UCB Acquires Ra Pharma, Adds Zilucoplan to Its MG Pipeline

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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Johnson & Johnson

UCB has completed its acquisition of Ra Pharmaceuticals, priced at about $2.3 billion, meaning that zilucoplan — an investigational treatment for myasthenia gravis (MG) — is being added to UCB’s product pipeline.

The transaction, whose start was announced by UCB last year, is expected to enhance the company’s potential in new therapies for MG, and to support its growth from 2024 onward.

“Zilucoplan gives us the opportunity to become a leader in treating people living with myasthenia gravis, an auto-antibody mediated neurological orphan disease with high unmet medical need. The acquisition also strengthens our neurology and immunology franchises with late and early-stage pipeline projects,” Jean-Christophe Tellier, CEO of UCB, said in a press release.

Zilucoplan is an artificial peptide that blocks a protein called complement 5 (C5), preventing the overactivation of the complement system — a set of more than 50 blood proteins that form part of the body’s immune defenses. The complement system is thought to be involved in the formation of harmful auto-reactive antibodies in MG.

With the acquisition, zilucoplan will now join UCB’s experimental antibody rozanolixizumab (UCB7665) in the company’s MG portfolio.

Rozanolixizumab is designed to block the activity of the human neonatal Fc receptor, a receptor that prevents MG autoantibodies from being destroyed upon binding. By blocking these receptors, rozanolixizumab is expected to lower the levels of circulating auto-reactive antibodies, helping to alleviate MG symptoms.

Both therapies are now being tested in Phase 3 trials.

Zilucoplan’s safety, tolerability, and efficacy are being tested in up to 130 adults with generalized MG (gMG) in a double-blind Phase 3 trial (NCT04115293) called RAISE.

Patients are being given either zilucoplan (0.3 mg/kg) or a placebo, both self-administered through an under-the-skin injection, for 12 weeks (three months).

RAISE’s primary goal is to determine the degree of change in patient scores on the myasthenia gravis activities of daily living (MG-ADL) scale from the study’s start (baseline measures) until week 12. MG-ADL will be used to measure the therapy’s ability to improve participants’ engagement in daily activities.

Rozanolixizumab is being evaluated in a double-blind Phase 3 trial (NCT03971422) expected to enroll around 240 adult gMG patients. Participants are being randomly assigned to one of two doses of rozanolixizumab, or a placebo, given via an under-the-skin infusion at pre-specified timepoints.

Its primary goal is also measures of changes in patients’ MG-ADL scores from baseline to day 43 (visit 10).

As a safety measure due to the current COVID-19 outbreak, enrollment in this global trial has been temporarily halted. A list of study locations is available here.