Track the companies that matter to you. It's FREE! Click one of these fan favorites to get started: Apple; Google; Ford.



Repligen, Continuing Overhaul, Flips Rare Disease Drugs to BioMarin

Don't let it get away!

Keep track of the stocks that matter to you.

Help yourself with the Fool's FREE and easy new watchlist service today.

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. 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 candidateto 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.

The Motley Fool's Top Stock for 2014
There's a huge difference between a good stock and a stock that can make you rich. The Motley Fool's chief investment officer has selected his No. 1 stock for 2014, and it's one of those stocks that could make you rich. You can find out which stock it is in the special free report "The Motley Fool's Top Stock for 2014." Just click here to access the report and find out the name of this under-the-radar company.

This article originally appeared on Xconomy, along with:

Read/Post Comments (0) | Recommend This Article (2)

Comments from our Foolish Readers

Help us keep this a respectfully Foolish area! This is a place for our readers to discuss, debate, and learn more about the Foolish investing topic you read about above. Help us keep it clean and safe. If you believe a comment is abusive or otherwise violates our Fool's Rules, please report it via the Report this Comment Report this Comment icon found on every comment.

Be the first one to comment on this article.

Sponsored Links

Leaked: Apple's Next Smart Device
(Warning, it may shock you)
The secret is out... experts are predicting 458 million of these types of devices will be sold per year. 1 hyper-growth company stands to rake in maximum profit - and it's NOT Apple. Show me Apple's new smart gizmo!

DocumentId: 2802406, ~/Articles/ArticleHandler.aspx, 9/1/2015 2:16:25 PM

Report This Comment

Use this area to report a comment that you believe is in violation of the community guidelines. Our team will review the entry and take any appropriate action.

Sending report...


Xconomy is dedicated to providing business and technology leaders with timely, insightful, close-to-the-scene information about the local personalities, companies, and technological trends that best exemplify today’s high-tech economy. Find out more at

Today's Market

updated Moments ago Sponsored by:
DOW 16,122.39 -405.64 -2.45%
S&P 500 1,924.66 -47.52 -2.41%
NASD 4,673.52 -102.99 -2.16%

Create My Watchlist

Go to My Watchlist

You don't seem to be following any stocks yet!

Better investing starts with a watchlist. Now you can create a personalized watchlist and get immediate access to the personalized information you need to make successful investing decisions.

Data delayed up to 5 minutes

Related Tickers

9/1/2015 2:00 PM
RGEN $33.64 Down -0.44 -1.29%
Repligen Corp CAPS Rating: ****