Intellia Therapeutics Announces Publication of Positive Interim Phase 1 Data for NTLA-2002 in Patients with Hereditary Angioedema in the New England Journal of Medicine
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Insights
The interim results from the Phase 1 study of NTLA-2002, as published in the New England Journal of Medicine, indicate a substantial mean reduction in monthly HAE attack rate post-treatment. This 95% reduction is significant, as HAE attacks can be life-threatening and severely impact quality of life. The fact that 90% of participants remained attack-free during the observation period suggests that NTLA-2002 could be a transformative treatment for HAE patients. This therapy, utilizing CRISPR-based gene editing, represents a leap forward in precision medicine, potentially offering a long-term solution where only symptomatic treatments were available previously.
From a research perspective, the well-tolerated nature of the therapy across all dose levels is equally important. It suggests a favorable safety profile, which is a critical consideration in the development of new treatments, especially those involving novel technologies like CRISPR. The long-term implications for the biotechnology industry could be profound, as success with NTLA-2002 might accelerate the development and acceptance of gene editing therapies for other genetic conditions.
The publication of these results in a prestigious journal and the positive data surrounding NTLA-2002 could have a noteworthy impact on Intellia Therapeutics' market position. Investors may view the potential of a single-dose, curative treatment for HAE—a chronic and debilitating condition—as an opportunity for significant market growth, especially considering the rarity of the disease and the lack of curative treatments. The company's stock could see increased investor interest, reflecting the high unmet medical need and the potential for NTLA-2002 to command premium pricing if approved.
However, it is essential to consider that the therapy is still in the early stages of clinical trials. The transition from Phase 1/2 to later-stage trials, regulatory hurdles and eventual market adoption are crucial milestones that will further influence the financial outlook. Investors should monitor upcoming trial results, FDA feedback and market readiness to integrate such advanced therapies. The long-term financial success will hinge on these factors and the ability of Intellia to scale production and navigate the complex reimbursement landscape for gene therapies.
The release of these clinical trial results is a pivotal moment for the field of gene editing and for the biotech market as a whole. The apparent efficacy and safety of NTLA-2002 could catalyze investment and interest in similar CRISPR-based therapies. For stakeholders, this could mean diversification of portfolios and increased funding for biotech companies specializing in gene editing. The data suggests not only a potential breakthrough in HAE management but also sets a precedent for the gene editing therapeutic approach.
While the clinical and financial potential is clear, the biotech market will be closely observing the scalability of such therapies. Manufacturing, distribution and the ability to meet global demand are non-trivial challenges that Intellia and its peers will need to address. Furthermore, the regulatory landscape for gene therapies is still evolving, with implications for market access and timing. The long-term impact on the biotech sector will depend on how these challenges are navigated and the eventual outcomes of broader clinical trials.
- Data reinforce the potential of NTLA-2002 to eliminate angioedema attacks in people living with hereditary angioedema (HAE) after a single dose
- A single dose of NTLA-2002 led to
95% mean reduction in monthly HAE attack rate with 9 of 10 patients remaining completely attack free following the 16-week primary observation period through the latest follow-up reported - NTLA-2002 was well-tolerated at all dose levels
- Second NEJM publication of initial clinical data for Intellia’s in vivo CRISPR-based investigational therapies
CAMBRIDGE, Mass., Jan. 31, 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 that interim results from the Phase 1 portion of the Phase 1/2 study of NTLA-2002 were published online in the New England Journal of Medicine (NEJM). 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.
“Despite currently available treatments, people living with hereditary angioedema continue to face frequent anxiety about their next swelling attack. The interim NTLA-2002 clinical data published suggest that a single dose of NTLA-2002 may eliminate angioedema attacks for people suffering from hereditary angioedema,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We are highly encouraged by these data and look forward to presenting extended follow-up from the Phase 1 and results from the Phase 2 portion later this year. Additionally, we remain on track to initiate a global pivotal study for NTLA-2002 in the second half of 2024, subject to regulatory feedback. This marks the second consecutive Intellia in vivo CRISPR-based program to have its initial clinical data published in the New England Journal of Medicine, further supporting the immense potential impact our proprietary gene editing platform could have on the future of human health.”
The reported data showed that a single dose of NTLA-2002 led to a
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 (HAE) pursuant to its clinical trial applications and investigational new drug application, including the initiation of a global pivotal study for NTLA-2002 in the second half of 2024 subject to regulatory feedback, the potential for NTLA-2002 to eliminate angioedema attacks after a single dose in people living with HAE, and the expected timing of future data releases such as the presentation of extended follow-up data from the Phase 1 portion and results from the Phase 2 portion of the Phase 1/2 study later this year; and the potential impact its proprietary gene editing platform could have on the future of human health.
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 NTLA-2002 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 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:
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
TBCIntellia@tenbridgecommunications.com
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