Caught off guard by the FDA’s approval – and ahead of schedule, to boot – of Orenitram (treprostinil) extended-release tablets for pulmonary arterial hypertension (PAH), United Therapeutics Corp. plans to launch the product within the next six months.
Intrexon Corp.’s $26 million takeover of Medistem Inc. brings aboard endometrial regenerative cells (ERCs), derived from menstrual blood, for use with integrated synthetic biology platforms developed by Intrexon, opening potential for cell-based therapeutic candidates across a range of conditions.
The ongoing effort by Bristol-Myers Squibb Co. (BMS) to become a specialty biopharma firm led to its $2.7 billion split from the joint venture for Type II diabetes with Astrazeneca plc, leaving investors in both firms to sort through details.
Ribavirin (RBV) could drop out of hepatitis C virus (HCV) therapy for genotype 1 (GT1) patients, as a result of Gilead Sciences Inc.’s triumph in the Phase III program known as ION, which tested the once-daily, fixed-dose combination of its approved nucleotide analog polymerase inhibitor Sovaldi (sofosbuvir) and the NS5A inhibitor ledipasvir, with and without RBV.
Targacept Inc.’s Phase II blowup of TC-5619 in schizophrenia, months after its fizzle in attention deficit hyperactivity disorder (ADHD), nixed the development program and turned investor eyes to a separate therapy for overactive bladder (OAB), also in Phase II, due to report data in the middle of next year.
Shopping among European collaborators for its drug-device pain combo known as Zalviso, Acelrx Pharmaceuticals Inc. “had a number of different suitors approach us,” with “a lot of very large-scale players interested,” said CEO Richard King.
<P>After Coronado Biosciences Inc. reported two trial failures this year in Crohn’s disease with its oral Trichuris suis ova (TSO, or pig whipworm eggs), investors’ faith had fallen low in the unusual immune system-based approach, but Wall Street sang a different song when the company unveiled positive data from a pilot study in the seemingly unlikely indication of autism. <!--break--></P>
The prevalence of sickle cell disease (SCD) is a challenge for the healthcare systems around the world. There are very limited treatment options and, as yet, no effective therapies that address the underlying cause of the disease.
After Coronado Biosciences Inc. reported two trial failures this year in Crohn’s disease with its oral Trichuris suis ova (TSO, or pig whipworm eggs), investors’ faith had fallen low in the unusual immune system-based approach, but Wall Street sang a different song when the company unveiled positive data from a pilot study in the seemingly unlikely indication of autism.
Oversized hopes for what the company designed as an exploratory Phase II trial led to Immunocellular Therapeutics Ltd.’s stock plunge, after the company disclosed mixed results with its dendritic cell-based vaccine in glioblastoma multiforme (GBM), said CEO Andrew Gengos.
