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- A development-stage biopharmaceutical company focused on the identification, licensing, acquisition, development and commercialization of drugs for rare and ultra-rare genetic and metabolic diseases.
- Price-to-book is 4.1. Low cash burn rate (P/e ratio of -144).
- 25% of the IPO is spoken for.
There are no Transcripts on ORPN.
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- The FDA grants Orphan Drug status to Bio Blast's (ORPN -5.6%) Cabaletta for the treatment of Spinocerebellar Ataxia Type 3 (SCA3 or Machado-Joseph disease). It is the second Orphan Drug tag for the product candidate.
- Cabaletta is a chemical chaperone that protects against pathological processes in cells. It has demonstrated efficacy in preclinical cells and animal models of SCA3 as well as other diseases. The company plans to proceed with clinical development in 2015.
- Among the benefits of Orphan Drug status is an additional seven-year period of market exclusivity if approved.
Mon, Sep. 22, 9:27 AM
- Health Canada approves the initiation of patient enrollment at the Montreal Neurological Institute at McGill University for BioBlast Pharma's (NASDAQ:ORPN) Phase 2/3 study evaluating Cabaletta in patients with occulopharyngeal muscular dystophy (OPMD). The study will be conducted across three centers in Canada, Israel and the U.S.
- Cabaletta is a chemical chaperone that protects against pathological processes in cells.
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Bio Blast Pharma Ltd is a development-stage biopharmaceutical company. It is engaged in the identification, licensing, acquisition, development and commercialization of drugs for rare and ultra-rare genetic and metabolic diseases.
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