STUDY DESIGN

The NEURO-TTRansform trial studied the efficacy and safety of WAINUA in adults with hATTR-PN1,2

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A Phase 3, randomized, open-label, externally controlled, multicenter study1-5

NEURO-TTRansform Trial Study Design NEURO-TTRansform Trial Study Design

All patients received supplemental doses of the recommended daily allowance of vitamin A.

The external placebo control from NEURO-TTR was appropriate because of similar eligibility criteria and endpoints1,3,6

  1. *Patients included in NEURO-TTRansform were 18 to 82 years of age, and had a confirmed diagnosis of hATTR-PN as defined by all of the following: Stage 1 (ambulatory without assistance) or Stage 2 (ambulatory with assistance) familial amyloid polyneuropathy or Coutinho stage; documented mutation of the TTR gene; and signs and symptoms consistent with hATTR-PN, including NIS ≥10 and ≤130. Patients must have discontinued active treatment with any other approved drug for hATTR 2 weeks prior to study.2,3

  2. †The inotersen reference group was intended to confirm sufficiently comparable disease progression and treatment response patterns between NEURO-TTR and NEURO-TTRansform trials.3

  3. ‡All endpoints were compared with the placebo arm of the earlier NEURO-TTR trial (NCT01737398).1,2,6

  4. §The final analysis endpoints were distributed over 2 visits (Week 65 and Week 66), as prespecified in the protocol.3

Discover the efficacy of WAINUA

WAINUA efficacy

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Explore the safety profile of WAINUA

WAINUA safety

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FOOTNOTES AND ABBREVIATIONS

COMPASS-31, Composite Autonomic Symptom Score-31; EOT, end of treatment; hATTR, hereditary transthyretin-mediated amyloidosis; hATTR-PN, polyneuropathy of hereditary transthyretin-mediated amyloidosis; mBMI, modified body mass index; mNIS+7, modified Neuropathy Impairment Score +7; NIS, Neuropathy Impairment Score; Norfolk QoL-DN, Norfolk Quality of Life-Diabetic Neuropathy; Q4W, every 4 weeks; QW, every week; SC, subcutaneous; TTR, transthyretin.

REFERENCES

  1. 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025.
  2. 2. 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.
  3. 3. 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.
  4. 4. 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.
  5. 5. Data on File, REF-297876, AZPLP.
  6. 6. Benson MD, Waddington-Cruz M, Berk JL, et al. Inotersen treatment for patients with hereditary transthyretin amyloidosis. N Engl J Med. 2018;379(1):22-31.