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TTR SUPPRESSION
WAINUA provided rapid and sustained TTR suppression between doses in adults with hATTR-PN*1,2
- Week 35/65
- Week 85
LSM % CHANGE FROM BASELINE IN SERUM TTR1,2
CO-PRIMARY ENDPOINT:
- -66.4% significant difference with WAINUA vs placebo at Week 35 (95% CI, -71.4 to -61.5, P<0.001)
- -70.4% difference with WAINUA vs placebo at Week 65 (95% CI, -75.2 to -65.7)
NEURO-TTRansform was a Phase 3, randomized, open-label trial. Data presented from baseline to Week 35 are from the interim analysis; data beyond Week 35 are from the final analysis. Because the primary and key secondary endpoints were statistically significant at Week 35, no further formal statistical analyses were performed at Week 65/66. See study design.1-4
Reductions in serum TTR levels were sustained through Week 85 in adults with hATTR-PN1,2
Post hoc analysis
MEAN % CHANGE FROM BASELINE IN SERUM TTR1,2
Note: From Week 66 to Week 85, there was no external placebo group because NEURO-TTR concluded at Week 66. Change in serum TTR levels was assessed as a post hoc outcome at Week 85. No formal statistical analyses were performed at Week 85.2
NEURO-TTRansform was a Phase 3, randomized, open-label trial. Data presented from baseline to Week 35 are from the interim analysis; data beyond Week 35 are from the final analysis. Because the primary and key secondary endpoints were statistically significant at Week 35, no further formal statistical analyses were performed at Week 65/66. See study design.1-4
Effect of WAINUA on serum TTR levels through 3 years from treatment initiation†5
Data from Week 162 interim analysis of NEURO-TTRansform extension study measured from treatment initiation in the index study
MEAN (SEM) % CHANGE FROM BASELINE IN SERUM TTR5
Note: The placebo group refers to the external placebo control arm from the NEURO-TTR trial. From Week 66 to Week 162, there was no external placebo group because the NEURO-TTR trial concluded at Week 66. No formal statistical analyses were performed.5,6
The NEURO-TTRansform extension is an ongoing, Phase 3, open-label study evaluating the long-term efficacy and safety of WAINUA in patients who completed the NEURO-TTRansform index study. The interim analysis includes only patients randomized to receive WAINUA in the index study and excludes patients from the inotersen switch group. The demographics and clinical characteristics at baseline were generally similar between the index and extension cohorts. See study design.5
†In the NEURO-TTRansform extension study, WAINUA 45 mg SC was administered 4-8 weeks following the last dose in NEURO-TTRansform, then dosed once every 4 weeks thereafter.5,6
*Serum TTR level was first measured at Week 5. WAINUA 45 mg SC was dosed at Week 1 and once every 4 weeks thereafter. Serum TTR levels were drawn at trough drug levels predose, except at Week 35 and Week 85, which were drawn between doses.2,3
Discover the efficacy of WAINUA
Explore the NEURO-TTRansform study design
FOOTNOTES AND ABBREVIATIONS
EOT, end of treatment; hATTR-PN, polyneuropathy of hereditary transthyretin-mediated amyloidosis; LS, least squares; LSM, least-squares mean; SC, subcutaneous; SEM, standard error of mean; TTR, transthyretin.
REFERENCES
- 1. Data on File, REF-205487, AZPLP.
- 2. Coelho T, Marques W Jr, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy [article and supplementary online content]. JAMA. 2023;330(15):1448-1458.
- 3. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025.
- 4. Coelho T, Ando Y, Benson MD, et al. Design and rationale of the global phase 3 NEURO-TTRansform study of antisense oligonucleotide AKCEA-TTR-LRx (ION-682884-CS3) in hereditary transthyretin-mediated amyloid polyneuropathy. Neurol Ther. 2021;10(1):375-389.
- 5. Berk J, Coelho T, Conceição I, et al. Long-term efficacy and safety of eplontersen in patients with hereditary transthyretin amyloidosis with polyneuropathy: initial report from the open-label extension of the NEURO-TTRansform study. Presented at: 5th International ATTR Amyloidosis Meeting 2025; September 25-26, 2025; Baveno, Italy.
- 6. Data on File, REF-297876, AZPLP.
