Rare Daily Staff

Belgian biopharmaceutical UCB has agreed to buy Ra Pharmaceuticals in a deal valued at $2.1 billion.

Ra Pharma shareholders will receive $48 per share in cash at closing, representing a 93 percent premium to Ra Pharma’s share price based on its average closing price for the 30 days prior to signing the agreement.

Cambridge, Massachusetts-based Ra Pharma is a clinical-stage biotech with a synthetic macrocyclic peptide platform that is developing drugs that target rare diseases caused by excessive or uncontrolled activation of the complement system, a critical component of the innate immune system.

Its lead compound, zilucoplan, is in late-stage development for generalized myasthenia gravis (gMG), a rare chronic autoimmune condition in which the body attacks specific proteins in the neuromuscular junction disrupting the way that nerves can communicate with muscles. It results in muscle weakness and fatigue. Both men and women are impacted equally, and it can occur at any age and in any race, and impacts almost 200,000 patients in the United States, Europe, and Japan. Those living with gMG can experience a variety of symptoms, including drooping eyelids and double vision as well as severe muscular weakness that can result in life threatening weakness of muscles of respiration.

Zilucoplan, if approved, will compete with Alexion’s Soliris. The difference will be that zilucoplan has been formulated as a once-daily self-administered subcutaneous injection, whereas Soliris must be administered by a physician.

In December 2018, Ra Pharma announced positive top-line results from a phase 2 trial of zilucoplan in patients with gMG, achieving clinically meaningful and statistically significant reductions in both primary and key secondary endpoints. Zilucoplan is currently being tested in phase 3 for the treatment of gMG with top-line results expected in early 2021. Further indications that are potentially addressable by zilucoplan include immune-mediated necrotizing myopathy (IMNM), amyotrophic lateral sclerosis (ALS) and other tissue-based complement-mediated disorders with high unmet medical need. Ra Pharma is also developing an extended release formulation of zilucoplan, as well as a potential first-in-class oral small molecule C5 inhibitor.

UCB CEO Jean-Christophe Tellier said the acquisition will add to its internal growth opportunities, which include six potential product launches in the next five years, and will strengthen its neurology and immunology pipeline.

The addition of Ra Pharma’s “pipeline in a product” investigational peptide C5 inhibitor zilucoplan alongside UCB’s anti-FcRn rozanolixizumab, could create an opportunity to provide more people living with myasthenia gravis with better treatment options, according to UCB.

Additionally, UCB gains access to Ra Pharma’s platform to produce synthetic macrocyclic peptides, which combines the diversity, specificity and high affinity of therapeutic antibodies with the attractive pharmacological properties of small molecules. It has the potential to augment UCB’s drug discovery capabilities and provide access to Ra Pharma’s proven expertise and talent in this area. The deal will also strengthen UCB’s presence in the United States as Ra will maintain its presence in the Boston area.

“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 Pharmaceuticals.

Photo: Doug Treco, president and CEO of Ra Pharmaceuticals

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