Intellia Therapeutics completes enrollment in phase‑3 ATTRv‑PN CRISPR trial
Intellia Therapeutics announced that enrollment is finished for its MAGNITUDE‑2 phase‑3 study of the CRISPR‑based therapy nex‑z in hereditary transthyretin amyloidosis with polyneuropathy (ATTRv‑PN). The double‑blind, placebo‑controlled trial includes about 60 adult patients who receive a single 55 mg infusion or placebo. Primary endpoints are serum TTR change at day 29 and modified neuropathy impairment score at month 18, with optional blinded crossover after month 18. The program is run in partnership with Regeneron and the drug holds orphan and RMAT designations from the FDA and an orphan designation from the European Commission.
Why it matters
The enrollment completion moves the study into its efficacy phase, a key milestone that could bring the therapy closer to regulatory review and market launch, potentially affecting Intellia’s valuation.
Key facts
- 1Intellia completed enrollment in the MAGNITUDE‑2 phase‑3 trial for nex‑z in ATTRv‑PN. investing.com
- 2The trial will involve approximately 60 adult patients. investing.com
- 3Patients are randomized 1:1 to receive a single 55 mg infusion of nex‑z or placebo. investing.com
- 4Primary endpoints are change in modified neuropathy impairment score at month 18 and change in serum TTR levels at day 29. investing.com
- 5Nex‑z has received orphan drug and regenerative medicine advanced therapy (RMAT) designations from the U.S. FDA and an orphan drug designation from the European Commission. investing.com
- 6Intellia leads development and commercialization of nex‑z under a partnership with Regeneron Pharmaceuticals. investing.com
Summary written by AlphAI AI Desk from 2 of 2 sources. Not investment advice. Figures are as stated by the linked sources.