Biomarin Pharmaceutical Inc. is considering the design to be sought for the pivotal phase III trial with BMN 111 (vosoritide), an analog of C-type natriuretic peptide (CNP), in children with achondroplasia, thanks to strong results with the highest of three doses – 15 µg/kg/daily – in a phase II experiment that enrolled 26 children with a mean age of 7.8 years.
While Cormedix Inc. fights for patent rights in Germany and shops for a partner that could help pay for trials with the antimicrobial catheter-lock solution Neutrolin, guidance from U.S. gatekeepers regarding the second pivotal, phase III trial with the product spiced shares of the Bedminster, N.J.-based firm (AMEX:CRMD), which closed Wednesday at $5.09, up 33 cents, having traded as high as $5.75.
Biocryst Pharmaceuticals Inc.'s licensing deal with CSL Ltd. for the FDA-approved influenza treatment Rapivab (peramivir) brings $33.7 million aboard, with as much as $12 million more possible if regulatory milestones are hit, while letting the firm keep rights to U.S. pandemic stockpile orders – "the greatest potential source of income," according to Piper Jaffray analyst Charles Duncan, who viewed the CSL agreement as a bonus.
Avalanche Biotechnologies Inc. touched off a Wall Street landslide by disclosing phase IIa results with gene therapy AVA-101 for wet age-related macular degeneration (AMD) that showed the trial's 12-month primary endpoint was met, based on ophthalmic and systemic safety.
Roche AG unit Genentech is placing a sizeable bet on the field of ubiquitin-specific protease (USP), "which is probably where kinases were 20 or 30 years ago," Tim Harrison, Almac Discovery Inc.'s vice president of discovery chemistry told BioWorld Today.
As hordes converge on the 2015 Biotechnology Industry Organization (BIO) International Convention in Philadelphia, a question on the minds of many early stage innovators is how to stimulate more investment by venture capital (VC) firms in the costly and risk-fraught discovery sector.
Company officials would not go into details, but Sunesis Pharmaceuticals Inc.'s drill-down on data from the narrowly failed phase III trial called Valor could mean the firm will take aim at patients ages 60 and beyond as regulatory efforts continue with the quinolone derivative vosaroxin plus cytarabine in adults with relapsed or refractory acute myeloid leukemia (AML).
Unum Therapeutics Inc. followed up last year's series A financing round with a series B that almost triples the amount, hauling down $65 million and adding seven fresh investors to its roster behind New Leaf Venture Partners, the debut backer that led the round.
Melinta Therapeutics Inc.'s $67 million equity financing will allow the company to finish the half-completed, second phase III trial for acute bacterial skin and skin structure infections with new fluoroquinolone antibiotic delafloxacin – the first in that class for 14 years – and file for FDA approval in early 2016 while moving ahead with other efforts, CEO Mary Szela said.
