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Intellia Readying for Commercial Activities, First Product Launch in 2027

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Intellia Therapeutics headquarters in Cambridge, Massachusetts

NEW YORK – Intellia Therapeutics expects to commercialize its first three therapeutic indications by 2030, the company's CEO said Monday at the JP Morgan Healthcare Conference in San Francisco.

Cambridge, Massachusetts-based Intellia, which was founded a decade ago by CRISPR pioneer Jennifer Doudna and others, doesn't yet have any marketed products. In Q3 2024, the most recent quarter for which it has reported financial results, the company posted $9.1 million in revenue from collaborations with other companies and a net loss of $135.7 million.

At the conference, Intellia President and CEO John Leonard shared the company's plans to commercialize its first products by the end of the decade. "We're at the point of the final lap before we move to commercialization, with three Phase III programs that are actively enrolling," Leonard said. "The actions that we took last week allow us to focus on the clinical progression here."

Intellia last week announced a strategic reorganization, including discontinuing development of an early-stage gene-editing candidate and laying off 27 percent of its workforce, which it said would set the foundation for "near-term value creation" and allow it to focus resources on advancing its late-stage candidates. The company in early 2024 had laid off 15 percent of its employees and cut certain research-stage programs as part of a plan to streamline operations.

Leonard said at the conference that Intellia is planning to submit a biologics license application (BLA) next year to the US Food and Drug Administration, seeking approval for a gene-editing treatment for hereditary angioedema (HAE). This could make the therapy, referred to as NTLA-2002, the first in vivo CRISPR gene-editing product to reach the market.

Unlike the first CRISPR gene-editing treatment to win FDA approval, Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy (exagamglogene autotemcel), which requires editing cells outside of the body, or ex vivo, Intellia's products don't require apheresis or myeloablative conditioning. As an in vivo therapy, the typical administration involves a single outpatient infusion, Leonard said.

Patients with HAE, a rare genetic disease, experience unpredictable and painful swelling attacks. NTLA-2002 aims to prevent and, ultimately, eliminate these attacks by stopping expression of the KLKB1 gene, which plays a critical role in the body's inflammatory response.

"We expect NTLA-2002 to be the first launch for the company starting in 2027, and that will be followed by two launches through 2030," Leonard said, adding that Intellia has sufficient cash to fund operations through the launch of NTLA-2002 in the first half of 2027. "We think our peak spend years are behind us, as we go forward to a point of commercialization."

The company's other late-stage investigational therapeutic, nexiguran ziclumeran, abbreviated as nex-z and previously known as NTLA-2001, is being developed in two indications related to transthyretin (ATTR) amyloidosis, a rare and severe progressive disease caused by abnormal and misfolded TTR proteins.

Intellia is developing nex-z as a treatment for ATTR amyloidosis with polyneuropathy (ATTR-PN), a hereditary disease that's caused by mutations in the TTR gene, and ATTR amyloidosis with cardiomyopathy (ATTR-CM), the more common type of the disease that can be either hereditary or associated with aging, in which case patients have the wild-type TTR gene.

Nex-z is designed to treat ATTR-PN and ATTR-CM by inactivating the TTR gene that encodes the eponymous protein, and clinical trial data so far suggest it may be able to not only halt disease progression but even reverse it, Leonard said. The gene-editing candidate was developed as part of a multitarget discovery, development, and commercialization collaboration with Regeneron.

Intellia expects to submit a BLA seeking approval for nex-z for treating ATTR-PN in 2028. In ATTR-CN, the company anticipates completing enrollment in a Phase III trial testing nex-z by early 2027 and filing a BLA by 2030.

In preparation for these activities, Intellia is planning to treat the first HAE patient with NTLA-2002 within the randomized, controlled pivotal Phase III HAELO trial early this year, as well as the first ATTR-PN patient with nex-z within the randomized, controlled Phase III MAGNITUDE-2 trial. The company launched the Phase III MAGNITUDE trial for patients with ATTR-CM last year.

If these clinical trials prove successful, NTLA-2002 could represent a notable revenue opportunity for the company beginning in 2027, and nex-z could provide such an opportunity beginning in 2029, according to Intellia. The company has said it is planning to have all of its commercial capabilities in place by the end of next year.

"As we stand back and look at the year behind us and the two years ahead, you see us on the final lap of approaching what we've wanted from the very beginning, which is to form a fully integrated, commercial-stage company," Leonard said.