Intellia Therapeutics, Inc. NTLA
Intellia Therapeutics, Inc. (Nasdaq: NTLA) is a rare genetic drug developer. Its lead program, lonvoguran ziclumeran (lonvo-z; NTLA-2002), is in Phase 3 for hereditary angioedema (HAE). Its next catalyst on record is lonvo-z: US commercial launch if approved (H1 2027).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: lonvo-z — US commercial launch if approved (H1 2027). 1 catalyst on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| lonvo-z (NTLA-2002) | hereditary angioedema | gene therapy | Phase 3 |
| nexiguran ziclumeran (nex-z; NTLA-2001) - MAGNITUDE | ATTR amyloidosis with cardiomyopathy | gene therapy | Phase 3 |
| nex-z (NTLA-2001) - MAGNITUDE-2 | hereditary ATTR amyloidosis with polyneuropathy | gene therapy | Phase 3 |
Recent results and decisions
- Sep 8, 2026accepted FDA accepted lonvo-z BLA with Priority Review; PDUFA date March 10, 2027; no AdComm currently planned.
FDA has accepted the Biologics License Application (BLA) for lonvo-z and granted the BLA Priority Review with a PDUFA target action date of March 10, 2027.
Filing - Apr 27, 2026met Met primary endpoint: 87% attack reduction vs placebo (0.26 vs 2.10 monthly attacks, p<0.0001); 62% attack-free vs 11%; no SAEs in lonvo-z arm.
The trial met its primary endpoint. For the six-month efficacy evaluation period (weeks 5 to 28), a one-time infusion of lonvo-z reduced attacks by 87% versus placebo, with a mean monthly attack rate of 0.26 in the lonvo-z arm compared with 2.10 in the placebo arm (p<0.0001).
Filing - Apr 27, 2026submitted Rolling BLA submission initiated with FDA; completion planned 2H 2026, US launch 1H 2027 if approved.
Intellia separately announced today that it has initiated a rolling BLA submission to the FDA to seek regulatory approval.
Filing
3 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 rare genetic companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.