Repligen, Continuing Overhaul, Flips Rare Disease Drugs to BioMarin

Xconomy Boston — 

Waltham, MA-based Repligen (NASDAQ: RGEN) decided a few years ago that it would be better off abandoning drug development and focusing instead on bioprocessing. Today it’s cut a deal to shed some of its remaining therapeutic baggage.

Repligen has licensed its portfolio of experimental, preclinical histone deacetylase inhibitors, or HDACs, to Novato, CA-based rare disease company BioMarin (NASDAQ: BMRN). Repligen is getting just $2 million from BioMarin up front, though it’s eligible to receive another $160 million in various milestone payments if the compounds in the deal make it through clinical testing and are approved by regulators. The Massachusetts biotech would also get royalties on the HDACs if they eventually hit the market.

Repligen had been developing an HDAC known as RG2833 to target Friedreich’s ataxia, a rare, inherited nervous-system disorder in which the spinal cord and peripheral nerves progressively degenerate, typically leading to difficulty walking and often a loss of sensation in the arms and legs. It’s the most common inherited form of ataxia, and affects about 20,000 people in the U.S. and Europe, according to Repligen. Friedreich’s ataxia is caused by a genetic defect leading to low production of the protein frataxin.

Repligen put RG2833 through a single early-stage trial in early 2013, and while it said that the results of that small study supported the approach of using an HDAC to treat Friedriech’s ataxia, researchers saw “the generation of potentially toxic metabolites” in patients dosed with the drug, according to regulatory filings. As a result, Repligen was unsure of its development path and said that its other HDAC compounds might be worth developing instead.

For Repligen, the deal the latest move in a strategic overhaul. The company initially started out in 1981 as a producer of industrial enzymes before trying its hand at drug development, attempting to fashion drugs for rare diseases and other conditions such as bipolar disorder. In 2011, Repligen changed course again, and decided to focus on bioprocessing—specifically, the manufacturing of protein A, a reagent used to purify monoclonal antibodies. It bought out its biggest competitor, the bioprocessing business of Novozymes, for about $26.4 million that year. Repligen has since been trying to shed, or find partners for, the experimental drugs in its portfolio. It handed over rights to a spinal muscular atrophy drug candidate to Pfizer in 2012, for instance.

“The outlicensing of our HDAC portfolio, which includes the Friedreich’s ataxia program, is consistent with our objective to realize financial value from discontinued therapeutic development programs as we fully focus on the expansion of Repligen’s bioprocessing business,” said Repligen president and CEO Walter Herlihy, in a statement.