Abstract Details

Title Phase 1 Study of Gefurulimab Pharmacokinetics (PK) and Safety Following Delivery Via Autoinjector in Healthy Adults

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P10 - Poster Session 10 (11:45 AM-12:45 PM)

Poster/Presentation
Number 11-004

Objective Compare gefurulimab PK exposure in healthy adults following a single subcutaneous (SC) dose administered by autoinjector versus manual prefilled syringe with safety device (PFS-SD). Safety, tolerability, immunogenicity, pharmacodynamics (PD), and device performance will also be assessed.

Background Monoclonal antibodies targeting terminal complement component 5 (C5) are approved for the treatment of acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR+ gMG). A phase 3 trial is investigating the safety and efficacy of PFS-SD–administered gefurulimab, a novel bispecific variable domain on a heavy chain (VHH) antibody that binds to C5 and albumin, in adults with AChR+ gMG (NCT05556096). Autoinjectors are prefilled, triggered devices designed for easy administration and have been shown to reduce pain at the injection site and improve adherence in individuals with chronic diseases versus alternatives. Autoinjectors represent an attractive option for at-home administration by the patient or caregiver.

Design/Methods This phase 1, open-label, randomized, parallel-group study will compare the PK of a single SC gefurulimab dose administered to the abdomen, thigh, or upper arm with either an autoinjector or PFS-SD in healthy adults aged 18-65 years. Approximately 174 participants will be enrolled, stratified into 3 weight groups, and randomized equally to one of six combinations of device and injection site. Participants are screened for ≤70 days prior to treatment on day 1 and remain at the clinic until day 5, after which visits occur throughout the 92-day evaluation period. Primary endpoints are serum gefurulimab PK parameters (areas under the curve and maximum observed concentration) for each device. Secondary endpoints are immunogenicity, safety, PD, additional PK parameters, and device performance

Results N/A

Conclusions This study will examine the PK, PD, safety, tolerability, immunogenicity, and device performance of the gefurulimab autoinjector in healthy adults. Results may support use of the autoinjector with the goals of providing convenient administration and enhancing patient experience in individuals with gMG.

Authors/Disclosures
Olivia Tong, MD PRESENTER	Dr. Tong has received personal compensation for serving as an employee of Alexion Pharmaceuticals.
Alanna McEneny (Alexion Pharmaceuticals)	Alanna McEneny has received personal compensation for serving as an employee of Alexion, AstraZeneca Rare Disease. Alanna McEneny has stock in AstraZeneca.
Torry Cong	No disclosure on file
Min Yee (Alexion)	Min Yee has nothing to disclose.

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