BioMarin has purchased the rights to Repligen’s histone deacetylase (HDAC) inhibitor portfolio, including the experimental FA drug RG2833, whose development MDA has supported
Experimental Friedreich's ataxia (FA) drug RG2833, which has been in early-stage development by Massachusetts company Repligen with support from MDA, is now owned by California biotechnology company BioMarin.
BioMarin announced the agreement in a Jan. 21, 2014, press release, as did Repligen, in a separate release. BioMarin has purchased the rights to develop the FA drug and other compounds of its class that to date have been in development by Repligen for FA and other neurological disorders. RG2833 is one of multiple compounds with the potential to treat FA that were sold by Repligen to BioMarin.
RG2833 is a histone deacetylase (HDAC) inhibitor and is designed to increase production of the frataxin protein, which is deficient in FA-affected cells.
In April 2013, Jim Rusche of Repligen announced results of a phase 1 study of the drug in 20 FA-affected adults, showing it was well-tolerated and appeared to increase frataxin gene activity.
MDA welcomes transition
"MDA is delighted that BioMarin, a company with a great commitment to developing drugs for rare diseases, has taken on development of the HDAC inhibitors for Friedreich’s ataxia," said Jane Larkindale, MDA's vice president of research."
Larkindale continued, "MDA funded this therapy through MDA Venture Philanthropy, a program designed to fund development of therapies at an early stage to make them more attractive to later-stage drug developers. We have succeeded in this regard with this series of compounds, and we hope that BioMarin can complete the final stage [with an FA drug] and prove that it can be safely and effectively delivered to patients.
"MDA worked closely with other funding organizations — the Friedreich's Ataxia Research Alliance (FARA) and Go FAR Friedreich's Ataxia Research in particular — to make this happen, and we are excited by this progress."