Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)

Summary

The purpose was to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of sapablursen administered subcutaneously to participants with non-transfusion dependent β-Thalassemia Intermedia.

Timeline

Start

2020-09-24

Primary completion

2023-03-28

Completion

2023-03-28

Outcome

Missed primary endpoint

Stopped: “Favorable safety and tolerability were seen, but efficacy results in the mid-stage study did not meet Ionis' minimum target product profile to justify further development.”

paper The study did not meet its primary or secondary endpoints of increased blood hemoglobin or reduced liver iron content (LIC). PMID 42241700 ↗

Publications

• Taher A, Diamantidis MD, Kattamis A, Aydinok Y, Kaplan ZS, Dibble A, Dorow S, Doherty S, Mathews J, Jung S, Goldberg YP, Tami J, Bhanot S, Schneider E, Monia BP, Barrett TD. Phase 2a randomized study to evaluate sapablursen in patients with non-transfusion dependent beta-thalassemia intermedia. Blood Adv. 2026 Jun 4:bloodadvances.2026019799. doi: 10.1182/bloodadvances.2026019799. Online ahead of print.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	sapablursen	Antisense oligonucleotide	30 mg	Subcutaneous
Subject	sapablursen	Antisense oligonucleotide	50 mg	Subcutaneous
Subject	sapablursen	Antisense oligonucleotide	80 mg	Subcutaneous
Subject	sapablursen	Antisense oligonucleotide	160 mg	Subcutaneous

Indications