The positive opinion Jan. 27 from the EMA’s Committee for Medicinal Products for Human Use regarding Reblozyl (luspatercept) from Bristol Myers Squibb Co. to treat adults with non‑transfusion-dependent beta-thalassemia marked an advance in the space, where several developers are jockeying for position. Reblozyl, a first-in-class erythroid maturation agent, was first approved in November 2019 in the hands of Celgene Corp., acquired by Princeton, N.J.-based BMS the same year.
The other shoe dropped in a good way for backers of Actinium Pharmaceuticals Inc. as the firm popped the lid off full data from the phase III study called Sierra testing Iomab-B in patients age 55 and older with active relapsed or refractory acute myeloid leukemia (r/r AML). Antibody radiation conjugate (ARC) Iomab-B met the primary endpoint of durable complete remission of six months following initial complete remission after bone-marrow transplant with a high degree of statistical significance.
Bridge Biotherapeutics Inc.’s small-molecule Pellino-1 inhibitor, BBT-401, failed to meet the primary efficacy endpoint in a phase II trial in ulcerative colitis (UC). The phase II mid-to-high dose cohort study included 38 patients with active UC enrolled at 37 clinical sites across the U.S., New Zealand, South Korea, Poland and Ukraine.
Denali Therapeutics Inc.’s extensive update on clinical programs in central nervous system diseases at the start of this year included plans for lead asset DNL-310 in mucopolysaccharidosis II (MPS II), also known as Hunter syndrome – a space where other notable players include such names as Regenxbio Inc. and Takeda Pharmaceutical Co. Ltd.
The A’s have it but the B’s don’t in Moderna Inc.’s pivotal phase III study of mRNA-1010, a seasonal flu vaccine for adults living in the southern hemisphere. Interim results showed the vaccine achieved superiority on seroconversion rates for influenzas A/H3N2 and A/H1N1, superiority on geometric mean titer ratios for influenza A/H3N2 and noninferiority on geometric mean titer ratios for influenza A/H1N1. However, noninferiority was not met for the endpoints against the influenza B/Victoria- and B/Yamagata-lineage strains.
Ascletis Pharma Inc. presented subgroup data from a phase II trial showing its subcutaneous PD-L1 antibody envafolimab (ASC-22) was able to functionally cure chronic hepatitis B, according to hepatitis B surface antigen decline following 24-week treatment.
The steadily percolating psychedelic drug space stands poised to generate a near-term stream of potentially encouraging developments in a range of mental health disorders, including depression, anxiety, post-traumatic stress disorder and more.
After gaining emergency regulatory approval in Japan for its oral COVID-19 antiviral, 3CL protease inhibitor Xocova (ensitrelvir/S-217622), Shionogi & Co. Ltd. is advancing several global phase III trials across a wide range of COVID-19 patient populations. Branded as Xocova in Japan, ensitrelvir received emergency regulatory approval from Japan’s Ministry of Health, Labour and Welfare in late November 2022 for SARS-CoV-2 infection based on the results from phase II/III trials in non-hospitalized patients in Asia.
Replay Holdings LLC said it will be first into the clinic with a T-cell receptor natural killer (TCR-NK) cancer immunotherapy, after forming its latest satellite company around technology from the University of Texas MD Anderson Cancer Center. No financial details were disclosed, but the newco, Syena, is funded to run a basket trial that will assess an NY-ESO-1-targeted TCR-NK in a number of hematological and solid tumors, and said it will start treating patients in the second half of 2023.
Updated MRI results from Clene Inc.’s phase II Visionary-MS trial testing gold nanocrystal therapy CNM-Au8 bolsters the company’s premise that targeting energy metabolism could protect neuronal function in patients with multiple sclerosis and lays the groundwork for a phase III trial as soon as a strategic partner is found.
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