Neuromuscular Disease · registered primary completion 2027-07-01 · NCT06672237
Registered design for NCT06672237: randomized, quadruple-masked, 60 participants (estimated). Primary outcome: Modified Neuropathy Impairment Score +7 (mNIS+7). 1 further primary outcome(s) registered.
Cash of $438M against operating outflow of $109M per quarter gives roughly 4.0 quarters of runway, clearing the registered primary completion 3.4 quarters out by 0.6 quarters.
140,126,693 shares outstanding against 152,265,693 if every outstanding option, warrant and convertible were exercised. In that case 8.0% of a gain in enterprise value would accrue to those holders rather than to current shareholders.
Share count up 30% year over year to 140,126,693 as of 2026-07-31.
Designations disclosed: RMAT, Breakthrough Therapy, Fast Track, Orphan Drug. These accelerate review and, for Orphan, confer exclusivity if approved; none is evidence of efficacy.
5 distinct molecules in 4 active company-sponsored trials; NTLA-2002 accounts for 2 of them at Phase 1/Phase 2/Phase 3.