Intellia Therapeutics, Inc. ( NTLA Quick Quote NTLA - Free Report) announced that that European Commission (“EC”) has granted orphan drug designation to its pipeline candidate, NTLA-2002, for the treatment of hereditary angioedema (“HAE”). Shares of the company were up 11.1% on Nov 14 in response to the news.
HAE is a rare genetic disorder characterized by recurring attacks of severe swelling, which can be painful and even life-threatening at times.
The EC grants orphan drug designation in the European Union (“EU”) to a drug or biologic based on the positive opinion rendered by the European Medicines Agency’s (“EMA”) Committee for Orphan Medical Products. The tag is granted to a product candidate intended to treat, prevent or diagnose a rare disease or condition that does not affect more than five in 10,000 people across the EU.
The designation also includes incentives, including financial aid, to develop treatments for rare diseases for which there are no satisfactory treatments available or significant benefits to those affected by the disease.
Shares of Intellia have declined 24.2% so far this year compared with the
industry’s decrease of 24.1%. Image Source: Zacks Investment Research
Last month, the EMA granted Priority Medicines designation to NTLA-2002 for the treatment of HAE.
We remind investors that the FDA has already granted Orphan Drug designation and the Regenerative Medicine Advanced Therapy designation to NTLA-2002 for the treatment of HAE.
NTLA-2002 also received the Innovation Passport from the United Kingdom Medicines and Healthcare products Regulatory Agency.
NTLA-2002 is Intellia’s in vivo CRISPR-based investigational therapy candidate, which is currently being evaluated in a phase I/II study for treating HAE.
Intellia remains focused on completing enrollment in the phase II portion of its early-mid-stage study of NTLA-2002 in HAE in the fourth quarter of 2023.
NTLA is also currently planning to initiate a pivotal phase III study, including U.S. patients, on NTLA-2002 in the HAE indication in the third quarter of 2024, contingent upon regulatory feedback.
Intellia is developing curative therapeutics using the CRISPR/Cas9 technology. The company is evaluating its other in-vivo genome-editing candidate NTLA-2001 for the treatment of transthyretin amyloidosis.
NTLA-2001 is part of the company’s co-development and co-promotion agreement with
Regeneron Pharmaceuticals ( REGN Quick Quote REGN - Free Report) . While NTLA is the lead party in the deal, REGN shares some of the development costs and commercial profits.
Intellia’s collaboration with Regeneron has given a boost to the former to develop its pipeline.
Zacks Rank & Stocks to Consider
Intellia currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the healthcare sector are
Dynavax Technologies Corporation ( DVAX Quick Quote DVAX - Free Report) and Ligand Pharmaceuticals Incorporated ( LGND Quick Quote LGND - Free Report) , sporting a Zacks Rank #1 (Strong Buy) each. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for Dynavax Technologies’ 2023 loss per share have narrowed from 24 cents to 12 cents. Meanwhile, during the same period, earnings per share estimates for 2024 have improved from 2 cents to 18 cents. Year to date, shares of DVAX have rallied 29.7%.
Earnings of Dynavax Technologies beat estimates in two of the last four quarters while missing the same on the remaining two occasions. DVAX delivered a four-quarter average earnings surprise of 293.21%.
In the past 60 days, Ligand Pharmaceuticals’ earnings per share estimates for 2023 have improved from $4.98 to $5.10. During the same period, earnings per share estimates for 2024 have moved up from $4.26 to $4.59. Year to date, shares of LGND have lost 15.5%.
Earnings of Ligand Pharmaceuticals beat estimates in each of the trailing four quarters. On average, LGND came up with a four-quarter earnings surprise of 67.19%.