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Rare Diseases And Orphan Drugs: Two Company’s Business Developments

March 12, 2014

This Blog Post presents recent business developments for two biotechnology companies in the rare disease and orphan drug space.

I– Versartis

Versartis, a Redwood City, California-based biotechnology company developing treatments for growth hormone deficiency, announces this week the terms for its Initial Public Offering (IPO). The company plans on selling 4.6 million shares for $16 – $19/share. Versartis plans on listing on the NASDAQ under the symbol “VSAR” with Morgan Stanley and Citi as the joint bookrunners. Versartis initially files confidentially in January 2014.

Versartis’ lead drug candidate, VRS-317, the company’s proprietary long-acting form of recombinant Human Growth Hormone (rhGH), is currently being investigated for its safety and efficacy in pediatric Growth Hormone Deficiency (GHD) patients, for up to once monthly dosing. In October 2013, VRS-317 receives FDA Orphan Drug Designation (ODD) for GHD at once-a-month dosing – EMA also gives VRS-317 ODD. Currently, growth hormone treatments require daily injections. The company is using the novel half-life extension technology, XTEN, for developing new therapeutic proteins.

II– Lumena Pharmaceuticals

Lumena Pharmaceuticals, founded in 2011, is a privately held San Diego company developing oral therapeutics for rare liver diseases. The company receives for their lead candidate, LUM001, ODD from both the FDA (September 2013) and the EMA (November 2013) for the following 4 rare cholestatic liver disease indications:


Orphan Designation


Primary Biliary Cirrhosis (PBC)


Alagille Syndrome (ALGS)


Progressive Familial Intrahepatic Cholestasis (PFIC)


Primary Sclerosing Cholangitis (PSC)


On March 11th, Lumena Pharmaceuticals announces that it has secured $45 million in Series B financing from New Enterprise Associates (NEA) and several other investors to advance the clinical development of LUM001 and LUM002 (for treatment of Nonalcoholic Steatohepatitis (NASH)).

Please Note “LuMaxArt Golden Guys Teamwork Gear Runners” by lumaxart [CC-BY-SA-2.0] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

    • Dr. Crasto – I want to thank you so much for this !! Your constant support of the Orphan Druganaut Blog is greatly appreciated. Also, your dedication to the pharmaceutical industry, the sciences, the constant providing and sharing of knowledge by your blogs is amazing. Hopefully one day we will meet. Kind Regards, Ann

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