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Orphan Drugs And Rare Diseases : New Start-up And IPOs (06.18.13)

June 18, 2013

This Blog Post discusses a new startup and Initial Public Offerings (IPOs) in the rare disease and orphan drug space:

I – New Startup – LAM Therapeutics

LAM Therapeutics, a Connecticut-based biopharmaceutical company, announces on June 6, 2013,  that it has closed a Series A financing with private investors to launch operations to develop drugs for a rare lung disease. The company has a disease-specific focus and mission to “advance the clinical development of new therapies to treat Lymphangioleiomyomatosis (LAM).” Proceeds from the Series A financing are to be used for the identification of clinical stage drugs with potential activity against LAM and to conduct clinical trials. The company has developed assays to identify clinical stage compounds having different mechanisms of action on a wide spectrum of targets with potential activity against LAM. LAM Therapeutics is the first biopharmaceutical company dedicated to identifying and developing drugs for the treatment of LAM.

LAM is a rare lung disease found mostly in women that results in progressive cystic lung destruction. Besides the lungs, LAM can affect other organs including lymph nodes and kidneys. LAM is a “destructive, metastasizing neoplasm of smooth muscle-like cells that leads to progressive cystic lung disease. The disorder causes shortness of breath, lung destruction, respiratory failure, and death.” The disease originates from mutations in tuberous sclerosis complex (TSC) genes or can occur in otherwise healthy women (sporadic LAM). There is currently no cure or FDA-approved drug for LAM. There are approximately 200,000 LAM patients worldwide. There are at least 200 new cases of LAM diagnosed each year in the US.

References

LAM Foundation

LAM Health Project.

II – IPOs Expected To Price Week Of 06/17/13

According to the Renaissance Capital IPO tracker, two US IPOs in the rare disease and orphan drug space are expected to price this week, the week of 06/17/13 :

•    PTC Therapeutics plans to raise $100 million by offering 6.9 million shares at a price range of $13 – $16.  At the $14.50 midpoint of the proposed range, the company would have a market value of $322 million. PTC Therapeutics, a New Jersey-based biotech, has orphan drug Ataluren in its pipeline targeting Duchenne Muscular Dystrophy (DMD)/Becker Muscular Dystrophy (BMD), and Cystic Fibrosis (CF).  The company plans to list on the NASDAQ under the symbol PTCT. J.P.Morgan and Credit Suisse are the bookrunners for the deal. Reference Blog Post for further information.

•     Bluebird bio, a Cambridge Massachusetts-based biotechnology company, plans to raise $75 million by offering 5 million shares at a price range of $14 – $16. At the midpoint of the proposed range, the company would command a market value of $374 million. Bluebird bio develops products based on gene therapy to treat severe genetic and rare diseases. The company plans to list on the NASDAQ under the symbol BLUE. J.P.Morgan and BofA Merrill Lynch are the bookrunners for the deal. Reference Blog Post for further information.  

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

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

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