Intellia Therapeutics, Inc. NTLA is developing its lead CRISPR-based, in vivo genome-editing candidate, nexiguran ziclumeran (nex-z, formerly known as NTLA-2001) for the treatment of transthyretin (ATTR) amyloidosis.
Intellia has collaborated with Regeneron Pharmaceuticals REGN 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.
Two separate phase III studies are evaluating nex-z for treating transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM) and hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN).
The phase III MAGNITUDE study is evaluating the safety and efficacy of nex-z in patients with ATTR-CM. If the data from the MAGNITUDE study is found to be positive, it will enable global regulatory filings for nex-z.
The phase III MAGNITUDE 2 study is actively screening patients with ATTRv-PN, with the first patient expected to be dosed shortly.
The collaboration with Regeneron for nex-z is a boost for Intellia as it provides the latter with resources to support the development of the candidate.
Intellia is developing its wholly owned CRISPR-based pipeline candidate, NTLA-2002, for the treatment of hereditary angioedema (“HAE”).
The pivotal phase III HAELO study is actively enrolling patients evaluating NTLA-2002 for treating HAE. Intellia dosed the first patient in the HAELO study in January 2025. Enrollment in the same is likely to be completed in the second half of 2025.
The company plans to submit a potential biologics license application for NTLA-2002 in HAE in the second half of 2026.
In January 2025, the company announced a strategic reorganization to prioritize its portfolio of late-stage pipeline candidates.
As part of the portfolio reorganization, NTLA is planning to prioritize the development of nex-z and NTLA-2002. The company will also reduce its workforce by 27% in 2025.
Intellia is gearing up to transition from a late-stage development company to a commercial-ready organization by the end of 2026.
The encouraging pipeline progress with nex-z and NTLA-2002 should maintain the momentum for Intellia in 2025.
Intellia’s pipeline candidates are still in early-to-mid-stage development and some years away from commercialization. Upon successful development and potential approval, the candidates are likely to face stiff competition from companies that are also using the CRISPR/Cas9 gene editing technology to address various diseases in specific areas.
The FDA approval of Vertex Pharmaceuticals VRTX and CRISPR Therapeutics’ CRSP CRISPR/Cas9 genome-edited cell therapy, Casgevy, for the treatment of sickle cell disease has put the limelight on the gene editing space.
The approval for Vertex and CRSP’s Casgevy was a breakthrough for medical science, as it was the first for a CRISPR-based gene-editing therapy in the world.
VRTX leads the global development and commercialization of Casgevy under the terms of the 2021 agreement, with support from CRISPR Therapeutics.
Meanwhile, owing to the latest portfolio reorganization, Intellia has decided to stop the development of its in vivo gene insertion candidate, NTLA-3001, for the treatment of alpha-1 antitrypsin deficiency-associated lung disease.
In the absence of a marketed product, the successful development of its pipeline candidates remains a key focus area for Intellia. Failure in the ongoing studies will be a big setback for the company.
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