Findings published in Nature showing that dual blockage of PD-L1 and CD47 can boost the therapeutic effects of oxaliplatin chemotherapy as well as the FOLFOX regimen – in a CT-26 mice tumor model, anyway – served to highlight CD47, a target that has spurred added efforts of late.
Esperion Therapeutics Inc.’s full results from the phase III outcomes trial called Cholesterol Lowering via Bempedoic Acid, an ACL-Inhibiting Regimen, known by the rough acronym CLEAR, inspired excitement in the mainstream media worldwide but not on Wall Street, as numbers from the experiment fell short of what some wanted. Though the findings proved unmistakably positive, shares of Ann Arbor, Mich.-based Esperion (NASDAQ:ESPR) dropped almost 20% or $1.27 to close March 6 at $5.08.
Praxis Precision Medicines Inc. CEO Marcio Souza said it would be “disingenuous not to move forward” – U.S. FDA willing – into a phase III effort with an alternate design targeted for the second half of this year, given top-line results from the phase IIb Essential-1 study with ulixacaltamide (PRAX-944) for essential tremor.
Inovio Pharmaceuticals Inc. has “more work to do” in understanding mixed results with the DNA-based immunotherapy VGX-3100 as a treatment for cervical high-grade squamous intraepithelial lesions associated with human papillomavirus (HPV)-16 or HPV-18, said Michael Sumner, chief medical officer for the company. “We only got the data in our hands about a week ago.”
If clinical efforts pan out, gamma delta T-cell specialist TC Biopharm plc could plant the space’s first U.S. regulatory flag. Founded 10 years ago, the Glasgow, U.K.-based firm is marching ahead with phase IIb work testing main asset Omnimmune, an allogeneic unmodified cell therapy, in acute myeloid leukemia (AML).
Biocryst Pharmaceuticals Inc.’s unveiling of real-world data Feb. 24 with its approved Orladeyo (berotralstat) for prophylactic treatment of hereditary angioedema (HAE) brought more attention to the space, due to yield findings from would-be competitors in the near term.
Aiming to start afresh under a new name, troubled Mei Pharma Inc. wants to merge with Infinity Pharmaceuticals Inc. in an all-stock transaction that would provide the combined outfit enough cash to fund operations through mid-2025. Specifically, coffers of the newly made firm would contain about $100 million for the advancement of three oncology candidates in a pipeline led by Infinity’s phase II-stage eganelisib for head and neck squamous cell carcinoma.
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.
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.
