Although a few skeptics remain, Esperion Inc.'s top-line data from its pivotal phase III study known as Study 2, or 1002-047, with bempedoic acid (BA) for lowering LDL-C seemed to lay mostly to rest the worries brought on by five neoplasm (lung cancer) deaths in Study 1. "When we think back to May [when data from Study 1 with BA were disclosed], the issue was really: What is the perceived meaningfulness of what we called and still call the spurious result?" CEO Timothy Mayleben told BioWorld.
In the company's second program with UX-007 (triheptanoin, or trihep), Ultragenyx Pharmaceutical Inc.'s data are "much more robust" – enough so that the FDA recently said the firm could file an NDA based on phase II findings, spokesperson Danielle Keatley told BioWorld.
Clementia Pharmaceuticals Inc. delighted Wall Street with news that the company would file an NDA in the second half of 2019 – a full year earlier than previously planned – based on data from the completed phase II trial with its lead candidate, oral retinoic acid receptor gamma agonist palovarotene in the ultra-rare bone disease fibrodysplasia ossificans progressiva (FOP).
Stoke Therapeutics Inc. CEO Edward Kaye told BioWorld that the company cranked its quest "into turbocharge mode, once we had the animal data [in June] that suggest we were having some pretty significant efficacy results," and clinical work is expected to begin by 2020.
When The Medicines Co. (MDCO) gained FDA approval in August 2017 for the antibiotic Vabomere (meropenem and vaborbactam), the win provided clear validation of the approach taken by new company Qpex Biopharma Inc., which has pulled down $33 million in series A money.
A spokesperson for Merrimack Pharmaceuticals Inc. said the company had little to add to its press release disclosing the halt of the phase II study with MM-121, but a pipeline review was promised during the third-quarter earnings call next month.
Although it's likely premature to use the word "trend" when talking about the industry overall, Novartis AG has been plenty active in business development, snatching up Endocyte Inc. for $2.1 billion most recently.
A survey of 48 oncologists by Leerink found that the use of a poly ADP-ribose polymerase (PARP) inhibitor in second-line maintenance of ovarian cancer could increase from about 45 percent to about 55 percent during the next 12 months. The class' utility there may be starting to plateau, but clinicians polled liked the prospects of expanded use into the front-line maintenance setting, as well as PARP retreatment.
With a just-begun phase III trial, Malvern, Pa.-based Galera Therapeutics Inc. is testing its small-molecule mimicker of superoxide dismutase, avasopasem manganese (GC-4419), against severe oral mucositis (SOM) in patients with head and neck cancer.
Matinas Biopharma Holdings Inc. CEO Jerome Jabbour told BioWorld that, with the company's omega-3 fatty acid-based MAT-9001 for high triglycerides, "we've come full circle," thanks to cardiovascular (CV) outcomes data disclosed last month by Amarin Corp. plc with omega-3 drug Vascepa (icosapent ethyl). "Now we find ourselves sitting on a potential best-in-class asset," which beat Dublin-based Amarin's compound in a head-to-head trial several years ago.
