Ultragenyx Pharmaceutical Inc.
NEWS
Shares of Ultragenyx are down more than 15 percent this morning after the company announced its Phase III drug candidate UX007 failed to hit the mark as a treatment for patients with glucose transporter type-1 deficiency syndrome.
CODA Biotherapeutics launched in South San Francisco with a $19 million Series A financing.
Maryland-based REGENXBIO is expanding its gene therapy pipeline to include a new treatment for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease.
Recently, Milton Packer, a renowned cardiologist at Baylor Scott & White Health, wrote a criticism of the Orphan Drug Act and the resulting problems caused by the biopharma industry. John LaMattina, formerly president of Pfizer Global Research and Development and a current senior partner at PureTech Ventures, responded to the editorial in Forbes. Let’s take a look.
Most investors thought 2018 would be a big year for mergers and acquisitions in the biopharma industry because of changes to the tax law, and so far they’ve been right.
Ultragenyx CEO Emil Kakkis said the sale of the PRV will provide the company with an “important source of non-dilutive capital” that will be used to advance the company’s pipeline of rare and ultra-rare therapies.
Shares of Ultragenyx are sharply climbing this morning after the company announced positive results from its Phase III study of burosumab in adults with XLH.
Genentech’s Hemlibra just got approved but is expected to bring in around $2 billion in peak sales.
Here’s a closer look at six drugs facing FDA decisions in November.
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