Intellia Therapeutics to Present Updated Data from Phase 1/2 Study of NTLA-2002 for the Treatment of Hereditary Angioedema (HAE) at the EAACI Congress 2024
Intellia Therapeutics, Inc. (NASDAQ: NTLA) will present updated data from the Phase 1/2 study of NTLA-2002 for the treatment of Hereditary Angioedema (HAE) at the EAACI Congress 2024. The presentation will cover safety, kallikrein reduction, and attack rate data. The company will also host an investor webcast on June 3, 2024.
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Investigational drugs, particularly those employing CRISPR technology for gene editing, hold significant promise for treating genetic disorders, such as hereditary angioedema (HAE). Intellia Therapeutics' NTLA-2002 is a prime example of how advancements in this field can potentially offer a single-dose treatment option for patients suffering from HAE. This type of therapy could disrupt current treatment paradigms, which often involve repeated administrations of medication.
Understanding the nuances of CRISPR and its implications is important for investors. The CRISPR-based gene editing aims to modify the DNA of a patient's cells to address the underlying genetic causes of a disease. Specifically, NTLA-2002 targets the KLKB1 gene to reduce plasma kallikrein, a protein that contributes to swelling in HAE patients. Reduction in kallikrein levels correlates with fewer attacks, which is a direct measure of efficacy.
It is worth noting for investors that the updated data encompassing safety, kallikrein reduction and attack rate is invaluable. Safety profiles determine the likelihood of a drug receiving regulatory approval and subsequently reaching the market. Long-term efficacy, as evidenced by sustained kallikrein reduction and decreased attack rates, could indicate the potential for improved quality of life for patients, which in turn can affect drug adoption and market penetration.
When evaluating the financial implications of the ongoing studies for Intellia Therapeutics, two key aspects should be considered: the current development phase and the potential market size for HAE treatments. As NTLA-2002 is still in Phase 1/2 of clinical trials, the investment required for its development is substantial. However, given the orphan drug status often granted to treatments for rare diseases like HAE, the pricing and market exclusivity could be favorable if NTLA-2002 is approved.
Market reactions to clinical trial updates can be significant, especially when they concern innovative treatments with large unmet needs. Strong safety and efficacy results could lead to a positive response from the investment community, reflected in the company's stock value. Conversely, any safety concerns might negatively impact investor sentiment.
Additionally, it's important to understand the competitive landscape. If NTLA-2002 shows promise, Intellia Therapeutics could capture a significant share of the HAE treatment market. However, competitors are also working on similar treatments and any delays or negative results could hinder Intellia's market positioning.
- Extended follow-up will include safety, kallikrein reduction and attack rate data, including number of patients who continue to be completely attack free through the latest follow-up
- Intellia to host investor webcast on Monday, June 3, at 8 a.m. ET
CAMBRIDGE, Mass., April 29, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced the acceptance of an oral presentation from the Phase 1 portion of the ongoing NTLA-2002 Phase 1/2 study at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2024, taking place May 31 – June 3 in Valencia, Spain. NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for hereditary angioedema (HAE), a rare genetic condition that leads to potentially life-threatening swelling attacks. The presentation will include updated safety and efficacy results from the Phase 1 study across all three dose cohorts (25 mg, 50 mg and 75 mg).
Presentation Details
Title: CRISPR-based gene editing of KLKB1 resulted in long-term plasma kallikrein protein reduction and decreased attack rate in patients with hereditary angioedema: Updated results from a phase 1 study
Session: Breakthroughs in management of Hereditary Angioedema
Date and Time: Sunday, June 2, 2024, from 8:30 – 10:00 a.m. CET
Presenter: Hilary Longhurst, M.D., Ph.D., FRCP, FRCPath, Senior Medical Officer at Auckland District Health Board and Honorary Associate Professor at University of Auckland, New Zealand, the trial’s principal investigator in New Zealand
Intellia Therapeutics Investor Webcast
Intellia will host a live webcast on Monday, June 3, 2024, at 8:00 a.m. ET to review the new data. To join the webcast, please visit this link, or the Events and Presentations page of the Investors & Media section of the company’s website at www.intelliatx.com. A replay of the webcast will be available on Intellia’s website for at least 30 days following the call.
About the NTLA-2002 Clinical Program
Intellia’s ongoing Phase 1/2 study is evaluating the safety and activity of NTLA-2002 in adults with Type I or Type II hereditary angioedema (HAE). The Phase 1/2 is an international, open-label study designed to identify a dose level of NTLA-2002 for further evaluation in a Phase 3 study. Enrollment of the Phase 1/2 is complete. Intellia plans to initiate the global, pivotal Phase 3 study in the second half of 2024, subject to regulatory feedback. Visit clinicaltrials.gov (NCT05120830) for more details.
About NTLA-2002
Based on Nobel-prize winning CRISPR/Cas9 technology, NTLA-2002 has the potential to become the first one-time treatment for hereditary angioedema (HAE). NTLA-2002 is designed to prevent HAE attacks by inactivating the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. Interim Phase 1 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. NTLA-2002 has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration, 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 by the European Commission.
About Hereditary Angioedema
Hereditary angioedema (HAE) is a rare, genetic disease characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. It is estimated that one in 50,000 people are affected by HAE. Although there is no known cure for HAE, there are preventative and on-demand treatment options to help manage the condition, including long- and short-term prophylaxis used to prevent swelling attacks. Current treatment options often include life-long therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. Kallikrein inhibition is a clinically validated strategy for the preventive treatment of HAE attacks.
About Intellia Therapeutics
Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. The company’s in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. Intellia’s ex vivo programs use CRISPR to engineer human cells outside the body for the treatment of cancer and autoimmune diseases. 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 intelliatx.com 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 regarding: the safety, efficacy, success and advancement of its clinical program for NTLA-2002 for the treatment of hereditary angioedema pursuant to its clinical trial applications and investigational new drug application, including its ability to initiate the global, pivotal Phase 3 study in the second half of 2024, subject to regulatory feedback and the potential of NTLA-2002 to become the first one-time treatment for hereditary angioedema.
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 licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; and uncertainties related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates, including NTLA-2002. 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. 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:
Ian Karp
Senior Vice President, Investor Relations and Corporate Communications
ian.karp@intelliatx.com
Lina Li
Senior Director, Investor Relations and Corporate Communications
lina.li@intelliatx.com
Media:
Matt Crenson
Ten Bridge Communications
media@intelliatx.com
mcrenson@tenbridgecommunications.com
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