Intellia Therapeutics NTLA announced the initiation of a phase I/II study to evaluate its investigational CRISPR/Cas9 therapy NTLA-2002 as a single-dose therapy to prevent angioedema attacks in patients with hereditary angioedema (HAE). The study will identify the dosage of NTLA-2002 for use in future studies.
Intellia designed the study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE.
While the phase I portion of the study will identify upto two doses of NTLA-2002, the phase II will evaluate the doses already identified in the phase I portion.
The stock has skyrocketed 110% so far this year against the industry’s 21.6% fall.
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Following initiation of the phase I/II study, NTLA-2002 is the first single-dose CRISPR/Cas9 therapy, designed to prevent attacks in HAE patients, to enter clinical studies.
A rare genetic disorder, HAE 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, which require chronic administration for disease control, HAE attacks still occur. Per management, one in 50,000 people is affected by HAE.
NTLA-2002, which utilizes the CRISPR/Cas9 technology, will be administered systematically via intravenous infusion. The therapy is designed to inactivate the kallikrein B1. This will lead to the reduction of plasma kallikrein activity and in turn, should prevent HAE attacks. A reduction in kallikrein activity is clinically validated in preventing HAE attacks.
NTLA-2002 is Intellia’s second candidate to move to the clinical studies. NTLA’s first candidate to enter into clinical development is NTLA-2001, which is being evaluated in a phase I study for transthyretin amyloidosis with polyneuropathy (ATTRv-PN). Intellia plans to present interim data from the study in first-quarter 2022.
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Zacks Rank & Other Stocks to Consider
Intellia presently carries a Zacks Rank #2 (Buy). Other top-ranked stocks in the biotech/drug sector include Endo International ENDP, IVERIC bio ISEE and Precision BioSciences DTIL. While Endo International currently sports a Zacks Rank #1 (Strong Buy), both IVERIC bio and Precision BioSciences carry a Zacks Rank of 2 at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
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Earnings of Endo International beat estimates in all the last four quarters, the average being 57.7%. Endo International’s stock price closed at $4.51 at the end of the Dec 13 trading session.
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Earnings of IVERIC bio missed estimates in three of the last four quarters and surpassed expectations once, the negative surprise being 5.6%, on average. IVERIC bio’s stock price closed at $15.16 at the end of the Dec 13 trading session.
Precision BioSciences’ loss per share estimates for 2021 have narrowed from $1.17 to $0.65 in the past 60 days. The same for 2022 has narrowed from $2.39 to $1.91 in the past 60 days.
Earnings of Precision BioSciences beat estimates in all the last four quarters, delivering a surprise of 76.9%, on average. Precision BioSciences’ stock price closed at $8.23 at the end of the Dec 13 trading session.
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