The
thrombopoietin mimetic peptide for injection is a second-generation
thrombopoietin receptor agonist (TPO-RA) used in the treatment of patients with
immune thrombocytopenia. The aim of the present study was to assess the safety, tolerance, pharmacokinetic and pharmacodynamic properties of
thrombopoietin mimetic peptide for injection in Chinese healthy volunteers. A randomized, placebo-controlled, double-blind, dose-escalation study was conducted in healthy Chinese subjects aged 18-50 years. Thirty subjects received single
subcutaneous injection of 0.3 μg/kg, 1.0 μg/kg, 2.0 μg/kg
thrombopoietin mimetic peptide or placebo.
Thrombopoietin mimetic peptide was safe and well tolerated at doses of 0.3-2.0 μg/kg. There was no significant change in mean platelet count (PLT) from baseline at the 0.3 μg/kg or placebo groups. The mean PLT of subjects in the 1.0 μg/kg and 2.0 μg/kg groups peaked at day 12 (± 1), began to decline around day 17, and returned to the baseline level at day 28 (± 1). Platelet aggregation rates of the three dose groups showed no significant change before and after administration. Serum concentrations of
thrombopoietin mimetic peptide in all subjects were below the quantization limit. This was the first study to demonstrate that
subcutaneous injection of
thrombopoietin mimetic peptide at doses of 0.3-2.0 μg/kg was safe and well tolerated in Chinese healthy subjects. As a second-generation TPO-RA,
thrombopoietin mimetic peptide is effective at improving PLT after single
subcutaneous injection at dose of ≥1 μg/kg.P lain l anguage s ummaryWhat is the context?●
Immune thrombocytopenia (
ITP) is a rare, serious autoimmune disorder characterized by low platelet count (PLT) without an alternate cause. The treatment goal of
ITP is to increase the platelet count to a safe level that can stop active
bleeding and reduce the risks of future
bleeding.●
Thrombopoietin receptor agonists (TPO-RAs, e.g.
eltrombopag,
avatrombopag,
hetrombopag, and
romiplostim) have shown high response rates in stimulating platelet production and reducing the risk of
bleeding. TPO-RAs provide
ITP patients with well-tolerated, long-term treatment choices.What is new?● The
thrombopoietin mimetic peptide for injection is a new TPO-RAs developed by Shandong Quangang
Pharmaceutical Co., Ltd. (China).● This study showed that
thrombopoietin mimetic peptide is effective at improving PLT after a single
subcutaneous injection.● The
thrombopoietin mimetic peptide is safe and well-tolerated in Chinese healthy subjects.What is the impact?● This study provides evidence for the further development potential of the
thrombopoietin mimetic peptide.