UCB Buys Ra Pharma, Adding Zilucoplan to Its MG Pipeline

UCB will add the investigational candidate zilucoplan to its therapeutic pipeline for the treatment of people with myasthenia gravis (MG), after announcing it will acquire the therapy’s developer, Ra Pharmaceuticals.

Two Phase 3 trials are currently recruiting participants with generalized myasthenia gravis: the RAISE trial (NCT04115293) will assess zilucoplan, and the other trial (NCT03971422) will test UCB’s MG candidate therapy rozanolixizumab (UCB7665).

“Ra Pharma is an excellent strategic fit addressing multiple areas of UCB’s patient value growth strategy,” Jean-Christophe Tellier, CEO of UCB, said in a news release.

“The acquisition will add to our strong internal growth opportunities — six potential product launches in the next five years, strengthening our neurology and immunology franchises with late- and early-stage pipeline projects,” he added.

Zilucoplan is an artificial peptide that specifically binds and inhibits the activity of complement 5 (C5), a member of the complement system.

The complement system, a group of 50 or so blood proteins, is part of the body’s immune defenses, and is thought to be involved in producing the harmful autoantibodies in myasthenia gravis, which attack healthy cells, leading to weakness and fatigue in voluntary muscles. By inhibiting C5, zilucoplan prevents the overactivation of the complement system.

Zilucoplan has been granted orphan drug status by the U.S. Food and Drug Administration for the treatment of myasthenia gravis.

The designation followed the results of a Phase 2 trial (NCT03315130) that showed that treating generalized myasthenia gravis patients with zilucoplan led to significantly improved scores in quantitative MG and myasthenia gravis activities of daily living tests compared with a placebo.

The effectiveness of zilucoplan is currently being assessed in a 12-week, global, pivotal, randomized, double-blind, placebo-controlled Phase 3 clinical trial known as RAISE (NCT04115293), which is currently enrolling patients.

The participants will self-administer an under-the-skin injection of 0.3 mg/kg of zilucoplan once daily. Top-line results are expected in early 2021. If zilucoplan is approved, UCB is expected to launch it worldwide.

UCB’s investigational antibody rozanolixizumab binds to human neonatal Fc receptor. This receptor normally binds the disease-causing IgG autoantibody and prevents its degradation. By selectively inhibiting this receptor, rozanolixizumab treatment decreases the serum levels of IgG, which is hoped will help reduce the symptoms of myasthenia gravis.

The efficacy and safety of the treatment in adult patients with generalized myasthenia gravis is being assessed in a Phase 3 clinical trial. The study (NCT03971422) is recruiting participants, a total of 240, and will be carried at eight clinical sites in the United States. More information on enrollment can be found on the trial’s page here.

With the merger, UCB will also gain access to Ra Pharma’s Extreme Diversity platform, which produces synthetic macrocyclic peptides. Large peptide libraries are produced by a technique known as mRNA display. These libraries can then be screened for desirable properties and potentially lead to the discovery of therapies with high affinity, potency, specificity, and stability, as well as improved bioavailability and tissue penetration.

“UCB shares our commitment to the rare disease patient community and our goal of developing novel, accessible, and cost-effective therapies in the areas of immunology and neurology,” said Doug Treco, president and CEO of Ra Pharma. “I firmly believe it is the right partner for us to advance new treatment options from our unique early- and late-stage pipeline to patients.”