Steadymed Ltd. CEO Jonathan Rigby told BioWorld that officials at his firm "feel more confident now than we ever have that we know exactly what the agency wants to see," after the FDA provided guidance regarding what's needed to resubmit the NDA for Trevyent (treprostinil), delivered by way of Patchpump technology for pulmonary arterial hypertension (PAH).
"There was always a question of whether or not there was something special about brexanolone," Sage Therapeutics Inc.'s chief medical officer (CMO), Steve Kanes, told BioWorld, referring to the phase III win in post-partum depression (PPD) last month. "It doesn't seem to be the case."
Madrigal Pharmaceuticals Inc. is waiting on further data to provide firm guidance about a potential phase III trial, but shares (NASDAQ:MDGL) meanwhile soared 88.3 percent, or $40.88, to close Wednesday at $87.18 on word of positive top-line findings from a phase II experiment in nonalcoholic steatohepatitis (NASH) with MGL-3196, a first-in-class, oral, once-daily, liver-directed, thyroid hormone receptor beta-selective agonist.
RT-002 achieved "a trifecta of positive clinical results," Revance Therapeutics Inc. CEO Dan Browne said, pointing to across-the-board success in a pair of phase III trials with next-generation neuromodulator DaxibotulinumtoxinA for injection (RT-002), which turned up positive top-line results in alleviating moderate to severe glabellar (frown) lines in the clinical program called Sakura.
The FDA has given Mylan NV the go-ahead for its Ogivri (trastuzumab-dkst), a biosimilar to Roche AG's blockbuster breast cancer therapy Herceptin (trastuzumab), for patients with breast or metastatic stomach cancer (gastric or gastroesophageal junction adenocarcinoma) whose tumors overexpress the HER2 gene.
Lumen Bioscience Inc. plans to use its $11.2 million in series A money – supplemented by $1.8 million in the form of a grant from the U.S. Department of Energy – to explore the use of spirulina in making therapeutic products, CEO and co-founder Brian Finrow told BioWorld Insight.
After Jefferies analyst Peter Welford said in a late August report that Puretech Health plc affiliate Restorbio Inc. was "fast grabbing our attention," investors are paying heed as well, helping the company to a $40 million series B round to advance the immunotherapy RTB-101, a mechanistic target of rapamycin complex 1 (mTORC1) inhibitor, through its phase IIb study and potentially into phase III for reducing the incidence of respiratory tract infections (RTIs) in elderly people.
Phaserx Inc. CEO Robert Overell told BioWorld the firm has paused its lead, preclinical effort in intracellular enzyme replacement therapy (iERT) as the search continues for "a strategic transaction, including a potential merger" to advance the pipeline. "The goal is to get this technology and these programs into the hands of a group who can adequately capitalize it and capitalize on it," he said.
Hopes had not exactly soared high for Regeneron Pharmaceuticals Inc.'s combo pairing the angiopoietin2 (Ang2) antibody nesvacumab with approved Eylea (aflibercept), although "nobody would like it to do a bit more" than Eylea monotherapy, said CEO Leonard Schleifer, referring to chief scientific officer George Yancopoulos, who discovered Ang2. "A lot of people have sort of forgotten that," he said.
American Society of Hematology (ASH) President Kenneth Anderson said that the annual meeting, set for next month in Atlanta, will include plenty more data with chimeric antigen receptor (CAR) T cells, highlighting the approach's much-celebrated ability to build for patients an "autologous immune army."
