Rozanolixizumab (UCB7665) is an investigational humanized monoclonal IgG antibody being developed by UCB for the treatment of myasthenia gravis (MG), a neuromuscular condition thought to be triggered by an autoimmune response.
In addition to MG, Rozanolixizumab is also being tested as a potential therapy for patients with idiopathic thrombocytopenic purpura, also known as immune thrombocytopenia (ITP).
How Rozanolixizumab works
The body’s immune system produces antibodies that normally help fight off infections and threats. In MG patients, pathogenic IgG autoantibodies mistakenly attack healthy cells and tissues, causing weakness and fatigue of voluntary muscles.
High concentrations of pathogenic IgG autoantibodies in the circulation occur due to the action of the neonatal Fc receptor (FcRn) that bind to IgG, inhibiting its degradation.
Rozanolixizumab works by binding with high-affinity to human neonatal Fc receptor (FcRn), selectively inhibiting IgG rescue and recycling. By reducing the serum levels of IgG, it is expected that Rozanolixizumab will help reduce the symptoms of MG.
Rozanolixizumab in clinical trials
The safety, tolerability, pharmacokinetics (movement in the body), and pharmacodynamics (effect on the body) of Rozanolixizumab were evaluated in a placebo-controlled Phase 1 clinical trial (NCT02220153). A total of 41 healthy adult volunteers were randomized to receive a single dose of Rozanolixizumab or placebo injected into the bloodstream or under the skin.
Preliminary results from the trial presented at the 2017 Peripheral Nerve Society Annual Meeting, showed that Rozanolixizumab effectively reduced serum IgG, with under-the-skin administration generally being better tolerated than injection into the bloodstream.
To further these observations, a Phase 2, multi-center, randomized, double-blind, placebo-controlled study (NCT03052751) was initiated to test the effectiveness, safety, and tolerability of Rozanolixizumab in patients with moderate-to-severe MG. The study is currently recruiting 42 patients in the U.S. and Europe and is expected to be completed by 2018.
Rozanolixizumab also is being tested in a Phase 2 trial (NCT02718716) for the treatment of immune thrombocytopenia. Interim results from the trial also confirmed the efficacy of Rozanolixizumab in the reductions of IgG levels.
The most common adverse effects associated with the use of Rozanolixizumab were headache and back pain.
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