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CAMBRIDGE, Mass. - Intellia Therapeutics, Inc. (NASDAQ:NTLA), a $1.85 billion market cap biotech company, reported today that its investigational gene therapy nexiguran ziclumeran (nex-z) demonstrated sustained reduction of transthyretin (TTR) protein in patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). The company’s stock has shown remarkable momentum, gaining over 100% in the past six months, according to InvestingPro data.
According to data presented at the 5th International ATTR Amyloidosis Annual Meeting in Baveno, Italy, patients who received a single dose of nex-z maintained mean TTR reductions of at least 90% through three years of follow-up. While the clinical results are promising, InvestingPro analysis reveals the company maintains a strong financial position with more cash than debt, though it’s currently burning through cash reserves as it advances its pipeline.
The Phase 1 study results showed that among patients receiving doses of 0.3 mg/kg or higher, mean serum TTR reduction was 92% at 24 months. For the 12 patients who reached the 36-month mark, mean reduction was 90%.
Clinical measures also showed positive trends, with 72% of evaluated patients demonstrating clinically meaningful improvements in modified Neuropathy Impairment Score +7 (mNIS+7) at 24 months. The company reported that 89% of patients showed improvement or stability in polyneuropathy disability scores compared to baseline.
The treatment has been generally well tolerated across all dose levels, with infusion-related reactions being the most common treatment-related adverse events, according to the company’s statement.
With 11 analysts recently revising their earnings expectations upward and price targets ranging from $7 to $106 per share, market sentiment appears optimistic about Intellia’s prospects. "The results from our ongoing Phase 1 study of nex-z support our belief that deeper and more consistent reductions in TTR translate to better outcomes for patients," said Intellia President and Chief Executive Officer John Leonard in the press release.
Intellia began dosing patients in its Phase 3 MAGNITUDE-2 trial in April 2025 and expects to complete enrollment in the first half of 2026. The company anticipates submitting a biologics license application for ATTRv-PN by 2028. For investors seeking deeper insights into Intellia’s financial health and growth prospects, InvestingPro offers comprehensive analysis through its Pro Research Report, available as part of its coverage of over 1,400 US stocks.
ATTR amyloidosis is a rare, progressive disease caused by the buildup of misfolded TTR protein in multiple tissues. The company estimates there are approximately 50,000 people worldwide living with ATTRv amyloidosis.
The data was simultaneously published in the New England Journal of Medicine.
In other recent news, Intellia Therapeutics has made significant strides in its clinical trials and received notable attention from analysts. The company announced the completion of patient enrollment in its global Phase 3 HAELO study for the treatment of hereditary angioedema (HAE) using its investigational CRISPR-based gene-editing therapy, lonvoguran ziclumeran. This milestone was achieved within nine months, with nearly half of the participants from the United States. Intellia plans to report topline data from this study in the first half of 2026, with a U.S. launch anticipated in the first half of 2027.
In addition, H.C. Wainwright has raised its price target for Intellia Therapeutics from $25 to $30, maintaining a Buy rating, citing the swift enrollment of the Phase 3 trial as a positive indicator of interest. Meanwhile, Citizens JMP has reiterated a Market Perform rating, emphasizing the rapid enrollment as a promising sign for the company’s gene-editing treatment. Furthermore, Intellia is set to present longer-term data from its Phase 1 trial for hereditary ATTR amyloidosis at an upcoming medical conference. These developments underscore the company’s ongoing progress in advancing its gene-editing therapies.
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