Orphan Drugs And Rare Diseases: Top 2014 IPOs
This week, GEN (Genetic Engineering & Biotechnology News), publishes an online list of the top 20 Initial Public Offerings (IPOs) of the first six months of 2014 that “raked in the most proceeds after going public”. The article cites several reasons for the increase in IPOs :
• FDA increase in its reviews and decisions on new drugs
• Jumpstart Our Businesses (JOBS) Act: 5-year exemption from Sarbanes-Oxley Act & confidential submission of draft registration statements.
The chart below identifies the top IPOs in the orphan drug and rare disease space for the first 6 months of 2014. The data in the chart is from the online article published in GEN (except for the last column which is from the Orphan Druganaut Blog).
Top IPOs In Orphan Drug/Rare Disease Space
|GEN Rank #||IPO Name||Amount Raised In Net Proceeds (US $ in Millions)||# of Shares Issued (In Millions)/ IPO Price||FDA Orphan Drug Designation/Rare Disease Indications In Development|
|3||Kite Pharma||$ 146.625||8.625/ $17||ODD in March 2014 For Diffuse Large B-Cell Lymphoma; Cancer Immunotherapy Products|
|4||Versartis||$ 132.2||6.9/ $21||ODD in October 2013 For Growth Hormone Deficiency; Endocrine Disorders|
|5||Ultragenyx Pharmaceutical||$ 126.4||6.624/ $21||Mucopolysaccharidosis Type VII (MPS VII or Sly Syndrome), ODD in September 2011 for Hereditary Inclusion Body Myopathy (HIBM), X-Linked Hypophosphatemia (XLH); Genetic Diseases|
|7||ZS Pharma||$ 112||6.836/ $18||Hyperkalemia|
|8||Zafgen||$ 110.4||6.9/ $ 16||ODD in January 2013 For Beloranib For Prader-Willi Syndrome; Obesity|
|11||Dicerna Pharmaceuticals||$ 92.9||6.9/ $15||Primary Hyperoxaluria 1 (PH1); Rare/Inherited Liver Diseases & Cancer|
|12||uniQure||$85.4||5.4/ $17||ODD in May 2007 For Glybera For Lipoprotein Lipase Deficiency (LPLD) – EU approval in October 2012, Porphyria, Sanfilippo B, Hemophilia B; Gene Therapies For Orphan Diseases|
|13||Auspex Pharmaceuticals||$ 87.1||8.05/ $12||Huntington’s Disease, Tourette Syndrome, Tardive Dyskinesia; Orphan Hyperkinetic Movement Disorders|
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