Rare Diseases And Orphan Drugs: New IPO And Patient Advocacy Foundation Success

This Blog Post discusses a new Initial Public Offering (IPO) and a patient advocacy foundation that has found success in the rare disease and orphan drug space.

I – Insmed

Insmed is a New Jersey biopharmaceutical company that is developing and commercializing novel, targeted inhaled therapies for orphan lung diseases. The company announces on July 15, 2013, that it is offering $60 million of its common stock in a proposed underwritten public offering. All of the shares in the offering are to be sold by Insmed. Leerink Swann LLC is the sole book-running manager for the offering.

Two days later on July 17, Insmed announces that it is pricing an offering of 6 million shares of its common stock at a price of $10.40/share in a “registered underwritten public offering for gross proceeds totaling $62.4 million.” Soon after, the company announces on July 22, the closing of a $71.8 million underwritten public offering of 6.9 million shares of common stock. Net proceeds to Insmed is about $67 million. Proceeds from the sale of the common stock is to fund the additional clinical development of its lead product candidate, Arikace for the following two orphan indications :

•   Cystic Fibrosis (CF) patients with Pseudomonas Aeruginosa lung infections
•   Patients with Non-Tuberculous Mycobacteria (NTM) lung infections.

II – Barth Syndrome Foundation Finds Success

The Barth Syndrome Foundation (BSF), is the only “world-wide volunteer organization dedicated to saving lives through education, advances in treatment, and the pursuit of a cure for Barth Syndrome (BTHS).” The foundation started in 2000 after the first international conference is held. At that first meeting, families from around the world decide to work together to find a cure for BTHS through a collaboration between families and scientists. Since 2003, the foundation has been funding research for BTHS.

According to the Foundation’s website:

“Barth syndrome (BTHS) is a rare, X-linked genetic disorder of lipid metabolism that primarily affects males across different ethnicities. Typically, boys with BTHS present with hypotonia (low muscle tone) and dilated cardiomyopathy (labored breathing, poor appetite, and/or slow weight gain) at or within the first few months after birth…. The gene for Barth syndrome, tafazzin (TAZ, also called G4.5), is located on the long arm of the X chromosome (Xq28). Mutations in the tafazzin gene lead to decreased production of an enzyme required for the synthesis of “cardiolipin,” a special lipid that is important in energy metabolism. There is no specific treatment for BTHS…”.

On July 24, 2013, the Barth Syndrome Foundation receives FDA Orphan Drug Designation (ODD) for Bezalip (Bezafibrate) for the therapeutic treatment of Barth Syndrome.

Please Note: “Global Thinking” by Benjamin D. Esham [Public domain] | Wikimedia Commons.

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