Intellia begins phase 3 trial for gene-editing therapy

CAMBRIDGE, Mass. - Intellia Therapeutics, Inc. (NASDAQ:NTLA), a pioneer in CRISPR-based gene editing treatments, has announced the initiation of a Phase 3 trial for its investigational therapy, nexiguran ziclumeran (nex-z), aimed at treating hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). The first patient has recently been dosed in the global, pivotal MAGNITUDE-2 study. According to InvestingPro data, the company maintains a strong financial position with more cash than debt and a healthy current ratio of 5.77x, though it’s currently experiencing rapid cash burn as it advances its clinical programs.

ATTRv-PN is a rare and progressive disease characterized by the buildup of abnormal amyloid proteins, leading to nerve damage and other serious complications. Nex-z, which utilizes the Nobel Prize-winning CRISPR/Cas9 technology, is designed to inactivate the gene responsible for the production of the mutated transthyretin (TTR) protein, which is the underlying cause of the disease. With a market capitalization of approximately $756 million and trading near its 52-week low, InvestingPro analysis suggests the stock is currently undervalued, despite recent market volatility.

The MAGNITUDE-2 study is a randomized, double-blind, placebo-controlled trial that will assess the efficacy and safety of a single 55 mg infusion of nex-z in approximately 50 patients. The primary endpoint includes a modified neuropathy impairment score and changes in serum TTR levels.

Dr. Paulo Sgobbi, Medical Director at PSEG Clinical Research Center, expressed optimism about the potential of nex-z to significantly alter the disease progression and improve the quality of life for patients, who currently rely on chronic treatment regimens.

Intellia’s President and CEO, John Leonard, M.D., highlighted the encouraging Phase 1 data, which showed rapid, deep, and durable reduction in serum TTR after a single dose of nex-z. The company anticipates presenting longer-term data from the Phase 1 studies later this year and aims to submit a biologics licensing application for ATTRv-PN by 2028.

Nex-z has received Regenerative Medicine Advanced Therapy designations from the U.S. FDA for both cardiomyopathy and polyneuropathy, as well as Orphan Drug Designation from the U.S. FDA and European Commission. Intellia is leading the development and commercialization of nex-z in collaboration with Regeneron Pharmaceuticals, Inc.

ATTR amyloidosis affects an estimated 50,000 individuals globally with the hereditary form and between 200,000 and 500,000 with the wild-type form. Current treatments focus on slowing the accumulation of misfolded TTR protein, but there is no known cure for the disease.

This news is based on a press release statement from Intellia Therapeutics, Inc.

In other recent news, Intellia Therapeutics has made significant strides with the U.S. Food and Drug Administration granting a Regenerative Medicine Advanced Therapy designation for its investigational gene-editing therapy, nexiguran ziclumeran, aimed at treating transthyretin amyloidosis with cardiomyopathy. This designation could accelerate the therapy’s development and review process. However, analysts have expressed mixed sentiments about the company’s prospects. Truist Securities maintained a Buy rating on Intellia but reduced its price target from $90 to $50, citing competitive pressures. Goldman Sachs took a more cautious stance, downgrading Intellia from Neutral to Sell and cutting the price target to $9, pointing to concerns over the competitive landscape and efficacy differentiation. Similarly, JPMorgan downgraded the stock from Overweight to Neutral, reducing the price target to $13 and noting the lack of immediate catalysts. These developments come amid a broader shakeup in the biotech sector following the resignation of FDA’s top vaccine official, Peter Marks, which has raised concerns about potential changes in regulatory approaches.

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