What could become the first nonsurgical therapy for ptosis, or droopy eyelid, inched closer to an NDA submission with upbeat top-line phase III efficacy results from Study 202, Osmotica Pharmaceuticals plc's test of RVL (oxymetazoline hydrochloride ophthalmic solution 0.1%), supplemented by new long-term phase III safety data, known as Study 203.

Almost nine years after its complete response letter (CRL) from the FDA for tafamidis, Pfizer Inc.'s approval of two forms of the drug well ahead of their PDUFA dates to treat wild-type or hereditary transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM) left investors in Pfizer and other firms speculating about what the win might mean.

Synthorx Inc. has rolled out intriguing data over the past couple of months and more is due ahead at the American Society of Clinical Oncology (ASCO) meeting in early June, when the San Diego-based firm unveils how synthetic biology can be used to reprogram the therapeutic activity of interleukin-2 (IL-2).

Exocrine pancreatic insufficiency (EPI) is one of the potential dreaded accompaniments of cystic fibrosis (CF) that patients must deal with, a condition that means downing as many as 40 pills per day if they take porcine-derived pancreatic enzymes (PPEs), the standard of care.

Saying he was "extraordinarily disappointed" by the complete response letter (CRL) from the FDA regarding HTX-011, Heron Therapeutics Inc. CEO Barry Quart called it "very discouraging" that the agency's objections could have been raised earlier during talks but were not.

Vividion Therapeutics Inc. CEO Diego Miralles told BioWorld that $82 million in new series B money will propel research to "clinical proof of concept in at least one of the three lead programs, if not more" as the company – just over a year after sealing a pact worth $101 million up front that brought Celgene Corp. aboard as a collaborator – pulled off an oversubscribed round with its partner on board.

Satsuma Pharmaceuticals Inc. CEO John Kollins told BioWorld Asia the migraine space may be in a stage of development similar to the depression therapeutic area before the advent of selective serotonin reuptake inhibitors, and his firm's dry-powder inhaled STS-101 (dihydroergotamine [DHE]) could help turn the corner in acute treatment of headaches.

The disclosure in late March by Blueprint Medicines Corp. that, based on talks with regulators, the Cambridge, Mass.-based firm will expedite development of avapritinib, BLU-667 and BLU-782 served to whet investor appetite and fuel speculation about the competitive chances of one candidate in particular.

The start in early April by Biohaven Pharmaceutical Holding Co. Ltd. of the phase II/III trial with BHV-3500 in acute treatment of migraine brought renewed attention to the calcitonin gene-related peptide (CGRP) receptor antagonist space, even as rumors swirled later in the month that the New Haven, Conn.-based firm was mulling a sale – if not of the company, then of its CGRP platform.