In May, when Aquinox Pharmaceuticals Inc. signed its potential $155 million deal with Astellas Pharma Inc. for once-daily, oral rosiptor (AQX-1125) for the treatment of interstitial cystitis/bladder pain syndrome (IC/BPS), hopes ran high for the first-in-class SHIP1 activator, which seemed on track to become the first such therapy to reach the market in 20 years.

The FDA's approval of GW Pharma plc's liquid Epidiolex – the first pharmaceutical formulation of purified, plant-based cannabidiol (CBD) to reach the market – in Dravet syndrome (DS) and Lennox-Gastaut syndromes (LGS) opens the door to a new therapeutic and regulatory world.

Gossamer Bio Inc.'s co-founder, president and chief operating officer, Sheila Gujrathi, told BioWorld that by signing a potential $420 million deal with Aerpio Pharmaceuticals Inc., her firm hopes to pursue "higher-hurdle endpoints of mucosal healing" in inflammatory bowel disease (IBD) and use Aerpio's hypoxia induced factor (HIF)-1 alpha stabilizer, AKB-4924, and related compounds to bring about longer-term remissions in such patients.

An upsurge in research on fibroblast growth factor 21 (FGF21) during the past decade or so – along with the advance of drug candidates targeting nonalcoholic steatohepatitis (NASH) through that mechanism and others – helped Akero Therapeutics Inc. nail down its handsome $65 million series A round as the field evolves.

Reporting positive and eagerly anticipated, potentially wider label-enabling data from a pair of phase IIb trials, Heron Therapeutics Inc.'s non-opioid HTX-011 took another leap forward on the road to marketing approval in postoperative pain, leaving farther behind any concerns regarding the recent add-on indication for Exparel (bupivacaine liposome injectable suspension) from Pacira Pharmaceuticals Inc.

Although Merck & Co. Inc. continues to chalk up investor-pleasing sales with Keytruda (pembrolizumab) and Bristol-Myers Squibb Co. (BMS) keeps doing well with Opdivo (nivolumab), researchers have yet to strike upon the best combinations with other agents.

At the American Society of Clinical Oncology (ASCO) annual meeting in Chicago, Roche Holding AG reported disappointing news from the phase III Sandpiper study testing the phosphoinositide 3-kinase (PI3K) inhibitor taselisib in breast cancer, news that rocked the beleaguered drug class yet again. MEI Pharma Inc., however, is keeping faith with its PI3K prospect, which also yielded results at ASCO – and impressive ones.

Hopes for Flex Pharma Inc.'s phase II FLX-787 swirled down the drain – and so did the share price – after the company said clinical work is stopping because of oral tolerability concerns. Flex (NASDAQ:FLKS) ended Wednesday at $1.04, a loss of $3.14, or 75 percent.

Sage Therapeutics Inc. came a step closer in its bid to "rewrite the textbook on how depression should be treated," CEO Jeff Jonas told BioWorld, as the FDA rubber-stamped an expedited development route for type A gamma-aminobutyric acid (GABAA) modulator SAGE-217 in major depressive disorder (MDD). "We think this is a fundamental brain mechanism" with even wider applications, he said.