Intellia gains FDA fast track for gene editing therapy

CAMBRIDGE, Mass. - Intellia Therapeutics, Inc. (NASDAQ:NTLA), a pioneer in CRISPR-based gene editing treatments with a market capitalization of $904 million, has secured a Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration for its investigational therapy, nexiguran ziclumeran (nex-z, also known as NTLA-2001), targeting transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM). According to InvestingPro data, the company maintains a strong liquidity position with a current ratio of 5.77, indicating robust short-term financial stability.

This RMAT designation is a significant regulatory milestone, potentially expediting nex-z’s development and review process. ATTR amyloidosis is a rare and often fatal disease characterized by the accumulation of misfolded transthyretin (TTR) protein in the body’s tissues, leading to nerve damage or heart failure. Nex-z, a one-time treatment using CRISPR/Cas9 technology to inactivate the TTR gene, has shown promising results in Phase 1 clinical trials, demonstrating substantial and sustained TTR reduction. While the company’s stock has faced recent headwinds, trading near its 52-week low of $8.30, InvestingPro analysis suggests the stock may be undervalued, with 8 analysts recently revising their earnings expectations upward.

The RMAT status, part of the 21st Century Cures Act, is granted to therapies with potential to address severe conditions and includes benefits such as early FDA interactions and the possibility of priority review for the product’s biologics license application. Nex-z has also received Orphan Drug Designation in the U.S. and Europe, recognizing its potential to treat a rare condition.

Intellia, in partnership with Regeneron, is leading the development of nex-z and has emphasized its commitment to advancing this therapy to patients swiftly. The company is leveraging its expertise in gene editing to create innovative medicines that could significantly improve patient care.

The information in this article is based on a press release statement from Intellia Therapeutics, Inc.

In other recent news, Intellia Therapeutics reported better-than-expected fourth-quarter results, with adjusted earnings per share of -$1.27, surpassing analyst estimates of -$1.34. Revenue for the quarter reached $12.87 million, significantly exceeding the consensus forecast of $9.28 million, and marking a substantial increase from the previous year’s figure of -$1.9 million. The company ended 2024 with a cash balance of $861.7 million, which is expected to fund operations into the first half of 2027.

In terms of mergers, there were no significant developments reported. Analyst activity has been notable, with Truist Securities reducing its price target for Intellia from $90.00 to $50.00 while maintaining a Buy rating, citing concerns about competition and a tempered commercial outlook for its ATTR amyloidosis treatment. Goldman Sachs downgraded Intellia to Sell with a new price target of $9.00, reflecting concerns over the competitive landscape and commercial viability of its treatments. JPMorgan also adjusted its stance, downgrading the stock to Neutral and lowering the price target to $13.00, expressing caution due to a lack of immediate catalysts. Meanwhile, Citi raised its price target to $14.00 from $12.00, maintaining a Neutral rating, following Intellia’s confirmation of its clinical stage execution plans for the year.

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