CAMBRIDGE, Mass. - Intellia Therapeutics, Inc. (NASDAQ:NTLA), a gene editing company, has announced positive Phase 2 data for its investigational therapy NTLA-2002, aimed at treating hereditary angioedema (HAE). The results, published in The New England Journal of Medicine, indicate significant reductions in monthly attack rates for HAE patients.
The Phase 2 study, which is part of an ongoing Phase 1/2 trial, demonstrated that a single 50 mg dose of NTLA-2002 led to a mean monthly attack rate reduction of 77% during weeks 1-16 and 81% during weeks 5-16, compared to placebo. Remarkably, eight out of eleven patients in the 50 mg arm were attack-free post-infusion at the latest follow-up, suggesting that NTLA-2002 could potentially serve as a functional cure for HAE.
HAE is a rare genetic condition characterized by severe, recurring, and unpredictable inflammatory attacks, which can be life-threatening. The current standard of care involves chronic administration of treatments that can reduce, but not always eliminate, these attacks. NTLA-2002, an in vivo CRISPR-based gene editing therapy, aims to inactivate the gene responsible for these attacks, offering a one-time treatment solution.
The study enrolled 27 participants who received either a 25 mg or 50 mg single dose of NTLA-2002 or a placebo via intravenous infusion. Those in the 50 mg dose group not only experienced the most significant attack rate reductions but also showed a greater reduction in kallikrein protein, a key player in HAE attacks.
NTLA-2002 was well tolerated across both dose levels, with the most frequent adverse events being mild, such as headache and fatigue. There were no serious adverse events related to the therapy.
These promising results have led Intellia to select the 50 mg dose for further evaluation in the global pivotal Phase 3 HAELO study, which is currently recruiting patients. The company aims to redefine the treatment paradigm for HAE, offering hope for a lifetime free from chronic therapy.
The information in this article is based on a press release statement from Intellia Therapeutics.
In other recent news, Intellia Therapeutics has shown significant progress in its clinical programs. The company successfully completed its Phase II studies for NTLA-2002, a hereditary angioedema (HAE) treatment, and received approval to begin the first-in-human study for NTLA-3001, aimed at treating Alpha-1 Antitrypsin Deficiency (AATD) associated lung disease.
Intellia also reported a strong financial position, with $939.9 million in cash reserves, expected to fund operations into late 2026. BMO Capital and RBC Capital Markets maintained their Outperform ratings on Intellia's stock, with BMO Capital setting a price target of $70.00 and RBC Capital adjusting its price target.
The company has initiated a pivotal Phase 3 trial for NTLA-2002 following promising results from Phase 1/2 trials. A pivotal trial for transthyretin amyloid cardiomyopathy (TTR-CM) has also started across 35 global sites.
These developments reflect Intellia's commitment to advancing its gene editing therapies and its robust financial position to support its ambitious clinical programs.
InvestingPro Insights
As Intellia Therapeutics (NASDAQ:NTLA) makes strides in its gene editing therapy for hereditary angioedema, investors should consider some key financial metrics and insights from InvestingPro.
According to InvestingPro data, Intellia's market capitalization stands at $2.03 billion, reflecting the market's valuation of the company's potential in the gene editing space. However, the company's financial position presents a mixed picture. An InvestingPro Tip notes that Intellia holds more cash than debt on its balance sheet, which could provide financial flexibility as it advances its clinical trials.
Despite the promising clinical results, Intellia faces some financial challenges. The company's revenue for the last twelve months as of Q2 2023 was $45.97 million, with a concerning revenue growth decline of 13.33% over the same period. This aligns with another InvestingPro Tip indicating that the company is quickly burning through cash, a common situation for biotech firms in the development stage.
Investors should also be aware that Intellia is not currently profitable, with a negative gross profit margin of -876.2% in the last twelve months as of Q2 2023. This is consistent with the InvestingPro Tip stating that analysts do not anticipate the company will be profitable this year.
The stock's performance has been volatile, with a year-to-date price total return of -34.6% as of the latest data. This volatility is highlighted in an InvestingPro Tip, which could be particularly relevant for risk-averse investors.
It's worth noting that InvestingPro offers 10 additional tips for Intellia Therapeutics, providing a more comprehensive analysis for investors interested in delving deeper into the company's financial health and market position.
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