Executive Summary

NTLA reported Q2 2024 collaboration revenue of $7.0M and a net loss of $147.0M, with diluted EPS of $(1.52); cash and marketable securities ended at ~$939.9M, funding operations into late 2026 .
Phase 2 for NTLA-2002 (HAE) met primary and all secondary endpoints; management selected 50 mg for a pivotal Phase 3 expected to start in 2H 2024 after a successful end-of-Phase 2 FDA meeting, positioning NTLA-2002 as a potential “functional cure” .
NTLA-2001 (ATTR-CM) MAGNITUDE Phase 3 enrollment is tracking ahead of prior projections, now cleared in 12+ countries and active at 35+ sites; updated Phase 1 data (CM and PN arms) are planned for 2H 2024 .
Platform advances: first-ever CRISPR redosing data for NTLA-2001 (55 mg follow-on dose → 90% median TTR reduction at day 28), and MHRA authorization for NTLA-3001 (AATD) with first patient dosing planned in 2H 2024 .
Near-term stock catalysts: detailed HAE Phase 2 data (Q4), HAE Phase 3 initiation, ATTR Phase 1 update and MAGNITUDE enrollment progress; S&P Global consensus estimates were unavailable at query time (note in Estimates Context) .

What Went Well and What Went Wrong

What Went Well

NTLA-2002 Phase 2 met primary and all secondary endpoints at both 25 mg and 50 mg; 50 mg chosen for Phase 3 after greater kallikrein reduction and more complete attack elimination vs 25 mg; successful FDA end-of-Phase 2 meeting .
NTLA-2001 MAGNITUDE Phase 3 (ATTR-CM) enrollment is “well ahead” of projections; regulatory clearance in 12+ countries and >35 active sites; management expects ~100 sites globally .
Platform validation: redosing proof-of-concept in NTLA-2001 showed 90% median TTR reduction at day 28 on follow-on 55 mg dosing for low-dose (0.1 mg/kg) patients; safety consistent with single-dose profile .

Management quote: “We anticipate that NTLA-2002 will be the first in vivo gene editing therapy to come to market… [and] reset the standard of care for HAE” .

What Went Wrong

Collaboration revenue declined year-over-year ($7.0M vs $13.6M in Q2 2023), driven by lower AvenCell agreement-related revenue .
Net loss widened year-over-year ($147.0M vs $123.7M in Q2 2023), reflecting operating loss and negative change in fair value of investments .
Estimates comparison unavailable from S&P Global at query time; unable to quantify beat/miss vs Street for Q2 2024 (see Estimates Context) [functions.GetEstimates errors].

Financial Results

Metric	Q2 2023	Q1 2024	Q2 2024
Collaboration Revenue ($USD Millions)	$13.6	$28.9	$7.0
R&D Expense ($USD Millions)	$115.3	$111.8	$114.2
G&A Expense ($USD Millions)	$30.7	$31.1	$31.8
Total Operating Expenses ($USD Millions)	$145.9	$142.9	$146.0
Operating Loss ($USD Millions)	$(132.3)	$(114.0)	$(139.0)
Net Loss ($USD Millions)	$(123.7)	$(107.4)	$(147.0)
Diluted EPS ($USD)	$(1.40)	$(1.12)	$(1.52)
Cash, Cash Equivalents & Marketable Securities ($USD Millions)	N/A	$953.4	$939.9
Net Income Margin % (Net Loss / Collaboration Revenue)	-910%	-371.5%	-2,112%

Notes: Net margin derived from reported collaboration revenue and net loss in each period .

KPIs and Operating Metrics

KPI	Q2 2023 / Earlier	Q1 2024	Q2 2024
MAGNITUDE Trial – Countries Cleared	N/A	N/A	12+
MAGNITUDE Trial – Active Sites	N/A	N/A	35+
HAE NTLA‑2002 Phase 2 Outcome	N/A	Enrollment complete (planned)	Met primary & all secondary endpoints
HAE NTLA‑2002 Phase 3 Dose	N/A	Dose to be selected post P2	50 mg selected
HAE NTLA‑2002 Phase 3 Size	N/A	N/A	Fewer than 70 patients (placebo-controlled, 6 months)
NTLA‑2001 Redosing PD Effect	N/A	Oral presentation planned	90% median TTR reduction at day 28 after 55 mg follow-on
Stock-Based Comp in R&D ($M)	$25.4 (Q2 2024 disclosed for current quarter)	$20.2	$25.4
Stock-Based Comp in G&A ($M)	$15.4 (Q2 2024 disclosed for current quarter)	$14.0	$15.4
Interest Income ($M)	N/A	$12.6	$12.4
Cash Runway	N/A	Into late 2026	Into late 2026

Segment breakdown: Not applicable; NTLA is pre-commercial with collaboration revenue only .

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
Cash runway	Company-level	Fund operations into late 2026	Fund operations into late 2026	Maintained
NTLA‑2002 HAE – Phase 3 initiation	2H 2024	On track to initiate in 2H 2024	On track; successful EOP2; 50 mg selected	Maintained/clarified
NTLA‑2001 ATTR‑PN – Phase 3 initiation	By year-end 2024	Expect to initiate by year-end	Plan to initiate by year-end; ex‑US ~50 patients	Maintained
NTLA‑3001 AATD – first patient dosing	2H 2024	Dose first patient in 2024	Dose first patient in 2H 2024; MHRA CTA authorized	Refined timing
NTLA‑2001 – updated Phase 1 data	2H 2024	Present in 2H 2024	Present in 2H 2024	Maintained

Earnings Call Themes & Trends

Topic	Previous Mentions (Q4 2023, Q1 2024)	Current Period (Q2 2024)	Trend
HAE dose selection & Phase 3 design	Dose to be selected from P2; P3 planned 2H 2024	EOP2 successful; 50 mg selected; P3 size <70; include women of childbearing globally	Progressing; design clarified
ATTR‑CM MAGNITUDE enrollment	Initiated; >30 dosed; >40 in screening; event‑driven endpoint; conservative powering	12+ countries cleared; >35 sites active; tracking ahead of projections; ~100+ sites planned	Accelerating
ATTR‑PN Phase 3	FDA alignment; ~50 patient ex‑US placebo‑controlled	Plan to initiate by year‑end; FDA does not require US dosing for PN; leverage CM exposure	Advancing
CRISPR redosing platform	Redosing oral presentation accepted (PNS)	First‑ever CRISPR redosing data; 90% median TTR reduction after 55 mg follow‑on; tolerability consistent	Validated
AATD NTLA‑3001	First‑in‑human expected in 2024; insertion via albumin locus	MHRA CTA authorization; first patient dosing in 2H 2024; 50 mg LNP, AAV dose‑escalation	Moving to clinic
Cash runway & balance sheet	Strong cash to late 2026; OpEx steady	~$939.9M cash at 6/30; runway late 2026	Maintained

Management Commentary

“We are on track to begin the Phase III [HAE] trial in the second half of this year… we anticipate that NTLA‑2002 will be the first in vivo gene editing therapy to come to market” — John Leonard, CEO .
“We now know the size of the [HAE Phase 3] trial… fewer than 70 patients… outcomes include attack rate reduction and patients who achieve complete response over a 6‑month interval” — John Leonard, CEO .
“Cash, cash equivalents and marketable securities were approximately $939.9 million as of June 30, 2024… expected to fund operating plans into late 2026” — Edward Dulac, CFO .
“We plan to include safety and TTR reduction data from both the CM and PN arms as well as other clinical endpoints, such as NT‑proBNP, 6‑minute walk test and mNIS+7” — John Leonard, CEO .

Q&A Highlights

Competition in HAE: management emphasized targeting a complete response and functional cure profile vs chronic prophylaxis, aiming to “reset the bar” versus competitors (e.g., Ionis) .
Women of childbearing potential: clarified there was no partial clinical hold; inclusion planned globally in Phase 3 with full FDA alignment .
HAE Phase 3 size/design: “fewer than 70 patients,” placebo‑controlled over six months; endpoints include complete response and attack rate reduction .
Redosing strategy: not planned for current amyloidosis or HAE programs; platform option could matter outside the liver or where additive effect desired .
ATTR‑CM bar vs stabilizers: NTLA‑2001 designed to achieve deeper, durable TTR reduction; management expects outcomes superior to existing agents; enrollment brisk with ~100 sites targeted .

Estimates Context

S&P Global consensus estimates (Primary EPS Consensus Mean, Revenue Consensus Mean; Q2 2024 and prior quarters) were unavailable at query time due to rate limits; we were unable to quantify beat/miss versus Street. We will update when access is available [functions.GetEstimates errors].
Given biotech’s collaboration‑driven revenue, Street models are likely to recalibrate timelines, Phase 3 design clarity (HAE size, 50 mg dose), and pipeline probabilities for NTLA‑2002 and NTLA‑2001 following successful EOP2 and ongoing rapid MAGNITUDE enrollment .

Key Takeaways for Investors

HAE Phase 2 success and 50 mg selection, coupled with a small Phase 3 (<70 patients) and FDA alignment, materially de‑risk NTLA‑2002’s path to a 2026 BLA; detailed P2 data in Q4 could be a major catalyst .
ATTR‑CM MAGNITUDE enrollment pace, breadth of global sites, and upcoming Phase 1 readouts (including clinical endpoints) support NTLA‑2001’s best‑in‑class TTR knockdown thesis; watch HELIOS‑B read‑throughs and potential monotherapy/add‑on dynamics .
First‑ever CRISPR redosing data strengthens Intellia’s platform defensibility and optionality; while not needed for current programs, it could be leveraged for future indications requiring additive effects .
NTLA‑3001’s MHRA authorization and planned 2H 2024 first dosing mark gene insertion entry into clinic; achieving normal AAT levels after single dose would be highly differentiated in AATD .
Financially, NTLA’s ~$939.9M cash and runway into late 2026 enable multi‑program Phase 3 execution without near‑term financing; Street will likely focus on OpEx discipline vs expanding clinical footprint .
Near‑term trading setup: Q4 HAE P2 detail and P3 start, ATTR Phase 1 update, and continued MAGNITUDE site adds are key narrative drivers; absence of Q2 estimate comparison doesn’t change the core pipeline‑driven story .
Medium‑term thesis: one‑time, in vivo gene editing therapies with deep target reductions, manageable safety, and scalable delivery position NTLA for potential category leadership in HAE and ATTR; execution on pivotal trials is paramount .

Intellia Therapeutics, Inc. (NTLA)·Q2 2024 Earnings Summary