Intellia Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to NTLA-2002 for the Treatment of Hereditary Angioedema
CAMBRIDGE, Mass., March 21, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is an in vivo CRISPR-based investigational therapy designed to inactivate the target gene, kallikrein B1 (KLKB1), to potentially prevent life-threatening swelling attacks in people with HAE.
“The RMAT designation is important recognition for our early clinical data. It indicates that a single dose of NTLA-2002 has the potential to address serious unmet medical need for people living with hereditary angioedema,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We look forward to continuing our productive dialogue with the FDA to accelerate the development of NTLA-2002, an investigational in vivo CRISPR-based therapy, with the goal of bringing forth a potentially transformative treatment to patients more quickly.”
The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising therapeutic candidates, including genetic therapies, which are intended to treat, modify, reverse or cure a serious or life-threatening disease. RMAT designation includes benefits, such as early interactions with the FDA, including discussions on surrogate or intermediate endpoints that could potentially support accelerated approval and satisfy post-approval requirements, and potential priority review of a product’s biologics license application (BLA).
The RMAT is the third special regulatory designation received by Intellia for NTLA-2002. NTLA-2002 was also granted Orphan Drug Designation by the FDA and the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency.
About the NTLA-2002 Clinical Program
Intellia’s multi-national Phase 1/2 study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II hereditary angioedema (HAE). This includes the measurement of plasma kallikrein protein and activity, as well as HAE attack rate. The Phase 1 portion of the study is an open-label, single-ascending dose design used to identify two dose levels of NTLA-2002 for further evaluation in the Phase 2, randomized, placebo-controlled portion of the study. This Phase 1/2 study will identify the dose of NTLA-2002 for use in future studies. In 2022, Intellia reported positive interim results from the Phase 1 study demonstrating deep, dose-dependent reductions in plasma kallikrein and robust reductions in patient HAE attacks. Patient screening in the Phase 2 portion of the study is ongoing. Visit clinicaltrials.gov (NCT05120830) for more details.
Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2002 is the first single-dose investigational treatment being explored in clinical trials for the potential to continuously reduce kallikrein activity and prevent attacks in people living with hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational CRISPR therapeutic candidate designed to inactivate the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. NTLA-2002 is Intellia’s second investigational CRISPR therapeutic candidate to be administered systemically, by intravenous infusion, to edit disease-causing genes inside the human body with a single dose of treatment. Intellia’s proprietary non-viral platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which together carry out the precision editing.
About Hereditary Angioedema
Hereditary angioedema (HAE) is a rare, genetic disorder 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, and 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, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics using CRISPR/Cas9 technology. To fully realize the transformative potential of CRISPR/Cas9, Intellia is pursuing two primary approaches. The company’s in vivo programs use intravenously administered CRISPR as the therapy, in which proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. Intellia’s ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of CRISPR/Cas9 to create new classes of genetic medicine. Learn more at intelliatx.com. Follow us on Twitter @intelliatx.
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 accelerate the development of NTLA-2002 pursuant to its Regenerative Medicine Advanced Therapy designation; and the expected timing of regulatory filings and the initiation, enrollment, dosing and completion of clinical trials for NTLA-2002.
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; uncertainties related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates, including NTLA-2002; the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized; and the risk that the results of preclinical studies or clinical studies, such as the clinical study of NTLA-2002, 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 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.
Senior Vice President, Investor Relations and Corporate Communications
Senior Director, Investor Relations and Corporate Communications
Ten Bridge Communications