HAE gene-editing therapy NTLA-2002 receives RMAT designation

Status paves way for early talks with the FDA about experimental treatment

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to NTLA-2002, an experimental gene-editing therapy that Intellia Therapeutics is developing to treat hereditary angioedema (HAE).

RMAT designation is granted to therapies with the potential to treat, reverse, or cure serious or life-threatening diseases to accelerate development and regulatory review. It provides a series of benefits, including early talks with the FDA about trial design, and potential for priority review of an eventual application seeking approval.

“The RMAT designation is important recognition for our early clinical data,” John Leonard, MD, president and CEO of Intellia, said in a press release. “It indicates that a single dose of NTLA-2002 has the potential to address serious unmet medical need for people living with hereditary angioedema.”

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NTLA-2002 is designed to disrupt the activity of the KLKB1 gene in liver cells, where the gene is most active. The KLKB1 gene provides instructions for making a precursor of the protein kallikrein, which is responsible for controlling the production of the signaling molecule bradykinin.

In HAE, swelling attacks are triggered by the excessive production of bradykinin. By interfering with the KLKB1 gene, NTLA-2002 aims to reduce levels of kallikrein, thereby lowering bradykinin levels and ultimately preventing swelling attacks.

Intellia is conducting a Phase 1/2 clinical trial (NCT05120830) to test NTLA-2002 in people with HAE. Early data from the first 10 participants in the open label Phase 1 portion of the trial indicated the therapy was generally well tolerated and capable of reducing swelling attack rates.

Intellia recently began screening for participants in the Phase 2 part of the study, in which participants will receive a single infusion of one of two doses of NTLA-2002, or a placebo. The main goal is to assess the effect of treatment on swelling attack rates at 16 weeks following treatment.

NTLA-2002 received orphan drug designation from the FDA in late 2022, and earlier this year it was awarded the innovation passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA).

“We look forward to continuing our productive dialogue with the FDA to accelerate the development of NTLA-2002 … with the goal of bringing forth a potentially transformative treatment to patients more quickly,” Leonard said.