Intellia Therapeutics, Inc. (NTLA) has put up a dismal performance in 2024. Shares of the company have plunged 60.4% compared with the industry’s decline of 13.7%. The deterioration was more pronounced in the past five months. The stock has also underperformed the sector and the S&P 500 Index during this time frame.Intellia is a leading clinical-stage gene editing company focused on developing innovative CRISPR-based therapies. While these innovative therapies have been in the spotlight following the FDA ap ...

Regulatory Designation and Impact - The FDA granted the Regenerative Medicine Advanced Therapy (RMAT) designation to Intellia Therapeutics' investigational in vivo genome-editing candidate, Nexiguran ziclumeran (nex-z, also known as NTLA-2001), for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN) [1] - The RMAT designation facilitates early interactions with the FDA, including discussions on surrogate or intermediate endpoints, and supports accelerated approval and potential priority review of the biologics license application (BLA) [2] - Shares of Intellia rose 3.4% following the announcement, although the stock has declined 51.6% year-to-date compared to the industry's 9% decline [3] Clinical Development and Collaborations - Interim data from a phase I study showed that a one-time treatment with nex-z led to rapid, deep, and durable TTR reduction, which is likely to halt and potentially reverse the disease [4] - Intellia has a co-development and co-promotion agreement with Regeneron Pharmaceuticals for nex-z, with Regeneron sharing 25% of the development costs and commercial profits [5] - The FDA cleared Intellia's investigational new drug (IND) application for the pivotal phase III MAGNITUDE-2 study, which will evaluate nex-z for ATTRv-PN, with patient enrollment expected to begin outside the U.S. by the end of 2024 [6] Ongoing Studies and Future Prospects - The phase III MAGNITUDE study is currently evaluating the safety and efficacy of nex-z in patients with ATTR amyloidosis with cardiomyopathy (ATTRv-CM), with enrollment ongoing [7] - Positive data from the MAGNITUDE study could enable global regulatory filings for nex-z, making its successful development crucial for Intellia [7] Industry Context and Peer Performance - Intellia currently holds a Zacks Rank 3 (Hold), while other biotech companies like Spero Therapeutics (SPRO) and Castle Biosciences (CSTL) are ranked 1 (Strong Buy) [8] - Spero Therapeutics' 2024 loss per share estimates narrowed from $1.59 to $1.13, and its 2025 estimates narrowed from $1.54 to $0.54, with shares declining 21.7% year-to-date [9] - Castle Biosciences' 2024 loss per share estimates narrowed from $0.58 to $0.08, and its 2025 estimates narrowed from $2.13 to $1.88, with shares surging 36.8% year-to-date [10] - Castle Biosciences has consistently beaten earnings estimates in the past four quarters, with an average surprise of 172.72% [11]

CAMBRIDGE, Mass, Nov. 25, 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 the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN). Nex-z is an in viv ...

Shares of biotech Intellia Therapeutics (NTLA 5.18%) are down by around 33% over the past 30 days, amid the publication of some new data from an early stage clinical trial on Nov. 16 and its third-quarter earnings on Nov. 7.Usually, updates like those two would act as positive catalysts for a stock, assuming there was good news to share.In this case, there wasn't exactly any bad news, but the stock is clearly still smarting from the damage in October, when some clinical results from its gene editing program ...

I'm an investment analyst with clinical experience in healthcare and an MBA, expanding my focus from biotech to a range of sectors. I've had the privilege of sharing my insights on Seeking Alpha since 2017, emphasizing financial modeling techniques like DCF analysis to identify underlying assumptions in stock valuations. I provide scenario-based forecasts to help readers gauge reasonable outcomes. Influenced by works like Superforecasting and Antifragile, I advocate for disciplined risk management through a ...

Consistently rapid, deep and durable reduction in serum TTR accompanied by evidence of disease stabilization or improvement after a one-time treatment of nex-z, supporting the hypothesis that greater TTR reduction may lead to a greater clinical benefit in ATTR amyloidosis Favorable trends consistently observed across multiple markers of cardiac disease progression at month 12 compared to baseline in an ATTR-CM population with a high proportion of advanced heart failure patientsConsistent trend observed to d ...

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 ...

Intellia Therapeutics, Inc. (NTLA) incurred third-quarter 2024 loss of $1.34 per share, which was narrower than the Zacks Consensus Estimate of a loss of $1.37. In the year-ago quarter, Intellia had incurred a loss of $1.38 per share.The company’s total revenues currently comprise only collaboration revenues. Intellia reported revenues of $9.1 million for the third quarter of 2024 compared with $12 million reported in the year-ago quarter. Revenues however beat the Zacks Consensus Estimate of $8 million.The ...

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]