[{"id":"bal-ep1","name":"Placebo-adjusted mean TG reduction at 6 months","type":"PRIMARY","unit":"percentage points","p_value":"<0.001 (80mg); 0.08 (50mg, NS)","results":[{"ci":"-69.1 to -17.9","arm":"bal-80","label":"-43.5 pp (80mg vs placebo)","value":-43.5},{"ci":"-47.2 to +2.5","arm":"bal-50","label":"-22.4 pp (50mg, NS, p=0.08)","value":-22.4}],"timepoint":"Month 6","description":"FCS patients have triglycerides often >2,000 mg/dL (normal <150) due to genetic lipoprotein lipase deficiency. They cannot clear dietary fat from their blood, leading to life-threatening pancreatitis. Olezarsen blocks apoC-III (which normally inhibits TG clearance), allowing an alternative TG clearance pathway."}]

Favorable safety profile. Injection site reactions ~19% (mild). Mild platelet count reductions ~12% (NO cases <50K - key differentiator vs volanesorsen which had 76% thrombocytopenia). Arthralgia ~9%. No REMS required. GalNAc conjugation dramatically reduces off-target toxicity vs first-gen volanesorsen.

BALANCE is the pivotal FCS study for Tryngolza. FDA approved Dec 2024. The 80mg dose is clearly superior (-43.5% at 6mo, -57% at 12mo). The 50mg dose did NOT reach significance (p=0.08). Pancreatitis reduction (1 episode vs 11) is the clinically meaningful endpoint. Zero severe thrombocytopenia is the key safety win vs volanesorsen. SOBI has ex-US rights from Ionis (up to mid-20% royalty to Ionis).

GalNAc3-conjugated antisense oligonucleotide targeting apoC-III mRNA. GalNAc conjugation enables liver-targeted delivery at lower doses and monthly dosing (vs weekly for volanesorsen), dramatically reducing thrombocytopenia risk. Published NEJM 2024.