A Belgian drugmaker is spending more than $2 billion to acquire a company developing a drug to treat several rare and serious diseases.
Brussels-based UCB said on Thursday that it would acquire Cambridge, Massachusetts-based Ra Pharmaceuticals for $2.1 billion, and up to $2.5 billion, or $48 per share. Ra’s lead product candidate is zilucoplan, a C5 inhibitor administered via subcutaneous injection that is in Phase III development.
Shares of Ra were up nearly 100 percent following the news, trading at $45.34 per share on the Nasdaq Thursday afternoon. Shares of UCB were up nearly 2 percent on the Brussels Stock Exchange.
“Ra Pharma is an excellent strategic fit addressing multiple areas of UCB’s patient value growth strategy,” UCB CEO Jean-Christophe Tellier said in a statement. “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-stage pipeline projects.”
Ra’s zilucoplan, previously known as as RA101495, has the same molecular target as Alexion Pharmaceuticals’ approved C5 inhibitors, Soliris (eculizumab) and Ultomiris (ravulizumab-cwvz). Both Alexion drugs are approved for paroxysmal nocturnal hemoglobinuria, or PNH, while Soliris is additionally approved for atypical hemolytic uremic syndrome, or aHUS.
Zilucoplan – a peptide inhibitor rather than a monoclonal antibody – is in a Phase III study for generalized myasthenia gravis, or gMG, a rare autoimmune disease that causes weakness in the skeletal muscles and affects about 60,000 people in the U.S. A Phase II/III study in amyotrophic lateral sclerosis is planned, as is a Phase II study in immune-mediated necrotizing myopathy.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
UCB said the acquisition would boost its own potential for leadership in gMG, as it has its own drug in Phase III development for the disease, the FcRn-targeting monoclonal antibody rozanolixizumab. The rozanolixizumab study began in June, while Ra’s study started in October, according to ClinicalTrials.gov. Alexion started a Phase III study of Ultomiris in gMG in March. However, a Phase III study of Soliris in refractory gMG failed in 2016. A study of Soliris in pediatric patients with gMG opened in December 2018.
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