Ocera Therapeutics Inc. CEO Linda Grais told BioWorld Today that a closer look at data from the failed-but-validating phase IIb STOP-HE study with intravenous (I.V.) OCR-002 (ornithine phenylacetate) gives the company "the basis for an argument that [ammonia levels] should at least be part of a potentially composite endpoint, but we do expect we would still need a clinical component as well" in the next experiments with the compound, in the works for hospitalized patients with hepatic encephalopathy (HE).
With enrollment about six months ahead of schedule, precision medicine specialist Dalcor Pharmaceuticals Inc. has randomized more than 1,000 patients of the expected 5,000 for the phase III dal-Gene trial, a cardiovascular outcomes (CVOT) study of dalcetrapib in patients with acute coronary syndrome (ACS) who have the AA genotype in the ADCY9 gene.
In a second-quarter meeting with the FDA, Scynexis Inc. will be talking about three mild to moderate thrombotic events in healthy phase I volunteers, and CEO Marco Taglietti told BioWorld Today that the company was already putting together a data package when informed by regulators that no further trials will be allowed with the intravenous (I.V.) form of the antifungal SCY-078 until questions are resolved.
The FDA gave its nod to Milford, Penn.-based Serenity Pharmaceuticals LLC's Noctiva (desmopressin acetate) nasal spray for nocturnal polyuria (overproduction of urine during the night) in adults who awaken at least two times per night to urinate. It's the first FDA-approved treatment for that condition.
Karyopharm Therapeutics Inc.'s phase II failure with lowered-dose selinexor in acute myeloid leukemia (AML) "put[s] to rest the sepsis question," which was "a big overhang on the stock, frankly, and on the drug," CEO Michael Kauffman told BioWorld Today, as trials continue with the compound in other, somewhat less intractable indications.
Myovant Sciences Inc.'s kickoff to 2017 with a phase III trial testing its oral gonadotropin-releasing hormone (GnRH) receptor antagonist relugolix in uterine fibroids (UFs) signaled a year of tighter focus on the condition, with a group of heavyweight drug developers involved in that indication as well as endometriosis.
With "solid, convincing evidence that the speculations will actually be realized" in hand, Kite Pharma Inc.'s chief medical officer (CMO), David Chang, told BioWorld Today that the company – just as it hit the primary endpoint in the pivotal trial – will meet the targeted filing of the BLA for its lead chimeric antigen receptor (CAR) T-cell candidate, axicabtagene ciloleucel.
Maybe some were less than surprised by the gist of phase III results tallied by La Jolla Pharmaceutical Co. in the ATHOS-3 study with LJPC-501 (angiotensin II) in patients with catecholamine-resistant hypotension (CRH) associated with distributive shock, but the data hinted at an even brighter picture and Wall Street rewarded the firm handsomely, sending shares (NASDAQ:LJPC) on a ride that ended Monday at $35.12, up $15.25, or 76.8 percent.
Myovant Sciences Inc.'s kickoff to 2017 with a phase III trial testing its oral gonadotropin-releasing hormone (GnRH) receptor antagonist relugolix in uterine fibroids (UFs) signaled a year of tighter focus on the condition, with a group of heavyweight drug developers involved in that indication as well as endometriosis.
Reata Pharmaceuticals Inc.'s chief medical officer, Colin Meyer, told BioWorld Today that an earlier phase III trial in chronic kidney disease (CKD) was stopped because of "a very specific safety finding that we did not understand at the time," and the company's more recently informed confidence – along with a blessing from the FDA – is fueling another attempt with bardoxolone methyl (bard).
