The FDA is taking more steps to protect consumers from false or misleading drug ads. The agency finalized its 2013 revised draft guidance on product name placement, size, prominence and frequency in promotional labeling and advertisements. Read More
ATLANTA – About what he called "incredible" data with BB-2121 in multiple myeloma (MM), Bluebird Bio Inc.'s chief medical officer, David Davidson, told BioWorld that his firm is "actually in a privileged position to have positive results across all of our clinical programs" at the scientific meeting, with data in severe sickle cell disease (SCD) and beta-thalassemia. "But clearly the myeloma results are really getting everyone excited," he said. Read More
Acceleron Pharma Inc., of Cambridge, Mass., reported preliminary results from an ongoing phase II trial testing luspatercept in 106 patients with lower-risk myelodysplastic syndromes. Luspatercept is a modified activin receptor type IIB fusion protein being developed with Summit, N.J.-based Celgene Corp. Read More
Wave Life Sciences Ltd., of Cambridge, Mass., said preclinical data of WVE-3972-01, a stereopure antisense oligonucleotide designed to target the pathogenic allele of the C90RF72 gene to treat amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), showed it demonstrated substantial reduction in disease-associated biomarkers and superior potency to stereorandom oligonucleotides. Read More
Oncotartis Inc., of Buffalo, N.Y., raised $6 million to support the phase I trial of OT-82, which has been isolated for its selective toxicity for a broad range of hematological malignancies. Read More
ATLANTA – Gilead Sciences Inc.'s Kite Pharma unit lifted the lid for investors on eagerly awaited long-term follow-up data from the pivotal Zuma-1 study with approved chimeric antigen receptor T (CAR T) therapy Yescarta (axicabtagene ciloleucel) in patients with relapsed or refractory non-Hodgkin's lymphoma (NHL). Read More
ATLANTA – Shares of Blueprint Medicines Corp. (NASDAQ:BPMC) popped 25 percent at Monday's opening bell, closing the day at $88.32 for a gain of $16.46, after the company wowed participants at the American Society of Hematology (ASH) annual meeting with a presentation of data from the ongoing phase I trial of BLU-285 – now named avapritinib – in advanced systemic mastocytosis (SM). Findings from the dose-escalation portion of the phase I study showed an overall response rate (ORR) of 72 percent and a disease control rate (DCR) of 100 percent in patients evaluable for response as of Oct. 4, based on the International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (IWG-MRT-ECNM) consensus criteria. At data cut-off, avapritinib was well tolerated and most adverse events (AEs) reported by investigators were grade 1 or 2. No discontinuations occurred due to treatment-related AEs, and 30 of 32 patients remained on therapy with a median treatment duration of nine months. Read More
ATLANTA – Forty years after the establishment of ABVD – adriamycin, bleomycin, vinblastine and dacarbazine – as standard-of-care front-line therapy in patients with previously untreated advanced classical Hodgkin lymphoma (cHL), Seattle Genetics Inc. (Seagen) revealed details supporting its contention that replacing bleomycin with Adcetris (brentuximab vedotin) could improve efficacy and reduce toxicity in those patients, improving their chance of a cure with the first round of treatment. Read More
LONDON – Hookipa Biotech AG has raised €50 million (US$59 million) in an oversubscribed series C, providing the means to advance clinical development of its vaccines technologies in both infectious diseases and cancer. Read More
Syros Pharmaceuticals shares (NASDAQ:SYRS) lost nearly a third of their value Monday after investors learned that, by the end of October, just one out of 48 evaluable patients enrolled in an ongoing study of the company's lead gene control therapy, SY-1425 (tamibarotene), had achieved a complete response. The ongoing trial is evaluating SY-1425 for the treatment of genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). No patients with lower-risk MDS had achieved transfusion independence, another endpoint of the study, by the cut-off for reporting. Read More
LONDON – The European pharma industry has united with patients groups as well as health care professionals in calling for a deal for the sector to be given priority now that the U.K. has cleared the barriers to enter phase two of the Brexit negotiations. Read More
DUBLIN – The B-cell antigen CD19 is, of course, the target of choice for the first wave of chimeric antigen receptor T-cell (CAR T-cell) therapies to secure FDA approval this year. ADC Therapeutics Sarl and Morphosys AG both reported clinical data at the American Society of Hematology Annual Meeting in Atlanta this week, which also confirms its potential as a target for antibody-based therapies as well – but only in the context of engineered antibodies that have additional properties beyond what conventional antibodies can offer. Read More
ATLANTA – With last month's early FDA nod for bispecific antibody Hemlibra (emicizumab) – more than three months ahead of its PDUFA date – and a rapidly advancing gene therapy candidate, the treatment landscape for hemophilia A is poised for a dramatic change. Read More
