- Robust reductions in plasma kallikrein ranges and HAE assault charges noticed in any respect doses examined
- All sufferers handled within the 25 mg and 75 mg cohorts have an ongoing attack-free interval via newest
follow-up - First three sufferers handled have an ongoing attack-free interval of 5.5 – 10.6 months after a single dose of NTLA-2002
- NTLA-2002 was usually well-tolerated in any respect dose ranges
- Intellia to host investor occasion to debate up to date information from Phase 1/2 research of NTLA-2002, its second systemically administered in vivo CRISPR candidate, on Monday, November 14, at 8:00 a.m. ET
CAMBRIDGE, Mass., Nov. 12, 2022 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a number one clinical-stage genome enhancing firm targeted on creating probably healing therapeutics leveraging CRISPR-based applied sciences, in the present day introduced up to date information from an ongoing Phase 1/2 scientific research of NTLA-2002 for the therapy of hereditary angioedema (HAE). The interim analyses had been shared in the present day in a Distinguished Industry Abstract oral presentation on the American College of Allergy, Asthma & Immunology (ACAAI) 2022 Annual Scientific Meeting, being held November 10 – 14 in Louisville, Kentucky.
The information offered are from 10 grownup sufferers with HAE within the Phase 1, dose-escalation portion of the research, with an information closing date of September 28, 2022. Single doses of 25 mg (n=3), 50 mg (n=4) and 75 mg (n=3) of NTLA-2002 had been administered by way of intravenous infusion, and modifications from baseline values of plasma kallikrein protein had been measured for every affected person.
Plasma Kallikrein Reduction
Administration of NTLA-2002 led to deep, dose-dependent reductions in plasma kallikrein as described beneath, primarily based on full cohort biomarker information availability. For the 25 mg and 75 mg cohorts, these deep reductions in plasma kallikrein had been sustained via the commentary interval, which ranged from week 16 to week 32.
| Cohort | Mean plasma kallikrein discount at newest follow-up |
| 25 mg (n=3) | 64% (week 32) |
| 50 mg (n=4) | 81% (day 22) |
| 75 mg (n=3) | 92% (week 16) |
HAE Attack Rate Reduction
HAE assault charges are measured within the dose-escalation portion of the research, with the primary evaluation occurring on the finish of the pre-specified 16-week major commentary interval. To date, all sufferers within the 25 mg and 75 mg dose cohorts have reached the tip of this preliminary commentary interval in ongoing follow-up as described beneath. Patients within the 50 mg cohort haven’t accomplished the first 16-week commentary interval.
| Cohort | Baseline assault price in screening interval | Mean HAE assault price discount – week 1 to 16 | Mean HAE assault price discount – week 5 to 16 | Duration of ongoing attack-free interval |
| 25 mg (n=3) | 1.1 to 7.2 assaults / month | 91% | 89% | 5.5 – 10.6 months |
| 75 mg (n=3) | 4.0 to five.9 assaults / month | 78% | 89% | 2.3 – 4.2 months |
“We see early evidence that our one-time CRISPR-based investigational therapy may offer patients suffering from hereditary angioedema a functional cure for their disease,” stated Intellia President and Chief Executive Officer John Leonard, M.D. “Based on the extended data across multiple dose cohorts, we are strongly encouraged that all patients who received a single dose of NTLA-2002 subsequently became attack-free. In the patients with the longest follow-up to date, their attack-free interval has been maintained 5 to 10 months from their last attack. Importantly, the safety data from all 10 patients are highly encouraging, further supporting NTLA-2002’s potential to change the future HAE treatment paradigm. As the second clinical program from our in vivo pipeline to demonstrate deep and consistent protein reduction following a one-time administration, the latest interim data further reinforce the enormous potential of our modular CRISPR genome editing platform to treat a host of genetic diseases.”
At all three dose ranges, NTLA-2002 was usually well-tolerated, and the vast majority of hostile occasions had been delicate in severity. The most frequent hostile occasions had been infusion-related reactions, which had been largely Grade 1 and resolved inside in the future. There have been no dose-limiting toxicities, no critical hostile occasions and no hostile occasions of Grade 3 or greater noticed thus far. No clinically important laboratory abnormalities had been noticed.
Intellia expects to pick as much as two doses to additional consider NTLA-2002 within the Phase 2, placebo-controlled, dose-expansion portion of the research, which is predicted to start within the first half of 2023. Intellia anticipates increasing nation and web site participation, together with U.S. scientific websites, as a part of the Phase 2 research.
Intellia Therapeutics Investor Event and Webcast Information
Intellia will host a reside webcast, Monday, November 14, 2022, at 8:00 a.m. ET, to evaluation the interim outcomes from NTLA-2002. To be part of the webcast, please go to this hyperlink, or the Events and Presentations web page of the Investors & Media part of the corporate’s web site at www.intelliatx.com. A replay of the webcast shall be out there on Intellia’s web site for at the least 30 days following the decision.
About the NTLA-2002 Clinical Program
Intellia’s multi-national Phase 1/2 research is evaluating the security, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II hereditary angioedema (HAE). This contains the measurement of plasma kallikrein protein ranges and exercise as decided by HAE assault price measures. The Phase 1 portion of the research is an open-label, single-ascending dose design used to determine as much as two dose ranges of NTLA-2002 that shall be additional evaluated within the randomized, placebo-controlled Phase 2 portion of the research. This Phase 1/2 research will determine the dose of NTLA-2002 to be used in future research. Visit clinicaltrials.gov (NCT05120830) for extra particulars.
About NTLA-2002
Based on Nobel Prize-winning CRISPR/Cas9 know-how, NTLA-2002 is the primary single-dose investigational therapy being explored in scientific trials for the potential to repeatedly scale back kallikrein exercise and stop assaults in individuals residing with hereditary angioedema (HAE). NTLA-2002 is an entirely 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 contained in the human physique with a single dose of therapy. Intellia’s proprietary non-viral platform deploys lipid nanoparticles to ship to the liver a two-part genome enhancing system: information RNA particular to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which collectively perform the precision enhancing.
About Hereditary Angioedema
Hereditary angioedema (HAE) is a uncommon, genetic dysfunction characterised by extreme, recurring and unpredictable inflammatory assaults in numerous organs and tissues of the physique, which will be painful, debilitating and life-threatening. It is estimated that one in 50,000 persons are affected by HAE, and present therapy choices usually embody life-long therapies, which can require continual intravenous (IV) or subcutaneous (SC) administration as usually as twice per week, or each day oral administration to make sure fixed pathway suppression for illness management. Despite continual administration, breakthrough assaults nonetheless happen. Kallikrein inhibition is a clinically validated technique for the preventive therapy of HAE assaults.
About Intellia Therapeutics
Intellia Therapeutics, a number one clinical-stage genome enhancing firm, is creating novel, probably healing therapeutics leveraging CRISPR-based applied sciences. To totally notice the transformative potential of CRISPR-based applied sciences, Intellia is pursuing two major approaches. The firm’s in vivo packages use intravenously administered CRISPR because the remedy, through which proprietary supply know-how permits extremely exact enhancing of disease-causing genes straight inside particular goal tissues. Intellia’s ex vivo packages use CRISPR to create the remedy through the use of engineered human cells to deal with most cancers and autoimmune illnesses. Intellia’s deep scientific, technical and scientific growth expertise, together with its sturdy mental property portfolio, have enabled the corporate to take a management function in harnessing the total potential of genome enhancing to create new lessons of genetic medication. Learn extra at intelliatx.com. Follow us on Twitter @intelliatx.
Forward-Looking Statements
This press launch accommodates “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) inside the that means of the Private Securities Litigation Reform Act of 1995. These forward-looking statements embody, however will not be restricted to, categorical or implied statements concerning Intellia’s beliefs and expectations concerning: its means to conduct and full scientific research for NTLA-2002 for the therapy of hereditary angioedema (“HAE”); its means to generate information to reveal NTLA-2002 as a possible single-dose therapy for HAE, together with security, kallikrein discount and assault price information; its perception that NTLA-2002 could supply sufferers struggling from HAE a useful treatment for his or her illness; its expectation to start the Phase 2 dose-expansion of the research within the first half of 2023, and its expectation to increase nation and web site participation, together with U.S. scientific websites, as a part of the Phase 2 research; its means to develop its modular CRISPR genome enhancing platform to deal with a bunch of genetic illnesses; the development and growth of its CRISPR/Cas9 know-how to develop human therapeutic merchandise; its means to keep up and increase its associated mental property portfolio, and keep away from or purchase rights to legitimate mental property of third events; its means to reveal its platform’s modularity and replicate or apply outcomes achieved in preclinical and scientific research, together with these in its NTLA-2002 program, in any future research, together with human scientific trials evaluating remedies for different genetic illnesses; its means to develop different in vivo or ex vivo cell therapeutics of every type, and NTLA-2002 particularly, utilizing CRISPR/Cas9 know-how; and the timing of regulatory filings and scientific trial execution, together with enrollment and dosing of sufferers.
Any forward-looking statements on this press launch are primarily based on administration’s present expectations and beliefs of future occasions, and are topic to numerous dangers and uncertainties that might trigger precise outcomes to vary materially and adversely from these set forth in or implied by such forward-looking statements. These dangers and uncertainties embody, however will not be restricted to: dangers associated to the profitable enrollment of sufferers within the Phase 1/2 research for NTLA-2002 for the therapy of HAE; dangers associated to Intellia’s means to guard and keep its mental property place; dangers associated to the authorization, initiation and conduct of research and different growth necessities, together with manufacturing, for its in vivo and ex vivo product candidates, together with NTLA-2002; the danger that anybody or extra of Intellia’s product candidates, together with NTLA-2002, won’t be efficiently developed and commercialized; the danger that the outcomes of preclinical research or scientific research, together with for NTLA-2002, won’t be predictive of future leads to reference to future research; and the danger that Intellia won’t be able to reveal its platform’s modularity and replicate or apply outcomes achieved in preclinical research to develop extra product candidates, together with to use its proprietary CRISPR/Cas9 know-how platform efficiently to extra product candidates to deal with different genetic illnesses. For a dialogue of those and different dangers and uncertainties, and different necessary elements, any of which may trigger Intellia’s precise outcomes to vary from these contained within the forward-looking statements, see the part entitled “Risk Factors” in Intellia’s most up-to-date annual report on Form 10-Okay and quarterly report on Form 10-Q filed with the U.S. Securities and Exchange Commission (“SEC”), in addition to discussions of potential dangers, uncertainties and different necessary elements in Intellia’s different filings with the SEC. All data on this press launch is as of the date of the discharge, and Intellia undertakes no obligation to replace this data until required by regulation.
Intellia Contacts:
Investors:
Ian Karp
Senior Vice President, Investor Relations and Corporate Communications
+1-857-449-4175
[email protected]
Lina Li
Senior Director, Investor Relations and Corporate Communications
+1-857-706-1612
[email protected]
Media:
Rebecca Spalding
Ten Bridge Communications
+1-646-509-3831
[email protected]
[email protected]
