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Intellia Therapeutics, Inc. ($NTLA) shares plunged hard on Thursday after the small-cap, clinical-stage biotech reported results from mid-stage study results of its CRISPR-based gene-editing therapy codenamed NTLA-2002 in patients with hereditary angioedema (HAE).
HAE is a rare genetic disorder that manifests as severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body. The condition could lead to potentially life-threatening swelling attacks.
The disorder has no curative therapies but current preventive therapies have to be administered lifelong.
Intellia said the results from the Phase 2 study showed NTLA-2002 has the potential to eliminate HAE attacks following a one-time infusion.
Among the findings from the study are:
“We are highly encouraged by these results, which we believe sets NTLA-2002 apart from other prophylaxis treatments. What was previously an unimaginable potential to be free of chronic therapy is one step closer to becoming a reality for the HAE community,” said John Leonard, CEO of Intellia.
The company said it has selected the 50 mg formulation for evaluation in the global Phase 3 study dubbed ‘HALEO.’ Screening for the study is currently ongoing, it said.
HAE therapeutics market was at $4.1 billion in 2023 and is estimated to grow at 9.2% compounded annual growth rate between 2024 and 2032, according to Global Market Insights.
The negative reaction could be due to the “buy-the-rumor, sell-the-news” phenomenon as the stock tacked on about 17% gains from a near-term (Oct. 10) low of $17.09.
On Stocktwits, retail trader sentiment was ‘extremely bearish’ (21/100), reversing from ‘bullish’ mood a day ago, with message volume spiking to ‘extremely high.’
At 11:05 am ET, the stock was down 19.51% at $16.05, although off the day’s low of $15.52, which marked the lowest level in over four years.
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