A single pivotal phase III trial of an enzyme engineered to treat a rare urea cycle disorder, arginase 1 deficiency (ARG1-D), could potentially support U.S. and European approvals of the therapy, according to Aeglea Biotherapeutics Inc. The Austin, Texas-based biotech expects to start dosing a small group of patients with the enzyme, its lead candidate pegzilarginase, during the second quarter of 2019 and to make top-line data available in the first quarter of 2021.
SAN DIEGO – First-line treatment of peripheral T-cell lymphomas (PTCL) with a quartet of drugs substituting Adcetris (brentuximab vedotin) for Oncovin (vincristine) in the decades-old standard of care for the disease, CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone), proved superior on both progression-free survival and overall survival, according to full data reported Tuesday by Seattle Genetics Inc. and partner Takeda Pharmaceutical Co. Ltd.
Macrogenics Inc. shares (NASDAQ:MGNX) sank 18 percent to $13.43 on Monday as investors fretted over the implications of a partial clinical hold the FDA slapped on a phase I test of the company's bispecific antibody candidate, MGD-009. High transaminase levels seen in some participants were "likely a cytokine-mediated event," said Macrogenics President and CEO Scott Koenig. Trial protocol amendments to mitigate the issue are under discussion, with Macrogenics planning to submit answers to questions from the regulator before the end of the year, he told BioWorld.
Just months after its Nasdaq debut in June, Meiragtx Holdings plc put its relatively new shares to work in an all-stock-based acquisition of Vector Neurosciences Inc. Announced in October, the transaction brought the London and New York-based company an adeno-associated virus (AAV) encoding glutamic acid decarboxylase (AAV-GAD) gene therapy candidate poised for continued phase II development in Parkinson's disease. Now the company has five ongoing clinical programs in eye, salivary gland and central nervous system disorders, advancing alongside a pipeline of preclinical and research programs and is further extending its ambitions.
More than a year after legal wrangling derailed a $2 billion deal to hasten development of its antibody drug conjugate (ADC) IMMU-132 (sacituzumab govitecan) for breast cancer, Immunomedics Inc. is racing toward new regulatory milestones. The company submitted updated phase II data on the ADC in metastatic triple-negative breast cancer (mTNBC) to support an ongoing FDA priority review while also reaching an accord with the agency on the design of a registration-enabling trial for the drug in HR+/HER2- metastatic breast cancer (mBC), it said.
SAN DIEGO – Interim results from a large global phase III trial have shown that adding the anti-CD38 monoclonal antibody Darzalex (daratumumab) to Revlimid (lenalidomide) and dexamethasone (dex) reduced by 44 percent the risk of death or disease progression in patients with newly diagnosed multiple myeloma (MM) who were ineligible for a stem cell transplant.
SAN DIEGO – First-line treatment of peripheral T-cell lymphomas (PTCL) with a quartet of drugs substituting Adcetris (brentuximab vedotin) for Oncovin (vincristine) in the decades-old standard of care for the disease, CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone), proved superior on both progression-free survival and overall survival, according to full data reported Tuesday by Seattle Genetics Inc. and partner Takeda Pharmaceutical Co. Ltd.
SAN DIEGO – New data from Cidara Therapeutics Inc. suggest the potential of its antifungal echinocandin rezafungin to prevent invasive fungal infections in bone marrow transplant (BMT) patients.
San Diego – New data showcased by Beigene Ltd. at the American Society of Hematology meeting (ASH) provided ample evidence of the company's progress in improving on drugs in two classes of medicine already known for their high-profile leaders. Its anti-PD-1 antibody, tislelizumab, and Bruton's tyrosine kinase (BTK) inhibitor, zanubrutinib, both yielded new data.
Marking its first regulatory win in the U.S. blood cancer space, Tokyo's Astellas Pharma Inc. has secured approval from the FDA for its oral acute myeloid leukemia (AML) therapy, Xospata (gilteritinib). Already approved in Japan, the FMS-like tyrosine kinase 3 (FLT3) inhibitor is now approved in the U.S. for the treatment of relapsed or refractory AML patients who test positive for the mutation, which is often associated with increased risk of relapse and poor survival.
