Trial reportBlood advances2026
Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria.
Trial report in Blood advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 1 paper.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
An Open-Label Study to Evaluate Efficacy and Safety of Long-Term Treatment With ACH-0144471 in Participants Who Completed Clinical Study ACH471-100
A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ALXN2050 (ACH-0145228) in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy
Who cites it
1 citing paper in PubMed.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
abstractParoxysmal nocturnal hemoglobinuria (PNH) is a rare, complement-mediated hematologic disease. We present the results of a phase 2, open-label clinical trial of vemircopan monotherapy in patients with PNH to explore the efficacy and safety of inhibiting the complement alternative pathway via factor D. Adults (aged ≥18 years) with PNH who were treatment-naïve and switched from eculizumab with hemoglobin (Hgb) of <10 g/dL or rolled over from danicopan monotherapy (ACH471-103 trial; ClinicalTrials.gov identifier: NCT03181633) were enrolled. The trial comprised a 60-day screening period, 12-week treatment period, and 148-week long-term extension. Participants received vemircopan 120 mg twice daily, with potential escalation to 180 mg twice daily. The primary endpoint was change in Hgb from baseline to week 12. Safety endpoints included treatment-emergent adverse events (TEAEs) and serious AEs. Twenty-nine participants were enrolled (treatment-naïve, n = 12; eculizumab-switch, n = 11; and danicopan-rollover, n = 6); all completed the 12-week treatment period, and 1 danicopan-rollover participant completed the long-term extension. Clinically meaningful improvements from baseline to week 12 in Hgb (change of ≥2 g/dL) were reported in the treatment-naïve (mean, 3.6 [standard deviation [SD], 1.5]) and eculizumab-switch (mean, 3.3 [SD, 2.0]) cohorts and maintained through the end of the trial. Eighty-two breakthrough intravascular hemolysis (BT-IVH) events in 25 participants were reported. Twenty-eight participants reported TEAEs, and 14 reported serious AEs; most were unrelated to vemircopan. No deaths occurred. This trial evaluating vemircopan monotherapy met its primary efficacy endpoint; however, concerns regarding BT-IVH risk and suboptimal, inconsistent control of IVH emerged, leading to early trial termination. This trial was registered at www.clinicaltrials.gov as #NCT04170023, and EudraCT as #2019-003830-17.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.