Intellia Up on Upbeat Long-Term Angioedema Study Data

Intellia Therapeutics' NTLA shares gained 10% on Jun 3 after the company reported encouraging long-term data from the phase I portion of its early to mid-stage study evaluating NTLA-2002 for treating hereditary angioedema. Shares of NTLA continued to gain another 1.1% in the after-market hours.

NTLA-2002 is Intellia's investigational single-dose CRISPR/Cas9 gene-editing therapy, which is being developed as a one-time cure for HAE. It is a rare genetic condition that is marked by severe inflammatory attacks in various body organs and tissues, which can be painful and even life-threatening. Despite the current treatment options that require chronic administration for disease control, HAE attacks still occur.

NTLA-2002 aims to prevent HAE attacks by suppressing the plasma kallikrein activity. Per the management, the long-term data presented at a recent medical conference has bolstered the candidate's potential as a groundbreaking treatment for HAE as patients experienced durable elimination of their attacks in the ongoing phase I/II study.

Year to date, shares of Intellia have plunged 22.9% compared with the industry's 6.6% decline.

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A total of 10 patients are being treated in the phase I portion of the study with either of the three one-time doses (25 mg, 50 mg, or 75 mg) of NTLA-2002,administered via intravenous infusion. Eight of these patients experienced no attacks during the initial 16-week observation period. These patients have experienced ongoing attack-free durations of greater than 18 months.

After the last assessment of HAE attacks as of Feb 12, 2024, a 98% mean reduction in monthly attack rate and a 99% mean reduction in moderate to severe attacks were observed after a single dose of NTLA-2002 in the overall patient population. Robust and sustainable reduction in HAE attack rate was observed upon treatment with all dose levels. 

Intellia reported that the longest attack-free interval for an individual patient post-infusion is more than 26 months and ongoing. Treatment with NTLA-2002 has also shown a persistent reduction in HAE attacksin patients with the most severe HAE symptoms.

In the phase I/II study's beginning, two patients had the highest historic monthly HAE attack rates (16.8 and 14.0 attacks per month, respectively). Both patients were attack-free by the end of the 16-week primary observation period and have remained free of attacks through the latest follow-up. 

Furthermore, Intellia reported that the longest attack-free duration among these two patients is 23.5 months and ongoing. All patients who discontinued prophylaxis treatment after NTLA-2002 remain free of chronic prophylaxis treatment.

At the time of the latest follow-up, the mean reduction in plasma kallikrein levels from baseline upon treatment with NTLA-2002 was 60% (25 mg, 88 weeks), 88% (50 mg, 72 weeks) and 95% (75 mg, 88 weeks).

Per Intellia, all three doses of the gene therapy candidate were well-tolerated in the phase I portion of the early to mid-stage study and most adverse events were mild in severity.

The company plans to assess HAE attacks and plasma kallikrein protein levelsthrough the remainder of the two-year follow-up period.

It has completed enrollment in the phase II portion of the study that will further evaluate the 25 mg and 50 mg doses of NTLA-2002 for HAE, expecting to report top-line data soon. Intellia also anticipates initiating a pivotal phase III study of the candidate for the same indication in the second half of 2024, subject to regulatory feedback.

Please note that the company's clinical-stage pipeline comprises another gene-editing candidate, NTLA-2001, which is currently being evaluated in a late-stage study in partnership with Regeneron to treat transthyretin amyloidosis with cardiomyopathy.

Intellia Therapeutics, Inc. Price and Consensus

Intellia Therapeutics, Inc. price-consensus-chart | Intellia Therapeutics, Inc. Quote

Zacks Rank and Stocks to Consider

Intellia currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks from the drug/biotech industry are ALX Oncology Holdings ALXO, Annovis Bio ANVS and Compugen CGEN, each carrying a Zacks Rank #2 (Buy) at present.

In the past 30 days, the Zacks Consensus Estimate for ALX Oncology's 2024 loss per share has narrowed from $3.33 to $2.89. During the same period, the consensus estimate for 2025 loss per share has narrowed from $2.85 to $2.73. Year to date, shares of ALXO have plunged 39.8%.

ALX Oncology beat estimates in two of the trailing four quarters and missed twice, delivering an average negative surprise of 8.83%.

In the past 30 days, the Zacks Consensus Estimate for Annovis' 2024 loss per share has narrowed from $2.93 to $2.46. During the same period, the consensus estimate for 2025 loss per share has narrowed from $2.83 to $1.95. Year to date, shares of ANVS have plunged 63.5%.

ANVS beat estimates in three of the trailing four quarters and missed once, delivering an average negative surprise of 1.39%.

In the past 30 days, the Zacks Consensus Estimate for Compugen's 2024 earnings per share has increased from 2 cents to 5 cents. The consensus estimate for 2025 loss per share is currently pegged at 11 cents. Year to date, shares of CGEN have gained 10.6%.

CGEN's earnings beat estimates in three of the trailing four quarters and missed once, delivering an average surprise of 5.79%.

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