About two months ahead of the priority review action date, Novartis AG scored FDA clearance for Adakveo (crizanlizumab), previously known as SEG-101, to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and pediatric patients ages 16 and older with sickle cell disease (SCD).
Any worries by Promedior Inc. investors after Bristol-Myers Squibb Co. (BMS) backed away last year from its option to acquire the firm were resolved in a big way by last week's news that Roche Holding AG is taking over the company in a transaction valued as high as $1.39 billion. Privately held, Lexington, Mass.-based Promedior's lead asset is PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that won breakthrough designation from the FDA earlier this year for idiopathic pulmonary fibrosis (IPF).
CEO David Giljohann said the first indication in Exicure Inc.'s deal with Allergan plc is antigenic alopecia, but his firm doesn't "have anything else to disclose yet" about other hair loss diseases that the pact will include.
Avidity Biosciences Inc. CEO Sarah Boyce told BioWorld it's "a little too soon" to say when the company's lead program in myotonic dystrophy type I might reach the clinic, but the firm's $100 million series C financing will help with that effort and the portfolio in rare muscle diseases, as the antibody-oligonucleotide conjugate (AOC) platform undergoes further development.
Solid Biosciences Inc.'s chief technology officer, Joel Schneider, said the company is "not going to leave any stone unturned" as investigators begin to explore the severe adverse event (SAE) that led to the FDA's clinical hold on the phase I/II study with SGT-001 in Duchenne muscular dystrophy (DMD).
Within the first few minutes of a key opinion leader webinar on Alzheimer's disease sponsored by AC Immune SA, CEO Andrea Pfeifer brought up the decision by Biogen Inc. to file for regulatory approval of amyloid beta targeter aducanumab in early AD, based on results from a subset of patients in the phase III study called Engage.
Supernus Pharmaceuticals Inc. CEO Jack Khattar said the phase III failure in the study called P301 with SPN-810 (molindone hydrochloride) was "very puzzling" to the Rockville, Md.-based firm, which will be "digging very deep into the data" with hopes of figuring out what went wrong. Shares (NASDAQ:SUPN) closed Wednesday at $19.93, down $9.20, or 31.6%.
Redhill Biopharma Ltd. CEO Dror Ben-Asher told BioWorld that the 2 million patients targeted by just-approved, delayed-release Talicia, previously known as RHB-105, for adult infection by Helicobacter pylori represents "the baseline where we start our promotion," and there's an opportunity to help many more patients. "When you combine the high prevalence of roughly 100 million Americans infected and more than half of the world's population with the association with gastric cancer, it's a major public health concern," he said. About 800,000 people worldwide die every year from gastric cancer, and about 90% of cases are caused by H. pylori.
San Diego-based Otonomy Inc.'s launch of the phase I/II trial for OTO-413, a sustained-exposure formulation of brain-derived neurotrophic factor (BDNF), in patients with hearing loss brought renewed investor attention to the company, consigned to the back burner after a late-stage failure.
RNAi specialist Dicerna Pharmaceuticals Inc.'s chief operating officer, James Weissman, told BioWorld that scientists inside and outside of the company have come to believe in what's called "the X hypothesis" in hepatitis B virus (HBV). "My view, though, as a person who has been in the pharmaceutical industry for decades, is who cares, anyway?" he said – as long as the drug works. He pointed out that, with the cholesterol therapy Lipitor (atorvastatin calcium) from New York-based Pfizer Inc., where he served in business development and marketing, "everybody had a different theory for why it lowered the incidence of heart disease. Nobody knew with certainty."
