In a boost to the drug discovery side of its business, Repligen (NASDAQ: RGEN) said it has found a very big pharma partner for its spinal muscular atrophy program—New York-based Pfizer (NYSE: PFE). Pfizer has agreed to pay up to $70 million to Waltham, MA-based Repligen for the licensing rights to any drugs that come out of the development effort, starting with an upfront payment of $5 million, plus royalties on sales of any approved drugs.
Repligen currently has a small molecule drug, RG3039, in the earliest stage of clinical testing for spinal muscular atrophy, a rare inherited disease of the central nervous system that occurs in approximately eight out of every 100,000 live births. Children born with the most serious form of the disease rarely live longer than two to three years.
Repligen, founded in 1981 as a manufacturer of industrial enzymes, has gone through a dramatic reinvention in recent years. After spending years unsuccessfully trying to develop drugs for bipolar disorder and other diseases, the company started building up its bioprocessing business and in 2011 bought out its leading competitor in that business, Novozymes. As a result of that purchase, Repligen now supplies over 95 percent of the world market for Protein A, used in the manufacture of monoclonal antibody-based drugs.
The company also hung on to its drug candidates for spinal muscular atrophy and Friedreich’s ataxia, another rare disease of the central nervous system, as well as an imaging agent for pancreatic cancer currently in a Phase III trial. But Repligen CEO Walter Herlihy told Xconomy in Sept. 2011 that the company needed larger partners to complete costly clinical trials. Herlihy said then that even with modest pricing, each of the two central nervous system drugs could bring in $500 million in revenues. “There’s a lot of interest in orphan drugs, so we think we’re well positioned to find a partner,” he told Xconomy.
In the press release announcing the Pfizer deal, Herlihy said the agreement “is consistent with the strategic decision we announced in August 2012 to focus Repligen’s internal efforts on the growth of our bioprocessing business, while seeking external partners for our therapeutic development programs.”
Under the terms of the agreement, Repligen is responsible for completing the first two cohorts of a Phase I trial of RG3039 in healthy volunteers, which it said should be occur in the next three months. Pfizer will then take over development of the spinal muscular atrophy drugs. Repligen has already received orphan-drug and fast-track designations for RG3039 from the FDA, and an orphan medicinal product designation from the European Union.
Families of SMA, a patient organization for spinal muscular atrophy, spent more than $13 million funding the preclinical development of RG3039.
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