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NTLA Up as FDA Grants RMAT Status to Genome-Editing Therapy
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Intellia Therapeutics, Inc. (NTLA - Free Report) announced that the FDA has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational in vivo genome-editing candidate, Nexiguran ziclumeran (nex-z, also known as NTLA-2001).
The regulatory body granted the RMAT designation to nex-z for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN).
The RMAT designation provides the candidate with increased opportunities to meet FDA officials, as well as arrange early meetings to discuss potential surrogate or intermediate endpoints. It opens up early interactions between the FDA and sponsors to facilitate accelerated approval and potential priority review of a product’s biologics license application (BLA).
Shares of Intellia were up 3.4% yesterday following the announcement of the news. However, the stock has plunged 51.6% so far this year compared with the industry’s decline of 9%.
Image Source: Zacks Investment Research
The FDA granted the RMAT designation after interim data from a phase I study showed that one-time treatment with nex-z led to rapid, deep and durable TTR reduction, which is likely to halt and potentially reverse the disease.
More Updates on NTLA's Development of Nex-Z
Intellia has collaborated with Regeneron Pharmaceuticals (REGN - Free Report) for the development of nex-z.
Nex-z is part of the company’s co-development and co-promotion agreement with Regeneron. While NTLA is the lead party in the deal for nex-z, REGN shares 25% of the development costs and commercial profits.
Earlier this month, the FDA cleared Intellia’s investigational new drug (IND) application for initiating the pivotal phase III MAGNITUDE-2 study evaluating nex-z for the treatment of ATTRv-PN.
The company plans to begin patient enrollment in the MAGNITUDE-2 study at sites outside the United States by 2024-end.
Besides ATTRv-PN, nex-z is also being developed for the treatment of ATTR amyloidosis with cardiomyopathy (ATTRv-CM).
The phase III MAGNITUDE study is currently evaluating the safety and efficacy of nex-z in patients with ATTRv-CM. Enrollment is currently ongoing in the study.
If the MAGNITUDE study's data are found to be positive, it will enable regulatory filings for the nex-z worldwide. As a result, the successful development of the candidate will be important for Intellia.
NTLA's Zacks Rank & Key Picks
Intellia currently carries a Zacks Rank #3 (Hold).
In the past 60 days, estimates for Spero Therapeutics’ 2024 loss per share have narrowed from $1.59 to $1.13. Loss per share estimates for 2025 have narrowed from $1.54 to 54 cents during the same time. Year to date, shares of SPRO have declined 21.7%.
SPRO’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average surprise being 94.42%.
In the past 60 days, estimates for Castle Biosciences’ 2024 loss per share have narrowed from 58 cents to 8 cents. Loss per share estimates for 2025 have narrowed from $2.13 to $1.88 during the same time. Year to date, shares of CSTL have surged 36.8%.
CSTL’s earnings beat estimates in each of the trailing four quarters, the average surprise being 172.72%.
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NTLA Up as FDA Grants RMAT Status to Genome-Editing Therapy
Intellia Therapeutics, Inc. (NTLA - Free Report) announced that the FDA has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational in vivo genome-editing candidate, Nexiguran ziclumeran (nex-z, also known as NTLA-2001).
The regulatory body granted the RMAT designation to nex-z for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN).
The RMAT designation provides the candidate with increased opportunities to meet FDA officials, as well as arrange early meetings to discuss potential surrogate or intermediate endpoints. It opens up early interactions between the FDA and sponsors to facilitate accelerated approval and potential priority review of a product’s biologics license application (BLA).
Shares of Intellia were up 3.4% yesterday following the announcement of the news. However, the stock has plunged 51.6% so far this year compared with the industry’s decline of 9%.
Image Source: Zacks Investment Research
The FDA granted the RMAT designation after interim data from a phase I study showed that one-time treatment with nex-z led to rapid, deep and durable TTR reduction, which is likely to halt and potentially reverse the disease.
More Updates on NTLA's Development of Nex-Z
Intellia has collaborated with Regeneron Pharmaceuticals (REGN - Free Report) for the development of nex-z.
Nex-z is part of the company’s co-development and co-promotion agreement with Regeneron. While NTLA is the lead party in the deal for nex-z, REGN shares 25% of the development costs and commercial profits.
Earlier this month, the FDA cleared Intellia’s investigational new drug (IND) application for initiating the pivotal phase III MAGNITUDE-2 study evaluating nex-z for the treatment of ATTRv-PN.
The company plans to begin patient enrollment in the MAGNITUDE-2 study at sites outside the United States by 2024-end.
Besides ATTRv-PN, nex-z is also being developed for the treatment of ATTR amyloidosis with cardiomyopathy (ATTRv-CM).
The phase III MAGNITUDE study is currently evaluating the safety and efficacy of nex-z in patients with ATTRv-CM. Enrollment is currently ongoing in the study.
If the MAGNITUDE study's data are found to be positive, it will enable regulatory filings for the nex-z worldwide. As a result, the successful development of the candidate will be important for Intellia.
NTLA's Zacks Rank & Key Picks
Intellia currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks from the biotech sector are Spero Therapeutics, Inc. (SPRO - Free Report) and Castle Biosciences, Inc. (CSTL - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for Spero Therapeutics’ 2024 loss per share have narrowed from $1.59 to $1.13. Loss per share estimates for 2025 have narrowed from $1.54 to 54 cents during the same time. Year to date, shares of SPRO have declined 21.7%.
SPRO’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average surprise being 94.42%.
In the past 60 days, estimates for Castle Biosciences’ 2024 loss per share have narrowed from 58 cents to 8 cents. Loss per share estimates for 2025 have narrowed from $2.13 to $1.88 during the same time. Year to date, shares of CSTL have surged 36.8%.
CSTL’s earnings beat estimates in each of the trailing four quarters, the average surprise being 172.72%.