Intellia Therapeutics (NASDAQ: NTLA) saw shares rise after the FDA lifted a clinical hold on one of its Phase 3 trials for nexiguran ziclumeran (nex-z), a CRISPR-based therapy for transthyretin amyloidosis (ATTR).
What Happened
Last October, U.S. regulators paused two trials after a participant died from severe liver toxicity. The cleared trial, MAGNITUDE-2, tests nex-z in patients with the nerve-related form of ATTR. Intellia plans to resume the study with tighter monitoring of liver health and enroll roughly 10 more patients.
The second trial, MAGNITUDE, which focuses on the heart-related form of ATTR, remains on hold. Since this form affects older patients and represents a larger market, the FDA may require more extensive safety measures before allowing it to continue.
Why Investors Should Care
- Positive regulatory signal: Restarting MAGNITUDE-2 shows Intellia can work with the FDA to address safety concerns.
- Market potential: The nerve-related trial is smaller, but a successful restart helps maintain investor confidence in Intellia’s CRISPR platform.
- Ongoing risks: The cardiomyopathy trial represents the bigger opportunity. Any delays or additional safety requirements could weigh on the stock.
Other Developments
Intellia is also waiting on Phase 3 results for lonvo-z, a therapy for hereditary angioedema. The company has positioned this as a potential “functional cure,” with results expected in the first half of 2026. Success there could provide another growth driver for the stock.
Investor Takeaway
Intellia’s FDA clearance is a short-term boost, but the bigger test will be the cardiomyopathy trial and lonvo-z data. Investors should weigh the potential upside of Intellia’s cutting-edge CRISPR therapies against the regulatory and safety risks that could keep the stock volatile.
