Enrollment in the phase IIb COBALT-1 trial of gemcabene (CI-1027) was small, due to the orphan population of homozygous familial hypercholesterolemia (HoFH), but meaningful, according to Lee Golden, chief medical officer of Gemphire Therapeutics Inc., who cited comparisons with phase II proof-of-concept studies for approved HoFH drugs Repatha (evolocumab, Amgen Inc.), Juxtapid (lomitapide, Aegerion Pharmaceuticals Inc.) and Kynamro (mipomersen, Kastle Therapeutics LLC).
Logicbio Therapeutics Inc. hung out its shingle for public display, extending its fundraising with an oversubscribed $45 million series B led by publicly traded Arix Bioscience Ltd. and Orbimed with participation from Edmond de Rothschild Investment Partners, Pontifax and SBI Japan-Israel Innovation Fund.
With two phase III studies of lead compound, KPI-121 1.0 percent, in the books to treat inflammation and pain following cataract surgery, Kala Pharmaceuticals Inc. looked to the public markets to fund pre-commercialization activities for the nanoparticle-based therapy by filing for an IPO of $86.25 million.
I missed BIO this year – the first time in 40 years as a journalist that I canceled attendance at a major conference due to a death in the family. About 12 hours before I was due to board a plane to San Diego, my brother in-law, Kevin, had a massive heart attack and died at the age of 64. In life, Kevin was what health care professionals likely would describe as a “challenging” patient. Like his father, he was subject to familial hypercholesterolemia. Unlike his dad, who had a proverbial “warning shot” in his 40s and then embraced a...
Novartis AG put its VEGF-A ligand inhibitor, brolucizumab (RTH-258), to the test in neovascular age-related macular degeneration (nAMD) against Regeneron Pharmaceuticals Inc.'s cash cow, Eylea (aflibercept), and appeared to come up a winner.
Six months after Rubraca (rucaparib) was granted accelerated approval by the FDA to treat BRCA mutation-associated advanced ovarian cancer in patients previously treated with two or more chemotherapies, Clovis Oncology Inc. basked in the bigger win it predicted all along.
Checkmate Pharmaceuticals Inc. might be the only immuno-oncology (I-O) company that's not attending the BIO International Convention in San Diego. Instead, the Cambridge, Mass.-based firm will spend the week plotting the next moves to advance its only asset, CMP-001, after completing a $27 million series B preferred financing led by new investor F-Prime Capital Partners.
Acceleron Pharma Inc. found itself in an enviable position considering the phase II DART study of dalantercept plus Inlyta (axitinib, Pfizer Inc.) missed its primary endpoint in advanced renal cell carcinoma (RCC). Instead of rocking the company's shares (NASDAQ:XLRN), the trial failure and decision to halt development of dalantercept seemed to allay concerns that the asset was a distraction from the company's lead program, luspatercept, partnered with Celgene Corp.
Regulus Therapeutics Inc. is halting development of its lead microRNA candidate, RG-101, an N-acetylgalactosamine (GalNAc)-conjugated asset targeting miR-122 in chronic hepatitis C virus (HCV), rather than pursuing resolution of a clinical hold placed by the FDA last June following a second case of jaundice in the phase II program.
The FDA stirred up a hornet's nest with an unprecedented request to Endo International plc to remove voluntarily its opioid pain medication, a tamper-resistant reformulation of Opana ER (oxymorphone hydrochloride), from the market.
