FDA Grants Orphan Drug Designation for Intellia’s NTLA-5001; Shares Up

Intellia Therapeutics, Inc. (NASDAQ: NTLA) disclosed that NTLA-5001 has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA).  

Following the news, shares of the biotechnology company, which develops biopharmaceuticals using a CRISPR gene editing system, rose 1.64% in the extended trading session on Wednesday after jumping about 3.7% at the close. 

Facts about NTLA-5001 

NTLA-5001 is Intellia’s ex vivo investigational T cell receptor (TCR)-T cell therapy. It is designed to treat patients with acute myeloid leukemia (AML). Currently, the therapy is undergoing a Phase 1/2a study, which will evaluate the safety and tolerability in adults suffering from persistent or recurrent AML. These patients include individuals who have undergone first-line therapy. 

ODD status is granted by the FDA to medicines that are used to treat and prevent rare diseases affecting less than 200,000 people in the U.S. Specifically, AML, a deadly cancer of the blood and bone marrow without immediate treatment, is the most common type of acute leukemia in adults in the U.S. Sadly, over 20,000 new cases were estimated in 2021. 

Official Comments 

Encouragingly, Intellia CEO John Leonard said, “As part of our full-spectrum genome editing strategy, we seek to leverage our proprietary CRISPR/Cas9-based platform to engineer differentiated cell therapies targeting cancers for which there are currently limited or no treatment options.” 

Wall Street’s Take 

Recently, H.C. Wainwright analyst Michael King maintained a Buy rating on Intellia and a price target of $182. This indicates a 175.8% upside potential from Wednesday’s closing price of $65.99 a piece. 

Shares of Intellia have rallied 11.15% over the past year, while the stock still scores a Strong Buy consensus rating, based on 15 Buys versus two Holds. That’s alongside an average Intellia price target of $148.15, which implies 124.5% upside potential to current levels. 

Risk Analysis  

According to the new TipRanks Risk Factors tool, the Intellia stock is at risk mainly from two factors: Finance & Corporate and Tech & Innovation, which contribute 20 and 19 risks, respectively. In total, 65 risks were identified for the stock. 

Intellia is at a higher risk from a financial and technical standpoint than other companies in its industry. Despite its high-risk profile, based on the positive clinical development and price performance, investors might want to consider adding Intellia to their portfolios.

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