Bio Blast Pharma Ltd.'s (ORPN: Quote) lead product candidate Cabaletta has been granted Orphan Drug Designation by FDA for the treatment of Spinocerebellar Ataxia Type 3 (commonly known as SCA3 and Machado Joseph disease).
Cabaletta is under phase II study in Spinocerebellar Ataxia Type 3 in Israel. A phase 2/3 study of Cabaletta in treatment of Oculopharyngeal Muscular Dystrophy is also ongoing. Cabaletta also has orphan drug designation for Occulopharyngeal Muscular Dystrophy.
Bio Blast went public on the NASDAQ Global Market on July 31, 2014, priced at $11.00 per share.
ORPN closed Wednesday's trading at $4.65, down 1.27%.
Cellular Biomedicine Group Inc. (CBMG: Quote) is a stock to keep on your radar as the company has a couple of catalysts coming its way.
The company expects to present twelve month follow-up data analysis from its phase I/IIa clinical trial of lead regenerative medicine product candidate ReJoin for Knee Osteoarthritis on December 5, 2014 at the World Stem Cell Summit in San Antonio, Texas.
The six-month follow-up data analysis, announced in June of this year, concluded that ReJoin cell therapy for Knee Osteoarthritis patients is safe and revealed an increase in cartilage volume as early as three months after the therapy.
A multi-center phase IIb clinical trial for ReJoin is underway, with interim data scheduled for release in Q1 2015.
CBMG closed Wednesday's trading at $15.60, down 0.57%.
Cempra Inc. (CEMP: Quote) is all set to receive a funding of $16.0 million under a contract with Biomedical Advanced Research and Development Authority (BARDA) to cover the cost of a phase I study and start-up activities of a phase 2/3 study of Solithromycin in pediatric patients.
Visit link:
CBMG To Keep On Your Radar, CEMP Gets Funding, MACK On Fast Track