Intellia pauses trials after serious liver event in gene therapy patient

CAMBRIDGE - Intellia Therapeutics (NASDAQ:NTLA), a $2.75 billion gene therapy company that has seen its stock surge nearly 200% over the past six months, has temporarily halted patient dosing and screening in two Phase 3 clinical trials of its CRISPR-based gene therapy nex-z following a serious adverse event, the company announced Monday. According to InvestingPro data, the company maintains a strong financial position with more cash than debt on its balance sheet.

The decision came after a patient in the MAGNITUDE trial for transthyretin amyloidosis with cardiomyopathy (ATTR-CM) developed Grade 4 liver transaminases and increased total bilirubin after receiving the treatment on September 30. The patient has been hospitalized and is receiving medical care. Despite this setback, InvestingPro analysis shows the company maintains a healthy current ratio of 5.19, indicating strong ability to meet short-term obligations.

"In line with our commitment to patient safety, we have taken immediate action to temporarily pause enrollment in MAGNITUDE and MAGNITUDE-2 as we investigate this recent event," said Intellia President and Chief Executive Officer John Leonard in a statement based on the company’s press release.

The trials were testing nex-z as a potential one-time treatment for ATTR amyloidosis, a progressive condition that affects multiple organs. The MAGNITUDE trial for patients with cardiac manifestations had enrolled over 650 participants, while the MAGNITUDE-2 trial for patients with polyneuropathy had enrolled 47 patients. Approximately 450 patients across both trials had already received the experimental therapy.

Intellia is now consulting with experts and regulatory authorities to develop a strategy to resume the trials. The company is also considering potential risk mitigation measures.

Nex-z is designed to use Nobel Prize-winning CRISPR/Cas9 technology to inactivate the TTR gene responsible for producing the protein that accumulates in ATTR amyloidosis. The therapy has received Orphan Drug and RMAT designations from the FDA.

Intellia is developing nex-z in collaboration with Regeneron Pharmaceuticals as part of a multi-target development partnership.

In other recent news, Intellia Therapeutics announced significant findings from its investigational gene therapy, nexiguran ziclumeran (nex-z), which demonstrated sustained reduction of transthyretin (TTR) protein in patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). The data, presented at an international meeting, showed that patients maintained mean TTR reductions of at least 90% through three years of follow-up. Additionally, Intellia plans to present further long-term data from its Phase 1 trial of nex-z at an upcoming medical conference. In analyst updates, Citizens upgraded Intellia’s stock rating from Market Perform to Market Outperform, citing a strong outlook for the company’s hereditary angioedema (HAE) opportunity. H.C. Wainwright also raised its price target for Intellia to $30.00, maintaining a Buy rating, noting the rapid enrollment of the company’s Phase 3 HAELO trial. Meanwhile, Citizens JMP reiterated a Market Perform rating, highlighting the swift completion of the Phase 3 study enrollment as a positive indicator of interest in Intellia’s gene-editing treatment. These developments reflect ongoing interest and progress in Intellia’s therapeutic pipeline.

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