Intellia Therapeutics Reports Q3 2024 Financial Results and Recent Progress

Intellia Therapeutics, Inc., a leader in gene editing, has announced significant progress in its CRISPR-based therapy pipeline during the third quarter of 2024. The company, headquartered in Cambridge, Massachusetts, and listed on NASDAQ under NTLA, is pushing forward with various clinical trials and gaining regulatory approvals for its innovative treatments.

Intellia's President and CEO, John Leonard, M.D., highlighted the major advancements, including the start of the HAELO Phase 3 trial, which aims to establish NTLA-2002 as a functional cure for hereditary angioedema (HAE). Leonard emphasized the accelerated enrollment in the ongoing MAGNITUDE Phase 3 trial for nexiguran ziclumeran (nex-z) targeting ATTR amyloidosis with cardiomyopathy. Following the FDA's approval for the MAGNITUDE-2 Phase 3 trial for ATTR amyloidosis with polyneuropathy, Intellia is gearing up to commence this trial within the year.

Nexiguran ziclumeran (nex-z) is an investigative CRISPR-based therapy designed to deactivate the TTR gene in liver cells, curbing the production of transthyretin (TTR) protein, which is implicated in ATTR amyloidosis. This approach aims to provide a long-lasting reduction in TTR protein levels after a single dose. The MAGNITUDE Phase 3 trial for ATTR amyloidosis with cardiomyopathy is progressing well with robust patient enrollment. The MAGNITUDE-2 Phase 3 trial will evaluate nex-z in 50 adults with hereditary ATTR amyloidosis with polyneuropathy, involving a single 55 mg infusion versus placebo. The primary goals are to assess changes in neuropathy impairment scores and serum TTR levels.

Intellia plans to present new data from the Phase 1 study of nex-z at the 2024 American Heart Association (AHA) Scientific Sessions. This presentation will cover safety, serum TTR reduction, and biomarkers related to disease progression and functional capacity in ATTR-CM patients.

In the domain of hereditary angioedema (HAE), NTLA-2002 is being evaluated in the HAELO Phase 3 study. This global, randomized, double-blind, placebo-controlled trial involves 60 adults with Type I or Type II HAE, treated with a single 50 mg infusion of NTLA-2002 or placebo. Positive Phase 2 data show that NTLA-2002 potentially offers a functional cure for HAE, with a majority of patients experiencing no attacks during the primary observation period and beyond, with minimal adverse events.

For alpha-1 antitrypsin deficiency (AATD)-associated lung disease, NTLA-3001 represents Intellia's first CRISPR-mediated gene insertion candidate. This therapy aims to insert the wild-type SERPINA1 gene to restore functional AAT protein levels. Intellia anticipates dosing the first patient in the Phase 1/2 study of NTLA-3001 by the end of the year.

Intellia is broadening its CRISPR-based technology applications beyond liver treatments to target diseases in the bone marrow, brain, muscle, lung, and eye. These efforts aim to significantly expand the scope of CRISPR therapies.

Financially, Intellia ended the third quarter of 2024 with approximately $945 million in cash, cash equivalents, and marketable securities. This funding is expected to support operations into late 2026. The company reported collaboration revenue of $9.1 million for the quarter, with a net loss of $135.7 million, primarily driven by research and development expenses.

Intellia's ongoing research and strategic collaborations, including its partnership with Regeneron, are pivotal in advancing its gene editing programs. The company continues to push the boundaries of CRISPR-based therapies, aiming to transform medical treatment for various genetic disorders.