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Business Upturn
a day ago
- Business
- Business Upturn
Intellia Therapeutics Announces Positive Three-Year Data from Phase 1 Trial of Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary Angioedema (HAE) at the European Academy of Allergy and Clinical Immunology Congress
With up to three years of follow-up, a single dose of lonvo-z led to a 98% mean reduction in monthly HAE attack rate in all 10 patients All 10 patients were attack-free and treatment-free for a median of 23 months through the latest follow-up, demonstrating the potential of lonvo-z to become the first one-time therapy for most HAE patients Lonvo-z was well tolerated and continues to demonstrate a favorable safety profile The global Phase 3 HAELO trial of lonvo-z has concluded screening ahead of schedule with more than half screened from U.S. sites; Intellia to provide an update on enrollment in the future CAMBRIDGE, Mass., June 15, 2025 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced three-year follow-up data from the Phase 1 portion of the ongoing Phase 1/2 study in patients with HAE after receiving a single dose of lonvoguran ziclumeran (lonvo-z, also known as NTLA-2002). Results were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, held June 13-16 in Glasgow, United Kingdom. 'Today's results underscore the promising potential of Intellia's approach to gene editing therapy – a one-time treatment that was well tolerated and offered a highly differentiated, durable effect for patients suffering from a serious disease,' said Intellia President and Chief Executive Officer John Leonard, M.D. 'Seeing all 10 patients in the Phase 1 portion of this study free from both HAE attacks and chronic therapy at nearly two years of median follow-up is incredibly encouraging. These data fuel our optimism for the outcomes of our ongoing Phase 3 HAELO study, which we expect to report in the first half of 2026, and highlight the strong value we believe it will offer patients, physicians and payers.' 'People living with HAE often report a reduced quality of life because they worry about the likelihood of their next attack, either because they still experience attacks or are reminded of it by their use of chronic therapy,' said Dr. Joshua Jacobs, Medical Director, Allergy and Asthma Clinical Research, Inc. 'Based on the data, it is reasonable to expect lonvo-z could offer patients the potential to be free from both physical HAE attacks and the burden of managing chronic HAE treatment.' In the Phase 1 portion of the study, a one-time dose of 25 mg (N=3), 50 mg (N=4) or 75 mg (N=3) of lonvo-z was administered via intravenous infusion and plasma kallikrein protein levels were measured along with HAE attacks. At the time of the February 12 data cutoff, patients were attack-free and treatment-free for a median of nearly two years. With up to three years of follow-up, a single dose of lonvo-z led to a mean reduction in monthly HAE attack rate of 98% over the study period, compared to pre-treatment baseline. For all 10 patients, deep, dose-dependent and durable reductions in plasma kallikrein protein continued to be observed through the latest assessment. Safety Across all three dose levels, lonvo-z has been well tolerated and continues to demonstrate a favorable safety profile consistent with earlier data presented at EAACI in 2024. The most frequent adverse events during the study period were infusion-related reactions (IRRs). IRRs were mostly Grade 1 and resolved with all patients receiving the full dose. With up to 3 years of follow-up, no treatment-emergent serious adverse events were observed, and no treatment-related adverse events were observed during the period following 28 days after dosing. Clinical Development Plans Intellia's global Phase 3, randomized, double-blind, placebo-controlled HAELO trial is ongoing to assess the safety and efficacy of lonvo-z at the 50 mg dosage. The Company announced today the HAELO trial has successfully completed screening ahead of schedule, with over half of the patients being screened in the United States. The study is no longer recruiting and Intellia will provide an update on enrollment in the future. New and longer-term data from the Phase 2 portion of the ongoing Phase 1/2 study is planned to be presented in the second half of 2025. Intellia expects to submit a biologics license application (BLA) in 2026 to support the Company's plans for a U.S. launch in 2027. For more information on HAELO (NCT06634420), please visit About the Lonvoguran Ziclumeran (lonvo-z, also known as NTLA-2002) Clinical Program Intellia's ongoing Phase 1/2 study is evaluating the safety and efficacy of lonvo-z in adults with Type I or Type II hereditary angioedema (HAE). The Phase 1 portion of the study is an international, open-label study designed to identify the dose level of lonvo-z selected for further evaluation in the Phase 2 portion of the study. Enrollment in both portions of the Phase 1/2 study is complete. Intellia dosed the first patient in the global Phase 3, randomized, double-blind, placebo-controlled HAELO trial in January of 2025. Visit (NCT05120830) for more details. About Lonvo-z Based on Nobel Prize-winning CRISPR/Cas9 technology, lonvo-z has the potential to become the first one-time treatment for hereditary angioedema (HAE). Lonvo-z is an investigational in vivo CRISPR-based gene editing therapy designed to prevent HAE attacks by inactivating the kallikrein B1 ( KLKB1 ) gene, which encodes for prekallikrein, the kallikrein precursor protein. Interim Phase 1/2 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. Lonvo-z has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration (FDA), the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation (ODD) by the European Commission. About Intellia Therapeutics Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. Since its inception, Intellia has focused on leveraging gene editing technology to develop novel, first-in-class medicines that address important unmet medical needs and advance the treatment paradigm for patients. Intellia's deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. Learn more at and follow us @intelliatx. Forward-Looking Statements This press release contains 'forward-looking statements' of Intellia Therapeutics, Inc. ('Intellia' or the 'Company') within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia's beliefs and expectations concerning: the safety, efficacy, success and advancement of its clinical programs for lonvoguran ziclumeran or 'lonvo-z' (also known as NTLA-2002) for hereditary angioedema ('HAE'), including the ability to successfully complete its global Phase 3 HAELO study; its expectation to present additional data regarding lonvo-z, including reporting outcomes of the Phase 3 HAELO study in the first half of 2026 and presenting new and longer-term data from the Phase 2 portion of the ongoing Phase 1/2 study of lonvo-z in the second half of 2025; and its expectation to be able to support a biologics license application for lonvo-z for the treatment of HAE by 2026 for a U.S. launch in 2027. 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, licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to valid third party intellectual property; risks related to Intellia's relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to regulatory agencies' evaluation of regulatory filings and other information related to our product candidates, including lonvo-z; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for our product candidates, including uncertainties related to regulatory approvals to conduct clinical trials, including our ability to complete the Phase 3 HAELO study for HAE; the risk that any one or more of Intellia's product candidates, including lonvo-z, will not be successfully developed and commercialized; and the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies for the same product candidate or Intellia's other product candidates. 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 of Form 10-K and quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in Intellia's other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law. Intellia Contacts: Investors:Brittany ChavesSenior Manager, Investor Relations [email protected]
Yahoo
08-05-2025
- Business
- Yahoo
Intellia Therapeutics Announces First Quarter 2025 Financial Results and Highlights Recent Company Progress
On track to complete enrollment of the global Phase 3 HAELO study in hereditary angioedema (HAE) in the third quarter of 2025 Dosed first patient in the global Phase 3 MAGNITUDE-2 study evaluating nexiguran ziclumeran (nex-z) in patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) Enrollment in the global Phase 3 MAGNITUDE trial of nex-z in patients with ATTR with cardiomyopathy (ATTR-CM) continues to track ahead of projections Presenting additional data from the Phase 1 portion of the ongoing Phase 1/2 study of NTLA-2002 in patients with HAE at EAACI Congress in June 2025 Expect to present longer-term data from both ATTR-CM and ATTRv-PN patients in the Phase 1 study of nex-z in the second half of 2025 Ended the first quarter with approximately $707.1 million in cash, cash equivalents and marketable securities; Expected to fund operations into the first half of 2027 CAMBRIDGE, Mass., May 08, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today reported operational highlights and financial results for the first quarter ended March 31, 2025. 'Intellia is full steam ahead and making excellent progress across its clinical programs,' said Intellia President and Chief Executive Officer John Leonard, M.D. 'Two key achievements in the first quarter were dosing the first patients in two of our Phase 3 studies: the HAELO study for hereditary angioedema and the MAGNITUDE-2 study for hereditary ATTR amyloidosis with polyneuropathy. Additionally, our Phase 3 MAGNITUDE study for ATTR with cardiomyopathy continues to enroll rapidly. Upcoming catalysts include longer-term data from the ongoing Phase 1 study of NTLA-2002 at the upcoming EAACI Congress in addition to updated data from the Phase 2 study of NTLA-2002 and longer-term Phase 1 data of nex-z in the second half of 2025.' First Quarter 2025 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. Intellia will present additional data from the ongoing Phase 1/2 study in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025 on Sunday, June 15 in Glasgow, United Kingdom. The presentation will include longer-term durability data from patients in the Phase 1 portion of the Phase 1/2 study. Enrollment is progressing in the global Phase 3 HAELO study and the Company expects to complete enrollment in the third quarter of 2025. Intellia expects to present new and longer-term data from the Phase 2 portion of the ongoing Phase 1/2 study in the second half of 2025. The data will include patients 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. The Company is on track to submit a Biologics License Application (BLA) in the second half of 2026. 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 events have been mild or moderate infusion reactions; all patients were able to receive the intended dose of nex-z. Intellia leads development and commercialization of nex-z in collaboration with Regeneron Pharmaceuticals, Inc. ATTR Amyloidosis with Cardiomyopathy (ATTR-CM): In March, Intellia announced the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) to nex-z for the treatment of ATTR-CM. Enrollment in the global Phase 3 MAGNITUDE trial is progressing ahead of the Company's target projections and anticipates enrollment to exceed 550 total patients by year-end. Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN): In April, the Company announced the first patient was randomized and dosed with nex-z in the global Phase 3 MAGNITUDE-2 study. Intellia expects enrollment to be completed in 2026. In May, the Company will present data at the European Society of Cardiology Heart Failure (ESC-HF) Congress and Peripheral Nerve Society (PNS) Annual Meeting. At ESC-HF, Intellia will show wildtype vs. variant data in patients with ATTR-CM. At PNS, the Company will present interim Phase 1 extended data in patients with ATTRv-PN. Intellia expects to present longer-term data from both ATTR-CM and ATTRv-PN patients in the Phase 1 study in the second half of 2025. The data will include updated measures of clinical efficacy and safety. Platform Update Intellia is pioneering novel technologies, such as CRISPR-based gene editing technologies and lipid nanoparticle (LNP) delivery technologies, to create highly differentiated, future in vivo and ex vivo product candidates. The Company's proprietary platform technologies are being researched and developed to expand therapeutics opportunities to support the mission of transforming lives of people with severe diseases, including the possibility of curative genome editing therapeutics. Upcoming Events The Company will participate in the following events during the second quarter of 2025: Bank of America Securities Health Care Conference, May 13, Las Vegas ESC Heart Failure Congress, May 17, Belgrade PNS Annual Meeting, May 18, Edinburgh RBC Capital Markets Global Healthcare Conference, May 21, New York EAACI Congress, June 15, Glasgow First Quarter 2025 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $707.1 million as of March 31, 2025, compared to $861.7 million as of December 31, 2024. The decrease in cash, cash equivalents and marketable securities was primarily driven by first quarter operations and approximately $51 million of non-recurring cash payments associated with the Company's previously announced portfolio prioritization, workforce reduction, and real estate consolidation. The Company's cash, cash equivalents and marketable securities as of March 31, 2025 are expected to fund operations into the first half of 2027. Collaboration Revenue: Collaboration revenue was $16.6 million during the first quarter of 2025, compared to $28.9 million during the first quarter of 2024. The $12.3 million decrease was mainly driven by a decrease in collaboration revenue under the AvenCell license and collaboration agreement. R&D Expenses: Research and development (R&D) expenses were $108.4 million during the first quarter of 2025, compared to $111.8 million during the first quarter of 2024. The $3.4 million decrease was primarily driven by employee-related expenses, stock-based compensation, research materials and contracted services offset by an increase in the advancement of our lead programs. Stock-based compensation expense included in R&D expenses was $12.6 million for the first quarter of 2025. G&A Expenses: General and administrative (G&A) expenses were $29.0 million during the first quarter of 2025, compared to $31.1 million during the first quarter of 2024. The $2.1 million decrease was primarily related to lower employee-related expenses due to a workforce reduction in January 2025 and lower stock-based compensation, partially offset by increases related to severance expenses recorded in the first quarter. Stock-based compensation expense included in G&A expenses was $9.2 million for the first quarter of 2025. Net Loss: Net loss was $114.3 million for the first quarter of 2025, compared to $107.4 million during the first quarter of 2024. Conference Call to Discuss First Quarter 2025 Results The Company will discuss these results on a conference call today, Thursday, May 8 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 beginning on May 8 at 12 p.m. ET. About Intellia TherapeuticsIntellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. Since its inception, Intellia has focused on leveraging gene editing technology to develop novel, first-in-class medicines that address important unmet medical needs and advance the treatment paradigm for patients. Intellia's deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. Learn more at and follow us @intelliatx. Forward-Looking Statements This press release contains 'forward-looking statements' of Intellia Therapeutics, Inc. ('Intellia' or the 'Company') within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia's beliefs and expectations concerning: the safety, efficacy, success and advancement of its clinical programs for NTLA-2001, also known as nexiguran ziclumeran or 'nex-z', for transthyretin ('ATTR') amyloidosis and NTLA-2002 for the treatment of hereditary angioedema ('HAE') pursuant to its clinical trial applications ('CTA') and investigational new drug application ('IND') submissions, including the expected timing of data releases from its ongoing clinical trials of nex-z and NTLA-2002, regulatory feedback, regulatory filings, and the initiation, enrollment, dosing and completion of clinical trials, such as the completion of enrollment of the Phase 3 HAELO study in the third quarter of 2025 and the submission of a biologics license application ('BLA') in the second half of 2026, its ability to enroll the Phase 3 MAGNITUDE study and exceed 550 total enrolled patients by the end of 2025, its ability to enroll the Phase 3 MAGNITUDE-2 study and complete enrollment in 2026, its plans to present new data from the Phase 2 portion of the Phase 1/2 study of NTLA-2002 and longer-term Phase 1 data of nex-z, including updated measures of clinical efficacy and safety, in the second half of 2025, the potential of nex-z to halt and reverse disease by driving a deep, consistent and potentially lifelong reduction in TTR protein after a single dose, and the potential of NTLA-2002 to provide lifelong control of HAE attacks after a single dose; its ability to apply novel technologies, such as CRISPR-based gene editing technologies and lipid nanoparticle delivery technologies, to develop in vivo and ex vivo product candidates, including its ability to use those technologies to expand therapeutic opportunities and the timing expectations of advancing such product candidates; its ability to optimize the impact of its collaborations on its development programs, including, but not limited to, its collaboration with Regeneron Pharmaceuticals, Inc. ('Regeneron') and their co-development programs for ATTR amyloidosis; and its growth as a company and expectations regarding its uses of capital, expenses, future accumulated deficit and financial results, including its ability to fund operations into the first half of 2027. 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 Securities and Exchange Commission, including its quarterly report on Form 10-Q. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law. INTELLIA THERAPEUTICS, INC. CONSOLIDATED STATEMENTS OF OPERATIONS (UNAUDITED) (Amounts in thousands, except per share data) Three Months ended March 31, 2025 2024 Collaboration revenue $ 16,627 $ 28,935 Operating expenses: Research and development 108,427 111,847 General and administrative 29,007 31,091 Total operating expenses 137,434 142,938 Operating loss (120,807 ) (114,003 ) Other income (expense), net: Interest income 8,603 12,632 Change in fair value of investments, net (2,125 ) (6,065 ) Total other income, net 6,478 6,567 Net loss $ (114,329 ) $ (107,436 ) Net loss per share, basic and diluted $ (1.10 ) $ (1.12 ) Weighted average shares outstanding, basic and diluted 103,500 95,502 INTELLIA THERAPEUTICS, INC. CONSOLIDATED BALANCE SHEET DATA (UNAUDITED) (Amounts in thousands) March 31,2025 December 31,2024 Cash, cash equivalents and marketable securities $ 707,100 $ 861,730 Total assets 986,163 1,191,015 Total liabilities 206,244 319,059 Total stockholders' equity 779,919 871,956 Investors:Brittany ChavesSenior Manager, Investor Media:Matt CrensonTen Bridge Communicationsmedia@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
