Shire plc reported that SHP-616, a ready-to-use subcutaneous formulation of its C1 esterase inhibitor, Cinryze, significantly reduced hereditary angioedema (HAE) attacks vs. placebo in a phase III study that laid the groundwork for the company to seek regulatory approvals of the candidate.
Kura Oncology Inc. shares (NASDAQ:KURA) shot 72.3 percent higher to $11.80 Friday on news that its lead candidate, tipifarnib, hit the primary endpoint of a phase II trial in HRAS-mutant relapsed or refractory squamous cell carcinomas of the head and neck (SCCHN) before the trial had even completed enrolling. Four confirmed partial responses and two patients with stable disease were observed among the first six evaluable patients enrolled, and two patients showed objective responses beyond one year. Kura said it will continue to enroll additional patients.
New data on tezepelumab, an experimental drug for people with severe uncontrolled asthma developed by Amgen Inc. in collaboration with Astrazeneca plc's Medimmune unit, showed that adding it to standard therapies for the condition significantly reduced annual asthma exacerbation rates vs. placebo.
Voyager Therapeutics Inc.'s shares (NASDAQ:VYGR) rose 24.7 percent to $13.63 on Wednesday as the company announced plans to embark on a pivotal phase II/III program testing its adeno-associated virus 2 (AAV2) gene therapy, VY-AADC-01, in advanced Parkinson's disease (PD) later this year, encouraged by early stage tests in a small group of PD patients. Dosing of the first patient in the pivotal program is expected in the first half of 2018.
Just weeks ahead of the planned resubmission of the FDA new drug application for a pain pill tailored to reduce opioid-induced nausea and vomiting (OINV), one-time backer Daiichi Sankyo Co. Ltd. handed rights to the drug back to Charleston Laboratories Inc. and booked a ¥27.8 billion (US$252.7 million) loss on the project as it reevaluates its U.S. pain drug strategy. An NDA submitted for the candidate, CL-108, drew a complete response letter (CRL) in February, though neither company revealed why.
The death of a 78-year-old man with a rare blood cancer in an early trial of Cellectis SA's gene-edited chimeric antigen receptor T-cell (CAR T) therapy, UCART-123, triggered an FDA clinical hold on the study and a second test of the experimental treatment. Following closely on last week's FDA approval of Novartis AG's CAR T immunotherapy, Kymriah (tisagenlecleucel), the fatality offered a stark reminder of the risks faced by both companies and patients participating in efforts to advance new gene-edited T-cell-based treatments.
Shares of Seattle-based Aptevo Therapeutics Inc. (NASDAQ:APVO) rose 58.7 percent on Friday to $2.11 as the company unveiled the sale of three noncore hematology drugs for up to $74.5 million to Saol Therapeutics and regained full rights to an experimental bispecific antibody immunotherapy for metastatic castration-resistant prostate cancer (mCRPC) from Morphosys AG.
Just weeks ahead of the planned resubmission of the FDA new drug application for a pain pill tailored to reduce opioid-induced nausea and vomiting (OINV), one-time backer Daiichi Sankyo Co. Ltd. is handing rights to the drug back to Charleston Laboratories Inc. and booking a ¥27.8 billion (US$252.7 million) loss on the project as it reevaluates its U.S. pain drug strategy.
More than seven years after first testing its Meniere's disease candidate, Otividex, in patients with the inner ear disorder, Otonomy Inc. is immediately suspending development of the specially formulated steroid after it proved no better than a placebo in reducing both the number and severity of vertigo episodes in the first of two phase III trials.
An FDA refuse-to-file (RTF) letter has derailed Acorda Therapeutics Inc.'s new drug application (NDA) for Inbrija, an inhaled levodopa candidate for improving motor function in Parkinson's disease (PD) patients.
