Intellia Therapeutics(NTLA) - 2025 Q2 - Quarterly Results

Q2 2025 Earnings Release Overview 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 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][4] - 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][9] - 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][14] Clinical Program Updates 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) 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] - 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] - The company remains on track to submit a Biologics License Application (BLA) for lonvo-z in the second half of 2026[6] Transthyretin (ATTR) Amyloidosis Program: Nexiguran ziclumeran (nex-z) 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][7] ATTR Amyloidosis with Cardiomyopathy (ATTR-CM) 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] - 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] Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN) 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] - 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][10] Platform and Company Updates 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 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] - 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] Upcoming Events 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] Second Quarter 2025 Financial Results The company reported its Q2 2025 financial performance, detailing collaboration revenue, operating expenses, net loss, and cash position Financial Summary 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] - 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] Consolidated Statements of Operations 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 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 |