On February 27, 2025 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, reported operational highlights and financial results for the fourth quarter and year ended December 31, 2024 (Press release, Intellia, FEB 27, 2025, View Source [SID1234650708]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"We are off to an excellent start in 2025 with renewed focus and strong operational execution across our three, pivotal Phase 3 studies," said Intellia President and Chief Executive Officer John Leonard, M.D. "We are excited by the clinical data presented during the fourth quarter. Our Phase 1/2 results in HAE suggest that NTLA-2002 could represent a functional cure for patients with HAE – for the first time a patient has the potential to be both free from attacks and free from chronic therapy. Similarly, the rapid, deep and durable reductions in serum TTR demonstrated to date in our Phase 1 study of nex-z in ATTR represent a highly differentiated profile that may offer patients an opportunity to stabilize or improve their clinical readouts in an otherwise unrelenting, progressive disease."
Fourth Quarter 2024 and Recent Operational Highlights
Hereditary Angioedema (HAE)
NTLA-2002: NTLA-2002 is a wholly owned, investigational in vivo CRISPR-based therapy designed to knock out the KLKB1 gene in the liver, with the goal of lifelong control of HAE attacks after a single dose.
In January, Intellia announced the first patient was dosed with NTLA-2002 in the global Phase 3 HAELO study. The Company expects to complete enrollment in the second half of 2025.
The Company plans to submit a Biologics License Application in the second half of 2026 to support plans for a U.S. launch in 2027.
Intellia expects to present longer-term data from the ongoing Phase 1/2 study in 2025. The data will include patients in the Phase 2 portion who initially received a 25 mg dose or placebo and were subsequently given the 50 mg dose of NTLA-2002 selected for the Phase 3 study.
Transthyretin (ATTR) Amyloidosis
Nexiguran ziclumeran (nex-z, also known as NTLA-2001): Nex-z is an investigational in vivo CRISPR-based therapy designed to inactivate the TTR gene in liver cells, thereby preventing the production of transthyretin (TTR) protein for the treatment of ATTR amyloidosis. Nex-z offers the possibility of halting and reversing the disease by driving a deep, consistent and potentially lifelong reduction in TTR protein after a single dose. Nex-z has been generally well tolerated across all patients and at all dose levels tested. The most common treatment-related adverse event was an infusion reaction, which were mild or moderate; all patients were able to receive the intended dose of nex-z. Intellia leads development and commercialization of nex-z in collaboration with Regeneron.
ATTR Amyloidosis with Cardiomyopathy (ATTR-CM):
Enrollment in the pivotal Phase 3 MAGNITUDE trial is progressing ahead of our target projections and we anticipate enrollment to exceed 550 total patients by year end.
Intellia presented data from the ongoing Phase 1 study at the 2024 American Heart Association (AHA) Scientific Sessions and published the findings online in the New England Journal of Medicine. Across all patients (n=36), a single dose of nex-z led to consistently rapid, deep and sustained serum TTR reduction, regardless of baseline levels, through the latest follow-up. At month 12, the mean serum TTR reduction was 90%, and the mean absolute residual serum TTR concentration was 17 µg/mL. With 11 patients who have reached 24 months of follow-up, all patients continued to show a sustained response with no evidence of a waning effect over time. The consistently low levels of serum TTR are anticipated to reduce the rate of ongoing amyloid formation and potentially allow for amyloid clearance and improvement in cardiac function. Nex-z was generally well tolerated across all patients.
Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN):
We are actively screening patients for the Phase 3 MAGNITUDE-2 study and are on track to dose the first patient in the first quarter of 2025.
Intellia presented data from the ongoing Phase 1 study in November. At month 12, patients who received a dose of 0.3 mg/kg or higher (n=33) had a mean serum TTR reduction of 91% and mean absolute residual serum TTR concentration of 20 µg/mL. For the 16 patients who reached 24 months of follow-up, there was no change to their post-dose TTR levels. It is anticipated that greater TTR reduction may lead to a greater clinical benefit in patients with ATTRv-PN. Nex-z was generally well tolerated across all patients and at all dose levels tested.
In November, Intellia announced the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) to nex-z for the treatment of ATTRv-PN.
Intellia expects to present longer-term data from both ATTR-CM and ATTRv-PN patients in the Phase 1 study in 2025. The data will include updated measures of clinical efficacy and safety.
Platform Update
Intellia continues to apply novel technologies, such as CRISPR-based gene editing technologies and lipid nanoparticle (LNP) delivery technologies, to develop in vivo and ex vivo product candidates. Treating—and potentially curing—a broad range of severe diseases requires the application of multiple technologies. With Intellia’s proprietary technology at the core of the platform, the Company continues to research and develop new gene editing and delivery technologies to expand the therapeutic opportunities, furthering progress on the frontier of genetic medicine.
Corporate Update
On January 9, 2025, the Company announced that, after a strategic review of its business, it elected to prioritize late-stage programs – NTLA-2002 for HAE and nex-z for ATTR amyloidosis – and select research investments to focus on near-term value creation. As a result, the Company discontinued NTLA-3001 and other, undisclosed programs, and is reducing its workforce by approximately 27% in 2025. The Company expects to incur charges of approximately $8.0 million for severance and other employee termination-related costs in the first quarter of 2025.
Upcoming Events
The Company will participate in the following events during the first quarter of 2025:
AAAAI/WAO Joint Congress, March 1, San Diego
TD Cowen 45th Annual Health Care Conference, March 4, Boston
Leerink 2025 Global Biopharma Conference, March 10, Miami
Barclays 27th Annual Global Healthcare Conference, March 11, Miami
Jefferies Biotech on the Beach Summit, March 12, Miami
Fourth Quarter and Full-Year 2024 Financial Results
Cash Position: Cash, cash equivalents and marketable securities were $861.7 million as of December 31, 2024, compared to $1.0 billion as of December 31, 2023. The Company’s cash position as of December 31, 2024, is expected to fund operations into the first half of 2027.
Collaboration Revenue: Collaboration revenue was $12.9 million during the fourth quarter of 2024, compared to negative $1.9 million during the fourth quarter of 2023. The $14.8 million increase was mainly driven by collaboration revenue received under the Regeneron agreements.
R&D Expenses: Research and development (R&D) expenses were $116.9 million during the fourth quarter of 2024, compared to $109.0 million during the fourth quarter of 2023. The $7.9 million increase was primarily driven by the advancement of our lead programs. Stock-based compensation expense included in R&D expenses was $24.4 million for the fourth quarter of 2024.
G&A Expenses: General and administrative (G&A) expenses were $32.4 million during the fourth quarter of 2024, compared to $29.0 million during the fourth quarter of 2023. The $3.4 million increase was primarily related to stock-based compensation. Stock-based compensation expense included in G&A expenses was $15.2 million for the fourth quarter of 2024.
Net Loss: Net loss was $128.9 million for the fourth quarter of 2024, compared to $132.2 million during the fourth quarter of 2023.
Conference Call to Discuss Fourth Quarter and Full-Year 2024 Results
The Company will discuss these results on a conference call today, Thursday, February 27 at 8 a.m. ET.
To join the call:
U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726, approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call.
Please visit this link for a simultaneous live webcast of the call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at intelliatx.com, beginning on February 27 at 12 p.m. ET.