Intellia Therapeutics Announces Third Quarter 2024 Financial Results and Highlights Recent Company Progress
NTLA 11.07.2024
Drug:NTLA-2001 nexiguran ziclumeran
Drug:NTLA-2002 NTLA-2002
Drug:NTLA-3001 NTLA-3001
Diseases:hereditary transthyretin (ATTR) amyloidosis
Diseases:hereditary angioedema (HAE)
Diseases:alpha-1 antitrypsin deficiency (AATD)
Date of Upcoming Event:2024-11-16
Name of Upcoming Event:2024 American Heart Association (AHA) Scientific Sessions
About Gravity Analytica
Recent News
- 01.22.2025 - Intellia Therapeutics Announces First Patient Dosed in the HAELO Phase 3 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema
- 01.13.2025 - J.P. Morgan Healthcare Conference
- 01.09.2025 - Intellia Therapeutics Announces Anticipated 2025 Milestones and Strategic Reorganization to Prioritize the Advancement of its Late-Stage Programs, NTLA-2002 and Nexiguran Ziclumeran (nex-z)
Recent Filings
- 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-end - Strong 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 Sessions
- Actively screening patients in the HAELO Phase 3 study of NTLA-2002 for hereditary angioedema (HAE)
- Reported positive results from the Phase 2 study supporting NTLA-2002’s potential to be a functional cure for HAE
- On track to dose the first patient by year-end in the Phase 1/2 study of NTLA-3001, anin vivogene insertion candidate for the treatment of alpha-1 antitrypsin deficiency (AATD)
- Ended the third quarter of 2024 with approximately
$945 million in cash, cash equivalents and marketable securities
“Our third quarter and recent period have been marked by outstanding progress in advancing our pipeline of CRISPR-based gene editing therapies,” said Intellia President and Chief Executive Officer
Third Quarter 2024 and Recent Operational Highlights
Transthyretin (ATTR) Amyloidosis
- Nexiguran ziclumeran (nex-z, also known as NTLA-2001): Nex-z is an investigationalin vivoCRISPR-based therapy designed to inactivate theTTRgene 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. 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 swiftly and continuing to track ahead of the Company’s target enrollment projections.
- Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN):
- Intellia announced today that the
U.S. Food and Drug Administration (FDA) has cleared its nex-z Investigational New Drug (IND) application to initiate the MAGNITUDE-2 pivotal Phase 3 trial for ATTRv-PN. MAGNITUDE-2 is an international, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of nex-z in 50 adults with ATTRv-PN. Patients will be randomized 1:1 to receive a single 55 mg infusion of nex-z or placebo. Patients randomized to the placebo arm will be eligible for optional crossover to receive nex-z. The primary endpoints are the change from baseline in modified Neuropathy Impairment Score +7 (mNIS+7) at month 18 and serum TTR at day 29. The mNIS+7 scale is a validated measure specifically designed to assess and quantify polyneuropathy impairment, including muscle weakness, muscle stretch reflexes, sensory loss and autonomic impairment. The Company expects to initiate patient enrollment in the MAGNITUDE-2 study at ex-U.S. sites by year-end.
- Intellia announced today that the
- Upcoming Clinical Data Update:
- Intellia will bepresentingnew data from the ongoing Phase 1 study in a late-breaking oral presentation at the 2024
American Heart Association (AHA) Scientific Sessions onSaturday, November 16 . The presentation will include safety, reduction in serum TTR and biomarkers of disease progression and functional capacity data in patients with ATTR-CM. The Company will host an investor webcast to review these data along with data from the ATTRv-PN arm onSaturday, November 16 .
- Intellia will bepresentingnew data from the ongoing Phase 1 study in a late-breaking oral presentation at the 2024
- ATTR Amyloidosis with Cardiomyopathy (ATTR-CM):
Hereditary Angioedema (HAE)
- NTLA-2002:NTLA-2002 is a wholly owned, investigationalin vivoCRISPR-based therapy designed to knock out theKLKB1gene in the liver, with the goal of lifelong control of HAE attacks after a single dose.
- Intellia is actively screening patients in the HAELO Phase 3 study. In October, Intelliaannouncedthe initiation of HAELO, a global, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of NTLA-2002 in 60 adults with Type I or Type II HAE. Patients will be randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo. Patients randomized to the placebo arm will be eligible for optional crossover to NTLA-2002 at week 28. The primary endpoint is the change in number of HAE attacks from week 5 through week 28.
- In October, the Companypresentedpositive Phase 2 data from the ongoing Phase 1/2 study, with results continuing to support the potential of NTLA-2002 to be a functional cure for HAE. Eight of 11 patients in the 50 mg arm ceased having any attacks during the 16-week primary observation period after a single dose of NTLA-2002. These eight patients continued to be attack-free through the reported follow-up and no further treatment has been required. NTLA-2002 was well tolerated. The most frequent adverse events (AEs) were headache, fatigue and nasopharyngitis. There have been no serious AEs, and all AEs were either Grade 1 or 2. These interim data werepublishedin the
New England Journal of Medicine and presented at the 2024American College of Allergy, Asthma & Immunology (ACAAI) Scientific Meeting inBoston, Massachusetts .
Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease
- NTLA-3001:NTLA-3001 is a first-in-class CRISPR-mediatedin vivotargeted gene insertion development candidate for the treatment of AATD-associated lung disease. It is designed to precisely insert the wild-typeSERPINA1gene, which encodes the alpha-1 antitrypsin (AAT) protein, with the potential to restore permanent expression of fully functional AAT protein to normal levels after a single dose. This is Intellia’s first wholly owned gene insertion program.
- Intellia expects to dose the first patient in the Phase 1/2 study of NTLA-3001 by year-end.
In VivoPlatform Expansion
- Intellia is expanding the range of diseases that can be targeted with its CRISPR-based technologies by deploying new editing and delivery innovations. This includes advancing gene editing programs in five different tissues outside the liver, either independently or in collaboration with partners. These research and preclinical programs are targeting diseases that originate in the bone marrow, brain, muscle, lung and eye, which, if successful, could dramatically expand the opportunities for CRISPR-based treatments.
Ex VivoProgram Updates
- Intellia is advancing multiple programs, wholly owned and in collaboration with partners, utilizing its allogeneic platform for the treatment of immuno-oncology and autoimmune diseases. The Company’s proprietary allogeneic cell engineering platform avoids both T cell- and NK cell-mediated rejection in preclinical models, a key unsolved challenge with other investigational allogeneic approaches. Cell therapies engineered with Intellia’s allogeneic platform, combined with edits to enhance cell function, offer a new approach to target both hematological and solid tumors.
Upcoming Events
The Company will participate in the following events during the fourth quarter of 2024:
- 2024
American Heart Association (AHA) Scientific Sessions,November 16 – 18,Chicago
Third Quarter 2024 Financial Results
- Cash Position:Cash, cash equivalents and marketable securities were
$944.7 million as ofSeptember 30, 2024 , compared to$1.0 billion as ofDecember 31, 2023 . The decrease was driven by cash used to fund operations of$335.0 million . The Company’s investments were offset in part by$176.9 million of net equity proceeds from the Company’s “At the Market” (ATM) program,$47.0 million of reimbursement from collaborators, including a one-time$30.0 million payment received inApril 2024 related to the Company’s technology collaboration with Regeneron,$37.2 million of interest income and$6.5 million in proceeds from employee-based stock plans. The cash position is expected to fund operations into late 2026. - Collaboration Revenue:Collaboration revenue was
$9.1 million during the third quarter of 2024, compared to$12.0 million during the third quarter of 2023. The$2.9 million decrease was mainly driven by a reduction in revenue related to the AvenCell license and collaboration agreement. - R&D Expenses:Research and development (R&D) expenses were
$123.4 million during the third quarter of 2024, compared to$113.7 million during the third quarter of 2023. The$9.7 million increase was primarily driven by the advancement of our lead programs. Stock-based compensation expense included in R&D expenses was$24.2 million for the third quarter of 2024. - G&A Expenses:General and administrative (G&A) expenses were
$30.5 million during the third quarter of 2024, compared to$29.4 million during the third quarter of 2023. The$1.1 million increase was primarily related to stock-based compensation. Stock-based compensation expense included in G&A expenses was$15.4 million for the third quarter of 2024. - Net Loss:Net loss was
$135.7 million for the third quarter of 2024, compared to$122.2 million during the third quarter of 2023.
Conference Call to Discuss Third Quarter 2024 Results
The Company will discuss these results on a conference call today,
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 theIntellia Therapeutics conference call.- Please visit thislinkfor 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 atintelliatx.com, beginning on
About
Forward-Looking StatementsThis press release contains “forward-looking statements” of
Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its contract manufacturers, collaborators, licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of preclinical and clinical studies and other development requirements for its product candidates, including uncertainties related to regulatory approvals to conduct clinical trials; risks related to the ability to develop and commercialize any one or more of Intellia’s product candidates successfully; risks related to the results of preclinical studies or clinical studies not being predictive of future results in connection with future studies; the risk that clinical study results will not be positive; risks related to the potential delay of planned clinical trials due to regulatory feedback or other developments; and risks related to Intellia’s collaborations with Regeneron, or its other collaborations not continuing or not being successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the
| CONSOLIDATED STATEMENTS OF OPERATIONS (UNAUDITED) | |||||||||||||||||||||
| (Amounts in thousands, except per share data) | |||||||||||||||||||||
| Three Months Ended | Nine Months Ended | ||||||||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||||||||
| Collaboration revenue | $ | 9,111 | $ | 11,992 | $ | 45,003 | $ | 38,192 | |||||||||||||
| Operating expenses: | |||||||||||||||||||||
| Research and development | 123,380 | 113,696 | 349,434 | 326,088 | |||||||||||||||||
| General and administrative | 30,501 | 29,403 | 93,385 | 87,503 | |||||||||||||||||
| Total operating expenses | 153,881 | 143,099 | 442,819 | 413,591 | |||||||||||||||||
| Operating loss | (144,770 | ) | (131,107 | ) | (397,816 | ) | (375,399 | ) | |||||||||||||
| Other income (expense), net: | |||||||||||||||||||||
| Interest income | 12,122 | 12,740 | 37,176 | 37,373 | |||||||||||||||||
| Change in fair value of investments, net | (3,064 | ) | - | (29,483 | ) | - | |||||||||||||||
| Loss from equity method investment | - | (3,857 | ) | - | (10,905 | ) | |||||||||||||||
| Change in fair value of contingent consideration | - | - | - | (100 | ) | ||||||||||||||||
| Total other income, net | 9,058 | 8,883 | 7,693 | 26,368 | |||||||||||||||||
| Net loss | $ | (135,712 | ) | $ | (122,224 | ) | $ | (390,123 | ) | $ | (349,031 | ) | |||||||||
| Net loss per share, basic and diluted | $ | (1.34 | ) | $ | (1.38 | ) | $ | (3.99 | ) | $ | (3.96 | ) | |||||||||
| Weighted average shares outstanding, basic and diluted | 101,002 | 88,645 | 97,842 | 88,204 | |||||||||||||||||
| CONSOLIDATED BALANCE SHEET DATA (UNAUDITED) | |||||||||||
| (Amounts in thousands) | |||||||||||
| Cash, cash equivalents and marketable securities | $ | 944,681 | $ | 1,012,087 | |||||||
| Total assets | 1,173,351 | 1,300,977 | |||||||||
| Total liabilities | 210,736 | 250,808 | |||||||||
| Total stockholders’ equity | 962,615 | 1,050,169 | |||||||||
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Source: Intellia Therapeutics, Inc.