Building an orphan pipeline, Horizon Pharma bags PhIII-ready rare disease drug in $145M buyout

Timothy P. Walbert

Eight months after a little-known subsidiary group named River Vision Development Corp. won a breakthrough therapy designation for its lead drug for a rare eye disease, Horizon Pharma $HZNP has swooped in to pluck its sole drug asset in a buyout deal powered with a $145 million upfront.

The company is a subsidiary of Narrow River, a Rockefeller Center-based biotech which was set up by biotech veteran David Madden, the chairman at Dicerna and a board member of Navitor. Madden got River Vision started with a $17 million round backed by GSK’s SR One along with Lundbeckfond in 2012. Then he followed up with a licensing deal with Roche on teprotumumab (RV001), which had been partnered with Genmab, which had discovered the treatment.

Horizon is paying for an antibody targeting rare cases of thyroid eye disease, an autoimmune inflammatory disorder. In the Phase II trial the drug looked positive in the intent-to-treat group: 29 of 42 patients who received teprotumumab (69%), as compared with 9 of 45 patients who received placebo (20%), had a response at week 24.

Horizon is well known for handling some of the highest cost meds in the industry, with two of the top 10 most expensive drugs on the planet — both orphan therapies. And they see this latest purchase as a key example of how they plan to build a pipeline of rare disease treatments.

The deal comes with an unspecified set of milestones.

Horizon’s investors, though, were more focused on the company’s disappointing Q1 performance this morning, with a big cut to the company’s 2017 forecast, which drove down the value of its shares by 22% in pre-market trading.

Their new drug inhibits insulin-like growth factor type 1 receptor (IGF-1R), and Horizon believes it has the potential to go on to an orphan approval and more than $250 million in annual sales. The BTD title was won on human data that the company snagged in its TED01RV trial.

“This acquisition is an important step in our strategy of pursuing and acquiring development-stage medicines targeting rare diseases,” said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma.  “With no approved medicines to treat Thyroid Eye Disease, there is a significant unmet treatment need among the approximately 10,000 patients in the United States with moderate to severe disease and we look forward to beginning the pivotal study with teprotumumab in the second half of this year.”

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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