UCB to acquire Ra Pharmaceuticals; joining forces to improve treatment options in rare diseases

UCB agrees to acquire Ra Pharmaceuticals with the goal of joining forces to improve treatment options for people living with myasthenia gravis and other rare diseases.

Ra specialises in using synthetic peptides to create novel medicines for serious diseases caused by excessive or uncontrolled activation of the complement system, a critical component of the innate immune system.

Ra Pharma’s phase III product candidate, zilucoplan, is a once-daily self-administered, subcutaneous peptide inhibitor of C5.

In December 2018, Ra Pharma announced positive top-line results from a phase II trial of zilucoplan in patients with generalised myasthenia gravis (gMG), achieving clinically meaningful and statistically significant reductions in both primary and key secondary endpoints.

It is being tested in a phase III trial in gMG and top-line results are expected in early 2021.

Zilucoplan is also being developed in patients with paroxysmal nocturnal haemoglobinuria (PNH), who have not responded well to Soliris.

There are further plans to develop zilucoplan in diseases including immune-mediated necrotising myopathy, amyotrophic later scleroisis, and other tissue-based disorders caused by issues with the complement system.

Ra is also developing an extended release formulation of zilucoplan, as well as a potential first-in-class oral small molecule C5 inhibitor.

UCB has made the acquisition as part of a strategic growth plan announced in January this year.

As well as potentially expanding its portfolio of products, the deal brings in Ra’s expertise with synthetic peptides, which combine the specificity and high affinity of therapeutic antibodies with pharmacological properties of small molecules.

Jean-Christophe Tellier, CEO UCB said: “Ra Pharma is an excellent strategic fit addressing multiple areas of UCB’s patient value growth strategy. Upon closing, 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-state pipeline projects.”

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