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Rare Diseases And Orphan Drugs: Initial Public Offerings (IPOs)

May 22, 2013

PwC’s (PricewaterhouseCoopers) Pharmaceutical and Life Sciences Industry Group, releases a new US May 2013 report, “Capital Crunch”, that reports on the venture capital dollars invested in biotechnology and medical device companies for the Q1 2013. According to the report, “fewer venture capital dollars are invested into life sciences companies in Q1 2013”, with venture capital dollars falling 14% as investors turn to less capital-intensive industries.

According to the PwC Report, for Initial Public Offerings (IPOs), during Q1 2013 :

•   8 venture-backed IPOs raised $672 million, a drop of 52% from the previous quarter

•   the # of listings represents a 3-year low, a decline of 58% from the Q1 2012.

The report concludes with the following from Tracy T. Lefteroff, Global Managing Partner of the Venture Capital practice at PwC US :

“… Investors are particularly interested in products that will command a premium price in the market, such as those that target orphan diseases. And the FDA’s recent efforts to streamline the regulatory approval process are encouraging for many companies. The agency has already granted several products ‘breakthrough’ status, giving them priority for quick approval. This activity could prompt investors to take a closer look at the sector.”

The following are recent IPOs in the rare disease and orphan drug space.

I – PTC Therapeutics

PTC Therapeutics, a South Plainfield, NJ-based biotech, files with the SEC to raise up to  $85 million in an initial public offering. The  company originally files for an IPO in March 2006 before ultimately withdrawing  its  offering in 2007.  PTC Therapeutics develops its lead drug Ataluren for genetic disorders with nonsense mutation. According to a May 16, 2013 Press Release, PTC Therapeutics announces that it has filed a registration statement on Form S-1 with the US Securities and Exchange Commission (SEC), relating to the proposed IPO of its common stock. The number of shares to be offered and the price range for the offering have not been determined. J.P. Morgan and Credit Suisse are acting as joint book-running managers for the offering. PTC Therapeutics wants to list its shares on the NASDAQ under the symbol “PTCT”.

PTC Therapeutics lead drug candidate is orphan drug Ataluren which has FDA Orphan Drug Designation (ODD) for three indications :

1) Muscular Dystrophy

2) Spinal Muscular Atrophy

3) Cystic Fibrosis.

PTC Therapeutics has launched a Phase III study of Ataluren for Duchenne Muscular Dystrophy and plans on starting a confirmatory Phase III study of Ataluren for Cystic Fibrosis in the second half of 2013.

II – Kamada

Kamada, an Israeli therapeutics company focusing on orphan drugs and plasma-derived protein therapeutics, announces on May 15, 2013, the terms for the $60 million US IPO. Kamada currently trades on the Tel Aviv Stock Exchange under the ticker KMDA and plans to list on the NASDAQ under the symbol KMDA. The company initially filed confidentially on January 24, 2013. Kamada files an initial Form F-1 with the US SEC in April 2013.

Kamada’s inhaled AAT (AAT IH) product is in advanced stages of clinical trials, under FDA and EU ODD for several indications :

•  AAT Deficiency
•  Cystic Fibrosis
•  Bronchiectasis.

III – Bluebird bio

On May 14, 2013, bluebird bio files a $86.25 million IPO. The company plans to list on the NASDAQ under the symbol “BLUE”. J.P. Morgan and Bank of America Merrill Lynch are leading the offering. Bluebird bio files on May 14, a registration statement on Form S-1 with the US SEC.

Bluebird bio, a Cambridge, Massachusetts biotechnology company, develops next generation products based on gene therapy to treat severe genetic and rare diseases. The company has two clinical stage products in development for rare diseases that have been granted US and European ODD :

•   Lenti-D for the treatment of Childhood Cerebral Adrenoleukodystrophy (CCALD)
•   LentiGlobin for the treatment of ß-thalassemia major and Sickle Cell Disease (SCD).

Bluebird bio expects to start in late 2013, a Phase II/III clinical study of their leading product candidate, orphan drug Lenti-D, for CCALD. CCALD is a rare, hereditary neurological disorder affecting young boys that is often fatal. The company also expects to start in mid-2013, Phase I/II clinical studies of orphan drug LentiGlobin, in patients with ß-thalassemia major and SCD.

Please Note: “Dollar Symbol Gold” by Rugby471 (Own work) [GFDL] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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