first-in-human study by year-end 2024, with expectations to dose the first patient in a Phase 1/2 study involving up to 30 patients[111]. - The median reduction in serum TTR was 90% at day 28 after redosing with NTLA-2001, indicating a significant pharmacodynamic effect[112]. Financial Performance - Collaboration revenue decreased by $2.9 million to $9.1 million for the three months ended September 30, 2024, primarily due to a reduction in revenue under the AvenCell license agreement[121]. - Research and development expenses increased by $9.7 million to $123.4 million for the three months ended September 30, 2024, compared to the same period in 2023[124]. - General and administrative expenses rose by $1.1 million to $30.5 million for the three months ended September 30, 2024, mainly due to an increase in stock-based compensation[125]. - Collaboration revenue increased by $6.8 million to $45.0 million for the nine months ended September 30, 2024, compared to $38.2 million in the same period of 2023[129]. - Operating loss for the three months ended September 30, 2024, was $144.8 million, compared to $131.1 million for the same period in 2023, reflecting an increase of $13.7 million[122]. - Total operating expenses for the three months ended September 30, 2024, were $153.9 million, an increase of $10.8 million from $143.1 million in the same period of 2023[122]. - Other income, net increased by $0.2 million to $9.1 million for the three months ended September 30, 2024, primarily due to changes in equity method losses and fair value of investments[126]. - Research and development expenses increased by $23.3 million to $349.4 million for the nine months ended September 30, 2024, compared to $326.1 million for the same period in 2023, representing a 7% increase[130]. - External development expenses for NTLA-2001 rose by $11.7 million (30%) to $51.2 million, while NTLA-2002 expenses increased by $14.7 million (90%) to $31.1 million[131][132]. - General and administrative expenses increased by $5.9 million to $93.4 million during the nine months ended September 30, 2024, primarily due to a $6.3 million increase in stock-based compensation[133]. - Net cash used in operating activities was $263.7 million for the nine months ended September 30, 2024, compared to $301.0 million for the same period in 2023[144][146]. - The company anticipates an increase in expenses for the remainder of 2024 as it continues to develop clinical programs[140].

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].