Rare Daily Staff
Strongbridge Biopharma, a company focused on the development and commercialization of therapies for rare diseases, has entered into an agreement for Novo Nordisk to acquire the rights to Macrilen in the United States and Canada. Macrilen is the only U.S. Food and Drug Administration-approved oral drug for the diagnosis of adult growth hormone deficiency (AGHD).
AGHD is a rare disorder associated with increased morbidity and mortality that affects more than 50,000 adults in the United States. People who have AGHD can include those who were GH deficient as children and become adults with AGHD, or adults who become GH deficient. In adults, GH deficiency can develop when the pituitary gland or hypothalamus is damaged due, for example, to tumors, surgery, radiation or traumatic brain injury. If left undiagnosed, AGHD may lead to increased risk for premature death and significant morbidities, including an increase in body fat, increased rate of fractures, a decrease in muscle mass, dyslipidemia, weakness and fatigue, cardiovascular disease, osteoporosis, and impaired psychological well-being. Except in the presence of multiple other pituitary hormone deficiencies, AGHD cannot be diagnosed by routine blood or other tests; growth hormone stimulation testing is required to diagnose AGHD.
As part of the agreement, Novo Nordisk will pay Strongbridge $145 million upfront and will purchase approximately 5.2 million shares of Strongbridge stock at $7 per share, a premium to its recent market price. Strongbridge will also receive tiered royalties on sales through 2027, and its commercial organization will continue to support Macrilen, in partnership with Novo Nordisk, for the next three years.
The upfront payment and equity investment from Novo Nordisk will significantly strengthen the company’s overall financial position and marks a tremendous step forward in Strongbridge’s continued evolution as a company dedicated to rare diseases,” said Matthew Pauls, president and CEO of Strongbridge Biopharma.
Besides Macrilen, Strongbridge’s rare disease pipeline includes Recorlev (levoketoconazole), a cortisol synthesis inhibitor in phase 3 studies for the treatment of endogenous Cushing’s syndrome, and veldoreotide extended release, a pre-clinical next-generation somatostatin analog being investigated for the treatment of acromegaly and potential additional applications in other conditions.
Both Recorlev and veldoreotide have received orphan drug designation from the FDA and the European Medicines Agency. The company’s rare neuromuscular franchise also includes the orphan drug Keveyis (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis.
November 5, 2018
Photo: Matthew Pauls, president and CEO of Strongbridge Biopharma