Intellia's Nexiguran Ziclumeran Receives FDA RMAT Designation for ATTR Cardiomyopathy

Intellia Therapeutics, Inc., a prominent name in the sphere of gene editing, has made a significant stride in its mission to transform medical treatment through its CRISPR-based therapies. The company announced that the U.S. Food and Drug Administration (FDA) has awarded Regenerative Medicine Advanced Therapy (RMAT) status to its innovative treatment, nexiguran ziclumeran (commonly known as nex-z or NTLA-2001). This treatment targets transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM), a severe condition affecting the heart.

John Leonard, M.D., the President and CEO of Intellia, expressed the importance of this milestone, highlighting how the RMAT designation is a critical step towards expediting the development of nex-z. The goal is to rapidly deliver this potentially groundbreaking treatment to patients. In clinical settings, nex-z has demonstrated exceptional results, producing rapid, sustained, and consistent reductions in serum transthyretin (TTR) levels after a single dose. Such reductions are crucial as they significantly influence markers that track the progression of the disease.

The RMAT designation, established under the 21st Century Cures Act, is designed to fast-track the development and review of promising therapies aimed at treating, modifying, reversing, or curing serious or life-threatening diseases. By receiving this designation, nex-z benefits from the opportunity of early interactions with the FDA and discussions about surrogate or intermediate endpoints that could facilitate accelerated approval processes. Furthermore, this status opens the door to potential priority review for the product's biologics license application (BLA).

Nex-z has not only received the RMAT designation for cardiomyopathy and polyneuropathy but has also been recognized as an Orphan Drug by both the U.S. FDA and the European Commission. This highlights its promise and underscores the critical unmet needs it aims to address in treating ATTR amyloidosis, a condition currently lacking in effective long-term solutions.

At the heart of nex-z's innovation is the CRISPR/Cas9 technology, renowned for earning a Nobel Prize. This therapy is designed to be a one-time treatment that targets the TTR gene responsible for producing the transthyretin protein. The Phase 1 clinical trials have provided interim data indicating that administering nex-z results in consistent, deep, and prolonged reductions in TTR levels. Intellia is spearheading the development and commercialization of nex-z through a strategic partnership with Regeneron, underscoring a collaborative effort to advance this groundbreaking treatment.

Transthyretin amyloidosis, or ATTR amyloidosis, is a rare but deadly condition caused by mutations in the TTR gene, leading to the production of misfolded proteins by the liver. These proteins can accumulate in the body's tissues, resulting in severe complications, including heart and nerve damage. There are two main forms of the disease: hereditary (ATTRv) and wild-type (ATTRwt). ATTRv is usually genetic, while ATTRwt develops from the instability of non-mutated proteins over time, affecting the heart primarily. Current estimates suggest that ATTRv amyloidosis affects about 50,000 individuals worldwide, while ATTRwt impacts between 200,000 and 500,000 people. Presently, there is no cure for ATTR amyloidosis, and existing treatments can only slow the disease's progression.

Intellia Therapeutics continues to lead the charge in gene editing, focusing on developing pioneering medicines that address significant unmet medical needs. By continuously enhancing its CRISPR-based platform with innovative editing and delivery technologies, Intellia aims to set a new benchmark in the field of gene therapy, offering hope to patients with complex genetic conditions.