Horizon Pharma Inc. had its eye fixed on raising $86 million in its initial public offering (IPO), but – like other biotechs this year – had to lower its horizons considerably. The Northbrook, Ill.-based company priced 5.5 million shares of common stock at $9 per share to raise $49.5 million – a 42 percent discount off the aggregate proceeds sought in the company's S-1 filing last year. (See BioWorld Today, Aug. 5, 2010.)

Dynavax Technologies Corp. reported Wednesday that top-line data from the Phase III trial comparing its investigational hepatitis B vaccine Heplisav met all three clinical endpoints, demonstrating noninferiority, superiority and safety in comparison to the marketed hep B vaccine Engerix-B (GlaxoSmithKline). Nevertheless, investors failed to respond with enthusiasm, perhaps reflecting nervousness about a second piece of news regarding the company's consistency analysis of manufacturing data.

Reformulations aren't exactly a novel proposition in the biotech world, but Madeira Therapeutics LLC is taking that strategy one step further. The company is one of the few biotechs focused on repositioning adult compounds for the pediatric market.

A bevy of U.S.-based firms – notably Alnylam Pharmaceuticals Inc., of Cambridge, Mass. – together with London-based Silence Therapeutics plc and, more recently, South Korean start-up BioMolecular Therapeutics, have been striving mightily to become first to market using RNAi-based platform technology to treat cancer.

Serious conditions attract serious attention. Acute myeloid leukemia (AML) is a devastating form of cancer with a poor prognosis and limited treatment options. Already, a raft of biotechs has thrown resources at the disease, but with more failure than success. Now, TORCell Therapeutics Inc., a biotech spun out of the Ontario Institute for Cancer Research (OICR) in Toronto, is taking aim at AML with a technology to boost a patient's own double-negative T cells (DNT) – a subpopulation of T lymphocytes that has potent anticancer properties, but comprises only 1 percent of a patient's peripheral blood mononuclear cells.

Rib-X Pharmaceuticals Inc. hooked a big fish for its first partnership, inking an exclusive worldwide research collaboration and licensing option with Sanofi SA, of Paris, for antibiotics resulting from its RX-04 program for the treatment of resistant Gram-positive and resistant Gram-negative pathogens.

Genentech Inc. came out fighting during the second day of the FDA's public hearing on its proposal to withdraw accelerated approval of Avastin (bevacizumab) for metastatic breast cancer, putting members of the Oncologic Drugs Advisory Committee and representatives from the Center for Drug Evaluation and Research on notice that the company wouldn't go away quietly.

A parade of three dozen physicians, nurses, patients, advocates and family members overwhelmingly endorsed the continued availability of Avastin (bevacizumab) for metastatic breast cancer during the first day of the FDA's unprecedented hearing on its proposal to withdraw the drug's approval for the indication. But in a slew of legal and scientific data, representatives from the FDA's Office of New Drugs and Center for Drug Evaluation and Research laid the groundwork for an unflinching rejection of the request by Genentech Inc., of South San Francisco, and parent company Roche AG, of Basel, Switzerland, to continue marketing the drug for metastatic breast cancer while Genentech pursues new studies to support Avastin's use in the indication.

Using technology acquired from Pfizer Inc., San Diego-based biotech MingSight Pharmaceuticals Inc. has its eye on an oral therapy for retinal diseases.

A small biotech that has flown under the radar for nearly a decade may be poised to make an impact in the global hepatitis B space. Spring Bank Pharmaceuticals Inc. expects to file an investigational new drug (IND) application with the FDA this year for SB 9200, an orally bioavailable hepatitis B drug based on the company's small molecule nucleic acid hybrid (SMNH) technology.