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
Intellia Therapeutics, Inc. (NASDAQ:NTLA) announced positive interim results from the Phase 1 portion of the Phase 1/2 study of NTLA-2002, an investigational in vivo CRISPR-based gene editing therapy for hereditary angioedema (HAE). The data revealed a 95% mean reduction in monthly HAE attack rate after a single dose of NTLA-2002, with 9 out of 10 patients remaining completely attack-free during the 16-week primary observation period. The results were published in the New England Journal of Medicine, reinforcing the potential of NTLA-2002 to eliminate angioedema attacks in people living with HAE. This marks a significant milestone in the development of revolutionary CRISPR-based therapies for rare genetic conditions.
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.
01/31/2024 - 05:00 PM
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 95% mean reduction in monthly HAE attack rate across all 10 patients in the Phase 1 portion. Nine out of 10 patients remained completely attack-free following the 16-week primary observation period through the latest follow-up. Further, all patients who discontinued concomitant long-term HAE prophylaxis treatment after NTLA-2002 administration (n=6) have reported no HAE attacks since discontinuation. NTLA-2002 has been well tolerated at all dose levels. The most frequent adverse events reported were mild, transient infusion-related reactions and fatigue. The data were previously shared in a late-breaking presentation at the 2023 European Academy of Allergy and Clinical Immunology Hybrid Congress.
About the NTLA-2002 Clinical Program clinicaltrials.gov (NCT05120830) for more details.
About NTLA-2002 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
About Intellia Therapeutics 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
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@intelliatx.com
Lina Lilina.li@intelliatx.com
Media: media@intelliatx.com TBCIntellia@tenbridgecommunications.com
What is the ticker symbol for Intellia Therapeutics, Inc.?
The ticker symbol for Intellia Therapeutics, Inc. is NTLA.
What is NTLA-2002?
NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for hereditary angioedema (HAE).
What were the results of the Phase 1 portion of the Phase 1/2 study of NTLA-2002?
The Phase 1 portion of the study revealed a 95% mean reduction in monthly HAE attack rate after a single dose of NTLA-2002, with 9 out of 10 patients remaining completely attack-free during the 16-week primary observation period.
Where were the interim results of the Phase 1 portion of the Phase 1/2 study of NTLA-2002 published?
The interim results were published online in the New England Journal of Medicine (NEJM).
What is hereditary angioedema (HAE)?
Hereditary angioedema (HAE) is a rare genetic condition that leads to potentially life-threatening swelling attacks.
