185 0 Kommentare Intellia Therapeutics Highlights its Three-Year Strategic Priorities and Anticipated 2024 Key Milestones

Core priorities through 2026 include completion of Phase 3 study and BLA submission for NTLA-2002, completion of patient enrollment in the pivotal study of NTLA-2001, clinical validation of modular in vivo gene insertion technology and advancement of earlier-stage pipeline and platform innovations
On track to dose the first patient in the pivotal Phase 3 MAGNITUDE trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy in Q1 2024
Expect to present results from the Phase 2 study of NTLA-2002 for the treatment of hereditary angioedema (HAE) and to initiate the Phase 3 study in 2024
On track to dose the first patient in the Phase 1 study of NTLA-3001 for the treatment of alpha-1 antitrypsin deficiency (AATD)-associated lung disease in 2024
Ended 2023 in a strong financial position with approximately $1.0 billion in cash; cash runway extended into mid-2026

CAMBRIDGE, Mass., Jan. 04, 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 its strategic priorities through 2026 and key anticipated 2024 milestones that support the company’s mission to transform the lives of patients and bring forth a new era in medicine.

“2023 was a momentous year for Intellia as our lead investigational therapy, NTLA-2001, advanced into the final stage of clinical development for the treatment of transthyretin amyloidosis with cardiomyopathy,” said Intellia President and Chief Executive Officer John Leonard, M.D. “Looking ahead, we will continue to turn groundbreaking science into real-world medicines by capitalizing on our extensive experience and capabilities to edit disease-causing genes. We are rapidly expanding the potential to treat an even larger range of diseases, including those that originate outside of the liver, by deploying our novel delivery and editing technologies. With our comprehensive gene editing toolbox, we are well-positioned to harness the full potential of CRISPR-based medicines for patients.”

Recent Pipeline Advancement and Corporate Updates

NTLA-2001 for Transthyretin (ATTR) Amyloidosis: Initiated and actively enrolling the pivotal Phase 3 MAGNITUDE trial in adults with ATTR amyloidosis with cardiomyopathy (ATTR-CM).
NTLA-2002 for Hereditary Angioedema (HAE): Completed enrollment and dosing in the Phase 2 portion of the Phase 1/2 study in adults with HAE.
NTLA-3001 for Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease: Submitted a clinical trial application to initiate a first-in-human Phase 1 study of NTLA-3001 ahead of previous Q1 2024 expectation.
Organizational Streamlining: Following an internal strategic review, Intellia will be streamlining company-wide operations to further focus resources on key strategic priorities and programs. These changes will result in a pause of select exploratory research-stage programs and a workforce reduction of approximately 15%.
Cash Runway: Intellia ended the fourth quarter of 2023 with approximately $1.0 billion in cash, cash equivalents and marketable securities. The cash position is expected to fund operations into mid-2026.
Board Update: In December, Intellia announced the departure of John Crowley from its board of directors following his appointment as Chief Executive Officer of the Biotechnology Innovation Organization (BIO), the life sciences industry’s leading trade and advocacy association.